Cost-utility analysis of 4% tetrasodium ethylenediaminetetraacetic acid, taurolidine, and heparin lock to prevent central line-associated bloodstream infections in children with intestinal failure.

BACKGROUND: Central line-associated bloodstream infections (CLABSI) are a serious complication in children with intestinal failure. This study assessed the incremental costs of 4% tetrasodium ethylenediaminetetraacetic acid (EDTA) compared with taurolidine lock and heparin lock per quality-adjusted life-year (QALY) gained in children with intestinal failure from the healthcare payer and societal perspective. METHODS: A Markov cohort model of a 1-year-old child with intestinal failure was simulated until the age of 17 years (time horizon), with a cycle length of 1 month. The health outcome measure was QALYs, with results expressed in terms of incremental costs and QALYs. Model parameters were obtained from published literature and institutional data. Deterministic, probabilistic, and scenario sensitivity analyses were performed. RESULTS: 4% Tetrasodium EDTA was dominant (more effective and less expensive) compared with taurolidine and heparin, yielding an additional 0.17 QALYs with savings of CAD$88,277 compared with heparin, and an additional 0.06 QALYs with savings of CAD$52,120 compared with taurolidine lock from the healthcare payer perspective. From the societal perspective, 4% tetrasodium EDTA resulted in savings of CAD$90,696 compared with heparin and savings of CAD$36,973 compared with taurolidine lock. CONCLUSIONS: This model-based analysis indicates that 4% tetrasodium EDTA can be considered the optimal strategy compared with taurolidine and heparin in terms of cost-effectiveness. The decision uncertainty can be reduced by conducting further research on the model input parameters. An expected value of perfect information analysis can identify what model input parameters would be most valuable to focus on.

Cost-utility analysis of teduglutide compared to standard care in weaning parenteral nutrition support in children with short bowel syndrome.

BACKGROUND & AIMS: A growing proportion of children with short bowel syndrome (SBS) remain dependent on long-term parenteral nutrition (PN). Teduglutide offers the potential for more children to decrease PN support and achieve enteral autonomy (EA), but at a significant expense. This study aims to assess the incremental costs of teduglutide plus standard of care compared to standard of care alone in weaning PN support per quality-adjusted life year (QALY) gained in children with SBS. METHODS: This is a cost-utility analysis comparing teduglutide with standard of care alone in children with SBS. A microsimulation model of children with SBS on PN aged 1-17 years was constructed over a time horizon of six years, with a cycle length of one month. The study adopted the healthcare system and societal payer perspectives in Ontario, Canada. The health outcome measure was QALYs, with results expressed in terms of incremental costs and QALYs. Scenario analyses were performed to examine the effects of different time horizons, timing of teduglutide initiation, and modeling cost of teduglutide based on pediatric weight-dosing. RESULTS: Incremental healthcare system costs for teduglutide compared to standard of care were CAD$441,314 (95% CI, 414,006 to 441,314) and incremental QALYs were 1.80 (95% CI, 1.70 to 1.89) resulting in an incremental cost-effectiveness ratio (ICER) of CAD$285,334 (95% CI, 178,209 to 392,459) per QALY gained. Incremental societal costs were CAD$418,504 (95% CI, 409,487 to 427,522) and incremental societal QALYs were 1.91 (95% CI, 1.85 to 1.98) resulting in an ICER of CAD$261,880 (95% CI, 136,887 to 386,874) per QALY gained. Scenario analysis showed that teduglutide was cost-effective when it was started two years after intestinal resection (ICER CAD$48,741, 95% CI, 17,317 to 80,165) and when its monthly cost was adjusted using weight-based dosing, avoiding wastage of the remaining 5 mg dose vial (Teduglutide dominated over SOC as the less costly and most effective strategy). CONCLUSIONS: Although teduglutide was not cost-effective in weaning PN support in children with SBS, starting teduglutide once natural intestinal adaptation is reduced and adjusting its monthly cost to reflect cost by volume as dictated by weight-based dosing rendered the intervention cost-effective relative to standard of care. These results indicate the potential for clinicians to re-assess optimal time for initiation of teduglutide after intestinal resection, drug manufacturers to consider the use of multi-dose or paediatric-dose vials, and the opportunity for decision-makers to re-evaluate teduglutide funding.

Assessment of body composition in pediatric intestinal failure: A comparison study.

