Successful haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide in a child with X-linked chronic granulomatous disease: A first report in Asia.
Journal
Asian Pacific journal of allergy and immunology
ISSN: 0125-877X
Titre abrégé: Asian Pac J Allergy Immunol
Pays: Thailand
ID NLM: 8402034
Informations de publication
Date de publication:
Sep 2022
Sep 2022
Historique:
pubmed:
5
11
2019
medline:
17
8
2022
entrez:
4
11
2019
Statut:
ppublish
Résumé
HLA-matched hematopoietic stem cell transplantation (HSCT) is the curative treatment for chronic granulomatous disease (CGD). To report a case of X-linked CGD with active infection successfully treated by haploidentical HSCT with post-transplant high dose cyclophosphamide (PTCY). 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. 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. Haploidentical HSCT with PTCY could be an effective treatment option for children with CGD.
Sections du résumé
BACKGROUND
BACKGROUND
HLA-matched hematopoietic stem cell transplantation (HSCT) is the curative treatment for chronic granulomatous disease (CGD).
OBJECTIVE
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
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
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
CONCLUSIONS
Haploidentical HSCT with PTCY could be an effective treatment option for children with CGD.
Identifiants
pubmed: 31677618
doi: 10.12932/AP-290419-0551
doi:
Substances chimiques
Cyclophosphamide
8N3DW7272P
Types de publication
Case Reports
Journal Article
Langues
eng
Sous-ensembles de citation
IM