Newborn Screening for Severe Combined Immunodeficiency (SCID); Early Diagnosis and Early Start of Treatment Can Prevent Severe Infections

According to a January 30, 2017 press release, the German Institute for Quality and Efficiency in Health Care (IQWiG) has assessed the benefit of newborn screening for severe combined immunodeficiency (SCID). Without treatment most young children with SCID die within 1 to 2 years, as their immune response fails. The benefit assessment provides a hint of a benefit of newborn screening for SCID: An early test combined with infection prophylaxis and subsequent curative treatment (allogeneic bone marrow or stem cell transplantation) can prevent severe or deadly infections in affected children. The failure of immune response in SCID is caused by a genetic disorder leading to inhibited development of vital immune cells (T lymphocytes, B lymphocytes, natural killer cells). Children with SCID are already highly susceptible to infections as babies and also show impairment of growth. Without treatment most young children with SCID die within 1 to 2 years. It is not exactly known how many children are born with SCID in Germany. For the year 2013, statistics of the statutory health insurance report 21 cases in children younger than a year old. SCID is currently treated with allogeneic bone marrow or stem cell transplantation: In this procedure, the inadequate stem cells of the child are replaced by those of a suitable donor in order to develop the child's immune function. The question of the optimum time of transplantation and the necessity of chemotherapy in newborns with SCID is currently the subject of controversy. Even before initiation of curative treatment the newborns must be stabilized by preventive and supportive measures, such as strict hygienic precautionary measures, infection prophylaxis, and substitution of antibodies.
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