Table 3.
Medical history by limb lengthening subgroup
| 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) |
*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
**Terms are coded using MedDRA dictionary and subsequently mapped into 1 of 10 predefined categories described in the Health Economic Exploratory Analysis Plan
ENT Ear, nose, throat