Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index
Abstract
:1. Introduction
2. Materials and Methods
3. Results
3.1. Study Population
3.2. Disease Burden
3.3. Treatments
3.4. Recreational Drug-Use
3.5. Patient Benefit Index
3.6. Patient Needs
4. Discussion
4.1. Burden of Disease
4.2. Therapies
4.3. Recreational Drug-Use
4.4. Patient Benefit Index (PBI)
4.5. Patient Needs: Items
4.6. Patient Needs: Correlations
4.7. Strenghts and Limitations
5. Conclusions
Supplementary Materials
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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All | EBS | JEB | DDEB | RDEB | |
---|---|---|---|---|---|
Participants, n (%) | 68 (100.0) | 29 (42.6) | 10 (14.7) | 23 (33.8) | 6 (8.8) |
Age, mean ± SD | 41.1 ± 16.4 | 39.7 ± 15.4 | 49.3 ± 19.4 | 41.5 ± 16.8 | 33.2 ± 12.5 |
Time since diagnosis (years), mean ± SD | 15.3 ± 8.0 | 12.8 ± 7.4 | 19.6 ± 5.9 | 16.1 ± 7.9 | 16.7 ± 11.3 |
Sex, n (%) | |||||
Female | 38 (55.9) | 17 (58.6) | 4 (40.0) | 13 (56.5) | 4 (66.7) |
Male | 30 (44.1) | 12 (41.4) | 6 (60.0) | 10 (43.5) | 2 (33.3) |
Presence of pain, n (%) | 46 (67.6) | 15 (51.7) | 10 (100) | 15 (65.2) | 6 (100) |
Presence of pruritus, n (%) | 50 (73.5) | 17 (58.6) | 8 (80) | 19 (82.6) | 6 (100) |
Pain NRS, median (IQR) | |||||
Morning | 2.0 (0.0–5.0) | 0.0 (0.0–2.5) * | 6.0 (5.0–7.0) *,** | 2.0 (0.0–3.0) ** | 2.5 (1.0–7.0) |
Afternoon | 2.0 (0.0–5.0) | 0.0 (0.0–2.0) * | 5.0 (3.0–7.3) *,** | 2.0 (0.0–4.0) ** | 3.0 (1.0–6.5) |
Evening | 2.0 (0.0–6.0) | 0.0 (0.0–2.5) * | 6.0 (5.0–7.3) *,** | 2.0 (0.0–6.0) ** | 4.0 (1.0–7.0) |
Mean all-day | 2.0 (0.0–5.0) | 0.7 (0.0–2.0) * | 5.8 (4.8–6.6) *,** | 1.3 (0.0–4.3) ** | 3.2 (1.0–7.1) |
Pruritus NRS, median (IQR) | |||||
Morning | 1.0 (0.0–3.5) | 0.0 (0.0–2.0) * | 1.5 (0.0–4.3) | 1.0 (0.0–4.0) | 4.5 (2.5–7.0) * |
Afternoon | 1.0 (0.0–3.5) | 0.0 (0.0–2.0) * | 1.0 (0.0–3.3) | 1.0 (1.0–4.0) | 3.5 (3.0–5.8) * |
Evening | 2.0 (0.0–6.0) | 1.0 (0.0–4.0) | 5.5 (0.8–7.0) | 3.0 (1.0–6.0) | 5.0 (2.8–7.5) |
Mean all-day | 1.7 (0.0–4.0) | 0.7 (0.0–2.3) * | 2.8 (0.5–4.1) | 2.0 (1.0–4.6) | 4.7 (2.6–6.3) * |
Self-reported Severity, n (%) | |||||
Mild | 37 (54.4) | 19 (65.5) | 2 (20.0) | 16 (69.6) | 0 (0.0) |
Moderate | 20 (29.4) | 9 (31.0) | 3 (30.0) | 6 (26.1) | 2 (33.3) |
Severe | 11 (16.2) | 1 (3.5) | 5 (50.0) | 1 (4.3) | 4 (66.7) |
(a) Treatment Indication | Drug Class | Number of Participants (%) | Number of (Simultaneous) Treatments | Total Count (%) (n = 68] | Count by EB-subtype | |||
EBS (%) (n = 29) | DDEB (%) (n = 23) | RDEB (%) (n = 6) | JEB (%) (n = 10) | |||||
Pain | Paracetamol | 24 (35.1) | None | 36 (52.9) | 21 (72.4) | 8 (34.8) | 2 (33.3) | 5 (50.0) |
NSAID | 8 (11.8) | 1 | 24 (35.3) | 7 (24.1) | 11 (47.8) | 4 (66.7) | 2 (20.0) | |
Opioid | 3 (4.4) | 2 | 5 (7.4) | 1 (3.4) | 3 (13.0) | - | 1 (10.0) | |
Anti-epileptic | 2 (2.9) | 3 | 1 (1.5) | - | 1 (4.3) | - | - | |
CBM | 1 (1.5) | 4 | 1 (1.5) | - | - | - | 1 (10.0) | |
5 | 1 (1.5) | - | - | - | 1 (10.0) | |||
Pruritus | Antihistamine | 14 (20.6) | None | 51 (75.0) | 24 (82.8) | 15 (65.2) | 4 (66.7) | 8 (80.0) |
Calcineurin Inhibitor | 3 (4.4) | 1 | 14 (20.6) | 5 (17.2) | 5 (21.7) | 2 (33.3) | 2 (20.0) | |
Corticosteroid | 3 (4.4) | 2 | 2 (2.9) | - | 2 (8.7) | - | - | |
5HT3-Antagonist | 1 (1.5) | 3 | 1 (1.5) | - | 1 (4.3) | - | - | |
