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  • 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.
    Author: Janjindamai C, Manuyakorn W, Anurathapan U, Pakakasama S, Wattanasirichaigoon D, Kamchaisatian W, Benjaponpitak S, Hongeng S.
    Journal: Asian Pac J Allergy Immunol; 2022 Sep; 40(3):278-282. PubMed ID: 31677618.
    Abstract:
    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.
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