A mitochondrial protein may help explain gender differences in cardiovascular risk, PPIs may boost the risk of C. difficile infections in hospitals, topical retinoids and benzoyl peroxide are tops for inflammatory acne, and the grueling prior authorization process may get easier.

Listen to the MDedge Daily News podcast for all the details on today’s top news. 

A mitochondrial protein may help explain gender differences in cardiovascular risk, PPIs may boost the risk of C. difficile infections in hospitals, topical retinoids and benzoyl peroxide are tops for inflammatory acne, and the grueling prior authorization process may get easier.

Listen to the MDedge Daily News podcast for all the details on today’s top news. 

A mitochondrial protein may help explain gender differences in cardiovascular risk, PPIs may boost the risk of C. difficile infections in hospitals, topical retinoids and benzoyl peroxide are tops for inflammatory acne, and the grueling prior authorization process may get easier.

Listen to the MDedge Daily News podcast for all the details on today’s top news. 

Alex M. Azar II has been confirmed as secretary of the Department of Health & Human Services following a Jan. 24 vote in the U.S. Senate.

His nomination passed by a vote of 55-43.

Mr. Azar has previously been confirmed to two posts at HHS, first as general counsel and later as deputy HHS secretary during the administration of President George W. Bush between 2001 and 2007. Both appointments were confirmed by unanimous consent in the Senate.

Wikimedia Commons/WWsgConnect/CC-SA 4.0

[email protected]

Alex M. Azar II has been confirmed as secretary of the Department of Health & Human Services following a Jan. 24 vote in the U.S. Senate.

His nomination passed by a vote of 55-43.

Mr. Azar has previously been confirmed to two posts at HHS, first as general counsel and later as deputy HHS secretary during the administration of President George W. Bush between 2001 and 2007. Both appointments were confirmed by unanimous consent in the Senate.

Wikimedia Commons/WWsgConnect/CC-SA 4.0

[email protected]

Alex M. Azar II has been confirmed as secretary of the Department of Health & Human Services following a Jan. 24 vote in the U.S. Senate.

His nomination passed by a vote of 55-43.

Mr. Azar has previously been confirmed to two posts at HHS, first as general counsel and later as deputy HHS secretary during the administration of President George W. Bush between 2001 and 2007. Both appointments were confirmed by unanimous consent in the Senate.

Wikimedia Commons/WWsgConnect/CC-SA 4.0

[email protected]

After decades of hype, dashed hopes, and setbacks, gene therapy has finally arrived and is poised to transform the treatment paradigm for many diseases, according to Cynthia E. Dunbar, MD, senior investigator at the Hematology Branch of the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

Hematologists can expect more developments that build on current successes with chimeric antigen receptor (CAR) T-cell therapy and gene therapy advances for hemophilia, as well as emerging advances in gene editing techniques including the CRISPR/Cas9 approach, Dr. Dunbar said in an interview.

Courtesy National Heart, Lung, and Blood Institute

Milestones

A new approach to cancer treatment was ushered in on Aug. 30, 2017, with the Food and Drug Administration approval of tisagenlecleucel, the first-ever gene therapy available in the United States. The CD19-directed CAR T-cell therapy is indicated for treatment of certain pediatric or young adult patients with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse.

Soon afterward, FDA approved another CD19-directed CAR T-cell therapy, axicabtagene ciloleucel, for adult patients with large B-cell lymphoma after two or more lines of systemic therapy.

“It’s a very interesting time for immunotherapies in general,” Dr. Dunbar said. “There’s a huge number of options in terms of PD-1 inhibitors and other pharmacologics or antibodies that allow the patient’s own immune system to attack tumors. CAR T-cell therapy is an obvious step beyond that, in terms of arming your own T cells to very specifically target tumor cells.”

But randomized trials or meta-analyses may be necessary to determine the place of CAR T-cell therapy in the treatment armamentarium for acute lymphoblastic leukemia and large B-cell lymphoma given their cost and the availability of other therapeutic options, Dr. Dunbar suggested.

“Gene therapies have a large upfront cost, but if they’re truly curative and a one-time treatment, then they may in the long run be much cheaper than doing failed multiple transplants or needing monoclonal antibody infusion every 2 weeks for the rest of your life,” she said.

Another major success story still in the works, according to Dr. Dunbar, is the treatment of hemophilia A and B with gene therapy approaches. The positive data include a recent report showing that transgene-derived factor IX coagulant activity allowed for the termination of baseline prophylaxis, and the near elimination of bleeding and factor use, in patients with hemophilia B (N Engl J Med. 2017 Dec 7;377[23]:2215-27).

While gene therapy for hemophilia A has been more challenging, another recent report nevertheless demonstrated sustained normalization of factor VIII activity level with a single intravenous infusion of adeno-associated virus serotype 5 vector encoding a B-domain–deleted human factor VIII (N Engl J Med. 2017 Dec 28;377[26]:2519-30).

“The proof-of-principle was already there in hemophilia B,” Dr. Dunbar said. “It really was just a question of figuring out a way to package and deliver a Factor VIII that would work in the constraints of an AAV [adeno-associated virus] vector.”

