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Table 3 Medical history by limb lengthening subgroup

From: Lifetime impact of achondroplasia study in Europe (LIAISE): findings from a multinational observational study

Medical history* n (%)/events (rate per 100 person-years)

Subgroup

Overall

Limb lengthening (N = 40)

No limb lengthening (N = 146)

N = 186

Patient historical years

663.0

1,721.6

2,384.6

Medical and surgical complications**

  ≥ 1 general medical history

35 (87.5%)/170 (25.6)

128 (87.7%)/773 (44.9)

163 (87.6%)/943 (39.5)

 Foramen magnum stenosis

2 (5.0%)/2 (0.3)

50 (34.2%)/56 (3.3)

52 (28.0%)/58 (2.4)

 Gastroesophageal issues

8 (20.0%)/8 (1.2)

24 (16.4%)/48 (2.8)

32 (17.2%)/56 (2.3)

 Genu varum/valgum

3 (7.5%)/3 (0.5)

37 (25.3%)/44 (2.6)

40 (21.5%)/47 (2.0)

 Infections/infestations

4 (10.0%)/8 (1.2)

23 (15.8%)/45 (2.6)

27 (14.5%)/53 (2.2)

 Other ENT issues (excluding otitis media)

24 (60.0%)/58 (8.7)

90 (61.6%)/261 (15.2)

114 (61.3%)/319 (13.4)

 Other orthopaedic problems

22 (55.0%)/44 (6.6)

47 (32.2%)/119 (6.9)

69 (37.1%)/163 (6.8)

 Otitis media

1 (2.5%)/1 (0.2)

11 (7.5%)/16 (0.9)

12 (6.5%)/17 (0.7)

 Pain (any type)

11 (27.5%)/25 (3.8)

34 (23.3%)/66 (3.8)

45 (24.2%)/91 (3.8)

 Spinal cord compression/stenosis

10 (25.0%)/10 (1.5)

37 (25.3%)/49 (2.8)

47 (25.3%)/59 (2.5)

 Spinal deformities (kyphosis/lordosis)

7 (17.5%)/11 (1.7)

45 (30.8%)/69 (4.0)

52 (28.0%)/80 (3.4)

Surgical burdenb

  ≥ 1 surgery/number of surgeries

40 (100%)/223 (33.6)

94 (64.4%)/290 (6.8)

134 (72.0%)/513 (21.5)

 Brainstem decompression

2 (5.0%)/2 (0.3)

24 (16.4%)/24 (1.4)

26 (14.0%)/26 (1.1)

 Limb lengthening

40 (100%)/113 (17.0)

0

40 (21.5%)/113 (4.7)

 Middle ear procedures

6 (15.0%)/8 (1.3)

37 (25.4%)/59 (3.3)

43 (23.1%)/67 (2.8)

 Nerve decompression

1 (2.5%)/1 (0.2)

6 (4.1%)/7 (0.4)

7 (3.8%)/8 (0.3)

 Shunt insertion

2 (5.0%)/2 (0.3)

5 (3.4%)/5 (0.3)

7 (3.8%)/7 (0.3)

 Shunt revision

0

4 (2.7%)/8 (0.5)

4 (2.2%)/8 (0.3)

Spinal correction

2 (5.0%)/4 (0.6)

8 (5.5%)/11 (0.6)

10 (5.4%)/15 (0.6)

 Tonsillectomy/adenoidectomy

10 (25.0%)/13 (2.0)

39 (26.7%)/55 (3.2)

49 (26.3%)/68 (2.9)

 Other

24 (60.0%)/80 (12.1)

58 (39.7%)/121 (7.0)

82 (44.1%)/201 (8.4)

Healthcare professional visits

  ≥ 1 health care professional visit/number of visits

16 (40.0%)/55 (8.3)

97 (66.4%)/460 (26.7)

113 (60.8%)/515 (21.6)

 Cardiologist

0

4 (2.7%)/6 (0.3)

4 (2.2%)/6 (0.3)

 Dentist

1 (2.5%)/1 (0.2)

2 (1.4%)/2 (0.1)

3 (1.6%)/3 (0.1)

 Dermatologist

1 (2.5%)/1 (0.2)

3 (2.1%)/4 (0.2)

4 (2.2%)/5 (0.2)

 Dietician

0

4 (2.7%)/4 (0.2)

4 (2.2%)/4 (0.2)

 ENT physician and/or surgeon

4 (10.0%)/5 (0.8)

24 (16.4%)/40 (2.3)

28 (15.1%)/45 (1.9)

 Emergency Doctor

0

5 (3.4%)/9 (0.5)

5 (2.7%)/9 (0.4)

