Red blood cells

The European Commission (EC) has granted orphan designation to BMN 270, an investigational gene therapy, for the treatment of patients with hemophilia A.

BMN 270 is an adeno-associated virus-factor VIII (FVIII) vector designed to restore FVIII plasma concentrations in these patients.

The EC grants orphan designation to products intended to treat, prevent, or diagnose a life-threatening condition affecting up to 5 in 10,000 people in the European Union.

The product must provide significant benefit to those affected by the condition.

Orphan designation from the EC provides companies with certain development incentives, including protocol assistance, a type of scientific advice specific for orphan drugs, and 10 years of market exclusivity once the drug is approved for use.

BMN 270 is under development by BioMarin Pharmaceutical Inc.

BioMarin is conducting a phase 1/2 study to evaluate the safety and efficacy of BMN 270 in up to 12 patients with severe hemophilia A.

Researchers are assessing the safety of a single infusion of BMN 270 and the change in FVIII expression level from baseline to 16 weeks after infusion.

The group is also assessing the impact of BMN 270 on the frequency of FVIII replacement therapy, the number of bleeding episodes requiring treatment, and any potential immune responses.

Patients will be monitored for safety and durability of effect for 5 years. BioMarin plans to provide an update on this trial in April.

BMN 270 also has orphan designation in the US.

Red blood cells

The European Commission (EC) has granted orphan designation to BMN 270, an investigational gene therapy, for the treatment of patients with hemophilia A.

BMN 270 is an adeno-associated virus-factor VIII (FVIII) vector designed to restore FVIII plasma concentrations in these patients.

The EC grants orphan designation to products intended to treat, prevent, or diagnose a life-threatening condition affecting up to 5 in 10,000 people in the European Union.

The product must provide significant benefit to those affected by the condition.

Orphan designation from the EC provides companies with certain development incentives, including protocol assistance, a type of scientific advice specific for orphan drugs, and 10 years of market exclusivity once the drug is approved for use.

BMN 270 is under development by BioMarin Pharmaceutical Inc.

BioMarin is conducting a phase 1/2 study to evaluate the safety and efficacy of BMN 270 in up to 12 patients with severe hemophilia A.

Researchers are assessing the safety of a single infusion of BMN 270 and the change in FVIII expression level from baseline to 16 weeks after infusion.

The group is also assessing the impact of BMN 270 on the frequency of FVIII replacement therapy, the number of bleeding episodes requiring treatment, and any potential immune responses.

Patients will be monitored for safety and durability of effect for 5 years. BioMarin plans to provide an update on this trial in April.

BMN 270 also has orphan designation in the US.

Red blood cells

The European Commission (EC) has granted orphan designation to BMN 270, an investigational gene therapy, for the treatment of patients with hemophilia A.

BMN 270 is an adeno-associated virus-factor VIII (FVIII) vector designed to restore FVIII plasma concentrations in these patients.

The EC grants orphan designation to products intended to treat, prevent, or diagnose a life-threatening condition affecting up to 5 in 10,000 people in the European Union.

The product must provide significant benefit to those affected by the condition.

Orphan designation from the EC provides companies with certain development incentives, including protocol assistance, a type of scientific advice specific for orphan drugs, and 10 years of market exclusivity once the drug is approved for use.

BMN 270 is under development by BioMarin Pharmaceutical Inc.

BioMarin is conducting a phase 1/2 study to evaluate the safety and efficacy of BMN 270 in up to 12 patients with severe hemophilia A.

Researchers are assessing the safety of a single infusion of BMN 270 and the change in FVIII expression level from baseline to 16 weeks after infusion.

The group is also assessing the impact of BMN 270 on the frequency of FVIII replacement therapy, the number of bleeding episodes requiring treatment, and any potential immune responses.

Patients will be monitored for safety and durability of effect for 5 years. BioMarin plans to provide an update on this trial in April.

BMN 270 also has orphan designation in the US.

SAN DIEGO — If you arrived late, or even right on time, to the session on becoming a better attending, you’d better have been ready to find a clear spot on the floor or have had the energy to stand for an hour.

Read more about the speakers at HM16.

But the dynamic talk, you could even say performance, by Jeffrey Wiese, MD, MHM, crackled with energy, so there was plenty of it to go around. The session, in its 10th year and now practically an institution of its own at the annual meeting, was a highlight among the offerings on career development at HM16.

Dr. Wiese liberally seasoned the session with role-playing and humor—the “patient” on the session-room floor but without a pillow meant “Press Ganey’s going to take a hit on this one,” he joked. He emphasized the importance of attending physicians to give reasons to support the expectations they have for their trainees.

“The key piece is giving that rationale. Once they have a reason for why they’re going to do it, now the expectation’s grounded, now it actually makes sense,” said Dr. Wiese, professor of medicine at Tulane University Health Sciences Center in New Orleans and a past SHM president. “You don’t do that, they’re going to fill in the gaps with their own expectations.”

Other points of emphasis:

• The importance of autonomy and choice so that trainees have a sense of purpose;
• The transition from self-interested medical students to residents who are concerned with the well-being of team members;
• The assurance of an endpoint so that hectic periods don’t spiral out of control; and
• Acts of ritual, such as using Purell before entering a patient’s room, as moments of “genuflect” to regain perspective.

Charles Kast, MD, an attending physician at Long Island Jewish Medical Center in New Hyde Park, N.Y., said the relationship theme resonated with him the most.

“It’s more the relationships the attendings have with their residents and with their students, and it’s more of an emotional connection,” he said. “Whether it’s education or mentoring or what have you, it’s all about developing that trust between the resident and the attending.”

But it’s a gradual process.

“It’s baby steps,” Dr. Kast added. “There were 17 different lists in there, right? So you’ve got to pick one and kind of go with it. I think it’s kind of an organic process, where one thing kind of leads to the next.”

Tactics to avoid burnout—by cultivating a sense of purpose while understanding and relating to trainees’ concerns—were a key part of Dr. Wiese’s message. Burnout was a topic that HM16 attendees returned to again and again when discussing their take-homes from the meeting. The subject popped up in almost every session to one degree or another.

David Nevin, MD, a hospitalist at ThedaCare in Wisconsin, said that he was reminded in one session—“Staying in the Game: Self-Care for Hospitalists”—that taking even brief moments for yourself can make a difference.

“Focusing on the positive for a moment and what’s good about your life, and doing that kind of exercise, helps sort of deal with burnout and bring things into perspective,” he said. “You get sort of worn down, you’re not as sharp, you miss things when you’re not at your peak in terms of looking at things.”

Ariana Peters, DO, who works at Mayo Clinic in Scottsdale, Ariz., said a similar message resonated with her, as well. There are ample situations when, if she doesn’t consciously take time for herself, things will seem to mushroom.

In just one recent example, she reflected on an especially difficult day.

“I had 18 patients, and it was horrible,” she said. “I left my office in the morning and didn’t come back until 8 p.m. that night. I was literally eating peanut butter and graham crackers on the floor.

“It doesn’t take long to just stop and take a breath. Twenty seconds is not a long time.”

Waiting for a session on personal productivity to start, Adam Garber, MD, assistant professor at Virginia Commonwealth University in Richmond, said that he found the introduction to the SMART approach (Specific, Measurable, Achievable, Relevant, and Time-Bound), meaning being able to be done within a certain period, was a good guideline to approaching projects of all kinds.

“I think you can apply it to any problem and career-development project you want to work on,” he said. “It just kind of gives you that framework of how to organize it, present it logically, and carry it out.” TH

SAN DIEGO — If you arrived late, or even right on time, to the session on becoming a better attending, you’d better have been ready to find a clear spot on the floor or have had the energy to stand for an hour.

Read more about the speakers at HM16.

But the dynamic talk, you could even say performance, by Jeffrey Wiese, MD, MHM, crackled with energy, so there was plenty of it to go around. The session, in its 10th year and now practically an institution of its own at the annual meeting, was a highlight among the offerings on career development at HM16.

