CLINICAL EVALUATIVE RESEARCH
The Center has examined the relationship between hospital volume and outcomes for several cardiovascular procedures (CABG, elective repairs of abdominal aortic aneurysms, and repair of cardiac congenital defects) in New York State between 1990 and 1995. We found that no such correlation exists in CABG surgery, where there is an active quality improvement program, but that significant inverse relationships exist both in abdominal aortic aneurysm surgery and congenital cardiac surgery, where no such programs have been implemented.
Heart failure is the largest medical DRG accounting for $35 billion in health care expenditures. The need for effective treatment options for advanced heart failure has stimulated the development of left ventricular assist devices (LVADs). LVADs assume the function of the damaged left ventricle, restoring normal hemodynamics and end-organ blood flow, and have been used in chronic advanced heart failure, to support patients awaiting cardiac transplantation. The FDA approved the Thoratec and Novacor devices for bridge-to-transplantation in 1998. The Center, in collaboration with the Division of Cardiothoracic surgery and the Division of Circulatory Physiology (Dept. of Medicine), has looked at the quality of life and economic implications of using left ventricular assist devices (LVADs) as a bridge to transplantation in patients with end-stage heart failure.
Favorable results in the bridge to transplant population encouraged the design of the multi-center REMATCH (randomized evaluation of mechanical assistance for the treatment of congestive heart failure) trial to evaluate the efficacy and safety of long-term LVAD support. InCHOIR has been the data and clinical coordinating center for this NIH-supported trial. Meeting its primary endpoint in 2001, the study randomized 129 patients to either the Thoratec Heartmate LVAD (n=68) or optimal medical management (n=61). Despite the limitation of an external driveline, LVAD implantation halved the one-year mortality as compared to medical management (from 75 to 51%). Moreover, these patients had a significant improvement in quality of life and functional status compared to their medical counterparts. On the basis of these results, the FDA approved the Heartmate LVAD for long-term use in the United States in November 2002. Although enrollment is complete, the trial is still ongoing, with data collection for all the major endpoints.
Acute Cardiogenic Shock -- We are extending our current work on left ventricular assist devices (LVAD) in the treatment of chronic heart failure by investigating the use of these devices for hemodynamic support of patients with cardiogenic shock, developed acutely in the setting of myocardial infarction and cardiomyotomy. The proposed investigation would be a multi-center, randomized controlled trial that would be coordinated at Columbia University, utilize the REMATCH network of investigators and derive support from the National Heart Lung and Blood Institute.
ARROW LionHeart Destination Therapy Trial -- Arrow International has developed a totally implantable LVAD, which may circumvent some of the problems associated with an external driveline, such as infections. This device has been evaluated in Europe, and we are conducting the statistical analyses. A feasibility study is now ongoing in the U.S., which we will analyze as well. InCHOIR will be the data and clinical coordinating center for the pivotal trial, expected to start in the fall of 2003.
MicroMed Axial Flow Pump -- This miniaturized device, which is based on NASA technology, is implanted in the chest only and does not require a reservoir to contain a volume of blood. This is because the blood is moving at high speed, continuously, and not in a pulsatile fashion, as is the case with a native heart. We will be designing, conducting and analyzing a trial testing the use of this pump in patients awaiting cardiac transplantation.
CABG in the elderly – The Center has documented the effectiveness and cost-effectiveness of CABG surgery in patients 80 years old and older. In collaboration with the NYPH Office of Outcomes Research, we demonstrated that elderly patients who underwent CABG surgery at CPMC had better survival and better quality of life than similar patients treated medically. CABG was also shown to be cost effective ($10,424 per quality-adjusted life year saved). This is an important finding because increasing numbers of elderly patients are undergoing CABG surgery and earlier clinical trials conducted of CABG excluded persons over 65.
Heart Failure Management In Women -- In collaboration with the Office of Outcomes Research of NYPH, we are launching a major study comparing the treatment outcomes of heart failure in women and men. This study will enroll 1,300 patients hospitalized for heart failure at the two campuses of NYPH. These patients will be followed longitudinally for quality of life, hospitalization frequency, costs of care, and survival.
Off-Pump Coronary Artery Bypass Surgery – A recent innovation in the technique of coronary artery surgery is to bypass stenotic vessels on a heart that is still beating, rather than first arresting the heart and putting the patient on a heart-lung bypass machine. The theoretical advantages to performing surgery in this manner include shorter operative times, less bleeding, and, most importantly, avoidance of the potential neurovascular complications from cardio-pulmonary bypass. In collaboration with cardiothoracic surgeons at New York-Presbyterian Hospital (Columbia Presbyterian Center) and St. Michael’s Hospital, the Center is initiating a multi-center randomized controlled trial comparing "off pump" to "on pump" coronary bypass surgery in first time recipients. Study endpoints will include cognitive function, stroke, coronary patency, quality of life, and cost-effectiveness, in addition to all-cause and cardiovascular mortality. The Center is seeking funding from the National Heart Lung and Blood Institute of the National Institutes of Health.
