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Treatment of Wiskott-Aldrich Syndrome
Last reviewed: 19.10.2021
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The first choice in the treatment of Wiskott-Aldrich syndrome is the transplantation of hematopoietic stem cells (TSCC). Survival of patients with Wiskott-Aldrich syndrome after TRNC from HLA-identical siblings reaches 80%. Transplantation from HLA-identical unrelated donors is most effective in children under 5 years of age. Unlike TSCC from an HLA-identical donor, the results of TSCS from partially compatible (haploidentical) related donors were not so impressive, although many angoras describe a 50-60% survival rate, which is quite acceptable, given the poor prognosis of the disease outside the TSCC.
Splenectomy reduces the likelihood of bleeding, but is accompanied by an increased risk of septicemia. Splenectomy leads to an increase in the number of circulating platelets and an increase in their size.
If the diagnosis of Wiskott-Aldrich syndrome is prenatal, then because of the danger of intracranial hemorrhage, labor is recommended to be performed by cesarean section.
Transfusions of platelets should be avoided if the patient does not have a serious threat to life and bleeding can be stopped in conservative ways. Hemorrhages in the central nervous system require immediate transfusion of platelets. Platelets and other blood products must be irradiated before transfusion to prevent transplantation against the host in the reaction.
Since patients with Wiskott-Aldrich syndrome have a disruption in the production of antibodies in response to many varieties of antigens, prophylactic treatment with intravenous immunoglobulin transfusion (IVIG) is indicated for patients with frequent infections. Since immunoglobulins in the serum are rapidly catabolized, the optimal prophylactic dose of IVIG can exceed the usual 400 mg / ng / month and infusions can be performed more often, for example once every 2-3 weeks.
Eczema, especially severe, may require complex treatment with the use of antibiotics. Steroid ointments and creams are usually effective in treating eczema, however, it may sometimes be necessary to take short courses of systemic steroids. Often the use of antibiotics leads to an improvement in the symptoms of eczema, which indicates the influence of the bacterial factor on its development. It is necessary to take into account the presence of food allergies and adjust the diet accordingly.
6 In the case of autoimmune component development, high doses of IVIG and systemic steroids can give a positive result, then a dose of steroids can be reduced.
Forecast
The average life expectancy of patients with Wiskott-Aldrich syndrome, without TSCC, was 3.5 years earlier, at the present time it is 11 years, but the majority of patients live more than 20 years. Unfortunately, at the third decade of life, the probability of malignant neoplasms, especially lymphomas, increases significantly. After successful TSCS, the patients completely recover, they do not have infections, bleeding and autoimmune diseases, and, at the moment, there is no increase in the incidence of malignant diseases.