Survival following allogeneic transplant in patients with myelofibrosis

Krisstina Gowin; Karen Ballen; Kwang Woo Ahn; Zhen Huan Hu; Haris Ali; Murat O. Arcasoy; Rebecca Devlin; Maria Coakley; Aaron T. Gerds; Michael Green; Vikas Gupta; Gabriela Hobbs; Tania Jain; Malathi Kandarpa; Rami Komrokji; Andrew T. Kuykendall; Kierstin Luber; Lucia Masarova; Laura C. Michaelis; Sarah Patches; Ashley C. Pariser; Raajit Rampal; Brady Stein; Moshe Talpaz; Srdan Verstovsek; Martha Wadleigh; Vaibhav Agrawal; Mahmoud Aljurf; Miguel Angel Diaz; Belinda R. Avalos; Ulrike Bacher; Asad Bashey; Amer M. Beitinjaneh; Jan Cerny; Saurabh Chhabra; Edward Copelan; Corey S. Cutler; Zachariah DeFilipp; Shahinaz M. Gadalla; Siddhartha Ganguly; Michael R. Grunwald; Shahrukh K. Hashmi; Mohamed A. Kharfan-Dabaja; Tamila Kindwall-Keller; Nicolaus Kröger; Hillard M. Lazarus; Jane L. Liesveld; Mark R. Litzow; David I. Marks; Sunita Nathan; Taiga Nishihori; Richard F. Olsson; Attaphol Pawarode; Jacob M. Rowe; Bipin N. Savani; Mary Lynn Savoie; Sachiko Seo; Melhem Solh; Roni Tamari; Leo F. Verdonck; Jean A. Yared; Edwin Alyea; Uday Popat; Ronald Sobecks; Bart L. Scott; Ryotaro Nakamura; Ruben Mesa; Wael Saber

doi:10.1182/bloodadvances.2019001084

Survival following allogeneic transplant in patients with myelofibrosis

Krisstina Gowin, Karen Ballen, Kwang Woo Ahn, Zhen Huan Hu, Haris Ali, Murat O. Arcasoy, Rebecca Devlin, Maria Coakley, Aaron T. Gerds, Michael Green, Vikas Gupta, Gabriela Hobbs, Tania Jain, Malathi Kandarpa, Rami Komrokji, Andrew T. Kuykendall, Kierstin Luber, Lucia Masarova, Laura C. Michaelis, Sarah PatchesAshley C. Pariser, Raajit Rampal, Brady Stein, Moshe Talpaz, Srdan Verstovsek, Martha Wadleigh, Vaibhav Agrawal, Mahmoud Aljurf, Miguel Angel Diaz, Belinda R. Avalos, Ulrike Bacher, Asad Bashey, Amer M. Beitinjaneh, Jan Cerny, Saurabh Chhabra, Edward Copelan, Corey S. Cutler, Zachariah DeFilipp, Shahinaz M. Gadalla, Siddhartha Ganguly, Michael R. Grunwald, Shahrukh K. Hashmi, Mohamed A. Kharfan-Dabaja, Tamila Kindwall-Keller, Nicolaus Kröger, Hillard M. Lazarus, Jane L. Liesveld, Mark R. Litzow, David I. Marks, Sunita Nathan, Taiga Nishihori, Richard F. Olsson, Attaphol Pawarode, Jacob M. Rowe, Bipin N. Savani, Mary Lynn Savoie, Sachiko Seo, Melhem Solh, Roni Tamari, Leo F. Verdonck, Jean A. Yared, Edwin Alyea, Uday Popat, Ronald Sobecks, Bart L. Scott, Ryotaro Nakamura, Ruben Mesa, Wael Saber

Research output: Contribution to journal › Article › peer-review

10 Scopus citations

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] 5 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 5 .006). However, after 1 year, OS was not significantly different (HR, 1.38, P 5 .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.

