Registry-based trials: a potential model for cost savings?

BACKGROUND/AIMS: Registry-based trials have emerged as a potentially cost-saving study methodology. Early estimates of cost savings, however, conflated the benefits associated with registry utilisation and those associated with other aspects of pragmatic trial designs, which might not all be as broadly applicable. In this study, we sought to build a practical tool that investigators could use across disciplines to estimate the ranges of potential cost differences associated with implementing registry-based trials versus standard clinical trials. METHODS: We built simulation Markov models to compare unique costs associated with data acquisition, cleaning, and linkage under a registry-based trial design versus a standard clinical trial. We conducted one-way, two-way, and probabilistic sensitivity analyses, varying study characteristics over broad ranges, to determine thresholds at which investigators might optimally select each trial design. RESULTS: Registry-based trials were more cost effective than standard clinical trials 98.6% of the time. Data-related cost savings ranged from $4300 to $600,000 with variation in study characteristics. Cost differences were most reactive to the number of patients in a study, the number of data elements per patient available in a registry, and the speed with which research coordinators could manually abstract data. Registry incorporation resulted in cost savings when as few as 3768 independent data elements were available and when manual data abstraction took as little as 3.4 seconds per data field. CONCLUSIONS: Registries offer important resources for investigators. When available, their broad incorporation may help the scientific community reduce the costs of clinical investigation. We offer here a practical tool for investigators to assess potential costs savings.

Anterior vertebral body tethering for immature adolescent idiopathic scoliosis: one-year results on the first 32 patients.

PURPOSE: This retrospective chart review evaluates the clinical and radiographic outcomes of anterior vertebral body tethering (VBT) at 1-year follow-up. Anterior VBT offers a fusionless treatment option for skeletally immature patients with adolescent idiopathic scoliosis. It is a growth-modulation technique, which utilizes patients' growth to attain progressive scoliosis correction. Numerous animal models support its promise; however, clinical data remain sparse. METHODS: Clinical and radiographic data were retrospectively analyzed. We reviewed 32 patients who underwent thoracic VBT with a minimum one-year follow-up. Pertinent clinical and radiographic data were collected. ANOVA, Student's t test and Fisher's exact test were utilized to compare different time points. RESULTS: 32 patients with thoracic idiopathic scoliosis (72 % female) with a minimum one-year follow-up were identified; mean age at surgery was 12 years. All patients were considered skeletally immature pre-operatively; mean Risser score 0.42, mean Sanders score 3.2. Patients underwent tethering of an average of 7.7 levels (range 7-11). Median blood loss was 100 cc. The mean pre-operative thoracic curve magnitude was 42.8° ± 8.0° which corrected to 21.0° ± 8.5° on first erect and 17.9° ± 11.4° at most recent. The pre-operative lumbar curve of 25.2° ± 7.3° demonstrated progressive correction (first erect = 18.0° ± 7.1°, 1 year = 12.6° ± 9.4°, p < 0.00001). Thoracic axial rotation measured 13.4° pre-operatively and 7.4° at the most recent measurement (p < 0.00001). One patient experienced prolonged atelectasis which required a bronchoscopy; otherwise, no major complications were observed. CONCLUSIONS: Our early results indicate that anterior VBT is a safe and potentially effective treatment option for skeletally immature patients with idiopathic scoliosis. These patients experienced an improvement of their scoliosis with minimal major complications. However, longer term follow-up of this cohort will reveal the true benefits of this promising technique. LEVEL OF EVIDENCE: IV.

Rapid Growth of Left Atrial Thrombus in a Pediatric Heart Transplant Recipient.

We report on a 10-year-old female patient who rapidly developed a left atrial (LA) mass 2 months after orthotopic heart transplant. Nine days prior to detection of the mass, she received high-dose corticosteroids for acute cellular rejection (grade 2). Despite negative echocardiogram 5 days prior to detection, a large echogenic mass was noted in the LA (18 x 12 x 24 mm); it was surgically resected after unsuccessful anticoagulation treatment. Pathogenesis of this LA thrombus remains uncertain, but immunosuppression, acute rejection, and high-dose steroid therapy may have contributed. Surgical thrombectomy is a safe and effective treatment option for LA thrombus.

Tailored strategy to match anatomy and physiology with intervention can improve outcomes of symptomatic neonates with Ebstein anomaly.