Meanwhile, myeloma trials of CAR T-cell therapy seem very promising so far, but the challenge in that disease could be finding a place for gene therapy in a “much more diverse treatment landscape” that includes multiple effective regimens, according to Dr. Dunbar.
 

Future trends, challenges

Looking forward, gene editing with methods including the CRISPR/Cas9 approach have the potential to revolutionize treatment in HIV, b-thalassemia, and sickle cell disease, she said.

Notably, genome editing approaches to treat sickle cell anemia are likely to move forward in the near future, according to Dr. Dunbar, following reports validating an erythroid enhancer of human BCL11A as a target for reinduction of fetal hemoglobin (Nature. 2015 Nov 12;527[7577]:192-7).

But all of this gene therapy development creates an educational challenge for frontline clinicians, even if the administration of CAR T-cell therapy and other advanced treatments is limited to highly specialized centers.

“There’s a lot of training that needs to go on with hematologists, oncologists, and other doctors about how to care for these patients after these treatments, in terms of what to look for and how to intervene early to prevent, for instance, severe toxicity from cytokine release syndrome,” Dr. Dunbar said.

Dr. Dunbar reported having no relevant financial disclosures.

After decades of hype, dashed hopes, and setbacks, gene therapy has finally arrived and is poised to transform the treatment paradigm for many diseases, according to Cynthia E. Dunbar, MD, senior investigator at the Hematology Branch of the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

Hematologists can expect more developments that build on current successes with chimeric antigen receptor (CAR) T-cell therapy and gene therapy advances for hemophilia, as well as emerging advances in gene editing techniques including the CRISPR/Cas9 approach, Dr. Dunbar said in an interview.

Courtesy National Heart, Lung, and Blood Institute

Milestones

A new approach to cancer treatment was ushered in on Aug. 30, 2017, with the Food and Drug Administration approval of tisagenlecleucel, the first-ever gene therapy available in the United States. The CD19-directed CAR T-cell therapy is indicated for treatment of certain pediatric or young adult patients with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse.

Soon afterward, FDA approved another CD19-directed CAR T-cell therapy, axicabtagene ciloleucel, for adult patients with large B-cell lymphoma after two or more lines of systemic therapy.

“It’s a very interesting time for immunotherapies in general,” Dr. Dunbar said. “There’s a huge number of options in terms of PD-1 inhibitors and other pharmacologics or antibodies that allow the patient’s own immune system to attack tumors. CAR T-cell therapy is an obvious step beyond that, in terms of arming your own T cells to very specifically target tumor cells.”

But randomized trials or meta-analyses may be necessary to determine the place of CAR T-cell therapy in the treatment armamentarium for acute lymphoblastic leukemia and large B-cell lymphoma given their cost and the availability of other therapeutic options, Dr. Dunbar suggested.

“Gene therapies have a large upfront cost, but if they’re truly curative and a one-time treatment, then they may in the long run be much cheaper than doing failed multiple transplants or needing monoclonal antibody infusion every 2 weeks for the rest of your life,” she said.

Another major success story still in the works, according to Dr. Dunbar, is the treatment of hemophilia A and B with gene therapy approaches. The positive data include a recent report showing that transgene-derived factor IX coagulant activity allowed for the termination of baseline prophylaxis, and the near elimination of bleeding and factor use, in patients with hemophilia B (N Engl J Med. 2017 Dec 7;377[23]:2215-27).

While gene therapy for hemophilia A has been more challenging, another recent report nevertheless demonstrated sustained normalization of factor VIII activity level with a single intravenous infusion of adeno-associated virus serotype 5 vector encoding a B-domain–deleted human factor VIII (N Engl J Med. 2017 Dec 28;377[26]:2519-30).

“The proof-of-principle was already there in hemophilia B,” Dr. Dunbar said. “It really was just a question of figuring out a way to package and deliver a Factor VIII that would work in the constraints of an AAV [adeno-associated virus] vector.”

Meanwhile, myeloma trials of CAR T-cell therapy seem very promising so far, but the challenge in that disease could be finding a place for gene therapy in a “much more diverse treatment landscape” that includes multiple effective regimens, according to Dr. Dunbar.
 

Future trends, challenges

Looking forward, gene editing with methods including the CRISPR/Cas9 approach have the potential to revolutionize treatment in HIV, b-thalassemia, and sickle cell disease, she said.

Notably, genome editing approaches to treat sickle cell anemia are likely to move forward in the near future, according to Dr. Dunbar, following reports validating an erythroid enhancer of human BCL11A as a target for reinduction of fetal hemoglobin (Nature. 2015 Nov 12;527[7577]:192-7).

But all of this gene therapy development creates an educational challenge for frontline clinicians, even if the administration of CAR T-cell therapy and other advanced treatments is limited to highly specialized centers.

“There’s a lot of training that needs to go on with hematologists, oncologists, and other doctors about how to care for these patients after these treatments, in terms of what to look for and how to intervene early to prevent, for instance, severe toxicity from cytokine release syndrome,” Dr. Dunbar said.

