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Table 2 Demographics and baseline disease by age category

From: 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

 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 scorea

 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 scoreb

 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)c

 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)c

 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

61,951 ± 75,221

159,303 ± 161,779

380,751 ± 363,142

651,913 ± 674,454

324,631 ± 440,977

 Median (min, max)

21,692 (0, 224,019)

130,509 (0, 828,262)

258,543 (0, 1,504,849)

481,524 (48,844, 2,833,946)

173,536 (0, 2,833,946)

Number of regions with HOd

 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. aCAJIS 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. bFOP-PFQ used transformed scores expressed as a percentage of the worst possible score. Lower percentages indicate better functioning; higher percentages indicate worse functioning
  3. cDistributions 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. dFifteen 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