Product information and disease description Place of product in therapy Disease description

Long-term morbidity

7 evidence topics · 6 sources

Comorbidities by organ system in a systematic review

“In our SLR, data on FOP comorbidity were extracted from 27 of the included studies, encompassing 73 distinct diseases and disorders. The three organ systems most affected by the conditions studied were, in order of severity, the cardiovascular (40–70%), skeletal (50–65%), and respiratory (20–42%) (Table 2).” (opens the source at this quote in a new tab)
“Falls and fractures were prevalent, affecting roughly 50% of the 260 patients with FOP collectively included in three of the eight studies reviewed” (opens the source at this quote in a new tab)

Comorbidities compared with matched controls in France

“Almost one-quarter (22.9%; n = 16) of individuals with FOP had a CCI score ≥ 1 compared with 3% (n = 21) of controls (Table 3). Notable long-term comorbidities occurred significantly more frequently among individuals with FOP compared with the control group and included neurological and muscular disorders (21.4% vs 0.9%; p < .0001), malignant tumors (4.3% vs 0.9%; p = .0407), inherited metabolic diseases (4.3% vs 0%; p = .0007), systemic disease (2.9% vs 0.1%; p = .0231), and ankylosing spondylitis (2.9% vs 0%; p = .0082; Table 3).” (opens the source at this quote in a new tab)

Disability severity by age

“According to these definitions, approximately half of all individuals with FOP (55.7%; n = 39/70) had a high severity of disability, whereas 37.1% (n = 26/70) had no identified severity between 2008 and 2019.” (opens the source at this quote in a new tab)

“FOP patients generally exhibited higher levels of disability, with 98.5% of them being disabled due to FOP and 64.2% holding disability certifications related to physical impairments.” (opens the source at this quote in a new tab)

Developmental arthropathy and degenerative joint disease

“Thus, the effects of the ACVR1 mutation on the normotopic skeletons of individuals who have FOP extend beyond malformation of the great toes and include both morphological defects and developmental arthropathy. Associated degenerative joint disease occurring at multiple sites starts in adolescence and progresses throughout life.” (opens the source at this quote in a new tab)

Hospitalization in the 12 months before registry enrollment

“in the 12 months prior to enrollment in the FOP Registry (a global disease registry operated by the International FOP Association [IFOPA] containing the largest and most detailed collection of clinical and medical information on people living with FOP), 27% of individuals had been hospitalized, with the most common reasons for admission being pain management (8.4%), fall/injury/accident/fracture (7.9%), and respiratory infection (4.4%)” (opens the source at this quote in a new tab)

Hospitalizations in a 10-year Swiss six-disease cohort

“Of the 2875 hospitalizations, 1146 were attributed to XLH, 925 to OI, 437 to FD, 219 to ACH, 87 to PHP, and 62 to FOP (Figure 1).” (opens the source at this quote in a new tab)
“Adult hospitalizations predominated in individuals with XLH (95.2%) and FOP (90.3%), while pediatric hospitalizations were common in individuals with ACH (52.1%) and OI (41.1%). Female sex was more prevalent in hospitalizations of individuals with ACH (63%) and FOP (61%), and similarly distributed in the other studied rare bone disease groups and the general population (48%-57%).” (opens the source at this quote in a new tab)

Comorbidity burden and lifelong hospitalization pattern in fibrodysplasia ossificans progressiva

“Individuals with FD and ACH showed comorbidity patterns broadly comparable to the general population. Estimates for PHP and FOP were variable due to small sample sizes, with a possible trend toward a higher comorbidity burden.” (opens the source at this quote in a new tab)
“Hospitalizations in PHP and FOP occurred throughout the lifespan, reflecting lifelong healthcare need, although clear trends were limited by small sample sizes.” (opens the source at this quote in a new tab)