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Table 2 Demographics and Baseline Disease by Age Category

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

 

<8 Yrs (N=17)

8 to <15 Yrs (N=36)

15 to <25 Yrs (N=34)

≥25 to ≤65 Yrs (N=27)

Total (N=114)

Males, n (%)

9 (52.9)

24 (66.7)

16 (47.1)

13 (48.1)

62 (54.4)

Age (years)

 Mean ±SD

5.9 ±1.1

11.4 ±2.1

18.9 ±3.1

31.7 ±6.7

17.6 ±9.7

 Median (min, max)

6.0 (4, 7)

11.0 (8, 14)

18.5 (15, 24)

30.0 (25, 56)

15.0 (4, 56)

Age at 1st flare-up

 Mean ±SD

2.9 ±2.1

4.4 ±3.6

5.6 ±4.8

7.1 ±5.0

5.2 ±4.4

 Median (min, max)

2.0 (1, 6)

4.0 (0, 13)

3.5 (0, 17)

5.0 (0, 20)

4.0 (0, 20)

Years since last flare-up

 Mean ±SD

0.7 ±0.9

1.5 ±2.8

1.5 ±1.9

2.3 ±3.5

1.6 ±2.6

 Median (min, max)

0.3 (0, 3)

0.5 (0, 14)

0.7 (0, 7)

0.9 (0, 15)

0.5 (0, 15)

Number of flare-ups in the past 12 months (in those with flare-ups)

 n

13

25

21

17

76

 Mean ±SD

2.9 ±2.6

6.8 ±11.2

2.2 ±1.8

1.9 ±1.6

3.8 ±6.9

 Median (min, max)

2.0 (1, 10)

2.0 (1, 40)

1.0 (1, 8)

1.0 (1, 7)

2.0 (1, 40)

CAJIS total score1

 Mean ±SD

5.6 ±3.9

9.2 ±4.6

13.9 ±6.7

16.5 ±7.3

11.8 ±7.0

 Median (min, max)

6.0 (1, 15)

8.5 (1, 20)

13.5 (1, 26)

18.0 (1, 30)

10.5 (1, 30)

FOP-PFQ % worst total score2

 Mean ±SD

34.8 ±26.1

44.4 ±20.2

43.1 ±31.0

58.4 ±35.8

46.3 ±27.1

 Median (min, max)

41.8 (1, 84.6)

45.2 (1.9, 82.7)

38.4 (0, 100)

54.5 (0, 100)

45.5 (0, 100)

PROMIS Global Physical Health (adult) or PROMIS Global Health (pediatric, parent proxy) (T-score)3

 Mean ±SD

47.6 ±9.4

41.8 ±8.7

44.1 ±9.0

42.5 ±7.9

NA

 Median (min, max)

48.3 (33, 24)

41.7 (24, 57)

44.9 (24, 68)

42.3 (27, 54)

 

PROMIS Global Mental Health (adult) (T-score)3

 Mean ±SD

NA

NA

53.6 ±9.9

51.4 ±8.5

52.6 ±9.3

 Median (min, max)

  

53.3 (28, 68)

53.3 (39, 68)

53.3 (28, 68)

Total body HO volume, excluding head (mm3)

 Mean ±SD

61951 ±75221

159303 ±161779

380751 ±363142

651913 ±674454

324631 ±440977

 Median (min, max)

21692 (0, 224019)

130509 (0, 828262)

258543 (0, 1504849)

481524 (48844, 2833946)

173536 (0, 2833946)

Number of regions with HO4

 Mean ±SD

3.1 ±2.6

5.1 ±2.7

7.4 ±3.4

8.8 ±3.7

6.4 ±3.7

 Median (min, max)

4.0 (0, 7)

5.0 (0, 14)

8.0 (0, 13)

8.5 (3, 14)

6.0 (0, 14)

  1. 1CAJIS assessed range of motion across 12 joints (left and right shoulders, elbows, wrists, hips, knees, and ankles) and three body regions (jaw, cervical spine [neck], and thoraco-lumbar spine). Each noted as: 0=uninvolved; 1=partially involved; 2=completely ankylosed. Total scores range from 0 to 30 with higher scores indicating more severe limitations in mobility.
  2. 2FOP-PFQ used transformed scores expressed as a percentage of the worst possible score. Lower percentages indicate better functioning; higher percentages indicate worse functioning.
  3. 3Distributions standardized such that a T-score of 50 (SD of 10) represents the average for the United States general population. Higher T-scores indicate better physical/mental health.
  4. 4Fifteen possible regions (neck, lower spine/abdomen, upper spine/chest; and both shoulders, elbows, wrists, hips, knees, and ankles).
  5. Abbreviations: CAJIS=Cumulative Analogue Joint Involvement Scale, HO=heterotopic ossification, max=maximum, min=minimum, FOP-PFQ=Fibrodysplasia Ossificans Progressiva-Patient Function Questionnaire, NA=not applicable, PROMIS=Patient-Reported Outcome Measure Information System, SD=standard deviation, Yrs=years
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172