A novel risk-based approach could be an effective way to triage women for mammography during times of limited capacity, such as the situation during the COVID-19 pandemic, according to new findings.

Researchers evaluated almost 2 million mammograms that had been performed at more than 90 radiology centers and found that 12% of mammograms with “high” and “very high” cancer risk rates accounted for 55% of detected cancers.

In contrast, 44% of mammograms with very low cancer risk rates accounted for 13% of detected cancers. The study was published online March 25, 2021, in JAMA Network Open.

Cancer screening programs dramatically slowed or even came to a screeching halt during 2020, when restrictions and lockdowns were in place. The American Cancer Society even recommended that “no one should go to a health care facility for routine cancer screening,” as part of COVID-19 precautions.

However, concern was voiced that the pause in screening would allow patients with asymptomatic cancers or precursor lesions to develop into a more serious disease state.

The authors pointed out that several professional associations had posted guidance for scheduling individuals for breast imaging services during the COVID-19 pandemic, but these recommendations were based on expert opinion. The investigators’ goal was to help imaging facilities optimize the number of breast cancers that could be detected during periods of reduced capacity using clinical indication and individual characteristics.

The result was a risk-based strategy for triaging mammograms during periods of decreased capacity, which lead author Diana L. Miglioretti, PhD, explained was feasible to implement. Dr. Miglioretti is division chief of biostatistics in the department of public health sciences at University of California, Davis.

“Our risk model used information that is commonly collected by radiology facilities,” she said in an interview. “Vendors of electronic medical records could create tools that pull the information from the medical record, or could create fields in the scheduling system to efficiently collect this information when the mammogram is scheduled.”

Dr. Miglioretti emphasized that, once the information is collected in a standardized manner, “it would be straightforward to use a computer program to apply our algorithm to rank women based on their likelihood of having a breast cancer detected.”

“I think it is worth the investment to create these electronic tools now, given the potential for future shutdowns or periods of reduced capacity due to a variety of reasons, such as natural disasters and cyberattacks – or another pandemic,” she said.

Some facilities are still working through backlogs of mammograms that need to be rescheduled, which would be another way that this algorithm could be used. “They could use this approach to determine who should be scheduled first by using data available in the electronic medical record,” she added.
 

Five risk groups

Dr. Miglioretti and colleagues conducted a cohort study using data that was prospectively collected from mammography examinations performed from 2014 to 2019 at 92 radiology facilities in the Breast Cancer Surveillance Consortium. The cohort included 898,415 individuals who contributed to 1.8 million mammograms.

Information that included clinical indication for screening, breast symptoms, personal history of breast cancer, age, time since last mammogram/screening interval, family history of breast cancer, breast density, and history of high-risk breast lesion was collected from self-administered questionnaires at the time of mammography or extracted from electronic health records.

Following analysis, the data was categorized into five risk groups: very high (>50), high (22-50), moderate (10-22), low (5-10), and very low (<5) cancer detection rate per 1,000 mammograms. These thresholds were chosen based on the observed cancer detection rates and clinical expertise.

Of the group, about 1.7 million mammograms were from women without a personal history of breast cancer and 156,104 mammograms were from women with a breast cancer history. Most of the cohort were aged 50-69 years at the time of imaging, and 67.9% were White (11.2% Black, 11.3% Asian or Pacific Islander, 7% Hispanic, and 2.2% were another race/ethnicity or mixed race/ethnicity).

Their results showed that 12% of mammograms with very high (89.6-122.3 cancers detected per 1,000 mammograms) or high (36.1-47.5 cancers detected per 1,000 mammograms) cancer detection rates accounted for 55% of all detected cancers. These included mammograms that were done to evaluate an abnormal test or breast lump in individuals of all ages regardless of breast cancer history.

On the opposite end, 44.2% of mammograms with very low cancer detection rates accounted for 13.1% of detected cancers and that included annual screening tests in women aged 50-69 years (3.8 cancers detected per 1,000 mammograms) and all screening mammograms in individuals younger than 50 years regardless of screening interval (2.8 cancers detected per 1000 mammograms).
 

Treat with caution

In an accompanying editorial, Sarah M. Friedewald, MD, and Dipti Gupta, MD, both from Northwestern University, Chicago, pointed out that, while the authors examined a large dataset to identify a subgroup of patients who would most likely benefit from breast imaging in a setting where capacity is limited, “these data should be used with caution as the only barometer for whether a patient merits cancer screening during a period of rationing.”

They noted that, in the context of an acute crisis, when patient volume needs to be reduced very quickly, it is often impractical for clinicians to sift through patient records in order to capture the information necessary for triage. In addition, asking nonclinical schedulers to accurately pull data at this level, at the time when the patient calls to make an appointment, is unrealistic.

In the context of the pandemic, the editorialists wrote that, while this model uses risk for breast cancer to prioritize those to be seen in the clinic, the risk for complications from COVID-19 may also be an important factor to consider. For example, an older patient may be at a higher risk for breast cancer but may also face a higher risk for COVID-related complications. Conversely, a younger woman at a lower risk for serious COVID-related disease but who has breast cancer detected early will gain more life-years than an older patient.

There are also no algorithms to account for each patient’s perceived risk for breast cancer or COVID-19, and “the downstream effect of delaying cancer diagnosis may similarly lead to unintended consequences but may take longer to become apparent,” they wrote. “Focusing efforts on the operations of accommodating as many patients as possible, such as extending clinic hours, would be preferable.”

Finally, Dr. Friedewald and Dr. Gupta concluded that “the practicality of this process during the COVID-19 pandemic and extrapolation to other emergent settings are less obvious.”

The study was supported through a Patient-centered Outcomes Research Institute program award. Dr. Miglioretti reported receiving royalties from Elsevier outside the submitted work. Several coauthors report relationships with industry. Dr. Friedewald reported receiving grants from Hologic Research during the conduct of the study. Dr. Gupta disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

A novel risk-based approach could be an effective way to triage women for mammography during times of limited capacity, such as the situation during the COVID-19 pandemic, according to new findings.

Researchers evaluated almost 2 million mammograms that had been performed at more than 90 radiology centers and found that 12% of mammograms with “high” and “very high” cancer risk rates accounted for 55% of detected cancers.

In contrast, 44% of mammograms with very low cancer risk rates accounted for 13% of detected cancers. The study was published online March 25, 2021, in JAMA Network Open.

Cancer screening programs dramatically slowed or even came to a screeching halt during 2020, when restrictions and lockdowns were in place. The American Cancer Society even recommended that “no one should go to a health care facility for routine cancer screening,” as part of COVID-19 precautions.

However, concern was voiced that the pause in screening would allow patients with asymptomatic cancers or precursor lesions to develop into a more serious disease state.

