Khanna Dinesh, Yan Xiaohong, Tashkin Donald P, Furst Daniel E, Elashoff Robert, Roth Michael D, Silver Richard, Strange Charlie, Bolster Marcy, Seibold James R, Riley David J, Hsu Vivien M, Varga John, Schraufnagel Dean E, Theodore Arthur, Simms Robert, Wise Robert, Wigley Fredrick, White Barbara, Steen Virginia, Read Charles, Mayes Maureen, Parsley Ed, Mubarak Kamal, Connolly M Kari, Golden Jeffrey, Olman Mitchell, Fessler Barri, Rothfield Naomi, Metersky Mark, Clements Philip J
University of Cincinnati, Cincinnati, Ohio 45267-0563, USA.
Arthritis Rheum. 2007 May;56(5):1676-84. doi: 10.1002/art.22580.
To assess the impact of cyclophosphamide (CYC) on the health-related quality of life (HRQOL) of patients with scleroderma after 12 months of treatment.
One hundred fifty-eight subjects participated in the Scleroderma Lung Study, with 79 each randomized to CYC and placebo arms. The study evaluated the results of 3 measures of health status: the Short Form 36 (SF-36), the Health Assessment Questionnaire (HAQ) disability index (DI), and Mahler's dyspnea index, and the results of 1 preference-based measure, the SF-6D. The differences in the HRQOL between the 2 groups at 12 months were calculated using a linear mixed model. Responsiveness was evaluated using the effect size. The proportion of subjects in each treatment group whose scores improved at least as much as or more than the minimum clinically important difference (MCID) in HRQOL measures was assessed.
After adjustment for baseline scores, differences in the HAQ DI, SF-36 role physical, general health, vitality, role emotional, mental health scales, and SF-36 mental component summary (MCS) score were statistically significant for CYC versus placebo (P < 0.05). Effect sizes were negligible (<0.20) for all of the scales of the SF-36, HAQ DI, and SF-6D at 12 months. In contrast, a higher proportion of patients who received CYC achieved the MCID compared with placebo in the HAQ DI score (30.9% versus 14.8%), transitional dyspnea index score (46.4% versus 12.7%), SF-36 MCS score (33.3% versus 18.5%), and SF-6D score (21.3% versus 3.8%).
One year of treatment with CYC leads to an improvement in HRQOL in patients with scleroderma lung disease.
评估环磷酰胺（CYC）治疗12个月后对硬皮病患者健康相关生活质量（HRQOL）的影响。
158名受试者参与了硬皮病肺部研究，其中79名随机分配至CYC组和安慰剂组。该研究评估了3项健康状况指标的结果：简短健康调查问卷（SF-36）、健康评估问卷（HAQ）残疾指数（DI）和马勒呼吸困难指数，以及1项基于偏好的指标SF-6D的结果。使用线性混合模型计算两组在12个月时HRQOL的差异。使用效应量评估反应性。评估每个治疗组中HRQOL指标得分改善至少达到或超过最小临床重要差异（MCID）的受试者比例。
在对基线分数进行调整后，CYC组与安慰剂组相比，HAQ DI、SF-36身体功能、总体健康、活力、情感职能、心理健康量表以及SF-36心理成分总结（MCS）得分的差异具有统计学意义（P<0.05）。在12个月时，SF-36、HAQ DI和SF-6D所有量表的效应量均可忽略不计（<0.20）。相比之下，与安慰剂组相比，接受CYC治疗的患者在HAQ DI得分（30.9%对14.8%）、过渡性呼吸困难指数得分（46.4%对12.7%）、SF-36 MCS得分（33.3%对18.5%）和SF-6D得分（21.3%对3.8%）方面达到MCID的比例更高。
CYC治疗一年可改善硬皮病肺病患者的HRQOL。
