Background: The phase 2 BRIGHT AML 1003 trial evaluated efficacy and safety of glasdegib + low-dose cytarabine (LDAC) in patients with acute myeloid leukemia ineligible for intensive chemotherapy. The multicenter, open-label study randomized patients to receive glasdegib + LDAC (n = 78) or LDAC alone (n = 38). The rate of complete remission (CR) was 19.2% in the glasdegib + LDAC arm versus 2.6% in the LDAC arm (P = 0.015). Methods: This post hoc analysis determines whether the clinical benefits of glasdegib are restricted to patients who achieve CR, or if they extend to those who do not achieve CR. Results: In patients who did not achieve CR, the addition of glasdegib to LDAC improved overall survival (OS) versus LDAC alone (hazard ratio = 0.63 [95% confidence interval, 0.41-0.98]; P = 0.0182; median OS, 5.0 vs 4.1 months). Additionally, more patients receiving glasdegib + LDAC achieved durable recovery of absolute neutrophil count (≥ 1000/μl, 45.6% vs 35.5%), hemoglobin (≥ 9 g/dl, 54.4% vs 38.7%), and platelets (≥ 100,000/μl, 29.8% vs 9.7%). Transfusion independence was achieved by 15.0% and 2.9% of patients receiving glasdegib + LDAC and LDAC alone, respectively. Conclusions: Collectively, these data suggest that there are clinical benefits with glasdegib in the absence of CR. Trial registration: ClinicalTrials.gov NCT01546038 (March 7, 2012)

Survival outcomes and clinical benefit in patients with acute myeloid leukemia treated with glasdegib and low-dose cytarabine according to response to therapy / Cortes, J. E.; Heidel, F. H.; Fiedler, W.; Smith, B. D.; Robak, T.; Montesinos, P.; Candoni, A.; Leber, B.; Sekeres, M. A.; Pollyea, D. A.; Ferdinand, R.; Ma, W. W.; O'Brien, T.; O'Connell, A.; Chan, G.; Heuser, M.. - In: JOURNAL OF HEMATOLOGY & ONCOLOGY. - ISSN 1756-8722. - 13:1(2020), pp. 1-10. [10.1186/s13045-020-00929-8]

Survival outcomes and clinical benefit in patients with acute myeloid leukemia treated with glasdegib and low-dose cytarabine according to response to therapy

Candoni A.;
2020-01-01

Abstract

Background: The phase 2 BRIGHT AML 1003 trial evaluated efficacy and safety of glasdegib + low-dose cytarabine (LDAC) in patients with acute myeloid leukemia ineligible for intensive chemotherapy. The multicenter, open-label study randomized patients to receive glasdegib + LDAC (n = 78) or LDAC alone (n = 38). The rate of complete remission (CR) was 19.2% in the glasdegib + LDAC arm versus 2.6% in the LDAC arm (P = 0.015). Methods: This post hoc analysis determines whether the clinical benefits of glasdegib are restricted to patients who achieve CR, or if they extend to those who do not achieve CR. Results: In patients who did not achieve CR, the addition of glasdegib to LDAC improved overall survival (OS) versus LDAC alone (hazard ratio = 0.63 [95% confidence interval, 0.41-0.98]; P = 0.0182; median OS, 5.0 vs 4.1 months). Additionally, more patients receiving glasdegib + LDAC achieved durable recovery of absolute neutrophil count (≥ 1000/μl, 45.6% vs 35.5%), hemoglobin (≥ 9 g/dl, 54.4% vs 38.7%), and platelets (≥ 100,000/μl, 29.8% vs 9.7%). Transfusion independence was achieved by 15.0% and 2.9% of patients receiving glasdegib + LDAC and LDAC alone, respectively. Conclusions: Collectively, these data suggest that there are clinical benefits with glasdegib in the absence of CR. Trial registration: ClinicalTrials.gov NCT01546038 (March 7, 2012)
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Survival outcomes and clinical benefit in patients with acute myeloid leukemia treated with glasdegib and low-dose cytarabine according to response to therapy / Cortes, J. E.; Heidel, F. H.; Fiedler, W.; Smith, B. D.; Robak, T.; Montesinos, P.; Candoni, A.; Leber, B.; Sekeres, M. A.; Pollyea, D. A.; Ferdinand, R.; Ma, W. W.; O'Brien, T.; O'Connell, A.; Chan, G.; Heuser, M.. - In: JOURNAL OF HEMATOLOGY & ONCOLOGY. - ISSN 1756-8722. - 13:1(2020), pp. 1-10. [10.1186/s13045-020-00929-8]
Cortes, J. E.; Heidel, F. H.; Fiedler, W.; Smith, B. D.; Robak, T.; Montesinos, P.; Candoni, A.; Leber, B.; Sekeres, M. A.; Pollyea, D. A.; Ferdinand, R.; Ma, W. W.; O'Brien, T.; O'Connell, A.; Chan, G.; Heuser, M.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/1294022
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