Objective: Neonatal presentation of Ebstein anomaly (EA) represents the most severe form of this condition. Despite significant advances, operative mortality remains high and management decisions represent a formidable challenge. We used a strategy aimed to match anatomy and physiology with type and time of intervention to increase survival. Methods: We performed a review of all patients with fetal or neonatal diagnosis of EA managed at a single center between 2007 and 2020. Results: Among 18 patients with EA, 8 underwent neonatal intervention. The most common indication included cyanosis and heart failure (8/8), end organ dysfunction (6/8), and maldistribution of cardiac output (6/8). Only 2/8 had antegrade pulmonary blood flow. Associated conditions included pulmonary regurgitation in 4/8, atrial tachyarrhythmia in 4, and a ventricular septal defect in 3. Three patients underwent initial stabilization with main pulmonary artery occlusion including bilateral pulmonary artery banding in 2. Five patients underwent biventricular repair with conversion to right ventricle exclusion in 2 cases. Three others underwent the Starnes procedure as initially planned. The median age at surgery was 10 days (range, 1-30) and median weight 2.6 kg (range, 1.9-4.0). The median duration of mechanical ventilation and intensive care unit stay were 9 days (range, 5-34) and 30 days (range, 15-100), respectively. Operative mortality was 1/8. At a median follow-up of 130 months (range, 5-146), there were no late deaths, and all survivors remain in functional class I and free of valvular reintervention. Conclusions: Symptomatic neonates with EA can be effectively managed with good outcomes. Preoperative stabilization and choice of management pathway on the basis of anatomy and physiology can help reduce morbidity and mortality.

An Approach to Diversity and Inclusion in Cardiothoracic Surgery.

EXECUTIVE SUMMARY: While the United States (US) population at large is rapidly diversifying, cardiothoracic surgery is among the least diverse specialties in terms of racial and gender diversity. Lack of diversity is detrimental to patient care, physician well-being, and the relevance of cardiothoracic surgery on our nation's health. Recent events, including the coronavirus disease 2019 pandemic and the Black Lives Matter protests, have further accentuated the gross inequities that underrepresented minorities face in our country and have reignited conversations on how to address bias and systemic racism within our institutions. The field of cardiothoracic surgery has a responsibility to adopt a culture of diversity and inclusion. This kind of systemic change is daunting and overwhelming. With bias ubiquitously entangled with everyday experiences, it can be difficult to know where to start. The Society of Thoracic Surgeons Workforce on Diversity and Inclusion presents this approach for addressing diversity and inclusion in cardiothoracic surgery. This framework was adapted from a model developed by the National Institute on Minority Health and Health Disparities and includes information and recommendations generated from our literature review on diversity and inclusion. A MEDLINE search was conducted using keywords "diversity," "inclusion," and "surgery," and approaches to diversity and inclusion were drawn from publications in medicine as well as non-healthcare fields. Recommendations were generated and approved by The Society of Thoracic Surgeons Executive Committee. We present an overarching framework that conceptualizes diversity and inclusion efforts in a series of concentric spheres of influence, from the global environment to the cardiothoracic community, institution, and the individual surgeon. This framework organizes the approach to diversity and inclusion, grouping interventions by level while maintaining a broader perspective of how each sphere is interconnected. We include the following key recommendations within the spheres of influence: It is important to note that each of the spheres of influence is interconnected. Interventions to improve diversity must be coordinated across spheres for concerted change. Altogether, this multilevel framework (global environment, cardiothoracic community, institution, and individual) offers an organized approach for cardiothoracic surgery to assess, improve, and sustain progress in diversity and inclusion.

Prospective Follow-up Report on Anterior Vertebral Body Tethering for Idiopathic Scoliosis: Interim Results from an FDA IDE Study.