The authors pointed out that several professional associations had posted guidance for scheduling individuals for breast imaging services during the COVID-19 pandemic, but these recommendations were based on expert opinion. The investigators’ goal was to help imaging facilities optimize the number of breast cancers that could be detected during periods of reduced capacity using clinical indication and individual characteristics.

The result was a risk-based strategy for triaging mammograms during periods of decreased capacity, which lead author Diana L. Miglioretti, PhD, explained was feasible to implement. Dr. Miglioretti is division chief of biostatistics in the department of public health sciences at University of California, Davis.

“Our risk model used information that is commonly collected by radiology facilities,” she said in an interview. “Vendors of electronic medical records could create tools that pull the information from the medical record, or could create fields in the scheduling system to efficiently collect this information when the mammogram is scheduled.”

Dr. Miglioretti emphasized that, once the information is collected in a standardized manner, “it would be straightforward to use a computer program to apply our algorithm to rank women based on their likelihood of having a breast cancer detected.”

“I think it is worth the investment to create these electronic tools now, given the potential for future shutdowns or periods of reduced capacity due to a variety of reasons, such as natural disasters and cyberattacks – or another pandemic,” she said.

Some facilities are still working through backlogs of mammograms that need to be rescheduled, which would be another way that this algorithm could be used. “They could use this approach to determine who should be scheduled first by using data available in the electronic medical record,” she added.
 

Five risk groups

Dr. Miglioretti and colleagues conducted a cohort study using data that was prospectively collected from mammography examinations performed from 2014 to 2019 at 92 radiology facilities in the Breast Cancer Surveillance Consortium. The cohort included 898,415 individuals who contributed to 1.8 million mammograms.

Information that included clinical indication for screening, breast symptoms, personal history of breast cancer, age, time since last mammogram/screening interval, family history of breast cancer, breast density, and history of high-risk breast lesion was collected from self-administered questionnaires at the time of mammography or extracted from electronic health records.

Following analysis, the data was categorized into five risk groups: very high (>50), high (22-50), moderate (10-22), low (5-10), and very low (<5) cancer detection rate per 1,000 mammograms. These thresholds were chosen based on the observed cancer detection rates and clinical expertise.

Of the group, about 1.7 million mammograms were from women without a personal history of breast cancer and 156,104 mammograms were from women with a breast cancer history. Most of the cohort were aged 50-69 years at the time of imaging, and 67.9% were White (11.2% Black, 11.3% Asian or Pacific Islander, 7% Hispanic, and 2.2% were another race/ethnicity or mixed race/ethnicity).

Their results showed that 12% of mammograms with very high (89.6-122.3 cancers detected per 1,000 mammograms) or high (36.1-47.5 cancers detected per 1,000 mammograms) cancer detection rates accounted for 55% of all detected cancers. These included mammograms that were done to evaluate an abnormal test or breast lump in individuals of all ages regardless of breast cancer history.

On the opposite end, 44.2% of mammograms with very low cancer detection rates accounted for 13.1% of detected cancers and that included annual screening tests in women aged 50-69 years (3.8 cancers detected per 1,000 mammograms) and all screening mammograms in individuals younger than 50 years regardless of screening interval (2.8 cancers detected per 1000 mammograms).
 

Treat with caution

In an accompanying editorial, Sarah M. Friedewald, MD, and Dipti Gupta, MD, both from Northwestern University, Chicago, pointed out that, while the authors examined a large dataset to identify a subgroup of patients who would most likely benefit from breast imaging in a setting where capacity is limited, “these data should be used with caution as the only barometer for whether a patient merits cancer screening during a period of rationing.”

They noted that, in the context of an acute crisis, when patient volume needs to be reduced very quickly, it is often impractical for clinicians to sift through patient records in order to capture the information necessary for triage. In addition, asking nonclinical schedulers to accurately pull data at this level, at the time when the patient calls to make an appointment, is unrealistic.

In the context of the pandemic, the editorialists wrote that, while this model uses risk for breast cancer to prioritize those to be seen in the clinic, the risk for complications from COVID-19 may also be an important factor to consider. For example, an older patient may be at a higher risk for breast cancer but may also face a higher risk for COVID-related complications. Conversely, a younger woman at a lower risk for serious COVID-related disease but who has breast cancer detected early will gain more life-years than an older patient.

There are also no algorithms to account for each patient’s perceived risk for breast cancer or COVID-19, and “the downstream effect of delaying cancer diagnosis may similarly lead to unintended consequences but may take longer to become apparent,” they wrote. “Focusing efforts on the operations of accommodating as many patients as possible, such as extending clinic hours, would be preferable.”

Finally, Dr. Friedewald and Dr. Gupta concluded that “the practicality of this process during the COVID-19 pandemic and extrapolation to other emergent settings are less obvious.”

The study was supported through a Patient-centered Outcomes Research Institute program award. Dr. Miglioretti reported receiving royalties from Elsevier outside the submitted work. Several coauthors report relationships with industry. Dr. Friedewald reported receiving grants from Hologic Research during the conduct of the study. Dr. Gupta disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

A novel risk-based approach could be an effective way to triage women for mammography during times of limited capacity, such as the situation during the COVID-19 pandemic, according to new findings.

Researchers evaluated almost 2 million mammograms that had been performed at more than 90 radiology centers and found that 12% of mammograms with “high” and “very high” cancer risk rates accounted for 55% of detected cancers.

In contrast, 44% of mammograms with very low cancer risk rates accounted for 13% of detected cancers. The study was published online March 25, 2021, in JAMA Network Open.

Cancer screening programs dramatically slowed or even came to a screeching halt during 2020, when restrictions and lockdowns were in place. The American Cancer Society even recommended that “no one should go to a health care facility for routine cancer screening,” as part of COVID-19 precautions.

However, concern was voiced that the pause in screening would allow patients with asymptomatic cancers or precursor lesions to develop into a more serious disease state.

The authors pointed out that several professional associations had posted guidance for scheduling individuals for breast imaging services during the COVID-19 pandemic, but these recommendations were based on expert opinion. The investigators’ goal was to help imaging facilities optimize the number of breast cancers that could be detected during periods of reduced capacity using clinical indication and individual characteristics.

The result was a risk-based strategy for triaging mammograms during periods of decreased capacity, which lead author Diana L. Miglioretti, PhD, explained was feasible to implement. Dr. Miglioretti is division chief of biostatistics in the department of public health sciences at University of California, Davis.

“Our risk model used information that is commonly collected by radiology facilities,” she said in an interview. “Vendors of electronic medical records could create tools that pull the information from the medical record, or could create fields in the scheduling system to efficiently collect this information when the mammogram is scheduled.”

Dr. Miglioretti emphasized that, once the information is collected in a standardized manner, “it would be straightforward to use a computer program to apply our algorithm to rank women based on their likelihood of having a breast cancer detected.”

