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
Famous quotes containing the word treatment:
“Ambivalence reaches the level of schizophrenia in our treatment of violence among the young. Parents do not encourage violence, but neither do they take up arms against the industries which encourage it. Parents hide their eyes from the books and comics, slasher films, videos and lyrics which form the texture of an adolescent culture. While all successful societies have inhibited instinct, ours encourages it. Or at least we profess ourselves powerless to interfere with it.”
—C. John Sommerville (20th century)
“I feel that any form of so called psychotherapy is strongly contraindicated for addicts.... The question “Why did you start using narcotics in the first place?” should never be asked. It is quite as irrelevant to treatment as it would be to ask a malarial patient why he went to a malarial area.”
—William Burroughs (b. 1914)
“Judge Ginsburg’s selection should be a model—chosen on merit and not ideology, despite some naysaying, with little advance publicity. Her treatment could begin to overturn a terrible precedent: that is, that the most terrifying sentence among the accomplished in America has become, “Honey—the White House is on the phone.””
—Anna Quindlen (b. 1952)