Hematopoietic cell transplant after 2010 no longer conveyed a survival advantage over earlier time intervals
By Elana Gotkine HealthDay Reporter
WEDNESDAY, June 28, 2023 (HealthDay News) — Newborn screening for severe combined immunodeficiency (SCID) facilitating early identification results in improved survival, according to a study published online June 20 in The Lancet.
Monica S. Thakar, M.D., from the Fred Hutchinson Cancer Center in Seattle, and colleagues analyzed transplantation-related data from children with SCID treated at 34 sites for 1982 to 1989, 1990 to 1999, 2000 to 2009, and 2010 to 2018 to examine factors affecting survival, focusing on SCID screening that was initiated in 2008 and expanded during 2010 to 2018. Data were included for 902 children with confirmed SCID.
The researchers found that the five-year overall survival remained stable for 28 years at 72 to 73 percent until 2010 to 2018, when it increased to 87 percent. Five-year overall survival was 92.5 percent for children identified as having SCID by newborn screening since 2010, which was better than that of children identified by clinical illness or family history during the same period (79.9 and 85.4 percent, respectively). During all time intervals, the factors of active infection, age 3.5 months or older at hematopoietic cell transplantation (HCT), Black or African-American race (hazard ratios, 2.41, 2.12, and 2.33, respectively), and certain SCID genotypes were associated with lower overall survival. HCT after 2010 no longer conveyed a survival advantage over earlier time intervals studied after adjustment for multiple variables.
“Our findings summarize the evolution of almost four decades of transplant practices and provide direct evidence that newborn screening for SCID has been the primary driver of improved survival in newborn babies in the USA and Canada since 2010,” the authors write.
Several authors disclosed ties to the biopharmaceutical industry.
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