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PubMed

Záznam pochází z PubMed

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

The Journal of allergy and clinical immunology. 2022 May ; 149 (5) : 1744-1754.e8. [epub] 20211027

J Allergy Clin Immunol
ISSN 1097-6825 | 0091-6749
Zdroj

Jazyk angličtina Země Spojené státy americké Médium print-electronic

Typ dokumentu časopisecké články, multicentrická studie, práce podpořená grantem

Perzistentní odkaz https://www.medvik.cz/link/pmid34718043

PubMed 34718043
DOI 10.1016/j.jaci.2021.10.017
PII: S0091-6749(21)01629-8
Knihovny.cz E-zdroje

Klíčová slova
SCID, conditioning, genetic subgroups, immune reconstitution, pretransplantation infections,
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.

BMT Unit Instituto Português de Oncologia de Lisboa Lisbon Portugal

Department for Children and Adolescents Medicine Division for Stem Cell Transplantation and Immunology University Hospital Frankfurt Frankfurt Germany

Department for Hematopoietic Stem Cell Transplantation Dmitriy Rogachev National Center for Pediatric Hematology Oncology and Immunology Moscow Russia

Department of Clinical Immunology and Transplantation Jagiellonian University Medical College Krakow Poland

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 Hematology Oncology Immunology Gene Therapy and Stem Cell Transplantation and Children's Research Center University Children's Hospital Zurich Switzerland

Department of Pediatric Hemato oncology and Stem Cell Transplant Ghent University Hospital Ghent Belgium

Department of Pediatric Hematology and Oncology Center for Pediatrics and Adolescent Medicine and Institute for Immunodeficiency Center for Chronic Immunodeficiency Faculty of Medicine Medical Center University of Freiburg Freiburg Germany

Department of Pediatric Hematology and Oncology Ege University Hospital Izmir Turkey

Department of Pediatric Hematology and Oncology Teaching Hospital Motol 2nd Medical School Charles University Motol Prague Czech Republic

Department of Pediatric Hematology and Oncology Universitätsklinikum Münster Münster Germany; Department of Pediatric Hematology and Oncology University of Greifswald Greifswald Germany