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Lenalidomide treatment in myelodysplastic syndrome with 5q deletion – Czech MDS group experience


Authors: Anna Jonášová 1;  Libor Červinek 2;  Petra Bělohlávková 3;  Jaroslav Čermák 4;  Monika Beličková 4;  Petr Rohoň 5;  Olga Černá 6;  Ivana Hochová 7;  Magda Šišková 1;  Karla Kačmářová 8;  Eva Janoušová 9
Authors‘ workplace: I. interní klinika – klinika hematologie 1. LK UK a VFN Praha, přednosta prof. MUDr. Marek Trněný, CSc. 1;  Interní hematologická a onkologická klinika LF MU a FN Brno, pracoviště Bohunice, přednosta prof. MUDr. Jiří Mayer, CSc. 2;  IV. interní hematologická klinika LF UK a FN Hradec Králové, přednosta doc. MUDr. Pavel Žák, Ph. D. 3;  Ústav hematologie a krevní transfuze Praha, ředitel prof. MUDr. Petr Cetkovský, Ph. D., MBA 4;  Hemato-onkologická klinika LF UP a FN Olomouc, přednosta prof. MUDr. Tomáš Papajík, CSc. 5;  Hematologická klinika 3. LF UK a FN Královské Vinohrady Praha, přednosta prof. MUDr. Tomáš Kozák, Ph. D., MBA 6;  Oddělení klinické hematologie 2. LF UK a FN Motol Praha, přednostka prim. MUDr. Ivana Hochová 7;  Česká MDS skupina 8;  Institut biostatistiky a analýz LF MU Brno, ředitel doc. RNDr. Ladislav Dušek, Ph. D. 9
Published in: Vnitř Lék 2015; 61(12): 1028-1033
Category: Original Contributions

Overview

Myelodysplastic syndrome (MDS) is a common hematological disease in patients over sixty. Despite intensive research, the therapy of this heterogeneous blood disease is complicated. In recent years, two new therapeutic approaches have been proposed: immunomodulation and demethylation therapy. Immunomodulation therapy with lenalidomide represents a meaningful advance in the treatment of anemic patients, specifically those with 5q- aberrations. As much as 60–70 % of patients respond and achieve transfusion independence. We present the initial lenalidomide experience of the Czech MDS group. We analyze Czech MDS register data of 34 (31 female; 3 male; median age 69 years) chronically transfused low risk MDS patients with 5q- aberration treated by lenalidomide. Twenty-seven (79.4 %) patients were diagnosed with 5q- syndrome, 5 patients with refractory anemia with multilineage dysplasia, 1 patient with refractory anemia with excess of blasts 1, and 1 patient with myelodysplastic/myeloproliferative unclassified. Response, as represented by achieving complete transfusion independence, was achieved in 91 % of patients. A true 5q- syndrome diagnosis in most our patients may be responsible for such a high response rate. Complete cytogenetic response was reached in 15 % of patients and partial cytogenetic response in 67 %, within a median time of 12 months. TP53 mutation was detected in 15 % (3 from 18 tested) and 2 of these patients progressed to higher grade MDS. The majority of patients tolerated lenalidomide very well. Based on this albeit small study, we present our findings of high lenalidomide efficacy as well as the basic principles and problems of lenalidomide therapy.

Key words:
Czech registry – lenalidomide – myelodysplastic syndrome – therapy


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