Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Lankester Arjan C, Neven Benedicte, Mahlaoui Nizar, von Asmuth Erik G J, 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, Güngör 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, Yeşilipek Mehmet A, Zecca Marco, Porta Fulvio, Schulz Ansgar, Veys Paul, Fischer Alain, Gennery Andrew R
Аннотация
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.