Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort
Makaleler > Tam Makale · 2021
Yazarlar: Arjan C. Lankester, Benedicte Neven, Nizar Mahlaoui, Erik G.J. von Asmuth, Virginie Courteille, Mikael Alligon, Michael H. Albert, Isabelle Badell Serra, Peter Bader, Dmitry Balashov, Rita Beier, Yves Bertrand, Stephane Blanche, Victoria Bordon, Robbert G. Bredius, Andrew Cant, Marina Cavazzana, Cristina Diaz-de-Heredia, Figen Dogu, Karoline Ehlert, Natacha Entz-Werle, Anders Fasth, Francesca Ferrua, Alina Ferster, Renata Formankova, Wilhelm Friedrich, Marta Gonzalez-Vicent, Jolanta Gozdzik, Tayfun Güngör, Manfred Hoenig, Aydan Ikinciogullari, Krzysztof Kalwak, Savas Kansoy, Alphan Kupesiz, Arnalda Lanfranchi, Caroline A. Lindemans, Roland Meisel, Gerard Michel, Nuno A.A. Miranda, Jose Moraleda, Despina Moshous, Herbert Pichler, Kanchan Rao, Petr Sedlacek, Mary Slatter, Elena Soncini, Carsten Speckmann, Mikael Sundin, Amos Toren, Kim Vettenranta, Austen Worth, Mehmet A. Yeşilipek, Marco Zecca, Fulvio Porta, Ansgar Schulz, Paul Veys, Alain Fischer, Andrew R. Gennery
Journal of Allergy and Clinical Immunology, Uluslararası, Wos, 2021, cilt 149, ss 1744-1754.e8
DOI: 10.1016/j.jaci.2021.10.017
Özet
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: /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.