Hematopoietic cell transplantation in severe combined immunodeficiency : The SCETIDE 2006-2014 European cohort
Lankester, Arjan C. (Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology. Willem-Alexander Children's Hospital. Leiden University Medical Center)
Neven, Benedicte (Institut Imagine. INSERM UMR1163. Laboratory of Immunogenetics of Pediatric Autoimmune Diseases)
Mahlaoui, Nizar (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))
von Asmuth, Erik G.J. (Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology. Willem-Alexander Children's Hospital. Leiden University Medical Center)
Courteille, Virginie (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))
Alligon, Mikael (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))
Albert, Michael H. (Dr von Haunersches University Children's Hospital)
Badell Serra, Isabel (Institut d'Investigació Biomèdica Sant Pau)
Bader, Peter (Department for Children and Adolescents Medicine. Division for Stem Cell Transplantation and Immunology. University Hospital Frankfurt)
Balashov, Dmitry (Department for Hematopoietic Stem Cell Transplantation. Dmitriy Rogachev National Center for Pediatric Hematology. Oncology and Immunology)
Beier, Rita (Klinik für Pädiatrische Hämatologie und Onkologie. Hannover Medical School)
Bertrand, Yves (Institut d'Hématologie et d'Oncologie Pédiatrique. Hospices Civils de Lyon and Université Claude Bernard Lyon 1)
Blanche, Stephane (Unité d'Immuno-hematologie et Rhumatologie Pédiatrique. Hôpital Universitaire Necker-Enfants Malades. Assistance Publique-Hôpitaux de Paris (AP-HP))
Bordon, Victoria (Department of Pediatric Hemato-oncology and Stem Cell Transplant. Ghent University Hospital)
Bredius, Robert G. (Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology. Willem-Alexander Children's Hospital. Leiden University Medical Center)
Cant, Andrew (Translational and Clinical Research Institute. Newcastle University. and the Paediatric Haematopoietic Stem Cell Transplant Unit. Great North Children's Hospital)
Cavazzana, Marina (Laboratory of Genomic Dynamics in the Immune System. Institut Imagine. INSERM UMR1163)
Díaz de Heredia, Cristina (Vall d'Hebron Institut d'Oncologia)
Dogu, Figen (Department of PIA and the BMT Unit. Ankara University)
Ehlert, Karoline (Department of Pediatric Hematology and Oncology. University of Greifswald)
Entz-Werle, Natacha (Pediatric Onco-hematology Department-Pediatrics III. University Hospital of Strasbourg)
Fasth, Anders (Department of Pediatrics. Institute of Clinical Sciences. University of Gothenburg)
Ferrua, Francesca (Pediatric Immunohematology and Bone Marrow Transplantation Unit and the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget). IRCCS San Raffaele Scientific Institute)
Ferster, Alina (Department of Hemato-oncology. Hôpital Universitaire des Enfants Reine Fabiola)
Formankova, Renata (Department of Pediatric Hematology and Oncology. Teaching Hospital Motol. 2nd Medical School. Charles University Motol)
Friedrich, Wilhelm (Department of Pediatrics. University Medical Center Ulm)
Gonzalez-Vicent, Marta (Hospital Infantil Universitario Niño Jesús (Madrid))
Gozdzik, Jolanta (Department of Clinical Immunology and Transplantation. Jagiellonian University Medical College)
Güngör, Tayfun (Department of Hematology. Oncology. Immunology. Gene Therapy and Stem Cell Transplantation. and Children's Research Center (CRC). University Children's Hospital)
Hoenig, Manfred (Department of Pediatrics. University Medical Center Ulm)
Ikinciogullari, Aydan (Department of PIA and the BMT Unit. Ankara University)
Kalwak, Krzysztof (Department of Pediatric Hematology. Oncology. and BMT. Wroclaw Medical University)
Kansoy, Savas (Department of Pediatric Hematology and Oncology. Ege University Hospital)