Dr. Dunbar reported having no relevant financial disclosures.

After decades of hype, dashed hopes, and setbacks, gene therapy has finally arrived and is poised to transform the treatment paradigm for many diseases, according to Cynthia E. Dunbar, MD, senior investigator at the Hematology Branch of the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

Hematologists can expect more developments that build on current successes with chimeric antigen receptor (CAR) T-cell therapy and gene therapy advances for hemophilia, as well as emerging advances in gene editing techniques including the CRISPR/Cas9 approach, Dr. Dunbar said in an interview.

Courtesy National Heart, Lung, and Blood Institute

Milestones

A new approach to cancer treatment was ushered in on Aug. 30, 2017, with the Food and Drug Administration approval of tisagenlecleucel, the first-ever gene therapy available in the United States. The CD19-directed CAR T-cell therapy is indicated for treatment of certain pediatric or young adult patients with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse.

Soon afterward, FDA approved another CD19-directed CAR T-cell therapy, axicabtagene ciloleucel, for adult patients with large B-cell lymphoma after two or more lines of systemic therapy.

“It’s a very interesting time for immunotherapies in general,” Dr. Dunbar said. “There’s a huge number of options in terms of PD-1 inhibitors and other pharmacologics or antibodies that allow the patient’s own immune system to attack tumors. CAR T-cell therapy is an obvious step beyond that, in terms of arming your own T cells to very specifically target tumor cells.”

But randomized trials or meta-analyses may be necessary to determine the place of CAR T-cell therapy in the treatment armamentarium for acute lymphoblastic leukemia and large B-cell lymphoma given their cost and the availability of other therapeutic options, Dr. Dunbar suggested.

“Gene therapies have a large upfront cost, but if they’re truly curative and a one-time treatment, then they may in the long run be much cheaper than doing failed multiple transplants or needing monoclonal antibody infusion every 2 weeks for the rest of your life,” she said.

Another major success story still in the works, according to Dr. Dunbar, is the treatment of hemophilia A and B with gene therapy approaches. The positive data include a recent report showing that transgene-derived factor IX coagulant activity allowed for the termination of baseline prophylaxis, and the near elimination of bleeding and factor use, in patients with hemophilia B (N Engl J Med. 2017 Dec 7;377[23]:2215-27).

While gene therapy for hemophilia A has been more challenging, another recent report nevertheless demonstrated sustained normalization of factor VIII activity level with a single intravenous infusion of adeno-associated virus serotype 5 vector encoding a B-domain–deleted human factor VIII (N Engl J Med. 2017 Dec 28;377[26]:2519-30).

“The proof-of-principle was already there in hemophilia B,” Dr. Dunbar said. “It really was just a question of figuring out a way to package and deliver a Factor VIII that would work in the constraints of an AAV [adeno-associated virus] vector.”

Meanwhile, myeloma trials of CAR T-cell therapy seem very promising so far, but the challenge in that disease could be finding a place for gene therapy in a “much more diverse treatment landscape” that includes multiple effective regimens, according to Dr. Dunbar.
 

Future trends, challenges

Looking forward, gene editing with methods including the CRISPR/Cas9 approach have the potential to revolutionize treatment in HIV, b-thalassemia, and sickle cell disease, she said.

Notably, genome editing approaches to treat sickle cell anemia are likely to move forward in the near future, according to Dr. Dunbar, following reports validating an erythroid enhancer of human BCL11A as a target for reinduction of fetal hemoglobin (Nature. 2015 Nov 12;527[7577]:192-7).

But all of this gene therapy development creates an educational challenge for frontline clinicians, even if the administration of CAR T-cell therapy and other advanced treatments is limited to highly specialized centers.

“There’s a lot of training that needs to go on with hematologists, oncologists, and other doctors about how to care for these patients after these treatments, in terms of what to look for and how to intervene early to prevent, for instance, severe toxicity from cytokine release syndrome,” Dr. Dunbar said.

Dr. Dunbar reported having no relevant financial disclosures.

Cisplatin and other platinum-based drugs are prescribed to 10% to 20% of patients with cancer. The drugs cause permanent hearing loss in as many as 80% of adult patients and at least half of children. Why is cisplatin so toxic to the inner ear?

Researchers from the National Institute on Deafness and other Communications Disorders may have found the answer. The inner ear readily takes up cisplatin but has little ability to remove the drug. In most areas of the body, cisplatin is eliminated within days or weeks after treatment; in the inner ear it remains much longer.

Using a mouse model, the researchers found cisplatin remained in the inner ear much longer than in most other body tissues and built up with each treatment. They also studied inner ear tissue donated by deceased patients who had been treated with cisplatin and found cisplatin remained in the inner ear many months or even years after treatment. They also examined inner ear tissue from one child, and found cisplatin buildup even higher than that seen in adults.

