Haplo-identical or mismatched unrelated donor hematopoietic cell transplantation for Fanconi anemia: Results from the Severe Aplastic Anemia Working Party of the EBMT.
Adolescent
Allografts
Bone Marrow Transplantation
/ statistics & numerical data
Child
Fanconi Anemia
/ genetics
Female
Graft Survival
Graft vs Host Disease
/ epidemiology
HLA Antigens
/ genetics
Haplotypes
Histocompatibility
/ genetics
Histocompatibility Testing
Humans
Kaplan-Meier Estimate
Living Donors
Lymphocyte Depletion
Male
Peripheral Blood Stem Cell Transplantation
/ statistics & numerical data
Primary Graft Dysfunction
/ epidemiology
Progression-Free Survival
Proportional Hazards Models
Prospective Studies
Siblings
T-Lymphocyte Subsets
/ immunology
Treatment Outcome
Journal
American journal of hematology
ISSN: 1096-8652
Titre abrégé: Am J Hematol
Pays: United States
ID NLM: 7610369
Informations de publication
Date de publication:
01 05 2021
01 05 2021
Historique:
revised:
16
02
2021
received:
10
11
2020
accepted:
18
02
2021
pubmed:
20
2
2021
medline:
14
5
2021
entrez:
19
2
2021
Statut:
ppublish
Résumé
Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for bone marrow failure or hematopoietic malignant diseases for Fanconi anemia (FA) patients. Although results have improved over the last decades, reaching more than 90% survival when a human leukocyte antigen (HLA)-identical donor is available, alternative HCT donors are still less reported. We compared HCT outcomes using HLA-mismatched unrelated donors (MMUD; n = 123) or haplo-identical donors (HDs), either using only in vivo T cell depletion (n = 33) or T cells depleted in vivo with some type of graft manipulation ex vivo (n = 59) performed for FA between 2000 and 2018. Overall survival (OS) by 24 months was 62% (53-71%) for MMUD, versus 80% (66-95%) for HDs with only in vivo T cell depletion and 60% (47-73%) for HDs with in vivo and ex vivo T cell depletion (p = .22). Event-free survival (EFS) was better for HD-transplanted FA patients with only in vivo T cell depletion 86% (73-99%) than for those transplanted from a MMUD 58% (48-68%) or those with graft manipulation 56% (42-69%) (p = .046). Grade II-IV acute graft-versus-host disease (GVHD) was 41% (MMUD) versus 40% (HDs with no graft manipulation) versus 17% (HDs with T cell depleted graft), (p = .005). No differences were found for the other transplant related outcomes. These data suggest that HDs might be considered as an alternative option for FA patients with better EFS using unmanipulated grafts.
Substances chimiques
HLA Antigens
0
Types de publication
Journal Article
Multicenter Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
571-579Informations de copyright
© 2021 Wiley Periodicals LLC.
Références
Fanconi G. Familial constitutional panmyelocytopathy, Fanconi's anemia (F.A.). I. Clinical aspects. Semin Hematol. 1967;4(3):233-240.
Butturini A, Gale RP, Verlander PC, Adler-Brecher B, Gillio AP, Auerbach AD. Hematologic abnormalities in Fanconi anemia: an International Fanconi Anemia Registry study. Blood. 1994;84(5):1650-1655.
Kutler DI, Singh B, Satagopan J, et al. A 20-year perspective on the International Fanconi Anemia Registry (IFAR). Blood. 2003;101(4):1249-1256.
Wagner JE, MacMillan ML, Auerbach AD. Hematopoietic cell transplantation for Fanconi's anemia. In: Appelbaum FR, Forman SJ, Negrin RS, Blume KG, eds. Thomas' Hematopoietic Cell Transplantation. 4th ed. Oxford: Blackwell Publishing Ltd; 2008:1178-1199.
Gluckman E, Auerbach AD, Horowitz MM, et al. Bone marrow transplantation for Fanconi anemia. Blood. 1995;86(7):2856-2862.
Guardiola P, Pasquini R, Dokal I, et al. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation. Blood. 2000;95(2):422-429.
Wagner JE, Eapen M, MacMillan ML, et al. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood. 2007;109(5):2256-2262.
Locatelli F, Zecca M, Pession A, et al. The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: a report from the Italian pediatric group. Haematologica. 2007;92(10):1381-1388.
Deeg HJ, Socié G, Schoch G, et al. Malignancies after marrow transplantation for aplastic anemia and fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients. Blood. 1996;87(1):386-392.
Tan PL, Wagner JE, Auerbach AD, Defor TE, Slungaard A, Macmillan ML. Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation. Pediatr Blood Cancer. 2006;46(5):630-636.
Gluckman E, Rocha V, Ionescu I, et al. Results of unrelated cord blood transplant in Fanconi anemia patients: risk factor analysis for engraftment and survival. Biol Blood Marrow Transplant. 2007;13(9):1073-1082.
