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Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort
AC. Lankester, B. Neven, N. Mahlaoui, EGJ. von Asmuth, V. Courteille, M. Alligon, MH. Albert, IB. Serra, P. Bader, D. Balashov, R. Beier, Y. Bertrand, S. Blanche, V. Bordon, RG. Bredius, A. Cant, M. Cavazzana, C. Diaz-de-Heredia, F. Dogu, K....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, multicentrická studie, práce podpořená grantem
- MeSH
- kohortové studie MeSH
- lidé MeSH
- nepříbuzný dárce MeSH
- příprava pacienta k transplantaci metody MeSH
- těžká kombinovaná imunodeficience * genetika terapie MeSH
- transplantace hematopoetických kmenových buněk * metody MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- práce podpořená grantem MeSH
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 × 10e3/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.
Biotherapy Clinical Investigation Center Assistance Publique Hopitaux de Paris INSERM Paris France
BMT Unit Instituto Português de Oncologia de Lisboa Lisbon Portugal
Department of Hemato oncology Hôpital Universitaire des Enfants Reine Fabiola Brussels Belgium
Department of Hematology and Hemotherapy Hospital Virgen de la Arrixaca IMIB Murcia Spain
Department of Pediatric Hematology and Oncology Ege University Hospital Izmir Turkey
Department of Pediatric Hematology and Oncology Universitätsklinikum Münster Münster Germany
Department of Pediatric Hematology and Oncology University of Greifswald Greifswald Germany
Department of Pediatric Hematology Oncology and BMT Wroclaw Medical University Wroclaw Poland
Department of Pediatrics Institute of Clinical Sciences University of Gothenburg Gothenburg Sweden
Department of Pediatrics University Medical Center Ulm Ulm Germany
Department of PIA and the BMT Unit Ankara University Ankara Turkey
Department of Stem Cell Transplantation Children's Cancer Institute St Anna Hospital Vienna Austria
Dr von Haunersches University Children's Hospital Munich Germany
French National Reference Center for Primary Immunodeficiencies Paris France
Hospital Clínic Sant Creu i Sant Pau Hospital Bone Marrow Transplantation Unit Barcelona Spain
Klinik für Pädiatrische Hämatologie und Onkologie Hannover Medical School Hannover Germany
Laboratory of Genomic Dynamics in the Immune System Institut Imagine INSERM UMR1163 Paris France
Paediatric Hemato oncology and BMT Sheba Medical Center Tel Hashomer Israel
Paris Biotherapy Department Necker Children's Hospital Assistance Paris France
Pediatric Hematology Oncology Fondazione IRCCS Policlinico San Matteo Pavia Italy
Pediatric Oncohaematology and BMT Unit Children's Hospital Brescia Brescia Italy
Service d'Hématologie Immunologie Oncologie Pédiatrique CHU La Timone Marseille France
Université de Paris Paris France
University of Helsinki and Children's Hospital University of Helsinki Helsinki Finland
Citace poskytuje Crossref.org
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- $a Lankester, Arjan C $u 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
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- $a Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort / $c AC. Lankester, B. Neven, N. Mahlaoui, EGJ. von Asmuth, V. Courteille, M. Alligon, MH. Albert, IB. Serra, P. Bader, D. Balashov, R. Beier, Y. Bertrand, S. Blanche, V. Bordon, RG. Bredius, A. Cant, M. Cavazzana, C. Diaz-de-Heredia, F. Dogu, K. Ehlert, N. Entz-Werle, A. Fasth, F. Ferrua, A. Ferster, R. Formankova, W. Friedrich, M. Gonzalez-Vicent, J. Gozdzik, T. Güngör, M. Hoenig, A. Ikinciogullari, K. Kalwak, S. Kansoy, A. Kupesiz, A. Lanfranchi, CA. Lindemans, R. Meisel, G. Michel, NAA. Miranda, J. Moraleda, D. Moshous, H. Pichler, K. Rao, P. Sedlacek, M. Slatter, E. Soncini, C. Speckmann, M. Sundin, A. Toren, K. Vettenranta, A. Worth, MA. Yeşilipek, M. Zecca, F. Porta, A. Schulz, P. Veys, A. Fischer, AR. Gennery
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- $a 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 × 10e3/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.
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