Dr. Wiese liberally seasoned the session with role-playing and humor—the “patient” on the session-room floor but without a pillow meant “Press Ganey’s going to take a hit on this one,” he joked. He emphasized the importance of attending physicians to give reasons to support the expectations they have for their trainees.

“The key piece is giving that rationale. Once they have a reason for why they’re going to do it, now the expectation’s grounded, now it actually makes sense,” said Dr. Wiese, professor of medicine at Tulane University Health Sciences Center in New Orleans and a past SHM president. “You don’t do that, they’re going to fill in the gaps with their own expectations.”

Other points of emphasis:

• The importance of autonomy and choice so that trainees have a sense of purpose;
• The transition from self-interested medical students to residents who are concerned with the well-being of team members;
• The assurance of an endpoint so that hectic periods don’t spiral out of control; and
• Acts of ritual, such as using Purell before entering a patient’s room, as moments of “genuflect” to regain perspective.

Charles Kast, MD, an attending physician at Long Island Jewish Medical Center in New Hyde Park, N.Y., said the relationship theme resonated with him the most.

“It’s more the relationships the attendings have with their residents and with their students, and it’s more of an emotional connection,” he said. “Whether it’s education or mentoring or what have you, it’s all about developing that trust between the resident and the attending.”

But it’s a gradual process.

“It’s baby steps,” Dr. Kast added. “There were 17 different lists in there, right? So you’ve got to pick one and kind of go with it. I think it’s kind of an organic process, where one thing kind of leads to the next.”

Tactics to avoid burnout—by cultivating a sense of purpose while understanding and relating to trainees’ concerns—were a key part of Dr. Wiese’s message. Burnout was a topic that HM16 attendees returned to again and again when discussing their take-homes from the meeting. The subject popped up in almost every session to one degree or another.

David Nevin, MD, a hospitalist at ThedaCare in Wisconsin, said that he was reminded in one session—“Staying in the Game: Self-Care for Hospitalists”—that taking even brief moments for yourself can make a difference.

“Focusing on the positive for a moment and what’s good about your life, and doing that kind of exercise, helps sort of deal with burnout and bring things into perspective,” he said. “You get sort of worn down, you’re not as sharp, you miss things when you’re not at your peak in terms of looking at things.”

Ariana Peters, DO, who works at Mayo Clinic in Scottsdale, Ariz., said a similar message resonated with her, as well. There are ample situations when, if she doesn’t consciously take time for herself, things will seem to mushroom.

In just one recent example, she reflected on an especially difficult day.

“I had 18 patients, and it was horrible,” she said. “I left my office in the morning and didn’t come back until 8 p.m. that night. I was literally eating peanut butter and graham crackers on the floor.

“It doesn’t take long to just stop and take a breath. Twenty seconds is not a long time.”

Waiting for a session on personal productivity to start, Adam Garber, MD, assistant professor at Virginia Commonwealth University in Richmond, said that he found the introduction to the SMART approach (Specific, Measurable, Achievable, Relevant, and Time-Bound), meaning being able to be done within a certain period, was a good guideline to approaching projects of all kinds.

“I think you can apply it to any problem and career-development project you want to work on,” he said. “It just kind of gives you that framework of how to organize it, present it logically, and carry it out.” TH

SAN DIEGO — If you arrived late, or even right on time, to the session on becoming a better attending, you’d better have been ready to find a clear spot on the floor or have had the energy to stand for an hour.

Read more about the speakers at HM16.

But the dynamic talk, you could even say performance, by Jeffrey Wiese, MD, MHM, crackled with energy, so there was plenty of it to go around. The session, in its 10th year and now practically an institution of its own at the annual meeting, was a highlight among the offerings on career development at HM16.

Dr. Wiese liberally seasoned the session with role-playing and humor—the “patient” on the session-room floor but without a pillow meant “Press Ganey’s going to take a hit on this one,” he joked. He emphasized the importance of attending physicians to give reasons to support the expectations they have for their trainees.

“The key piece is giving that rationale. Once they have a reason for why they’re going to do it, now the expectation’s grounded, now it actually makes sense,” said Dr. Wiese, professor of medicine at Tulane University Health Sciences Center in New Orleans and a past SHM president. “You don’t do that, they’re going to fill in the gaps with their own expectations.”

Other points of emphasis:

• The importance of autonomy and choice so that trainees have a sense of purpose;
• The transition from self-interested medical students to residents who are concerned with the well-being of team members;
• The assurance of an endpoint so that hectic periods don’t spiral out of control; and
• Acts of ritual, such as using Purell before entering a patient’s room, as moments of “genuflect” to regain perspective.

Charles Kast, MD, an attending physician at Long Island Jewish Medical Center in New Hyde Park, N.Y., said the relationship theme resonated with him the most.

“It’s more the relationships the attendings have with their residents and with their students, and it’s more of an emotional connection,” he said. “Whether it’s education or mentoring or what have you, it’s all about developing that trust between the resident and the attending.”

But it’s a gradual process.

“It’s baby steps,” Dr. Kast added. “There were 17 different lists in there, right? So you’ve got to pick one and kind of go with it. I think it’s kind of an organic process, where one thing kind of leads to the next.”

Tactics to avoid burnout—by cultivating a sense of purpose while understanding and relating to trainees’ concerns—were a key part of Dr. Wiese’s message. Burnout was a topic that HM16 attendees returned to again and again when discussing their take-homes from the meeting. The subject popped up in almost every session to one degree or another.

David Nevin, MD, a hospitalist at ThedaCare in Wisconsin, said that he was reminded in one session—“Staying in the Game: Self-Care for Hospitalists”—that taking even brief moments for yourself can make a difference.

“Focusing on the positive for a moment and what’s good about your life, and doing that kind of exercise, helps sort of deal with burnout and bring things into perspective,” he said. “You get sort of worn down, you’re not as sharp, you miss things when you’re not at your peak in terms of looking at things.”

Ariana Peters, DO, who works at Mayo Clinic in Scottsdale, Ariz., said a similar message resonated with her, as well. There are ample situations when, if she doesn’t consciously take time for herself, things will seem to mushroom.

In just one recent example, she reflected on an especially difficult day.

“I had 18 patients, and it was horrible,” she said. “I left my office in the morning and didn’t come back until 8 p.m. that night. I was literally eating peanut butter and graham crackers on the floor.

“It doesn’t take long to just stop and take a breath. Twenty seconds is not a long time.”

Waiting for a session on personal productivity to start, Adam Garber, MD, assistant professor at Virginia Commonwealth University in Richmond, said that he found the introduction to the SMART approach (Specific, Measurable, Achievable, Relevant, and Time-Bound), meaning being able to be done within a certain period, was a good guideline to approaching projects of all kinds.

“I think you can apply it to any problem and career-development project you want to work on,” he said. “It just kind of gives you that framework of how to organize it, present it logically, and carry it out.” TH

Micrograph showing MDS

Researchers have found the fusion protein APG101 can rescue erythropoiesis in bone marrow samples from patients with lower-risk myelodysplastic syndromes (MDS).

Previous research suggested that CD95—a receptor that can induce apoptosis when triggered by the CD95 ligand—is overexpressed in two-thirds of patients with lower-risk MDS, and overexpression of CD95 is predictive of a lower response to erythropoiesis-stimulating agents (ESAs).

APG101, which consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody, is designed to block the CD95 ligand.

The new study showed that APG101 can inhibit apoptosis in erythrocyte precursor cells and improve their overall proliferation rate. The drug increased the number of burst-forming unit-erythroid (BFU-E) progenitors in samples from MDS patients with low BFU-E numbers at baseline.

“APG101 added to cellular assays efficiently rescued the growth of erythroid progenitors in MDS patients harboring a profound defect of erythropoiesis, independent of the expression level of CD95 or CD95 ligand,” said Michaela Fontenay, MD, PhD, of the Institut Cochin in Paris, France.

Dr Fontenay and her colleagues described these results in Oncotarget. The research was funded by Apogenix, the company developing APG101.

By comparing bone marrow samples from MDS patients and healthy control subjects, the researchers found that CD95, but not CD95 ligand, was overexpressed in patients with lower-risk MDS.