“I think it is worth the investment to create these electronic tools now, given the potential for future shutdowns or periods of reduced capacity due to a variety of reasons, such as natural disasters and cyberattacks – or another pandemic,” she said.

Some facilities are still working through backlogs of mammograms that need to be rescheduled, which would be another way that this algorithm could be used. “They could use this approach to determine who should be scheduled first by using data available in the electronic medical record,” she added.
 

Five risk groups

Dr. Miglioretti and colleagues conducted a cohort study using data that was prospectively collected from mammography examinations performed from 2014 to 2019 at 92 radiology facilities in the Breast Cancer Surveillance Consortium. The cohort included 898,415 individuals who contributed to 1.8 million mammograms.

Information that included clinical indication for screening, breast symptoms, personal history of breast cancer, age, time since last mammogram/screening interval, family history of breast cancer, breast density, and history of high-risk breast lesion was collected from self-administered questionnaires at the time of mammography or extracted from electronic health records.

Following analysis, the data was categorized into five risk groups: very high (>50), high (22-50), moderate (10-22), low (5-10), and very low (<5) cancer detection rate per 1,000 mammograms. These thresholds were chosen based on the observed cancer detection rates and clinical expertise.

Of the group, about 1.7 million mammograms were from women without a personal history of breast cancer and 156,104 mammograms were from women with a breast cancer history. Most of the cohort were aged 50-69 years at the time of imaging, and 67.9% were White (11.2% Black, 11.3% Asian or Pacific Islander, 7% Hispanic, and 2.2% were another race/ethnicity or mixed race/ethnicity).

Their results showed that 12% of mammograms with very high (89.6-122.3 cancers detected per 1,000 mammograms) or high (36.1-47.5 cancers detected per 1,000 mammograms) cancer detection rates accounted for 55% of all detected cancers. These included mammograms that were done to evaluate an abnormal test or breast lump in individuals of all ages regardless of breast cancer history.

On the opposite end, 44.2% of mammograms with very low cancer detection rates accounted for 13.1% of detected cancers and that included annual screening tests in women aged 50-69 years (3.8 cancers detected per 1,000 mammograms) and all screening mammograms in individuals younger than 50 years regardless of screening interval (2.8 cancers detected per 1000 mammograms).
 

Treat with caution

In an accompanying editorial, Sarah M. Friedewald, MD, and Dipti Gupta, MD, both from Northwestern University, Chicago, pointed out that, while the authors examined a large dataset to identify a subgroup of patients who would most likely benefit from breast imaging in a setting where capacity is limited, “these data should be used with caution as the only barometer for whether a patient merits cancer screening during a period of rationing.”

They noted that, in the context of an acute crisis, when patient volume needs to be reduced very quickly, it is often impractical for clinicians to sift through patient records in order to capture the information necessary for triage. In addition, asking nonclinical schedulers to accurately pull data at this level, at the time when the patient calls to make an appointment, is unrealistic.

In the context of the pandemic, the editorialists wrote that, while this model uses risk for breast cancer to prioritize those to be seen in the clinic, the risk for complications from COVID-19 may also be an important factor to consider. For example, an older patient may be at a higher risk for breast cancer but may also face a higher risk for COVID-related complications. Conversely, a younger woman at a lower risk for serious COVID-related disease but who has breast cancer detected early will gain more life-years than an older patient.

There are also no algorithms to account for each patient’s perceived risk for breast cancer or COVID-19, and “the downstream effect of delaying cancer diagnosis may similarly lead to unintended consequences but may take longer to become apparent,” they wrote. “Focusing efforts on the operations of accommodating as many patients as possible, such as extending clinic hours, would be preferable.”

Finally, Dr. Friedewald and Dr. Gupta concluded that “the practicality of this process during the COVID-19 pandemic and extrapolation to other emergent settings are less obvious.”

The study was supported through a Patient-centered Outcomes Research Institute program award. Dr. Miglioretti reported receiving royalties from Elsevier outside the submitted work. Several coauthors report relationships with industry. Dr. Friedewald reported receiving grants from Hologic Research during the conduct of the study. Dr. Gupta disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

The OS was similar between the two arms, although the analysis was underpowered.  Nausea, diarrhea, and vomiting were the most frequently occurring treatment-emergent adverse events for oral azacitidine vs placebo (76% vs 23%, 68% vs 23%, 63% vs 9%). While oral azacitidine is not approved for MDS and trial was stopped early, it can provide meaningful reduction in RBC transfusions in low grade MDS, and further evaluation is needed in MDS.

Paroxysmal nocturnal hemoglobinuria (PNH) clones have been observed in bone marrow failure syndromes including MDS and aplastic anemia (AA). Fattizzo et al analyzed PNH clone size with clinical outcomes in MDS and AA in a cohort of 3085 patients tested for PNH at King’s College Hospital in London. 20.3% of MDS patients had presence of PNH clone; PNH cases occurred more in hypoplastic MDS, and lower risk IPSS MDS patients. The presence of PNH clone was a predictor of response to immunosuppressive therapy and to allogeneic stem cell transplant (84% vs 45%, p=0.01, 71% vs 57%, p=0.09), but not to azacitidine. MDS patients with PNH clone had lower rate of progression by IPSS and AML transformation, and higher OS accounting for MDS with excess blasts. Each 10% increase in clone size resulted in a 1% decrease in cumulative incidence of death. PNH testing is often done especially in cases with hypoplastic MDS; the underlying mechanism and role for monitoring PNH clones in MDS needs further investigation.

Outcomes in MDS patients after azacitidine therapy are generally poor. Clavio et al analyzed outcomes of 402 MDS patients post-azacitidine in the Italian MDS Registry. At azacitidine discontinuation, 20% of patients still derived response to azacitidine, 35% had primary resistance, and 44% had adaptive resistance, which was defined as loss of response or progression after achievement of a response. As expected, OS was longer for patients who stopped treatment due to planned allogeneic stem cell transplant (median OS not reached), compared to patients with primary resistance (median OS 4 months), or adaptive resistance (median OS 5 months), or patients intolerant or noncompliant to azacitidine (median OS 4 months, p=0.004). The North American MDS Consortium scoring system was evaluated in the study cohort; patients with high risk had worse OS than low risk (3 and 7 months, p<0.001). This retrospective analysis confirms the poor outlook of MDS patients after treatment of azacitidine, and effective therapy in this patient population is an unmet need.

The OS was similar between the two arms, although the analysis was underpowered.  Nausea, diarrhea, and vomiting were the most frequently occurring treatment-emergent adverse events for oral azacitidine vs placebo (76% vs 23%, 68% vs 23%, 63% vs 9%). While oral azacitidine is not approved for MDS and trial was stopped early, it can provide meaningful reduction in RBC transfusions in low grade MDS, and further evaluation is needed in MDS.

