Division of Hematology Oncology, Department of Medicine
Clinical Trials | Health Services Administration | Health Services Research | Hematology | Hemic and Lymphatic Diseases | Neoplasms | Oncology
Allogeneic hematopoietic cell transplantation (HCT) is the only curative therapy for myelofibrosis (MF). In this large multicenter retrospective study, overall survival (OS) in MF patients treated with allogeneic HCT (551 patients) and without HCT (non-HCT) (1377 patients) was analyzed with Cox proportional hazards model. Survival analysis stratified by the Dynamic International Prognostic Scoring System (DIPSS) revealed that the first year of treatment arm assignment, due to upfront risk of transplant-related mortality (TRM), HCT was associated with inferior OS compared with non-HCT (non-HCT vs HCT: DIPSS intermediate 1 [Int-1]: hazard ratio [HR] = 0.26, P < .0001; DIPSS-Int-2 and higher: HR, 0.39, P < .0001). Similarly, in the DIPSS low-risk MF group, due to upfront TRM risk, OS was superior with non-HCT therapies compared with HCT in the first-year post treatment arm assignment (HR, 0.16, P = .006). However, after 1 year, OS was not significantly different (HR, 1.38, P = .451). Beyond 1 year of treatment arm assignment, an OS advantage with HCT therapy in Int-1 and higher DIPSS score patients was observed (non-HCT vs HCT: DIPSS-Int-1: HR, 2.64, P < .0001; DIPSS-Int-2 and higher: HR, 2.55, P < .0001). In conclusion, long-term OS advantage with HCT was observed for patients with Int-1 or higher risk MF, but at the cost of early TRM. The magnitude of OS benefit with HCT increased as DIPSS risk score increased and became apparent with longer follow-up.
clinical trials and observations, Myeloid Neoplasia, transplantation, myelofibrosis, follow-up
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DOI of Published Version
Gowin K, Ballen K, Ahn KW, Hu ZH, Ali H, Arcasoy MO, Devlin R, Coakley M, Gerds AT, Green M, Gupta V, Hobbs G, Jain T, Kandarpa M, Komrokji R, Kuykendall AT, Luber K, Masarova L, Michaelis LC, Patches S, Pariser AC, Rampal R, Stein B, Talpaz M, Verstovsek S, Wadleigh M, Agrawal V, Aljurf M, Angel Diaz M, Avalos BR, Bacher U, Bashey A, Beitinjaneh AM, Cerny J, Chhabra S, Copelan E, Cutler CS, DeFilipp Z, Gadalla SM, Ganguly S, Grunwald MR, Hashmi SK, Kharfan-Dabaja MA, Kindwall-Keller T, Kröger N, Lazarus HM, Liesveld JL, Litzow MR, Marks DI, Nathan S, Nishihori T, Olsson RF, Pawarode A, Rowe JM, Savani BN, Savoie ML, Seo S, Solh M, Tamari R, Verdonck LF, Yared JA, Alyea E, Popat U, Sobecks R, Scott BL, Nakamura R, Mesa R, Saber W. Survival following allogeneic transplant in patients with myelofibrosis. Blood Adv. 2020 May 12;4(9):1965-1973. doi: 10.1182/bloodadvances.2019001084. PMID: 32384540; PMCID: PMC7218417. Link to article on publisher's site
Gowin K, Cerny J. (2020). Survival following allogeneic transplant in patients with myelofibrosis. Open Access Articles. https://doi.org/10.1182/bloodadvances.2019001084. Retrieved from https://escholarship.umassmed.edu/oapubs/4234