Excellent Outcome Following Sibling Peripheral Blood Hematopoietic Stem Cell Transplantation for Glanzmann Thrombasthenia: A Case Report

Jian hua Li; Jian hua Li; Shu wen Sun; Shu wen Sun; Yuan Ai; Yuan Ai; Xue Yang; Xue Yang; Yi ping Zhu; Yi ping Zhu

doi:10.3389/fped.2021.776927

Frontiers in Pediatrics (Feb 2022)

Excellent Outcome Following Sibling Peripheral Blood Hematopoietic Stem Cell Transplantation for Glanzmann Thrombasthenia: A Case Report

Jian hua Li,
Jian hua Li,
Shu wen Sun,
Shu wen Sun,
Yuan Ai,
Yuan Ai,
Xue Yang,
Xue Yang,
Yi ping Zhu,
Yi ping Zhu

Affiliations

Jian hua Li: Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China
Jian hua Li: Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu, China
Shu wen Sun: Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China
Shu wen Sun: Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu, China
Yuan Ai: Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China
Yuan Ai: Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu, China
Xue Yang: Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China
Xue Yang: Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu, China
Yi ping Zhu: Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China
Yi ping Zhu: Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu, China

DOI: https://doi.org/10.3389/fped.2021.776927
Journal volume & issue: Vol. 9

Abstract

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Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet disorder due to a qualitative or quantitative anomaly of the platelet membrane glycoprotein GPIIb/IIIa. Its clinical manifestations include mild to severe bleeding. GT diagnosis mainly depends on platelet function analysis, flow cytometry, and gene detection. Treatment methods include conservative symptomatic treatment and allogeneic hematopoietic stem cell transplantation (allo-HSCT). Allo-HSCT is the only clinical radical method for GT. Herein, we report a 2-year-old boy with GT successfully cured by related identical peripheral blood stem cell transplantation (PBSCT). The platelet disorder was corrected to a normal level after PBSCT, with no significant complication related to the transplantation. Hematopoietic stem cell transplantation with full-matched donor in early stage could be a treatment option for GT.

Published in Frontiers in Pediatrics

ISSN: 2296-2360 (Online)
Publisher: Frontiers Media S.A.
Country of publisher: Switzerland
LCC subjects: Medicine: Pediatrics
Website: http://journal.frontiersin.org/journal/pediatrics

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