Better Survival Chances for Children with Rare Immunodeficiency

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A recent study highlights critical need to improve on treatment strategies for babies suffering from a rare immunodeficiency known as severe combined immune deficiency (SCID). This deficiency also known as bubble baby disease is an uncommon body syndrome connected with the non-functional immune system. Children with this disease are prone to get easily affected by severe infection caused by viruses, fungi, and bacteria as they have essentially a non-functional immune system. If not treated properly within the first few months of child’s birth, this disease can become fatal. SCID can be attributed to various gene mutations responsible for functioning of the immune system.

Researchers found that the nature of this mutated gene has a key influence on the patient’s immune system with respect to the rebuilding and survival after the transplantation of bone marrow done to restore their immunity. As per the study, the nature of the mutated gene should be considered while implementing treatment strategies for each patient. One of the researchers also said that the study greatly benefitted from the access of rare treatment data on patients for over a long period, as the incidence of the disease has been quite low in Quebec. Furthermore, the result from their study also revealed that the survival rate was higher due to the cell transplantation matching a family member donor. In fact, cell transplantation from other donor types also had a substantial impact on immune rebuilding and survival.

The study has been published in the medical journal Blood on August 2018.

Patient-Specific Treatment Strategies need to be developed for Treating SCID

A lead researcher stated that it is important to start developing treatment strategies specific to each patient. This should start at neonatal screening where the children suffering with the disease are appropriately isolated, measures are implemented for preventing the infection, and ensure that the patients get bone marrow transplant with ease. The study remarkably noted that is also important to closely monitor such patients post treatment so as to find out if they need extra medication and avoid poor prognosis. In the future, such studies will help identify factors that hamper the early immune rebuilding, and eventually pave way for more effective treatment strategies for SCID.