 Endocrinologist

0

3 (2.1%)/7 (0.4)

3 (1.6%)/7 (0.3)

 Gastroenterologist

0

3 (2.1%)/5 (0.3)

3 (1.6%)/5 (0.2)

 General practitioner

4 (10.0%)/16 (2.4)

23 (15.8%)/120 (7.0)

27 (14.5%)/136 (5.7)

 Geneticist

0

5 (3.4%)/7 (0.4)

5 (2.7%)/7 (0.3)

 Gynaecologist

0

3 (2.1%)/4 (0.2)

3 (1.6%)/4 (0.2)

 Mental Health

1 (2.5%)/1 (0.2)

3 (2.1%)/3 (0.2)

4 (2.2%)/4 (0.2)

 Neurologist

1 (2.5%)/1 (0.2)

8 (5.5%)/9 (0.5)

9 (4.8%)/10 (0.4)

 Neuropediatric

1 (2.5%)/1 (0.2)

2 (1.4%)/12 (0.7)

3 (1.6%)/13 (0.5)

 Neurosurgeon

0

5 (3.4%)/6 (0.3)

5 (2.7%)/6 (0.3)

 Occupational therapist

1 (2.5%)/1 (0.2)

5 (3.4%)/5 (0.3)

6 (3.2%)/6 (0.3)

 Ophthalmologist

2 (5.0%)/2 (0.3)

7 (4.8%)/7 (0.4)

9 (4.8%)/9 (0.4)

 Orthopaedic

3 (7.5%)/6 (0.9)

21 (14.4%)/30 (1.7)

24 (12.9%)/36 (1.5)

 Paediatrician

1 (2.5%)/1 (0.2)

7 (4.8%)/56 (3.3)

8 (4.3%)/57 (2.4)

 Physiotherapist

7 (17.5%)/10 (1.5)

52 (35.6%)/75 (4.4)

59 (31.7%)/85 (3.6)

 Respiratory physician

2 (5.0%)/3 (0.5)

4 (2.7%)/5 (0.3)

6 (3.2%)/8 (0.3)

 Speech therapist

4 (10.0%)/4 (0.6)

21 (14.4%)/27 (1.6)

25 (13.4%)/31 (1.3)

 Other

2 (5.0%)/2 (0.3)

14 (9.6%)/17 (1.0)

16 (8.6%)/19 (0.8)

Medical or surgical examination

  ≥ 1 medical or surgical examination/number of examinations

40 (100%)/821 (123.8)

145 (99.3%)/2,491(144.7)

185 (99.5%)/3,312 (138.9)

 Audiology

2 (5.0%)/3 (0.5)

37 (25.3%)/82 (4.8)

39 (21.0%)/85 (3.6)

 Cardiovascular

6 (15.0%)/21 (3.2)

36 (24.7%)/75 (4.4)

42 (22.6%)/96 (4.0)

 ENT

12 (30.0%)/31 (4.7)

69 (47.3%)/323 (18.8)

81 (43.5%)/354 (14.8)

 Gastrointestinal

5 (12.5%)/12 (1.8)

13 (8.9%)/30 (1.7)

18 (9.7%)/42 (1.8)

 General surgical

5 (12.5%)/24 (3.6)

7 (4.8%)/17 (1.0)

12 (6.5%)/41 (1.7)

 Neurological

12 (30.0%)/46 (6.9)

61 (41.8%)/257 (14.9)

73 (39.2%)/303 (12.7)

 Orthopaedic

23 (57.5%)/247 (37.3)

78 (53.4%)/366 (21.3)

101 (54.3%)/613 (25.7)

 Radiological

32 (80.0%)/218 (32.9)

98 (67.1%)/519 (30.1)

130 (69.9%)/737 (30.9)

 Respiratory

7 (17.5%)/10 (1.5)

31 (21.1%)/79 (4.6)

38 (20.4%)/89 (3.7)

 Urological

1 (2.5%)/1 (0.2)

10 (6.8%)/19 (1.1)

11 (5.9%)/20 (0.8)

 Other

24 (60.0%)/208 (31.4)

108 (74.0%)/724 (42.1)

132 (71.0%)/932 (39.1)

  1. *Each patient may have had several medical history events. Percentages are based on the overall number of subjects in the Full Analysis Set. Event rates per 100 person-years are calculated as the number of events divided by the total number of patient historical years multiplied by 100. Patient historical years are defined as the time between earliest collected history and enrolment date
  2. **Terms are coded using MedDRA dictionary and subsequently mapped into 1 of 10 predefined categories described in the Health Economic Exploratory Analysis Plan
  3. ENT Ear, nose, throat