HLA-haploidentical stem cell transplantation in children with inherited bone marrow failure syndromes: A retrospective analysis on behalf of EBMT severe aplastic Anemia and pediatric diseases working parties.
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:
18 Mar 2024
18 Mar 2024
Historique:
revised:
23
02
2024
received:
11
10
2023
accepted:
04
03
2024
medline:
18
3
2024
pubmed:
18
3
2024
entrez:
18
3
2024
Statut:
aheadofprint
Résumé
Haploidentical stem cell transplantation (haplo-SCT) represents the main alternative for children with inherited bone marrow failure syndrome (I-BMF) lacking a matched donor. This retrospective study, conducted on behalf of the EBMT SAAWP and PDWP, aims to report the current outcomes of haplo-SCT in I-BMFs, comparing the different in vivo and ex vivo T-cell depletion approaches. One hundred and sixty-two I-BMF patients who underwent haplo-SCT (median age 7.4 years) have been registered. Fanconi Anemia was the most represented diagnosis (70.1%). Based on different T-cell depletion (TCD) approaches, four categories were identified: (1) TCRαβ
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.
Références
Wilson DB, Link DC, Mason PJ, Bessler M. Inherited bone marrow failure syndromes in adolescents and young adults. Ann Med. 2014;46(6):353-363. doi:10.3109/07853890.2014.915579
Dokal I, Tummala H, Vulliamy T. Inherited bone marrow failure in the pediatric patient. Blood. 2022;140(6):556-570. doi:10.1182/blood.2020006481
Alter BP, Giri N, Savage SA, Rosenberg PS. Cancer in the National Cancer Institute inherited bone marrow failure syndrome cohort after fifteen years of follow-up. Haematologica. 2018;103(1):30-39. doi:10.3324/haematol.2017.178111
Shimamura A, Alter BP. Pathophysiology and management of inherited bone marrow failure syndromes. Blood Rev. 2010;24(3):101-122.
Bluteau O, Sebert M, Leblanc T, et al. A landscape of germ line mutations in a cohort of inherited bone marrow failure patients. Blood. 2018;131(7):717-732. doi:10.1182/blood-2017-09-806489
Dalle JH, Peffault de Latour R. Allogeneic hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Int J Hematol. 2016;103(4):373-379. doi:10.1007/s12185-016-1951-0
Pierri F, Faraci M, Giardino S, Dufour C. Hematopoietic stem cell transplantation for classical inherited bone marrow failure syndromes: an update. Expert Rev Hematol. 2021 Oct;14(10):911-925. doi:10.1080/17474086.2021.1977119
Benajiba L, Salvado C, Dalle JH, et al. HLA-matched related-donor HSCT in Fanconi anemia patients conditioned with cyclophosphamide and fludarabine. Blood. 2015;125(2):417-418. doi:10.1182/blood-2014-10-605113
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 Dec 19;122(26):4279-4286. doi:10.1182/blood-2013-01-479733
Fioredda F, Iacobelli S, Korthof ET, et al. Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita. Br J Haematol. 2018;183(1):110-118. doi:10.1111/bjh.15495
Miano M, Eikema DJ, de la Fuente J, et al. Stem cell transplantation for diamond-Blackfan Anemia. A retrospective study on behalf of the severe aplastic Anemia working Party of the European Blood and Marrow Transplantation Group (EBMT). Transplant Cell Ther. 2021;27(3):274.e1-274.e5. doi:10.1016/j.jtct.2020.12.024
Cancio M, Hebert K, Kim S, et al. Outcomes in hematopoietic stem cell transplantation for congenital Amegakaryocytic thrombocytopenia. Transplant Cell Ther. 2022;28(2):101.e1-101.e6. doi:10.1016/j.jtct.2021.10.009
Giardino S, de Latour RP, Aljurf M, et al. Outcome of patients with Fanconi anemia developing myelodysplasia and acute leukemia who received allogeneic hematopoietic stem cell transplantation: a retrospective analysis on behalf of EBMT group. Am J Hematol. 2020;95(7):809-816. doi:10.1002/ajh.25810
Inamoto Y, Shah NN, Savani BN, et al. Secondary solid cancer screening following hematopoietic cell transplantation. Bone Marrow Transplant. 2015;50(8):1013-1023. doi:10.1038/bmt.2015.63
Peffault de Latour R, Peters C, Gibson B, et al. Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Bone Marrow Transplant. 2015;50(9):1168-1172. doi:10.1038/bmt.2015.117
Zubicaray J, Pagliara D, Sevilla J, et al. Haplo-identical or mismatched unrelated donor hematopoietic cell transplantation for Fanconi anemia: results from the severe aplastic Anemia working party of the EBMT. Am J Hematol. 2021;96(5):571-579. doi:10.1002/ajh.26135
Mallhi KK, Srikanthan MA, Baker KK, et al. HLA-haploidentical hematopoietic cell transplantation for treatment of nonmalignant diseases using nonmyeloablative conditioning and post-transplant cyclophosphamide. Biol Blood Marrow Transplant. 2020;26(7):1332-1341. doi:10.1016/j.bbmt.2020.03.018
Bertaina A, Merli P, Rutella S, et al. HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders. Blood. 2014;124(5):822-826. doi:10.1182/blood-2014-03-563817
Bacigalupo A, Ballen K, Rizzo D, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009;15:1628-1633.
