Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia

Blood Advances - Tập 3 Số 20 - Trang 3123-3131 - 2019
Nelli Bejanyan1, Soyoung Kim2, Kyle Hebert3, Natasha Kekre4, Hisham Abdel‐Azim5, Ibrahim Ahmed6, Mahmoud Aljurf7, Sherif M. Badawy8, Amer Beitinjaneh9, Jaap Jan Boelens10, Miguel Ángel Díaz11, Christopher C. Dvorak12, Shahinaz M. Gadalla13, James Gajewski14, Robert Peter Gale15, Siddhartha Ganguly16, Andrew R. Gennery17, Biju George18, Usama Gergis19, David Gómez‐Almaguer20, Marta González‐Vicent11, Hasan Hashem21, Rammurti T. Kamble22, Kimberly A. Kasow23, Hillard M. Lazarus24, Vikram Mathews18, Paul J. Orchard25, Michael A. Pulsipher5, O Ringdén26, Kirk R. Schultz27, Pierre Teira28, Ann E. Woolfrey29, Blachy J. Dávila Saldaña30, Bipin N. Savani31, Jacek Winiarski26, Jean A. Yared32, Daniel J. Weisdorf25, Joseph H. Antin33, Mary Eapen3
1Department of Blood and Marrow Transplant and Cellular Therapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL;
2Division of Biostatistics, and
3Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI
4The Ottawa Hospital Blood & Marrow Transplant Program, University of Ottawa, Ottawa, ON, Canada;
5Children's Center for Cancer and Blood Diseases, Children’s Hospital of Los Angeles, Los Angeles, CA;
6Division of Pediatric Hematology/Oncology, Children's Mercy Hospital, Kansas City, MO;
7King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia
8Hematology, Oncology and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital of Chicago, Northwestern University, Evanston, IL;
9Department of Medicine, University of Miami, Miami, FL
10Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY
11Hospital Niño Jesus, Madrid, Spain
12Division of Pediatric Blood and Marrow Transplantation, University of California San Francisco Medical Center, San Francisco, CA;
13Clinical Genetics Branch, National Cancer Institute, Rockville, MD;
14Department of Medicine, Oregon Health & Science University, Portland, OR
15Division of Experimental Medicine, Department of Medicine, Imperial College London, London, United Kingdom;
16Division of Hematologic Malignancies and Cellular Therapeutics, University of Kansas, Kansas City, KS;
17Institute of Cellular Medicine, Newcastle University, Newcastle Upon Tyne, United Kingdom
18Department of Hematology, Christian Medical College Hospital, Vellore, India
19Department of Medicine, Weill Cornell Medical College, New York, NY
20Hospital Universitario José E. González, Universidad Autónoma de Nuevo León, Monterrey, Mexico;
21Division of Hematology and Oncology, Nationwide Children’s Hospital, Columbus, OH;
22Department of Medicine. Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX;
23Department of Pediatrics, University of North Carolina Hospitals, Chapel Hill, NC
24Department of Medicine, Case Western Reserve University, Cleveland, OH
25Department of Medicine, University of Minnesota, Minneapolis, MN
26Division of Therapeutic Immunology, Karolinska Institutet, Stockholm, Sweden
27British Columbia Children's Hospital, University of British Columbia, Vancouver, BC, Canada
28Centre Hospitalier Universitaire Sainte-Justine, Montreal, QC, Canada;
29Fred Hutchinson Cancer Research Center, Seattle, WA
30Children's National Medical Center, Washington, DC
31Vanderbilt University Medical Center, Nashville, TN
32Greenebaum Cancer Center, University of Maryland, Baltimore, MD; and
33Stem Cell Transplantation, Dana-Farber Cancer Institute, Boston, MA.

Tóm tắt

Key PointsFlu/Cy/ATG and Cy/ATG regimens offer the best survival for matched-sibling BMT. Transplantation in patients aged ≥30 years is associated with higher mortality after matched-sibling and unrelated donor BMT.

