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Treatment of Wiskott-Aldrich syndrome
Last reviewed: 04.07.2025
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The first choice of treatment for Wiskott-Aldrich syndrome is hematopoietic stem cell transplantation (HSCT). Survival rates for patients with Wiskott-Aldrich syndrome after HSCT from HLA-identical siblings are as high as 80%. Transplantation from HLA-identical unrelated donors is most effective in children under 5 years of age. In contrast to HSCT from an HLA-identical donor, the results of HSCT from partially matched (haploidentical) related donors have not been as impressive, although many Angoras report 50-60% survival, which is quite acceptable given the poor prognosis of the disease without HSCT.
Splenectomy reduces the risk of bleeding but is associated with an increased risk of septicemia. Splenectomy results in an increase in the number of circulating platelets and their size.
If Wiskott-Aldrich syndrome is diagnosed prenatally, then, due to the risk of intracranial bleeding, delivery is recommended by cesarean section.
Platelet transfusions should be avoided unless the patient is in serious danger of death and bleeding can be controlled conservatively. CNS hemorrhages require immediate platelet transfusion. Platelets and other blood products should be irradiated before transfusion to prevent graft-versus-host disease.
Because patients with Wiskott-Aldrich syndrome have impaired antibody production in response to many types of antigens, prophylactic treatment with intravenous immunoglobulin (IVIG) is indicated for patients with frequent infections. Because serum immunoglobulins are rapidly catabolized, the optimal prophylactic dose of IVIG may exceed the usual 400 mg/ng/month, and infusions may be given more frequently, such as once every 2-3 weeks.
Eczema, especially severe eczema, may require complex treatment with antibiotics. Steroid ointments and creams are usually effective in treating eczema, but sometimes short courses of systemic steroids may be required. Often, antibiotics improve the symptoms of eczema, indicating that a bacterial factor is involved in its development. It is necessary to take into account the presence of food allergies and adjust the diet accordingly.
In case of development of an autoimmune component, high doses of IVIG and systemic steroids can give a positive result, then the dose of steroids can be reduced.
Forecast
The average life expectancy of patients with Wiskott-Aldrich syndrome, without HSCT, was previously 3.5 years, currently it is 11 years, but most patients live more than 20 years. Unfortunately, in the third decade of life, the likelihood of malignant neoplasms, especially lymphomas, increases significantly. After successful HSCT, patients fully recover, they do not have infections, bleeding and autoimmune diseases, and, at present, there is no increase in the incidence of malignant diseases.