Publication details

Transplantace hematopoetických buněk u pěti pacientů s chronickou granulomatózní nemocí v České republice

Title in English Transplantation of hematopoietic cells in five patients with chronic granulomatous disease in the Czech Republic
Authors

JANDA A. KESLOVÁ P. FORMÁNKOVÁ R. LITZMAN Jiří ŠEDIVÁ A. HOUŠŤKOVÁ H. ROZSÍVAL P. PAŘÍZKOVÁ E. STARÝ J. SEDLÁČEK P.

Year of publication 2012
Type Article in Periodical
Magazine / Source Česko-slovenská pediatrie
MU Faculty or unit

Faculty of Medicine

Citation
Field Paediatry
Keywords chronic granulomatous disease; primary immunodeficiency; hematopoietic stem cell transplantation
Attached files
Description Chronic granulomatous disease (CGD) is a primary immunodeficiency caused by defective phagocytosis. Conservative treatment is applied in most of the patients. Currently, the only available curative treatment is allogeneic hematopoietic stem cell transplantation (HSCT). Thirty-seven patients were diagnosed with CGD in the Czech and Slovak Republic between 1957 and 2011. Calculated incidence of the disease in the Czech Republic is 1:280,000 live births. Five patients with serious course of the disease underwent allogeneic HSCT in 2007–2010. Median age of CGD diagnosis in these patients was 6 months (6–17 months), median age at transplantation was 21 months (1–17 years). One patient was transplanted in myeloablative regimen, 4 patients in regimen with reduced intensity. All of the grafts were from HLA-matched unrelated donors. The procedure was complicated with graft versus host disease – GvHD (3 patients), infections (4 patients), autoimmunity (3 patients) and toxic side effects (3 patients). Median follow-up after the transplantation was 12 months (6–44 months). In all of the patients normalization of granulocyte function was documented within 2 months after the transplantation. Mixed chimerismwas present in all of them. The oldest patient (transplanted at the age of 17 years, 4months) died 6 months after transplantation due to intracranial haemorrhage after operational revision of healed brain abscess. All the other patients (4) are in a good clinical condition, without signs of CGD or GvHD. In concordance with other transplantation centres we show that early allogeneic HSCT is a suitable treatment of patients with severe CGD.

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