Azacitidine Is Effective in Patients with Myelodysplastic Syndrome Managed in Community-Based Practice: Preliminary Data From the Spanish Azacitidine Compassionate Use Registry.

Abstract Abstract 4836 Background Azacitidine (AZA), a hypomethylating agent recently approved in Europe for the treatment of MDS, prolongs the median survival time in patients enrolled in clinical trials (Fenaux et al 2009). Decisions regarding treatment are based on performance status, age, patient preference and concomitant illnesses. NCCN-recommended treatment approaches vary according to International Prognostic Scoring System (IPSS) classification. AZA was available in Spain under compassionate use before its regulatory approval in May 2009. Material and Methods We present the preliminary analysis of the clinical results of the data from a longitudinal, multicenter Spanish patient registry, which retrospectively collected data from community-based hematology clinics on the disease course and management of patients with MDS treated with AZA in a compassionate use setting, in whom the dosing schedule administered was documented. AZA dosing was applied to the patients in different regimens in 28-day cycles. As of August 1, 2009, data from 147 patients with MDS diagnosed according to WHO criteria had been collected. Results Median age was 71 years and 66% were men. Most of the patients had primary MDS (129 patients; 88%). The most prevalent ECOG performance status was 0-1 (125 patients; 85%). An IPSS risk classification of low/intermediate-1 was documented in 80 patients (55%). An initial AZA dose of 75 mg/m2 was used in 124 patients (84%). Most of the patients (57%) received a 7-day regimen (either with or without a weekend rest). A 5-day regimen was used in 50 patients (31%). AZA was administered mostly subcutaneously (122 patients, 84%). The mean number of cycles administered was 6 (range 2-29). The overall treatment response was 60% (International Working Group 2006 criteria): 18% complete response, 16% complete bone marrow response, 7% partial response, 23% hematological response. Stable disease was documented in an additional 17% of the patients, who did not achieve a response. AZA was generally well tolerated. Grade 3/4 adverse events documented in these patients, regardless of their relationship to the active treatment, were neutropenia (42%), thrombocytopenia (31%), anemia (22%), febrile neutropenia (10%), rash (1%), vomiting or nausea (1%), septic shock (1%), injection site reaction (1%), and constitutional symptoms (1%) Conclusion These preliminary data do not differ from those previously obtained from clinical trials (Silverman et al 2006, Fenaux et al 2009). These results demonstrate that in a community-based setting, patients with MDS respond to AZA treatment. In line with previous clinical trials, we expect this could point to a better prognosis for MDS patients in Spain, with prolonged survival and a better quality of life. Disclosures No relevant conflicts of interest to declare.