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Published on: September 9, 2011
Phase I/II study of decitabine in patients with myelodysplastic syndrome: a multi-center study in Japan
Yasuhiro Oki1, Yutaka Kondo, Kazuhito Yamamoto
1Department of Hematology and Cell Therapy, Aichi Cancer Center Hospital, Nagoya, Japan. yoki@mdanderson.org
Abstract:
The management of myelodysplastic syndrome (MDS) remains challenging. We performed a phase I/II study to evaluate the safety and efficacy of decitabine in patients with MDS in Japan. Patients with MDS with red cell transfusion dependence or 5-30% blasts in marrow and with an International Prognostic Scoring System score of intermediate-1 or higher were eligible. Patients received intravenous decitabine at 15 or 20 mg/m(2) daily for 5 days every 4 weeks. A total of 37 patients were enrolled. Three patients received 15 mg/m(2) and experienced no dose limiting toxicity during the first cycle. Thirty-four patients received 20 mg/m(2) . Grade 3 or greater non-hematologic toxicities included cerebral infarction (n = 1), subdural hematoma (n = 1), elevated blood glucose (n = 1), and pulmonary hypertension (n = 1). At 20 mg/m(2) , complete response, partial response, and hematologic improvement were observed in 7 (20.6%), 2 (5.9%), and 7 (20.6%) patients, respectively. Complete cytogenetic response was observed in 30% of evaluable 20 patients. The median number of cycles to clinical response was 4 (range 4-8), and duration of remission was 474+ days (range 294-598+). The 2-year rate of acute myeloid leukemia-free survival was 52%. Correlative studies revealed hypomethylation in multiple genes in peripheral blood cells after treatment. Hypomethylation was generally more profound in CD15 + peripheral blood cells, which reflects myeloid cells, than in peripheral blood mononuclear cells. In summary, decitabine was safe and demonstrated efficacy in Japanese patients with high-risk MDS. This trial was registered at ClinicalTrials.gov (NCT00796003).
Insights
Decitabine showed safety and efficacy in Japanese patients with high-risk myelodysplastic syndrome (MDS). The treatment led to responses and improved survival, with observed gene hypomethylation.
Area of Science:
- Hematology
- Oncology
- Clinical Pharmacology
Background:
- Myelodysplastic syndromes (MDS) present significant management challenges.
- High-risk MDS requires effective therapeutic strategies.
Purpose of the Study:
- To evaluate the safety and efficacy of decitabine in Japanese patients with myelodysplastic syndrome (MDS).
- To assess treatment response, survival rates, and molecular changes associated with decitabine therapy.
Main Methods:
- A phase I/II clinical trial involving 37 Japanese patients with intermediate-1 or higher risk MDS.
- Intravenous decitabine administered at 15 or 20 mg/m(2)/day for 5 days every 4 weeks.
- Monitoring for dose-limiting toxicities, response rates (complete response, partial response, hematologic improvement), cytogenetic response, and survival outcomes.
Main Results:
- Decitabine at 20 mg/m(2) demonstrated efficacy, with complete response in 20.6%, partial response in 5.9%, and hematologic improvement in 20.6% of patients.
- Complete cytogenetic response was observed in 30% of evaluable patients.
- The 2-year acute myeloid leukemia-free survival rate was 52%, with a median duration of remission of 474+ days.
- Correlative studies indicated gene hypomethylation in peripheral blood cells post-treatment.
Conclusions:
- Decitabine is a safe and effective treatment option for Japanese patients with high-risk MDS.
- The drug induces clinical and cytogenetic responses and improves leukemia-free survival.
- Decitabine's mechanism involves hypomethylation of genes in myeloid cells.