The highest buildup of cisplatin was in the stria vascularis, which helps maintain the positive electrical charge in inner ear fluid that certain cells need to detect sound, the researchers say. They believe that accumulation contributed to the hearing loss. “If we can prevent cisplatin from entering the stria vascularis in the inner ear during treatment,” Lisa Cunningham, PhD, lead investigator says, “we may be able to protect cancer patients from developing cisplatin-induced hearing loss.”

Cisplatin and other platinum-based drugs are prescribed to 10% to 20% of patients with cancer. The drugs cause permanent hearing loss in as many as 80% of adult patients and at least half of children. Why is cisplatin so toxic to the inner ear?

Researchers from the National Institute on Deafness and other Communications Disorders may have found the answer. The inner ear readily takes up cisplatin but has little ability to remove the drug. In most areas of the body, cisplatin is eliminated within days or weeks after treatment; in the inner ear it remains much longer.

Using a mouse model, the researchers found cisplatin remained in the inner ear much longer than in most other body tissues and built up with each treatment. They also studied inner ear tissue donated by deceased patients who had been treated with cisplatin and found cisplatin remained in the inner ear many months or even years after treatment. They also examined inner ear tissue from one child, and found cisplatin buildup even higher than that seen in adults.

The highest buildup of cisplatin was in the stria vascularis, which helps maintain the positive electrical charge in inner ear fluid that certain cells need to detect sound, the researchers say. They believe that accumulation contributed to the hearing loss. “If we can prevent cisplatin from entering the stria vascularis in the inner ear during treatment,” Lisa Cunningham, PhD, lead investigator says, “we may be able to protect cancer patients from developing cisplatin-induced hearing loss.”

Cisplatin and other platinum-based drugs are prescribed to 10% to 20% of patients with cancer. The drugs cause permanent hearing loss in as many as 80% of adult patients and at least half of children. Why is cisplatin so toxic to the inner ear?

Researchers from the National Institute on Deafness and other Communications Disorders may have found the answer. The inner ear readily takes up cisplatin but has little ability to remove the drug. In most areas of the body, cisplatin is eliminated within days or weeks after treatment; in the inner ear it remains much longer.

Using a mouse model, the researchers found cisplatin remained in the inner ear much longer than in most other body tissues and built up with each treatment. They also studied inner ear tissue donated by deceased patients who had been treated with cisplatin and found cisplatin remained in the inner ear many months or even years after treatment. They also examined inner ear tissue from one child, and found cisplatin buildup even higher than that seen in adults.

The highest buildup of cisplatin was in the stria vascularis, which helps maintain the positive electrical charge in inner ear fluid that certain cells need to detect sound, the researchers say. They believe that accumulation contributed to the hearing loss. “If we can prevent cisplatin from entering the stria vascularis in the inner ear during treatment,” Lisa Cunningham, PhD, lead investigator says, “we may be able to protect cancer patients from developing cisplatin-induced hearing loss.”

of Western Australia

Researchers say they have developed a new technique for assessing chromosomal abnormalities in chronic lymphocytic leukemia (CLL).

The team believes their method, called immuno-flowFISH, could be used at the time of CLL diagnosis for disease stratification and after treatment to assess residual disease.

Kathryn A. Fuller, PhD, of The University of Western Australia in Crawley, Australia, and her colleagues described immuno-flowFISH in the journal Methods.

The name “immuno-flowFISH” acknowledges what has been incorporated into this technology.

“Immuno” recognizes that immunology testing is used to identify the CLL cells. “Flow” is used because the machine is an imaging flow cytometer. And “FISH” is the test that identifies the chromosomes inside the cells.

The researchers said they found that immuno-flowFISH could detect trisomic chromosomal abnormalities in cells with the phenotype of CLL.

And immuno-flowFISH provided greater specificity and sensitivity than standard FISH.

In particular, the researchers were able to analyze 10,000 to 20,000 cells in each sample, which is 100 to 200 times greater than traditional FISH methods.

“The imaging cytometer can analyze samples at a rate of up to 2000 cells per second, which means we can investigate a large number of cells in a relatively short amount of time, giving us greater sensitivity,” Dr Fuller said.

“This immuno-flowFISH method is an exciting development in personalizing pathology testing for leukemia,” added study author Wendy N. Erber, MD, DPhil, PhD, of The University of Western Australia.

Dr Erber and her colleagues are now expanding immuno-flowFISH so it can be applied to other malignancies as well.

of Western Australia

Researchers say they have developed a new technique for assessing chromosomal abnormalities in chronic lymphocytic leukemia (CLL).

The team believes their method, called immuno-flowFISH, could be used at the time of CLL diagnosis for disease stratification and after treatment to assess residual disease.

Kathryn A. Fuller, PhD, of The University of Western Australia in Crawley, Australia, and her colleagues described immuno-flowFISH in the journal Methods.

The name “immuno-flowFISH” acknowledges what has been incorporated into this technology.

“Immuno” recognizes that immunology testing is used to identify the CLL cells. “Flow” is used because the machine is an imaging flow cytometer. And “FISH” is the test that identifies the chromosomes inside the cells.

