Patient-reported outcome measures in dysphagia: a systematic review of instrument development and validation.

OBJECTIVE: Patient-reported outcome (PRO) measures are commonly used to capture patient experience with dysphagia and to evaluate treatment effectiveness. Inappropriate application can lead to distorted results in clinical studies. A systematic review of the literature on dysphagia-related PRO measures was performed to (1) identify all currently available measures and (2) to evaluate each for the presence of important measurement properties that would affect their applicability. DESIGN: MEDLINE via the PubMed interface, the Cumulative Index of Nursing and Allied Health Literature, and the Health and Psychosocial Instrument database were searched using relevant vocabulary terms and key terms related to PRO measures and dysphagia. Three independent investigators performed abstract and full text reviews. Each study meeting criteria was evaluated using an 18-item checklist developed a priori that assessed multiple domains: (1) conceptual model, (2) content validity, (3) reliability, (4) construct validity, (6) scoring and interpretation, and (7) burden and presentation. RESULTS: Of 4950 abstracts reviewed, a total of 34 dysphagia-related PRO measures (publication year 1987-2014) met criteria for extraction and analysis. Several PRO measures were of high quality (MADS for achalasia, SWAL-QOL and SSQ for oropharyngeal dysphagia, PROMIS-GI for general dysphagia, EORTC-QLQ-OG25 for esophageal cancer, ROMP-swallowing for Parkinson's Disease, DSQ-EoE for eosinophilic esophagitis, and SOAL for total laryngectomy-related dysphagia). In all, 17 met at least one criterion per domain. Thematic deficiencies in current measures were evident including: (1) direct patient involvement in content development, (2) empirically justified dimensionality, (3) demonstrable responsiveness to change, (4) plan for interpreting missing responses, and (5) literacy level assessment. CONCLUSION: This is the first comprehensive systematic review assessing developmental properties of all available dysphagia-related PRO measures. We identified several instruments with robust measurement properties in multiple diseases including achalasia, oropharyngeal dysphagia, post-surgical dysphagia, esophageal cancer, and dysphagia related to neurological diseases. Findings herein can assist clinicians and researchers in making more informed decisions in selecting the most fundamentally sound PRO measure for a given clinical, research, or quality initiative.

Evaluating the Impact of Performance Improvement Initiatives on Transplant Center Reporting Compliance and Patient Follow-Up After Living Kidney Donation.

With the changing demographics of the living donor population and increased regulatory oversight, it is important that transplant centers report outcomes accurately. The aim of our retrospective cohort study of 312 living donors who underwent nephrectomy between 2008 and 2013 was to evaluate the impact of living donor program performance improvement initiatives on: (i) transplant center program reporting compliance; (ii) patient compliance with postdonation follow-up and its associated factors; and (iii) overall financial costs to the transplant center. The effect of the initiatives (donation eras 2008-2010 and 2011-2013) on compliance at key reporting points (6 months, 1 year, 2 years) was analyzed using correlation coefficients, χ(2) and Fisher's exact tests. Multivariable logistic regression models tested the initiatives' effect on the likelihood of patient follow-up. The initiatives were associated with significant improvement in form reporting compliance (r ≥ 0.862, p ≤ 0.027; 1 and 2 year Fisher's Exact p ≤ 0.002) and patient follow-up (χ(2) p ≤ 0.009) with acceptable transplant center costs. Multivariable analyses demonstrated that donation era was consistently and significantly (p < 0.001) associated with increased likelihood of postdonation patient follow-up. Institution of performance improvement initiatives with dedicated program resources is financially feasible and leads to more accurate and complete form reporting and improved patient follow-up after nephrectomy.

A web-based application for initial screening of living kidney donors: development, implementation and evaluation.

