Clinical Edge Journal Scan

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.

Key clinical point: Red blood cell transfusion independence (TI) among patients with myelodysplastic syndrome (MDS) was associated with longer overall survival and lower risk for acute myeloid leukemia (AML) progression.

Major finding: Risk of death was 61.8% (hazard ratio [HR], 0.382; 95% credible interval [CrI], 0.201-0.666) lower in patients with TI vs. transfusion dependence (TD) and 63.4% (HR, 0.366; 95% CrI, 0.217-0.553) lower in patients who acquired TI through treatment vs. those who did not. Patients with TD vs. TI showed a trend toward worse prognosis and higher risk for AML progression (HR, 0.754; 95% CrI, 0.311-1.811).

Study details: Findings are from a meta-analysis of pooled studies that estimated the effect of transfusion sensitivity on clinical outcomes in patients with MDS.

Disclosures: The study was supported by Bristol-Myers Squibb Company. D Tang reported being an employee and equity holder of Bristol-Myers Squibb. T Schroeder reported being a consultant for Celgene International, a Bristol-Myers Squibb Company. The other authors declared no conflicts of interest.

Source: Lemos MB et al. Eur J Haematol. 2021 Mar 14. doi: 10.1111/ejh.13619.

Key clinical point: Red blood cell transfusion independence (TI) among patients with myelodysplastic syndrome (MDS) was associated with longer overall survival and lower risk for acute myeloid leukemia (AML) progression.

Major finding: Risk of death was 61.8% (hazard ratio [HR], 0.382; 95% credible interval [CrI], 0.201-0.666) lower in patients with TI vs. transfusion dependence (TD) and 63.4% (HR, 0.366; 95% CrI, 0.217-0.553) lower in patients who acquired TI through treatment vs. those who did not. Patients with TD vs. TI showed a trend toward worse prognosis and higher risk for AML progression (HR, 0.754; 95% CrI, 0.311-1.811).

Study details: Findings are from a meta-analysis of pooled studies that estimated the effect of transfusion sensitivity on clinical outcomes in patients with MDS.

Disclosures: The study was supported by Bristol-Myers Squibb Company. D Tang reported being an employee and equity holder of Bristol-Myers Squibb. T Schroeder reported being a consultant for Celgene International, a Bristol-Myers Squibb Company. The other authors declared no conflicts of interest.

Source: Lemos MB et al. Eur J Haematol. 2021 Mar 14. doi: 10.1111/ejh.13619.

Key clinical point: Red blood cell transfusion independence (TI) among patients with myelodysplastic syndrome (MDS) was associated with longer overall survival and lower risk for acute myeloid leukemia (AML) progression.

Major finding: Risk of death was 61.8% (hazard ratio [HR], 0.382; 95% credible interval [CrI], 0.201-0.666) lower in patients with TI vs. transfusion dependence (TD) and 63.4% (HR, 0.366; 95% CrI, 0.217-0.553) lower in patients who acquired TI through treatment vs. those who did not. Patients with TD vs. TI showed a trend toward worse prognosis and higher risk for AML progression (HR, 0.754; 95% CrI, 0.311-1.811).

Study details: Findings are from a meta-analysis of pooled studies that estimated the effect of transfusion sensitivity on clinical outcomes in patients with MDS.

Disclosures: The study was supported by Bristol-Myers Squibb Company. D Tang reported being an employee and equity holder of Bristol-Myers Squibb. T Schroeder reported being a consultant for Celgene International, a Bristol-Myers Squibb Company. The other authors declared no conflicts of interest.

Source: Lemos MB et al. Eur J Haematol. 2021 Mar 14. doi: 10.1111/ejh.13619.

Key clinical point: Oral azacitidine (CC-486) was associated with a significantly higher red blood cell (RBC) transfusion independence (TI) rate in patients with low-risk myelodysplastic syndrome (MDS) and high-risk disease features. The rate of early death was higher in the CC-846 arm.

Major finding: A significantly higher proportion of patients achieved RBC TI for 56 consecutive days in CC-486 vs. placebo arms (30.8% vs. 11.1%; odds ratio, 3.6; P = .0002). Overall, the death rate was similar, but death within the first 56 days was higher with CC-486 vs. placebo (16 vs. 6), most related to infections in patients with significant pretreatment neutropenia.

Study details: Findings are from AZA-MDS-003, a phase 3 trial involving 216 patients with low-risk MDS and RBC transfusion-dependent anemia and thrombocytopenia who were randomly allocated to either CC-486 (n=107) or placebo (n=109) for 21 days/28-day cycle.

Disclosures: The study was supported by Celgene Corporation, a Bristol-Myers Squibb company. Some of the authors declared receiving research funding, honoraria, travel and accommodation expenses, consulting/advisory role, being an employee of and/or stock/other ownership interest in various pharmaceutical companies including Celgene.

Source: Garcia-Manero G et al. J Clin Oncol. 2021 Mar 25. doi: 10.1200/JCO.20.02619. 

Key clinical point: Oral azacitidine (CC-486) was associated with a significantly higher red blood cell (RBC) transfusion independence (TI) rate in patients with low-risk myelodysplastic syndrome (MDS) and high-risk disease features. The rate of early death was higher in the CC-846 arm.