Further analysis revealed that a patient’s CD95 expression level at diagnosis could predict response to an ESA. Specifically, CD95 overexpression was predictive of a lower response rate to ESAs in patients with low- or intermediate-1-risk MDS.

Next, the researchers tested bone marrow erythroid progenitors from 3 control subjects and 5 patients with MDS and found that CD95 expression increased during MDS erythroid progenitor amplification but remained lower in control cultures.

On day 5 of culture, the mean number of BFU-Es was significantly lower in MDS patient samples than in controls. And treatment with APG101 prompted a dose-dependent increase in BFU-E growth in MDS samples but not in controls.

When the researchers added APG101 (at 10 μg/mL) to the cultures over 7 days, they observed an improvement in the proliferation of erythroblasts but no significant effect on the kinetics of differentiation.

They also found that APG101 reduced apoptosis in immature precursors by 30% but had no effect on apoptosis in mature precursors.

Baseline BFU-E number affects response

The researchers then assessed the effects of APG101 in samples from 5 control subjects and 20 MDS patients with varying responses to ESAs and varying baseline levels of BFU-Es.

Fifteen of the MDS patients had a significantly lower number of baseline BFU-Es than controls (P=0.005), but 5 MDS patients had a mean number of BFU-Es that was comparable to controls (P=0.429). There was no significant difference in WHO classification or CD95 expression between the 2 groups of MDS patients (P=0.612).

However, 11 of the 15 patients with low erythropoiesis had received an ESA, and all of them were resistant to this treatment. In all, 15 of the MDS patients had received an ESA, and 14 were resistant to it (6 primary and 8 secondary).

The researchers found that APG101 induced a dose-dependent increase of BFU-Es in samples from the 15 MDS patients with low erythropoiesis but not in samples from the 5 patients with normal erythropoiesis or in the control samples (P<0.001).

The team said that a low BFU-E number at baseline was significantly associated with in vitro response to APG101 among the 20 MDS patients (P=0.031) and the 14 ESA-resistant patients (P=0.027).

Further investigation confirmed that a low clonogenic progenitor number at baseline, but not the level of CD95 or CD95 ligand expression, was predictive of the response to APG101.

“This study provides a rationale for further clinical investigation of this potential new therapeutic option in patients with severely impaired erythropoiesis who are resistant to erythropoiesis-stimulating agents,” Dr Fontenay said.

Apogenix has conducted a phase 1 trial of APG101 in transfusion-dependent patients with low- to intermediate-1-risk MDS. The company expects the results of this trial will be available in the coming months.

Micrograph showing MDS

Researchers have found the fusion protein APG101 can rescue erythropoiesis in bone marrow samples from patients with lower-risk myelodysplastic syndromes (MDS).

Previous research suggested that CD95—a receptor that can induce apoptosis when triggered by the CD95 ligand—is overexpressed in two-thirds of patients with lower-risk MDS, and overexpression of CD95 is predictive of a lower response to erythropoiesis-stimulating agents (ESAs).

APG101, which consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody, is designed to block the CD95 ligand.

The new study showed that APG101 can inhibit apoptosis in erythrocyte precursor cells and improve their overall proliferation rate. The drug increased the number of burst-forming unit-erythroid (BFU-E) progenitors in samples from MDS patients with low BFU-E numbers at baseline.

“APG101 added to cellular assays efficiently rescued the growth of erythroid progenitors in MDS patients harboring a profound defect of erythropoiesis, independent of the expression level of CD95 or CD95 ligand,” said Michaela Fontenay, MD, PhD, of the Institut Cochin in Paris, France.

Dr Fontenay and her colleagues described these results in Oncotarget. The research was funded by Apogenix, the company developing APG101.

By comparing bone marrow samples from MDS patients and healthy control subjects, the researchers found that CD95, but not CD95 ligand, was overexpressed in patients with lower-risk MDS.

Further analysis revealed that a patient’s CD95 expression level at diagnosis could predict response to an ESA. Specifically, CD95 overexpression was predictive of a lower response rate to ESAs in patients with low- or intermediate-1-risk MDS.

Next, the researchers tested bone marrow erythroid progenitors from 3 control subjects and 5 patients with MDS and found that CD95 expression increased during MDS erythroid progenitor amplification but remained lower in control cultures.

On day 5 of culture, the mean number of BFU-Es was significantly lower in MDS patient samples than in controls. And treatment with APG101 prompted a dose-dependent increase in BFU-E growth in MDS samples but not in controls.

When the researchers added APG101 (at 10 μg/mL) to the cultures over 7 days, they observed an improvement in the proliferation of erythroblasts but no significant effect on the kinetics of differentiation.

They also found that APG101 reduced apoptosis in immature precursors by 30% but had no effect on apoptosis in mature precursors.

Baseline BFU-E number affects response

The researchers then assessed the effects of APG101 in samples from 5 control subjects and 20 MDS patients with varying responses to ESAs and varying baseline levels of BFU-Es.

Fifteen of the MDS patients had a significantly lower number of baseline BFU-Es than controls (P=0.005), but 5 MDS patients had a mean number of BFU-Es that was comparable to controls (P=0.429). There was no significant difference in WHO classification or CD95 expression between the 2 groups of MDS patients (P=0.612).

However, 11 of the 15 patients with low erythropoiesis had received an ESA, and all of them were resistant to this treatment. In all, 15 of the MDS patients had received an ESA, and 14 were resistant to it (6 primary and 8 secondary).

The researchers found that APG101 induced a dose-dependent increase of BFU-Es in samples from the 15 MDS patients with low erythropoiesis but not in samples from the 5 patients with normal erythropoiesis or in the control samples (P<0.001).

The team said that a low BFU-E number at baseline was significantly associated with in vitro response to APG101 among the 20 MDS patients (P=0.031) and the 14 ESA-resistant patients (P=0.027).

Further investigation confirmed that a low clonogenic progenitor number at baseline, but not the level of CD95 or CD95 ligand expression, was predictive of the response to APG101.

“This study provides a rationale for further clinical investigation of this potential new therapeutic option in patients with severely impaired erythropoiesis who are resistant to erythropoiesis-stimulating agents,” Dr Fontenay said.

Apogenix has conducted a phase 1 trial of APG101 in transfusion-dependent patients with low- to intermediate-1-risk MDS. The company expects the results of this trial will be available in the coming months.

Micrograph showing MDS

Researchers have found the fusion protein APG101 can rescue erythropoiesis in bone marrow samples from patients with lower-risk myelodysplastic syndromes (MDS).

Previous research suggested that CD95—a receptor that can induce apoptosis when triggered by the CD95 ligand—is overexpressed in two-thirds of patients with lower-risk MDS, and overexpression of CD95 is predictive of a lower response to erythropoiesis-stimulating agents (ESAs).

APG101, which consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody, is designed to block the CD95 ligand.

The new study showed that APG101 can inhibit apoptosis in erythrocyte precursor cells and improve their overall proliferation rate. The drug increased the number of burst-forming unit-erythroid (BFU-E) progenitors in samples from MDS patients with low BFU-E numbers at baseline.

“APG101 added to cellular assays efficiently rescued the growth of erythroid progenitors in MDS patients harboring a profound defect of erythropoiesis, independent of the expression level of CD95 or CD95 ligand,” said Michaela Fontenay, MD, PhD, of the Institut Cochin in Paris, France.

Dr Fontenay and her colleagues described these results in Oncotarget. The research was funded by Apogenix, the company developing APG101.

By comparing bone marrow samples from MDS patients and healthy control subjects, the researchers found that CD95, but not CD95 ligand, was overexpressed in patients with lower-risk MDS.

Further analysis revealed that a patient’s CD95 expression level at diagnosis could predict response to an ESA. Specifically, CD95 overexpression was predictive of a lower response rate to ESAs in patients with low- or intermediate-1-risk MDS.

Next, the researchers tested bone marrow erythroid progenitors from 3 control subjects and 5 patients with MDS and found that CD95 expression increased during MDS erythroid progenitor amplification but remained lower in control cultures.

