Azacitidine Is Effective in Patients with Intermediate-2 / High IPSS Risk Myelodysplastic Syndrome Managed in Community Based Practice: Preliminary Data From the Spanish Azacitidine Compassionate Use Registry.

Myelodysplastic syndrome (MDS) is comprised of a group of heterogeneous hematological disorders. Although the decision regarding treatment of a patient with MDS is based on performance status, age, patient preference and concomitant illnesses, NCCN-recommended treatment approaches vary according to IPSS risk score. Azacitidine (AZA) is a hypomethylating agent recently approved in Europe for the treatment of MDS. AZA was available in Spain under compassionate use before its regulatory approval and marketing authorization from the Spanish Medicines Agency in May 2009.

Material and

We present the preliminary analysis of the clinical data from a longitudinal, multicenter Spanish patient registry. Data on the disease course and management of patients with MDS treated with AZA under compassionate use conditions were retrospectively collected from community-based hematology clinics. As of August 1, 2009, 65 patients with intermediate-2 / high IPSS-risk MDS diagnosed according to WHO criteria had been included.

At baseline the median age was 69 years, the male/female ratio was 60/40, and the majority of patients had primary MDS (56 patients; 88%) and an ECOG performance status of 0-1 (43 patients; 67%). The most frequent initial dose of AZA applied was 75 mg/m² (53 patients, 83%), and the most common dosing schedules were as follows: days 1-7 (82%), and days 1-5 and 8-9 (5%) in a 28-day cycle. AZA was administered mostly subcutaneously (56 patients, 89%). The mean number of cycles administered was 6 (range 2-29). The overall treatment response was 59% (International Working Group 2006 criteria): 16% complete response, 13% complete bone marrow response, 13% partial response and 18% hematological response. In addition, 23% achieved stable disease. AZA was generally well tolerated. The grade 3/4 adverse events documented in these patients, regardless of their relationship to active treatment, were neutropenia (37%), thrombocytopenia (26%), anemia (17%), febrile neutropenia (8%), rash (2%), vomiting or nausea (2%), and constitutional symptoms (2%).

These preliminary data do not differ from those previously obtained from clinical trials (Silverman et al 2006, Fenaux et al 2009). Our results demonstrate that in a community-based setting, patients with intermediate-2 / high-risk MDS respond to treatment with AZA. 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.

No relevant conflicts of interest to declare.