Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort.

Autor: Lankester AC; Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands. Electronic address: a.lankester@lumc.nl., Neven B; Unité d'Immuno-hematologie et Rhumatologie Pédiatrique, Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; Université de Paris, Paris, France; Institut Imagine, INSERM UMR1163, Laboratory of Immunogenetics of Pediatric Autoimmune Diseases, Paris, France., Mahlaoui N; French National Reference Center for Primary Immunodeficiencies (CEREDIH) and European Registry for Stem Cell Transplantation for Primary Immunodeficiencies (SCETIDE), Hôpital Universitaire Necker-Enfants malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France., von Asmuth EGJ; Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands., Courteille V; French National Reference Center for Primary Immunodeficiencies (CEREDIH) and European Registry for Stem Cell Transplantation for Primary Immunodeficiencies (SCETIDE), Hôpital Universitaire Necker-Enfants malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France., Alligon M; French National Reference Center for Primary Immunodeficiencies (CEREDIH) and European Registry for Stem Cell Transplantation for Primary Immunodeficiencies (SCETIDE), Hôpital Universitaire Necker-Enfants malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France., Albert MH; Dr von Haunersches University Children's Hospital, Munich, Germany., Serra IB; Hospital Clínic, Sant Creu i Sant Pau Hospital, Bone Marrow Transplantation Unit, Barcelona, Spain., Bader P; Department for Children and Adolescents Medicine, Division for Stem Cell Transplantation and Immunology, University Hospital Frankfurt, Frankfurt, Germany., Balashov D; Department for Hematopoietic Stem Cell Transplantation, Dmitriy Rogachev National Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia., Beier R; Klinik für Pädiatrische Hämatologie und Onkologie, Hannover Medical School, Hannover, Germany., Bertrand Y; Institut d'Hématologie et d'Oncologie Pédiatrique, Hospices Civils de Lyon and Université Claude Bernard Lyon 1, Lyon, France., Blanche S; Unité d'Immuno-hematologie et Rhumatologie Pédiatrique, Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France., Bordon V; Department of Pediatric Hemato-oncology and Stem Cell Transplant, Ghent University Hospital, Ghent, Belgium., Bredius RG; Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands., Cant A; Translational and Clinical Research Institute, Newcastle University, and the Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom., Cavazzana M; Université de Paris, Paris, France; Paris Biotherapy Department, Necker Children's Hospital Assistance, Paris, France; Biotherapy Clinical Investigation Center, Assistance Publique Hopitaux de Paris, INSERM, Paris, France; Laboratory of Genomic Dynamics in the Immune System, Institut Imagine, INSERM UMR1163, Paris, France., Diaz-de-Heredia C; Department of Pediatric Oncology and Hematology, and Hematopoietic Stem Cell Transplantation, Hospital Universitario Vall d'Hebron, Barcelona, Spain., Dogu F; Department of PIA and the BMT Unit, Ankara University, Ankara, Turkey., Ehlert K; Department of Pediatric Hematology and Oncology, Universitätsklinikum Münster, Münster, Germany; Department of Pediatric Hematology and Oncology, University of Greifswald, Greifswald, Germany., Entz-Werle N; Pediatric Onco-hematology Department-Pediatrics III, University Hospital of Strasbourg, Strasbourg, France., Fasth A; Department of Pediatrics, Institute of Clinical Sciences, University of Gothenburg, Gothenburg, Sweden., Ferrua F; Pediatric Immunohematology and Bone Marrow Transplantation Unit and the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy., Ferster A; Department of Hemato-oncology, Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium., Formankova R; Department of Pediatric Hematology and Oncology, Teaching Hospital Motol, 2nd Medical School, Charles University Motol, Prague, Czech Republic., Friedrich W; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany., Gonzalez-Vicent M; Hematopoietic Stem Cell Transplantation and Cellular Therapy Unit, Hospital Infantil Universitario 'Niño Jesus,' Madrid, Spain., Gozdzik J; Department of Clinical Immunology and Transplantation, Jagiellonian University Medical College, Krakow, Poland., Güngör T; Department of Hematology, Oncology, Immunology, Gene Therapy and Stem Cell Transplantation, and Children's Research Center (CRC), University Children's Hospital, Zurich, Switzerland., Hoenig M; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany., Ikinciogullari A; Department of PIA and the BMT Unit, Ankara University, Ankara, Turkey., Kalwak K; Department of Pediatric Hematology, Oncology, and BMT, Wroclaw Medical University, Wroclaw, Poland., Kansoy S; Department of Pediatric Hematology and Oncology, Ege University Hospital, Izmir, Turkey., Kupesiz A; Department of Pediatrics, Hematology, and Oncology, Akdeniz University School of Medicine, Antalya, Turkey., Lanfranchi A; Diagnostic Department, Stem Cell Laboratory, Section of Hematology and Blood Coagulation, Clinical Chemistry Laboratory, ASST Spedali Civili, Brescia, Italy., Lindemans CA; Department of