Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults

Chiesa, R.; Wang, J.F.; Blok, H.J.; Hazelaar, S.; Neven, B.; Moshous, D.; Schulz, A.; Hoenig, M.; Hauck, F.; Seraihy, A. al; Gozdzik, J.; Ljungman, P.; Lindemans, C.A.; Fernandes, J.F.; Kalwak, K.; Strahm, B.; Schanz, U.; Sedlacek, P.; Sykora, K.W.; Aksoylar, S.; Locatelli, F.; Stepensky, P.; Wynn, R.; Lurn, S.H.; Zecca, M.; Porta, F.; Taskinen, M.; Gibson, B.; Matthes, S.; Karakukcu, M.; Hauri-Hohl, M.; Veys, P.; Gennery, A.R.; Lucchini, G.; Felber, M.; Albert, M.H.; Balashov, D.; Lankester, A.; Gungor, T.; Slatter, M.A.; EBMT Inborn Errors Working Party

doi:doi:10.1182/blood.2020005590

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...Show moreChronic 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.Show less

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Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults