Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms – results of the EUROPE trial by EMSCO

Kubasch, Anne Sophie; Giagounidis, Aristoteles; Metzgeroth, Georgia; Jonášová, Anna; Herbst, Regina; Diaz, José Miguel Torregrosa; Renzis, Benoît de; Götze, Katharina; Huetter-Kroenke, Marie-Luise; Gourin, Marie-Pierre; Slama, Borhane; Dimicoli‐Salazar, Sophie; Cony‐Makhoul, Pascale; Laribi, Kamel; Park, Sophie; Jersemann, Katja; Schipp, Dorothea; Metzeler, Klaus H.; Tiebel, Oliver; Sockel, Katja; Gloaguen, Silke; Mies, Anna; Chermat, Fatiha; Thiede, Christian; Sapena, Rosa; Schlenk, Richard F.; Fenaux, Pierre; Platzbecker, Uwe; Adès, Lionel

doi:10.1038/s41375-022-01669-z

Cited by 3 publications

(2 citation statements)

References 38 publications

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“…Five-year followup for transformation to AML and death did not differ between the 2 groups [29]. A subsequent trial of romiplostim in LR MDS with <5% marrow blasts demonstrated a hematologic improvement-platelet response (HI-P) rate of 42% with a median response duration of 48.5 weeks [30]. Predictors of response were SRSF2 mutation status and baseline hemoglobin levels, but not endogenous TPO levels or platelet transfusion history.…”

Section: Therapeutic Optionsmentioning

confidence: 92%

Management of Patients with Lower-Risk Myelodysplastic Neoplasms (MDS)

2023

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Myelodysplastic neoplasms (MDS) are a heterogenous group of clonal hematologic disorders characterized by morphologic dysplasia, ineffective hematopoiesis, and cytopenia. In the past year, the classification of MDS has been updated in the 5th edition of the World Health Organization (WHO) Classification of Haematolymphoid Tumours and the International Consensus Classification (ICC) of Myeloid Neoplasms and Acute Leukemia with incorporation of morphologic, clinical, and genomic data. Furthermore, the more comprehensive International Prognostic Scoring System-Molecular (IPSS-M) allows for improved risk stratification and prognostication. These three developments allow for more tailored therapeutic decision-making in view of the expanding treatment options in MDS. For patients with lower risk MDS, treatment is aimed at improving cytopenias, usually anemia. The recent approval of luspatercept and decitabine/cedazuridine have added on to the current armamentarium of erythropoietic stimulating agents and lenalidomide (for MDS with isolated deletion 5q). Several newer agents are being evaluated in phase 3 clinical trials for this group of patients, such as imetelstat and oral azacitidine. This review provides a summary of the classification systems, the prognostic scores and clinical management of patients with lower risk MDS.

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Section: Therapeutic Optionsmentioning

confidence: 92%

Management of Patients with Lower-Risk Myelodysplastic Neoplasms (MDS)

2023

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“…9,19 Notably, in low-risk MDS, SRSF2 may predict response to romiplostim. 20 Our study is limited by the small sample size and its retrospective nature. While all patients did have pathogenic SRSF2 mutations with significant VAFs, we cannot conclude that this genetic abnormality defines CCUS-IT.…”

Section: Wementioning

confidence: 94%

Clinical and pathological features of clonal cytopenia of undetermined significance presenting with isolated thrombocytopenia (CCUS‐IT)

O'Neill,

Nwachukwu,

Vergara‐Lluri

et al. 2023

European J of Haematology

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BackgroundClonal cytopenia of undetermined significance (CCUS) is defined as somatic mutations of myeloid malignancy‐associated genes in the blood or bone marrow with one or more persistent unexplained cytopenias that do not meet diagnostic criteria for a defined myeloid neoplasm. CCUS with isolated thrombocytopenia (CCUS‐IT) is rare.MethodsThis is a retrospective case series of patients with prolonged isolated thrombocytopenia, a pathogenic mutation on a myeloid molecular panel, and a bone marrow biopsy with morphologic atypia below the WHO‐defined diagnostic threshold for dysplasia.ResultsFive male patients were identified with a median age at CCUS‐IT diagnosis of 61 years (56–74). Median duration of thrombocytopenia prior to CCUS‐IT diagnosis was 4 years (3–12), and median platelet count at CCUS‐IT diagnosis was 41 × 103/μL (26–80). All patients had megakaryocytic hyperplasia and megakaryocytes with hyperchromasia and high nuclear‐cytoplasmic ratio. Pathogenic SRSF2 mutations were identified in all 5 patients with median variant allele frequency of 36% (28%–50%). Three patients were treated with IVIg and/or steroids with no response; one of three responded to thrombopoietin receptor agonists. Three patients progressed to MDS and one to AML.DiscussionWe describe the clinicopathological features of CCUS‐IT which can mimic immune thrombocytopenia.

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Myelodysplastische Neoplasien (MDS)

Kubasch,

Platzbecker

2024

Facharztwissen Hamatologie Onkologie

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Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms – results of the EUROPE trial by EMSCO

Cited by 3 publications

References 38 publications

Management of Patients with Lower-Risk Myelodysplastic Neoplasms (MDS)

Management of Patients with Lower-Risk Myelodysplastic Neoplasms (MDS)

Clinical and pathological features of clonal cytopenia of undetermined significance presenting with isolated thrombocytopenia (CCUS‐IT)

Myelodysplastische Neoplasien (MDS)

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