Ruxolitinib before allogeneic hematopoietic transplantation in patients with myelofibrosis on behalf SFGM-TC and FIM groups

被引：25

作者：

Robin, Marie ^{[1
]}

Porcher, Raphael ^{[2
,3
]}

Orvain, Corentin ^{[4
]}

Bay, Jacques-Olivier ^{[5
]}

Barraco, Fiorenza ^{[6
]}

Huynh, Anne ^{[7
]}

Charbonnier, Amandine ^{[8
]}

Forcade, Edouard ^{[9
]}

Chantepie, Sylvain ^{[10
]}

Bulabois, Claude ^{[11
]}

Yakoub-Agha, Ibrahim ^{[12
]}

Detrait, Marie ^{[13
]}

Michonneau, David ^{[14
]}

Turlure, Pascal ^{[15
]}

Raus, Nicole ^{[16
]}

Boyer, Francoise ^{[4
]}

Suarez, Felipe ^{[17
]}

Vincent, Laure ^{[18
]}

Guyen, Stephanie N. ^{[19
]}

Cornillon, Jerome ^{[20
]}

Villate, Alban ^{[21
]}

Dupriez, Brigitte ^{[22
]}

Cassinat, Bruno ^{[23
]}

Rolland, Valerie ^{[24
]}

Schlageter, Marie Helene ^{[23
]}

Socie, Gerard ^{[14
]}

Kiladjian, Jean-Jacques ^{[25
]}

机构：

[1] Hop St Louis, AP HP, Serv Hematol Greffe, Paris, France

[2] Univ Paris, INSERM, Epidemiol & Stat Res Ctr CRESS, INRA, Paris, France

[3] Hop Hotel Dieu, AP HP, Ctr Epidemiol Clin, Paris, France

[4] CHU Angers, Angers, France

[5] CHU Clermont Ferrand, Clermont Ferrand, France

[6] CHU Lyon Sud, Lyon, France

[7] CHU Toulouse, Toulouse, France

[8] CHU Amiens, Amiens, France

[9] CHU Bordeaux, Serv Hematol & Therapie Cellulaire, Bordeaux, France

[10] CHU Caen, Caen, France

[11] CHU Grenoble Alpes, Grenoble, France

[12] Univ Lille, CHU Lille, INSERM U1286, Infinite, Lille, France

[13] Univ Lorraine, Inst Louis Mathieu, CHRU Nancy, Vandoeuvre Les Nancy, France

[14] Univ Paris, Serv Hematol Greffe, Hop St Louis, AP HP,U976, Paris, France

[15] CHU Limoges, Limoges, France

[16] Hop Lyon Sud, SFGM TC, Lyon, France

[17] CHU, Hop Necker, AP HP, Paris, France

[18] CHU Montpellier, Hop St Eloi, Montpellier, France

[19] Hop La Pitie Salpetriere, APHP, Paris, France

[20] Inst Cancerol Loire, St Etienne, France

[21] CHRU Tours, Hop Bretonneau, Tours, France

[22] Ctr Hosp, Route Bassee, Lens, France

[23] Hop St Louis, AP HP, Lab Biol Cellulaire, Paris, France

[24] CHU Lyon Sud, FILO, Lyon, France

[25] Hop St Louis, AP HP, Ctr Invest Clin, Paris, France

来源：

BONE MARROW TRANSPLANTATION | 2021年 / 56卷 / 08期

关键词：

D O I：

10.1038/s41409-021-01252-7

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

This multicenter prospective phase 2 trial analyzed disease-free survival (DFS) in myelofibrosis patients receiving ruxolitinib for 6 months before transplantation. Seventy-six patients were recruited. Age-adjusted dynamic international prognostic scoring system was intermediate-1, intermediate-2, and high in 27 (36%), 31 (41%), and 18 (24%) patients. All patients received ruxolitinib from inclusion to conditioning regimen (fludarabine-melphalan) or to progression. A donor was found in 64 patients: 18 HLA-matched sibling donor (MSD), 32 HLA-matched unrelated (UD10/10), and 14 HLA mismatched unrelated donor (UD9/10. Among 64 patients with a donor, 20 (31%) achieved a partial response before transplantation and 59 (92%) could be transplanted after ruxolitinib therapy (18/18 MSD, 30/21 UD10/10, 11/34 UD9/10), of whom 19 (32%) were splenectomized. Overall survival from inclusion was 68% at 12 months. One-year DFS after transplantation was 55%: 83%, 40%, and 34% after MSD, UD10/10 or UD9/10, respectively. Cumulative incidence of grade 2-4 acute graft-versus-host disease (GVHD) was 66% and non-relapse-mortality was 42% at 12 months. Short course of ruxolitinib before transplantation is followed by a high rate of transplantation. With the platform used in this protocol, outcome was much better in patients transplanted with HLA-matched sibling donor as compared to unrelated donor.

