Is allogeneic stem cell transplantation for myelofibrosis still indicated at the time of molecular markers and JAK inhibitors era?

被引:7
作者
Lestang, Elsa [1 ]
Peterlin, Pierre [1 ]
Le Bris, Yannick [2 ]
Dubruille, Viviane [1 ]
Delaunay, Jacques [1 ]
Godon, Catherine [2 ]
Theisen, Olivier [2 ]
Blin, Nicolas [1 ]
Mahe, Beatrice [1 ]
Gastinne, Thomas [1 ]
Garnier, Alice [1 ]
Touzeau, Cyrille [1 ]
Voldoire, Maud [1 ]
Bene, Marie C. [2 ]
Le Gouill, Steven [1 ]
Milpied, Noel [1 ,3 ]
Mohty, Mohamad [1 ,4 ]
Moreau, Philippe [1 ]
Guillaume, Thierry [1 ]
Chevallier, Patrice [1 ]
机构
[1] CHU Hotel Dieu, Hematol Dept, Nantes, France
[2] CHU Hotel Dieu, Hematol Biol Dept, Nantes, France
[3] CHU Bordeaux, Dept Hematol, Bordeaux, France
[4] Hosp St Antoine, Dept Hematol, Paris, France
关键词
allogeneic transplantation; calreticuline; intent-to-treat analysis; JAK inhibitor; JAK2; myeloproliferative leukemia virus; primary myelofibrosis; secondary myelofibrosis; INTERNATIONAL WORKING GROUP; MYELOPROLIFERATIVE NEOPLASMS RESEARCH; PROGNOSTIC SCORING SYSTEM; VERSUS-HOST-DISEASE; CELLULAIRE SFGM-TC; POLYCYTHEMIA-VERA; ESSENTIAL THROMBOCYTHEMIA; MYELOID METAPLASIA; SOCIETE FRANCAISE; IWG-MRT;
D O I
10.1111/ejh.12891
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
ObjectiveThe role of allogenic stem cell transplantation (ASCT) is still debated in myelofibrosis (MF). MethodsA retrospective analyzed was performed to compare the outcome of 71 patients with intermediate-2 or high-risk Dynamic International Prognosis Scoring System+ (DIPSS+) primary (PMF) or secondary (SMF) myelofibrosis with an indication of ASCT as they ultimately underwent the procedure (n=34) or not (n=37). ResultsFive-year overall survival (OS) was not statistically different between both groups (allograft: 52% vs no allograft: 34%, P=.12). However, progression to myelodysplastic syndrome or acute myeloid leukemia at 5years was significantly lower in transplanted patients (14% vs 50%, P=.01). In univariate analysis, 5-year OS was significantly higher for transplanted vs non-transplanted patients with unfavorable karyotype (75% vs 0%, P=.001), SMF (71% vs 20%, P=.001) or high DIPSS+ score (46% vs 15%, P=.03). There was also a trend for better 5-year OS in allografted patients with high JAK2(V617F) burden (>65%) (75% vs 8%, P=.07). Interestingly, the survival of patients who did not proceed to ASCT was dramatically increased by the use of ruxolitinib. ConclusionsNot all intermediate-2/high-risk DIPSS+ MF patients benefit from ASCT, especially since the introduction of JAK2 inhibitors.
引用
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页码:60 / 69
页数:10
相关论文
共 48 条
[1]   The outcome of allo-HSCT for 92 patients with myelofibrosis in the Nordic countries [J].
Abelsson, J. ;
Merup, M. ;
Birgegard, G. ;
WeisBjerrum, O. ;
Brinch, L. ;
Brune, M. ;
Johansson, P. ;
Kauppila, M. ;
Lenhoff, S. ;
Liljeholm, M. ;
Malm, C. ;
Remes, K. ;
Vindelov, L. ;
Andreasson, B. .
BONE MARROW TRANSPLANTATION, 2012, 47 (03) :380-386
[2]   Impact of JAK2V617F mutation status, allele burden, and clearance after allogeneic stem cell transplantation for myelofibrosis [J].
Alchalby, Haefaa ;
Badbaran, Anita ;
Zabelina, Tatjana ;
Kobbe, Guido ;
Hahn, Joachim ;
Wolff, Daniel ;
Bornhaeuser, Martin ;
Thiede, Christian ;
Baurmann, Herrad ;
Bethge, Wolfgang ;
Hildebrandt, York ;
Bacher, Ulrike ;
Fehse, Boris ;
Zander, Axel R. ;
Kroeger, Nicolaus .
BLOOD, 2010, 116 (18) :3572-3581
[3]   Digital-PCR assay for screening and quantitative monitoring of calreticulin (CALR) type-2 positive patients with myelofibrosis following allogeneic stem cell transplantation [J].