Major finding: A significantly higher proportion of patients achieved RBC TI for 56 consecutive days in CC-486 vs. placebo arms (30.8% vs. 11.1%; odds ratio, 3.6; P = .0002). Overall, the death rate was similar, but death within the first 56 days was higher with CC-486 vs. placebo (16 vs. 6), most related to infections in patients with significant pretreatment neutropenia.

Study details: Findings are from AZA-MDS-003, a phase 3 trial involving 216 patients with low-risk MDS and RBC transfusion-dependent anemia and thrombocytopenia who were randomly allocated to either CC-486 (n=107) or placebo (n=109) for 21 days/28-day cycle.

Disclosures: The study was supported by Celgene Corporation, a Bristol-Myers Squibb company. Some of the authors declared receiving research funding, honoraria, travel and accommodation expenses, consulting/advisory role, being an employee of and/or stock/other ownership interest in various pharmaceutical companies including Celgene.

Source: Garcia-Manero G et al. J Clin Oncol. 2021 Mar 25. doi: 10.1200/JCO.20.02619. 

Key clinical point: Oral azacitidine (CC-486) was associated with a significantly higher red blood cell (RBC) transfusion independence (TI) rate in patients with low-risk myelodysplastic syndrome (MDS) and high-risk disease features. The rate of early death was higher in the CC-846 arm.

Major finding: A significantly higher proportion of patients achieved RBC TI for 56 consecutive days in CC-486 vs. placebo arms (30.8% vs. 11.1%; odds ratio, 3.6; P = .0002). Overall, the death rate was similar, but death within the first 56 days was higher with CC-486 vs. placebo (16 vs. 6), most related to infections in patients with significant pretreatment neutropenia.

Study details: Findings are from AZA-MDS-003, a phase 3 trial involving 216 patients with low-risk MDS and RBC transfusion-dependent anemia and thrombocytopenia who were randomly allocated to either CC-486 (n=107) or placebo (n=109) for 21 days/28-day cycle.

Disclosures: The study was supported by Celgene Corporation, a Bristol-Myers Squibb company. Some of the authors declared receiving research funding, honoraria, travel and accommodation expenses, consulting/advisory role, being an employee of and/or stock/other ownership interest in various pharmaceutical companies including Celgene.

Source: Garcia-Manero G et al. J Clin Oncol. 2021 Mar 25. doi: 10.1200/JCO.20.02619. 

Key clinical point: Bone marrow stromal cells in patients with myelodysplastic syndromes and acute myeloid leukemia showed a reduced frequency of colony forming unit-fibroblasts.

Major finding: The frequencies of positive/positive bone marrow stromal cells were significantly lower in AML patient samples compared to healthy individuals and MDS bone marrow samples. The median cell numbers of bone marrow stromal cells in diagnostic samples were 3,200 for healthy samples, 476 for MDS samples, and 70 for AML samples.

Study details: The data come from bone marrow samples in AML patients, MDS patients, and healthy donors.

Disclosures: The study was supported in part by Projekt DEAL and the Deutsche Forschungsgemeinschaft SFB 1243 for projects A09 and the FOR2033 project B3, as well as the Deutsche Jose Carreras Leukämie Stiftung. The researchers had no financial conflicts to disclose. 

Source: Weickert M-T et al. Sci Rep. 2021 Mar 15. doi: 10.1038/s41598-021-85122-8.

Key clinical point: Bone marrow stromal cells in patients with myelodysplastic syndromes and acute myeloid leukemia showed a reduced frequency of colony forming unit-fibroblasts.

Major finding: The frequencies of positive/positive bone marrow stromal cells were significantly lower in AML patient samples compared to healthy individuals and MDS bone marrow samples. The median cell numbers of bone marrow stromal cells in diagnostic samples were 3,200 for healthy samples, 476 for MDS samples, and 70 for AML samples.

Study details: The data come from bone marrow samples in AML patients, MDS patients, and healthy donors.

Disclosures: The study was supported in part by Projekt DEAL and the Deutsche Forschungsgemeinschaft SFB 1243 for projects A09 and the FOR2033 project B3, as well as the Deutsche Jose Carreras Leukämie Stiftung. The researchers had no financial conflicts to disclose. 

Source: Weickert M-T et al. Sci Rep. 2021 Mar 15. doi: 10.1038/s41598-021-85122-8.

Key clinical point: Bone marrow stromal cells in patients with myelodysplastic syndromes and acute myeloid leukemia showed a reduced frequency of colony forming unit-fibroblasts.

Major finding: The frequencies of positive/positive bone marrow stromal cells were significantly lower in AML patient samples compared to healthy individuals and MDS bone marrow samples. The median cell numbers of bone marrow stromal cells in diagnostic samples were 3,200 for healthy samples, 476 for MDS samples, and 70 for AML samples.

Study details: The data come from bone marrow samples in AML patients, MDS patients, and healthy donors.

Disclosures: The study was supported in part by Projekt DEAL and the Deutsche Forschungsgemeinschaft SFB 1243 for projects A09 and the FOR2033 project B3, as well as the Deutsche Jose Carreras Leukämie Stiftung. The researchers had no financial conflicts to disclose. 

Source: Weickert M-T et al. Sci Rep. 2021 Mar 15. doi: 10.1038/s41598-021-85122-8.