Kupesiz, Alphan (Department of Pediatrics. Hematology. and Oncology. Akdeniz University School of Medicine)
Lanfranchi, Arnalda (Diagnostic Department. Stem Cell Laboratory. Section of Hematology and Blood Coagulation. Clinical Chemistry Laboratory. ASST Spedali Civili)
Lindemans, Caroline A. (Department of Pediatrics. University Medical Center Utrecht. University of Utrecht)
Meisel, Roland (Department of Pediatric Oncology. Hematology. and Clinical Immunology. Medical Faculty. Division of Pediatric Stem Cell Therapy. Heinrich-Heine-University)
Michel, Gerard (Service d'Hématologie Immunologie Oncologie Pédiatrique. CHU La Timone)
Miranda, Nuno A. A. (BMT Unit. Instituto Português de Oncologia de Lisboa)
Moraleda, Jose (Instituto Murciano de Investigación Biosanitaria)
Moshous, Despina (Laboratory of Genomic Dynamics in the Immune System. Institut Imagine. INSERM UMR1163)
Pichler, Herbert (Department of Stem Cell Transplantation. Children's Cancer Institute. St Anna Hospital)
Rao, Kanchan (Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health)
Sedlacek, Petr (Department of Pediatric Hematology and Oncology. Teaching Hospital Motol. 2nd Medical School. Charles University Motol)
Slatter, Mary (Translational and Clinical Research Institute. Newcastle University. and the Paediatric Haematopoietic Stem Cell Transplant Unit. Great North Children's Hospital)
Soncini, Elena (Pediatric Oncohaematology and BMT Unit. Children's Hospital Brescia)
Speckmann, Carsten (Department of Pediatric Hematology and Oncology. Center for Pediatrics and Adolescent Medicine. Institute for Immunodeficiency. Center for Chronic Immunodeficiency (CCI). Faculty of Medicine. Medical Center. University of Freiburg)
Sundin, Mikael (Section of Pediatric Hematology. Immunology. and HCT. Astrid Lindgren Children's Hospital. Karolinska University Hospital. and Division of Pediatrics. CLINTEC. Karolinska Institutet)
Toren, Amos (Paediatric Hemato-oncology and BMT. Sheba Medical Center)
Vettenranta, Kim (University of Helsinki and Children's Hospital. University of Helsinki)
Worth, Austen (Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health)
Yeşilipek, Mehmet A. (Pediatric Hematology. Oncology. and Pediatric Stem Cell Transplantation Unit. Medicalpark Antalya & Göztepe Hospitals)
Zecca, Marco (Pediatric Hematology/Oncology. Fondazione IRCCS Policlinico San Matteo)
Porta, Fulvio (Pediatric Oncohaematology and BMT Unit. Children's Hospital Brescia)
Schulz, Ansgar (Department of Pediatrics. University Medical Center Ulm)
Veys, Paul (Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health)
Fischer, Alain (Université de Paris)
Gennery, Andrew R. (Translational and Clinical Research Institute. Newcastle University. and the Paediatric Haematopoietic Stem Cell Transplant Unit. Great North Children's Hospital)

Fecha:	2022
Resumen:	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/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.
Nota:	Altres ajuts: Dutch Rare Disease Foundation/Stichting Zeldzame Ziekten Fonds.
Derechos:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Lengua:	Anglès
Documento:	Article ; recerca ; Versió publicada
Materia:	SCID ; Genetic subgroups ; Conditioning ; Pretransplantation infections ; Immune reconstitution
Publicado en:	The Journal of Allergy and Clinical Immunology, Vol. 149 Núm. 5 (may 2022) , p. 1744-1754.e8, ISSN 1097-6825

DOI: 10.1016/j.jaci.2021.10.017
PMID: 34718043

19 p, 3.2 MB

El registro aparece en las colecciones:
Documentos de investigación > Documentos de los grupos de investigación de la UAB > Centros y grupos de investigación (producción científica) > Ciencias de la salud y biociencias > Institut de Recerca Sant Pau
Artículos > Artículos de investigación
Artículos > Artículos publicados