引用

页码：1888 / 1899

页数：12

共 41 条

[11]

Dupriez B, 1996, BLOOD, V88, P1013

[12] Standard graft-versus-host disease prophylaxis with or without anti-T-cell globulin in haematopoietic cell transplantation from matched unrelated donors: a randomised, open-label, multicentre phase 3 trial [J].

Finke, Juergen ;

Bethge, Wolfgang A. ;

Schmoor, Claudia ;

Ottinger, Hellmut D. ;

Stelljes, Matthias ;

Zander, Axel R. ;

Volin, Liisa ;

Ruutu, Tapani ;

Heim, Dominik A. ;

Schwerdtfeger, Rainer ;

Kolbe, Karin ;

Mayer, Jiri ;

Maertens, Johan A. ;

Linkesch, Werner ;

Holler, Ernst ;

Koza, Vladimir ;

Bornhaeuser, Martin ;

Einsele, Hermann ;

Kolb, Hans-Jochem ;

Bertz, Hartmut ;

Egger, Matthias ;

Grishina, Olga ;

Socie, Gerard .

LANCET ONCOLOGY, 2009, 10 (09) :855-864

[13] Classification and Personalized Prognosis in Myeloproliferative Neoplasms [J].

Grinfeld, J. ;

Nangalia, J. ;

Baxter, E. J. ;

Wedge, D. C. ;

Angelopoulos, N. ;

Cantrill, R. ;

Godfrey, A. L. ;

Papaemmanuil, E. ;

Gundem, G. ;

MacLean, C. ;

Cook, J. ;

O'Neil, L. ;

O'Meara, S. ;

Teague, J. W. ;

Butler, A. P. ;

Massie, C. E. ;

Williams, N. ;

Nice, F. L. ;

Andersen, C. L. ;

Hasselbalch, H. C. ;

Guglielmelli, P. ;

McMullin, M. F. ;

Vannucchi, A. M. ;

Harrison, C. N. ;

Gerstung, M. ;

Green, A. R. ;

Campbell, P. J. .

NEW ENGLAND JOURNAL OF MEDICINE, 2018, 379 (15) :1416-1430

[14] Ruxolitinib Therapy Followed by Reduced-Intensity Conditioning for Hematopoietic Cell Transplantation for Myelofibrosis: Myeloproliferative Disorders Research Consortium 114 Study [J].

Gupta, Vikas ;

Kosiorek, Heidi E. ;

Mead, Adam ;

Klisovic, Rebecca B. ;

Galvin, John P. ;

Berenzon, Dmitriy ;

Yacoub, Abdulraheem ;

Viswabandya, Auro ;

Mesa, Ruben A. ;

Goldberg, Judith ;

Price, Leah ;

Salama, Mohamed E. ;

Weinberg, Rona Singer ;

Rampal, Raajit ;

Farnoud, Noushin ;

Dueck, Amylou C. ;

Mascarenhas, John O. ;

Hoffman, Ronald .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2019, 25 (02) :256-264

[15] Reduced-Intensity Hematopoietic Cell Transplantation for Patients with Primary Myelofibrosis: A Cohort Analysis from the Center for International Blood and Marrow Transplant Research [J].

Gupta, Vikas ;

Malone, Adriana K. ;

Hari, Parameswaran N. ;

Ahn, Kwang Woo ;

Hu, Zhen-Huan ;

Gale, Robert Peter ;

Ballen, Karen K. ;

Hamadani, Mehdi ;

Olavarria, Eduardo ;

Gerds, Aaron T. ;

Waller, Edmund K. ;

Costa, Luciano J. ;

Antin, Joseph H. ;

Kamble, Rammurti T. ;

van Besien, Koen M. ;

Savani, Bipin N. ;

Schouten, Harry C. ;

Szer, Jeffrey ;

Cahn, Jean-Yves ;

de Lima, Marcos J. ;

Wirk, Baldeep ;

Aljurf, Mahmoud D. ;

Popat, Uday ;

Bejanyan, Nelli ;

Litzow, Mark R. ;

Norkin, Maxim ;

Lewis, Ian D. ;

Hale, Gregory A. ;

Woolfrey, Ann E. ;

Miller, Alan M. ;

Ustun, Celalettin ;

Jagasia, Madan H. ;

Lill, Michael ;

Maziarz, Richard T. ;

Cortes, Jorge ;

Kalaycio, Matt E. ;

Saber, Wael .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2014, 20 (01) :89-97

[16] Determinants of survival in myelofibrosis patients undergoing allogeneic hematopoietic cell transplantation [J].

