Evaluation of Quality of Life in Patients With Narcolepsy Treated With Sodium Oxybate: Use of the 36-Item Short-Form Health Survey in a Clinical Trial

Richard Bogan, Todd Swick, Mortimer Mamelak, Ruzica Kovacevic-Ristanovic, Chinglin Lai, Jed Black, Kathleen F Villa, Jacques Montplaisir, Richard Bogan, Todd Swick, Mortimer Mamelak, Ruzica Kovacevic-Ristanovic, Chinglin Lai, Jed Black, Kathleen F Villa, Jacques Montplaisir

Abstract

Introduction: The present post hoc analysis was designed to evaluate health-related quality of life (HRQoL) using the 36-item Short Form Health Status Survey (SF-36) during an 8-week trial of sodium oxybate (SXB).

Methods: SF-36 was assessed in a phase 3 placebo-controlled trial in patients with narcolepsy (N = 228) randomized to placebo or SXB in doses of 4.5, 6, or 9 g nightly for 8 weeks. Changes from baseline in SF-36 (last observation carried forward) were compared between SXB and placebo, and effect sizes (ES) were estimated.

Results: Baseline SF-36 values were lower than normative values for the US general population. After 8 weeks of treatment, mean (±standard deviation) improvement from baseline on the Physical Component Summary (PCS) was significantly greater with SXB 9 g/night than placebo (6.3 ± 9.1 vs. 1.5 ± 6.2; p = 0.005), with moderate ES; no significant difference was found between the SXB and placebo groups on the Mental Component Summary. SXB 9 g/night resulted in significantly (p < 0.05) greater improvements than placebo in Physical Functioning (4.4 ± 9.2 vs. 1.0 ± 8.0), General Health (GH; 3.1 ± 7.0 vs. 0.4 ± 6.8), and Social Functioning (6.8 ± 16.8 vs. 1.1 ± 9.6). All SXB doses resulted in significant improvement (p < 0.05) relative to placebo for Vitality, with moderate ES. No significant differences versus placebo were observed for Role-Physical, Role-Emotional, or Mental Health domains.

Conclusion: Treatment with SXB was associated with a dose-dependent improvement in HRQoL, with the 9-g nightly dose demonstrating a positive impact on PCS and individual SF-36 domains of Vitality, GH, and Physical and Social Functioning.

Trial registration: NCT00049803.

Funding: Jazz Pharmaceuticals.

Keywords: Narcolepsy; Quality of life; SF-36; Sodium oxybate.

Figures

Fig. 1
Fig. 1
Change from baseline at 8 weeks in SF-36 component summary scores. Data are for the intent-to-treat population using last observation carried forward; broken horizontal line represents the minimum clinically important difference (MCID)
Fig. 2
Fig. 2
Change from baseline at 8 weeks in SF-36 domain scores. Data are for the intent-to-treat population using last observation carried forward; broken horizontal line represents the minimum clinically important difference (MCID). PF Physical Functioning, RP Role–Physical, BP Bodily Pain, GH General Health, V Vitality, SF Social Functioning, RE Role–Emotional, MH Mental Health

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