On day 5 of culture, the mean number of BFU-Es was significantly lower in MDS patient samples than in controls. And treatment with APG101 prompted a dose-dependent increase in BFU-E growth in MDS samples but not in controls.

When the researchers added APG101 (at 10 μg/mL) to the cultures over 7 days, they observed an improvement in the proliferation of erythroblasts but no significant effect on the kinetics of differentiation.

They also found that APG101 reduced apoptosis in immature precursors by 30% but had no effect on apoptosis in mature precursors.

Baseline BFU-E number affects response

The researchers then assessed the effects of APG101 in samples from 5 control subjects and 20 MDS patients with varying responses to ESAs and varying baseline levels of BFU-Es.

Fifteen of the MDS patients had a significantly lower number of baseline BFU-Es than controls (P=0.005), but 5 MDS patients had a mean number of BFU-Es that was comparable to controls (P=0.429). There was no significant difference in WHO classification or CD95 expression between the 2 groups of MDS patients (P=0.612).

However, 11 of the 15 patients with low erythropoiesis had received an ESA, and all of them were resistant to this treatment. In all, 15 of the MDS patients had received an ESA, and 14 were resistant to it (6 primary and 8 secondary).

The researchers found that APG101 induced a dose-dependent increase of BFU-Es in samples from the 15 MDS patients with low erythropoiesis but not in samples from the 5 patients with normal erythropoiesis or in the control samples (P<0.001).

The team said that a low BFU-E number at baseline was significantly associated with in vitro response to APG101 among the 20 MDS patients (P=0.031) and the 14 ESA-resistant patients (P=0.027).

Further investigation confirmed that a low clonogenic progenitor number at baseline, but not the level of CD95 or CD95 ligand expression, was predictive of the response to APG101.

“This study provides a rationale for further clinical investigation of this potential new therapeutic option in patients with severely impaired erythropoiesis who are resistant to erythropoiesis-stimulating agents,” Dr Fontenay said.

Apogenix has conducted a phase 1 trial of APG101 in transfusion-dependent patients with low- to intermediate-1-risk MDS. The company expects the results of this trial will be available in the coming months.

Men potentially exposed to Zika virus should use a condom during all sex or abstain from sex for at least 8 weeks, according to new recommendations from the Centers for Disease Control and Prevention on reducing the risk of sexual transmission of the virus.

Men with confirmed infections or clinical symptoms of Zika should similarly abstain or use a condom for at least 6 months, the CDC recommends in the Morbidity and Mortality Weekly Report released on March 25 (MMWR 2016. Mar 25. doi: http://dx.doi.org/10.15585/mmwr.mm6512e3er).

These recommendations update and replace those issued by the CDC on Feb. 5 and include new guidance for men who live in, or have traveled to, an area with active Zika virus transmission. The recommendations apply to all types of sexual activity involving the penis, including vaginal intercourse, anal intercourse, or fellatio.

“The previous recommendations focused on women who were already pregnant,” Dr. Denise J. Jamieson, co-lead of the Pregnancy and Birth Defects Team of the CDC Zika Virus Response Team, said during a press briefing. “What’s new is that we are now concerned about the periconceptional period, around the time the woman conceives.”

For men with pregnant sex partners, the agency recommends consistent and accurate use of condoms during any type of sex, or abstinence during the length of the pregnancy.

“This course is the best way to avoid even a minimal risk of sexual transmission of Zika virus, which could have adverse fetal effects when contracted during pregnancy,” the CDC report states, adding that pregnant women should ask their male sex partners about recent travel to areas with currently circulating Zika virus.

For couples not expecting a child, but concerned about sexual transmission of Zika, men with a confirmed Zika infection or clinical symptoms of Zika infection should consider using condoms or abstaining from sex for at least 6 months after their symptoms appear. This recommendation is based on tripling 62 days – the longest time interval after infection during which the virus was successfully isolated from semen.

If men have traveled to areas with active Zika transmission but have not developed symptoms, the CDC recommends condom use or abstinence for at least 8 weeks after leaving the area. Those living in areas with active transmission should also consider condom use during sex or abstaining from sex until active transmission has ceased.

These recommendations come as more evidence points to a link between Zika infection and fetal abnormalities, including microcephaly and fetal mortality.

“I think we’re learning more every day, and I think the evidence of a link between Zika and a range of poor pregnancy outcomes is becoming stronger and stronger,” Dr. Jamieson said. “At this point, we’re not using causal language, but the evidence is mounting.”

The CDC also released two other reports focusing on the need to increase access to contraception for residents of Puerto Rico and interim guidance for health care providers of women of childbearing age who have been potentially exposed to Zika virus.

As of March 25, the CDC has reported 273 U.S. cases of Zika virus infections from 35 states and Washington, D.C. All of these – except six sexually transmitted cases – are travel related.

Additionally, Puerto Rico’s most recent case total is 261, all locally transmitted by mosquitoes, except for three travel-associated cases. American Samoa has 14 cases, and the U.S. Virgin Islands have 11 cases, all thought to be locally transmitted.

“Long-acting contraception methods are not readily available in Puerto Rico, and from our health care provider colleagues in Puerto Rico, there is a desire to provide a more broad range of contraception options to women in Puerto Rico,” Dr. Jamieson said.

She said the CDC is developing a plan to make long-acting contraceptive methods more available in Puerto Rico.

When advising couples who wish to become pregnant after the man has had confirmed or suspected Zika infection, the CDC recommends waiting at least 6 months after the man’s onset of Zika symptoms or confirmed infection before attempting to conceive.

Although no evidence suggests that Zika virus will cause congenital infections in pregnancies conceived after a woman’s infection has resolved, data on the virus’s incubation period is limited, according to the CDC.

“Women with Zika virus disease should wait until at least 8 weeks after symptom onset before attempting conception,” wrote Dr. Emily E. Petersen and her colleagues in the guidance on caring for women of reproductive age with possible Zika virus exposure. “No data are available regarding the risk for congenital infection among pregnant women with asymptomatic infection.”

Similarly, asymptomatic women potentially exposed to Zika virus should also wait at least 8 weeks after the possible exposure date before trying to conceive.

Men potentially exposed to Zika virus should use a condom during all sex or abstain from sex for at least 8 weeks, according to new recommendations from the Centers for Disease Control and Prevention on reducing the risk of sexual transmission of the virus.

Men with confirmed infections or clinical symptoms of Zika should similarly abstain or use a condom for at least 6 months, the CDC recommends in the Morbidity and Mortality Weekly Report released on March 25 (MMWR 2016. Mar 25. doi: http://dx.doi.org/10.15585/mmwr.mm6512e3er).

These recommendations update and replace those issued by the CDC on Feb. 5 and include new guidance for men who live in, or have traveled to, an area with active Zika virus transmission. The recommendations apply to all types of sexual activity involving the penis, including vaginal intercourse, anal intercourse, or fellatio.

“The previous recommendations focused on women who were already pregnant,” Dr. Denise J. Jamieson, co-lead of the Pregnancy and Birth Defects Team of the CDC Zika Virus Response Team, said during a press briefing. “What’s new is that we are now concerned about the periconceptional period, around the time the woman conceives.”

For men with pregnant sex partners, the agency recommends consistent and accurate use of condoms during any type of sex, or abstinence during the length of the pregnancy.

“This course is the best way to avoid even a minimal risk of sexual transmission of Zika virus, which could have adverse fetal effects when contracted during pregnancy,” the CDC report states, adding that pregnant women should ask their male sex partners about recent travel to areas with currently circulating Zika virus.

For couples not expecting a child, but concerned about sexual transmission of Zika, men with a confirmed Zika infection or clinical symptoms of Zika infection should consider using condoms or abstaining from sex for at least 6 months after their symptoms appear. This recommendation is based on tripling 62 days – the longest time interval after infection during which the virus was successfully isolated from semen.

If men have traveled to areas with active Zika transmission but have not developed symptoms, the CDC recommends condom use or abstinence for at least 8 weeks after leaving the area. Those living in areas with active transmission should also consider condom use during sex or abstaining from sex until active transmission has ceased.

