ReviewLimitations of randomized clinical trials for evaluating emerging operations: the case of lung volume reduction surgery
Section snippets
The LVRS story
Pulmonary emphysema affects approximately 2 million adults in the United States and three to four times that number worldwide. Medical treatment offers only limited palliation and does not slow or halt progression of the disease. Of the many operations introduced during the past century to remedy the consequences of advanced emphysema, almost all failed to achieve the desired objectives and were abandoned [5]. One of the exceptions, lung transplantation, is of limited value because of the
Methods for evaluating new operations
At the time HCFA and the National Institutes of Health began to organize a clinical trial on LVRS, indications for the operation, the basics of operative technique, and details of perioperative management had been moderately well standardized. Operative mortality in different centers varied widely but most surgeons reported figures between 5% and 8%. Observational studies provided impressive evidence about the short-term effectiveness of the operation in a relatively well defined subset of
Choice of studies for advanced evaluation of LVRS
In May of 1996 HCFA and the National Institutes of Health announced joint sponsorship of the National Emphysema Treatment Trial (NETT) on LVRS. The study was designed to randomize 4,700 Medicare patients between LVRS and the best available medical treatment for 5 years at 18 clinical sites throughout the United States. Patient eligibility criteria for NETT resembles the characteristics of the population in which the short-term effectiveness of LVRS had already been established. The duration of
Progress of the clinical trials and utilization of LVRS
Fewer than one third of the anticipated number of patients have entered NETT and OBEST. As there appears to be no prospect for a change in the pace of enrollment in the foreseeable future, the recruitment phase of the two ongoing randomized trials may not be completed for another 6 to 10 years. NETT has already cut back its original enrollment goal from 4,700 to 2,500 patients [38]. OBEST has joined forces with the Canadian Lung Volume Reduction Trial in an attempt to obtain statistically
Comment
In spite of declining LVRS utilization, a steady stream of data from uncontrolled case series have continued to confirm the short and midterm effectiveness of the operation 42, 43. Two RCTs from the United States (37 and 49 patients), and one each from the United Kingdom (48 patients), Sweden (38 patients), and Italy (60 patients) compared LVRS with the best available medical treatment. All five studies reported better outcomes in the surgical arms 39, 44, 45, 46, 47, 48. Observational studies
Acknowledgements
The research component of OBEST is supported by the Thoracic Foundation, United States Surgical Corp and Biovascular Corp.
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Cited by (43)
An evidence-based estimate on the size of the potential patient pool for lung volume reduction surgery
2012, Annals of Thoracic SurgeryRandomized clinical trials for new surgical operations: Square peg in a round hole?
2010, Journal of Thoracic and Cardiovascular SurgeryLung volume reduction therapies for advanced emphysema: An update
2010, ChestCitation Excerpt :Cooper and colleagues2 concluded from their experience that favorable outcomes from LVRS require (1) selection of appropriate patients, and (2) multidisciplinary team approach by pulmonologists, thoracic surgeons, and other relevant specialists. Adherence to these standards resulted in reproducible symptomatic and physiologic improvements.3–5,9 The basics of inclusion and exclusion criteria proposed by Cooper et al2 and NETT have been widely adopted.
Evaluation and stages of surgical innovations
2009, The LancetSurgical research: act 3, answers
2009, The LancetProspective Multicenter Study of Surgical Correction of Pectus Excavatum: Design, Perioperative Complications, Pain, and Baseline Pulmonary Function Facilitated by Internet-Based Data Collection
2007, Journal of the American College of SurgeonsCitation Excerpt :Equipoise also did not exist with the surgeons or with the parents of patients, most of whom arrived for surgical consultation with strongly held opinions about the best procedure for correction of their child’s problem.17,18 Recently, calls for expertise-based randomized controlled trials recognize the limitations of surgeons in providing equivalent results with different procedures11,19 but do not address the strongly held opinions of patients and families. We believe that our approach and design allowed useful evaluation of a new surgical approach to pectus excavatum in a pragmatic way.