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Haploidentical hematopoietic stem cell transplantation for a case with X-linked chronic granulomatous disease

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PEDIATRIC TRANSPLANTATION
卷 21, 期 1, 页码 -

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WILEY
DOI: 10.1111/petr.12861

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haploidentical; myeloablative conditioning; X-linked chronic granulomatous disease

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  1. Shanghai City Health Bureau [20124209]

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CGD is a rare primary immunodeficiency with high mortality rates when treated conventionally, especially for the X-chromosome-linked form. HSCT is the only curative therapy for CGD; however, haploidentical transplantation in CGD is rare. Here, we report a case of X-linked CGD treated successfully by haploidentical HSCT. The patient showed a positive result with full donor chimerism, good quality of life, and the absence of recurrent infectious diseases at follow-up (68months). Thus, haploidentical HSCT may serve as an acceptable treatment approach for patients who have CGD, but no HLA-matched related or unrelated donor.

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