These recommendations come as more evidence points to a link between Zika infection and fetal abnormalities, including microcephaly and fetal mortality.

“I think we’re learning more every day, and I think the evidence of a link between Zika and a range of poor pregnancy outcomes is becoming stronger and stronger,” Dr. Jamieson said. “At this point, we’re not using causal language, but the evidence is mounting.”

The CDC also released two other reports focusing on the need to increase access to contraception for residents of Puerto Rico and interim guidance for health care providers of women of childbearing age who have been potentially exposed to Zika virus.

As of March 25, the CDC has reported 273 U.S. cases of Zika virus infections from 35 states and Washington, D.C. All of these – except six sexually transmitted cases – are travel related.

Additionally, Puerto Rico’s most recent case total is 261, all locally transmitted by mosquitoes, except for three travel-associated cases. American Samoa has 14 cases, and the U.S. Virgin Islands have 11 cases, all thought to be locally transmitted.

“Long-acting contraception methods are not readily available in Puerto Rico, and from our health care provider colleagues in Puerto Rico, there is a desire to provide a more broad range of contraception options to women in Puerto Rico,” Dr. Jamieson said.

She said the CDC is developing a plan to make long-acting contraceptive methods more available in Puerto Rico.

When advising couples who wish to become pregnant after the man has had confirmed or suspected Zika infection, the CDC recommends waiting at least 6 months after the man’s onset of Zika symptoms or confirmed infection before attempting to conceive.

Although no evidence suggests that Zika virus will cause congenital infections in pregnancies conceived after a woman’s infection has resolved, data on the virus’s incubation period is limited, according to the CDC.

“Women with Zika virus disease should wait until at least 8 weeks after symptom onset before attempting conception,” wrote Dr. Emily E. Petersen and her colleagues in the guidance on caring for women of reproductive age with possible Zika virus exposure. “No data are available regarding the risk for congenital infection among pregnant women with asymptomatic infection.”

Similarly, asymptomatic women potentially exposed to Zika virus should also wait at least 8 weeks after the possible exposure date before trying to conceive.

Men potentially exposed to Zika virus should use a condom during all sex or abstain from sex for at least 8 weeks, according to new recommendations from the Centers for Disease Control and Prevention on reducing the risk of sexual transmission of the virus.

Men with confirmed infections or clinical symptoms of Zika should similarly abstain or use a condom for at least 6 months, the CDC recommends in the Morbidity and Mortality Weekly Report released on March 25 (MMWR 2016. Mar 25. doi: http://dx.doi.org/10.15585/mmwr.mm6512e3er).

These recommendations update and replace those issued by the CDC on Feb. 5 and include new guidance for men who live in, or have traveled to, an area with active Zika virus transmission. The recommendations apply to all types of sexual activity involving the penis, including vaginal intercourse, anal intercourse, or fellatio.

“The previous recommendations focused on women who were already pregnant,” Dr. Denise J. Jamieson, co-lead of the Pregnancy and Birth Defects Team of the CDC Zika Virus Response Team, said during a press briefing. “What’s new is that we are now concerned about the periconceptional period, around the time the woman conceives.”

For men with pregnant sex partners, the agency recommends consistent and accurate use of condoms during any type of sex, or abstinence during the length of the pregnancy.

“This course is the best way to avoid even a minimal risk of sexual transmission of Zika virus, which could have adverse fetal effects when contracted during pregnancy,” the CDC report states, adding that pregnant women should ask their male sex partners about recent travel to areas with currently circulating Zika virus.

For couples not expecting a child, but concerned about sexual transmission of Zika, men with a confirmed Zika infection or clinical symptoms of Zika infection should consider using condoms or abstaining from sex for at least 6 months after their symptoms appear. This recommendation is based on tripling 62 days – the longest time interval after infection during which the virus was successfully isolated from semen.

If men have traveled to areas with active Zika transmission but have not developed symptoms, the CDC recommends condom use or abstinence for at least 8 weeks after leaving the area. Those living in areas with active transmission should also consider condom use during sex or abstaining from sex until active transmission has ceased.

These recommendations come as more evidence points to a link between Zika infection and fetal abnormalities, including microcephaly and fetal mortality.

“I think we’re learning more every day, and I think the evidence of a link between Zika and a range of poor pregnancy outcomes is becoming stronger and stronger,” Dr. Jamieson said. “At this point, we’re not using causal language, but the evidence is mounting.”

The CDC also released two other reports focusing on the need to increase access to contraception for residents of Puerto Rico and interim guidance for health care providers of women of childbearing age who have been potentially exposed to Zika virus.

As of March 25, the CDC has reported 273 U.S. cases of Zika virus infections from 35 states and Washington, D.C. All of these – except six sexually transmitted cases – are travel related.

Additionally, Puerto Rico’s most recent case total is 261, all locally transmitted by mosquitoes, except for three travel-associated cases. American Samoa has 14 cases, and the U.S. Virgin Islands have 11 cases, all thought to be locally transmitted.

“Long-acting contraception methods are not readily available in Puerto Rico, and from our health care provider colleagues in Puerto Rico, there is a desire to provide a more broad range of contraception options to women in Puerto Rico,” Dr. Jamieson said.

She said the CDC is developing a plan to make long-acting contraceptive methods more available in Puerto Rico.

When advising couples who wish to become pregnant after the man has had confirmed or suspected Zika infection, the CDC recommends waiting at least 6 months after the man’s onset of Zika symptoms or confirmed infection before attempting to conceive.

Although no evidence suggests that Zika virus will cause congenital infections in pregnancies conceived after a woman’s infection has resolved, data on the virus’s incubation period is limited, according to the CDC.

“Women with Zika virus disease should wait until at least 8 weeks after symptom onset before attempting conception,” wrote Dr. Emily E. Petersen and her colleagues in the guidance on caring for women of reproductive age with possible Zika virus exposure. “No data are available regarding the risk for congenital infection among pregnant women with asymptomatic infection.”

Similarly, asymptomatic women potentially exposed to Zika virus should also wait at least 8 weeks after the possible exposure date before trying to conceive.

The global infectious disease and clinical microbiology community meets every year at the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID), the world’s largest congress on infectious diseases and medical microbiology, to present and discuss recent research results and to offer solutions to the most pressing infection problems.

The 2016 ECCMID annual conference, organized by the European Society of Clinical Microbiology and Infectious Diseases (ESCMID), will take place April 9-12 in Amsterdam. Discussions at this event not only help translate research findings into diagnostic tools, guidelines, best practices, and international policies; they also raise awareness of emerging health care challenges.

At ECCMID 2016, researchers will present more than 3,000 abstracts with the latest findings and recommendations to help improve diagnosis, prevention, and the clinical care given to patients. The Congress offers more than 150 oral presentations, including keynote lectures, symposia, oral sessions, educational workshops, and meet-the-experts sessions, as well as more than 2,000 poster presentations.

The main topics this year are strategies to detect and tackle antimicrobial resistance in various settings, approaches for prevention involving vaccines and infection control, as well as descriptions of novel diagnostic technologies. Always among the most popular sessions are lectures by winners of the ESCMID Award for Excellence and the Young Investigator Awards, as well as oral presentations on groundbreaking research, and late-breaking abstracts.

Also included will be mini oral “e-poster” presentations. Printed posters will be presented, but they will also be available at e-poster viewing stations, where visitors can scroll through abstracts of paper presentations.

Keynote speeches this year will feature innovative approaches to vaccines; microbiome and tuberculosis therapies; lectures on how nonhuman antibiotics affect public health; and an economic perspective on antimicrobial resistance.

Special topics

This year, the ECCMID Program Committee has decided to offer two special tracks for the late-breaking abstract sessions, focused on topics requiring a coordinated response from infection specialists across all disciplines.

The first topic is refugee and migrant health. The thousands of people who are currently migrating challenge public health systems in transition and the host countries. Clinicians and public health specialists need to develop strategies for the screening, prevention, and treatment of infectious diseases that were largely eradicated in Europe but are now gradually being reintroduced.

