Eteplirsen for paediatric patients with Duchenne muscular dystrophy: A pooled-analysis.

BACKGROUND

Duchenne Muscular Dystrophy is a paediatric disorder resulting from a defective dystrophin gene. It causes progressive loss of muscle fibres, muscle weakness, and eventually loss of ambulation during adolescence, with death due to respiratory or cardiovascular complications soon afterwards. The drug eteplirsen has received support from medical experts and parents of affected children, but the FDA has delayed their decision for approval of this drug.

OBJECTIVE

This study analysed the results of previous studies to assess the safety and efficacy of the eteplirsen, and is the first pooled-analysis of its kind.

METHODS

A literature search of electronic databases was performed. Only human studies using eteplirsen were eligible.

RESULTS

A total of four relevant clinical studies were identified. A pooled-analysis was performed using data relating to percentage dystrophin-positive fibres obtained from muscle biopsy, and the six-minute walk test (6 MWT). The average increase in percentage dystrophin-positive fibres after treatment with eteplirsen was 24.23% (range -4 to 78; SD 24.44%). The average rate of decline in distance walked was 65metres (range -335 to 83; SD 100.08 m).

CONCLUSIONS

Whether or not this increase in percentage dystrophin-positive fibres and distance walked is clinically significant is unclear, and there is therefore a need for more clinical trials.