Clinical trial · Observational
Observational Study on Eltrombopag in Hypocellular Myelodysplastic Neoplasms
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 18, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260918-000001
Summary
Brief summary (as posted)
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
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myelodysplastic Neoplasm | Myelodysplastic Syndrome | ALIAS | 0.90 |
| Myelodysplastic Syndrome | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Eltrombopag (ELT) | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- label
- Adult patients diagnosed with h-MDS according to WHO 2022 and treated with eltrombopag
- description
- Adult patients diagnosed with h-MDS according to WHO 2022 and treated with eltrombopag included in FISiM registry * IPSS-R very-low to intermediate * De novo or therapy related Available bone marrow sample for central revision
- interventionNames
- Drug: Eltrombopag (ELT)
Primary outcomes (1)
- measure
- Overall Response Rate (ORR)
- timeFrame
- From initiation of eltrombopag treatment through the last available response assessment, up to study completion.
- description
- Overall response rate to eltrombopag according to the International Working Group (IWG) response criteria (Cheson 2006)
Secondary outcomes (6)
- measure
- Overall Response Rate (ORR) of eltrombopag combined with immunosuppressive drugs
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Adult patients (i.e. aged ≥18 years) diagnosed with MDS-h according to WHO 2022 * IPSS-R risk very-low to intermediate * De novo or therapy related MDS * Availability of bone marrow sample for central revision * Treatment with eltrombopag, in monotherapy or associated with IST, as first -line therapy or as a subsequent therapy to the first line Exclusion Criteria: * Unavailability of bone marrow sample for central revision
References
Publications (0)
Data not yet available