Carobbio, Alessandra, Guglielmelli, Paola ORCID: 0000-0003-1809-284X, Rumi, Elisa, Cavalloni, Chiara, De Stefano, Valerio ORCID: 0000-0002-5178-5827, Betti, Silvia, Rambaldi, Alessandro ORCID: 0000-0002-3739-7502, Finazzi, Maria Chiara, Thiele, Juergen, Vannucchi, Alessandro M., Tefferi, Ayalew and Barbui, Tiziano (2020). A multistate model of survival prediction and event monitoring in prefibrotic myelofibrosis. Blood Cancer J., 10 (10). LONDON: SPRINGERNATURE. ISSN 2044-5385

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Abstract

Among 382 patients with WHO-defined prefibrotic myelofibrosis (pre-PMF) followed for a median of 6.9 years, fibrotic or leukemic transformation or death accounts for 15, 7, and 27% of cases, respectively. A multistate model was applied to analyze survival data taking into account intermediate states that are part of the clinical course of pre-PMF, including overt PMF and acute myeloid leukemia (AML). Within this multistate framework, multivariable models disclosed older age (>65 years) and leukocytosis (>15 x 10(9)/L) as predictors of death and leukemic transformation. The risk factors for fibrotic progression included anemia and grade 1 bone marrow fibrosis. The outcome was further affected by high molecular risk (HMR) but not driver mutations. Direct transition to overt PMF, AML, or death occurred in 15.2, 4.7, and 17.3% of patients, respectively. The risk of AML was the highest in the first 5 years (7%), but leveled off thereafter. Conversely, the probability of death from overt PMF or AML increased more rapidly over time, especially when compared to death in the pre-PMF state without disease progression. The probability of being alive with pre-PMF status decreased to 70 and 30% at 10 and 20 years, respectively. This study highlights the aspects of the clinical course and estimates of disease progression in pre-PMF.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Carobbio, AlessandraUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Guglielmelli, PaolaUNSPECIFIEDorcid.org/0000-0003-1809-284XUNSPECIFIED
Rumi, ElisaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cavalloni, ChiaraUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
De Stefano, ValerioUNSPECIFIEDorcid.org/0000-0002-5178-5827UNSPECIFIED
Betti, SilviaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Rambaldi, AlessandroUNSPECIFIEDorcid.org/0000-0002-3739-7502UNSPECIFIED
Finazzi, Maria ChiaraUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Thiele, JuergenUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Vannucchi, Alessandro M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Tefferi, AyalewUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Barbui, TizianoUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-314998
DOI: 10.1038/s41408-020-00368-1
Journal or Publication Title: Blood Cancer J.
Volume: 10
Number: 10
Date: 2020
Publisher: SPRINGERNATURE
Place of Publication: LONDON
ISSN: 2044-5385
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
WORLD-HEALTH-ORGANIZATION; ESSENTIAL THROMBOCYTHEMIA; MYELOID NEOPLASMS; CLASSIFICATION; DIAGNOSIS; REVISIONMultiple languages
Oncology; HematologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/31499

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