Keywords
How to Cite
Abstract
In a multicenter retrospective study, we evaluated the outcome of patients with myelofibrosis (MF) who underwent allogeneic hematopoietic stem cell transplantation (AHCT) in Argentina. A total of 43 patients were included (median age 53; range 23-70; males 56%) who received an AHSCT between 2002 and 2016 in 12 centers; 33 patients had primary MF and 10 patients MF secondary to PV or ET. Donors were related in 30 cases and full match in 38 cases, and the source of stem cells was peripheral blood for all patients. Conditioning regimen was myeloblative (MAC; n=16; Bu ≥9.6 mg/kg) or reduced intensity (RIC; n=27; 13 Bu, 10 Mel and four TBI based). 1-year cumulative incidence of acute grade 2-4 graft versus host disease (GVHD) was 49% and chronic GVHD 11% and the corresponding relapse incidence was 20% (higher in patients with a high DIPSS PLUS; p=0.015). 100-days and 1-year nonrelapse mortality (NRM) was 19% and 37% respectively with the GVHD being the main cause of death. 1-year overall survival (OS) was 55%; in the multivariate analysis pre-transplant factors associated with inferior OS were the HCT-CI (p=0.019), splenomegaly ≥10 cm (p=0.021) and the leukemic transformation (p=0.019). Patients with low HCT-CI (n=17) had better 1-year OS with MAC (MAC vs RIC: 83% vs 41%; p=0.025) whereas those with intermediate-high HCT-CI (n=23) had an inferior 1-year OS with MAC (MAC vs RIC: 22% vs 69%; p=0.040). Our data show that NRM is the main determiner of OS in patients with MF. We should make efforts to control the GVHD and the related infections.
References
2014; 124:2507-13.
2. Gupta V, Hari P, Hoffman R. Allogeneic hematopoietic cell transplantation for myelofibrosis in the era of JAK inhibitors. Blood. 2012;120:1367-79.
3. Alchalby H, Yunus DR, Zabelina T et al. Risk models predicting survival after reduced-intensity transplantation for mielofibrosis. Br J Haematol. 2012;157(1):75-85.
4. PanagiotaV, TholF, MarkusB et al. Prognostic effect of calreticulin mutations in patients with myelofibrosis after allogeneic hematopoietic stem cell transplantation. Leukemia.
2014;28(7):1552-5.
5. Kröger N, Panagiota V, Badbaran A et al. Impact of Molecular Genetics on Outcome in Myelofibrosis Patients after Allogeneic Stem Cell Transplantation. Biol Blood Marrow Transplant.
2017 Apr 4. pii: S1083-8791(17)30385-3. doi: 10.1016/j.bbmt.2017.03.034.
6. Kröger N, Holler E, Kobbe G et al. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic
LeukemiaWorking Party of the European Group for Blood and Marrow Transplantation. Blood. 2009 ;114(26):5264-70.
7. Gupta V, Malone AK, Hari PN et al. Reduced-intensity hematopoietic cell transplantation for patients with primary myelofibrosis: a cohort analysis from the center for international blood
and marrow transplant research. Biol Blood Marrow Transplant. 2014;20(1):89-97.
8. Rondelli D, Goldberg JD, Isola L et al. MPDRC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with mielofibrosis. Blood.
2014;124(7):1183-91.
9. Keyzner A, Han S, Shapiro S et al. Outcome of allogeneic hematopoietic stem cell transplantation for patients with chronic and advanced phase myelofibrosis. Biol Blood Marrow Transplant.
2016;22(12):2180-2186.
10.Bacigalupo A, Soraru M, Dominietto A et al. Allogeneic hemopoietic SCT for patients with primary myelofibrosis: a predictive transplant score based on transfusion requirement, spleen
size and donor type. Bone Marrow Transplant. 2010;45(3):458-63.
11.Kerbauy DM, Gooley TA, Sale GE et al. Hematopoietic cell transplantation as curative therapy for idiopathic myelofibrosis, advanced polycythemia vera, and essential thrombocythemia. Biol Blood
Marrow Transplant. 2007;13(3):355-65.
12.Schmohl JU, Groh C, Faul C et al. Allogeneic hematopoietic cell transplantation in patients with myelofibrosis: A single center experience. Ann Hematol. 2016;95(6):973-83.
13.Robin M, Tabrizi R, Mohty M et al. Allogeneic haematopoietic stem cell transplantation for myelofibrosis: a report of the Société Française de Greffe de Moelle et de Therapie Cellulaire
(SFGM-TC). Br J Haematol. 2011;152(3):331-9.
14.Gangat N, Caramazza D, Vaidya R et al. DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J Clin Oncol. 2011;29(4):392-7.
15.Kröger NM, Deeg JH, Olavarria E et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015;29(11):2126-33.
16.Arber DA, Orazi A, Hasserjian R et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127(20):2391-405.
17.Bacigalupo A, Ballen K, Rizzo D, Giralt S, Lazarus H, Ho V et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009;15(12):1628-33.
18.Kanda Y. Investigation of the freely available easy-to-use software ‘EZR’ for medical statistics. Bone Marrow Transplant. 2013;48(3):452-8.
19.Shanavas M, Messner HA, Atenafu EG et al. Allogeneic hematopoietic cell transplantation for myelofibrosis using fludarabine-, intravenous busulfan- and low-dose TBI-based conditioning. Bone Marrow Transplant. 2014;49(9):1162-9.
20.Shanavas M, Popat U, Michaelis LC et al. Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients with Myelofibrosis with Prior Exposure to Janus Kinase 1/2 Inhibitors. Biol Blood Marrow Transplant. 2016;22(3):432-40.
All material published in the journal HEMATOLOGÍA (electronic and print version) is transferred to the Argentinean Society of Hematology. In accordance with the copyright Act (Act 11 723), a copyright transfer form will be sent to the authors of approved works, which has to be signed by all the authors before its publication. Authors should keep a copy of the original since the journal is not responsible for damages or losses of the material that was submitted. Authors should send an electronic version to the email: revista@sah.org.ar