In part one of the study, patients received an initial cycle of subcutaneous azacitidine (75 mg/m2 daily for the first 7 days of a 28-day cycle). Thereafter, they were assigned to different doses of oral azacitidine (120-600 mg daily) for the first seven days of repeated 28-day schedules. In part two, patients were assigned to one of four dosing schedules of oral azacitidine: 300 mg daily or 200 mg twice per day, each for either 14 or 21 days of each 28-day cycle. Patients remained on therapy until disease progression or study withdrawal.
Of the 15 patients receiving any of the part two extended dosing schedules, four (27%, 4/15 patients) achieved hematologic improvement, including patients who had complex cytogenetics and/or had failed prior therapy for AML. One of the eight patients in part one of the study treated with 7-day once daily oral azacitidine achieved a complete response with incomplete blood count recovery (CRi).
Grade 3/4 adverse events occurring in at least 10% of the 23 patients enrolled in the study included febrile neutropenia (35%, 8/23), pneumonia (17%, 4/23), syncope (17%, 4/23), and nausea (13%, 3/23). Three patients died due to causes unrelated to oral azacitidine.
Oral azacitidine is an investigational agent and has not been 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 Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of myeloid blood cells that often transforms from MDS upon disease progression. AML is the proliferation of abnormal cells that accumulate in the bone marrow and interfere with all types of normal blood cell production (also known as multi-lineage dysplasia). AML has traditionally been treated with high intensity chemotherapy, which is poorly tolerated by the majority of the patients who are afflicted - the elderly. Many of these patients may go untreated, and because they are ineligible for curative therapy, life expectancy is short and often measured in weeks to months.
About Celgene International Sàrl
Celgene International Sàrl, located in Boudry, in the
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Source: Celgene International Sàrl