Stem Cell Transplantation, Princess Maxima Center for Pediatric Oncology, Utrecht, The Netherlands; Department of Pediatrics, University Medical Center Utrecht, University of Utrecht, Utrecht, The Netherlands., Meisel R; Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Division of Pediatric Stem Cell Therapy, Heinrich-Heine-University, Düsseldorf, Germany., Michel G; Service d'Hématologie Immunologie Oncologie Pédiatrique, CHU La Timone, Marseille, France., Miranda NAA; BMT Unit, Instituto Português de Oncologia de Lisboa, Lisbon, Portugal., Moraleda J; Department of Hematology and Hemotherapy, Hospital Virgen de la Arrixaca-IMIB, Murcia, Spain., Moshous D; Unité d'Immuno-hematologie et Rhumatologie Pédiatrique, Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; Université de Paris, Paris, France; Laboratory of Genomic Dynamics in the Immune System, Institut Imagine, INSERM UMR1163, Paris, France., Pichler H; Department of Stem Cell Transplantation, Children's Cancer Institute, St Anna Hospital, Vienna, Austria., Rao K; Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health, London, United Kingdom., Sedlacek P; Department of Pediatric Hematology and Oncology, Teaching Hospital Motol, 2nd Medical School, Charles University Motol, Prague, Czech Republic., Slatter M; Translational and Clinical Research Institute, Newcastle University, and the Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom., Soncini E; Pediatric Oncohaematology and BMT Unit, Children's Hospital Brescia, Brescia, Italy., Speckmann C; Department of Pediatric Hematology and Oncology, Center for Pediatrics and Adolescent Medicine, and Institute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Faculty of Medicine, Medical Center, University of Freiburg, Freiburg, Germany., Sundin M; Section of Pediatric Hematology, Immunology, and HCT, Astrid Lindgren Children's Hospital, Karolinska University Hospital, and Division of Pediatrics, CLINTEC, Karolinska Institutet, Stockholm, Sweden., Toren A; Paediatric Hemato-oncology and BMT, Sheba Medical Center, Tel-Hashomer, Israel., Vettenranta K; University of Helsinki and Children's Hospital, University of Helsinki, Helsinki, Finland., Worth A; Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health, London, United Kingdom., Yeşilipek MA; Pediatric Hematology, Oncology, and Pediatric Stem Cell Transplantation Unit, Medicalpark Antalya & Göztepe Hospitals, Antalya, Turkey., Zecca M; Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy., Porta F; Pediatric Oncohaematology and BMT Unit, Children's Hospital Brescia, Brescia, Italy., Schulz A; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany., Veys P; Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health, London, United Kingdom., Fischer A; Unité d'Immuno-hematologie et Rhumatologie Pédiatrique, Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; Université de Paris, Paris, France., Gennery AR; Translational and Clinical Research Institute, Newcastle University, and the Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom.
Jazyk: angličtina
Zdroj: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2022 May; Vol. 149 (5), pp. 1744-1754.e8. Date of Electronic Publication: 2021 Oct 27.
DOI: 10.1016/j.jaci.2021.10.017
Abstrakt: Background: Hematopoietic stem cell transplantation (HSCT) represents a curative treatment for patients with severe combined immunodeficiency (SCID), a group of monogenic immune disorders with an otherwise fatal outcome.
Objective: We performed a comprehensive multicenter analysis of genotype-specific HSCT outcome, including detailed analysis of immune reconstitution (IR) and the predictive value for clinical outcome.
Methods: HSCT outcome was studied in 338 patients with genetically confirmed SCID who underwent transplantation in 2006-2014 and who were registered in the SCETIDE registry. In a representative subgroup of 152 patients, data on IR and long-term clinical outcome were analyzed.
Results: Two-year OS was similar with matched family and unrelated donors and better than mismatched donor HSCT (P < .001). The 2-year event-free survival (EFS) was similar in matched and mismatched unrelated donor and less favorable in mismatched related donor (MMRD) HSCT (P < .001). Genetic subgroups did not differ in 2-year OS (P = .1) and EFS (P = .073). In multivariate analysis, pretransplantation infections and use of MMRDs were associated with less favorable OS and EFS. With a median follow-up of 6.2 years (range, 2.0-11.8 years), 73 of 152 patients in the IR cohort were alive and well without Ig dependency. IL-2 receptor gamma chain/Janus kinase 3/IL-7 receptor-deficient SCID, myeloablative conditioning, matched donor HSCT, and naive CD4 T lymphocytes >0.5 × 10 e3 /μL at +1 year were identified as independent predictors of favorable clinical and immunologic outcome.
Conclusion: Recent advances in HSCT in SCID patients have resulted in improved OS and EFS in all genotypes and donor types. To achieve a favorable long-term outcome, treatment strategies should aim for optimal naive CD4 T lymphocyte regeneration.
(Copyright © 2021 The Authors. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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