Prognostic stratification in MDS/MPN syndromes other than CMML: an Italian multicenter real-world study
Abstract MDS/MPN syndromes comprise a heterogeneous group of myeloid neoplasms. While CMML has been extensively characterized, data on other MDS/MPN entities remain limited, particularly in real-world settings. We conducted a nationwide, multicenter retrospective study including adult patients with MDS/MPN syndromes other than CMML from 17 Italian centers. Patients were classified as MDS/MPN with SF3B1 mutation and thrombocytosis ( SF3B1 -T), atypical chronic myeloid leukemia/chronic neutrophilic leukemia (aCML/CNL), or MDS/MPN not otherwise specified (NOS). A total of 101 patients were included (median age 71 years). MDS/MPN SF3B1 -T showed a significant survival advantage compared with MDS/MPN NOS and aCML/CNL (OS p = 0.0058, LFS p = 0.012). aCML/CNL displayed the worst outcomes and most genetic complexity. Across the overall cohort, IPSS-M demonstrated the highest discriminative ability and calibration accuracy for both OS and LFS, outperforming CMML-directed models, including BLAST-clinical and BLAST-molecular whereas disease-specific scores provided optimal stratification within individual entities but failed to adequately predict outcomes in MDS/MPN NOS. In this real-world analysis, IPSS-M emerged as the most robust prognostic framework across MDS/MPN entities other than CMML. The inability of current tools to reliably stratify patients with MDS/MPN NOS underscores the biological heterogeneity of this category and highlights the urgent need for integrated clinical and genomic prognostic models.
Authors
- Luca Guarnera (ORCID: https://orcid.org/0000-0001-8293-0663)
- Michelina Santopietro (ORCID: https://orcid.org/0000-0001-6979-8186)
- Francesco Muriano (ORCID: https://orcid.org/0009-0001-4737-4451)
- Roberto Latagliata (ORCID: https://orcid.org/0000-0002-7741-862X)
- Paola Guglielmelli (ORCID: https://orcid.org/0000-0003-1809-284X)
- Luca Maurillo (ORCID: https://orcid.org/0000-0001-7297-4988)
- Lorenzo Rizzo (ORCID: https://orcid.org/0000-0002-8778-8317)
- Caterina Tatarelli
- Mara Memoli (ORCID: https://orcid.org/0000-0002-1922-6048)
- Alessandro M. Vannucchi (ORCID: https://orcid.org/0000-0001-5755-0730)
- Ambra Di Veroli (ORCID: https://orcid.org/0000-0002-2876-4833)
- Giovanni Iaquinta (ORCID: https://orcid.org/0009-0004-6616-5884)
- Antonio Spadea (ORCID: https://orcid.org/0000-0002-3651-2951)
- Maria Teresa Voso (ORCID: https://orcid.org/0000-0002-6164-4761)
- Federica Catania (ORCID: https://orcid.org/0000-0002-0443-5197)
- Paola Grammatico (ORCID: https://orcid.org/0000-0002-6864-2574)
- Anna Lina Piccioni
- Sabrina Leonetti Crescenzi
- Costanza Andriola
- Francesca Romana Iovene
- Sabrina Mariani (ORCID: https://orcid.org/0009-0004-1448-8112)
- Giuseppe Gaetano Loscocco (ORCID: https://orcid.org/0000-0002-6241-1206)
- Prassede Salutari
- Katia Paciaroni
- Emilia Scalzulli
- Alessandro Andriani
- Adriano Venditti
- Marta Riva
- Massimo Breccia
- Emiliano Fabiani
- Gianluca Albanese
- Bruno Fattizzo
- Annalisa Biagi
- Novella Pugliese
- Alfredo Molteni
Institutions
- University of Rome Tor Vergata (IT)
- Università Campus Bio-Medico (IT)
- University of Milan (IT)
- Azienda Ospedaliera San Giovanni Addolorata (IT)
- Policlinico Umberto I (IT)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- ASL Roma (IT)
- Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT)
- Saint Camillus International University of Health and Medical Sciences (IT)
- Federico II University Hospital (IT)
- Azienda Ospedaliera Sant'Andrea (IT)
- Ospedale di Belcolle (IT)
- Villa Pineta Hospital (IT)
- Ospedale Santa Maria Goretti (IT)
- Azienda USL di Pescara (IT)
- Carlo Forlanini Hospital (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Azienda Sanitaria Locale Roma 3 (IT)
- Villa Torri Hospital (IT)
- Istituti Ospitalieri di Cremona (IT)
- Azienda Socio Sanitaria Territoriale di Cremona
- University of Florence (IT)
- European Institute of Oncology (IT)
Publication Details
- Journal
- Clinical and Experimental Medicine
- Published
- 2026-09-01
- DOI
- https://doi.org/10.1007/s10238-026-02287-0
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Università degli Studi di Roma Tor Vergata