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IN INFANTS, LAL-D IS A MEDICAL EMERGENCY THAT MAY CAUSE DEATH1
Nearly 90% mortality within 12 months after birth in infants with LAL-D1
a A retrospective chart review and data extraction of 35 patients diagnosed with LAL-D before age 2 (26 with growth failure before 6 months of life, 9 without).1
b Although the protocol allowed for enrollment of living patients, all patients in the overall population were deceased.1
Mortality in Infants with LAL-D
Infants with LAL-D experienced early and severe symptom onset at a median age of approximately 1 month (for patients with and without early growth failure).1
Among the overall population studied, the median age of death was similar to that of patients with early growth failure (3.7 months [range, 1.4-46.3 months] versus 3.5 months [range, 1.4-37.3 months], respectively).1,i
of infant patients with LAL-D experience symptom onset before 1 month of age1,ii
LAL-D in infants is fatal; rapid diagnosis is critical1
i Early growth failure was defined as fulfillment of 1 or more of the following criteria before 6 months of age: (1) decreased body weight across ≥2 of the 11 major percentiles on a standard World Health Organization (WHO) weight-for-age chart; (2) body weight below the tenth percentile on a standard WHO weight-for-age chart and no weight gain within the previous 2 weeks; (3) loss of ≥5% of birth weight in children >2 weeks of age.1
ii Four patients survived beyond 12 months of age; of those, 2 underwent HSCT, 1 underwent liver transplant and HSCT, and 1 was untreated.1
References: 1. Jones SA, et al. Genet Med. 2016;18:452-8. doi:10.1038/gim.2015.108.