BACKGROUND: Anterior vertebral body tethering (aVBT) has emerged as a novel treatment option for patients with idiopathic scoliosis. We present the results from the first U.S. Food and Drug Administration (FDA) Investigational Device Exemption (IDE) study on aVBT. METHODS: In this prospective review of a retrospective data set, eligible patients underwent aVBT at a single center from August 2011 to July 2015. Inclusion criteria included skeletally immature patients with Lenke type-1A or 1B curves between 30° and 65°. Clinical and radiographic parameters were collected, with the latter measured by an independent reviewer. RESULTS: Fifty-seven patients (49 girls and 8 boys), with a mean age (and standard deviation) of 12.4 ± 1.3 years (range, 10.1 to 15.0 years), were enrolled in the study. The patients had a mean of 7.5 ± 0.6 levels tethered, the mean operative time was 223 ± 79 minutes, and the mean estimated blood loss was 106 ± 86 mL. The patients were followed for an average of 55.2 ± 12.5 months and had a mean Risser grade of 4.2 ± 0.9 at the time of the latest follow-up. The main thoracic Cobb angle was a mean of 40.4° ± 6.8° preoperatively and was corrected to 18.7° ± 13.4° at the most recent follow-up. In the sagittal plane, T5-T12 kyphosis measured 15.5° ± 10.0° preoperatively, 17.0° ± 10.1° postoperatively, and 19.6° ± 12.7° at the most recent follow-up. Eighty percent of patients had curves of <30° at the most recent follow-up. The most recent Scoliosis Research Society (SRS) scores averaged 4.5 ± 0.4, and scores on the self-image questionnaire averaged 4.4 ± 0.7. No major neurologic or pulmonary complications occurred. Seven (12.3%) of 57 patients had a revision: 5 were done for overcorrection and 2, for adding-on. CONCLUSIONS: Anterior VBT is a promising technique that has emerged as a treatment option for patients with immature idiopathic scoliosis. We present the results from the first FDA-approved IDE study on aVBT, which formed the basis for the eventual Humanitarian Device Exemption approval. The findings affirm the safety and efficacy of this technique and suggest opportunities for improvement, particularly with respect to reoperation rates. LEVEL OF EVIDENCE: Therapeutic Level IV. See Instructions for Authors for a complete description of levels of evidence.

An Exploration of Myths, Barriers, and Strategies for Improving Diversity Among STS Members.

Diversity within health care organizations has many proven benefits, yet women and other groups remain underrepresented in cardiothoracic surgery. We sought to explore responses from a Society of Thoracic Surgeons (STS) survey to identify myths and barriers for informing organizational strategies in the STS and cardiothoracic surgery. We performed a qualitative review of narrative survey responses within three domains surrounding diversity in cardiothoracic surgery: myths, barriers, and strategies for improvement. Common diversity myths included diversity as a pipeline problem (24%), diversity equated to exclusivity (21%), and diversity not supporting meritocracy (18%). The most frequent barrier code was perceived prejudice (22%). Suggested strategies toward improvement were culture change prioritizing diversity (22%) and training the leaders (14%). Notably, 15% of response codes reflected the belief that disparities do not exist; thus, the issue should not be prioritized by the organization. The results do not necessarily reflect the beliefs of most of the STS membership; nonetheless, they provide important insight critical to guide any efforts toward eliminating disparities within cardiothoracic surgery and improving the care of our patients.

Protocols associated with no mortality in 100 consecutive Fontan procedures.

OBJECTIVES: Results of Fontan's procedure have improved considerably, but perioperative mortality still occurs, attributed to ventricular dysfunction, stroke, arrhythmia, thromboembolism, and multi-organ dysfunction. Our protocols of operative and intensive care unit management address these potential issues, and have been associated with zero mortality, even with many high-risk candidates. METHODS: From 1996 to 2006, all Fontan patients were managed as follows: operative strategy based on aortic and single atrial cannulation, cooling on full-flow bypass, and hypothermic circulatory arrest to create the Fontan pathway. No direct caval cannulation. Use of central venous lines was completely avoided. Fresh whole blood was used for pump prime and for volume restoration. Inotropic and vasodilator therapy was continued for at least 48 h. Aspirin was used exclusively as anti-thrombotic therapy. Postoperative pleural drainage was accomplished with small pigtail catheters. The usual Fontan pathway was by lateral atrial tunnel (84), with extra-cardiac conduit when dictated by anatomy (16). RESULTS: One hundred Fontan operations were performed with no mortality. All patients were extubated by postoperative day 1. Hospital stay was 10+/-5 days. Complications were: bleeding (1), reintubation (1), emergent fenestration closure (1), pericardial effusion (4), and seizures (1). Risk factors included Fontan connection to one lung (3), diminutive pulmonary arteries (PAs) and unifocalized major aortopulmonary collateral arteries (MAPCAs) (1), discontinuous PAs (3), right ventricle dependent coronaries (3), neonatal pulmonary venous obstruction (3), Trisomy 21 (1), preoperative pacemaker dependence (2), and heterotaxy (10). No candidate was excluded. CONCLUSIONS: While many surgeons try to avoid bypass or aortic clamping when performing Fontan operations, the strategies we have employed facilitate safe accomplishment of Fontan's operation in diverse anatomic groups with multiple risk factors, with avoidance of operative mortality in 100 consecutive cases.