Most centers utilize phone or written surveys to screen candidates who self-refer to be living kidney donors. To increase efficiency and reduce resource utilization, we developed a web-based application to screen kidney donor candidates. The aim of this study was to evaluate the use of this web-based application. Method and time of referral were tabulated and descriptive statistics summarized demographic characteristics. Time series analyses evaluated use over time. Between January 1, 2011 and March 31, 2012, 1200 candidates self-referred to be living kidney donors at our center. Eight hundred one candidates (67%) completed the web-based survey and 399 (33%) completed a phone survey. Thirty-nine percent of donors accessed the application on nights and weekends. Postimplementation of the web-based application, there was a statistically significant increase (p < 0.001) in the number of self-referrals via the web-based application as opposed to telephone contact. Also, there was a significant increase (p = 0.025) in the total number of self-referrals post-implementation from 61 to 116 per month. An interactive web-based application is an effective strategy for the initial screening of donor candidates. The web-based application increased the ability to interface with donors, process them efficiently and ultimately increased donor self-referral at our center.

The impact of a laparoscopic colorectal surgeon on the laparoscopic colectomy experience of a single academic center.

BACKGROUND: The objective of this study was to evaluate the impact of a laparoscopic colorectal surgeon (LCRS) on the laparoscopic colectomy experience of a single academic center. METHODS: We performed a retrospective review of case complexity, patient characteristics, operative and preparation time, and trends over time for the LCRS compared to two veteran laparoscopic surgeons (VLS). RESULTS: The LCRS performed 48 of the procedures (83%) and the VLS 10 (17%) for a total of 58 laparoscopic colon cases. The LCRS handled a greater number of complex cases (p = 0.07). For less complex cases, overall operative time differed for the two groups (LCRS = 220 +/- 11 vs VLS = 152 +/- 15 min, p = 0.004). Overall hospital stay was 4.8 +/- 0.6 days (range, 2-33). Minor complications occurred in 12 cases (21%); major complications in occurred in seven cases (12%). Among procedures performed by the LCRS, comparison of the first 24 cases to the second 24 demonstrated that operative and preparation time decreased in the second cohort (all p < 0.05). CONCLUSION: The addition of an LCRS had a significant impact on this center's experience with laparoscopic colectomies in terms of both volume and case complexity.

Weight gain and nutritional efficacy in anorexia nervosa.

To evaluate the usefulness of interval weight change in assessing nutritional support efficacy, we studied four anorexia nervosa patients (52% ideal body weight) requiring long-term total parenteral nutrition (TPN) for 63 +/- 18 days. Fluid and electrolyte deficits were corrected before the initiation of nutritional support. Resting energy expenditure was measured before the initiation of TPN and weekly thereafter, using indirect calorimetry. Daily caloric expenditure was estimated at 1.1 X resting energy expenditure, based on previous studies of continuous heart rate monitoring in this patient population. Daily excess calories were calculated as caloric intake minus caloric expenditure. Each patient was weighed daily and linear regression analysis (excess calories versus weight change) was performed for individual patients and the group over intervals of varying length. There was no individual or group correlation between excess calories and weight gain on a daily or weekly interval basis. Cumulative weight changes over the long-term course of TPN correlated significantly with cumulative excess calories for each patient and the whole group (r = +0.82, p less than 0.01). The excess calories required to gain a kilogram body weight ranged from 5569 to 15619 kcal/kg with a mean of 9768. Cumulative long-term weight changes during nutritional repletion in anorexia nervosa are meaningful indicators of caloric balance, but short interval weight changes (daily, weekly) are not. The caloric cost of weight gain is variable in this population.

Controlled trial of the metabolic effects of a very-low-calorie diet: short- and long-term effects.

