Human growth hormone stabilizes walking and improves strength in a patient with dominantly inherited calpainopathy

- Muscular dystrophies are characterized by progressive muscle wasting.
- There are no disease-specific treatments available for muscular dystrophies.
- Growth hormone was hypothesized to improve muscle strength in muscular dystrophies.
- Growth hormone supplementation may improve muscle strength in calpainopathies.

The aim was to investigate if daily low-dose treatment with recombinant human growth hormone (somatropine) can stabilize or improve muscle strength and walking capability in a patient with dominantly inherited calpainopathy. The patient was treated with daily injections of somatropine, except for a 6-month pause, over a period of 4.5 years. Efficacy was assessed by repeated muscle dynamometry tests and 6-minute walk tests (6MWT). Strength improved in most muscle groups on treatment, deteriorated in the 6-month off treatment, and improved again when treatment was resumed. The 6MWT stabilized during the initial 18-month treatment period, then deteriorated in the 6 months off treatment and improved to pre-trial levels when treatment was resumed.The findings suggest that supplementation with somatropine, within physiological ranges, may improve muscle strength and stabilize walking capability in a patient with calpainopathy. This finding calls for testing of somatropine supplementation in muscular dystrophies in a randomized study.