Children with this disorder are initially hospitalized and monitored until they are stable. No single treatment approach is effective and unfortunately, responses to various treatments are inconsistent. Because of the complexity of this condition, a multidisciplinary approach (having pediatric specialists in different fields working together) is required.
Initially, corticosteroids such as prednisolone are given at varying doses. If this approach is not effective, chemotherapy with vincristine (a drug widely used to treat cancers) is started. If this regimen is not effective, other drugs such as interferon, Cytoxan, Amicar and Rapamycin are used.
When the tumor does not respond to drug therapy, embolization (a highly specialized procedure in which particles are injected into the blood vessels to stop the blood flow), surgical removal, or both may be necessary. Blood products such as platelets and packed red blood cells are administered only if absolutely necessary.
The lesion may take many years to decrease in size. Studies suggest that some patients have residual lesions even 10 years after diagnosis. Improvement in platelet counts initially occurs with a decrease in the size of the lesion occurring later.