Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplant Cyclophosphamide for Primary Immunodeficiencies and Inherited Disorders in Children

Biology of Blood and Marrow Transplantation - Tập 25 Số 7 - Trang 1363-1373 - 2019
Bénédicte Neven1,2,3, Jean‐Sébastien Diana4,2,3, Martin Castelle3, Alessandra Magnani4,1,2, Jérémie Rosain1,2,5, Fabien Touzot4,1,2, Baptiste Moreira6, Marie‐Louise Frémond1,2,3, Coralie Briand2,3, Matthieu Bendavid2,3, Romain Lévy2,3, Guillaume Morelle2,3, Marc Vincent1,2, Elsa Magrin4,1,2, Philippe Bourget7, Lucienne Chatenoud6, Capucine Pïcard1,2,3,5, Alain Fischer8,1,2,3, Despina Moshous1,2,3, Stéphane Blanche2,3
1INSERM U1163 and Institut Imagine, Paris, France
2Paris-Descartes University, Sorbonne Paris Cité, Paris, France
3Pediatric Hematology-Immunology and Rheumatology Unit, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
4Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
5Study Center for Primary Immunodeficiencies, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
6Immunology Laboratory, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
7Functional explorations Unit, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
8College de France, Paris, France

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Gragert, 2014, HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. Registry Abstract, N Engl J Med, 371, 339, 10.1056/NEJMsa1311707

Gennery, 2010, Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?, J Allergy Clin Immunol, 126, 602, 10.1016/j.jaci.2010.06.015

Locatelli, 2011, Strategies to optimize the outcome of children given T-cell depleted HLA-haploidentical hematopoietic stem cell transplantation, Best Pract Res Clin Haematol, 24, 339, 10.1016/j.beha.2011.04.004

Reisner Y, Kapoor N, Kirkpatrick D, et al. Transplantation for severe combined immunodeficiency with HLA-A, B, D, DR incompatible parental marrow cells fractionated by soybean agglutinin and sheep red blood cells. Blood. 1983;61:341–348

Bethge, 2006, Haploidentical allogeneic hematopoietic cell transplantation in adults with reduced-intensity conditioning and CD3/CD19 depletion: fast engraftment and low toxicity, Exp Hematol, 34, 1746, 10.1016/j.exphem.2006.08.009

Chaleff, 2007, A large-scale method for the selective depletion of αβ T lymphocytes from PBSC for allogeneic transplantation, Cytotherapy, 9, 746, 10.1080/14653240701644000

Locatelli, 2013, Negative depletion of α/β+ T cells and of CD19+ B lymphocytes: a novel frontier to optimize the effect of innate immunity in HLA-mismatched hematopoietic stem cell transplantation, Immunol Lett, 155, 21, 10.1016/j.imlet.2013.09.027

Shah, 2018, T-cell receptor αβ+ and CD19+ cell-depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency, J Allergy Clin Immunol, 141, 1417, 10.1016/j.jaci.2017.07.008

Bertaina, 2014, HLA-haploidentical stem cell transplantation after removal of αβ+T and B cells in children with nonmalignant disorders, Blood, 124, 822, 10.1182/blood-2014-03-563817

Balashov, 2015, 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, 21, 1955, 10.1016/j.bbmt.2015.07.008

Touzot, 2015, CD45RA depletion in HLA-mismatched allogeneic hematopoietic stem cell transplantation for primary combined immunodeficiency: a preliminary study, J Allergy Clin Immunol, 135, 1303, 10.1016/j.jaci.2014.08.019

Bollard, 2016, review series advances in cell-based immune therapeutics in hematology T cells for viral infections after allogeneic hematopoietic stem cell transplant, Blood, 127, 3331, 10.1182/blood-2016-01-628982

Kanakry, 2014, Multi-institutional study of post-transplantation cyclophosphamide as single-agent graft-versus-host disease prophylaxis after allogeneic bone marrow transplantation using myeloablative busulfan and fludarabine conditioning, J Clin Oncol, 32, 3497, 10.1200/JCO.2013.54.0625

O'Donnell P, 2002, Nonmyeloablative bone marrow transplantation from partially HLA-mismatched related donors using posttransplantation cyclophosphamide, Biol Blood Marrow Transplant, 8, 377, 10.1053/bbmt.2002.v8.pm12171484

Brodsky, 2008, Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases, Bone Marrow Transplant, 42, 523, 10.1038/bmt.2008.203

Shah, 2017, Haploidentical related donor hematopoietic stem cell transplantation for dedicator-of-cytokinesis 8 deficiency using post-transplantation cyclophosphamide, Biol Blood Marrow Transplant, 23, 980, 10.1016/j.bbmt.2017.03.016

Klein, 2016, Alternative-donor hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for nonmalignant disorders, Biol Blood Marrow Transplant, 22, 895, 10.1016/j.bbmt.2016.02.001

Bahr, 2016, Haploidentical transplantation with post-transplant cyclophosphamide following reduced-intensity conditioning for osteopetrosis: outcomes in three children, Bone Marrow Transplant, 51, 1546, 10.1038/bmt.2016.180

Rastogi, 2018, Reduced‐toxicity alternate‐donor stem cell transplantation with posttransplant cyclophosphamide for primary immunodeficiency disorders, Pediatr Blood Cancer, 65, 10.1002/pbc.26783

Paci, 2012, Pharmacokinetic behavior and appraisal of intravenous busulfan dosing in infants and older children: the results of a population pharmacokinetic study from a large pediatric cohort undergoing hematopoietic stem-cell transplantation, Ther Drug Monit, 34, 198, 10.1097/FTD.0b013e31824c2f60

Dignan, 2013, BCSH/BSBMT guideline: diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation, Br J Haematol, 163, 444, 10.1111/bjh.12558

Ho VT, Cutler C, Carter S, et al. Blood and Marrow Transplant Clinical Trials Network Toxicity Committee consensus summary: thrombotic microangiopathy after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2005;11:571–575.