Paroxysmal nocturnal hemoglobinuria (PNH) clones have been observed in bone marrow failure syndromes including MDS and aplastic anemia (AA). Fattizzo et al analyzed PNH clone size with clinical outcomes in MDS and AA in a cohort of 3085 patients tested for PNH at King’s College Hospital in London. 20.3% of MDS patients had presence of PNH clone; PNH cases occurred more in hypoplastic MDS, and lower risk IPSS MDS patients. The presence of PNH clone was a predictor of response to immunosuppressive therapy and to allogeneic stem cell transplant (84% vs 45%, p=0.01, 71% vs 57%, p=0.09), but not to azacitidine. MDS patients with PNH clone had lower rate of progression by IPSS and AML transformation, and higher OS accounting for MDS with excess blasts. Each 10% increase in clone size resulted in a 1% decrease in cumulative incidence of death. PNH testing is often done especially in cases with hypoplastic MDS; the underlying mechanism and role for monitoring PNH clones in MDS needs further investigation.

Outcomes in MDS patients after azacitidine therapy are generally poor. Clavio et al analyzed outcomes of 402 MDS patients post-azacitidine in the Italian MDS Registry. At azacitidine discontinuation, 20% of patients still derived response to azacitidine, 35% had primary resistance, and 44% had adaptive resistance, which was defined as loss of response or progression after achievement of a response. As expected, OS was longer for patients who stopped treatment due to planned allogeneic stem cell transplant (median OS not reached), compared to patients with primary resistance (median OS 4 months), or adaptive resistance (median OS 5 months), or patients intolerant or noncompliant to azacitidine (median OS 4 months, p=0.004). The North American MDS Consortium scoring system was evaluated in the study cohort; patients with high risk had worse OS than low risk (3 and 7 months, p<0.001). This retrospective analysis confirms the poor outlook of MDS patients after treatment of azacitidine, and effective therapy in this patient population is an unmet need.

The OS was similar between the two arms, although the analysis was underpowered.  Nausea, diarrhea, and vomiting were the most frequently occurring treatment-emergent adverse events for oral azacitidine vs placebo (76% vs 23%, 68% vs 23%, 63% vs 9%). While oral azacitidine is not approved for MDS and trial was stopped early, it can provide meaningful reduction in RBC transfusions in low grade MDS, and further evaluation is needed in MDS.

Paroxysmal nocturnal hemoglobinuria (PNH) clones have been observed in bone marrow failure syndromes including MDS and aplastic anemia (AA). Fattizzo et al analyzed PNH clone size with clinical outcomes in MDS and AA in a cohort of 3085 patients tested for PNH at King’s College Hospital in London. 20.3% of MDS patients had presence of PNH clone; PNH cases occurred more in hypoplastic MDS, and lower risk IPSS MDS patients. The presence of PNH clone was a predictor of response to immunosuppressive therapy and to allogeneic stem cell transplant (84% vs 45%, p=0.01, 71% vs 57%, p=0.09), but not to azacitidine. MDS patients with PNH clone had lower rate of progression by IPSS and AML transformation, and higher OS accounting for MDS with excess blasts. Each 10% increase in clone size resulted in a 1% decrease in cumulative incidence of death. PNH testing is often done especially in cases with hypoplastic MDS; the underlying mechanism and role for monitoring PNH clones in MDS needs further investigation.

Outcomes in MDS patients after azacitidine therapy are generally poor. Clavio et al analyzed outcomes of 402 MDS patients post-azacitidine in the Italian MDS Registry. At azacitidine discontinuation, 20% of patients still derived response to azacitidine, 35% had primary resistance, and 44% had adaptive resistance, which was defined as loss of response or progression after achievement of a response. As expected, OS was longer for patients who stopped treatment due to planned allogeneic stem cell transplant (median OS not reached), compared to patients with primary resistance (median OS 4 months), or adaptive resistance (median OS 5 months), or patients intolerant or noncompliant to azacitidine (median OS 4 months, p=0.004). The North American MDS Consortium scoring system was evaluated in the study cohort; patients with high risk had worse OS than low risk (3 and 7 months, p<0.001). This retrospective analysis confirms the poor outlook of MDS patients after treatment of azacitidine, and effective therapy in this patient population is an unmet need.

Key clinical point: Adding stanozolol to decitabine after effective decitabine treatment may improve progression-free survival (PFS) and reduce the severity of neutropenia in patients with high-risk myelodysplastic syndrome (MDS).

Major finding: PFS was significantly longer in the stanozolol group vs. stanozolol+decitabine group (15.0 vs. 9.0 months; hazard ratio [HR], 0.35; P = .0005). The proportion of patients with grade 3-4 neutropenia was lower in stanozolol group (76.2% vs. 95.1%; P = .039).

Study details: Findings are from a retrospective analysis of 62 patients with newly diagnosed high-risk MDS who achieved at least partial remission after 4 cycles of decitabine. For maintenance treatment, 21 patients received stanozolol and decitabine and 41 patients received decitabine alone.

Disclosures: This study was partly supported by the National Natural Science Foundation of China, the Natural Science Foundation of Tianjin China, Key Technology Research and Development Program of Tianjin China, Beijing Natural Science Foundation, and Chinese Academy of Medical Sciences innovation for medical sciences. The authors declared no conflicts of interest.

Source: Liu Y et al. Int J Hematol. 2021 Mar 1. doi: 10.1007/s12185-021-03115-9.

Key clinical point: Adding stanozolol to decitabine after effective decitabine treatment may improve progression-free survival (PFS) and reduce the severity of neutropenia in patients with high-risk myelodysplastic syndrome (MDS).

Major finding: PFS was significantly longer in the stanozolol group vs. stanozolol+decitabine group (15.0 vs. 9.0 months; hazard ratio [HR], 0.35; P = .0005). The proportion of patients with grade 3-4 neutropenia was lower in stanozolol group (76.2% vs. 95.1%; P = .039).

Study details: Findings are from a retrospective analysis of 62 patients with newly diagnosed high-risk MDS who achieved at least partial remission after 4 cycles of decitabine. For maintenance treatment, 21 patients received stanozolol and decitabine and 41 patients received decitabine alone.

Disclosures: This study was partly supported by the National Natural Science Foundation of China, the Natural Science Foundation of Tianjin China, Key Technology Research and Development Program of Tianjin China, Beijing Natural Science Foundation, and Chinese Academy of Medical Sciences innovation for medical sciences. The authors declared no conflicts of interest.

Source: Liu Y et al. Int J Hematol. 2021 Mar 1. doi: 10.1007/s12185-021-03115-9.

Key clinical point: Adding stanozolol to decitabine after effective decitabine treatment may improve progression-free survival (PFS) and reduce the severity of neutropenia in patients with high-risk myelodysplastic syndrome (MDS).