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Tài liệu tham khảo

Scheinberg, 2012, How I treat acquired aplastic anemia, Blood, 120, 1185, 10.1182/blood-2011-12-274019

Bacigalupo, 2017, How I treat acquired aplastic anemia, Blood, 129, 1428, 10.1182/blood-2016-08-693481

Young, 2018, Aplastic anemia, N Engl J Med, 379, 1643, 10.1056/NEJMra1413485

Townsley, 2017, Elthrombopag added to standard immunosuppression for aplastic anemia, N Engl J Med, 376, 1540, 10.1056/NEJMoa1613878

Gupta, 2010, Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors, Haematologica, 95, 2119, 10.3324/haematol.2010.026682

Rice, 2019, Allogeneic hematopoietic cell transplantation in patients aged 50 years or older with severe aplastic anemia, Biol Blood Marrow Transplant, 25, 488, 10.1016/j.bbmt.2018.08.029

Bacigalupo, 2005, Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party, Bone Marrow Transplant, 36, 947, 10.1038/sj.bmt.1705165

Deeg, 2006, Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy, Blood, 108, 1485, 10.1182/blood-2006-03-005041

Champlin, 2007, Bone marrow transplantation for severe aplastic anemia: a randomized controlled study of conditioning regimens, Blood, 109, 4582, 10.1182/blood-2006-10-052308

Marsh, 2011, Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft-versus-host disease after allogeneic stem cell transplantation for acquired aplastic anemia, Blood, 118, 2351, 10.1182/blood-2010-12-327536

Tolar, 2012, Fludarabine-based conditioning for marrow transplantation from unrelated donors in severe aplastic anemia: early results of a cyclophosphamide dose deescalation study show life-threatening adverse events at predefined cyclophosphamide dose levels, Biol Blood Marrow Transplant, 18, 1007, 10.1016/j.bbmt.2012.04.014

Anderlini, 2015, Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study, Lancet Haematol, 2, e367, 10.1016/S2352-3026(15)00147-7

Schrezenmeier, 2007, Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA-matched sibling donor transplants for young patients with severe acquired aplastic anemia, Blood, 110, 1397, 10.1182/blood-2007-03-081596

Eapen, 2011, Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia, Blood, 118, 2618, 10.1182/blood-2011-05-354001

Bacigalupo, 2012, Bone marrow versus peripheral blood as the stem cell source for sibling transplants in acquired aplastic anemia: survival advantage for bone marrow in all age groups, Haematologica, 97, 1142, 10.3324/haematol.2011.054841

Olsson, 2013, Graft failure in the modern era of allogeneic hematopoietic SCT [published correction appears in Bone Marrow Transplant. 2013;48(4):616], Bone Marrow Transplant, 48, 537, 10.1038/bmt.2012.239

Przepiorka, 1995, 1994 Consensus Conference on Acute GVHD Grading, Bone Marrow Transplant, 15, 825

Atkinson, 1989, Consensus among bone marrow transplanters for diagnosis, grading and treatment of chronic graft-versus-host disease, Bone Marrow Transplant, 4, 247

Lin, 1997, Non-parametric inference for cumulative incidence functions in competing risks studies, Stat Med, 16, 901, 10.1002/(SICI)1097-0258(19970430)16:8<901::AID-SIM543>3.0.CO;2-M

Cox, 1972, Regression models and life tables, J R Stat Soc Series A, 34, 187, 10.1111/j.2517-6161.1972.tb00899.x

Zhang, 2007, A SAS macro for estimation of direct adjusted survival curves based on a stratified Cox regression model, Comput Methods Programs Biomed, 88, 95, 10.1016/j.cmpb.2007.07.010

Fine, 1999, A proportional hazards model for the subdistribution of a competing risk, J Am Stat Assoc, 94, 496, 10.1080/01621459.1999.10474144

Andersen, 1999, Testing for centre effects in multi-centre survival studies: a Monte Carlo comparison of fixed and random effects tests, Stat Med, 18, 1489, 10.1002/(SICI)1097-0258(19990630)18:12<1489::AID-SIM140>3.0.CO;2-#

Aljurf, 2013, Standard treatment of acquired SAA in adult patients 18-40 years old with an HLA-identical sibling donor, Bone Marrow Transplant, 48, 178, 10.1038/bmt.2012.223

Killick, 2016, Guidelines for the diagnosis and management of adult aplastic anaemia, Br J Haematol, 172, 187, 10.1111/bjh.13853

Kekre, 2017, Effect of antithymocyte globulin source on outcomes of bone marrow transplantation for severe aplastic anemia, Haematologica, 102, 1291, 10.3324/haematol.2017.164459

Vaht, 2017, Incidence and outcome of acquired aplastic anemia: real-world data from patients diagnosed in Sweden from 2000-2011, Haematologica, 102, 1683, 10.3324/haematol.2017.169862

Horan, 2012, Evaluation of HLA matching in unrelated hematopoietic stem cell transplantation for nonmalignant disorders, Blood, 120, 2918, 10.1182/blood-2012-03-417758

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Blood Advances

Tập 3 Số 20

3123-3131

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