Use of Latent Class Analysis to Predict Intensive Care Unit Admission and Mortality in Children with a Major Congenital Anomaly.

OBJECTIVE: To define major congenital anomaly (CA) subgroups and assess outcome variability based on defined subgroups. STUDY DESIGN: This population-based cohort study used registries in Denmark for children born with a major CA between January 1997 and December 2016, with follow-up until December 2018. We performed a latent class analysis (LCA) using child and family clinical and sociodemographic characteristics present at birth, incorporating additional variables occurring until age of 24 months. Cox proportional hazards regression models estimated hazard ratios (HRs) of pediatric mortality and intensive care unit (ICU) admissions for identified LCA classes. RESULTS: The study included 27 192 children born with a major CA. Twelve variables led to a 4-class solution (entropy = 0.74): (1) children born with higher income and fewer comorbidities (55.4%), (2) children born to young mothers with lower income (24.8%), (3) children born prematurely (10.0%), and (4) children with multiorgan involvement and developmental disability (9.8%). Compared with those in Class 1, mortality and ICU admissions were highest in Class 4 (HR = 8.9, 95% CI = 6.4-12.6 and HR = 4.1, 95% CI = 3.6-4.7, respectively). More modest increases were observed among the other classes for mortality and ICU admissions (Class 2: HR = 1.7, 95% CI = 1.1-2.5 and HR = 1.3, 95% CI = 1.1-1.4, respectively; Class 3: HR = 2.5, 95% CI = 1.5-4.2 and HR = 1.5, 95% CI = 1.3-1.9, respectively). CONCLUSIONS: Children with a major CA can be categorized into meaningful subgroups with good discriminative ability. These groupings may be useful for risk-stratification in outcome studies.

Small and large bowel anatomy is associated with enteral autonomy in infants with short bowel syndrome: A retrospective cohort study.

BACKGROUND: Achievement of enteral autonomy (EA) is the ultimate treatment goal in pediatric intestinal failure (IF). We aimed to assess predictors of EA in pediatric short bowel syndrome (SBS) and explore the impact of residual small bowel (SB) and large bowel (LB) length on EA. METHODS: A retrospective cohort study was performed on infants aged <12 months (n = 367, six centers) with SBS referred between 2010 and 2015. The cohort was stratified based on the achievement of EA. Statistical testing was completed using t-test, chi-square, Cox proportional hazards regression model, and Kaplan-Meier analysis. RESULTS: EA was achieved in 229 patients. In the multivariable analysis, the percentage of residual LB (hazard ratio [HR] = 1.02; 95% CI = 1.01-1.02) and SB (HR = 1.01; 95% CI = 1.01-1.02) length, presence of the ileocecal valve (HR = 2.02; 95% CI=1.41-2.88), and not coming from a high-volume transplantation center (HR = 2.42; 95% CI = 1.68-3.49) were positively associated with EA, whereas a negative association was seen with the presence of stoma at the time when shortest remnant was documented (HR = 0.72; 95% CI = 0.52-1.00). EA achievement was significantly different between the anatomical subgroups (log-rank test P < 0.001) with an EA rate of 80.4% in infants with ≥50% SB and LB (median time 209 days); 62.5% with ≥50% SB and <50% LB (397 days); 58.3% with <50% SB and ≥50% LB (1192 days), and 25.9% with <50% SB and LB. Necrotizing enterocolitis (NEC) was not associated with a better achievement of EA (NEC vs other etiologies: log-rank test P = 0.33). CONCLUSIONS: Overall, 62% of infants with IF secondary to SBS achieved EA over a mean time of follow-up of 2.3 years. A colon length of >50% can compensate for the loss of small bowel (<50%) and account for similar EA rates as those in children with residual SB > 50%.

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.

Severe Respiratory Disease Among Children With and Without Medical Complexity During the COVID-19 Pandemic.