Major finding: PFS was significantly longer in the stanozolol group vs. stanozolol+decitabine group (15.0 vs. 9.0 months; hazard ratio [HR], 0.35; P = .0005). The proportion of patients with grade 3-4 neutropenia was lower in stanozolol group (76.2% vs. 95.1%; P = .039).

Study details: Findings are from a retrospective analysis of 62 patients with newly diagnosed high-risk MDS who achieved at least partial remission after 4 cycles of decitabine. For maintenance treatment, 21 patients received stanozolol and decitabine and 41 patients received decitabine alone.

Disclosures: This study was partly supported by the National Natural Science Foundation of China, the Natural Science Foundation of Tianjin China, Key Technology Research and Development Program of Tianjin China, Beijing Natural Science Foundation, and Chinese Academy of Medical Sciences innovation for medical sciences. The authors declared no conflicts of interest.

Source: Liu Y et al. Int J Hematol. 2021 Mar 1. doi: 10.1007/s12185-021-03115-9.

The Food and Drug Administration has approved dasiglucagon (Zegalogue 0.6 mg/0.6 mL, Zealand Pharma) autoinjector and prefilled syringe for the treatment of severe hypoglycemia in people with diabetes aged 6 years and older.

Olivier Le Moal/Getty Images

The product has a shelf-life of 36 months at refrigerated temperatures and is stable for up to 12 months at room temperature.

“This approval will help enable appropriate children and adults with diabetes to be able to address sudden and severe hypoglycemia, which can quickly progress from a mild event to an emergency,” Jeremy Pettus, MD, assistant professor of medicine at the University of California, San Diego, said in a company statement.

The approval marks the latest step in the development of newer glucagon formulations that are easier to use in hypoglycemic emergencies than the traditional formulation that requires several steps for reconstitution.

The first intranasal glucagon (Baqsimi, Eli Lilly) was approved in the United States in July 2019 for people with diabetes age 4 years and older.

In September 2019, the FDA approved another prefilled glucagon rescue pen (Gvoke HypoPen, Xeris Pharmaceuticals) for the treatment of severe hypoglycemia in adult and pediatric patients age 2 years and older with diabetes.

Dasiglucagon is currently in phase 3 trials as a subcutaneous infusion for treating congenital hyperinsulinemia, and in phase 2 trials as part of a bihormonal artificial pancreas pump system.

The FDA approval was based on results from three randomized, double-blind, placebo-controlled, phase 3 studies of dasiglucagon in children age 6-17 years and adults with type 1 diabetes.

The primary endpoint was time to achieving an increase in blood glucose of 20 mg/dL or greater from time of administration without additional intervention within 45 minutes. That endpoint was achieved in all three studies, with a median time to blood glucose recovery of 10 minutes overall, with 99% of adults recovering within 15 minutes.

The most common adverse events reported in 2% or more of study participants were nausea, vomiting, headache, and injection-site pain in both children and adults. Diarrhea was also reported in adults.  

Full launch is expected in late June 2021.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved dasiglucagon (Zegalogue 0.6 mg/0.6 mL, Zealand Pharma) autoinjector and prefilled syringe for the treatment of severe hypoglycemia in people with diabetes aged 6 years and older.

Olivier Le Moal/Getty Images

The product has a shelf-life of 36 months at refrigerated temperatures and is stable for up to 12 months at room temperature.

“This approval will help enable appropriate children and adults with diabetes to be able to address sudden and severe hypoglycemia, which can quickly progress from a mild event to an emergency,” Jeremy Pettus, MD, assistant professor of medicine at the University of California, San Diego, said in a company statement.

The approval marks the latest step in the development of newer glucagon formulations that are easier to use in hypoglycemic emergencies than the traditional formulation that requires several steps for reconstitution.

The first intranasal glucagon (Baqsimi, Eli Lilly) was approved in the United States in July 2019 for people with diabetes age 4 years and older.

In September 2019, the FDA approved another prefilled glucagon rescue pen (Gvoke HypoPen, Xeris Pharmaceuticals) for the treatment of severe hypoglycemia in adult and pediatric patients age 2 years and older with diabetes.

Dasiglucagon is currently in phase 3 trials as a subcutaneous infusion for treating congenital hyperinsulinemia, and in phase 2 trials as part of a bihormonal artificial pancreas pump system.

The FDA approval was based on results from three randomized, double-blind, placebo-controlled, phase 3 studies of dasiglucagon in children age 6-17 years and adults with type 1 diabetes.

The primary endpoint was time to achieving an increase in blood glucose of 20 mg/dL or greater from time of administration without additional intervention within 45 minutes. That endpoint was achieved in all three studies, with a median time to blood glucose recovery of 10 minutes overall, with 99% of adults recovering within 15 minutes.

The most common adverse events reported in 2% or more of study participants were nausea, vomiting, headache, and injection-site pain in both children and adults. Diarrhea was also reported in adults.  

Full launch is expected in late June 2021.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved dasiglucagon (Zegalogue 0.6 mg/0.6 mL, Zealand Pharma) autoinjector and prefilled syringe for the treatment of severe hypoglycemia in people with diabetes aged 6 years and older.

Olivier Le Moal/Getty Images

The product has a shelf-life of 36 months at refrigerated temperatures and is stable for up to 12 months at room temperature.

“This approval will help enable appropriate children and adults with diabetes to be able to address sudden and severe hypoglycemia, which can quickly progress from a mild event to an emergency,” Jeremy Pettus, MD, assistant professor of medicine at the University of California, San Diego, said in a company statement.

The approval marks the latest step in the development of newer glucagon formulations that are easier to use in hypoglycemic emergencies than the traditional formulation that requires several steps for reconstitution.

The first intranasal glucagon (Baqsimi, Eli Lilly) was approved in the United States in July 2019 for people with diabetes age 4 years and older.

In September 2019, the FDA approved another prefilled glucagon rescue pen (Gvoke HypoPen, Xeris Pharmaceuticals) for the treatment of severe hypoglycemia in adult and pediatric patients age 2 years and older with diabetes.

Dasiglucagon is currently in phase 3 trials as a subcutaneous infusion for treating congenital hyperinsulinemia, and in phase 2 trials as part of a bihormonal artificial pancreas pump system.

The FDA approval was based on results from three randomized, double-blind, placebo-controlled, phase 3 studies of dasiglucagon in children age 6-17 years and adults with type 1 diabetes.

The primary endpoint was time to achieving an increase in blood glucose of 20 mg/dL or greater from time of administration without additional intervention within 45 minutes. That endpoint was achieved in all three studies, with a median time to blood glucose recovery of 10 minutes overall, with 99% of adults recovering within 15 minutes.

The most common adverse events reported in 2% or more of study participants were nausea, vomiting, headache, and injection-site pain in both children and adults. Diarrhea was also reported in adults.  

