Romiplostim in Low/INT-1-Risk MDS Results in Reduced Bleeding without Impacting Leukemic Progression: Final Results from a Randomized, Double-Blind, Placebo-Controlled Study

Kantarjian, H.; Fenaux, Pierre; Sekeres, Mikkael A.; Szer, Jeffrey; Platzbecker, Uwe; Kuendgen, Andrea; Gaidano, G; Jedrzejczak, W.W.; Carpenter, Nancy; Mehta, Bhakti; Franklin, Janet; Giagounidis, Aristoteles

doi:10.1016/s0145-2126(17)30136-4

Cited by 4 publications

(3 citation statements)

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“…Earlier findings with romiplostim in patients with MDS showed increased bone marrow blast counts, and suggested a potential risk of AML progression . More recently, it was reported that patients with low‐risk MDS who received romiplostim for 58 weeks did not have significantly higher AML progression rates 5 years after discontinuation, indicating that the effects of romiplostim on blast counts and, potentially, AML progression are largely reversible . In contrast, clinical studies in patients with either low‐risk or high‐risk MDS did not show an increased risk of disease progression in those receiving eltrombopag monotherapy as compared with those receiving placebo .…”

Section: Safety Considerationsmentioning

confidence: 97%

Thrombopoietin receptor agonists in hereditary thrombocytopenias

Rodeghiero

Pecci

Balduini

2018

Journal of Thrombosis and Haemostasis

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Hereditary thrombocytopenias (HTPs) constitute a heterogeneous group of diseases characterized by a reduction in platelet count and a potential bleeding risk. As a result of advances in diagnostic methods, HTPs are increasingly being identified, and appear to be less rare than previously thought. Most HTPs do not have effective treatments, except for platelet transfusion when bleeding occurs and in preparation for procedures associated with a risk of bleeding. Preliminary clinical evidence suggests that thrombopoietin receptor agonists (TPO-RAs) with an established use in the treatment of certain acquired thrombocytopenias are well tolerated and provide clinical benefits in patients with some forms of HTP. These drugs may therefore be considered for the treatment of HTPs in clinical practice. However, caution and close monitoring are recommended, owing to the absence of long-term safety data and the potential risks posed by prolonged bone marrow stimulation in certain HTPs. In this review, we summarize the available clinical data on TPO-RAs in the treatment of HTPs, and discuss their use in patients with these disorders. We believe that TPO-RAs will play a major role in the treatment of HTPs, particularly myosin heavy chain 9-related disease, Wiskott-Aldrich syndrome, X-linked thrombocytopenia, and thrombocytopenia caused by THPO mutations.

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Section: Safety Considerationsmentioning

confidence: 97%

Thrombopoietin receptor agonists in hereditary thrombocytopenias

Rodeghiero

Pecci

Balduini

2018

Journal of Thrombosis and Haemostasis

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“…There are other publications that do not find a pro-leukemic effect [96]. In more recent MDS studies, TRAs had no effect on the rate of leukemic transformations [97]. Some authors recommend a bone marrow biopsy to exclude MDS (and fibrosis, see below) before the administration of TRAs.…”

Section: Tras -Side Effectsmentioning

confidence: 99%

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

Matzdorff¹,

Meyer²,

Ostermann³

et al. 2018

Oncol Res Treat

108

156

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“…The TPO-RA romiplostim has been studied in a double-blind, randomized, placebo-controlled clinical trial, the final long-term results of which were presented earlier this year. 40 Due to an unfortunate data monitoring committee decision, the trial was terminated early before recruitment of all patients had taken place, and robust conclusions cannot be drawn as to romiplostim's definite role in this situation. When the data monitoring committee met, the data suggested that more AMLs had happened in the romiplostim arm of the study.…”

Section: Patients With Predominant Thrombocytopeniamentioning

confidence: 99%

Current treatment algorithm for the management of lower-risk MDS

Giagounidis

2017

Hematology

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Lower risk myelodysplastic syndromes (MDS), defined as MDS with a Revised International Prognostic Scoring System score ≤3.5 points, will remain a challenging entity in 2018. Supportive care continues to be the linchpin of treatment, although the options to reduce transfusion needs are broadening. To achieve red blood cell transfusion independence in non-del(5q) patients, erythropoiesis-stimulating agents remain a mainstay of therapy as long as endogenous erythropoietin levels are <500 U/L (and preferably <200 U/L). Experimental strategies for patients ineligible for erythropoiesis-stimulating agents or relapsing after gaining transfusion independence include immunosuppressive agents, transforming growth factor β inhibitors, and lenalidomide. All these alternatives have shown reasonable response rates in selected patient populations with lower risk MDS. Patients with del(5q) disease can derive long-term benefit from lenalidomide, and some patients remain transfusion free for extended periods even after discontinuation of the drug. In rare cases in which thrombocytopenia is the main clinical problem leading to clinically significant bleeding events, thrombopoietin receptor analogues may alleviate bleeding, increase platelet counts, and rarely lead to trilineage responses. It seems prudent to use these drugs only in patients with confirmed bone marrow blast counts <5%. Allogeneic stem cell transplantation is reasonable for patients with high molecular risk of progression and those failing several lines of treatment with signs of progression toward higher-risk MDS.

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Romiplostim in Low/INT-1-Risk MDS Results in Reduced Bleeding without Impacting Leukemic Progression: Final Results from a Randomized, Double-Blind, Placebo-Controlled Study

Cited by 4 publications

References 0 publications

Thrombopoietin receptor agonists in hereditary thrombocytopenias

Thrombopoietin receptor agonists in hereditary thrombocytopenias

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

Current treatment algorithm for the management of lower-risk MDS

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