Successful haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide in a child with X-linked chronic granulomatous disease: A first report in Asia (2019)

Title           :  Successful haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide in a child with X-linked chronic granulomatous disease: A first report in Asia

Researcher       : Clin.Prof.Suwat Benjaponpitak 

Department     : Faculty of Medicine, Siam University, Bangkok, Thailand

E-mail                : med@siam.edu

Abstract            :  Backgound: HLA-matched hematopoietic stem cell transplantation (HSCT) is the curative treatment for chronic granulomatous disease (CGD).

Objective: To report a case of X-linked CGD with active infection successfully treated by haploidentical HSCT with post-transplant high dose cyclophosphamide (PTCY).

Methods: A 5-year-old Thai boy with CGD was undergone for haploidentical HSCT using PTCY with correction of the phagocytic function. He presented with Chromobacterium violaceum liver abscess at the age of 9 months and experienced recurrent perianal abscess and invasive pulmonary aspergillosis even receiving antimicrobial prophylaxis. PTCY was given on day 3 and 4, after CD34+ cells infusion.

Results: The peripheral blood-nucleated cell chimerism showed 100% on day 16 and remained 100%. Dihydrorhodamine (DHR) assay on day 108 and day 214 showed normal results. Currently at 22 months post HSCT, he does not receive antibiotic and anti-fungal prophylaxis.

Conclusions: Haploidentical HSCT with PTCY could be an effective treatment option for children with CGD.

Key words: Haploidentical transplantation, Hematopoietic stem cell transplantation. Chronic granulomatous disease, Cyclophosphamide, Graft-Versus-Host Disease

Link to Academic article: DOI: 10.12932/AP-290419-0551

Journal : Asian Pacific journal of allergy and immunology 

Bibliography     : Janjindamai, C., Manuyakorn, W., Anurathapan, U., Pakakasama, S., Wattanasirichaigoon, D., Kamchaisatian, W., Benjaponpitak, S., & Hongeng, S. (2019, November 3). Successful haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide in a child with X-linked chronic granulomatous disease: A first report in Asia. Asian Pac J Allergy Immunol, doi: 10.12932/AP-290419-0551. Epub ahead of print. PMID: 31677618.