Full launch is expected in late June 2021.

A version of this article first appeared on Medscape.com.

Key clinical point: Over 13 years of observation reveals the presence of comorbidities, including a prior malignancy, in majority of patients with myelodysplastic syndrome (MDS). A combination of comorbidities was significantly associated with worse overall survival (OS).

Major finding: A comorbidity was reported in 67% of patients, of which 24.4% had a prior malignancy. Patients with Charlson Comorbidity Index score of 4 or higher vs. lower score had significantly impaired OS (P less than .01). Both therapy-related (hazard ratio [HR], 1.51) and secondary (HR, 1.58) vs. de novo MDS were associated with worse OS (P = .04).

Study details: Data come from an observational population-based study of 291 patients with newly diagnosed MDS.

Disclosures: No source of funding was declared. The lead author reported receiving financial support from Celgene for attending the MDS Foundation meeting in 2019. The remaining authors declared no competing financial interests.

Source: Rozema J et al. Blood Adv. 2021 Mar 3. doi: 10.1182/bloodadvances.2020003381.

Key clinical point: Over 13 years of observation reveals the presence of comorbidities, including a prior malignancy, in majority of patients with myelodysplastic syndrome (MDS). A combination of comorbidities was significantly associated with worse overall survival (OS).

Major finding: A comorbidity was reported in 67% of patients, of which 24.4% had a prior malignancy. Patients with Charlson Comorbidity Index score of 4 or higher vs. lower score had significantly impaired OS (P less than .01). Both therapy-related (hazard ratio [HR], 1.51) and secondary (HR, 1.58) vs. de novo MDS were associated with worse OS (P = .04).

Study details: Data come from an observational population-based study of 291 patients with newly diagnosed MDS.

Disclosures: No source of funding was declared. The lead author reported receiving financial support from Celgene for attending the MDS Foundation meeting in 2019. The remaining authors declared no competing financial interests.

Source: Rozema J et al. Blood Adv. 2021 Mar 3. doi: 10.1182/bloodadvances.2020003381.

Key clinical point: Over 13 years of observation reveals the presence of comorbidities, including a prior malignancy, in majority of patients with myelodysplastic syndrome (MDS). A combination of comorbidities was significantly associated with worse overall survival (OS).

Major finding: A comorbidity was reported in 67% of patients, of which 24.4% had a prior malignancy. Patients with Charlson Comorbidity Index score of 4 or higher vs. lower score had significantly impaired OS (P less than .01). Both therapy-related (hazard ratio [HR], 1.51) and secondary (HR, 1.58) vs. de novo MDS were associated with worse OS (P = .04).

Study details: Data come from an observational population-based study of 291 patients with newly diagnosed MDS.

Disclosures: No source of funding was declared. The lead author reported receiving financial support from Celgene for attending the MDS Foundation meeting in 2019. The remaining authors declared no competing financial interests.

Source: Rozema J et al. Blood Adv. 2021 Mar 3. doi: 10.1182/bloodadvances.2020003381.

Key clinical point: Evolutionary Action score (EAp53), a mutation-dependent, stable predictive biomarker, can help identify prognostic subsets within TP53-mutated myelodysplastic syndrome (MDS).

Major finding: High (more than 52) vs. low (52 or lesser) EAp53 score predicted worse overall survival (10 vs. 47.8 months; P = .01). Frequency of TP53 mutations (P = .027) and multi-allelic TP53 alterations (P = .0087) was higher in high-EAp53-MDS, whereas cytogenetic abnormalities (P = .019), complex karyotype (P = .0241), and monosomal karyotype (P = .0043) were lower in low-EAp53-MDS.

Study details: Findings are from a retrospective study of 270 patients with newly diagnosed MDS or oligoblastic acute myeloid leukemia (AML; less than 30% blasts) with 1 or more missense TP53 mutation.

Disclosures: This study was partly supported by institutional start-up funds, research grant awarded to the lead author, University of Texas MD Anderson Cancer Center Support grant, and generous philanthropic donations to the University of Texas MD Anderson MDS/AML Moon Shot Program. Some of the authors declared advisory roles with; consultancies with; research support, honoraria, personal fees, and/or equity ownership from various pharmaceutical companies.

Source: Kanagal-Shamanna R et al. Blood Cancer J. 2021 Mar 6. doi: 10.1038/s41408-021-00446-y.

Key clinical point: Evolutionary Action score (EAp53), a mutation-dependent, stable predictive biomarker, can help identify prognostic subsets within TP53-mutated myelodysplastic syndrome (MDS).

Major finding: High (more than 52) vs. low (52 or lesser) EAp53 score predicted worse overall survival (10 vs. 47.8 months; P = .01). Frequency of TP53 mutations (P = .027) and multi-allelic TP53 alterations (P = .0087) was higher in high-EAp53-MDS, whereas cytogenetic abnormalities (P = .019), complex karyotype (P = .0241), and monosomal karyotype (P = .0043) were lower in low-EAp53-MDS.

Study details: Findings are from a retrospective study of 270 patients with newly diagnosed MDS or oligoblastic acute myeloid leukemia (AML; less than 30% blasts) with 1 or more missense TP53 mutation.

Disclosures: This study was partly supported by institutional start-up funds, research grant awarded to the lead author, University of Texas MD Anderson Cancer Center Support grant, and generous philanthropic donations to the University of Texas MD Anderson MDS/AML Moon Shot Program. Some of the authors declared advisory roles with; consultancies with; research support, honoraria, personal fees, and/or equity ownership from various pharmaceutical companies.

Source: Kanagal-Shamanna R et al. Blood Cancer J. 2021 Mar 6. doi: 10.1038/s41408-021-00446-y.

Key clinical point: Evolutionary Action score (EAp53), a mutation-dependent, stable predictive biomarker, can help identify prognostic subsets within TP53-mutated myelodysplastic syndrome (MDS).

Major finding: High (more than 52) vs. low (52 or lesser) EAp53 score predicted worse overall survival (10 vs. 47.8 months; P = .01). Frequency of TP53 mutations (P = .027) and multi-allelic TP53 alterations (P = .0087) was higher in high-EAp53-MDS, whereas cytogenetic abnormalities (P = .019), complex karyotype (P = .0241), and monosomal karyotype (P = .0043) were lower in low-EAp53-MDS.

Study details: Findings are from a retrospective study of 270 patients with newly diagnosed MDS or oligoblastic acute myeloid leukemia (AML; less than 30% blasts) with 1 or more missense TP53 mutation.

Disclosures: This study was partly supported by institutional start-up funds, research grant awarded to the lead author, University of Texas MD Anderson Cancer Center Support grant, and generous philanthropic donations to the University of Texas MD Anderson MDS/AML Moon Shot Program. Some of the authors declared advisory roles with; consultancies with; research support, honoraria, personal fees, and/or equity ownership from various pharmaceutical companies.