Hernandez-Boluda, Juan Carlos ;

Pereira, Arturo ;

Kroeger, Nicolaus ;

Beelen, Dietrich ;

Robin, Marie ;

Bornhaeuser, Martin ;

Angelucci, Emanuele ;

Vitek, Antonin ;

Blau, Igor Wolfgang ;

Niittyvuopio, Riitta ;

Finke, Juergen ;

Cornelissen, Jan J. ;

Passweg, Jakob ;

Dreger, Peter ;

Petersen, Eefke ;

Kanz, Lothar ;

Sanz, Jaime ;

Zuckerman, Tsila ;

Zinger, Nienke ;

Iacobelli, Simona ;

Hayden, Patrick ;

Czerw, Tomasz ;

McLornan, Donal ;

Yakoub-Agha, Ibrahim .

LEUKEMIA, 2021, 35 (01) :215-224

[17] Hematopoietic cell transplantation as curative therapy for idiopathic myelofibrosis, advanced polycythemia vera, and essential thrombocythemia [J].

Kerbauy, Daniella M. B. ;

Gooley, Theodore A. ;

Sale, George E. ;

Flowers, Mary E. D. ;

Doney, Kristine C. ;

Georges, George E. ;

Greene, Joanne E. ;

Linenberger, Michael ;

Petersdorf, Effie ;

Sandmaier, Brenda M. ;

Scott, Bart L. ;

Sorror, Mohamed ;

Stirewalt, Derek L. ;

Stewart, F. Marc ;

Witherspoon, Robert P. ;

Storb, Rainer ;

Appelbaum, Frederick R. ;

Deeg, H. Joachim .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2007, 13 (03) :355-365

[18] Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group [J].

Kroeger, N. M. ;

Deeg, J. H. ;

Olavarria, E. ;

Niederwieser, D. ;

Bacigalupo, A. ;

Barbui, T. ;

Rambaldi, A. ;

Mesa, R. ;

Tefferi, A. ;

Griesshammer, M. ;

Gupta, V. ;

Harrison, C. ;

Alchalby, H. ;

Vannucchi, A. M. ;

Cervantes, F. ;

Robin, M. ;

Ditschkowski, M. ;

Fauble, V. ;

McLornan, D. ;

Ballen, K. ;

Popat, U. R. ;

Passamonti, F. ;

Rondelli, D. ;

Barosi, G. .

LEUKEMIA, 2015, 29 (11) :2126-2133

[19] Impact of primary disease on outcome after allogeneic stem cell transplantation for transformed secondary acute leukaemia [J].

Kroeger, Nicolaus ;

Eikema, Diderik-Jan ;

Koster, Linda ;

Beelen, Dietrich ;

de Wreede, Liesbeth C. ;

Finke, Juergen ;

Koenecke, Christian ;

Niederwieser, Dietger ;

Bornhaeuser, Martin ;

Schoenland, Stefan ;

Potter, Victoria ;

Wolschke, Christine ;

Maertens, Johan ;

Theobald, Matthias ;

Kobbe, Guido ;

Itala-Remes, Maija ;

Wulf, Gerald ;

Kahls, Peter ;

Forcade, Edouard ;

Greinix, Hildegard ;

Masszi, Tamas ;

Yakoub-Agha, Ibrahim ;

Chalandon, Yves ;

Robin, Marie .

BRITISH JOURNAL OF HAEMATOLOGY, 2019, 185 (04) :725-732

[20] Peritransplantation Ruxolitinib Prevents Acute Graft-versus-Host Disease in Patients with Myelofibrosis Undergoing Allogenic Stem Cell Transplantation [J].

Kroeger, Nicolaus ;

Abd Kadir, Sharifah Shahnaz Syed L. ;

Zabelina, Tatjana ;

Badbaran, Anita ;

Christopeit, Maximilian ;

Ayuk, Francis ;

Wolschke, Christine .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2018, 24 (10) :2152-2156

← 1 2 3 4 5 →