Abstract
BackgroundLenalidomide has been approved for the treatment of lower-risk myelodysplastic syndrome (MDS) with 5q deletion (del(5q)). We present for the first time a retrospective analysis of low-risk MDS with isolated del5q treated with lenalidomide, outside the clinical trials.Methods36 red blood cell (RBC) transfusion-dependent patients have been included in the study. Patients received lenalidomide 10 mg/day on days 1–21 of 28-day cycles.Results91.7 % of patients responded to lenalidomide treatment: 72.2 % achieved erythroid response, 19.4 % achieved minor erythroid response and 8.4 % of patients did not respond to treatment. Response depended on number of previous treatment lines (p = 0.0101), International Prognostic System Score (IPSS; p = 0.0067) and RBC transfusion frequency (p = 0.0139). Median duration of response was 16 months (range 6–60 months). Treatment was well tolerated. We observed hematological toxicity (grade 3 and 4): neutropenia in 16 (44.4 %) patients and thrombocytopenia in 9 (25 %) patients. Two patients (5.5 %) progressed to high-risk MDS and two subsequent progressed to acute myeloid leukemia. A Kaplan-Meier estimate for overall survival at 5 years in the study group was 79.0 ± 8.8 %.ConclusionsLenalidomide in this group of patients was beneficial for the treatment of RBC transfusion-dependency with well-known safety profile.
Highlights
Lenalidomide has been approved for the treatment of lower-risk myelodysplastic syndrome (MDS) with 5q deletion (del(5q))
In 2005, lenalidomide as an immunomodulating agent was approved by U.S Food and Drug Administration (FDA) in treatment of transfusion-dependent patients with low-risk myelodysplastic syndrome (MDS) with 5q deletion (del(5q))
Chromosome 5q deletion is one of the most frequent rearrangements observed in myelodysplastic syndrome, which may exist as an independent aberration or as a complex of cytogenetic disorders
Summary
Lenalidomide has been approved for the treatment of lower-risk myelodysplastic syndrome (MDS) with 5q deletion (del(5q)). We present for the first time a retrospective analysis of low-risk MDS with isolated del5q treated with lenalidomide, outside the clinical trials. In 2005, lenalidomide as an immunomodulating agent was approved by U.S Food and Drug Administration (FDA) in treatment of transfusion-dependent patients with low-risk myelodysplastic syndrome (MDS) with 5q deletion (del(5q)). In the majority of patients with classic del(5q) syndrome, a transfusion dependency accompanied by iron overload developed over time [3]. In the paper by Patnaik et al, it was revealed that age, transfusion needs at diagnosis and dysgranulopoiesis were independent factors affecting survival reduction in patients with chromosome 5 long arm deletion syndrome [4]. The drug modifies immunological pathways involved in pathomechanism of the disease [10]
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