Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8453260 | Leukemia Research | 2018 | 7 Pages |
Abstract
Thrombocytopenia is among the strongest predictors of decreased survival for patients with myelodysplastic syndromes (MDS) across all prognostic risk groups. The safety and efficacy of CC-486 (oral azacitidine) was investigated in early-phase studies; we assessed clinical outcomes among subgroups of MDS patients from these studies, defined by presence or lack of pretreatment thrombocytopenia (â¤75âÃâ109/L platelet count). Patients received CC-486 300âmgâonce-daily for 14 or 21 days of repeated 28-day cycles. Overall, 81 patients with MDS, median age 72 years, comprised the Low Platelets (nâ=â45) and High Platelets (nâ=â36) cohorts. Pretreatment median platelet counts were 34âÃâ109/L and 198âÃâ109/L, respectively. Grade 3-4 bleeding events occurred in 2 patients in the Low Platelets and 1 patient in the High Platelets groups; events resolved without sequelae. Treatment-related mortality was reported for 7 patients, 5 of whom had pretreatment platelet values <25âÃâ109/L. Overall response rates were 38% and 46% in the Low Platelets and High Platelets groups, respectively. Five thrombocytopenic patients attained complete remission and 9 attained platelet hematologic improvement. In both cohorts, platelet counts dropped during the first CC-486 treatment cycle, then increased thereafter. Extended CC-486 dosing was generally well tolerated and induced hematologic responses in these patients regardless of pretreatment thrombocytopenia.
Keywords
HMANCI-CTCAERA with excess blastsAMLRCMDInternational Working GroupIWGRAEBCMMLIPSSTPONCCNANCECoGWBCORRMDSRBCMCRRARsTEAEoverall survivalpartial remissionThrombocytopeniaThrombopoietinAbsolute neutrophil countbaselinecomplete remissionWorld Health OrganizationMyelodysplastic syndromesRefractory cytopenia with multilineage dysplasiaInternational Prognostic Scoring SystemNational Comprehensive Cancer Networkeastern cooperative oncology groupHypomethylating agenttreatment-emergent adverse eventpharmacokineticconfidence intervalsacute myeloid leukemiachronic myelomonocytic leukemiaNational Cancer Institute Common Terminology Criteria for Adverse Eventsbone marrowoverall response rateRefractory anemiaWHOwhite blood cellred blood cell
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Authors
Guillermo Garcia-Manero, Bart L. Scott, Christopher R. Cogle, Thomas E. Boyd, Suman Kambhampati, Joel Hetzer, Qian Dong, Keshava Kumar, Stacey M. Ukrainskyj, CL Beach, Barry S. Skikne,