An international survey (NCT04665323), sponsored by the pharmaceutical company Ipsen, which develops palovarotene, and translated into 11 languages was available online between January and April 2021.
It was completed by 219 people with FOP and 244 family members from 15 countries including France (20 patients and 21 family members). Almost half of the patients were 25 years old or older. The results were:
On the patient side
- Greater joint retractions in patients over 25 years of age than in those under 8 years of age, with the Patient-Reported Mobility Assessment (PRMA) score, which assesses the mobility of 12 joints and three body regions
- A loss of function (FOP Physical Function Questionnaire) of 76.1% in the over 25s and 29.6% in the under 8s.
- Alteration of quality of life correlated with the extent of joint limitations and worsening with age.
On the family side
- The quality of life of caregivers (more than 85% of whom were women) and family members was not correlated with either the degree of functional limitation or the age of the patient.
- The Zarit Burden Interwiew (ZBI) showed a low to moderate impact of POF on the health and psychological well-being of caregivers.
- Almost 80% of family members found it very difficult to learn about the diagnosis of POF.
- More than 85% reported significant stress when a flare-up of POF occurred.
- The majority of family members felt that caring for someone with FOP had little or no impact on their social interactions (74.7%), ability to make friends (83.5%) or to have a love life (82.9%).
Travel and career choices strongly affected
- Joint limitations have a strong impact on the ability to travel: 50% of people with a PRMA level 4 score cannot fly, more than a third cannot travel by car.
- More than 80% of adults with FOP report that the disease has affected their career choices.
- One third of family members and more than half of carers have had to adapt their working life to care for their relative with FOP.
First international Fibrodysplasia Ossificans Progressiva (FOP) Burden of Illness Survey reveals extent of social, economic, and quality of life impact of disease, Ipsen, 5 Sept 2022 press release.