Resting energy expenditure (REE), weight, and body composition were measured up to seven times in 13 obese women during a 24-wk study. Patients were randomly assigned to a very-low-calorie diet (VLCD, 500 kcal/d) or a balanced-deficit diet (BDD, 1200 kcal/d). After 8 wk of supplemented fasting, REE of the VLCD patients decreased by 17% whereas that of the BDD patients was virtually unchanged. REE of the VLCD patients increased during 12 subsequent weeks of realimentation such that differences in REE between the two groups were not statistically significant at week 24 (VLCD = -11%, BDD = -2%). Reductions in weight and fat-free mass (FFM) were 12.1% and 3.6% for the VLCD patients and 10.6% and 4.1% for the BDD patients, respectively. There were no significant differences between the groups in pre- to posttreatment changes in REE normalized to FFM. Results suggest that REE recovers partially after consumption of a VLCD. They also provide evidence of a possible metabolic advantage of weight loss by a more moderate restriction.

Plasma cholecystokinin levels in Prader-Willi syndrome and obese subjects.

The cardinal feature of individuals with Prader-Willi syndrome (PWS) is severe hyperphagia-mediated obesity resulting from a faulty satiety mechanism. PWS is the most common genetic cause of marked obesity. Cholecystokinin (CCK) is a 33-amino-acid peptide found in high levels in the gut and brain involved in mediating the satiety response to meals. Free fatty acids (FFA) are responsible for the stimulation of CCK release after a fatty meal, and CCK and plasma FFA levels rise in tandem in normal individuals. Fasting plasma CCK levels were measured by radio-immunoassay in 33 PWS subjects with a mean age of 22.2 years +/- 8.1 years and 24 obese control subjects without a known cause of their obesity with a mean age of 28.7 years +/- 12.9 years. Consistent with previous findings, neither fasting plasma FFA levels (617.5 versus 486.8 microm/mL) or CCK levels (21.0 versus 19.1 pg/mL) were significantly different in PWS or control subjects, respectively. However, there was a significant correlation between fasting plasma FFA and CCK levels in obese subjects (r = 0. 64, P < 0.01), this correlation was completely lacking in PWS subjects (r = -0.06, P = 0.79). This difference in correlation coefficients constitutes a large effect. There were no significant effects observed for genetic subtypes (15q11-q13 deletion or maternal disomy 15), body mass index, percentage of fat, plasma levels of insulin, C-peptide, glucagon or leptin, age, or gender on CCK levels in our PWS subjects. These results suggest that differences in the peripheral CCK response to FFA levels may be a factor contributing to the altered satiety response in PWS subjects.

Decreased bone mineral density in Prader-Willi syndrome: comparison with obese subjects.

Bone density, anthropometric data, and markers of bone turnover were collected on 21 subjects diagnosed with Prader-Willi syndrome (PWS) and compared with 9 subjects with obesity of unknown cause. In addition, urinary N-telopeptide levels were obtained in all subjects. N-telopeptides are the peptide fragments of type I collagen, the major bone matrix material. During periods of active bone degradation or high bone turnover, high levels of N-telopeptides are excreted in the urine. However, no significant difference was detected in the urinary N-telopeptide levels when corrected for creatinine excretion (raw or transformed data) between our subjects with obesity or PWS and the observed effect size of the between-group difference was small. Although N-telopeptide levels were higher but not significantly different in the subjects with PWS compared with obese controls, the subjects with PWS had significantly decreased total bone and spine mineral density and total bone mineral content (all P < 0.001). No differences in N-telopeptide levels or bone mineral density were observed between subjects with PWS and chromosome 15q deletion or maternal disomy. Thus, decreased bone mineral density in subjects with PWS may relate to the lack of depositing bone mineral during growth when bones are becoming more dense (e.g., during adolescence), possibly because of decreased production of sex or growth hormones and/or long-standing hypotonia. It may not be caused by loss, or active degradation, of bone matrix measurable by the methods described in this study further supporting the possible need for hormone therapy during adolescence.

Resting energy expenditure, body composition, and excess weight in the obese.

