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Observational Study on Eltrombopag in Hypocellular Myelodysplastic Neoplasms
Sponsor: Fondazione Italiana Sindromi Mielodisplastiche-ETS
Summary
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
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
100
Start Date
2026-05-01
Completion Date
2027-07-31
Last Updated
2026-09-17
Healthy Volunteers
No
Interventions
Eltrombopag (ELT)
Eltrombopag administered as part of routine clinical practice for the treatment of hypocellular myelodysplastic neoplasms. The study retrospectively and prospectively evaluates the effectiveness and safety of eltrombopag in patients treated with eltrombopag monotherapy or in combination with immunosuppressive therapy.
Locations (26)
SCDU di Ematologia, AO SS. Antonio e Biagio e Cesare Arrigo
Alessandria, Italy
UO Ematologia con Trapianto, Dipartimento di Medicina di Precisione e Rigenerativa e Area Ionica, AOU Consorziale Policlinico, Università degli Studi Aldo Moro
Bari, Italy
Istituto di Ematologia L. e A. Seràgnoli, Policlinico di S. Orsola, IRCCS AOU di Bologna
Bologna, Italy
Comprehensive Cancer Center, Spedali Civili di Brescia
Brescia, Italy
Oncologia Medica, INOC
Candiolo, Italy
MDS Unit, AOU Careggi
Florence, Italy
UO Ematologia e Terapie Cellulari, IRCCS Ospedale Policlinico San Martino
Genova, Italy
SC Immunoematologia e Medicina Trasfusionale, Ospedale Carlo Poma, ASST di Mantova
Mantua, Italy
Hematology Unit, IRCCS Istituto Romagnolo per lo Studio dei Tumori Dino Amadori
Meldola (FC), Italy
Oncoematologia, PO Fatebenefratelli, ASST Fatebenefratelli Sacco
Milan, Italy
SC Medicina II, PO San Paolo, ASST Santi Paolo e Carlo
Milan, Italy
Unità di Ematologia e TMO, IRCCS Ospedale San Raffaele
Milan, Italy
UOC Ematologia, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Milan, Italy
UOC Ematologia, Dipartimento di Medicina Clinica e Chirurgia, Università di Napoli Federico II
Naples, Italy
SCDU Medicina interna ad indirizzo ematologico, AOU San Luigi Gonzaga, Università degli Studi di Torino
Orbassano, Italy
UO Ematologia e Immunologia clinica, Dipartimento di Medicina, AOU di Padova
Padova, Italy
UOC Ematologia e CTMO, AOU Parma
Parma, Italy
UO Ematologia e CTMO, Ospedale Guglielmo da Saliceto
Piacenza, Italy
UOC Ematologia, Ospedale S. Eugenio, ASL Roma 2
Roma, Italy
Ematologia, Dipartimento di Medicina, Chirurgia e Farmacia, Università degli Studi di Sassari
Sassari, Italy
SC Ematologia U, AOU Città della Salute e della Scienza, Università degli Studi di Torino
Torino, Italy
SC Ematologia, Dipartimento di Oncologia, AOU Città della Salute e della Scienza
Torino, Italy
SCDU Ematologia e Terapie cellulari, AO Ordine Mauriziano, Università degli Studi di Torino
Torino, Italy
UOC Ematologia e TMO, Pia Fondazione di Culto e Religione Card. G. Panico
Tricase, Italy
UOC Ematologia, Ospedale Maggiore, ASU Giuliano Isontina
Trieste, Italy
Hematology Unit, Ospedale di Circolo e Fondazione Macchi, ASST Sette Laghi
Varese, Italy