In the study, patients with lower-risk (IPSS Low or INT-1) MDS who were red blood cell transfusion-dependent and/or thrombocytopenic were enrolled and sequentially assigned to receive oral azacitidine 300 mg once daily for either 14 or 21 days of each 28-day cycle. Patients were assessed for safety and hematologic response.
At study entry, 40% of patients had received no prior MDS treatment (outside of transfusions), 45% had received erythropoiesis-stimulating agents, and 15% had received white blood cell growth factors. At the time of the analysis, 53 patients had been enrolled.
After a median seven treatment cycles for the 14-day treatment arm and five for the 21-day treatment arm, the most frequent grade 3/4 hematologic adverse events in the 14-day arm were anemia (11.5%), thrombocytopenia (11.5%) and neutropenia (7.7%), and in the 21-day arm were neutropenia (18.5%), febrile neutropenia (11.1%) and anemia (7.4%). The most frequent grade 3/4 non-hematologic adverse events were pneumonia (15.4%), vomiting (7.7%), diarrhea (7.7%) and cellulitis (7.7%) in the 14-day arm, and diarrhea (11.1%) and vomiting (7.4%) in the 21-day arm. At data cut-off, 36 patients had discontinued treatment, including eight who discontinued due to adverse events. Three patients in the 21-day arm and two patients in the 14-day arm received reduced doses of oral azacitidine (200 mg) due to adverse events.
The overall response rate for patients in the 14-day arm was 42.3%
(11/26) and 37.0% (10/27) in the 21-day arm. Additionally, the
percentage of patients showing any hematologic improvement was 26.9%
(7/26) in the 14-day arm and 29.6% (8/27) in the 21-day arm. The
percentage of patients who sustained
Based on these and other early-stage data evaluating CC-486,
These results are from an investigational study. CC-486 is not approved for any indication.
About Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) are a group of hematologic malignancies
that affect approximately 300,000 people worldwide. Myelodysplastic
syndromes occur when blood cells remain in an immature or “blast” stage
within the bone marrow and never develop into mature cells capable of
performing their necessary functions. Eventually, the bone marrow may be
filled with blast cells, suppressing normal cell development. According
About Celgene International Sàrl
Celgene International Sàrl, located in Boudry, in the
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