Badbaran, A. ;
Fehse, B. ;
Christopeit, M. ;
Aranyossy, T. ;
Ayuk, F. A. ;
Wolschke, C. ;
Kroeger, N. .
BONE MARROW TRANSPLANTATION, 2016, 51 (06) :872-873
[4]   Outcome of Transplantation for Myelofibrosis [J].
Ballen, Karen K. ;
Shrestha, Smriti ;
Sobocinski, Kathleen A. ;
Zhang, Mei-Jie ;
Bashey, Asad ;
Bolwell, Brian J. ;
Cervantes, Francisco ;
Devine, Steven M. ;
Gale, Robert Peter ;
Gupta, Vikas ;
Hahn, Theresa E. ;
Hogan, William J. ;
Kroeger, Nicolaus ;
Litzow, Mark R. ;
Marks, David I. ;
Maziarz, Richard T. ;
McCarthy, Philip L. ;
Schiller, Gary ;
Schouten, Harry C. ;
Roy, Vivek ;
Wiernik, Peter H. ;
Horowitz, Mary M. ;
Giralt, Sergio A. ;
Arora, Mukta .
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2010, 16 (03) :358-367
[5]   How we manage JAK inhibition in allogeneic transplantation for myelofibrosis [J].
Ballinger, Tarah J. ;
Savani, Bipin N. ;
Gupta, Vikas ;
Kroger, Nicolaus ;
Mohty, Mohamad .
EUROPEAN JOURNAL OF HAEMATOLOGY, 2015, 94 (02) :115-119
[6]   Philadelphia-Negative Classical Myeloproliferative Neoplasms: Critical Concepts and Management Recommendations From European LeukemiaNet [J].
Barbui, Tiziano ;
Barosi, Giovanni ;
Birgegard, Gunnar ;
Cervantes, Francisco ;
Finazzi, Guido ;
Griesshammer, Martin ;
Harrison, Claire ;
Hasselbalch, Hans Carl ;
Hehlmann, Rudiger ;
Hoffman, Ronald ;
Kiladjian, Jean-Jacques ;
Kroeger, Nicolaus ;
Mesa, Ruben ;
McMullin, Mary F. ;
Pardanani, Animesh ;
Passamonti, Francesco ;
Vannucchi, Alessandro M. ;
Reiter, Andreas ;
Silver, Richard T. ;
Verstovsek, Srdan ;
Tefferi, Ayalew .
JOURNAL OF CLINICAL ONCOLOGY, 2011, 29 (06) :761-770
[7]   Proposed criteria for the diagnosis of post-polycythemia vera and post-essential thrombocythemia myelofibrosis: a consensus statement from the international working group for myelofibrosis research and treatment [J].
Barosi, G. ;
Mesa, R. A. ;
Thiele, J. ;
Cervantes, F. ;
Campbell, P. J. ;
Verstovsek, S. ;
Dupriez, B. ;
Levine, R. L. ;
Passamonti, F. ;
Gotlib, J. ;
Reilly, J. T. ;
Vannucchi, A. M. ;
Hanson, C. A. ;
Solberg, L. A. ;
Orazi, A. ;
Tefferi, A. .
LEUKEMIA, 2008, 22 (02) :437-438
[8]   SRSF2 and U2AF1 mutations in primary myelofibrosis are associated with JAK2 and MPL but not calreticulin mutation and may independently reoccur after allogeneic stem cell transplantation [J].
Bartels, S. ;
Lehmann, U. ;
Buesche, G. ;
Schlue, J. ;
Mozer, M. ;
Stadler, J. ;
Triviai, I. ;
Alchalby, H. ;
Kroeger, N. ;
Kreipe, H. .
LEUKEMIA, 2015, 29 (01) :253-255
[9]   Allo-SCT for Philadelphia-negative myeloproliferative neoplasms in blast phase: a study from the Societe Francaise de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC) [J].
Cahu, X. ;
Chevallier, P. ;
Clavert, A. ;
Suarez, F. ;
Michallet, M. ;
Vincent, L. ;
Vigouroux, S. ;
Blaise, D. ;
Mariette, C. ;
Bilger, K. ;
Robin, M. ;
Yakoub-Agha, I. ;
de Latour, R. Peffault ;
Mohty, M. .
BONE MARROW TRANSPLANTATION, 2014, 49 (06) :756-760
[10]   New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment [J].
Cervantes, Francisco ;
Dupriez, Brigitte ;
Pereira, Arturo ;
Passamonti, Francesco ;
Reilly, John T. ;
Morra, Enrica ;
Vannucchi, Alessandro M. ;
Mesa, Ruben A. ;
Demory, Jean-Loup ;
Barosi, Giovanni ;
Rumi, Elisa ;
Tefferi, Ayalew .
BLOOD, 2009, 113 (13) :2895-2901