Myelodysplastic syndromes (MDS) comprise a heterogeneous group of hematopoietic stem cell disorders characterized by dysplasia resulting in cytopenia in one or more cell lineages. Th e World Health Organization (WHO) classifi cation recognizes MDS associated with an isolated partial deletion of the long arm of chromosome 5 [del(5q)] as a distinct entity. Th e so-called del(5q) syndrome is characterized by macrocytic anemia, with or without other cytopenias, and less than 5% blasts in the bone marrow. Treatment of MDS consists traditionally of red blood cell (RBC) transfusions. In 2005 the Food and Drug Administration approved lenalidomide for the treatment of RBC transfusion-dependent MDS with del(5q). In Europe, lenalidomide is not yet approved for the treatment of patients with MDS and del(5q).Th e mechanisms of action include direct anti-tumor activity (e.g. via secreted protein acidic and rich in cysteine [SPARC], inhibitory eff ects on cdc25c and pp2ac and others), inhibition of angiogenesis and immune modulation [1,2]. Patients with del(5q) who respond to therapy with lenalidomide benefi t from improvement of anemia and transfusion independence, as shown in the MDS001 and MDS003 trials [3,4]. Recently, the French named patient program (NPP) and MDS004 trial confi rmed the high rate of transfusion independence upon treatment with lenalidomide in MDS with del(5q) [5,6]. In this study, we describe the evaluation of the fi rst Dutch NPP, investigating the effi cacy of lenalidomide in patients with MDS and del(5q).Patients were eligible for inclusion if diagnosed with MDS and del(5q), with or without additional cytogenetic abnormalities. Between 2007 and 2009, 40 patients were enrolled in the program after informed consent, in accordance with the Declaration of Helsinki. Case report forms could not be retrieved from 13 patients. Diagnoses were made using the WHO 2001 classifi cation. Karyotypes were examined and described according to the International System for Human Cytogenetic Nomenclature guidelines [7]. If conventional karyotyping failed due to lack of metaphases, a fl uorescence in situ hybridization (FISH) procedure was performed. Eight patients were excluded: one patient had a duplicate identity, fi ve patients had a normal karyotype at baseline and two patients received lenalidomide for less than 1 month. Among the 19 patients that could be evaluated, two patients were already receiving lenalidomide treatment in the context of the MDS004 trial. Both patients were included and continued treatment in the Dutch NPP after termination of the MDS004 trial. Patients received lenalidomide at a daily dose of 10 mg or 5 mg for every 21 days or continuously on a 28-day cycle. Th e dose and schedule were adjusted based on drug toxicity and blood counts according to standard guidelines. Response was evaluated using the International Working Group (IWG) 2006 response criteria [8]. Th e endpoints were disease status, hematologic improvement and cytogenetic response. Response was categorized as complete remission (CR)...
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