溶组织梭状芽孢杆菌胶原酶治疗掌腱膜挛缩症结节的疗效与安全性：一项随机对照试验
Efficacy and safety of collagenase clostridium histolyticum for Dupuytren disease nodules: a randomized controlled trial.
作者信息
Costas Bronier, Coleman Stephen, Kaufman Greg, James Robert, Cohen Brian, Gaston R Glenn
机构信息
The Hand and Upper Extremity Center of Georgia, 980 Johnson Ferry Rd, NE, Suite 1020, Atlanta, GA, 30342, USA.
Brisbane Hand and Upper Limb Clinic, 259 Wickham Terrace, Spring Hill, Queensland, 4000, Australia.
出版信息
BMC Musculoskelet Disord. 2017 Aug 30;18(1):374. doi: 10.1186/s12891-017-1713-z.
BACKGROUND
To determine the safety and efficacy of collagenase clostridium histolyticum (CCH) injection for the treatment of palmar Dupuytren disease nodules.
METHODS
In this 8-week, double-blind trial, palpable palmar nodules on one hand of adults with Dupuytren disease were selected for treatment. Patients were randomly assigned using an interactive web response system to receive a dose of 0.25 mg, 0.40 mg, or 0.60 mg (1:1:1 ratio) and then allocated to active treatment (CCH) or placebo (4:1 ratio). All patients and investigators were blinded to treatment. One injection was made in the selected nodule on Day 1. Caliper measurements of nodule length and width were performed at screening and at Weeks 4 and 8. Investigator-reported nodular consistency and hardness were evaluated at baseline and Weeks 1, 4, and 8. Investigator-rated patient improvement (1 [very much improved] to 7 [very much worse]) and patient satisfaction were assessed at study end.
RESULTS
In the efficacy population (n = 74), percentage changes in area were significantly greater with CCH 0.40 mg (-80.1%, P = 0.0002) and CCH 0.60 mg (-78.2%, P = 0.0003), but not CCH 0.25 mg (-58.3%, P = 0.079), versus placebo (-42.2%) at post-treatment Week 8. Mean change in nodular consistency and hardness were significantly improved with CCH versus placebo at Weeks 4 and 8 (P ≤ 0.0139 for all). At Week 8, investigator global assessment of improvement was significantly greater with CCH 0.40 mg and 0.60 mg (P ≤ 0.0014) but not statistically significant with CCH 0.25 mg versus placebo (P = 0.13). Most patients were "very satisfied" or "quite satisfied" with CCH 0.40 mg and 0.60 mg. Contusion/bruising (50.0% to 59.1%) was the most common adverse event with CCH treatment.
CONCLUSION
In patients with Dupuytren disease, a single CCH injection significantly improved palmar nodule size and hardness. The safety of CCH was similar to that observed previously in patients with Dupuytren contracture.
TRIAL REGISTRATION
ClinicalTrials.gov identifier: NCT02193828 . Date of trial registration: July 2, 2014 to December 5, 2014.
背景
确定溶组织梭状芽孢杆菌胶原酶（CCH）注射治疗掌部杜普伊特伦病结节的安全性和有效性。
方法
在这项为期8周的双盲试验中，选择患有杜普伊特伦病的成年人一只手上可触及的掌部结节进行治疗。使用交互式网络响应系统将患者随机分配，以接受0.25毫克、0.40毫克或0.60毫克的剂量（1:1:1比例），然后分配至活性治疗组（CCH）或安慰剂组（4:1比例）。所有患者和研究人员均对治疗不知情。在第1天对选定的结节进行一次注射。在筛查时以及第4周和第8周对结节的长度和宽度进行卡尺测量。在基线以及第1、4和8周评估研究人员报告的结节质地和硬度。在研究结束时评估研究人员评定的患者改善情况（1[改善非常明显]至7[恶化非常明显]）和患者满意度。
结果
在疗效人群（n = 74）中，与安慰剂组（-42.2%）相比，治疗后第8周时，0.40毫克CCH组（-80.1%，P = 0.0002）和0.60毫克CCH组（-78.2%，P = 0.0003）的面积百分比变化显著更大，但0.25毫克CCH组（-58.3%，P = 0.079）并非如此。在第4周和第8周时，与安慰剂相比，CCH组的结节质地和硬度的平均变化有显著改善（所有P≤0.0139）。在第8周时，0.40毫克和0.60毫克CCH组的研究人员总体改善评估显著更高（P≤0.0014），但0.25毫克CCH组与安慰剂组相比无统计学意义（P = 0.13）。大多数患者对0.40毫克和0.60毫克CCH“非常满意”或“比较满意”。挫伤/瘀斑（50.0%至59.1%）是CCH治疗最常见的不良事件。
结论
在患有杜普伊特伦病的患者中，单次CCH注射可显著改善掌部结节大小和硬度。CCH的安全性与先前在患有杜普伊特伦挛缩的患者中观察到的相似。
试验注册
ClinicalTrials.gov标识符：NCT02193828。试验注册日期：2014年7月2日至2014年12月5日。
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