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Multicenter Study
. 2020 May 12;4(9):1965-1973.
doi: 10.1182/bloodadvances.2019001084.

Survival following allogeneic transplant in patients with myelofibrosis

Krisstina Gowin  1 Karen Ballen  2 Kwang Woo Ahn  3   4 Zhen-Huan Hu  3 Haris Ali  5 Murat O Arcasoy  6 Rebecca Devlin  7 Maria Coakley  8 Aaron T Gerds  9 Michael Green  6 Vikas Gupta  10 Gabriela Hobbs  11 Tania Jain  12 Malathi Kandarpa  13 Rami Komrokji  14 Andrew T Kuykendall  14 Kierstin Luber  15 Lucia Masarova  16 Laura C Michaelis  17 Sarah Patches  18 Ashley C Pariser  19 Raajit Rampal  12 Brady Stein  19 Moshe Talpaz  16 Srdan Verstovsek  16 Martha Wadleigh  18 Vaibhav Agrawal  20 Mahmoud Aljurf  21 Miguel Angel Diaz  22 Belinda R Avalos  23 Ulrike Bacher  24 Asad Bashey  25 Amer M Beitinjaneh  26 Jan Cerny  27 Saurabh Chhabra  3 Edward Copelan  28 Corey S Cutler  29 Zachariah DeFilipp  30 Shahinaz M Gadalla  31 Siddhartha Ganguly  32 Michael R Grunwald  23 Shahrukh K Hashmi  21   33 Mohamed A Kharfan-Dabaja  34 Tamila Kindwall-Keller  2 Nicolaus Kröger  35 Hillard M Lazarus  36 Jane L Liesveld  37 Mark R Litzow  38 David I Marks  39 Sunita Nathan  40 Taiga Nishihori  41 Richard F Olsson  42   43 Attaphol Pawarode  44 Jacob M Rowe  45 Bipin N Savani  46 Mary Lynn Savoie  47 Sachiko Seo  48 Melhem Solh  49 Roni Tamari  12 Leo F Verdonck  50 Jean A Yared  51 Edwin Alyea  52 Uday Popat  16 Ronald Sobecks  53 Bart L Scott  54 Ryotaro Nakamura  55 Ruben Mesa  56 Wael Saber  3
Affiliations
Multicenter Study

Survival following allogeneic transplant in patients with myelofibrosis

Krisstina Gowin et al. Blood Adv. .

Erratum in

Abstract

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.

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Conflict of interest statement

Conflict-of-interest disclosure: A.T.K. received research funding from Incyte, AbbVie, Janssen, and Colgene. A.T.G. received research funding from Celgene, Incyte, CTI Biopharma, and Pfizer. G.H. received research funding from Bayer, Meirck, Incyte, and Celgene. R.F.O. received research funding from AstraZeneca. Z.D. received research funding from Incyte Corp. M.O.A. received research funding from Incyte, Gilead, Samus Therapeautics, CTI Biopharma, and Janssen. B.R.A. contributed to Best Practice guidelines for the British Med J and received research funding from Juno. V.G. received research funding from Novartis, Celgene, Sierra Oncology, and Incyte. L.C.M. received research funding from ASTEX, Bioline, BMS, Celgene, Janssen, Jazz, Macrogenics, Millennium, Novartis, Pfizer, and TG Therapeutics. J.C. received research funding from Inctye, Daichi-Sankyo, and Jaze Pharmaceuticals. K.G. received consultancy fees from and is a member of the scientific advisory board for Incyte Corporation. R.N. received research funding from Alexion, Celgene, Kirin Kyowa, and Merck. M.T. received research funding from Celgene, CTI Biopharma, Imago, and Constellation. S.V. received research funding from Incyte Corporation, Roche, NS Pharma, Celgene, Gilead, Promisor, CTI BioPharma, Genentech, Blueprint Medicines, Novartis, Sierra Oncology, Pharma Essentia, AstraZeneca, Ital Pharma, and Protagonist Therapeutics. M.G. received consultancy fees from Incyte, Amgen, AbbVie, Astellas, BMS/Celgene, Merck, Pfizer, Premier, Trovagene, Daiichi Sankyo, and Cardinal Health and research funding from Incyte, Amgen, Novartis, Janssen, Genentech/Roche, and Forma Therapeutics. T.J. received consultancy fees from Takeda Oncology and is a member of the CareDx advisory board. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Survival probabilities for the DIPSS risk groups in MF receiving HCT vs non-HCT therapy. (A) DIPSS low risk. (B) DIPSS Int-1. (C) DIPSS Int-2 or higher. (D) Overall (all DIPSS groups). The survival curves presented here, stratified by DIPSS risk score, are a representation of the interventions (ie, HCT vs non-HCT therapy) over a median follow-up of ∼6 years. The curves cross much later in the clinical course than 12 months; however, the slope of the curves changes much earlier (12 months) and then plateaus, indicating the OS benefit associated with HCT begins much earlier than when the curves actually cross. A long-term survival advantage with HCT was observed for patients with Int-1 or higher risk MF, but at the cost of early mortality. The magnitude of OS benefit increased as DIPSS risk score increased.

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