Importance: Severe respiratory disease declined during the COVID-19 pandemic, partially due to decreased circulation of respiratory pathogens. However, the outcomes of children with higher risk have not been described using population-based data. Objective: To compare respiratory-related hospitalizations, intensive care unit (ICU) admissions, and mortality during the pandemic vs prepandemic, among children with medical complexity (CMC) and without medical complexity (non-CMC). Design, Setting, and Participants: This population-based repeated cross-sectional study used Canadian health administrative data of children aged younger than 18 years in community and pediatric hospitals during a pandemic period (April 1, 2020, to February 28, 2022) compared with a 3-year prepandemic period (April 1, 2017, to March 31, 2020). The pandemic period was analyzed separately for year 1 (April 1, 2020, to March 31, 2021) and year 2 (April 1, 2021, to February 28, 2022). Statistical analysis was performed from October 2022 to April 2023. Main Outcomes and Measures: Respiratory-related hospitalizations, ICU admissions, and mortality before and during the pandemic among CMC and non-CMC. Results: A total of 139 078 respiratory hospitalizations (29 461 respiratory hospitalizations for CMC and 109 617 for non-CMC) occurred during the study period. Among CMC, there were fewer respiratory hospitalizations in both 2020 (rate ratio [RR], 0.44 [95% CI, 0.42-0.46]) and 2021 (RR, 0.55 [95% CI, 0.51-0.62]) compared with the prepandemic period. Among non-CMC, there was an even larger relative reduction in respiratory hospitalizations in 2020 (RR, 0.18 [95% CI, 0.17-0.19]) and a similar reduction in 2021 (RR, 0.55 [95% CI, 0.54-0.56]), compared with the prepandemic period. Reductions in ICU admissions for respiratory illness followed a similar pattern for CMC (2020: RR, 0.56 [95% CI, 0.53-0.59]; 2021: RR, 0.66 [95% CI, 0.63-0.70]) and non-CMC (2020: RR, 0.22 [95% CI, 0.20-0.24]; RR, 0.65 [95% CI, 0.61-0.69]). In-hospital mortality for these conditions decreased among CMC in both 2020 (RR, 0.63 [95% CI, 0.51-0.77]) and 2021 (RR, 0.72 [95% CI, 0.59-0.87]). Conclusions and Relevance: This cross-sectional study found a substantial decrease in severe respiratory disease resulting in hospitalizations, ICU admissions, and mortality during the first 2 years of the pandemic compared with the 3 prepandemic years. These findings suggest that future evaluations of the effect of public health interventions aimed at reducing circulating respiratory pathogens during nonpandemic periods of increased respiratory illness may be warranted.

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.

Composite lipid emulsion use and essential fatty acid deficiency in pediatric patients with intestinal failure with high parenteral nutrition dependence: A retrospective cohort study.

BACKGROUND: Reports of essential fatty acid deficiency (EFAD) in patients receiving parenteral nutrition (PN) and a composite lipid (mixed oil intravenous lipid emulsion [MO ILE]) are predominantly when managed by lipid restriction. The objective of this study was to determine the prevalence of EFAD in patients with intestinal failure (IF) who are PN dependent without lipid restriction. METHODS: We retrospectively evaluated patients, ages 0-17 years, followed by our intestinal rehabilitation program between November 2020 and June 2021 with PN dependency index (PNDI) of >80% on a MO ILE. Demographic data, PN composition, PN days, growth, and plasma fatty acid profile were collected. A plasma triene-tetraene (T:T) ratio >0.2 indicated EFAD. Summary statistics and Wilcoxon rank sum test evaluated to compare between PNDI category and ILE administration (grams/kilograms/day). P < 0.05 was considered significant. RESULTS: Twenty-six patients (median age, 4.1 years [interquartile range (IQR) = 2.4-9.6]) were included. The median duration of PN was 1367 days (IQR = 824-3195). Sixteen patients had a PNDI of 80%-120% (61.5%). Fat intake for the group was 1.7 g/kg/day (IQR = 1.3-2.0). The median T:T ratio was 0.1 (IQR = 0.1-0.2) with no values >0.2. Linoleic and arachidonic acid were low in 85% and 19% of patients, respectively; however, Mead acid was normal in all patients. CONCLUSION: This report is the largest to date on the EFA status of patients with IF on PN. These results suggest that, in the absence of lipid restriction, EFAD is not a concern when using MO ILEs in children receiving PN for IF.

Body Composition and Physical Activity in Pediatric Intestinal Failure On and Off Parenteral Nutrition.

OBJECTIVES: Data on the relationship between body composition (BC) and physical activity (PA) in children with intestinal failure (IF) are lacking. The objectives were to collect data on PA and BC in children with IF, both parenterally and enterally fed, and to assess the relationship between PA and BC. METHODS: Cross-sectional study in children 5-18 years with IF including those receiving parenteral nutrition (PN) and those fully enterally fed. PA levels were measured using accelerometry. BC was measured by dual-energy X-ray absorptiometry. Data were compared to age- and sex-matched population norms using t tests. Regression analysis assessed the relationship between BC and PA. RESULTS: Fifty-eight children with IF (38 males), mean (SD) age of 10.0 (3.5) years, 20 dependent on PN were included. Patients with IF had significantly fewer steps per day ( P ≤ 0.001) compared with literature controls, with a mean (SD) of 7,972 (3,008) and 11,749 (1,106), respectively. There were no significant differences between patients receiving PN and those enterally fed, but both groups were significantly less active than literature controls ( P < 0.001). Patients with IF had higher fat mass and lower fat-free mass compared to literature controls ( P = 0.008). PA had a significant effect on BC ( r2 = 0.32, P < 0.001). CONCLUSIONS: Children with IF, those receiving PN and those fully enterally fed, are at risk of decreased PA and altered BC. PA should be part of ongoing rehabilitation and management to optimize outcomes.