The researchers said they found that immuno-flowFISH could detect trisomic chromosomal abnormalities in cells with the phenotype of CLL.

And immuno-flowFISH provided greater specificity and sensitivity than standard FISH.

In particular, the researchers were able to analyze 10,000 to 20,000 cells in each sample, which is 100 to 200 times greater than traditional FISH methods.

“The imaging cytometer can analyze samples at a rate of up to 2000 cells per second, which means we can investigate a large number of cells in a relatively short amount of time, giving us greater sensitivity,” Dr Fuller said.

“This immuno-flowFISH method is an exciting development in personalizing pathology testing for leukemia,” added study author Wendy N. Erber, MD, DPhil, PhD, of The University of Western Australia.

Dr Erber and her colleagues are now expanding immuno-flowFISH so it can be applied to other malignancies as well.

of Western Australia

Researchers say they have developed a new technique for assessing chromosomal abnormalities in chronic lymphocytic leukemia (CLL).

The team believes their method, called immuno-flowFISH, could be used at the time of CLL diagnosis for disease stratification and after treatment to assess residual disease.

Kathryn A. Fuller, PhD, of The University of Western Australia in Crawley, Australia, and her colleagues described immuno-flowFISH in the journal Methods.

The name “immuno-flowFISH” acknowledges what has been incorporated into this technology.

“Immuno” recognizes that immunology testing is used to identify the CLL cells. “Flow” is used because the machine is an imaging flow cytometer. And “FISH” is the test that identifies the chromosomes inside the cells.

The researchers said they found that immuno-flowFISH could detect trisomic chromosomal abnormalities in cells with the phenotype of CLL.

And immuno-flowFISH provided greater specificity and sensitivity than standard FISH.

In particular, the researchers were able to analyze 10,000 to 20,000 cells in each sample, which is 100 to 200 times greater than traditional FISH methods.

“The imaging cytometer can analyze samples at a rate of up to 2000 cells per second, which means we can investigate a large number of cells in a relatively short amount of time, giving us greater sensitivity,” Dr Fuller said.

“This immuno-flowFISH method is an exciting development in personalizing pathology testing for leukemia,” added study author Wendy N. Erber, MD, DPhil, PhD, of The University of Western Australia.

Dr Erber and her colleagues are now expanding immuno-flowFISH so it can be applied to other malignancies as well.

Essential thrombocythemia

The US Food and Drug Administration (FDA) has cleared use of QIAGEN’s ipsogen JAK2 RGQ PCR Kit (ipsogen JAK2 assay) for the diagnosis of all myeloproliferative neoplasms (MPNs).

The ipsogen JAK2 assay is a qualitative, in vitro diagnostic test designed to detect the JAK2 V617F/G1849T allele in genomic DNA extracted from EDTA whole blood.

The assay was previously cleared by the FDA for use in conjunction with other clinicopathological factors to aid the diagnosis of polycythemia vera.

Now, the FDA has cleared use of the assay for 2 additional MPNs—essential thrombocythemia and primary myelofibrosis.

“We are eager to expand the use of our ipsogen JAK2 assay, which is already available in Europe and other markets, for use in a wider range of patients in the US,” said Thierry Bernard, senior vice president and head of QIAGEN’s Molecular Diagnostics Business Area.

“Our JAK2 assay makes it easier for hematologists and oncologists to follow recommended diagnostic testing algorithms and international guidelines for their patients suspected of having MPNs.”

The ipsogen JAK2 assay is a real-time PCR test performed on the QIAGEN Rotor-Gene Q MDx instrument.

The test is intended for use as an adjunct to the evaluation of suspected MPNs, in conjunction with other clinicopathological factors.

The ipsogen JAK2 assay does not detect less common JAK2 mutations associated with MPNs, including mutations in exon 12, and is not intended for stand-alone diagnosis of MPNs.

Essential thrombocythemia

The US Food and Drug Administration (FDA) has cleared use of QIAGEN’s ipsogen JAK2 RGQ PCR Kit (ipsogen JAK2 assay) for the diagnosis of all myeloproliferative neoplasms (MPNs).

The ipsogen JAK2 assay is a qualitative, in vitro diagnostic test designed to detect the JAK2 V617F/G1849T allele in genomic DNA extracted from EDTA whole blood.

The assay was previously cleared by the FDA for use in conjunction with other clinicopathological factors to aid the diagnosis of polycythemia vera.

Now, the FDA has cleared use of the assay for 2 additional MPNs—essential thrombocythemia and primary myelofibrosis.

“We are eager to expand the use of our ipsogen JAK2 assay, which is already available in Europe and other markets, for use in a wider range of patients in the US,” said Thierry Bernard, senior vice president and head of QIAGEN’s Molecular Diagnostics Business Area.

“Our JAK2 assay makes it easier for hematologists and oncologists to follow recommended diagnostic testing algorithms and international guidelines for their patients suspected of having MPNs.”

The ipsogen JAK2 assay is a real-time PCR test performed on the QIAGEN Rotor-Gene Q MDx instrument.