BACKGROUND: The objective of the study was to compare bioelectrical impedance analysis (BIA) and skinfolds with dual energy x-ray absorptiometry (DXA) in the assessment of body composition of children with intestinal failure. DXA is the reference method for body composition assessment in clinical settings. METHODS: Children aged 1-18 years with intestinal failure whohave DXA as part of routine clinical monitoring were eligible. BIA measured total body water on the same day as DXA. Skinfold measurements were taken at four sites: triceps, biceps, subscapular, and suprailiac. Percentage of fat mass (%FM) and fat-free mass (%FFM) were derived from resistance and reactance measured by BIA by using age-specific equations. Percentage of FM was calculated from skinfold measures by using age-specific equations. Data on patient characteristics, intestinal failure-related factors, and feeding method were collected. Paired t test examined differences in %FM and %FFM and Bland-Altman analysis determined the agreement between BIA, skinfolds, and DXA. Marginal linear model assessed the effect of age, sex, and feeding method on the difference in body composition obtained between DXA and BIA and between DXA and skinfolds. RESULTS: Sixty-eight children with intestinal failure, mean age 8.9 ± 4.2 years, were studied. There was no difference between %FFM and %FM obtained by DXA and BIA (P = 0.26), with a mean bias (95% CI) of -0.69 (-1.9 to 0.5) for %FFM. Sex and age were individually and jointly associated with the bias observed between DXA and BIA (P < 0.05). Skinfold and DXA measurements were significantly different (P < 0.05). CONCLUSIONS: BIA is an acceptable clinical tool for assessing body composition in pediatric intestinal failure.

Health-related quality of life in neonates and infants: a conceptual framework.

PURPOSE: With reduced mortality of neonatal conditions, health-related quality of life (HRQOL) has become an important clinical outcome. However, since the meaning of HRQOL in dependent, non-autonomous infants and neonates remains largely undefined, HRQOL measurement and economic evaluation are limited due to the lack of age-specific methodology. The objective was to construct a conceptual framework of neonatal and infant HRQOL (NIHRQOL) which identifies factors relevant to the neonate and infant, their relationship with each other and the caregiving environment. METHODS: Using qualitative methods, a concept was developed based on in-depth analysis of verbatim records of two focus groups (6 caregivers, 6 healthcare providers) and five interviews with caregivers of chronically ill neonates/infants (n = 2), and healthcare professionals of a pediatric tertiary healthcare center (n = 3). Two analysts independently performed thematic analysis using an inductive and contextual approach. RESULTS: The majority of participants regarded NIHRQOL as an individual entity, which was closely related and strongly influenced by caregivers and family. It may be gauged by the perceived degree of effort required to achieve expected normalcy in everyday life for the neonate/infant and its family. The importance of individual HRQOL factors is developmental stage-dependent. CONCLUSION: Neonatal and infant HRQOL is a multidimensional, multilayered and interconnected concept, where the child's needs contribute most directly, and the caregiver's and society's ability to meet those needs characterize the interdependence between the child and its caregiving environment. Developmental stage-specific HRQOL instruments for premature and mature neonates, and infants are warranted to allow for valid HRQOL measurement.

Corrigendum to "Assessment of School Readiness in Chronic Cholestatic Liver Disease: A Pilot Study Examining Children with and without Liver Transplantation".

[This corrects the article DOI: 10.1155/2017/9873945.].

Assessment of School Readiness in Chronic Cholestatic Liver Disease: A Pilot Study Examining Children with and without Liver Transplantation.

Background. Assessment of school readiness evaluates physical, social-emotional, and neuropsychological domains essential for educational success. Cognitive testing of preschool aged children with chronic liver disease may guide more timely interventions and focused efforts by health care providers. Patients and Methods. Children with chronic cholestatic liver disease diagnosed as an infant and still with their native liver (NL) and children who received a liver transplant (LT) before age of 2 years underwent testing with a battery of well-validated pediatric psychometric measures. Results. Eighteen (13 LT, 5 NL) patients (median age of 4.45 and 4.05 years, resp.) were tested. Median Full-Scale IQ was 98 (range 102-116) for LT and 116 [(range 90-106), p = 0.35, NS] for NL subjects. LT recipients had significantly greater visual based difficulties, poorer caregiver rated daily living skills (p = 0.04), and higher levels of executive function based difficulties (e.g., inattention, inhibition). Conclusion. This pilot study highlights the risk of neuropsychological difficulties in early school age children who were under 2 years of age at time of LT. Comprehensive early school age assessment should integrate psychometric measures to identify children at greatest risk, thus allowing for proactive educational intervention.

Cost of ambulatory care for the pediatric intestinal failure patient: One-year follow-up after primary discharge.