Peffault de Latour R, Porcher R, Dalle JH, et al. Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European Group for Blood and Marrow Transplantation experience. Blood. 2013;122(26):4279-4286.
Zecca M, Strocchio L, Pagliara D, et al. HLA-haploidentical T cell-depleted allogeneic hematopoietic stem cell transplantation in children with Fanconi anemia. Biol Blood Marrow Transplant. 2014;20(4):571-576.
Bierings M, Bonfim C, Peffault De Latour R, et al. Transplant results in adults with Fanconi anaemia. Br J Haematol. 2018;180(1):100-109.
MacMillan ML, DeFor TE, Young JA, et al. Alternative donor hematopoietic cell transplantation for Fanconi anemia. Blood. 2015;125(24):3798-3804.
Smetsers SE, Smiers FJ, Bresters D, Sonnevelt MC, Bierings MB. Four decades of stem cell transplantation for Fanconi anaemia in the Netherlands. Br J Haematol. 2016;174(6):952-961.
Ebens CL, DeFor TE, Tryon R, Wagenr JE, MacMillan ML. Comparable outcomes after HLA-matched sibling and alternative donor hematopoietic cell transplantation for children with Fanconi anemia and severe aplastic anemia. Biol Blood Marrow Transplant. 2018;24(4):765-771.
Anur P, Friedman DN, Sklar C, et al. Late effects in patients with Fanconi anemia following allogeneic hematopoietic stem cell transplantation from alternative donors. Bone Marrow Transplant. 2016;51(7):938-944.
Chaudhury S, Auerbach AD, Kernan NA, et al. Fludarabine-based cytoreductive regimen and T-cell-depleted grafts from alternative donors for the treatment of high-risk patients with Fanconi anaemia. Br J Haematol. 2008;140(6):644-655.
Mehta PA, Davies SM, Leemhuis T, et al. Radiation-free, alternative-donor HCT for Fanconi anemia patients: results from a prospective multi-institutional study. Blood. 2017;129(16):2308-2315.
Rossi G, Giorgiani G, P Comoli P, et al. Successful T-cell-depleted, related haploidentical peripheral blood stem cell transplantation in a patient with Fanconi anaemia using a fludarabine-based preparative regimen without radiation. Bone Marrow Transplant. 2003;31(6):437-440.
Mitchell R, Cole T, Shaw PJ, Mechinaud F, O'Brien T, Fraser C. TCR α+β+/CD19+ cell-depleted hematopoietic stem cell transplantation for pediatric patients. Pediatr Transplant. 2019;23(6):e13517.
Bonfim C, Ribeiro L, Nichele S, et al. Haploidentical bone marrow transplantation with post-transplant cyclophosphamide for children and adolescents with Fanconi anemia. Biol Blood Marrow Transplant. 2017;23(2):310-317.
Uppuluri R, Swaminathan VV, Ramanan KM, et al. Haploidentical stem cell transplantation with post-transplant cyclophosphamide in Fanconi anemia: improving outcomes with improved supportive care in India. Biol Blood Marrow Transplant. 2020;26(12):2292-2298.
Elhasid R, Ben Arush MW, Katz T, et al. Successful haploidentical bone marrow transplantation in Fanconi anemia. Bone Marrow Transplant. 2000;26(11):1221-1223.
Bertaina A, Merli P, Rutella S, et al. HLA-haploidentical stem cell transplantation after removal of alphabeta+ T and B cells in children with nonmalignant disorders. Blood. 2014;124(5):822-826.
Przepiorka D, Weisdorf D, Martin P, et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant. 1995;15(6):825-828.
Shulman HM, Sullivan KM, Weiden PL, et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med. 1980;69(82):204-217.
MacMillan ML, Weisdorf DJ, Wagner JE, et al. Response of 443 patients to steroids as primary therapy for acute graft-versus-host disease: comparison of grading systems. Biol Blood Marrow Transplant. 2002;8(7):387-394.
Ruggeri A, de Latour RP, Rocha V, et al. Double cord blood transplantation in patients with high risk bone marrow failure syndromes. Br J Haematol. 2008;143(3):404-408.
Ayas M, Al-Seraihi A, El-Solh H, et al. The Saudi experience in fludarabine-based conditioning regimens in patients with Fanconi anemia undergoing stem cell transplantation: excellent outcome in recipients of matched related stem cells but not in recipients of unrelated cord blood stem cells. Biol Blood Marrow Transplant. 2012;18(4):627-632.
Pagliuca S, Ruggeri A, Régis Peffault de Latour R. Cord blood transplantation for bone marrow failure syndromes: state of art. Stem Cell Investig. 2019;5(6):39. https://doi.org/10.21037/sci.2019.10.04.