Treatment of Chediak-Higashi syndrome

Treatment of the syndrome of Chediak-Higashi before the development of the accelerating phase is reduced to adequate control of emerging infections. In the case of deep abscesses, in addition to antibiotic therapy, surgical treatment is indicated. The therapy in the accelerated phase is carried out according to the protocol intended for PGLH, with the mandatory subsequent allogeneic TSCC from the HLA-identical or haploidentical donor. In 2006, Tardieu et al. Published the results of more than 20-year experience of transplantation of patients with Chediak-Higashi syndrome, conducted in the hospital Necker-Enfants Matades in Paris. Five-year survival was observed in 10 out of 14 patients, but 2 out of 10 surviving patients had neurological disorders in the early post-transplant period, and in 3 more patients they debuted 20 years or more after transplantation. Interestingly, in one patient transplanted from an incompatible donor with 1 HLA antigen, this symptomatology appeared only at the age of 21, and during this period only 4% of donor cells were detected during the entire posttransplant period. In the absence of gene therapy, TSCC remains the only radical treatment for patients suffering from CHS.

Forecast

The prognosis, in the absence of TSCC, is unfavorable, since children with Chediak-Higashi syndrome usually do not live to the age of ten. The causes of death, as a rule, are severe infections, hemorrhagic syndrome. Several patients at the age of 20 years who have not undergone TSCS have been described, however, in such patients, lymphoproliferation increases and the risk of malignant lymphoma increases significantly over the years.