Acute liver failure worsened after respiratory syncytial virus infection in an infant with spinal muscular atrophy type I after receiving onasemnogene abeparvovec

Abstract

Background

Onasemnogene abeparvovec (OA) is an adeno-associated viral type 9 (AAV9) vector-based gene replacement therapy for infants with spinal muscular atrophy (SMA) if they are negative for anti-AAV9 antibodies. However, serious adverse events were reported after OA treatment.

Case presentation

A 2-month-old infant received a diagnosis of SMA type I because of progressive weakness and the result of genetic screening. The detectable anti-AAV9 antibody titers prompted us to start risdiplam immediately as the first-line treatment. OA was administered 7 months after birth when the titers declined to be negative. Fever and slightly elevated levels of transaminases were found one week after OA and improved spontaneously. Two months after OA, acute liver failure developed in association with respiratory syncytial virus infection. Intensive care with steroid therapy rescued this patient from life-threatening hepatopathy.

Discussion/conclusion

The anti-AAV9 antibody delayed OA in the early diagnosed case of SMA. The literature review found that all cases of liver failure occurred within the first 2 months of OA. Hepatopathy needs to be controlled in SMA cases with OA because of the potential factors to augment liver damage during infection.