Anterior vertebral body tethering for idiopathic scoliosis: two-year results.

STUDY DESIGN: Retrospective review. OBJECTIVE: To report the 2-year results of the initial cohort undergoing anterior vertebral body tethering (VBT). SUMMARY OF BACKGROUND DATA: Anterior VBT is a promising new technique with abundant preclinical studies but very few clinical results. It is a growth modulation technique, which utilizes patients' growth to attain progressive correction of their scoliosis. We report 2-year results of the initial cohort undergoing this procedure. METHODS: After obtaining institutional review board approval, we retrospectively reviewed our first 11 consecutive patients who underwent anterior VBT with 2-year follow-up. We collected pertinent preoperative, intraoperative, and most recent clinical and radiographical data. Student t test and Fisher exact test were utilized to compare different time points. RESULTS: Eleven patients with thoracic idiopathic scoliosis (8 females) were identified, with a mean age of 12.3 ± 1.6 years. Preoperatively, all were skeletally immature (Sanders mean = 3.4 ± 1.1; Risser mean = 0.6 ± 1.1). All underwent tethering of an average of 7.8 ± 0.9 (range: 7-9) levels, with the most proximal being T5 and the most distal L2. Preoperative thoracic Cobb angle averaged 44.2 ± 9.0° and corrected to 20.3 ± 11.0° on first erect, with progressive improvement at 2 years (Cobb angle = 13.5 ± 11.6°, % correction = 70%; P < 0.00002). Similarly, the preoperative lumbar curve of 25.1 ± 8.7° demonstrated progressive correction (first erect = 14.9 ± 4.9°, 2 yr = 7.2 ± 5.1°, % correction = 71%; P < 0.0002). Thoracic axial rotation as measured by a scoliometer went from 12.4 ± 3.3° preoperatively to 6.9 ± 3.4° at the most recent measurement (P < 0.01). No major complications were observed. As anticipated, 2 patients returned to the operating room at 2 years postoperatively for loosening of the tether to prevent overcorrection. CONCLUSION: Anterior VBT is a promising technique for skeletally immature patients with idiopathic scoliosis. This technique can be performed safely and can result in progressive correction. LEVEL OF EVIDENCE: 4.

Coronary steal syndrome after coronary artery bypass for anomalous aortic origin of a coronary artery.

Anomalous aortic origin of a coronary artery found in a symptomatic 9-year-old boy was initially treated with coronary artery bypass grafting using a left internal mammary artery anastomoses to the left anterior descending coronary artery, but resulted in coronary ischemia, likely from a steal phenomenon. Subsequent transection of the proximal left internal mammary artery with anastomosis to the ascending aorta, and coronary ostial enlargement, resulted in a durable treatment. We recommend caution in choosing coronary artery bypass grafting using a left internal mammary artery pedicle graft for the treatment of anomalous aortic origin of a coronary artery.

Electromagnetic Interference (EMI) and arrhythmic events in ICD patients undergoing gastrointestinal procedures.

BACKGROUND: The objective was to determine the effect of electromagnetic interference (EMI) in patients undergoing gastrointestinal endoscopy. The implantable cardioverter-defibrillator (ICD) is the primary therapeutic modality for patients at risk for sudden cardiac death. One potential problem with ICDs is interactions with electrical devices and medical procedures causing EMI or triggering arrhythmic events. Endoscopy frequently employs electrocautery (EC) for diagnosis and treatment of gastrointestinal diseases. Current guidelines advise inactivating ICDs before any surgical procedure. There is limited information on management of ICDs during endoscopy with or without EC. We prospectively evaluated patients with ICDs undergoing endoscopic procedures at our institution. METHODS AND RESULTS: Forty-one ICD patients underwent 52 gastrointestinal endoscopies over 17 months. The mean age of the population was 66 years (51-83). There were 28 men and 13 women. Thirteen patients had single chamber devices, 25 had dual chamber devices, and 2 had biventricular ICDs. The mean tachyarrhythmia detection rate programmed was 164.7 bpm (125-188). Eighteen procedures (43.9%) required biopsy, coagulation, or polypectomy. Of these, 10 (55%) required the use of EC. Only unipolar EC with mean current 19.6 mA was used. All ICDs were programmed to detection-only with therapies off. Sensitivity was left at nominal programmed settings. Post procedure interrogation showed no detection of EMI or tachyarrhythmic events. CONCLUSIONS: Our study shows no EMI or arrhythmic events triggered during endoscopic procedures in patients with pectorally implanted transvenous ICDs. Routine practice of programming ICDs off for gastrointestinal procedures may not be necessary. However, larger studies are needed before change in current recommendations.