The test is intended for use as an adjunct to the evaluation of suspected MPNs, in conjunction with other clinicopathological factors.

The ipsogen JAK2 assay does not detect less common JAK2 mutations associated with MPNs, including mutations in exon 12, and is not intended for stand-alone diagnosis of MPNs.

Essential thrombocythemia

The US Food and Drug Administration (FDA) has cleared use of QIAGEN’s ipsogen JAK2 RGQ PCR Kit (ipsogen JAK2 assay) for the diagnosis of all myeloproliferative neoplasms (MPNs).

The ipsogen JAK2 assay is a qualitative, in vitro diagnostic test designed to detect the JAK2 V617F/G1849T allele in genomic DNA extracted from EDTA whole blood.

The assay was previously cleared by the FDA for use in conjunction with other clinicopathological factors to aid the diagnosis of polycythemia vera.

Now, the FDA has cleared use of the assay for 2 additional MPNs—essential thrombocythemia and primary myelofibrosis.

“We are eager to expand the use of our ipsogen JAK2 assay, which is already available in Europe and other markets, for use in a wider range of patients in the US,” said Thierry Bernard, senior vice president and head of QIAGEN’s Molecular Diagnostics Business Area.

“Our JAK2 assay makes it easier for hematologists and oncologists to follow recommended diagnostic testing algorithms and international guidelines for their patients suspected of having MPNs.”

The ipsogen JAK2 assay is a real-time PCR test performed on the QIAGEN Rotor-Gene Q MDx instrument.

The test is intended for use as an adjunct to the evaluation of suspected MPNs, in conjunction with other clinicopathological factors.

The ipsogen JAK2 assay does not detect less common JAK2 mutations associated with MPNs, including mutations in exon 12, and is not intended for stand-alone diagnosis of MPNs.

Photo by Chad McNeeley

The European Commission (EC) has granted orphan designation to NLA101 as an adjunct to hematopoietic stem cell transplant (HSCT).

NLA101 is a universal, off-the-shelf, stem and progenitor cell therapy intended to provide a short-term bridge for hematopoietic recovery while also providing long-term immunologic and clinical benefits in HSCT recipients.

NLA101 is a product of Nohla Therapeutics Inc.

The company says more than 125 infusions of NLA101 have been administered since 2009. The therapy is under investigation in patients receiving intensive chemotherapy as well as in HSCT recipients.

Results from a pilot study of NLA101 in HSCT recipients were presented at the 2014 ASH Annual Meeting.

In this study, 15 patients with hematologic malignancies underwent myeloablative cord blood transplant, with or without NLA101.

Patients who received NLA101 had a significantly reduced median time to platelet and neutrophil recovery, compared to controls. At 5 years, disease-free survival was 86% in the NLA101 group and 67% in the control group.

The rate of grade 3-4 acute graft-versus-host disease was 0% in the NLA101 group and 29% in the control group. The rate of transplant-related mortality was 0% and 22%, respectively.

Phase 2 studies of NLA101 in chemotherapy and HSCT recipients are ongoing.

About orphan designation

Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.

Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.

The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.

Photo by Chad McNeeley

The European Commission (EC) has granted orphan designation to NLA101 as an adjunct to hematopoietic stem cell transplant (HSCT).

NLA101 is a universal, off-the-shelf, stem and progenitor cell therapy intended to provide a short-term bridge for hematopoietic recovery while also providing long-term immunologic and clinical benefits in HSCT recipients.

NLA101 is a product of Nohla Therapeutics Inc.

The company says more than 125 infusions of NLA101 have been administered since 2009. The therapy is under investigation in patients receiving intensive chemotherapy as well as in HSCT recipients.

Results from a pilot study of NLA101 in HSCT recipients were presented at the 2014 ASH Annual Meeting.

In this study, 15 patients with hematologic malignancies underwent myeloablative cord blood transplant, with or without NLA101.

Patients who received NLA101 had a significantly reduced median time to platelet and neutrophil recovery, compared to controls. At 5 years, disease-free survival was 86% in the NLA101 group and 67% in the control group.

The rate of grade 3-4 acute graft-versus-host disease was 0% in the NLA101 group and 29% in the control group. The rate of transplant-related mortality was 0% and 22%, respectively.

Phase 2 studies of NLA101 in chemotherapy and HSCT recipients are ongoing.

About orphan designation

Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.

Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.

The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.

Photo by Chad McNeeley

The European Commission (EC) has granted orphan designation to NLA101 as an adjunct to hematopoietic stem cell transplant (HSCT).

NLA101 is a universal, off-the-shelf, stem and progenitor cell therapy intended to provide a short-term bridge for hematopoietic recovery while also providing long-term immunologic and clinical benefits in HSCT recipients.

NLA101 is a product of Nohla Therapeutics Inc.

The company says more than 125 infusions of NLA101 have been administered since 2009. The therapy is under investigation in patients receiving intensive chemotherapy as well as in HSCT recipients.

Results from a pilot study of NLA101 in HSCT recipients were presented at the 2014 ASH Annual Meeting.

