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Table 1 Timing of standard assessments over the 3-year study

From: Natural history of fibrodysplasia ossificans progressiva: cross-sectional analysis of annotated baseline phenotypes

Assessment/Procedure Screening/Baseline First 3 weeks Every 3 months Every 6 months Every 12 months
Clinic visit X     X
Telephone contact   X X X  
Informed consent/assent X     
Assess for eligibility X     
Medical and FOP history X     
Prior/concomitant medications X X X X X
Post-baseline medical events     X X
Physical examination X     X
Linear height (≥ 18 years old) X     X
Knee and sitting height (< 18 years old) X     X
Body weight X     X
Vital signs X     X
Electrocardiogram X     X
Pulmonary function tests X     X
Pulse oximetry X     X
Clinical laboratory assessmentsa X     X
Blood and urine for biomarkers samplesb X     X
CAJIS X     X
Knee and hand/wrist x-rays (< 18 years old)      X
WBCT (excluding head) X     X
FOP assistive devices assessment X    X X
FOP-PFQ assessment X    X X
PROMIS Global Scales X    X X
Columbia-Suicide Severity Rating Scale X     X
Genotyping X     
Adverse events   X X X X
  1. aIncludes triglycerides, alanine aminotransferase, aspartate aminotransferase, amylase, total cholesterol, lipase, total bilirubin, alkaline phosphatase, hemoglobin, hematocrit, platelets, and white blood cell count
  2. bBone and cartilage biomarkers include osteocalcin, bone specific alkaline phosphatase, P1CP-C-terminal propeptide of type 1 procollagen, P1NP N-terminal propeptide of type 1 procollagen, cartilage-derived retinoic acid-sensitive protein, and c-terminal telopeptide. The angiogenesis biomarker was urinary basic fibroblast growth factor. The inflammation biomarkers included erythrocyte sedimentation rate, c-reactive protein, interleukin-6, interleukin-1 beta, tumor necrosis factor-alpha, creatinine phosphokinase, and lactate dehydrogenase
  3. Abbreviations: CAJIS = Cumulative Analogue Joint Involvement Scale, FOP = Fibrodysplasia Ossificans Progressiva, FOP-PFQ = Fibrodysplasia Ossificans Progressiva-Patient Function Questionnaire, PROMIS = Patient-Reported Outcome Measure Information System, WBCT = whole body computed tomography