Przepiorka, 1995, Consensus conference on acute GVHD grading, Bone Marrow Transplant, 15, 825

Lee SJ, Vogelsang G, Flowers MED, Lee S. Chronic graft-versus-host disease. 2003.

Tischer, 2014, Evaluation of suitable target antigens and immunoassays for high-accuracy immune monitoring of cytomegalovirus and Epstein-Barr virus-specific T cells as targets of interest in immunotherapeutic approaches, J Immunol Methods, 408, 101, 10.1016/j.jim.2014.05.011

Orchard, 2015, Hematopoietic stem cell transplantation for infantile osteopetrosis, Blood, 126, 270, 10.1182/blood-2015-01-625541

Ouachée-Chardin, 2006, Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients, Pediatrics, 117, e743, 10.1542/peds.2005-1789

Hamadani, 2016, Reduced-intensity transplantation for lymphomas using haploidentical related donors versus hla-matched sibling donors: a Center for International Blood and Marrow Transplant research analysis, J Clin Oncol, 34, 3141, 10.1200/JCO.2015.66.3476

Kasamon, 2017, Prospective study of nonmyeloablative, HLA-mismatched unrelated BMT with high-dose posttransplantation cyclophosphamide, Blood Adv, 1, 288, 10.1182/bloodadvances.2016002766

Güngör, 2014, Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study, Lancet, 383, 436, 10.1016/S0140-6736(13)62069-3

Marsh, 2015, Experience with alemtuzumab, fludarabine, and melphalan reduced-intensity conditioning hematopoietic cell transplantation in patients with nonmalignant diseases reveals good outcomes and that the risk of mixed chimerism depends on underlying disease, stem, Biol Blood Marrow Transplant, 21, 1460, 10.1016/j.bbmt.2015.04.009

Morillo-Gutierrez, 2016, Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience, Blood, 128, 440, 10.1182/blood-2016-03-704015

Berger, 2016, Feasibility and outcome of haploidentical hematopoietic stem cell transplantation with post-transplant high-dose cyclophosphamide for children and adolescents with hematologic malignancies: an AIEOP-GITMO retrospective multicenter study, Biol Blood Marrow Transplant, 22, 902, 10.1016/j.bbmt.2016.02.002

Balasubramanian, 2012, Population pharmacokinetics of cyclophosphamide in patients with thalassemia major undergoing HSCT, Bone Marrow Transplant, 47, 1178, 10.1038/bmt.2011.254

Crocchiolo, 2016, The patient's CMV serological status affects clinical outcome after T-cell replete haplo-HSCT and post-transplant cyclophosphamide, Bone Marrow Transplant, 5169, 1134, 10.1038/bmt.2016.69

Slade, 2017, Epidemiology of infections following haploidentical peripheral blood hematopoietic cell transplantation, Transpl Infect Dis, 19, 10.1111/tid.12629

Kosulin, 2018, Intestinal adenovirus shedding before allogeneic stem cell transplantation is a risk factor for invasive infection post-transplant, E Bio Medicine, 28, 114

Hiwarkar, 2018, Management of adenovirus infection in patients after haematopoietic stem cell transplantation: state‐of‐the‐art and real‐life current approach, Rev Med Virol, 28, 10.1002/rmv.1980

Kanakry, 2016, Origin and evolution of the T cell repertoire after posttransplantation cyclophosphamide, Ref Inf JCI Insight, 1, 86252

Marsh, 2017, Clinical research: pediatric pretransplant absolute lymphocyte counts impact the pharmacokinetics of alemtuzumab, Biol Blood Marrow Transplant, 23, 635, 10.1016/j.bbmt.2017.01.071

Admiraal, 2015, Paediatric Blood and Marrow Transplant Program association between anti-thymocyte globulin exposure and CD4+ immune reconstitution in paediatric haemopoietic cell transplantation: a multicentre, retrospective pharmacodynamic cohort analysis, Lancet Haematol, 2, e194, 10.1016/S2352-3026(15)00045-9

Willemsen, 2015, Impact of serotherapy on immune reconstitution and survival outcomes after stem cell transplantations in children: thymoglobulin versus alemtuzumab, Biol Blood Marrow Transplant, 21, 473, 10.1016/j.bbmt.2014.11.674

Chiesa, 2012, Omission of in vivo T-cell depletion promotes rapid expansion of naïve CD4+ cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant, Br J Haematol, 156, 656, 10.1111/j.1365-2141.2011.08994.x

Parikh, 2014, A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases, Biol Blood Marrow Transplant, 20, 326, 10.1016/j.bbmt.2013.11.021