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

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.

• Authors: Chawisa Janjindamai, Wiparat Manuyakorn, Usanarat Anurathapan, Samart Pakakasama, Duangrurdee Wattanasirichaigoon, Wasu Kamchaisatian, Suwat Benjaponpitak, Suradej Hongeng
• Journal: Asian Pacific journal of allergy and immunology (Asian Pac J Allergy Immunol) Vol. 40 Issue 3 Pg. 278-282 (Sep 2022) ISSN: 0125-877X [Print] Thailand
• PMID: 31677618 (Publication Type: Case Reports, Journal Article)

Chemical References

• Cyclophosphamide

Topics

• Child
• Child, Preschool
• Cyclophosphamide (therapeutic use)
• Graft vs Host Disease (drug therapy, prevention & control)
• Granulomatous Disease, Chronic (complications, diagnosis, therapy)
• Hematopoietic Stem Cell Transplantation (methods)
• Humans
• Infant
• Male
• Transplantation Conditioning (methods)