A study of 3,963 patients with myasthenia gravis included in the National Rare Diseases Database (BNDMR) provides valuable insights into patient care at rare disease reference centres:
- the median interval between diagnosis and inclusion in the BNDMR is 3.8 months,
- the age at symptom onset ranges from 32 to 69 years, and is 65 years or older for a quarter of patients,
- the overall follow-up rate is one contact (of any kind) with the centre every 4.1 months, and the rate of consultations with a neurologist is 1.7 per year per patient, a rate that is almost in line with current recommendations (twice a year),
- only 13% of patients were able to benefit from teleconsultations,
- 12.9% were hospitalised at least once over an average follow-up period of six years,
- their survival rate stands at 92.2% at five years and 82.7% at ten years, figures higher than those recorded in Nordic registries (84% at five years and 70% at ten years) as well as in a study conducted in France using the National Health Data System (SNDS) (87% at five years),
- this difference could indicate better quality of care in Reference Centres, even though they are likely to treat a higher number of patients with severe forms of the disease.
