Ravulizumab in juvenile myasthenia gravis: An effective treatment in a 17‐year‐old girl
Domizia Vecchio, Amanda Papa, Giada Filisetti, Francesca Brustia, Cristoforo Comi, Roberto CantelloAbstract
Background
Ravulizumab, a monoclonal antibody against C5, is not currently approved for juvenile myasthenia gravis (MG).
Case Presentation
We are presenting a 14‐year‐old Moroccan girl diagnosed with antibody‐positive generalized MG first treated with pyridostigmine 240 mg, prednisone 1 mg/kg/day, and thymectomy. Her clinical status remained unchanged (MG Foundation of America Clinical Classification: IIIa) with azathioprine 3 mg/kg, and steroids up to 2 mg/kg/day. She also required three rescue therapies over the subsequent 8 months, gained 10 kg, and had insomnia. We started compassionate ravulizumab treatment, as part of Alexion Pharmaceuticals expanded access program. At screening, the Myasthenia Gravis–Activities of Daily Living scale total score was 4, and Quantitative Myasthenia Gravis total scores were 13 (handgrip and forced vital capacity not performed). She continued her treatment every 8 weeks, and, at week 60, her Myasthenia Gravis–Activities of Daily Living scale total score was 0, and Quantitative Myasthenia Gravis total score was 1. This status was achieved despite an ongoing steroid tapering, that was possibly allowed using a rapidly‐acting treatment. No significant infection or adverse effects were collected.
Conclusions
We are presenting the first case of juvenile MG treated with ravulizumab. Clinical assessment showed at week 60 an improvement of 12 points according to the Quantitative Myasthenia Gravis scoring system, and 3 points to the Myasthenia Gravis–Activities of Daily Living scale total score. This case report supports the efficacy and safety of ravulizumab in MG patients aged <18 years.