This study investigated differences between measured and predicted resting energy expenditure (REE) in 80 women who averaged 104.6 kg in weight and were 49 kg and 88% overweight. Body composition analysis showed that 68% of the excess weight was fat and 32% was fat-free mass (FFM). Normalized for height, total body potassium (TBK) was 113 +/- 15% and total body water (TBW) was 133 +/- 21% of values in nonobese controls. The health of the FFM, defined as the potassium content per kg of FFM, was 84 +/- 13% of normal. Measured and predicted REE were only modestly related (r = +.59), and only 59% of measured REEs were within 10% of predicted values. A stepwise multiple regression indicated that weight was the single best predictor of measured REE and that the size of the FFM made a significantly greater contribution to REE than did the size of the fat mass. Commonly used equations for the prediction of REE are not appropriate for moderately or severely obese patients. Caloric prescription for weight reduction must be tailored to individuals rather than recommending the same caloric intake to persons with varying metabolic rates.

Ablation of abnormal energy expenditure by curative tumor resection.

Resting energy expenditure is abnormal in most patients with cancer and may contribute to cancer cachexia. These metabolic abnormalities may be a direct measure of tumor metabolism, or represent alterations in the size or activity of the body cell mass, or both. To unravel this pathogenesis, we prospectively studied 68 preoperative patients with cancer about to undergo curative resection by measuring resting energy expenditure before and after tumor resection. The preoperative measured resting energy expenditure was compared with expected resting energy expenditure based on Harris-Benedict resting energy expenditure predictions: 10 patients were hypometabolic (less than 90% Harris-Benedict); 35 were normometabolic (90% to 110% Harris-Benedict); and 23 were hypermetabolic (greater than 110% Harris-Benedict). Using each patient as his or her own control, resting energy expenditure normalized or remained normal following curative resection. In contrast, after palliative resection, resting energy expenditure remained hypermetabolic or significantly increased toward hypermetabolism. Tumor induces an abnormal metabolic rate, since tumor removal results in prompt normalization of resting energy expenditure. The abnormal energy expenditure of patients with cancer cannot be solely attributed to abnormal host body composition.

Energy expenditure in malnourished patients with colorectal cancer.

To evaluate energy expenditure in patients who have colorectal cancer with varying stages of disease and to examine the possible determinants of energy expenditure in a group of patients with cancer who have the same type of tumor, we studied 73 patients with biopsy proven and pathologically staged adenocarcinomas of the colon and rectum. Resting energy expenditure (REE) was measured by indirect calorimetry and compared with predicted energy expenditure (PEE), which was calculated from the Harris-Benedict formulas. Nutritional and tumor characteristics were examined. Forty-nine percent of patients had abnormal REE (normal = PEE +/- 10%). One quarter of the patients were hypometabolic (REE less than 90% PEE). The abnormalities persisted despite normalization of REE to metabolic body size (kg0.75) or predicted values based on weight, height, age, and sex. There were no differences in nutritional status, as judged by the percent of weight loss and visceral protein levels, between those patients in the hypometabolic, normometabolic, or hypermetabolic categories, and there were no significant relationships between energy expenditure and the tumor burden. The mean duration of disease in the normometabolic group was 4.5 months, while the hypometabolic and hypermetabolic groups had mean durations of 9.5 and 14.2 months, respectively. The tumor site and duration of the disease are important variables in studies of energy expenditure in patients with cancer.

Energy expenditure in patients with inflammatory bowel disease.

The basal energy expenditure (BEE) calculated from the Harris-Benedict equation was compared with the resting energy expenditure (REE) measured by indirect calorimetry in 12 consecutive hospitalized patients with inflammatory bowel disease. The BEE proved to be equivalent to the REE in the prediction of daily caloric requirements. Patients weighing less than 90% of their ideal body weight had significantly increased energy requirements on a per-kilogram-weight basis as compared with those weighing more than 90% of their ideal body weight. Daily caloric requirements based on 1.75 X BEE would provide 45 kcal/kg/24 hr, which is consistent with reported values necessary for anabolism.

Automated, eight-cage indirect calorimetry in rats.

