Abstract

Lenalidomide has demonstrated remarkable efficacy for therapy of lower-risk myelodysplastic syndromes (MDS) associated with 5q(-). The present evaluation aimed to describe the characteristics and outcomes of low-risk MDS patients treated with lenalidomide in Austria. For this retrospective, multicenter, observational analysis of MDS patients who received lenalidomide, data were collected at various hospitals in Austria over a period of 3 years. MDS classification, previous and current MDS therapies, and outcome and safety of lenalidomide were evaluated. Forty-six percent of the patients (n= 23) had a 5q(-) syndrome, while 12% (n= 6) exhibited 5q(-) plus additional aberrations or isolated 5q(-) but≥ 5% blasts in the bone marrow (10%, n= 5). The remaining 32% of patients (n= 16) had MDS with other World Health Organization classifications. Seventy percent belonged to lower International Prognostic Scoring System risk classes. Sixteen centers participated, involving a total of 50 patients. Most frequently used lenalidomide doses were 10 mg and 5 mg on days 1 to 21 of a 28-day cycle. Seventy-five percent of the patients received 11 months of treatment, with a median therapy period of 3.5 months; median follow-up was 3.9 months (range, 0-26 months). Response rate, defined as transfusion independence during the 2 months after lenalidomide therapy, was 64%. Median overall survival was not reached. Lenalidomide was well tolerated and is an effective and well-tolerated option for therapy of patients with 5q(-) syndrome but also lower-risk MDS patients with other World Health Organization classifications in clinical practice.

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