Original language	English (US)
Pages (from-to)	1966-1973
Number of pages	8
Journal	Blood Advances
Volume	4
Issue number	9
DOIs	https://doi.org/10.1182/bloodadvances.2019001084
State	Published - May 12 2020
Externally published	Yes

ASJC Scopus subject areas

Hematology

Access to Document

10.1182/bloodadvances.2019001084

Cite this

Gowin, K., Ballen, K., Ahn, K. W., Hu, Z. H., Ali, H., Arcasoy, M. O., Devlin, R., Coakley, M., Gerds, A. T., Green, M., Gupta, V., Hobbs, G., Jain, T., Kandarpa, M., Komrokji, R., Kuykendall, A. T., Luber, K., Masarova, L., Michaelis, L. C., ... Saber, W. (2020). Survival following allogeneic transplant in patients with myelofibrosis. Blood Advances, 4(9), 1966-1973. https://doi.org/10.1182/bloodadvances.2019001084

Survival following allogeneic transplant in patients with myelofibrosis. / Gowin, Krisstina; Ballen, Karen; Ahn, Kwang Woo; Hu, Zhen Huan; Ali, Haris; Arcasoy, Murat O.; Devlin, Rebecca; Coakley, Maria; Gerds, Aaron T.; Green, Michael; Gupta, Vikas; Hobbs, Gabriela; Jain, Tania; Kandarpa, Malathi; Komrokji, Rami; Kuykendall, Andrew T.; Luber, Kierstin; Masarova, Lucia; Michaelis, Laura C.; Patches, Sarah; Pariser, Ashley C.; Rampal, Raajit; Stein, Brady; Talpaz, Moshe; Verstovsek, Srdan; Wadleigh, Martha; Agrawal, Vaibhav; Aljurf, Mahmoud; Diaz, Miguel Angel; Avalos, Belinda R.; Bacher, Ulrike; Bashey, Asad; Beitinjaneh, Amer M.; Cerny, Jan; Chhabra, Saurabh; Copelan, Edward; Cutler, Corey S.; DeFilipp, Zachariah; Gadalla, Shahinaz M.; Ganguly, Siddhartha; Grunwald, Michael R.; Hashmi, Shahrukh K.; Kharfan-Dabaja, Mohamed A.; Kindwall-Keller, Tamila; Kröger, Nicolaus; Lazarus, Hillard M.; Liesveld, Jane L.; Litzow, Mark R.; Marks, David I.; Nathan, Sunita; Nishihori, Taiga; Olsson, Richard F.; Pawarode, Attaphol; Rowe, Jacob M.; Savani, Bipin N.; Savoie, Mary Lynn; Seo, Sachiko; Solh, Melhem; Tamari, Roni; Verdonck, Leo F.; Yared, Jean A.; Alyea, Edwin; Popat, Uday; Sobecks, Ronald; Scott, Bart L.; Nakamura, Ryotaro; Mesa, Ruben; Saber, Wael.

In: Blood Advances, Vol. 4, No. 9, 12.05.2020, p. 1966-1973.

Research output: Contribution to journal › Article › peer-review

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, Diaz, MA, 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 2020, 'Survival following allogeneic transplant in patients with myelofibrosis', Blood Advances, vol. 4, no. 9, pp. 1966-1973. https://doi.org/10.1182/bloodadvances.2019001084

Gowin, Krisstina ; Ballen, Karen ; Ahn, Kwang Woo ; Hu, Zhen Huan ; Ali, Haris ; Arcasoy, Murat O. ; Devlin, Rebecca ; Coakley, Maria ; Gerds, Aaron T. ; Green, Michael ; Gupta, Vikas ; Hobbs, Gabriela ; Jain, Tania ; Kandarpa, Malathi ; Komrokji, Rami ; Kuykendall, Andrew T. ; Luber, Kierstin ; Masarova, Lucia ; Michaelis, Laura C. ; Patches, Sarah ; Pariser, Ashley C. ; Rampal, Raajit ; Stein, Brady ; Talpaz, Moshe ; Verstovsek, Srdan ; Wadleigh, Martha ; Agrawal, Vaibhav ; Aljurf, Mahmoud ; Diaz, Miguel Angel ; Avalos, Belinda R. ; Bacher, Ulrike ; Bashey, Asad ; Beitinjaneh, Amer M. ; Cerny, Jan ; Chhabra, Saurabh ; Copelan, Edward ; Cutler, Corey S. ; DeFilipp, Zachariah ; Gadalla, Shahinaz M. ; Ganguly, Siddhartha ; Grunwald, Michael R. ; Hashmi, Shahrukh K. ; Kharfan-Dabaja, Mohamed A. ; Kindwall-Keller, Tamila ; Kröger, Nicolaus ; Lazarus, Hillard M. ; Liesveld, Jane L. ; Litzow, Mark R. ; Marks, David I. ; Nathan, Sunita ; Nishihori, Taiga ; Olsson, Richard F. ; Pawarode, Attaphol ; Rowe, Jacob M. ; Savani, Bipin N. ; Savoie, Mary Lynn ; Seo, Sachiko ; Solh, Melhem ; Tamari, Roni ; Verdonck, Leo F. ; Yared, Jean A. ; Alyea, Edwin ; Popat, Uday ; Sobecks, Ronald ; Scott, Bart L. ; Nakamura, Ryotaro ; Mesa, Ruben ; Saber, Wael. / Survival following allogeneic transplant in patients with myelofibrosis. In: Blood Advances. 2020 ; Vol. 4, No. 9. pp. 1966-1973.