Aversa F, Tabilio A, Velardi A, et al. Hematopoietic stem cell transplantation from alternative donors for high-risk acute leukemia: the haploidentical option. Curr Stem Cell Res Ther. 2007 Jan;2(1):105-112. doi:10.2174/157488807779316973
Ciceri F, Labopin M, Aversa F, et al. A survey of fully haploidentical hematopoietic stem cell transplantation in adults with high-risk acute leukemia: a risk factor analysis of outcomes for patients in remission at transplantation. Blood. 2008;112(9):3574-3581. doi:10.1182/blood-2008-02-140095
Reisner Y, Hagin D, Martelli MF. Haploidentical hematopoietic transplantation: current status and future perspectives. Blood. 2011;118(23):6006-6017. doi:10.1182/blood-2011-07-338822
Li Pira G, Malaspina D, Girolami E, et al. Selective depletion of αβ T cells and B cells for human leukocyte antigen-haploidentical hematopoietic stem cell transplantation. A three-year follow-up of procedure efficiency. Biol Blood Marrow Transplant. 2016;22(11):2056-2064. doi:10.1016/j.bbmt.2016.08.006
Luznik L, Jalla S, Engstrom LW, Iannone R, Fuchs EJ. Durable engraftment of major histocompatibility complex-incompatible cells after nonmyeloablative conditioning with fludarabine, low-dose total body irradiation, and posttransplantation cyclophosphamide. Blood. 2001;98(12):3456-3464. doi:10.1182/blood.v98.12.3456
Wolff SN. Second hematopoietic stem cell transplantation for the treatment of graft failure, graft rejection or relapse after allogeneic transplantation. Bone Marrow Transplant. 2002;29:545-552.
Teltschik H-M, Heinzelmann F, Gruhn B, et al. Treatment of graft failure with TNI-based reconditioning and haploidentical stem cells in paediatric patients. Br J Haematol. 2016;175:115-122. doi:10.1111/bjh.14190
Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18(4):295-304.
Jagasia MH, Greinix HT, Arora M, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. The 2014 diagnosis and staging working group report. Biol Blood Marrow Transplant. 2015;21(3):389-401.
Giardino S, Pierri F, Faraci M. How to optimize outcome of patients undergoing HLA-matched related haematopoietic stem cell transplantation in acquired and inherited bone marrow failure syndromes. Br J Haematol. 2023;203:158-160. doi:10.1111/bjh.19047
Luznik L, O'Donnell PV, Symons HJ, et al. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008;14:641-650. doi:10.1016/j.bbmt.2008.03.005
Eichholz T, Döring M, Giardino S, et al. Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas. Front Oncol. 2023;13:1064190. doi:10.3389/fonc.2023.1064190
Flaadt T, Ladenstein RL, Ebinger M, et al. Anti-GD2 antibody Dinutuximab Beta and low-dose interleukin 2 after haploidentical stem-cell transplantation in patients with relapsed neuroblastoma: a multicenter, phase I/II trial. J Clin Oncol. 2023;41(17):3135-3148. doi:10.1200/JCO.22.01630
Schumm M, Lang P, Bethge W, et al. Depletion of T-cell receptor alpha/beta and CD19 positive cells from apheresis products with the CliniMACS device. Cytotherapy. 2013 Oct;15(10):1253-1258. doi:10.1016/j.jcyt.2013.05.014
Peters C, Matthes-Martin S, Fritsch G, et al. Transplantation of highly purified peripheral blood CD34+ cells from HLA-mismatched parental donors in 14 children: evaluation of early monitoring of engraftment. Leukemia. 1999;13(12):2070-2078. doi:10.1038/sj.leu.2401577
Platzbecker U, Ehninger G, Bornhäuser M. Allogeneic transplantation of CD34+ selected hematopoietic cells-clinical problems and current challenges. Leuk Lymphoma. 2004;45(3):447-453. doi:10.1080/10428190310001615684
Kleinschmidt K, Lv M, Yanir A, Palma J, Lang P, Eyrich M. T-cell-replete versus ex vivo T-cell-depleted haploidentical Haematopoietic stem cell transplantation in children with acute lymphoblastic Leukaemia and other Haematological malignancies. Front Pediatr. 2021;9:794541. doi:10.3389/fped.2021.794541
Merli P, Algeri M, Galaverna F, et al. Immune modulation properties of zoledronic acid on TcRγδ T-lymphocytes after TcRαβ/CD19-depleted haploidentical stem cell transplantation: an analysis on 46 pediatric patients affected by acute leukemia. Front Immunol. 2020;11:699. doi:10.3389/fimmu.2020.00699
Arnold DE, MacMath D, Seif AE, et al. Immune reconstitution following TCRαβ/CD19-depleted hematopoietic cell transplantation for hematologic malignancy in pediatric patients. Transplant Cell Ther. 2021;27(2):169.e1-169.e9. doi:10.1016/j.jtct.2020.10.006
