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.

Key clinical point: Among patients with a solid tumor, the use of poly(adenosine diphosphate-ribose) polymerase inhibitor (PARPi) was associated with an increased incidence of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) in those receiving a PARPi early in their disease course.

Major finding: Use of PARPi was associated with an increased incidence of MDS/AML in the frontline setting (incidence rate ratio [IRR], 5.43; 95% confidence interval [CI], 1.51–19.60) but not in the recurrent setting.

Study details: Findings are from a meta-analysis of 14 phase 2 and 3 clinical trials that randomly allocated 5,739 patients with solid tumors to either PARPi or control therapy.

Disclosures: This study was supported by the National Institutes of Health, National Cancer Institute, and National Center for Advancing Translational Sciences. Some of the authors declared receiving research funding, grants, and/or personal fees from various pharmaceutical companies.

Source: Nitecki R et al. Gynecol Oncol. 2021 Mar 15. doi: 10.1016/j.ygyno.2021.03.011.

Key clinical point: Among patients with a solid tumor, the use of poly(adenosine diphosphate-ribose) polymerase inhibitor (PARPi) was associated with an increased incidence of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) in those receiving a PARPi early in their disease course.

Major finding: Use of PARPi was associated with an increased incidence of MDS/AML in the frontline setting (incidence rate ratio [IRR], 5.43; 95% confidence interval [CI], 1.51–19.60) but not in the recurrent setting.

Study details: Findings are from a meta-analysis of 14 phase 2 and 3 clinical trials that randomly allocated 5,739 patients with solid tumors to either PARPi or control therapy.

Disclosures: This study was supported by the National Institutes of Health, National Cancer Institute, and National Center for Advancing Translational Sciences. Some of the authors declared receiving research funding, grants, and/or personal fees from various pharmaceutical companies.

Source: Nitecki R et al. Gynecol Oncol. 2021 Mar 15. doi: 10.1016/j.ygyno.2021.03.011.

Key clinical point: Among patients with a solid tumor, the use of poly(adenosine diphosphate-ribose) polymerase inhibitor (PARPi) was associated with an increased incidence of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) in those receiving a PARPi early in their disease course.

Major finding: Use of PARPi was associated with an increased incidence of MDS/AML in the frontline setting (incidence rate ratio [IRR], 5.43; 95% confidence interval [CI], 1.51–19.60) but not in the recurrent setting.

Study details: Findings are from a meta-analysis of 14 phase 2 and 3 clinical trials that randomly allocated 5,739 patients with solid tumors to either PARPi or control therapy.

Disclosures: This study was supported by the National Institutes of Health, National Cancer Institute, and National Center for Advancing Translational Sciences. Some of the authors declared receiving research funding, grants, and/or personal fees from various pharmaceutical companies.

Source: Nitecki R et al. Gynecol Oncol. 2021 Mar 15. doi: 10.1016/j.ygyno.2021.03.011.