Hematopoietic cell transplantation in chronic granulomatous ...

TRANSPLANTATION| SEPTEMBER 3, 2020

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 for the EBMT Inborn Errors Working Party

Blood (2020) 136 (10): 1201–1211.

https://doi.org/10.1182/blood.2020005590

Key Points

An excellent outcome was obtained after allo-HCT in 712 patients with CGD, with a low incidence of graft failure and mortality.

HCT for CGD should be strongly considered in young patients, particularly in the presence of a well-matched donor.

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.

Subjects:

Phagocytes, Granulocytes, and Myelopoiesis, Transplantation

Topics:

allopurinol, child, donors, granulomatous disease, chronic, hematopoietic stem cell transplantation, transplantation, tissue transplants, infections, graft-versus-host disease, chronic, antigens

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