The second focus of the late-breaking abstracts is on emerging colistin resistance. Reports about the emergence of plasmid-borne resistance to this last-resort antibiotic have come from China, Canada, the United Kingdom, and most countries in continental Europe. Colistin resistance can spread easily between different types of bacteria, says Dr. Murat Akova, current ESCMID president and professor of medicine at Hacettepe University in Ankara, Turkey, and the world needs to wake up and take note.

In terms of viral infections, experts at the Congress will evaluate HIV and hepatitis C treatments in several interesting sessions. Researchers will also present results on emerging infections, including those caused by the Zika virus. Dr. Jean Paul Stahl, vice chairman of the ESCMID Study Group for Infectious Diseases of the Brain and professor of infectious diseases at University Hospital in Grenoble, France, says the current Zika virus epidemic is an important example of the great need we have for new evidence-based approaches on how to best manage emerging infections.

The outbreaks of Zika and Ebola in the last few years have seen the international community mobilize on infectious disease issues in a more collaborative manner than ever before, which should help reduce the severity of future outbreaks. But viral infections extend far beyond the recent outbreaks of unusual pathologies, and there are a number of important developments taking place among some of the more common viruses.

For more information on ECCMID 2016, visit http://www.eccmid.org/.

Dr. Winfried V. Kern is professor of medicine at the Albert Ludwigs University of Freiburg and head of the division of infectious diseases, department of medicine, and Centre for Infectious Diseases and Travel Medicine, University Hospital, Freiburg, Germany. His professional interests include bacterial multidrug resistance mechanisms and epidemiology, hospital antibiotic stewardship programs, health care–associated infections including infections in the immunocompromised host.

The global infectious disease and clinical microbiology community meets every year at the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID), the world’s largest congress on infectious diseases and medical microbiology, to present and discuss recent research results and to offer solutions to the most pressing infection problems.

The 2016 ECCMID annual conference, organized by the European Society of Clinical Microbiology and Infectious Diseases (ESCMID), will take place April 9-12 in Amsterdam. Discussions at this event not only help translate research findings into diagnostic tools, guidelines, best practices, and international policies; they also raise awareness of emerging health care challenges.

At ECCMID 2016, researchers will present more than 3,000 abstracts with the latest findings and recommendations to help improve diagnosis, prevention, and the clinical care given to patients. The Congress offers more than 150 oral presentations, including keynote lectures, symposia, oral sessions, educational workshops, and meet-the-experts sessions, as well as more than 2,000 poster presentations.

The main topics this year are strategies to detect and tackle antimicrobial resistance in various settings, approaches for prevention involving vaccines and infection control, as well as descriptions of novel diagnostic technologies. Always among the most popular sessions are lectures by winners of the ESCMID Award for Excellence and the Young Investigator Awards, as well as oral presentations on groundbreaking research, and late-breaking abstracts.

Also included will be mini oral “e-poster” presentations. Printed posters will be presented, but they will also be available at e-poster viewing stations, where visitors can scroll through abstracts of paper presentations.

Keynote speeches this year will feature innovative approaches to vaccines; microbiome and tuberculosis therapies; lectures on how nonhuman antibiotics affect public health; and an economic perspective on antimicrobial resistance.

Special topics

This year, the ECCMID Program Committee has decided to offer two special tracks for the late-breaking abstract sessions, focused on topics requiring a coordinated response from infection specialists across all disciplines.

The first topic is refugee and migrant health. The thousands of people who are currently migrating challenge public health systems in transition and the host countries. Clinicians and public health specialists need to develop strategies for the screening, prevention, and treatment of infectious diseases that were largely eradicated in Europe but are now gradually being reintroduced.

The second focus of the late-breaking abstracts is on emerging colistin resistance. Reports about the emergence of plasmid-borne resistance to this last-resort antibiotic have come from China, Canada, the United Kingdom, and most countries in continental Europe. Colistin resistance can spread easily between different types of bacteria, says Dr. Murat Akova, current ESCMID president and professor of medicine at Hacettepe University in Ankara, Turkey, and the world needs to wake up and take note.

In terms of viral infections, experts at the Congress will evaluate HIV and hepatitis C treatments in several interesting sessions. Researchers will also present results on emerging infections, including those caused by the Zika virus. Dr. Jean Paul Stahl, vice chairman of the ESCMID Study Group for Infectious Diseases of the Brain and professor of infectious diseases at University Hospital in Grenoble, France, says the current Zika virus epidemic is an important example of the great need we have for new evidence-based approaches on how to best manage emerging infections.

The outbreaks of Zika and Ebola in the last few years have seen the international community mobilize on infectious disease issues in a more collaborative manner than ever before, which should help reduce the severity of future outbreaks. But viral infections extend far beyond the recent outbreaks of unusual pathologies, and there are a number of important developments taking place among some of the more common viruses.

For more information on ECCMID 2016, visit http://www.eccmid.org/.

Dr. Winfried V. Kern is professor of medicine at the Albert Ludwigs University of Freiburg and head of the division of infectious diseases, department of medicine, and Centre for Infectious Diseases and Travel Medicine, University Hospital, Freiburg, Germany. His professional interests include bacterial multidrug resistance mechanisms and epidemiology, hospital antibiotic stewardship programs, health care–associated infections including infections in the immunocompromised host.

The global infectious disease and clinical microbiology community meets every year at the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID), the world’s largest congress on infectious diseases and medical microbiology, to present and discuss recent research results and to offer solutions to the most pressing infection problems.

The 2016 ECCMID annual conference, organized by the European Society of Clinical Microbiology and Infectious Diseases (ESCMID), will take place April 9-12 in Amsterdam. Discussions at this event not only help translate research findings into diagnostic tools, guidelines, best practices, and international policies; they also raise awareness of emerging health care challenges.

At ECCMID 2016, researchers will present more than 3,000 abstracts with the latest findings and recommendations to help improve diagnosis, prevention, and the clinical care given to patients. The Congress offers more than 150 oral presentations, including keynote lectures, symposia, oral sessions, educational workshops, and meet-the-experts sessions, as well as more than 2,000 poster presentations.

The main topics this year are strategies to detect and tackle antimicrobial resistance in various settings, approaches for prevention involving vaccines and infection control, as well as descriptions of novel diagnostic technologies. Always among the most popular sessions are lectures by winners of the ESCMID Award for Excellence and the Young Investigator Awards, as well as oral presentations on groundbreaking research, and late-breaking abstracts.

Also included will be mini oral “e-poster” presentations. Printed posters will be presented, but they will also be available at e-poster viewing stations, where visitors can scroll through abstracts of paper presentations.

Keynote speeches this year will feature innovative approaches to vaccines; microbiome and tuberculosis therapies; lectures on how nonhuman antibiotics affect public health; and an economic perspective on antimicrobial resistance.

Special topics

This year, the ECCMID Program Committee has decided to offer two special tracks for the late-breaking abstract sessions, focused on topics requiring a coordinated response from infection specialists across all disciplines.

The first topic is refugee and migrant health. The thousands of people who are currently migrating challenge public health systems in transition and the host countries. Clinicians and public health specialists need to develop strategies for the screening, prevention, and treatment of infectious diseases that were largely eradicated in Europe but are now gradually being reintroduced.

The second focus of the late-breaking abstracts is on emerging colistin resistance. Reports about the emergence of plasmid-borne resistance to this last-resort antibiotic have come from China, Canada, the United Kingdom, and most countries in continental Europe. Colistin resistance can spread easily between different types of bacteria, says Dr. Murat Akova, current ESCMID president and professor of medicine at Hacettepe University in Ankara, Turkey, and the world needs to wake up and take note.

In terms of viral infections, experts at the Congress will evaluate HIV and hepatitis C treatments in several interesting sessions. Researchers will also present results on emerging infections, including those caused by the Zika virus. Dr. Jean Paul Stahl, vice chairman of the ESCMID Study Group for Infectious Diseases of the Brain and professor of infectious diseases at University Hospital in Grenoble, France, says the current Zika virus epidemic is an important example of the great need we have for new evidence-based approaches on how to best manage emerging infections.

The outbreaks of Zika and Ebola in the last few years have seen the international community mobilize on infectious disease issues in a more collaborative manner than ever before, which should help reduce the severity of future outbreaks. But viral infections extend far beyond the recent outbreaks of unusual pathologies, and there are a number of important developments taking place among some of the more common viruses.

For more information on ECCMID 2016, visit http://www.eccmid.org/.

Dr. Winfried V. Kern is professor of medicine at the Albert Ludwigs University of Freiburg and head of the division of infectious diseases, department of medicine, and Centre for Infectious Diseases and Travel Medicine, University Hospital, Freiburg, Germany. His professional interests include bacterial multidrug resistance mechanisms and epidemiology, hospital antibiotic stewardship programs, health care–associated infections including infections in the immunocompromised host.

In the March podcast for The Journal of Community and Supportive Oncology, Dr David Henry discusses a number of articles that center on patient quality of life and overall quality of care, among them, an article on opioid risk assessment in palliative care and three Original Reports, one on the impact of trimodality treatment on arm function and QoL in patients with superior sulcus tumors, a second on patient perceptions and the challenges of oral anticancer therapy, and a third on the use of voluntary reporting to assess symptom burden in cancer patients. A Review article by Jose de Souza and colleagues on financial toxicity in cancer care spans the QoL and quality of care spectrum as it details the implications of the increasing cost of cancer care for the delivery of high-quality, patient-centered care and discusses potential predictors of financial toxicity and instruments that could help quantify financial burden. Also in the line-up are articles on encapsulated irinotecan for hard-to-treat cancer and Case Reports on an uncommon presentation of lung cancer and on acute promyelocytic leukemia presenting as a paraspinal mass.

Listen to the podcast below.

In the March podcast for The Journal of Community and Supportive Oncology, Dr David Henry discusses a number of articles that center on patient quality of life and overall quality of care, among them, an article on opioid risk assessment in palliative care and three Original Reports, one on the impact of trimodality treatment on arm function and QoL in patients with superior sulcus tumors, a second on patient perceptions and the challenges of oral anticancer therapy, and a third on the use of voluntary reporting to assess symptom burden in cancer patients. A Review article by Jose de Souza and colleagues on financial toxicity in cancer care spans the QoL and quality of care spectrum as it details the implications of the increasing cost of cancer care for the delivery of high-quality, patient-centered care and discusses potential predictors of financial toxicity and instruments that could help quantify financial burden. Also in the line-up are articles on encapsulated irinotecan for hard-to-treat cancer and Case Reports on an uncommon presentation of lung cancer and on acute promyelocytic leukemia presenting as a paraspinal mass.

Listen to the podcast below.

In the March podcast for The Journal of Community and Supportive Oncology, Dr David Henry discusses a number of articles that center on patient quality of life and overall quality of care, among them, an article on opioid risk assessment in palliative care and three Original Reports, one on the impact of trimodality treatment on arm function and QoL in patients with superior sulcus tumors, a second on patient perceptions and the challenges of oral anticancer therapy, and a third on the use of voluntary reporting to assess symptom burden in cancer patients. A Review article by Jose de Souza and colleagues on financial toxicity in cancer care spans the QoL and quality of care spectrum as it details the implications of the increasing cost of cancer care for the delivery of high-quality, patient-centered care and discusses potential predictors of financial toxicity and instruments that could help quantify financial burden. Also in the line-up are articles on encapsulated irinotecan for hard-to-treat cancer and Case Reports on an uncommon presentation of lung cancer and on acute promyelocytic leukemia presenting as a paraspinal mass.

Listen to the podcast below.

Click on the PDF icon at the top of this introduction to read the full article. 

Click on the PDF icon at the top of this introduction to read the full article. 

Click on the PDF icon at the top of this introduction to read the full article. 

An open-label pilot study has found adjunct treatment with the SSRI antidepressant sertraline is associated with accelerated clearance of Cryptococcus from the cerebrospinal fluid in HIV-infected individuals with cryptococcal meningitis.

The dose-finding study of 172 HIV-infected Ugandan adults with cryptococcal meningitis showed any dose of sertraline, in combination with standard amphotericin B and high-dose fluconazole therapy, was associated with an average clearance rate of –0.37 log10 colony-forming U/mL CSF per day, compared with –0.30 observed in a previous study in individuals taking standard therapy without sertraline.

©grandeduc/Thinkstock

According to a paper published online in Lancet Infectious Diseases, the incidence of paradoxical immune reconstitution inflammatory syndrome was 5%, compared with rates of 17%-30% observed in other studies.

The sertraline doses used in the study ranged from 100 mg/day to 400 mg/day but researchers did not observe a significant difference in serious adverse events between the lower and higher doses (Lancet Infect Dis. 2016 Mar 9. doi: 10.1016/S1473-3099[16]00074-8).

Sertraline has previously shown potent fungicidal activity against Cryptococcus in vitro and in mice, suggesting it could be a promising therapeutic option for cryptococcal meningitis, a disease that accounts for 15%-20% of AIDS-related deaths in Africa.

“The current standard therapy for cryptococcal meningitis is based on antifungal regimens that are a half-century old, are associated with a range of toxic effects, and are largely inaccessible in areas of the world where they are needed most,” wrote Dr. Joshua Rhein of the University of Minnesota, Minneapolis, and his coauthors. “For this reason, the discovery of a widely available, nontoxic, and affordable drug effective against Cryptococcus would represent a substantial advance in preventing deaths.”

The study was supported by the National Institutes of Health, Fogarty International Center, Grand Challenges Canada, and the Doris Duke Charitable Foundation. No conflicts of interest were declared.

An open-label pilot study has found adjunct treatment with the SSRI antidepressant sertraline is associated with accelerated clearance of Cryptococcus from the cerebrospinal fluid in HIV-infected individuals with cryptococcal meningitis.

The dose-finding study of 172 HIV-infected Ugandan adults with cryptococcal meningitis showed any dose of sertraline, in combination with standard amphotericin B and high-dose fluconazole therapy, was associated with an average clearance rate of –0.37 log10 colony-forming U/mL CSF per day, compared with –0.30 observed in a previous study in individuals taking standard therapy without sertraline.

©grandeduc/Thinkstock

According to a paper published online in Lancet Infectious Diseases, the incidence of paradoxical immune reconstitution inflammatory syndrome was 5%, compared with rates of 17%-30% observed in other studies.

The sertraline doses used in the study ranged from 100 mg/day to 400 mg/day but researchers did not observe a significant difference in serious adverse events between the lower and higher doses (Lancet Infect Dis. 2016 Mar 9. doi: 10.1016/S1473-3099[16]00074-8).

Sertraline has previously shown potent fungicidal activity against Cryptococcus in vitro and in mice, suggesting it could be a promising therapeutic option for cryptococcal meningitis, a disease that accounts for 15%-20% of AIDS-related deaths in Africa.

“The current standard therapy for cryptococcal meningitis is based on antifungal regimens that are a half-century old, are associated with a range of toxic effects, and are largely inaccessible in areas of the world where they are needed most,” wrote Dr. Joshua Rhein of the University of Minnesota, Minneapolis, and his coauthors. “For this reason, the discovery of a widely available, nontoxic, and affordable drug effective against Cryptococcus would represent a substantial advance in preventing deaths.”

The study was supported by the National Institutes of Health, Fogarty International Center, Grand Challenges Canada, and the Doris Duke Charitable Foundation. No conflicts of interest were declared.

An open-label pilot study has found adjunct treatment with the SSRI antidepressant sertraline is associated with accelerated clearance of Cryptococcus from the cerebrospinal fluid in HIV-infected individuals with cryptococcal meningitis.

The dose-finding study of 172 HIV-infected Ugandan adults with cryptococcal meningitis showed any dose of sertraline, in combination with standard amphotericin B and high-dose fluconazole therapy, was associated with an average clearance rate of –0.37 log10 colony-forming U/mL CSF per day, compared with –0.30 observed in a previous study in individuals taking standard therapy without sertraline.

©grandeduc/Thinkstock

According to a paper published online in Lancet Infectious Diseases, the incidence of paradoxical immune reconstitution inflammatory syndrome was 5%, compared with rates of 17%-30% observed in other studies.