@article{f9f589ea449c4037873d35535dbd0f73,

title = "Survival following allogeneic transplant in patients with myelofibrosis",

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] 5 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 5 .006). However, after 1 year, OS was not significantly different (HR, 1.38, P 5 .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.",

author = "Krisstina Gowin and Karen Ballen and Ahn, {Kwang Woo} and Hu, {Zhen Huan} and Haris Ali and Arcasoy, {Murat O.} and Rebecca Devlin and Maria Coakley and Gerds, {Aaron T.} and Michael Green and Vikas Gupta and Gabriela Hobbs and Tania Jain and Malathi Kandarpa and Rami Komrokji and Kuykendall, {Andrew T.} and Kierstin Luber and Lucia Masarova and Michaelis, {Laura C.} and Sarah Patches and Pariser, {Ashley C.} and Raajit Rampal and Brady Stein and Moshe Talpaz and Srdan Verstovsek and Martha Wadleigh and Vaibhav Agrawal and Mahmoud Aljurf and Diaz, {Miguel Angel} and Avalos, {Belinda R.} and Ulrike Bacher and Asad Bashey and Beitinjaneh, {Amer M.} and Jan Cerny and Saurabh Chhabra and Edward Copelan and Cutler, {Corey S.} and Zachariah DeFilipp and Gadalla, {Shahinaz M.} and Siddhartha Ganguly and Grunwald, {Michael R.} and Hashmi, {Shahrukh K.} and Kharfan-Dabaja, {Mohamed A.} and Tamila Kindwall-Keller and Nicolaus Kr{\"o}ger and Lazarus, {Hillard M.} and Liesveld, {Jane L.} and Litzow, {Mark R.} and Marks, {David I.} and Sunita Nathan and Taiga Nishihori and Olsson, {Richard F.} and Attaphol Pawarode and Rowe, {Jacob M.} and Savani, {Bipin N.} and Savoie, {Mary Lynn} and Sachiko Seo and Melhem Solh and Roni Tamari and Verdonck, {Leo F.} and Yared, {Jean A.} and Edwin Alyea and Uday Popat and Ronald Sobecks and Scott, {Bart L.} and Ryotaro Nakamura and Ruben Mesa and Wael Saber",

