Product information and disease description Place of product in therapy Disease description Burden of Fibrodysplasia ossificans progressiva

Humanistic burden and health-related quality of life

11 evidence topics · 7 sources

EQ-5D-5L and PROMIS Global Health in an international burden of illness survey

“Mean (SD) EQ-5D-5L index score for patients (≥13 years; n = 152) was 0.24 (0.36). Patients with greater restrictions in joint mobility had worse QoL, with mean (SD) EQ-5D-5L index score decreasing from 0.61 (0.26; n = 20) for patients with PRMA total scores in Level 1 to 0.05 (0.26; n = 62) for those in Level 4 (Figure 3).” (opens the source at this quote in a new tab)
“Mean (SD) PROMIS (pediatric) global total T-score for patients aged 5–14 years was 42.3 (9.9; n = 50).” (opens the source at this quote in a new tab)
“Mean (SD) PROMIS global physical and mental health T-scores for patients (≥15 years) were 37.6 (9.0; n = 140) and 44.9 (9.5; n = 137), respectively.” (opens the source at this quote in a new tab)

PROMIS Global Health Scale scores by age in the IFOPA registry

“The mean total scores for age groups 0– < 9 years, 9–15 years, and > 15 years were 25.4, 26.6, and 32, respectively.” (opens the source at this quote in a new tab)
“There is a strong, statistically significant association between the total PROMIS Global Health score and the number of anatomic sites subjectively reported as being affected by HO.” (opens the source at this quote in a new tab)

FOP-PFQ physical function by age

“There was a clear trend for worsening mobility and joint function with age; 55.3% of patients aged ≥25 years had PRMA total scores in Level 4, compared with no patients aged <8 years (Figure 2(a)), and mean (SD) FOP-PFQ score increased from 29.6 (25.1) for patients aged <8 years (n = 25) to 76.1 (26.7) for patients aged ≥25 years (n = 104; Supplementary Figure S1).” (opens the source at this quote in a new tab)

Validation of the FOP Physical Function Questionnaire

“Factor analysis confirmed a two-factor solution: Mobility and Upper Extremity.” (opens the source at this quote in a new tab)
“The FOP-PFQ was demonstrated to be a reliable, valid measure that may be responsive to change in individuals with FOP, although some results were inconclusive for pediatric versions.” (opens the source at this quote in a new tab)

Development and factor structure of the FOP-PFQ

“Development of the adult and pediatric versions of the FOP-PFQ involved a literature review, two iterative phases of qualitative work with participants, and clinical expert review.” (opens the source at this quote in a new tab)
“The comprehensiveness, relevance, and understandability of the adult measure was evaluated throughout and resulted in the final 28-item questionnaire.” (opens the source at this quote in a new tab)
“Initial exploratory factor analysis models based on retention of factors with eigenvalues of ≥1 suggested a two-factor solution (Factor 1: Upper Extremity function; Factor 2: Mobility function), which was confirmed by follow-up models that pre-specified two factors.” (opens the source at this quote in a new tab)

Reliability of the FOP-PFQ

“The FOP-PFQ yielded high alphas and ICCs at Baseline and Month 12.” (opens the source at this quote in a new tab)
“This indicates that FOP-PFQ scores were stable over the 1–3-week interval between questionnaires for individuals with no flare-ups.” (opens the source at this quote in a new tab)

Convergent and known-groups validity of the FOP-PFQ

“Adult and pediatric FOP-PFQ scores were worse for individuals with worse physical function and correlated with other measures of physical function, supporting convergent validity (Table 3).” (opens the source at this quote in a new tab)
“FOP-PFQ scores were significantly worse in groups of individuals with more severe disease, supporting known-groups validity, suggesting the instrument performs effectively as a measure of severity and potentially disease progression over time.” (opens the source at this quote in a new tab)

Correlation of HO volume with physical function

“There were significant (p < 0.0001) correlations between the objective assessment of HO volume and clinical assessments of CAJIS (r = 0.57) and FOP-PFQ (r = 0.52).” (opens the source at this quote in a new tab)

Pain severity and quality of life in the FOP Registry (PROMIS-based)

“Furthermore, independent of the flare‐up status, the severity of pain in FOP patients was found to be significantly anti‐correlated with emotional health, physical health, and overall quality‐of‐life.” (opens the source at this quote in a new tab)

EQ-5D health utility by age in China

“The average health utility values of patients < 8 and 8–15 years were 0.700 ± 0.163, 0.618 ± 0.202 measured by EQ-5D-Y. The utility values of patients ≥ 16 years were 0.221 ± 0.336 measured by EQ-5D-5L, significantly lower than the younger patients (Table 3).” (opens the source at this quote in a new tab)

SF-36 in a Mexican cohort

“Eight patients, mean age 30.2 years, were included. The physical dimension was the most affected, with an average of 25.5 points.” (opens the source at this quote in a new tab)