Peimer Clayton A, Skodny Paul, Mackowiak John I
Department of Surgery, College of Human Medicine, Michigan State University, Lansing; Marquette General Healthcare, Marquette, MI; Health Economics & Outcomes Research, Auxilium Pharmaceuticals, Inc., Malvern, PA; and Center for Outcomes Research, Nashville, TN.
J Hand Surg Am. 2013 Dec;38(12):2370-6. doi: 10.1016/j.jhsa.2013.08.114. Epub 2013 Oct 17.
To collect data on the real-world effectiveness of collagenase clostridium histolyticum (CCH) during its first year of use following U.S. Food and Drug Administration approval and compare those results with clinical trial efficacy data.
This retrospective chart review was conducted at 10 U.S. community and academic practice sites with major experience using CCH. Charts of patients treated with CCH between February and December 2010 were abstracted, and anonymized data were analyzed. Clinical use, including number of injections per cord and effectiveness outcomes (joint contracture and range of motion) were compared with results from 2 registration trials.
Data were collected from 501 patients (74% male; 48% employed; mean [SD] age, 65 [10] y); 463 patients had sufficient data for analysis. We found that 1.08 CCH injections were used per treated joint, compared with a mean of 1.7 injections in registration trials. Ninety-three percent of joints received only 1 injection. The mean (SD) number of visits per injection was 2.92 (1.0). Mean (SD) contracture was reduced by 75% from 49° (21) at baseline to 12° (17), similar to the 71% to 79% reduction in clinical trials. Mean (SD) range of motion was improved by 37° from 44° (20) at baseline to 81° (14), similar to the increase of 35° and 37° in the 2 clinical trials; and 67% of first injections resulted in full correction to 0° to 5°, compared with the clinical trial rate of 39%.
Despite a lower injection rate, correction of joint contracture and range of motion was similar to findings from clinical trials. Effectiveness reports using this kind of surveillance design could provide patients, physicians, and payers with the information needed to make better treatment and reimbursement decisions.
TYPE OF STUDY/LEVEL OF EVIDENCE: Therapeutic III.
收集溶组织梭状芽孢杆菌胶原酶（CCH）在美国食品药品监督管理局批准后的第一年实际应用效果的数据，并将这些结果与临床试验疗效数据进行比较。
这项回顾性图表审查在美国10个具有使用CCH丰富经验的社区和学术医疗机构进行。对2010年2月至12月期间接受CCH治疗的患者图表进行摘要提取，并对匿名数据进行分析。将临床应用情况，包括每条肌腱的注射次数和疗效结果（关节挛缩和活动范围）与两项注册试验的结果进行比较。
收集了501例患者的数据（74%为男性；48%有工作；平均[标准差]年龄为65[10]岁）；463例患者有足够的数据用于分析。我们发现，每个接受治疗的关节使用1.08次CCH注射，而注册试验中的平均注射次数为1.7次。93%的关节仅接受1次注射。每次注射的平均（标准差）就诊次数为2.92（1.0）。平均（标准差）挛缩从基线时的49°（21）降至12°（17），减少了75%，与临床试验中71%至79%的减少幅度相似。平均（标准差）活动范围从基线时的44°（20）增加了37°至81°（14），与两项临床试验中35°和37°的增加幅度相似；67%的首次注射导致完全矫正至0°至5°，而临床试验率为39%。
尽管注射率较低，但关节挛缩的矫正和活动范围与临床试验结果相似。使用这种监测设计的有效性报告可为患者、医生和支付方提供做出更好的治疗和报销决策所需的信息。
研究类型/证据水平：治疗性III级。