note = "Funding Information: The CIBMTR is supported primarily by Public Health Service grant/cooperative agreement U24CA076518 with the National Institutes of Health (NIH), National Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI); and National Institute of Allergy and Infectious Diseases (NHLBI and NCI grant/cooperative agreement U24HL138660; NHLBI grants OT3HL147741, R21HL140314, and U01HL128568; Health Resources and Services Administration [HRSA] contract HHSH250201700006C; Office of Naval Research grants N00014-18-1-2888 and N00014-17-1-2850; HRSA subaward from prime contract award SC1MC31881-01-00; NHLBI subawards from prime grant awards R01HL131731 and R01HL126589; NIH subawards from prime grant awards 5P01CA111412, 5R01HL129472, R01CA152108, 1R01HL131731, 1U01AI126612, and 1R01CA231141) and commercial funds from Actinium Pharmaceuticals, Adaptive Biotechnologies, Allovir, Amgen, and anonymous donation to the Medical College of Wisconsin, Anthem, Astellas Pharma US, Atara Biotherapeutics, BARDA, Be the Match Foundation, bluebird bio, Boston Children{\textquoteright}s Hospital, Bristol Myers Squibb, Celgene, Children{\textquoteright}s Hospital of Los Angeles, Chimerix, City of Hope Medical Center, CSL Behring, CytoSen Therapeutics, Inc., Daiichi Sankyo, Dana Farber Cancer Institute, Enterprise Science and Computing, Fred Hutchinson Cancer Research Center, Gamida-Cell, Genzyme, Gilead Sciences, GlaxoSmithKline, HistoGenetics, Immucor, Incyte Corporation, Janssen Biotech, Janssen Pharmaceuticals, Janssen Research & Development, Janssen Scientific Affairs, Japan Hematopoietic Cell Transplantation Data Center, Jazz Pharmaceuticals, Karius, Karyopharm Therapeutics, Kite (a Gilead Company), Kyowa Kirin, Magenta Therapeutics, Mayo Clinic and Foundation Rochester, Medac GmbH, Mediware, Memorial Sloan Kettering Cancer Center, Merck & Company, Mesoblast, MesoScale Diagnostics, Millennium, the Takeda Oncology Co., Miltenyi Biotec, Mundipharma EDO, National Marrow Donor Program, Novartis Oncology, Novartis Pharmaceuticals Corporation, Omeros Corporation, Oncoimmune, OptumHealth, Orca Bio-systems, PCORI, Pfizer, Phamacyclics, PIRCHE AG, Regen-eron Pharmaceuticals, REGiMMUNE, Sanofi Genzyme, Seattle Genetics, Shire, Sobi, Spectrum Pharmaceuticals, St. Baldrick{\textquoteright}s Foundation, Swedish Orphan Biovitrum, Takeda Oncology, The Medical College of Wisconsin, University of Minnesota, University of Pittsburgh, University of Texas MD Anderson Cancer Center, University of Wisconsin-Madison, Viracor Eurofins, and Xenikos BV. Funding Information: 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, Publisher Copyright: {\textcopyright} 2020 American Society of Hematology. All rights reserved.",

year = "2020",

month = may,

day = "12",

doi = "10.1182/bloodadvances.2019001084",

language = "English (US)",

volume = "4",

pages = "1966--1973",

journal = "Blood advances",

issn = "2473-9529",

publisher = "American Society of Hematology",

number = "9",

}

TY - JOUR

T1 - Survival following allogeneic transplant in patients with myelofibrosis

AU - Gowin, Krisstina

AU - Ballen, Karen

AU - Ahn, Kwang Woo

AU - Hu, Zhen Huan

AU - Ali, Haris

AU - Arcasoy, Murat O.

AU - Devlin, Rebecca

AU - Coakley, Maria

AU - Gerds, Aaron T.

AU - Green, Michael

AU - Gupta, Vikas

AU - Hobbs, Gabriela

AU - Jain, Tania

AU - Kandarpa, Malathi

AU - Komrokji, Rami

AU - Kuykendall, Andrew T.

AU - Luber, Kierstin

AU - Masarova, Lucia

AU - Michaelis, Laura C.

AU - Patches, Sarah

AU - Pariser, Ashley C.

AU - Rampal, Raajit

AU - Stein, Brady

AU - Talpaz, Moshe

AU - Verstovsek, Srdan

AU - Wadleigh, Martha

AU - Agrawal, Vaibhav

AU - Aljurf, Mahmoud

AU - Diaz, Miguel Angel

AU - Avalos, Belinda R.

AU - Bacher, Ulrike

AU - Bashey, Asad

AU - Beitinjaneh, Amer M.

AU - Cerny, Jan

AU - Chhabra, Saurabh

AU - Copelan, Edward

AU - Cutler, Corey S.

AU - DeFilipp, Zachariah

AU - Gadalla, Shahinaz M.

AU - Ganguly, Siddhartha

AU - Grunwald, Michael R.

AU - Hashmi, Shahrukh K.

AU - Kharfan-Dabaja, Mohamed A.

AU - Kindwall-Keller, Tamila

AU - Kröger, Nicolaus

AU - Lazarus, Hillard M.

AU - Liesveld, Jane L.

AU - Litzow, Mark R.

AU - Marks, David I.

AU - Nathan, Sunita

AU - Nishihori, Taiga

AU - Olsson, Richard F.

AU - Pawarode, Attaphol

AU - Rowe, Jacob M.

AU - Savani, Bipin N.

