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Author Spotlight: Exploring the Lifespan Dynamics of Healthy Human Hematopoiesis
Published on: December 8, 2023
Haploidentical hematopoietic stem cell transplantation for a case with X-linked chronic granulomatous disease
Ling Zhou1,2, Lu-Jia Dong2, Zhi-Yong Gao2
1The Fifth People's Hospital of Shanghai, Fudan University, Shanghai, China.
Insights
Haploidentical HSCT offers a curative option for X-linked Chronic Granulomatous Disease (CGD) when matched donors are unavailable. This case demonstrates successful treatment with long-term positive outcomes, improving quality of life.
Area of Science:
- Immunology
- Hematology
- Genetics
Background:
- Chronic Granulomatous Disease (CGD) is a rare primary immunodeficiency with significant mortality.
- X-linked CGD is the most common form, often associated with severe outcomes.
- Hematopoietic Stem Cell Transplantation (HSCT) is the only known cure for CGD.
Abstract:
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 (68 months). 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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