Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study.
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.
Ferrua Francesca, Galimberti Stefania, Courteille Virginie, Slatter Mary Anne, Booth Claire, Moshous Despina, Neven Benedicte, Blanche Stephane, Cavazzana Marina, Laberko Alexandra, Shcherbina Anna, Balashov Dmitry, Soncini Elena, Porta Fulvio, Al-Mousa Hamoud, Al-Saud Bandar, Al-Dhekri Hasan, Arnaout Rand, Formankova Renata, Bertrand Yves, Lange Andrzej, Smart Joanne, Wolska-Kusnierz Beata, Aquino Victor M, Dvorak Christopher C, Fasth Anders, Fouyssac Fanny, Heilmann Carsten, Hoenig Manfred, Schuetz Catharina, Kelečić Jadranka, Bredius Robbert G M, Lankester Arjan C, Lindemans Caroline A, Suarez Felipe, Sullivan Kathleen E, Albert Michael H, Kałwak Krzysztof, Barlogis Vincent, Bhatia Monica, Bordon Victoria, Czogala Wojciech, Alonso Laura, Dogu Figen, Gozdzik Jolanta, Ikinciogullari Aydan, Kriván Gergely, Ljungman Per, Meyts Isabelle, Mustillo Peter, Smith Angela R, Speckmann Carsten, Sundin Mikael, Keogh Steven John, Shaw Peter John, Boelens Jaap Jan, Schulz Ansgar S, Sedlacek Petr, Veys Paul, Mahlaoui Nizar, Janda Ales, Davies E Graham, Fischer Alain, Cowan Morton J, Gennery Andrew Richard, SCETIDE, PIDTC, EBMT & ESID IEWP
Аннотация
OBJECTIVE: We performed an international collaborative study to improve patients' management, aiming to individualize risk factors and determine optimal HSCT characteristics.
METHODS: We retrospectively collected data on 130 patients who underwent HSCT for CD40L deficiency between 1993-2015. We analyzed outcome and variables' relevance with respect to survival and cure.
RESULTS: Overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 78.2%, 58.1%, and 72.3% 5 years after HSCT. Results were better in transplantations performed in 2000 or later and in children less than 10 years old at the time of HSCT. Pre-existing organ damage negatively influenced outcome. Sclerosing cholangitis was the most important risk factor. After 2000, superior OS was achieved with matched donors. Use of myeloablative regimens and HSCT at 2 years or less from diagnosis associated with higher OS and DFS. EFS was best with matched sibling donors, myeloablative conditioning (MAC), and bone marrow-derived stem cells. Most rejections occurred after reduced-intensity or nonmyeloablative conditioning, which associated with poor donor cell engraftment. Mortality occurred mainly early after HSCT, predominantly from infections. Among survivors who ceased immunoglobulin replacement, T-lymphocyte chimerism was 50% or greater donor in 85.2%.
CONCLUSION: HSCT is curative in patients with CD40L deficiency, with improved outcome if performed before organ damage development. MAC is associated with better OS, EFS, and DFS. Prospective studies are required to compare the risks of HSCT with those of lifelong supportive therapy.