We have constructed an automated, eight-cage indirect calorimeter (AIC) for the measurement of energy expenditure in rats. We compared the measurements of resting energy expenditure (REE) in rats during a 30-h fast obtained with the AIC with those obtained with a manual indirect calorimetry (MIC) system. There was both a high degree of correlation between the two techniques during the initial 18 h of the fast (r = 0.90, P < 0.05) and strong intertechnique agreement. REE (AIC) decreased during the final 12 h of the 30-h fast (79.6 +/- 2.7-72.0 +/- 4.4 kcal.kg-0.75.d-1 [mean +/- SD, P < 0.01]). REE (MIC) did not show a significant decrease during this part of the fast (79.7 +/- 2.6 - 75.2 +/- 4.7 kcal.kg-0.75.d-1 [P = NS]). During the final 12 h of the fast agreement between the two systems gradually dissipated and correlation was poor (r = 0.375, P < 0.05). The frequency of animal handling necessitated by MIC may have resulted in a stress-induced increase in metabolic work that would mask the animals' adaptive response to starvation. This investigation demonstrates the advantages of the AIC and calls into question the accuracy of manual methods under long-term starvation conditions.

Spontaneous retraction of indwelling catheters: previously unreported complications.

The development of totally implantable reservoir central venous access systems has been an important advance in patients requiring long-term central venous access. With lower rates of infection and thrombosis than those with external catheters, they have potential for greater longevity and patient acceptance. Complications such as subcutaneous prosthetic infection, infusate extravasation, and difficulty in locating and puncturing the port have been reported. We present two cases of catheter migration and withdrawal from the subclavian vein. This presumably occurred from the action of the underlying pectoralis muscle on the reservoir, as well as a "jetting effect" during flushing. We make suggestions for prevention of similar problems in the future.

Examination of DSM-IV criteria for attention deficit/hyperactivity disorder in a county-wide sample.

This study replicated, in the subsequent academic year, teacher-reported prevalence rates for attention deficit/hyperactivity disorder (ADHD) based on DSM-IV. Teachers in grades K-5 in a Tennessee county (10 schools, 214 teachers, and 4323 children) completed questionnaires on all their students consisting of the DSM-IV symptoms for disruptive behavior disorders, except for eight conduct disorders symptoms, seven symptoms screening for anxiety or depression, ratings of performance, and questions about the presence of ADHD, stimulant medication treatment, and behavioral or academic problems. The prevalence rates were 16.1% for ADHD-all types, 8.8% for ADHD-inattentive type (AD), 2.6% for ADHD-hyperactive/impulsive type (HI), and 4.7% for ADHD-combined type and 6.8, 3.2, 0.6, and 2.9%, respectively, when impairment was taken into consideration. The rates of problems differed mostly between ADHD-AD AND ADHD-HI (30% vs. 68%) for behavior and (56% vs. 16%) for academics. Few (11-33%) had an ADHD diagnosis or were treated with stimulant treatment (8-26%). DSM-IV criteria are likely to increase the prevalence but may better characterize the heterogeneity of this disorder.

Obtaining systematic teacher reports of disruptive behavior disorders utilizing DSM-IV.

This study examines the psychometric properties of the Vanderbilt AD/HD Diagnostic Teacher Rating Scale (VADTRS) and provides preliminary normative data from a large, geographically defined population. The VADTRS consists of the complete list of DSM-IV AD/HD symptoms, a screen for other disruptive behavior disorders, anxiety and depression, and ratings of academic and classroom behavior performance. Teachers in one suburban county completed the scale for their students during 2 consecutive years. Statistical methods included (a) exploratory and confirmatory latent variable analyses of item data, (b) evaluation of the internal consistency of the latent dimensions, (c) evaluation of latent structure concordance between school year samples, and (d) preliminary evaluation of criterion-related validity. The instrument comprises four behavioral dimensions and two performance dimensions. The behavioral dimensions were concordant between school years and were consistent with a priori DSM-IV diagnostic criteria. Correlations between latent dimensions and relevant, known disorders or problems varied from .25 to .66.

Emergence of compulsive behavior and tantrums in children with Prader-Willi syndrome.

