Schwartz C E, Coulthard-Morris L, Cole B, Vollmer T
Frontier Science and Technology Research Foundation Inc., Chestnut Hill, Mass., USA.
Arch Neurol. 1997 Dec;54(12):1475-80. doi: 10.1001/archneur.1997.00550240029009.
A recombinant form of interferon beta-1b (Betaseron) was given Food and Drug Administration approval for use in the treatment of relapsing-remitting multiple sclerosis in 1993 based on a documented reduction in exacerbation rate. However, its effect on disease progression is less clear. It costs $11,000 per year and has documented adverse effects such as fatigue, feverlike symptoms, and depression.
To evaluate a recombinant form of interferon beta-1b in the treatment of relapsing-remitting multiple sclerosis and to discuss treatment trade-offs and comprehensive quality-of-life (QOL) outcomes.
We present a randomized evaluation of treatment with a recombinant form of interferon beta-1b in 79 patients with multiple sclerosis who participated in a random allocation lottery and were followed up for 12 months, during which data on QOL and clinical outcomes were collected. The data were analyzed using the Extended Quality-Adjusted Time Without Symptoms and Toxicity (Q-TWiST) method, which evaluates treatment trade-offs by incorporating several QOL domains and patient preferences regarding these domains.
Over the 12 months of follow-up, the case patients reported 10.6 months of quality-adjusted time, while the control patients reported 10.4 months of quality-adjusted time (P = .50).
Thus, the first year of treatment with interferon beta-1b did not significantly improve or detract from QOL. Results are discussed in terms of acceptable trade-offs depending on the nature of therapy. Future observational and clinical studies should incorporate measures of patient preference.
1993年，重组β-1b干扰素（倍泰龙）经美国食品药品监督管理局批准用于治疗复发缓解型多发性硬化症，依据是有记录显示其能降低疾病发作率。然而，其对疾病进展的影响尚不清楚。该药每年花费11,000美元，且有诸如疲劳、类似发热症状及抑郁等已记录在案的不良反应。
评估重组β-1b干扰素治疗复发缓解型多发性硬化症的效果，并讨论治疗的利弊权衡及综合生活质量（QOL）结果。
我们对79例参与随机分配抽签的多发性硬化症患者进行了重组β-1b干扰素治疗的随机评估，并对其进行了12个月的随访，在此期间收集了生活质量和临床结果的数据。使用扩展的无症状和无毒性质量调整时间（Q-TWiST）方法对数据进行分析，该方法通过纳入多个生活质量领域以及患者对这些领域的偏好来评估治疗的利弊权衡。
在12个月的随访期间，病例组患者报告的质量调整时间为10.6个月，而对照组患者报告的质量调整时间为10.4个月（P = 0.50）。
因此，使用β-1b干扰素治疗的第一年并未显著改善或损害生活质量。根据治疗的性质，就可接受的利弊权衡对结果进行了讨论。未来的观察性和临床研究应纳入患者偏好的测量指标。