Radjocic V, Luznik L. Mechanism of action of posttransplantation cyclophosphamide: more than meets the eye. J Clin Invest. 2019;129:2189-2191. doi:10.1172/JCI128710
Williams L, Cirrone F, Cole K, Abdul-Hay M, Luznik L, Al-Homsi AS. Post-transplantation cyclophosphamide: from HLA-haploidentical to matched-related and matched-unrelated donor blood and marrow transplantation. Front Immunol. 2020;11:636. doi:10.3389/fimmu.2020.00636
Hashem H, Najjar R, Abu-Shanap M, et al. Haploidentical hematopoietic cell transplantation using post-transplant cyclophosphamide for children with non-malignant diseases. J Clin Immunol. 2021;41(8):1754-1761. doi:10.1007/s10875-021-01113-4
Balashov D, Shcherbina A, Maschan M, et al. Single-center experience of unrelated and haploidentical stem cell transplantation with TCRαβ and CD19 depletion in children with primary immunodeficiency syndromes. Biol Blood Marrow Transplant. 2015;21(11):1955-1962. doi:10.1016/j.bbmt.2015.07.008
Merli P, Pagliara D, Galaverna F, et al. TCRαβ/CD19 depleted HSCT from an HLA-haploidentical relative to treat children with different nonmalignant disorders. Blood Adv. 2022;6(1):281-292. doi:10.1182/bloodadvances.2021005628
Giardino S, Bagnasco F, Falco M, et al. Haploidentical stem cell transplantation after TCR-αβ+ and CD19+ cells depletion in children with congenital non-malignant disease. Transplant Cell Ther. 2022;28(7):394.e1-394.e9. doi:10.1016/j.jtct.2022.04.002
Shah RM, Elfeky R, Nademi Z, et al. T-cell receptor αβ+ and CD19+ cell-depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency. J Allergy Clin Immunol. 2018;141(4):1417-1426.e1. doi:10.1016/j.jaci.2017.07.008 Epub 2017 Aug 3. Erratum in: J Allergy Clin Immunol. 2019 May;143(5):1977. PMID: 28780238.
Ayas M, Siddiqui K, Al-Jefri A, et al. Successful outcome in patients with Fanconi Anemia undergoing T cell-replete mismatched related donor hematopoietic cell transplantation using reduced-dose cyclophosphamide post-transplantation. Biol Blood Marrow Transplant. 2019 Nov;25(11):2217-2221. doi:10.1016/j.bbmt.2019.07.010
Thakar MS, Bonfim C, Walters MC, et al. Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia. Bone Marrow Transplant. 2017;52(4):570-573. doi:10.1038/bmt.2016.301
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 Feb;23(2):310-317. doi:10.1016/j.bbmt.2016.11.006
Strocchio L, Pagliara D, Algeri M, et al. HLA-haploidentical TCRαβ+/CD19+-depleted stem cell transplantation in children and young adults with Fanconi anemia. Blood Adv. 2021;5(5):1333-1339. doi:10.1182/bloodadvances.2020003707
Handgretinger R, Arendt AM, Maier CP, Lang P. Ex vivo and in vivo T-cell depletion in allogeneic transplantation: towards less or non-cytotoxic conditioning regimens. Expert Rev Clin Immunol. 2022;18(12):1285-1296. doi:10.1080/1744666X.2022.2134857
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. doi:10.1016/j.bbmt.2020.08.019
Uppuluri R, Sivasankaran M, Patel S, et al. Haploidentical stem cell transplantation with post-transplant cyclophosphamide for primary immune deficiency disorders in children: challenges and outcome from a tertiary Care Center in South India. J Clin Immunol. 2019;39(2):182-187. doi:10.1007/s10875-019-00600-z
Ricci A, Jin Z, Bourgeois W, et al. Financial impact of post-transplant complications among children undergoing allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2020;55(7):1421-1429. doi:10.1038/s41409-020-0899-0
Lima ACM, Bonfim C, Getz J, et al. The impact of donor-specific anti-human leukocyte antigen antibodies in salvage haploidentical hematopoietic cell transplantation with posttransplant cyclophosphamide in patients with nonmalignant disorders. HLA. 2021;97(6):493-504. doi:10.1111/tan.14277
Dietz AC, Savage SA, Vlachos A, et al. Late effects screening guidelines after hematopoietic cell transplantation for inherited bone marrow failure syndromes: consensus statement from the second pediatric blood and marrow transplant consortium international conference on late effects after pediatric HCT. Biol Blood Marrow Transplant. 2017;23(9):1422-1428. doi:10.1016/j.bbmt.2017.05.022
Svahn J, Bagnasco F, Cappelli E, et al. Somatic, hematologic phenotype, long-term outcome, and effect of hematopoietic stem cell transplantation. An analysis of 97 Fanconi anemia patients from the Italian national database on behalf of the marrow failure study group of the AIEOP (Italian Association of Pediatric Hematology-Oncology). Am J Hematol. 2016;91(7):666-671. doi:10.1002/ajh.24373