Transplantation of hematopoietic cells in five patients with chronic granulomatous disease in the Czech Republic
Abstract
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.