Source: Kanagal-Shamanna R et al. Blood Cancer J. 2021 Mar 6. doi: 10.1038/s41408-021-00446-y.

Key clinical point: Single-nucleotide polymorphism array (SNP-A)-based whole genome sequencing capable of detecting uniparental disomy (UPD) offers more diagnostic and prognostic information than conventional metaphase cytogenetic analysis (MC) in patients with myelodysplastic syndrome (MDS).

Major finding: SNP-A had a higher positivity for significant chromosomal aberrations than MC (58.2% vs. 36.9%; P less than .05). SNP-A detected 78 chromosomal alterations including 38 UPDs that were undetected by MC in 40 patients. Additionally, patients with vs. without UPD had a worse prognosis (P = .01).

Study details: Data come from a comparative analysis of SNP-A and MC in 127 patients with MDS (n=110) and related diseases, including myelodysplastic/myeloproliferative neoplasm (n=6) and transformed acute myeloid leukemia (n=11).

Disclosures: This study received experimental research funding from the National Natural Science Foundation of China, the Sichuan Provincial Academic and Technical Leadership Support Funding Project, and Innovation Development Program of Chengdu. The authors declared no conflicts of interest.

Source: Ou Y et al. Int J Lab Hematol. 2021 Mar 2. doi: 10.1111/ijlh.13502.

Key clinical point: Single-nucleotide polymorphism array (SNP-A)-based whole genome sequencing capable of detecting uniparental disomy (UPD) offers more diagnostic and prognostic information than conventional metaphase cytogenetic analysis (MC) in patients with myelodysplastic syndrome (MDS).

Major finding: SNP-A had a higher positivity for significant chromosomal aberrations than MC (58.2% vs. 36.9%; P less than .05). SNP-A detected 78 chromosomal alterations including 38 UPDs that were undetected by MC in 40 patients. Additionally, patients with vs. without UPD had a worse prognosis (P = .01).

Study details: Data come from a comparative analysis of SNP-A and MC in 127 patients with MDS (n=110) and related diseases, including myelodysplastic/myeloproliferative neoplasm (n=6) and transformed acute myeloid leukemia (n=11).

Disclosures: This study received experimental research funding from the National Natural Science Foundation of China, the Sichuan Provincial Academic and Technical Leadership Support Funding Project, and Innovation Development Program of Chengdu. The authors declared no conflicts of interest.

Source: Ou Y et al. Int J Lab Hematol. 2021 Mar 2. doi: 10.1111/ijlh.13502.

Key clinical point: Single-nucleotide polymorphism array (SNP-A)-based whole genome sequencing capable of detecting uniparental disomy (UPD) offers more diagnostic and prognostic information than conventional metaphase cytogenetic analysis (MC) in patients with myelodysplastic syndrome (MDS).

Major finding: SNP-A had a higher positivity for significant chromosomal aberrations than MC (58.2% vs. 36.9%; P less than .05). SNP-A detected 78 chromosomal alterations including 38 UPDs that were undetected by MC in 40 patients. Additionally, patients with vs. without UPD had a worse prognosis (P = .01).

Study details: Data come from a comparative analysis of SNP-A and MC in 127 patients with MDS (n=110) and related diseases, including myelodysplastic/myeloproliferative neoplasm (n=6) and transformed acute myeloid leukemia (n=11).

Disclosures: This study received experimental research funding from the National Natural Science Foundation of China, the Sichuan Provincial Academic and Technical Leadership Support Funding Project, and Innovation Development Program of Chengdu. The authors declared no conflicts of interest.

Source: Ou Y et al. Int J Lab Hematol. 2021 Mar 2. doi: 10.1111/ijlh.13502.

Key clinical point: Real-world evidence suggests that a combination regimen of decitabine, idarubicin, and cytarabine (D-IA) was effective and well tolerated in patients with high-risk myelodysplastic syndrome (MDS) who cannot receive intensive chemotherapy.

Major finding: The overall response rate and complete remission after 2 cycles were 76.6% and 23.4%, respectively. The 2-year estimated overall survival and event-free survival rates were 31% and 18%, respectively. Most patients showed the occurrence of grade 3/4 neutropenia and thrombocytopenia.

Study details: This retrospective study evaluated 154 patients with acute myeloid leukemia or high-risk MDS ineligible for intensive chemotherapy who were prescribed D-IA regimen (decitabine 15-20 mg/m² for 1 to 3-5 days, followed by idarubicin 3 mg/m² for 5-7 days and cytarabine 30 mg/m² for 7-14 days).

Disclosures: The study was supported by the National Natural Science Foundation of China. The authors declared no potential conflicts of interest.

Source: Xu W et al. Leuk Lymphoma. 2021 Mar 7. doi: 10.1080/10428194.2021.1891230.

Key clinical point: Real-world evidence suggests that a combination regimen of decitabine, idarubicin, and cytarabine (D-IA) was effective and well tolerated in patients with high-risk myelodysplastic syndrome (MDS) who cannot receive intensive chemotherapy.

Major finding: The overall response rate and complete remission after 2 cycles were 76.6% and 23.4%, respectively. The 2-year estimated overall survival and event-free survival rates were 31% and 18%, respectively. Most patients showed the occurrence of grade 3/4 neutropenia and thrombocytopenia.

Study details: This retrospective study evaluated 154 patients with acute myeloid leukemia or high-risk MDS ineligible for intensive chemotherapy who were prescribed D-IA regimen (decitabine 15-20 mg/m² for 1 to 3-5 days, followed by idarubicin 3 mg/m² for 5-7 days and cytarabine 30 mg/m² for 7-14 days).

Disclosures: The study was supported by the National Natural Science Foundation of China. The authors declared no potential conflicts of interest.

Source: Xu W et al. Leuk Lymphoma. 2021 Mar 7. doi: 10.1080/10428194.2021.1891230.

Key clinical point: Real-world evidence suggests that a combination regimen of decitabine, idarubicin, and cytarabine (D-IA) was effective and well tolerated in patients with high-risk myelodysplastic syndrome (MDS) who cannot receive intensive chemotherapy.

Major finding: The overall response rate and complete remission after 2 cycles were 76.6% and 23.4%, respectively. The 2-year estimated overall survival and event-free survival rates were 31% and 18%, respectively. Most patients showed the occurrence of grade 3/4 neutropenia and thrombocytopenia.

Study details: This retrospective study evaluated 154 patients with acute myeloid leukemia or high-risk MDS ineligible for intensive chemotherapy who were prescribed D-IA regimen (decitabine 15-20 mg/m² for 1 to 3-5 days, followed by idarubicin 3 mg/m² for 5-7 days and cytarabine 30 mg/m² for 7-14 days).

Disclosures: The study was supported by the National Natural Science Foundation of China. The authors declared no potential conflicts of interest.