The treatment of vascular disease, like many other areas in surgery, is being transformed by new technologies that give endovascular access to vessels. These less invasive approaches have reopened the issues of whether routine screening would be a cost-effective way to spend health care resources.
Open versus Endovascular Abdominal Aortic Aneurysm (AAA) -- The Center has designed a prospective cohort study to be conducted at NYPH to analyze the mortality, adverse events, quality of life, cost and sexual function associated with the repair of AAAs.
Epidemiological Analyses of Vascular Disease --We are currently conducting a twenty-year national retrospective study of the treatment of vascular disease, using the National Discharge Database. This study looks at the evolution of catheter and endovascular based approaches to a variety of vascular diseases, specifically identifying differential use in several groups of patients, impact on hospital utilization, and evidence of a volume outcome relationship.
Cost-Effectiveness of Screening for Vascular Disease --Using case finding data from a recent nationwide screening effort, we are putting together a cost-effectiveness model to assess the long-term cost effectiveness of one-time ultrasound screening for carotid, peripheral vascular and aortic disease.
National Emphysema Treatment Trial (NETT) -- InCHOIR faculty collaborated with the Division of Thoracic Surgery and the Division of Pulmonary Medicine to successfully compete for an investigative position in the NHLBI sponsored National Emphysema Treatment Trial. Columbia University is the only site in the Mid-Atlantic States and is one of 17 in the U.S. The trial is a randomized comparison of lung volume reduction surgery and maximal medical therapy in the treatment of patients with end-stage emphysema. The trial is one of the largest randomized surgical trials conducted in this country with nearly 2600 patients to be randomized. Moreover, it is a landmark with respect to its financial structure: the NIH and HCFA will support trial administration and patient care, respectively. The comparative endpoints will include survival, quality of life, pulmonary function, and cost-effectiveness. Dr. Moskowitz represents Columbia on the Cost-effectiveness Steering Committee.
Abdominal Aortic Aneurysm Treatment -- The faculty of InCHOIR are collaborating with members of the division of vascular surgery at Presbyterian Hospital, Mt Sinai Medical Center, and UCSF to study outcomes of two alternative treatments for abdominal aortic aneurysms: the traditional open repair and endovascular stenting. Through prospective and retrospective data collection efforts, the Center is comparing the effectiveness, cost, and quality of life (particularly sexual dysfunction) associated with each approach. In addition, the Center is reexamining the question of whether to routinely screen for the presence of aneurysms, using ultrasound, in high-risk populations – a practice currently not subscribed to by general medical societies (e.g. the American College of Physician) and third party payers in this country.
Obesity is well recognized as a major cause of morbidity, which has reached epidemic proportions in this country. In patients with morbid obesity, surgical measures to restrict food intake and decrease intestinal absorption have been the only approaches with demonstrable success. We designed a pilot randomized trial comparing laparoscopic adjustable gastric banding to laparoscopic gastric bypass. This trial has been funded, by the Office of Clinical Trials, and by the Minimal Access Surgery Center.
In conjunction with the Department of Pediatrics and the Office of Outcomes Research of NYPH, the Center is developing a comprehensive outcomes program in pediatrics. Within pediatrics, the initial focus is on (1) orthopedic disorders, (2) cardiac disease, and (3) otologic disease.
Efforts to measure quality of life in children have lagged behind such efforts for adults. Investigators at InCHOIR have been involved in pediatric quality of life instrument validation in the area of musculoskeletal diseases. We are currently one of 5 pediatric trauma centers participating in a Web-based trauma registry measuring the morbidity associated with childhood trauma, as assessed by quality of life instruments. In addition, we are conducting studies of quality of life among children with scoliosis and cerebral Palsy and have collected data on over 1500 patients.
Orthopedic Disorders -- Recently, there has been a growing focus on pediatric trials both at the NIH and within industry. The Division of Pediatric Orthopedic Surgery and InCHOIR have developed a research program in pediatric orthopedic outcomes research. In a retrospective cohort study, for example, we documented the effectiveness of pre-operative erythropoeitin in averting perioperative blood transfusions. In light of the fact that autologous donation is an expensive way of obtaining survival benefits, we are currently examining the cost-effectiveness of using erythropoeitin as a strategy to avert transfusion-related illnesses, such as hepatitis C.
Cardiac disease -- We recently presented the first quality of life study of children with hypoplastic heart syndrome to surgeons at the annual meeting of the American Association of Thoracic and Cardiovascular Surgeons. Our work demonstrated that longer circulatory arrest times not only had a detrimental effect on developmental status but also caused a significant reduction in the quality of life of patients. In collaboration with the Pediatric Cardiac Surgery Program, Division of Pediatric Cardiology, and The NYPH Office of Outcomes Research, we are launching a major study of the outcomes of pediatric cardiac surgery and interventional cardiology. This study will assess the clinical effectiveness, cost-effectiveness and quality-of-life of specific treatments for major cardiac disease in children.