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.

Intestinal failure among adults and children: Similarities and differences.

Intestinal failure (IF) is a complex medical condition that is caused by a constellation of disorders, resulting in the gut's inability to adequately absorb fluids and nutrients to sustain hydration, growth, and survival, thereby requiring the use of parenteral fluid and/or nutrition. Significant advancements in intestinal rehabilitation have resulted in improved survival rates for individuals with IF. There are distinct differences, however, related to etiology, adaptive potential and complications, and medical and surgical management when comparing children with adults. The purpose of this review is to contrast the similarities and differences between these two distinct groups and provide insight for future directions, as a growing population of pediatric patients will cross into the adult world for IF management.

Use of a combined transient elastography and biochemical strategy to determine liver fibrosis in pediatric intestinal failure.

BACKGROUND & AIMS: Non-invasive monitoring of intestinal failure (IF) associated liver disease is an ongoing challenge in children with IF. Our objective was to develop a combined algorithm of clinical, transient elastography (TE) and biochemical parameters to identify liver fibrosis in this population. METHODS: A retrospective cohort study of IF patients followed by our intestinal rehabilitation program between November 2015 to October 2019. Patients with a liver biopsy and TE were included. Demographic and liver function tests were collected. Fibrosis on liver biopsies was graded using the modified Scheuer score. Decision tree based algorithms classified low (F0-F1) versus high (F2-F4) fibrosis scores based on a combination of TE, biochemical and demographic parameters, using 6-fold classification error, sensitivity and specificity cross-validation (CV) scores. RESULTS: 42 patients (74% male, median age 7.6 (4.6; 42.7) months) were evaluated. Median length of PN therapy was 182 (121; 556) days. High fibrosis was present in 40.5% with a median TE of 12.1 (6.7; 12.9) kPa in high fibrosis children. An algorithm, based on cut-off values for TE of 11.3 kPa and AST of 40 U/L, and grouping of the underlying etiology resulted in a correct classification of 88.1% of the pathology scores; with sensitivity 0.82 (95% CI 0.57; 0.96), specificity 0.92 (95% CI 0.74; 0.99), positive predictive value 0.88 (95% CI 0.64; 0.96) and negative predictive value 0.88 (95% CI 0.73; 0.96). The CV classification error was 28.6%, CV sensitivity 72.2% and CV specificity 75.5%. CONCLUSIONS: This algorithm shows promising results that could simplify non-invasive monitoring of liver fibrosis in children with IF. Validation in additional IF cohorts is needed.

Predictors of Social-Emotional Development and Adaptive Functioning in School-Age Children with Intestinal Failure.

OBJECTIVES: Despite a focus on neurocognition in pediatric intestinal failure (IF) to date, we examined social-emotional and adaptive functioning. METHODS: Children (N = 63) in our IF rehabilitation program underwent neuropsychological assessments including caregiver- and teacher-reported questionnaires. Results were compared to norms using z-tests. Caregiver and teacher reports were compared using t tests. Medical and demographic factors were examined in an exploratory manner using correlation and targeted regression analyses, adjusting for gestational age and full-scale IQ. RESULTS: Caregiver and teacher reports indicated poorer executive, internalizing, behavioral, and adaptive functioning compared to norms. Teachers reported more executive dysfunctions than caregivers. Necrotizing enterocolitis diagnosis predicted internalizing emotional problems. Immigrant status predicted poorer social and practical adaptive functioning. Living with biological parents predicted fewer externalizing emotional and behavioral problems. CONCLUSIONS: The group displayed social-emotional and adaptive functioning concerns. Identifying medical and demographic risks can allow for screening and intervention.

Stress, anxiety, depression, and health-related quality of life in caregivers of children with intestinal failure receiving parenteral nutrition: A cross-sectional survey study.