BACKGROUND: Survival of children with intestinal failure has improved over the last decade, resulting in increased health care expenditures. Our objective was to determine outpatient costs for the first year after primary discharge. METHODS: A retrospective analysis was performed in pediatric intestinal failure (PIF) patients between 2010 and 2012. Patients were stratified into 3 groups (1=enteral support with no devices [7 patients], 2=enteral support with devices (gastrostomy and/or ostomy) [19 patients], 3=home parenteral nutrition (HPN) [22 patients]). Data abstraction included clinical characteristics and costs related to medication, enteral/parenteral nutrition, and supplies were calculated. Data were analyzed using one way ANOVA. RESULTS: Forty-eight patients (mean age 7.6months; 31 males [65%]) were studied. See attached table for results. HPN patients had significantly more ambulatory visits (p<0.0001), number of admitted days (p=0.01), and productive days lost (p<0.0001). Total cost of care was significantly higher for HPN patients (mean=$320,368.50, p<0.0001) when compared to other groups. Costs covered by the health care system were significantly higher for patients on HPN (mean=$316,101.56, p<0.0001). CONCLUSION: The outpatient expenditures to care for PIF patients in the first year post primary discharge are significant. Our single payer health care system supports the majority of costs, but families are also incurring expenses related to travel and lost productivity. Children on HPN have more visits to hospital, but have access to more funding options. Children solely on gastrostomy or stoma therapy, however, have a significantly greater personal financial burden.

Biliary complications in pediatric liver transplantation: Incidence and management over a decade.

This study analyzed how features of a liver graft and the technique of biliary reconstruction interact to affect biliary complications in pediatric liver transplantation. A retrospective analysis was performed of data collected from 2001 to 2011 in a single high-volume North American pediatric transplant center. The study cohort comprised 173 pediatric recipients, 75 living donor (LD) and 98 deceased donor (DD) recipients. The median follow-up was 70 months. Twenty-nine (16.7%) patients suffered a biliary complication. The majority of leaks (9/12, 75.0%) and the majority of strictures (18/22, 81.8%) were anastomotic. There was no difference in the rate of biliary complications associated with DD (18.4%) and LD (14.7%) grafts (P = 0.55). Roux-en-Y (RY) reconstruction was associated with a significantly lower rate of biliary complications compared to duct-to-duct reconstruction (13.3% versus 28.2%, respectively; P = 0.048). RY anastomosis was the only significant factor protecting from biliary complications in our population (hazard ratio, 0.30; 95% confidence interval, 0.1-0.85). The leaks were managed primarily by relaparotomy (10/12, 83.3%), and the majority of strictures were managed by percutaneous biliary intervention (14/22, 63.6%). Patients suffering biliary complications had inferior graft survival (P = 0.04) at 1, 5, and 10 years compared to patients without biliary complications. Our analysis demonstrates a lower incidence of biliary complications with RY biliary reconstruction, and patients with biliary complications have decreased graft survival.

Resting energy expenditure in children with cyanotic and noncyanotic congenital heart disease before and after open heart surgery.

BACKGROUND: Failure to thrive is a common problem in children with congenital heart disease (CHD). Resting energy expenditure (REE) in cyanotic and noncyanotic children with CHD before and after open heart surgery has hardly been investigated. METHODS: Twenty-nine children younger than 3 years of age with CHD (14 cyanotic and 15 noncyanotic CHD) who were referred for open heart surgery were enrolled. Data on dietary intake, anthropometric measurements, and indirect calorimetry parameters were measured 24 hours before the surgery, (day -1), and on day 5 after surgery. The measured REE was compared with the Schofield and World Health Organization (WHO) REE prediction equations. RESULTS: The mean +/- SD measured REE was similar in the cyanotic and noncyanotic children before and after surgery (before surgery: 57 +/- 13 and 58 +/- 9 kcal/kg per day, respectively; 5 days after surgery: 59 +/- 10 and 62 +/- 10 kcal/kg per day, respectively). Oxygen consumption (VO2) and carbon dioxide production (VCO2) did not change significantly before and after surgery and were similar in both groups. The measured REE for all children on day -1 and day 5 was similar to the calculated REE using the Schofield equation but was significantly different from the calculated REE using the WHO equation (p < .01). CONCLUSIONS: Significant changes in REE, VCO2, and VO2 were not observed before and 5 days after open heart surgery in children with CHD. These parameters (REE, VCO2, and VO2) were also similar in children with cyanotic versus noncyanotic CHD. The Schofield equation is more accurate than the WHO equation in predicting energy needs of children with CHD, but measurement of REE is preferred over calculation of REE.