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.

Chiesa, R., Wang, J., Blok, H. -., Hazelaar, S., Neven, B., Moshous, D., Schulz, A., Hoenig, M., Hauck, F., Seraihy, A. A., Gozdzik, J., Ljungman, P., Lindemans, C. A., Fernandes, J. F., Kalwak, K., Strahm, B., Schanz, U., Sedlacek, P., Sykora, K. -., Aksoylar, S., Locatelli, F., Stepensky, P., Wynn, R., Lum, 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., Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults, <<BLOOD>>, 2020; 136 (10): 1201-1211. [doi:10.1182/blood.2020005590] [https://hdl.handle.net/10807/228101]

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

Locatelli, Franco
Writing – Original Draft Preparation
;
2020

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
Inglese
Chiesa, R., Wang, J., Blok, H. -., Hazelaar, S., Neven, B., Moshous, D., Schulz, A., Hoenig, M., Hauck, F., Seraihy, A. A., Gozdzik, J., Ljungman, P., Lindemans, C. A., Fernandes, J. F., Kalwak, K., Strahm, B., Schanz, U., Sedlacek, P., Sykora, K. -., Aksoylar, S., Locatelli, F., Stepensky, P., Wynn, R., Lum, 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., Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults, <<BLOOD>>, 2020; 136 (10): 1201-1211. [doi:10.1182/blood.2020005590] [https://hdl.handle.net/10807/228101]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/228101
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