Abstract
The results of hematopoietic stem cell transplant (HSCT) for primary immunodeficiency diseases (PID) have been improving over time. Unfortunately, develo** countries do not experience the same results. This first report of Brazilian experience of HSCT for PID describes the development and results in the field. We included data from transplants in 221 patients, performed at 11 centers which participated in the Brazilian collaborative group, from July 1990 to December 2015. The majority of transplants were concentrated in one center (n = 123). The median age at HSCT was 22 months, and the most common diseases were severe combined immunodeficiency (SCID) (n = 67) and Wiskott-Aldrich syndrome (WAS) (n = 67). Only 15 patients received unconditioned transplants. Cumulative incidence of GVHD grades II to IV was 23%, and GVHD grades III to IV was 10%. The 5-year overall survival was 71.6%. WAS patients had better survival compared to other diseases. Most deaths (n = 53) occurred in the first year after transplantation mainly due to infection (55%) and GVHD (13%). Although transplant for PID patients in Brazil has evolved since its beginning, we still face some challenges like delayed diagnosis and referral, severe infections before transplant, a limited number of transplant centers with expertise, and resources for more advanced techniques. Measures like newborn screening for SCID may hasten the diagnosis and ameliorate patients’ conditions at the moment of transplant.
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The authors thank all the nurses, physicians, and multidisciplinary staff of the participating centers.
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JFF designed the study, enrolled and cared for patients, reviewed and analyzed data, and wrote the paper. CB and SN designed the study, enrolled and cared for patients, and reviewed the paper. All the other authors contributed to data collection, patient care, and reviewed the paper.
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This study was approved by the Institutional Review Boards of each center and performed in accordance with the 1964 Declaration of Helsinki.
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Fernandes, J.F., Nichele, S., Daudt, L.E. et al. Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Develo** Countries. J Clin Immunol 38, 917–926 (2018). https://doi.org/10.1007/s10875-018-0564-1
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DOI: https://doi.org/10.1007/s10875-018-0564-1