The sertraline doses used in the study ranged from 100 mg/day to 400 mg/day but researchers did not observe a significant difference in serious adverse events between the lower and higher doses (Lancet Infect Dis. 2016 Mar 9. doi: 10.1016/S1473-3099[16]00074-8).

Sertraline has previously shown potent fungicidal activity against Cryptococcus in vitro and in mice, suggesting it could be a promising therapeutic option for cryptococcal meningitis, a disease that accounts for 15%-20% of AIDS-related deaths in Africa.

“The current standard therapy for cryptococcal meningitis is based on antifungal regimens that are a half-century old, are associated with a range of toxic effects, and are largely inaccessible in areas of the world where they are needed most,” wrote Dr. Joshua Rhein of the University of Minnesota, Minneapolis, and his coauthors. “For this reason, the discovery of a widely available, nontoxic, and affordable drug effective against Cryptococcus would represent a substantial advance in preventing deaths.”

The study was supported by the National Institutes of Health, Fogarty International Center, Grand Challenges Canada, and the Doris Duke Charitable Foundation. No conflicts of interest were declared.

Objective To assess the symptom burden experienced by Yemeni cancer patients by using VR and systematic assessment (SA).

Methods 50 cancer patients were asked an open question to voluntarily report their symptoms. This was followed by an SA of a list of 20 common physical symptoms that was drawn up based on the literature.

Results From 375 symptom entries related to the 20 symptoms, VR accounted for 66 entries (18%) and SA for 309 (82%). The mean number of VR symptoms/patient was 1.3, and the mean number of VR plus SA symptoms was 7.5 (P < .001). In all, 74% of VR symptoms and 57% of SA symptoms were moderate or severe. For each symptom, the percentage of patients who experienced it and did not report it voluntarily (missed) was 100% for bleeding, constipation, early satiety, hoarseness, taste changes, and weight loss. These were followed by anorexia (97%), skin symptoms (92%), dry mouth (91%), edema (89%), dyspnea (88%), sore mouth (88%), fatigue/weakness (85%), diarrhea (80%), dysphagia (80%), nausea (76%), cough (75%), urinary symptoms (75%), vomiting (62%), and pain (18%). Pain was the most common voluntarily reported symptom (56% of patients), the most commonly distressing (42%), and the least under-reported (18%).

Limitations Relatively small sample size; the SA included only 20 symptoms.

Conclusions SA of symptoms yields a more accurate estimation of symptom burden than does VR. As with many developing countries where the majority of cancer patients present at an incurable disease stage, Yemeni cancer patients suffer a high symptom burden, especially pain.

Click on the PDF icon at the top of this introduction to read the full article. 

Objective To assess the symptom burden experienced by Yemeni cancer patients by using VR and systematic assessment (SA).

Methods 50 cancer patients were asked an open question to voluntarily report their symptoms. This was followed by an SA of a list of 20 common physical symptoms that was drawn up based on the literature.

Results From 375 symptom entries related to the 20 symptoms, VR accounted for 66 entries (18%) and SA for 309 (82%). The mean number of VR symptoms/patient was 1.3, and the mean number of VR plus SA symptoms was 7.5 (P < .001). In all, 74% of VR symptoms and 57% of SA symptoms were moderate or severe. For each symptom, the percentage of patients who experienced it and did not report it voluntarily (missed) was 100% for bleeding, constipation, early satiety, hoarseness, taste changes, and weight loss. These were followed by anorexia (97%), skin symptoms (92%), dry mouth (91%), edema (89%), dyspnea (88%), sore mouth (88%), fatigue/weakness (85%), diarrhea (80%), dysphagia (80%), nausea (76%), cough (75%), urinary symptoms (75%), vomiting (62%), and pain (18%). Pain was the most common voluntarily reported symptom (56% of patients), the most commonly distressing (42%), and the least under-reported (18%).

Limitations Relatively small sample size; the SA included only 20 symptoms.

Conclusions SA of symptoms yields a more accurate estimation of symptom burden than does VR. As with many developing countries where the majority of cancer patients present at an incurable disease stage, Yemeni cancer patients suffer a high symptom burden, especially pain.

Click on the PDF icon at the top of this introduction to read the full article. 

Objective To assess the symptom burden experienced by Yemeni cancer patients by using VR and systematic assessment (SA).

Methods 50 cancer patients were asked an open question to voluntarily report their symptoms. This was followed by an SA of a list of 20 common physical symptoms that was drawn up based on the literature.

Results From 375 symptom entries related to the 20 symptoms, VR accounted for 66 entries (18%) and SA for 309 (82%). The mean number of VR symptoms/patient was 1.3, and the mean number of VR plus SA symptoms was 7.5 (P < .001). In all, 74% of VR symptoms and 57% of SA symptoms were moderate or severe. For each symptom, the percentage of patients who experienced it and did not report it voluntarily (missed) was 100% for bleeding, constipation, early satiety, hoarseness, taste changes, and weight loss. These were followed by anorexia (97%), skin symptoms (92%), dry mouth (91%), edema (89%), dyspnea (88%), sore mouth (88%), fatigue/weakness (85%), diarrhea (80%), dysphagia (80%), nausea (76%), cough (75%), urinary symptoms (75%), vomiting (62%), and pain (18%). Pain was the most common voluntarily reported symptom (56% of patients), the most commonly distressing (42%), and the least under-reported (18%).

Limitations Relatively small sample size; the SA included only 20 symptoms.

Conclusions SA of symptoms yields a more accurate estimation of symptom burden than does VR. As with many developing countries where the majority of cancer patients present at an incurable disease stage, Yemeni cancer patients suffer a high symptom burden, especially pain.

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Background Oral anticancer agents are more convenient to use and better tolerated than traditional intravenous therapy but come with significant concerns about patient noncompliance, adverse effects, and high cost. Identifying areas for improvement in the medication use process may help ensure optimal use of these agents.

Results Most of the patients were satisfied with their oral treatment, reporting ease of use with minimal side effect occurrence. Oncologists and pharmacists were equally named as sources of drug information.

Limitations Sample size was small and patients were overwhelmingly male. Response bias may be partially responsible for the observed results for regimen management, side effect occurrence, missed doses, and overall treatment satisfaction.

Conclusion Oral anticancer therapy represents a significant therapeutic advance for many types of cancer. Pharmacists can serve as vital informational resources to these patients. Further studies examining the role of pharmacist-led educational programs in terms of overall patient outcomes are warranted.

Background Oral anticancer agents are more convenient to use and better tolerated than traditional intravenous therapy but come with significant concerns about patient noncompliance, adverse effects, and high cost. Identifying areas for improvement in the medication use process may help ensure optimal use of these agents.

Results Most of the patients were satisfied with their oral treatment, reporting ease of use with minimal side effect occurrence. Oncologists and pharmacists were equally named as sources of drug information.

Limitations Sample size was small and patients were overwhelmingly male. Response bias may be partially responsible for the observed results for regimen management, side effect occurrence, missed doses, and overall treatment satisfaction.

Conclusion Oral anticancer therapy represents a significant therapeutic advance for many types of cancer. Pharmacists can serve as vital informational resources to these patients. Further studies examining the role of pharmacist-led educational programs in terms of overall patient outcomes are warranted.

Background Oral anticancer agents are more convenient to use and better tolerated than traditional intravenous therapy but come with significant concerns about patient noncompliance, adverse effects, and high cost. Identifying areas for improvement in the medication use process may help ensure optimal use of these agents.

Results Most of the patients were satisfied with their oral treatment, reporting ease of use with minimal side effect occurrence. Oncologists and pharmacists were equally named as sources of drug information.

Limitations Sample size was small and patients were overwhelmingly male. Response bias may be partially responsible for the observed results for regimen management, side effect occurrence, missed doses, and overall treatment satisfaction.

Conclusion Oral anticancer therapy represents a significant therapeutic advance for many types of cancer. Pharmacists can serve as vital informational resources to these patients. Further studies examining the role of pharmacist-led educational programs in terms of overall patient outcomes are warranted.