From: Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease
All | Untreated group | Received GD-specific therapy | p value** | |
---|---|---|---|---|
1. Dependent on others because of your Gaucher disease? | 1.31 (1.2–1.4) | 1.09 (0.9–1.28) | 1.45 (1.23–1.68) | 0.001 |
2. Visibly big or swollen abdomen because of your Gaucher disease? | 2.05 (1.8–2.3) | 1.70 (1.29–2.12) | 2.18 (1.85–2.52) | 0.06 |
3. Fatigued because of your Gaucher disease? | 2.88 (2.5–3.2) | 1.69 (1.37–2.00) | 3.59 (3.09–4.08) | < 0.001 |
4. Physically weak because of your Gaucher disease? | 2.73 (2.4–3.1) | 1.70 (1.38–2.03) | 3.39 (2.95–3.83) | < 0.001 |
5. Severity of bone pain because of your Gaucher disease? | 2.40 (2.1–2.7) | 1.57 (1.26–1.89) | 2.88 (2.41–3.36) | < 0.001 |
6. Depressed because of your Gaucher disease? | 1.62 (1.4–1.8) | 1.35 (1.14–1.56) | 1.80 (1.48–2.12) | 0.18 |
7. Worried because of your Gaucher disease? | 2.01 (1.7–2.3) | 1.39 (1.17–1.60) | 2.39 (1.99–2.78) | 0.001 |
8. How have you felt about your future with Gaucher disease? | 2.47 (2.2–2.8) | 1.57 (1.28–1.87) | 2.98 (2.55–3.41) | < 0.001 |