A randomized controlled trial of romiplostim in patients with low- or intermediate-risk myelodysplastic syndrome (MDS) receiving decitabine

Leukemia & lymphoma (Impact Factor: 2.89). 08/2012; 54(2). DOI: 10.3109/10428194.2012.713477
Source: PubMed


Abstract MDS patients receiving hypomethylating agents commonly develop thrombocytopenia. This double-blind study evaluated the efficacy and safety of romiplostim, a peptibody protein that increases platelets, in MDS patients receiving decitabine. Patients received romiplostim 750μg (n=15) or placebo (n=14) and decitabine. Median platelet counts at the beginning of each decitabine cycle trended lower in placebo treated than romiplostim treated patients. Bleeding events occurred in 43% of placebo and 27% of romiplostim patients; platelet transfusions were administered to 57% of placebo and 47% of romiplostim patients. Overall clinical therapeutic response was achieved by 21% of placebo and 33% of romiplostim patients. Treatment was generally well tolerated. Progression to AML occurred in one patient per group. Adding romiplostim to decitabine treatment is well tolerated and may be beneficial, as indicated by trends toward higher platelet counts at the beginning of each treatment cycle and lower platelet transfusion rates and percentages of patients with bleeding events.

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    • "Une augmentation du pourcentage de blastes (le récepteur de la TPO étant exprimé par les cellules souches hématopoïétiques), réversible à l'arrêt du traitement, a été observée chez 10 à 15 % des patients, amenant à arrêter son utilisation dans les SMD. Le romiplostin a également montré qu'il pouvait réduire la thrombopénie quand il était associé à l'administration d'azacitidine , de décitabine et de lénalidomide dans les SMD [73] [74] [75]. Mais l'augmentation du pourcentage de blastes sous ce traitement limite son utilisation dans les SMD. "
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    ABSTRACT: Myelodysplastic syndromes are heterogeneous diseases whose molecular characteristics have only been identified in recent years. Better identification of prognostic factors, larger access to allogeneic stem cell transplantation and the advent of new drugs notably hypomethylating agents (azacitidine, decitabine) and lenalidomide have improved patient outcome.
    Bulletin du cancer 09/2015; DOI:10.1016/j.bulcan.2015.07.007 · 0.60 Impact Factor
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    • "Do zaburzeń niepożądanych, uznanych za związane z leczeniem, należą: przejściowe zwiększenie ilości włókien retikulinowych w szpiku, trombocytoza , pogorszenie małopłytkowości po zaprzestaniu leczenia oraz ryzyko zakrzepicy. Z uwagi na fakt pobudzania komórek prekursorowych, romiplostim może stymulować progresję istniejących zespołów mielodysplastycznych do ostrej białaczki szpikowej [14] [15]. Częstość występowania działań niepożądanych w postaci bólów głowy, zmęczenia, krwawień z nosa, bólów stawów oraz wybroczyn skórnych jest podobna do grupy placebo [10] [16]. "
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    ABSTRACT: The most common cause of isolated thrombocytopenia is primary immune thrombocytopenia (ITP). For patients failing initial corticosteroid-based treatment and with refractory ITP post-splenectomy, thrombopoietin receptor agonists are indicated. Two of this thrombopoiesis-stimulating agents have been approved for use in ITP – eltrombopag, formulated for oral administration, once a day and romiplostim, which is administered weekly as a subcutaneous injection.
    Acta haematologica Polonica 04/2013; 44(2):130-134. DOI:10.1016/j.achaem.2013.02.017
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    • "Romiplostim is indicated for the treatment of thrombocytopenia in patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy [18]. It has been investigated for treatment of MDS patients with or without del (5q) [19-23]. In phase 1/2 studies, 46–65% of lower-risk thrombocytopenic MDS patients receiving romiplostim achieved an International Working Group (IWG)-defined platelet response [19,20,24]. "
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    ABSTRACT: Lenalidomide treatment in myelodysplastic syndrome (MDS) may lead to thrombocytopenia and dose reductions/delays. This study evaluated the safety and tolerability of the thrombopoietin mimetic romiplostim and its effects on the incidence of clinically significant thrombocytopenic events (CSTEs) in lower risk MDS patients receiving lenalidomide. Patients were assigned to weekly placebo (n = 12) or romiplostim 500 μg (n = 14) or 750 μg (n = 13) for four 28-day lenalidomide cycles. The treatment groups were generally similar with respect to baseline disease characteristics. Del(5q) abnormalities were noted in 1 (8%) patient in the placebo group, 3 (21%) in the romiplostim 500 μg group, and two (15%) in the 750 μg group. CSTEs were noted in 8 (67%) patients in the placebo group, 4 (29%) in the romiplostim 500 μg group, and 8 (62%) in the romiplostim 750 μg group. Throughout the study, median platelet counts trended lower in placebo-treated than in romiplostim-treated patients. Thrombocytopenia-related adjustments in lenalidomide occurred in 6 (50%) patients in the placebo group, 5 (36%) in the romiplostim 500 μg group, and 2 (15%) in the 750 μg group. Although the percentages of patients who received platelet transfusions were similar across treatment groups, there was a trend toward lower numbers of transfusions in both romiplostim groups during each treatment cycle. There were two serious treatment-related adverse events during the treatment period (cerebrovascular accident, placebo; worsening thrombocytopenia, romiplostim 500 μg). Two patients (romiplostim 500 and 750 μg, respectively) had an increase in bone marrow blasts to >20% during treatment, but had no post-treatment biopsy to confirm or exclude the diagnosis of progression to AML. These data suggest that romiplostim administered to MDS patients during lenalidomide treatment may decrease the frequency of dose reductions/delays due to thrombocytopenia. Additional study is needed to confirm the results of this preliminary trial. Trial registration NCT00418665
    Journal of Hematology & Oncology 11/2012; 5(1):71. DOI:10.1186/1756-8722-5-71 · 4.81 Impact Factor
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