Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults
Robert Chiesa, Junfeng Wang, Henric-Jan Blok, Sheree Hazelaar, Benedicte Neven, Despina Moshous, Ansgar Schulz, Manfred Hoenig, Fabian Hauck, Amal Al Seraihy, Jolanta Gozdzik, Per Ljungman, Caroline A Lindemans, Juliana F Fernandes, Krzysztof Kalwak, Brigitte Strahm, Urs Schanz, Petr Sedlacek, Karl-Walter Sykora, Serap Aksoylar, Franco Locatelli, Polina Stepensky, Robert Wynn, Su Han Lum, Marco Zecca, Fulvio Porta, Mervi Taskinen, Brenda Gibson, Susanne Matthes, Musa Karakukcu, Mathias Hauri-Hohl, Paul Veys, Andrew R Gennery, Giovanna Lucchini, Matthias Felber, Michael H Albert, Dmitry Balashov, Arjan Lankester, Tayfun Güngör, Mary A Slatter, Robert Chiesa, Junfeng Wang, Henric-Jan Blok, Sheree Hazelaar, Benedicte Neven, Despina Moshous, Ansgar Schulz, Manfred Hoenig, Fabian Hauck, Amal Al Seraihy, Jolanta Gozdzik, Per Ljungman, Caroline A Lindemans, Juliana F Fernandes, Krzysztof Kalwak, Brigitte Strahm, Urs Schanz, Petr Sedlacek, Karl-Walter Sykora, Serap Aksoylar, Franco Locatelli, Polina Stepensky, Robert Wynn, Su Han Lum, Marco Zecca, Fulvio Porta, Mervi Taskinen, Brenda Gibson, Susanne Matthes, Musa Karakukcu, Mathias Hauri-Hohl, Paul Veys, Andrew R Gennery, Giovanna Lucchini, Matthias Felber, Michael H Albert, Dmitry Balashov, Arjan Lankester, Tayfun Güngör, Mary A Slatter
Abstract
Chronic granulomatous disease (CGD) is a primary immunodeficiency resulting in life-threatening infections and inflammatory complications. Allogeneic hematopoietic cell transplantation (allo-HCT) can cure the disease, but the indication to transplant remains controversial. We performed a retrospective multicenter study of 712 patients with CGD who underwent allo-HCT transplantation from March 1993 through December 2018. We studied 635 children (aged <18 years) and 77 adults. Median follow-up was 45 months. Median age at transplantation was 7 years (range, 0.1-48.6). Kaplan-Meier estimates of overall survival (OS) and event-free survival (EFS) at 3 years were 85.7% and 75.8%, respectively. In multivariate analysis, older age was associated with reduced survival and increased chronic graft-versus-host disease. Nevertheless, OS and EFS at 3 years for patients ≥18 years were 76% and 69%, respectively. Use of 1-antigen-mismatched donors was associated with reduced OS and EFS . No significant difference was found in OS, but a significantly reduced EFS was noted in the small group of patients who received a transplant from a donor with a >1 antigen mismatch. Choice of conditioning regimen did not influence OS or EFS. In summary, we report an excellent outcome after allo-HCT in CGD, with low incidence of graft failure and mortality in all ages. Older patients and recipients of 1-antigen-mismatched grafts had a less favorable outcome. Transplantation should be strongly considered at a younger age and particularly in the presence of a well-matched donor.
© 2020 by The American Society of Hematology.
Source: PubMed