Source: Xu W et al. Leuk Lymphoma. 2021 Mar 7. doi: 10.1080/10428194.2021.1891230.

Key clinical point: Paroxysmal nocturnal hemoglobinuria (PNH) clones are highly prevalent in myelodysplastic syndrome (MDS). PNH positivity predicted higher response to immunosuppressive therapy (IST) and stem cell transplants along with a favorable impact on overall survival (OS).

Major finding: PNH clones were present in 20.3% of MDS cases. PNH-positive vs. PNH-negative patients showed significantly higher response to IST (84% vs. 44.7%; P = .01) and stem cell transplant (71% vs. 56.6%; P = .09). PNH positivity had a favorable impact on disease progression (P less than .01) and overall survival (P less than .0001).

Study details: Data come from a large single-center study involving 3,085 patients with suspected underlying myeloid disorders, cytopenia, or unexplained thrombosis who underwent a first-time PNH test. The cohort had 869 cases of MDS.

Disclosures: The authors did not declare any source of funding. The authors declared no competing interests.

Source: Fattizzo B et al. Leukemia. 2021 Mar 4. doi: 10.1038/s41375-021-01190-9.

Key clinical point: Paroxysmal nocturnal hemoglobinuria (PNH) clones are highly prevalent in myelodysplastic syndrome (MDS). PNH positivity predicted higher response to immunosuppressive therapy (IST) and stem cell transplants along with a favorable impact on overall survival (OS).

Major finding: PNH clones were present in 20.3% of MDS cases. PNH-positive vs. PNH-negative patients showed significantly higher response to IST (84% vs. 44.7%; P = .01) and stem cell transplant (71% vs. 56.6%; P = .09). PNH positivity had a favorable impact on disease progression (P less than .01) and overall survival (P less than .0001).

Study details: Data come from a large single-center study involving 3,085 patients with suspected underlying myeloid disorders, cytopenia, or unexplained thrombosis who underwent a first-time PNH test. The cohort had 869 cases of MDS.

Disclosures: The authors did not declare any source of funding. The authors declared no competing interests.

Source: Fattizzo B et al. Leukemia. 2021 Mar 4. doi: 10.1038/s41375-021-01190-9.

Key clinical point: Paroxysmal nocturnal hemoglobinuria (PNH) clones are highly prevalent in myelodysplastic syndrome (MDS). PNH positivity predicted higher response to immunosuppressive therapy (IST) and stem cell transplants along with a favorable impact on overall survival (OS).

Major finding: PNH clones were present in 20.3% of MDS cases. PNH-positive vs. PNH-negative patients showed significantly higher response to IST (84% vs. 44.7%; P = .01) and stem cell transplant (71% vs. 56.6%; P = .09). PNH positivity had a favorable impact on disease progression (P less than .01) and overall survival (P less than .0001).

Study details: Data come from a large single-center study involving 3,085 patients with suspected underlying myeloid disorders, cytopenia, or unexplained thrombosis who underwent a first-time PNH test. The cohort had 869 cases of MDS.

Disclosures: The authors did not declare any source of funding. The authors declared no competing interests.

Source: Fattizzo B et al. Leukemia. 2021 Mar 4. doi: 10.1038/s41375-021-01190-9.

Key clinical point: Findings confirm dismal outcomes after azacitidine (AZA) discontinuation in patients with myelodysplastic syndrome (MDS); however, outcomes were best in those who discontinued AZA while being in response to undergo planned hematopoietic stem cell transplantation (HSCT).

Major finding: At discontinuation, 20.3% of the patients were still responding to AZA, 35.4% had primary resistance, and 44.3% developed adaptive resistance. Long-term survival was significantly better in patients who discontinued AZA while in response vs. those with primary or adaptive resistance (P = .004) with best outcomes in patients who discontinued to undergo planned HSCT with the median survival not reached and a 5-year survival rate of 56% (P less than .001).

Study details: This retrospective study evaluated 414 patients with MDS consecutively enrolled in the Italian MDS Registry of the Fondazione Italiana Sindromi Mielodisplastiche (FISiM) and treated with AZA.

Disclosures: FISiM received support from Celgene Corporation to carry on the analysis. Some of the authors declared receiving personal fees, grants, and/or honoraria from various pharmaceutical companies including Celgene/Bristol-Myers Squibb.

Source: Clavio M et al. Cancer. 2021 Mar 19. doi: 10.1002/cncr.33472.

Key clinical point: Findings confirm dismal outcomes after azacitidine (AZA) discontinuation in patients with myelodysplastic syndrome (MDS); however, outcomes were best in those who discontinued AZA while being in response to undergo planned hematopoietic stem cell transplantation (HSCT).

Major finding: At discontinuation, 20.3% of the patients were still responding to AZA, 35.4% had primary resistance, and 44.3% developed adaptive resistance. Long-term survival was significantly better in patients who discontinued AZA while in response vs. those with primary or adaptive resistance (P = .004) with best outcomes in patients who discontinued to undergo planned HSCT with the median survival not reached and a 5-year survival rate of 56% (P less than .001).

Study details: This retrospective study evaluated 414 patients with MDS consecutively enrolled in the Italian MDS Registry of the Fondazione Italiana Sindromi Mielodisplastiche (FISiM) and treated with AZA.

Disclosures: FISiM received support from Celgene Corporation to carry on the analysis. Some of the authors declared receiving personal fees, grants, and/or honoraria from various pharmaceutical companies including Celgene/Bristol-Myers Squibb.

Source: Clavio M et al. Cancer. 2021 Mar 19. doi: 10.1002/cncr.33472.

Key clinical point: Findings confirm dismal outcomes after azacitidine (AZA) discontinuation in patients with myelodysplastic syndrome (MDS); however, outcomes were best in those who discontinued AZA while being in response to undergo planned hematopoietic stem cell transplantation (HSCT).

Major finding: At discontinuation, 20.3% of the patients were still responding to AZA, 35.4% had primary resistance, and 44.3% developed adaptive resistance. Long-term survival was significantly better in patients who discontinued AZA while in response vs. those with primary or adaptive resistance (P = .004) with best outcomes in patients who discontinued to undergo planned HSCT with the median survival not reached and a 5-year survival rate of 56% (P less than .001).

Study details: This retrospective study evaluated 414 patients with MDS consecutively enrolled in the Italian MDS Registry of the Fondazione Italiana Sindromi Mielodisplastiche (FISiM) and treated with AZA.

Disclosures: FISiM received support from Celgene Corporation to carry on the analysis. Some of the authors declared receiving personal fees, grants, and/or honoraria from various pharmaceutical companies including Celgene/Bristol-Myers Squibb.

Source: Clavio M et al. Cancer. 2021 Mar 19. doi: 10.1002/cncr.33472.