The survival rate jumped to 92.5 percent in children whose SCID was detected and treated at birth, whereas it was much lower in children diagnosed after the onset of symptoms. Furthermore, the proportion of babies transplanted and who never contracted an infection was significantly higher, an aspect that played a major role in increasing their survival.
Also known as “bubble-baby” disease, SCID causes major immune cell dysfunction and affects 40 to 80 children annually in North America. Children with SCID appear perfectly healthy at birth but are extremely susceptible to infection. The disease is fatal (usually within the first year of life) unless the child receives treatment, such as a bone marrow transplant,
or enzyme therapy.
“The improvement in post-transplant survival is staggering and alone justifies the implementation of a newborn screening test for SCID and other life-threatening diseases of the immune system,” said Haddad, who is also the recipient of the BMO Chair in Pediatric Immunology and the head of the Immuno-allergy and Rheumatology division at CHU Sainte-Justine. “This study highlights the importance of prevention and of collaboration in precision health.”
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Done in collaboration with numerous research centres in Canada and the U.S., Haddad’s shows that the increase in the survival rate of children with transplants coincides with the rollout of a screening test in various North American regions begun in 2008. Since 2018, all U.S. states have been conducting preventive screening for SCID. In Canada, seven provinces and territories have followed suit; in Quebec, the process is underway.
The continuing adoption of newborn screening has made it possible to detect the disease earlier before symptoms appear, and to take all possible steps to prevent infection and quickly provide treatment. Previous research already showed that being younger than 3.5 months at the time of transplant and the absence of infection significantly improved survival rates.
“Given that we are preventing children from dying and from undergoing much more difficult treatments with subsequent consequences,” said Haddad, “these results will encourage other countries around the world to adopt newborn screening for SCID and other life-threatening immune diseases that the test can detect.”
Reference :
- Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency – (https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(23)00731-6/fulltext)
Source: Eurekalert
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