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

Lankester, Arjan C.; Neven, Benedicte; Mahlaoui, Nizar; Asmuth, Erik G. J. von; Courteille, Virginie; Alligon, Mikael; Albert, Michael H.; Serra, Isabelle Badell; Bader, Peter; Balashov, Dmitry; Beier, Rita; Bertrand, Yves; Blanche, Stephane; Bordon, Victoria; Bredius, Robbert G.; Cant, Andrew; Cavazzana, Marina; Diaz-de-Heredia, Cristina; Dogu, Figen; Ehlert, Karoline; Entz-Werle, Natacha; Fasth, Anders; Ferrua, Francesca; Ferster, Alina; Formankova, Renata; Friedrich, Wilhelm; Gonzalez-Vicent, Marta; Gozdzik, Jolanta; Gungor, Tayfun; Hoenig, Manfred; Ikinciogullari, Aydan; Kalwak, Krzysztof; Kansoy, Savas; Kupesiz, Alphan; Lanfranchi, Arnalda; Lindemans, Caroline A.; Meisel, Roland; Michel, Gerard; Miranda, Nuno A. A.; Moraleda, Jose; Moshous, Despina; Pichler, Herbert; Rao, Kanchan; Sedlacek, Petr; Slatter, Mary; Soncini, Elena; Speckmann, Carsten; Sundin, Mikael; Toren, Amos; Vettenranta, Kim; Worth, Austen; Yesilipek, Mehmet A.; Zecca, Marco; Porta, Fulvio; Schulz, Ansgar; Veys, Paul; Fischer, Alain und Gennery, Andrew R. (2022): Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort. In: Journal of Allergy and Clinical Immunology, Bd. 149, Nr. 5: S. 1744-1754

Abstract

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 longterm 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 3 10(-3)/mL at 11 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.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 0091-6749
• Sprache: Englisch