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

被引:0
作者
Janjindamai, Chawisa [1 ]
Manuyakorn, Wiparat [1 ,2 ]
Anurathapan, Usanarat [1 ]
Pakakasama, Samart [1 ]
Wattanasirichaigoon, Duangrurdee [1 ]
Kamchaisatian, Wasu [1 ]
Benjaponpitak, Suwat [1 ]
Hongeng, Suradej [1 ]
机构
[1] Mahidol Univ, Fac Med, Dept Pediat, Ramathibodi Hosp, Bangkok, Thailand
[2] Mahidol Univ, Fac Med, Dept Pediat, Div Allergy & Immunol,Ramathibodi Hosp, Rama 6 Rd, Bangkok 10400, Thailand
关键词
Haploidentical transplantation; Hematopoietic stem cell transplantation; Chronic granulomatous disease; Cyclophosphamide; Graft-Versus-Host Disease;
D O I
10.12932/ap-290419-0551
中图分类号
R392 [医学免疫学];
学科分类号
100102 ;
摘要
Background: 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.
引用
收藏
页码:278 / 282
页数:5
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