Many adults with Prader-Willi syndrome are affected by behaviors such as tantrums, skin-picking, and compulsions. The nature and extent of these problems suggest more attention be directed to their emergence in childhood. Our purpose was to investigate behavior problems in children with this syndrome and identify the age at which these behaviors emerge. Parents of children with Prader-Willi syndrome, Down syndrome, and those developing typically completed questionnaires. Children with Prader-Willi syndrome exhibited more compulsions, skin-picking, and tantrums than did the other groups. A discriminant analysis of behavior variables derived two statistically significant functions that were interpreted as developmental milestones and problematic behavior. These functions correctly predicted membership for 79% of grouped cases.

Self-injurious behavior and Prader-Willi syndrome: behavioral forms and body locations.

With few exceptions (e.g., Lesch-Nyhan syndrome), the specific nature of self-injury in relation to identified genetic syndromes associated with mental retardation is poorly understood. In the present study we surveyed the families of 62 persons with Prader-Willi syndrome to determine the prevalence, topographies, and specific body locations of self-injurious behavior. Self-injury was reported for 81% of the participants. Skin-picking was the most prevalent form, with the front of the legs and head being disproportionately targeted as preferred self-injury body sites. Individuals with the 15q11-q13 deletion injured significantly more body sites than did individuals with maternal disomy 15. Results are discussed in relation to previous self-injury body site findings and implications for the relevance of syndrome-specific behavioral phenotypes.

Visual capacity and Prader-Willi syndrome.

PURPOSE: Prader-Willi syndrome (PWS) refers to a genetic disorder induced by an anomaly on chromosome 15 occurring with a frequency of one in 10,000 to 20,000. It is characterized by a unique set of features including infantile hypotonia, obesity in childhood, small hands and feet, hypogonadism, and mental retardation. Reported here are the results of ophthalmic examinations of persons with PWS, together with results from controls comparable in age, percentage of body fat, and intelligence. These data bear on the hypothesis that the ocular anomalies in PWS are unique to this syndrome. METHOD: A comprehensive investigation of PWS brought children and adults to Vanderbilt University for extended testing, which included an ophthalmic examination. Genetic analysis determined unequivocally the PWS diagnosis and identified subgroups-deletion and maternal disomy. A group of persons without PWS but generally comparable in age, body composition, and intelligence served as controls. RESULTS: Significant differences between the deletion and disomy subgroups were not found for the clinical ophthalmic measures. The incidence of anomalies in the combined PWS was similar to those reported in previous studies. A similar pattern was present in the control group except for myopia and stereopsis. An effect of genetic subgroup, however, was observed for random element stereopsis with the maternal disomy group having a greater degree of impairment. CONCLUSION: The overall similarity between the PWS and control groups on all measures except myopia and stereopsis suggest that many of the anomalies in PWS found in prior studies are due to factors inherent in a general dysfunctional population, rather than reflective of an ocular signature unique to PWS.

Health-related quality of life before and after solid organ transplantation. Measurement consideration, reported outcomes, and future directions.

The initial focus in organ transplantation clinical research was demonstrating acceptable technical and survival outcomes. Both patient and graft survival have reached well-documented, laudable levels, and solid organ (liver, heart, kidney, lung) transplantation procedures are now relatively common. As with any complex medical procedure that entails relatively high risk, financial costs, and life-long follow-up care, reliable and valid assessments of the "quality" of the extended life years are of interest to patients, their families, policy makers, and payers. This review focuses on health-related quality of life (HRQOL) and functional performance in adults following solid organ transplantation, with an emphasis on: 1) instruments and methods; 2) outcomes in liver, heart, kidney, and lung transplant recipients; and 3) future research directions. Practical considerations for developing longitudinal HRQOL assessment strategies are reviewed. The current emphasis on modeling demographic and clinical factors that promote or limit optimal HRQOL is illustrated. These lines of research will help identify potential interventions designed to promote better HRQOL in organ transplant recipients.