Effectiveness of recombinant human platelet-derived growth factor for the treatment of diabetic neuropathic foot ulcers
Effectiveness of recombinant human platelet-derived growth factor for the treatment of diabetic neuropathic foot ulcers
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DOI：
10.1111/j.1524-475x.2005.00074.x
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发表时间：
2005-11-01
影响因子：
2.9
通讯作者：
Berlin, JA
中科院分区：
文献类型：
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作者：
Margolis, DJ;Bartus, C;Berlin, JA
本研究的目的是评估重组人血小板源性生长因子（rhPDGF）治疗糖尿病神经性足溃疡（DNFU）的临床效果。先前发表的关键性试验表明，在治疗的第20周，随机接受rhPDGF的组比未接受rhPDGF的组愈合的溃疡多35%（即，相对危险度（RR）约为1.35）。这代表了在随机临床试验的严格对照条件下对rhPDGF疗效的估计。在一项回顾性队列研究中估计了标准临床实践下的治疗有效性，使用倾向评分控制治疗选择偏倚。我们注意到24，898名DNFU患者，其中9.6%接受了rhPDGF。我们成功地创建了一个倾向评分模型，该模型在接受rhPDGF的患者和未接受rhPDGF的患者之间平衡了许多伤口特征。我们创建了五个组，从最不可能接受rhPDGF的组到最可能接受rhPDGF的组。与标准治疗相比，rhPDGF治疗后愈合伤口接受rhPDGF治疗的RR（控制倾向）为1.32（1.22，1.38）。关于截肢，与未接受rhPDGF的患者相比，接受rhPDGF后截肢的RR为0.65（0.54，0.78）。在我们研究的限制范围内，rhPDGF在帮助伤口愈合和预防截肢方面比标准疗法更有效，其效果与先前发表的随机对照试验的疗效估计相似。
The goal of this study was to estimate the effectiveness in actual clinical practice of recombinant human platelet-derived growth factor (rhPDGF) for the treatment of diabetic neuropathic foot ulcer (DNFU). Previously published pivotal trials have shown that by the 20th week of care 35 percent more ulcers healed in the group randomized to receive rhPDGF than those who did not receive rhPDGF (i.e., a relative risk [RR] of about 1.35). This represents an estimate of the efficacy of rhPDGF under the tightly controlled conditions of randomized clinical trials. Treatment effectiveness under standard clinical practice was estimated in a retrospective cohort study, controlling for treatment selection bias using propensity scores. We noted 24,898 individuals with a DNFU, of whom 9.6 percent received rhPDGF. We successfully created a propensity score model that evenly balanced many wound characteristics between those who received rhPDGF and those who did not. We created five groups, which varied from those least likely to receive rhPDGF to those most likely to receive rhPDGF. The RR, controlling for the propensity, to receive rhPDGF for a healed wound after treatment with rhPDGF as compared with standard care was 1.32 (1.22, 1.38). With respect to amputation, the RR for undergoing amputation after receiving rhPDGF was 0.65 (0.54, 0.78) as compared with those who did not receive rhPDGF. Within the limitations of our study, rhPDGF is more effective than standard therapy in both helping a wound to heal and preventing amputation, and its effect is similar to the efficacy estimates from previously published randomized controlled trials.