Treatment
Treatment for X-linked SCID can be divided into prophylactic treatment (i.e. before curative treatment can be applied) and curative treatment. The former attempts to manage the opportunistic infections common to SCID patients and the latter aims at reconstituting healthy T-lymphocyte function.
Prophylactic treatment for X-linked SCID is similar to that of traditional primary immunodeficiencies. Drugs are administered to control opportunistic infections, such as fluconazole for candidiasis, and acyclovir to prevent herpes virus infection. The patient can also undergo immunoglobulin supplementation.
Bone marrow transplantation (BMT) is standard procedure for immune reconstitution. Though the BMT process is ‘common’, it requires immunodeficiency specialists for maximum potential. Infants, in which there is no donor, can undergo BMT, only if the marrow is first depleted of its mature T-cells. The depletion of T-cells in the donor tissue aims to prevent graft-versus-host disease. The T-cells must be depleted in order to remove mismatched T-cells which would cause a reaction in the infant.
Gene therapy is also available to replace the mutant allele. Though, this process can be achieved through various processes, it has been successful in treating X-SCID by insertion of functional, healthy genes with a retrovirus. This, coupled with the bone marrow stem cells, has been successful in treating individuals with X-SCID. In one particular trial, 10 children were treated at infancy for X-SCID. Nine of the 10 were cured of X-SCID. However, about three years after treatment, two of the children developed T-cell leukemia due to insertion of the IL2RG-containing virus near LMO2 (a known oncogene). A third child of the 10 developed leukemia within two years of that study being published, probably as a direct result of the therapy. There is currently no approved gene therapy on the market, but there are many clinical trials into which X-SCID patients may enroll.
Read more about this topic: X-linked Severe Combined Immunodeficiency
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