Report of the 2005 STS Congenital Heart Surgery Practice and Manpower Survey.

BACKGROUND: Limited information is available concerning the congenital heart surgery workforce in North America. To obtain reliable data, The Society of Thoracic Surgeons (STS) Workforce on Congenital Heart Surgery undertook a subspecialty focused survey. METHODS: Preliminary research of websites and databases revealed a potential target group of 263 congenital heart surgeons, including 248 surgeons at 121 US centers and 15 at eight Canadian sites. Surveys were sent to these, plus any additional individuals who listed an interest in congenital or pediatric heart surgery on CTSNet or STS websites. Responders include active congenital heart surgeons, inactive, or retired surgeons, adult cardiac surgeons, and surgeons in training. Analysis is based exclusively on the responses of 217 active congenital heart surgeons (>80% of target). RESULTS: Average age was 48.3 +/- 8.3 years (range, 33 to 78). Ten were female (5%), 207 male (95%). American Medical School graduates were 170 (79%), with 9.2 +/- 1.6 years of postgraduate training. Eighty-five percent are Thoracic Board certified. Congenital heart surgery training outside the United States or Canada was obtained by 29 (14%). One hundred twenty-eight (59%) do exclusively congenital heart surgery. One third perform fewer than 100 congenital cases per year, one third perform 100 to 199, and one third perform 200 or more. Congenital heart surgeons have been in their current positions for 9.5 +/- 8.2 years. Eleven are in their first year of practice. Mean anticipated years to retirement is 15.7 +/- 7.5 (range, 1 to 34). Three anticipate retirement within 1 year. At the same time, 39 fellows will complete postgraduate training at 28 centers this year, and 19 will seek positions in North America. CONCLUSIONS: These data should help to facilitate rational plans to meet manpower needs, including evolving policies concerning training and certification.

Late left pulmonary artery stenosis after the Norwood procedure is prevented by a modification in shunt construction.

BACKGROUND: Late left pulmonary artery (LPA) stenosis occurs commonly after the Norwood procedure, and complicates subsequent stages. Compression by the neoaorta and ductal stump may favor flow into the right pulmonary artery, resulting in LPA hypoplasia. We hypothesize that an early compromise of LPA flow contributes to late LPA stenosis, and have modified our shunt to compensate. METHODS: We reviewed 34 consecutive neonates undergoing the Norwood procedure between 1999 and 2002, and morphometric data from angiograms obtained before the bidirectional cavopulmonary anastomosis (BDCPA). The Norwood technique included an autologous arch reconstruction with or without augmentation, and a polytetrafluoroethylene Blalock-Taussig shunt (BTS). Starting February 2001, the distal shunt was modified from an end-to-side construction to an oblique anastomosis directed into the retroaortic LPA. RESULTS: Norwood survival was 82%. LPA stenosis required plasty in 10 of 13 (77%) premodification survivors, and in 2 of 9 (22%) postmodification (p = 0.027). Bypass time was 151 +/- 65 minutes with LPA plasty versus 95 +/- 50 minutes without. Mortality (15% vs 0%), hospital stay (25 +/- 35 vs 9 +/- 6 days), and incidence of subsequent interventions were correspondingly higher with LPA stenosis. Ten of 13 patients (77%) with a BTS insertion point outside the central region of the pulmonary artery required LPA plasty, versus 2 of 9 (22%) with an insertion nearer to the center (p = 0.027). CONCLUSIONS: An oblique distal BTS anastomosis directed leftward onto the retroaortic pulmonary artery at the time of the Norwood procedure may prevent late LPA stenosis and its attendant morbidity.