Observational Study on Eltrombopag in Hypocellular Myelodysplastic Neoplasms

Brief summary (registry, in English)

Hypocellular myelodysplastic neoplasms (h-MDS) are a distinct entity introduced in the 2022 WHO classification. They are characterized by reduced bone marrow cellularity and may share several clinical, morphological, immunological, and molecular features with aplastic anemia (AA), making the differential diagnosis between the two conditions challenging. Eltrombopag, a thrombopoietin receptor agonist, has demonstrated efficacy in patients with severe AA and has also shown activity in lower-risk MDS, with bone marrow hypocellularity identified as a potential predictor of response. In Italy, eltrombopag has been used off-label in patients with h-MDS and clinically relevant cytopenias. However, systematic real-world data on its effectiveness and safety in this patient population, either as monotherapy or in combination with immunosuppressive therapy (IST), remain limited. This is an observational study. We plan to analyse all patients with h-MDS consecutively diagnosed and included in the FISIM registry in 26 Italian centres. Study duration: 12 months Data to be collected: demographic, disease characteristics at diagnosis (blood count, morphology, histopathology, cytogenetics, multiparameter flow cytometry, presence of PNH clone, molecular data), response to treatment according to IWG criteria, progression to AML rate and survival. Centralized revision of bone marrow biopsy: bone marrow biopsy at diagnosis will be centrally reviewed

What you commit to

• Study type: Observational
• Planned participants: 100
• From 1 May 2026 to the primary completion date, 30 Nov 2026
• 26 sites in 1 country
• Visits and tests: ask the trial team, the registry does not list them

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Who cannot take part

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Who the study is looking for

Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (26)

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