Otologic Disease – The NYPH Office of Outcomes Research is collaborating with the Division of Pediatric Otolaryngology to study the role of tonsillectomies in sleep disorders of children in a multi-center prospective outcomes study.
Ductal carcinoma in situ (DCIS) -- DCIS of the breast is a diagnosis that has exhibited exponential growth over the last decade, largely due to improved performance and use of diagnostic imaging. This growth in diagnostic capabilities has greatly outpaced the accrual of therapeutic knowledge. Thus, information concerning the long-term outcomes of alternative treatments for this disease is limited. What does exist suggests that the difference in outcomes between aggressive (mastectomy) and limited (lumpectomy) is small. Despite this, national treatment patterns suggest a predominance of mastectomy, which would seem to be at odds with overall concerns about breast preservation, survival and the low risk of developing invasive cancer, faced by women in this situation. The Center has developed a project, which examines the risk preferences of both patients and their physicians to see how these attitudes affect therapeutic decision-making, which has been submitted to National Cancer Institute (NCI) of the National Institutes of Health (NIH).
Breast Cancer -- Although breast cancer incidence rates have declined in the US in the past decade, breast cancer mortality rates have actually increased over the past 10 years among African-Americans and other minority American women. Tamoxifen, used as a prophylactic treatment, has been shown to reduce the incidence of invasive breast cancer by nearly 50% in select groups of women in clinical trials. InCHOIR faculty, in collaboration with the Irving Comprehensive Cancer Center, are developing a decision analysis model that will determine the relative benefits (in terms of survival, quality adjusted survival and cost-effectiveness) of treating women at high of developing breast cancer with prophylactic tamoxifen. The Center is seeking support from the NCI for this project.
Oral cancer -- The Center, in collaboration with the American Dental Association, is submitting a 10-year grant to NCI to design and evaluate an educational curriculum to prevent oropharyngeal cancer by modifying risk behaviors and to improve the rate of early detection. Approximately 30,000 new patients with oral cancer are diagnosed annually in the United States, with 25% fatality. This educational intervention will be directed toward dental students and practicing dentists as part of a multi-school randomized clinical trial. The Center, in conjunction with The School of Dental and Oral Surgery of Columbia University, has begun a pilot study to examine the effectiveness of training non-dental health care workers to screen for oral cancer. The pilot study will target the residents of a nursing home, a population at high risk this disease. The results of this pilot study will be used to design a large scale, randomized clinical trial.
ARTHRITIS OF THE SHOULDER --Along with the aging of the population has come an increase in large joint arthritis requiring joint replacement to reestablish a more functional life style. Investigators at Columbia’s Department of Orthopedics have developed the next generation of shoulder prosthesis and InCHOIR is conducting a multi-center trial to evaluate its efficacy.
Shoulder Surgery -- Large data-set analysis is a powerful tool to identify areas that need further clinical evaluation. Our research, for example, documented striking variations among states within the United States in the rates of shoulder procedures, which could not be easily explained by differences in disease prevalence or the density of orthopedists and shoulder surgeons. This analysis showed the need for well- designed clinical research to further define the factors that account for these variations and to examine the effectiveness and appropriate indications for these procedures, including rotator cuff tears.
In follow-up of this research, the Orthopedic Research and Education Foundation awarded a two-year grant to the Shoulder Service of the Department of Orthopedics and InCHOIR to examine the outcomes of repair of the partially torn rotator cuff. We recently completed data collection on a prospective cohort study of patients who received rotator-cuff repair at NYP hospital during the last two years, and are in the midst of analyzing the data. In addition, we are designing a multi-center clinical trial for a new shoulder prosthesis, developed at Columbia Presbyterian Medical Center.
Myasthenia Gravis -- We recently completed guidelines for conducting outcome analysis in the field of myesthenia gravis. These guidelines will appear in a task force paper of the Myasthenia Gravis Foundation of America and is being submitted to Neurology for review.
Planning Grant for a Multipurpose Clinical Research Center -- In collaboration with Dr. John Bilzekian, we have received an NIH grant to plan a Multipurpose Clinical Research Center (MCRC) for studies of arthritis and musculo-skeletal and skin diseases. This Center would bring together clinical researchers in arthritis, skin, and musculoskeletal disease at Columbia’s Health Sciences Campus with outcomes research experts at InCHOIR.
Pneumonia continues to be a major public health problem, partly because of the under-utilization of the pneumococcal vaccine. This maybe related to concerns that the vaccine may not be effective in preventing pneumonia in the elderly; recent research, however, found that it does prevent the consequences of pneumonia (i.e., sepsis). Our work shows that, even if sepsis prevention is the only benefit of the vaccine, its application in the elderly is cost-effective and, in fact, is one of the very few technologies that are cost saving.