Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency.
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.
Aydin Susanne E, Freeman Alexandra F, Al-Herz Waleed, Al-Mousa Hamoud A, Arnaout Rand K, Aydin Roland C, Barlogis Vincent, Belohradsky Bernd H, Bonfim Carmem, Bredius Robbert G, Chu Julia I, Ciocarlie Oana C, Doğu Figen, Gaspar Hubert B, Geha Raif S, Gennery Andrew R, Hauck Fabian, Hawwari Abbas, Hickstein Dennis D, Hoenig Manfred, Ikinciogullari Aydan, Klein Christoph, Kumar Ashish, Ifversen Marianne R S, Matthes Susanne, Metin Ayse, Neven Benedicte, Pai Sung-Yun, Parikh Suhag H, Picard Capucine, Renner Ellen D, Sanal Özden, Schulz Ansgar S, Schuster Friedhelm, Shah Nirali N, Shereck Evan B, Slatter Mary A, Su Helen C, van Montfrans Joris, Woessmann Wilhelm, Ziegler John B, Albert Michael H, Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and the European Society for Primary Immunodeficiencies
Аннотация
OBJECTIVE: To determine outcome of HSCT for DOCK8 deficiency and define possible outcome variables.
METHODS: We performed a retrospective study of the results of HSCT in a large international cohort of DOCK8-deficient patients.
RESULTS: We identified 81 patients from 22 centers transplanted at a median age of 9.7 years (range, 0.7-27.2 years) between 1995 and 2015. After median follow-up of 26 months (range, 3-135 months), 68 (84%) patients are alive. Severe acute (III-IV) or chronic graft versus host disease occurred in 11% and 10%, respectively. Causes of death were infections (n = 5), graft versus host disease (5), multiorgan failure (2), and preexistent lymphoma (1). Survival after matched related (n = 40) or unrelated (35) HSCT was 89% and 81%, respectively. Reduced-toxicity conditioning based on either treosulfan or reduced-dose busulfan resulted in superior survival compared with fully myeloablative busulfan-based regimens (97% vs 78%; P = .049). Ninety-six percent of patients younger than 8 years at HSCT survived, compared with 78% of those 8 years and older (P = .06). Of the 73 patients with chimerism data available, 65 (89%) had more than 90% donor T-cell chimerism at last follow-up. Not all disease manifestations responded equally well to HSCT: eczema, infections, and mollusca resolved quicker than food allergies or failure to thrive.
CONCLUSIONS: HSCT is curative in most DOCK8-deficient patients, confirming this approach as the treatment of choice. HSCT using a reduced-toxicity regimen may offer the best chance for survival.