Table 2 Participant demographics for ALD cross-sectional study
From: Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD)
Cross-sectional study participants, n | 109 |
Sex, n (%) | |
 Male | 47 (43.1) |
 Female | 62 (56.9) |
Age in years | |
 Mean ± 1 SD | 51 ± 17 |
 Range | 18–83 |
Race, n (%) | |
 Asian | 3 (2.8) |
 Black/African American | 1 (0.9) |
 White | 98 (89.9) |
 Other | 7 (6.4) |
Hispanic or Latino, n (%) | |
 Yes | 15 (13.8) |
 No | 94 (86.2) |
Country, n (%) (16 total countries represented) | |
 United States | 54 (49.5) |
 Canada | 5 (4.6) |
 Australia | 10 (9.7) |
 United Kingdom | 16 (14.7) |
 France | 2 (1.8) |
 Argentina | 7 (6.4) |
 Bolivia | 2 (1.8) |
 Chile | 1 (0.9) |
 India | 1 (0.9) |
 Iran | 1 (0.9) |
 Ireland | 3 (2.8) |
 Mexico | 1 (0.9) |
 New Zealand | 3 (2.8) |
 Poland | 1 (0.9) |
 South Korea | 1 (0.9) |
 Sweden | 1 (0.9) |
U.S. states represented, n | 21 |
Canadian provinces represented (Ontario), n | 1 |
Employment status, n (%) | |
 Employed full-time | 29 (26.6) |
 Employed part-time | 9 (8.3) |
 On disability | 16 (14.7) |
 Not working/not on disability | 8 (7.3) |
 Retired | 30 (27.5) |
 Student | 6 (5.5) |
 Stay-at-home parent | 1 (0.9) |
 Self-employed | 7 (6.4) |
 Other | 3 (2.8) |
Has ALD impacted your employment status or choice?, n (%) | |
 Yes | 60 (55.1) |
 No | 40 (36.7) |
 I don't know | 8 (7.3) |
 Prefer not to answer | 1 (0.9) |
Highest level of education, n (%) | |
 Grade school | 2 (1.8) |
 High school | 29 (26.6) |
 Technical degree | 19 (17.5) |
 College | 33 (30.3) |
 Master's or Doctorate | 24 (22.0) |
 None | 2 (1.8) |
Has ALD prevented you from pursuing additional education?, n (%) | |
 Yes | 18 (16.5) |
 No | 87 (79.8) |
 I don't know | 4 (3.7) |
Marital status, n (%) | |
 Married | 61 (56.0) |
 Single | 26 (23.9) |
 Widowed | 3 (2.7) |
 Divorced | 13 (12.0) |
 Separated | 3 (2.7) |
 Registered partnership | 3 (2.7) |
Has ALD impacted your marital status or decision to pursue relationships?, n (%) | |
 Yes | 35 (32.1) |
 No | 70 (64.2) |
 I don't know | 4 (3.7) |
Diagnosed with ALD?, n (%) | |
 Yes | 59 (54.1) |
 No | 43 (39.5) |
 I don't know | 7 (6.4) |
Diagnosed with AMN?, n (%) | |
 Yes | 71 (65.1) |
 No | 27 (24.8) |
 I don't know | 11 (10.1) |
Diagnosed with cerebral form of ALD?, n (%) | |
 Yes | 18 (16.5) |
 No | 81 (74.3) |
 I don't know | 10 (9.2) |
Diagnosed with Addison's disease?, n (%) | |
 Yes | 35 (32.1) |
 No | 71 (65.1) |
 I don't know | 3 (2.8) |
Ever been in adrenal crisis?, n (%) | |
 Yes | 22 (20.2) |
 No | 82 (75.2) |
 I don't know | 5 (4.6) |
Years since diagnosis | |
 Mean ± 1 SD | 16 ± 11 |
 Range | 1–40 |
Years since first noticed symptoms | |
 Mean ± 1 SD | 15 ± 11 |
 Range | 0–50 |
Ever misdiagnosed?, n (%) | |
 Yes | 38 (34.9) |
 No | 68 (62.4) |
 I don't know | 3 (2.7) |
Ambulation, n (%) | |
 Walk independently without assistance | 50 (45.9) |
 Primarily use a cane or crutches | 34 (31.2) |
 Primarily use a walker | 10 (9.2) |
 Use a wheelchair or motorized scooter sometimes and walk sometimes | 7 (6.4) |
 Primarily use a wheelchair or motorized scooter | 8 (7.3) |
Hours of home health aide per week, n (%) | |
 None | 75 (68.8) |
 1–5 h | 15 (13.8) |
 6–10 h | 6 (5.5) |
 16–20 h | 3 (2.7) |
 Greater than 20 h | 10 (9.2) |
Speech, n (%) | |
 Talk clearly and have no changes in speech | 89 (81.7) |
 Some speech changes | 17 (15.6) |
 Impaired speech, and people occasionally ask to repeat words or phrases | 2 (1.8) |
 Impaired speech that is often not understood by others | 1 (0.9) |
Positive genetic test for ABCD1 gene mutation?, n (%) | |
 Yes | 89 (81.6) |
 No | 5 (4.6) |
 No genetic testing | 11 (10.1) |
 I don't know | 4 (3.7) |
Functional ability, n (%) | |
 No symptoms | 4 (3.7) |
 No significant disability | 26 (23.8) |
 Slight disability | 32 (29.4) |
 Moderate disability | 29 (26.6) |
 Moderately severe disability | 18 (16.5) |
Ever received bone marrow or stem cell transplant?, n (%) | |
 Yes | 5 (4.6) |
 No | 104 (95.4) |
Current treatments, n (%)a | |
 Hormone replacement, steroid medications, or corticosteroids | 34 (31.2) |
 High dose antioxidants (OTC) | 4 (3.7) |
Lorenzo's oil | 2 (1.8) |
 Spasticity-reducing medications (Baclofen, Tazanidine, Botox, etc.) | 33 (30.3) |
 Neuropathic pain medications or anti-epileptic medications (Neurontin e.g. Gabapentin) | 32 (29.4) |
 Medications for overactive bladder or bowel | 21 (19.3) |
 Cannabidiol (CBD) | 15 (13.8) |
 Anti-depressants or anti-anxiety medications | 24 (22.0) |
 Physical therapy | 32 (29.4) |
 None | 20 (18.4) |