AU - Savoie, Mary Lynn

AU - Seo, Sachiko

AU - Solh, Melhem

AU - Tamari, Roni

AU - Verdonck, Leo F.

AU - Yared, Jean A.

AU - Alyea, Edwin

AU - Popat, Uday

AU - Sobecks, Ronald

AU - Scott, Bart L.

AU - Nakamura, Ryotaro

AU - Mesa, Ruben

AU - Saber, Wael

N1 - Funding Information: The CIBMTR is supported primarily by Public Health Service grant/cooperative agreement U24CA076518 with the National Institutes of Health (NIH), National Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI); and National Institute of Allergy and Infectious Diseases (NHLBI and NCI grant/cooperative agreement U24HL138660; NHLBI grants OT3HL147741, R21HL140314, and U01HL128568; Health Resources and Services Administration [HRSA] contract HHSH250201700006C; Office of Naval Research grants N00014-18-1-2888 and N00014-17-1-2850; HRSA subaward from prime contract award SC1MC31881-01-00; NHLBI subawards from prime grant awards R01HL131731 and R01HL126589; NIH subawards from prime grant awards 5P01CA111412, 5R01HL129472, R01CA152108, 1R01HL131731, 1U01AI126612, and 1R01CA231141) and commercial funds from Actinium Pharmaceuticals, Adaptive Biotechnologies, Allovir, Amgen, and anonymous donation to the Medical College of Wisconsin, Anthem, Astellas Pharma US, Atara Biotherapeutics, BARDA, Be the Match Foundation, bluebird bio, Boston Children’s Hospital, Bristol Myers Squibb, Celgene, Children’s Hospital of Los Angeles, Chimerix, City of Hope Medical Center, CSL Behring, CytoSen Therapeutics, Inc., Daiichi Sankyo, Dana Farber Cancer Institute, Enterprise Science and Computing, Fred Hutchinson Cancer Research Center, Gamida-Cell, Genzyme, Gilead Sciences, GlaxoSmithKline, HistoGenetics, Immucor, Incyte Corporation, Janssen Biotech, Janssen Pharmaceuticals, Janssen Research & Development, Janssen Scientific Affairs, Japan Hematopoietic Cell Transplantation Data Center, Jazz Pharmaceuticals, Karius, Karyopharm Therapeutics, Kite (a Gilead Company), Kyowa Kirin, Magenta Therapeutics, Mayo Clinic and Foundation Rochester, Medac GmbH, Mediware, Memorial Sloan Kettering Cancer Center, Merck & Company, Mesoblast, MesoScale Diagnostics, Millennium, the Takeda Oncology Co., Miltenyi Biotec, Mundipharma EDO, National Marrow Donor Program, Novartis Oncology, Novartis Pharmaceuticals Corporation, Omeros Corporation, Oncoimmune, OptumHealth, Orca Bio-systems, PCORI, Pfizer, Phamacyclics, PIRCHE AG, Regen-eron Pharmaceuticals, REGiMMUNE, Sanofi Genzyme, Seattle Genetics, Shire, Sobi, Spectrum Pharmaceuticals, St. Baldrick’s Foundation, Swedish Orphan Biovitrum, Takeda Oncology, The Medical College of Wisconsin, University of Minnesota, University of Pittsburgh, University of Texas MD Anderson Cancer Center, University of Wisconsin-Madison, Viracor Eurofins, and Xenikos BV. Funding Information: 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, Publisher Copyright: © 2020 American Society of Hematology. All rights reserved.

PY - 2020/5/12

Y1 - 2020/5/12

N2 - 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] 5 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 5 .006). However, after 1 year, OS was not significantly different (HR, 1.38, P 5 .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.

AB - 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] 5 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 5 .006). However, after 1 year, OS was not significantly different (HR, 1.38, P 5 .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.

UR - http://www.scopus.com/inward/record.url?scp=85086108920&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85086108920&partnerID=8YFLogxK

U2 - 10.1182/bloodadvances.2019001084

DO - 10.1182/bloodadvances.2019001084

M3 - Article

C2 - 32384540

AN - SCOPUS:85086108920

VL - 4

SP - 1966

EP - 1973

JO - Blood advances

JF - Blood advances

SN - 2473-9529

IS - 9

ER -

Survival following allogeneic transplant in patients with myelofibrosis

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