In this study, 15 patients with hematologic malignancies underwent myeloablative cord blood transplant, with or without NLA101.

Patients who received NLA101 had a significantly reduced median time to platelet and neutrophil recovery, compared to controls. At 5 years, disease-free survival was 86% in the NLA101 group and 67% in the control group.

The rate of grade 3-4 acute graft-versus-host disease was 0% in the NLA101 group and 29% in the control group. The rate of transplant-related mortality was 0% and 22%, respectively.

Phase 2 studies of NLA101 in chemotherapy and HSCT recipients are ongoing.

About orphan designation

Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.

Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.

The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.

Hypertension discovery in pediatric patients more than doubled for physicians using a clinical decision support (CDS) tool connected to the EHR, results of a study found.

Elyse O. Kharbanda, MD, MPH, a researcher at the HealthPartners Institute, Minneapolis, and her fellow investigators assert that using such a tool will help rectify the trend of underreported hypertension in adolescents, which remains a serious concern despite providers’ routinely taking blood pressure measurements during outpatient visits.

Vishnu Kumar/Thinkstock

[email protected]

Source: Kharbanda EO et al. Pediatrics. 2018. doi: 10.1542/peds.2017- 2954.

Hypertension discovery in pediatric patients more than doubled for physicians using a clinical decision support (CDS) tool connected to the EHR, results of a study found.

Elyse O. Kharbanda, MD, MPH, a researcher at the HealthPartners Institute, Minneapolis, and her fellow investigators assert that using such a tool will help rectify the trend of underreported hypertension in adolescents, which remains a serious concern despite providers’ routinely taking blood pressure measurements during outpatient visits.

Vishnu Kumar/Thinkstock

[email protected]

Source: Kharbanda EO et al. Pediatrics. 2018. doi: 10.1542/peds.2017- 2954.

Hypertension discovery in pediatric patients more than doubled for physicians using a clinical decision support (CDS) tool connected to the EHR, results of a study found.

Elyse O. Kharbanda, MD, MPH, a researcher at the HealthPartners Institute, Minneapolis, and her fellow investigators assert that using such a tool will help rectify the trend of underreported hypertension in adolescents, which remains a serious concern despite providers’ routinely taking blood pressure measurements during outpatient visits.

Vishnu Kumar/Thinkstock

[email protected]

Source: Kharbanda EO et al. Pediatrics. 2018. doi: 10.1542/peds.2017- 2954.

At follow-up 2 weeks later, pathology revealed a diagnosis of sebaceous hyperplasia (SH). On close examination of the face and nose, the FP noted other nonpigmented SHs and telangiectasias, which confirmed an overall diagnosis of rosacea.

The FP explained to the patient that SH is benign and that there are treatments for rosacea. In this case, the FP prescribed 1% metronidazole gel for the rosacea. At follow-up one month later, the shave biopsy had effectively removed the SH and the site had healed well. The metronidazole gel was also improving the appearance of the patient’s face. The patient indicated that he’d been avoiding the sun and drinking less alcohol, which was helping the rosacea and was, of course, good for his overall health.

Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Smith M. Sebaceous hyperplasia. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013: 931-934.

To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/

You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com

At follow-up 2 weeks later, pathology revealed a diagnosis of sebaceous hyperplasia (SH). On close examination of the face and nose, the FP noted other nonpigmented SHs and telangiectasias, which confirmed an overall diagnosis of rosacea.

The FP explained to the patient that SH is benign and that there are treatments for rosacea. In this case, the FP prescribed 1% metronidazole gel for the rosacea. At follow-up one month later, the shave biopsy had effectively removed the SH and the site had healed well. The metronidazole gel was also improving the appearance of the patient’s face. The patient indicated that he’d been avoiding the sun and drinking less alcohol, which was helping the rosacea and was, of course, good for his overall health.

Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Smith M. Sebaceous hyperplasia. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013: 931-934.

To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/

You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com

At follow-up 2 weeks later, pathology revealed a diagnosis of sebaceous hyperplasia (SH). On close examination of the face and nose, the FP noted other nonpigmented SHs and telangiectasias, which confirmed an overall diagnosis of rosacea.

The FP explained to the patient that SH is benign and that there are treatments for rosacea. In this case, the FP prescribed 1% metronidazole gel for the rosacea. At follow-up one month later, the shave biopsy had effectively removed the SH and the site had healed well. The metronidazole gel was also improving the appearance of the patient’s face. The patient indicated that he’d been avoiding the sun and drinking less alcohol, which was helping the rosacea and was, of course, good for his overall health.

Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Smith M. Sebaceous hyperplasia. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013: 931-934.

To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/

You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com

LOS ANGELES – For every 10 adult patients with type 2 diabetes, three are likely to have moderate to severe liver fibrosis, according to Kenneth Cusi, MD, FACP, FACE.

“The question is, How are we going to tackle this problem? My academic goal is that we incorporate screening for NASH [nonalcoholic steatohepatitis], or for fibrosis more specifically, in the same way we do for retinopathy or nephropathy [in diabetes], because we do have a way to treat it,” he said at the World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease.