(b) Treatment Indication | Drug Class | Number of Participants (%) | Number of Treatments Previously Used | Total Count (%) [n = 68] | Count by EB-subtype | |||
EBS (%) (n = 29) | DDEB (%) (n = 23) | RDEB (%) (n = 6) | JEB (%) (n = 10) | |||||
Pain and Pruritus | Paracetamol | 55 (80.9) | None | 25 (36.8) | 11 (37.9) | 10 (43.5) | 2 (33.3) | 2 (20.0) |
NSAID | 46 (67.6) | 1 | 6 (8.8) | 2 (6.9) | 3 (13.0) | - | 1 (10.0) | |
Opioid | 28 (41.2) | 2 | 9 (13.2) | 4 (13.8) | 3 (13.0) | 1 (16.7) | 1 (10.0) | |
Antihistamine | 25 (36.8) | 3 | 11 (16.2) | 5 (17.2) | 4 (17.4) | 1 (16.7) | 1 (10.0) | |
Steroid | 16 (23.5) | 4 | 9 (13.2) | 4 (13.8) | 1 (4.3) | 1 (16.7) | 3 (30.0) | |
Benzodiazepine | 13 (19.1) | 5 | 4 (5.9) | 2 (6.9) | - | 1 (16.7) | 1 (10.0) | |
CBM | 12 (17.6) | 6 | 1 (1.5) | - | 1 (4.3) | - | - | |
Antidepressant | 10 (14.7) | 7 | 3 (4.4) | 1 (3.4) | 1 (4.3) | - | 1 (10.0) | |
Anti-epileptic | 4 (5.9) | |||||||
Ketamine | 1 (1.5) | |||||||
(c) Type of Recreational Drug | Number of Participants (%) | Effect on EB | ||||||
EB Symptom Reduction | EB Symptom Worsening | Ability to Relax | No effect on EB | Missing | ||||
n (%) | ||||||||
Alcohol | 49 (72.1) | 7 (14.3) | 4 (8.2) | 4 (8.2) | 6 (12.2) | 28 (57.1) | ||
Tobacco | 12 (17.6) | - | - | 5 (41.7) | 3 (25.0) | 4 (30.0) | ||
Cannabis | 8 (11.8) | 5 (62.5) | 1 (12.5) | 3 (37.5) | 1 (12.5) | - | ||
Other drugs | 1 (1.5) | - | - | - | 1 (100.0) | - | ||
Does not use | 14 (20.6) | - |
n | Median (IQR) | Range (Min–Max) | PBI ≥ 1 * (%) | ||
---|---|---|---|---|---|
(a) PBI | Global | 68 | 1.34 (0.68–2.58) | 0.00–4.00 | 60.3 |
EBS | 29 | 0.96 (0.59–2.20) | 0.00–3.92 | 48.3 | |
RDEB | 6 | 1.35 (0.43–2.34) | 0.31–3.52 | 66.7 | |
DDEB | 23 | 1.61 (0.80–2.93) | 0.00–4.00 | 60.9 | |
JEB | 10 | 2.28 (0.84–2.79) | 0.32–3.81 | 80.0 | |
Male | 30 | 1.41 (0.83–2.86) | 0.00–4.00 | 60.0 | |
Female | 38 | 1.31 (0.61–2.38) | 0.00–4.00 | 60.5 | |
(b) PBI subscales | Reducing Social Impairments | 45 | 1.43 (0.37–2.67) | 0.00–4.00 | 60.0 |
Reducing Psychological Impairments | 45 | 2.00 (0.79–3.00) | 0.00–4.00 | 73.3 | |
Reducing Impairments due to Therapy | 39 | 1.00 (0.50–3.00) | 0.00–4.00 | 53.8 | |
Reducing Physical Impairments | 63 | 1.55 (0.63–3.00) | 0.00–4.00 | 71.4 | |
To have Confidence in Healing | 41 | 1.33 (1.00–3.00) | 0.00–4.00 | 82.9 |
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Schräder, N.H.B.; Korte, E.W.H.; Duipmans, J.C.; Stewart, R.E.; Bolling, M.C.; Wolff, A.P. Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index. J. Clin. Med. 2021, 10, 5836. https://doi.org/10.3390/jcm10245836
Schräder NHB, Korte EWH, Duipmans JC, Stewart RE, Bolling MC, Wolff AP. Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index. Journal of Clinical Medicine. 2021; 10(24):5836. https://doi.org/10.3390/jcm10245836
Chicago/Turabian StyleSchräder, Nicholas H. B., Eva W. H. Korte, José C. Duipmans, Roy E. Stewart, Maria C. Bolling, and André P. Wolff. 2021. "Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index" Journal of Clinical Medicine 10, no. 24: 5836. https://doi.org/10.3390/jcm10245836
APA StyleSchräder, N. H. B., Korte, E. W. H., Duipmans, J. C., Stewart, R. E., Bolling, M. C., & Wolff, A. P. (2021). Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index. Journal of Clinical Medicine, 10(24), 5836. https://doi.org/10.3390/jcm10245836