OBJECTIVES: Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder of progressive heterotopic ossification, causing cumulative disability. Most individuals with FOP become immobilized by the third decade of life, requiring lifelong assistance with activities of daily living. Here, we characterize the use of aids, assistive devices and adaptations (AADAs) as a surrogate measure of FOP progression in individuals with FOP enrolled in a 36‑month, global natural history study (NHS; NCT02322255).

METHODS: Individuals with FOP aged ≤65 years with a documented ACVR1^R206H mutation participated in the study for up to 36 months. Baseline and new-onset use of AADAs at 36 months are reported. AADA use was indicated via a list grouped into 12 categories. Data were analyzed using descriptive statistics.

RESULTS: Data on AADA usage were available for 108/114 participants at Baseline, and use of new AADAs for 37/41 participants with any available data on AADA use at Month 36. The most common AADA categories at Baseline (n=108) were personal care tools/aids (71.3%), care attendants (61.1%), bedroom aids/devices (53.7%), home adaptations (49.1%), mobility aids (48.1%), and medical therapies for daily living (45.4%). The same AADA categories were used at Month 36 (n=41) by 65.9%, 65.9%, 73.2%, 48.8%, 51.2%, and 51.2% of participants, respectively. At Month 36, 37/41 (90.2%) participants reported new-onset AADAs; the most common new AADAs across age groups were care attendants (48.6%), personal care tools/aids, and bathroom aids/devices (both 45.9%), bedroom aids/devices, home adaptations, and medical therapies for daily living (all 43.2%). The majority of participants aged 25–≤65 years (90.9% [10/11]) reported using personal care tools/aids and bathroom aids/devices at Month 36.

CONCLUSIONS: Specific types of AADAs used varied across age groups and over 36 months. Overall, reported new-onset AADA use increased at Month 36 versus Baseline, suggesting worsening disability over time.