Doug Brunk/Frontline Medical News

Dr. Cusi, chief of the division of endocrinology, diabetes, and metabolism at the University of Florida, Gainesville, predicted that obesity will become the No. 1 cause of liver transplantation. “It’s a real epidemic; you’re not seeing it because the inflexion of obesity happened just 2 decades ago,” he said. “Patients with diabetes face the greatest risk of fatty liver and of fibrosis. Untreated, it’s the equivalent of having macroalbuminuria. If you do nothing and they don’t die of cardiovascular disease, they’re going to have a good chance of getting fibrosis.”

As part of the large population-based Rotterdam study of individuals aged 45 years and older, researchers found that liver stiffness of 8 kPa or more by transient elastography was present in 5.6% of the study participants and was strongly associated with steatosis and diabetes (Hepatology. 2016;63:138-47). According to Dr. Cusi, individuals who have steatosis without diabetes face a 5%-10% risk of fibrosis, while those with steatosis and diabetes face a 15%-20% risk. “It’s well established in a number of studies that if you have fibrosis, you’re at high risk not only of cirrhosis, but also of hepatocellular carcinoma,” he said. “The key thing is not detecting fat, which is not really the target. The target is if there’s fibrosis or not.” Three ways to assess for fibrosis include MR elastography, transient elastography (which is the most commonly used), and fibrosis marker panels.

[email protected]
 

LOS ANGELES – For every 10 adult patients with type 2 diabetes, three are likely to have moderate to severe liver fibrosis, according to Kenneth Cusi, MD, FACP, FACE.

“The question is, How are we going to tackle this problem? My academic goal is that we incorporate screening for NASH [nonalcoholic steatohepatitis], or for fibrosis more specifically, in the same way we do for retinopathy or nephropathy [in diabetes], because we do have a way to treat it,” he said at the World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease.

Doug Brunk/Frontline Medical News

Dr. Cusi, chief of the division of endocrinology, diabetes, and metabolism at the University of Florida, Gainesville, predicted that obesity will become the No. 1 cause of liver transplantation. “It’s a real epidemic; you’re not seeing it because the inflexion of obesity happened just 2 decades ago,” he said. “Patients with diabetes face the greatest risk of fatty liver and of fibrosis. Untreated, it’s the equivalent of having macroalbuminuria. If you do nothing and they don’t die of cardiovascular disease, they’re going to have a good chance of getting fibrosis.”

As part of the large population-based Rotterdam study of individuals aged 45 years and older, researchers found that liver stiffness of 8 kPa or more by transient elastography was present in 5.6% of the study participants and was strongly associated with steatosis and diabetes (Hepatology. 2016;63:138-47). According to Dr. Cusi, individuals who have steatosis without diabetes face a 5%-10% risk of fibrosis, while those with steatosis and diabetes face a 15%-20% risk. “It’s well established in a number of studies that if you have fibrosis, you’re at high risk not only of cirrhosis, but also of hepatocellular carcinoma,” he said. “The key thing is not detecting fat, which is not really the target. The target is if there’s fibrosis or not.” Three ways to assess for fibrosis include MR elastography, transient elastography (which is the most commonly used), and fibrosis marker panels.

[email protected]
 

LOS ANGELES – For every 10 adult patients with type 2 diabetes, three are likely to have moderate to severe liver fibrosis, according to Kenneth Cusi, MD, FACP, FACE.

“The question is, How are we going to tackle this problem? My academic goal is that we incorporate screening for NASH [nonalcoholic steatohepatitis], or for fibrosis more specifically, in the same way we do for retinopathy or nephropathy [in diabetes], because we do have a way to treat it,” he said at the World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease.

Doug Brunk/Frontline Medical News

Dr. Cusi, chief of the division of endocrinology, diabetes, and metabolism at the University of Florida, Gainesville, predicted that obesity will become the No. 1 cause of liver transplantation. “It’s a real epidemic; you’re not seeing it because the inflexion of obesity happened just 2 decades ago,” he said. “Patients with diabetes face the greatest risk of fatty liver and of fibrosis. Untreated, it’s the equivalent of having macroalbuminuria. If you do nothing and they don’t die of cardiovascular disease, they’re going to have a good chance of getting fibrosis.”

As part of the large population-based Rotterdam study of individuals aged 45 years and older, researchers found that liver stiffness of 8 kPa or more by transient elastography was present in 5.6% of the study participants and was strongly associated with steatosis and diabetes (Hepatology. 2016;63:138-47). According to Dr. Cusi, individuals who have steatosis without diabetes face a 5%-10% risk of fibrosis, while those with steatosis and diabetes face a 15%-20% risk. “It’s well established in a number of studies that if you have fibrosis, you’re at high risk not only of cirrhosis, but also of hepatocellular carcinoma,” he said. “The key thing is not detecting fat, which is not really the target. The target is if there’s fibrosis or not.” Three ways to assess for fibrosis include MR elastography, transient elastography (which is the most commonly used), and fibrosis marker panels.

[email protected]