BACKGROUND: Improved survival rates for children with intestinal failure (IF) have resulted in an increased population of children receiving long-term parenteral nutrition (PN). Our objective was to determine burden on caregivers of children with IF receiving long-term PN. METHODS: We performed a cross-sectional study of caregivers of children with IF receiving long-term PN in our intestinal rehabilitation program. A healthy comparison group matched on age of the child was enrolled. All participants completed standardized questionnaires, including the Parental Stress Index - Short Form (PSI-SF), Hospital Anxiety and Depression Scale (HADS), and PedsQL Family Impact Module (PedsQL FIM). Univariate analysis was completed using a Student t test and chi-square, with an alpha value of <0.05 considered significant. RESULTS: Thirty-eight caregivers of children with IF and 29 caregivers of healthy children consented, with response rates of 89% and 96.5%, respectively. Our study demonstrated increased stress for caregivers compared with comparison parents (PSI-SF total score of 83 [SD = 26.8] vs 62.9 [SD = 13.5]; P < 0.01). Caregivers had increased anxiety (HADS anxiety score of 9.3 [SD = 4.8] vs 6.7 [SD = 3.2]; P = 0.02) and higher depression scores (HADS depression score of 6.3 [SD = 4.3] vs 4.1 [SD = 2.6]; P = 0.02) compared with the comparison group. Caregivers of children with IF demonstrated decreased health-related quality of life (HRQoL) (reduced PedsQL FIM total score of 50.6 [SD = 18.2] vs 84.1 [SD = 20.5]; P < 0.01). CONCLUSIONS: Our results demonstrated significant burden of care in caregivers of children with IF receiving long-term PN, with elevated stress, anxiety, and depression and decreased HRQoL.

An international multicenter validation study of the Toronto listing criteria for pediatric intestinal transplantation.

Deciding which patients would benefit from intestinal transplantation (IT) remains an ethical/clinical dilemma. New criteria* were proposed in 2015: ≥2 intensive care unit (ICU) admissions, loss of ≥3 central venous catheter (CVC) sites, and persistently elevated conjugated bilirubin (CB ≥ 75 µmol/L) despite 6 weeks of lipid modification strategies. We performed a retrospective, international, multicenter validation study of 443 children (61% male, median gestational age 34 weeks [IQR 29-37]), diagnosed with IF between 2010 and 2015. Primary outcome measure was death or IT. Sensitivity, specificity, NPV, PPV, and probability of death/transplant (OR, 95% confidence intervals) were calculated for each criterion. Median age at IF diagnosis was 0.1 years (IQR 0.03-0.14) with median follow-up of 3.8 years (IQR 2.3-5.3). Forty of 443 (9%) patients died, 53 of 443 (12%) were transplanted; 11 died posttransplant. The validated criteria had a high predictive value of death/IT; ≥2 ICU admissions (p < .0001, OR 10.2, 95% CI 4.0-25.6), persistent CB ≥ 75 µmol/L (p < .0001, OR 8.2, 95% CI 4.8-13.9). and loss of ≥3 CVC sites (p = .0003, OR 5.7, 95% CI 2.2-14.7). This large, multicenter, international study in a contemporary cohort confirms the validity of the Toronto criteria. These validated criteria should guide listing decisions in pediatric IT.

Muscle Strength, Agility, and Body Composition in Children With Intestinal Failure on Parenteral Nutrition.

OBJECTIVES: With increasing number of children with intestinal failure (IF) on long-term parenteral nutrition (PN), this study assesses the impact of IF on muscle strength, speed, and agility and body composition (BC), identifying clinical factors that may predict impairment. METHODS: Cross-sectional study in children 5-18 years with IF on PN. Assessments included Bruininks-Oseretsky Test of Motor Proficiency-2 strength and agility subtest (BOT-2), and grip strength. BC data from dual-energy x-ray absorptiometry (DXA) measurements and clinical variables were collected by chart abstraction. Data were compared to age and sex matched controls and population norms. RESULTS: Twenty-one children with IF (14 males), median age 8.33 (IQR: 6.96-11.04) years and 33 controls (20 males), 8.25 (6.67-10.79) years were included. Strength and agility ( P < 0.001) and grip strength ( P = 0.001) differed between groups. Nine of 21 (43%) of children with IF scored >1 standard deviation (SD) below mean on BOT-2 and 13 of 21 (62%) had grip strength >1 SD below mean. DXA measurements showed 10 of 18 (56%) of children had lower fat-free mass (FFM)% and higher fat mass (FM)% than reference norms. Decreased FFM% was associated with lower BOT-2 scores ( r = 0.479; P = 0.044) and grip strength scores >1 SD below mean ( P = 0.047). Additional clinical factors significantly impacting strength and agility included prematurity, height, hospitalizations, sepsis, and small bowel length. CONCLUSIONS: Children with IF are at risk of decreased muscle strength and agility, along with altered BC. Ongoing medical, nutritional, and rehabilitation intervention is vital to optimize outcomes.

Management of pediatric intestinal failure related to short bowel syndrome.

Intestinal failure (IF) secondary to short bowel syndrome is a challenging and complex medical condition with significant risk for surgical and medical complications. Significant advancements in the care of this patient population have led to improved survival rates. Due to their intensive medical needs children with IF are at risk for long-term complications that require comprehensive management and close monitoring. The purpose of this paper is to review the available literature emphasizing the surgical aspects of care for children with IF secondary to short bowel syndrome. A key priority in the surgical care of this patient population includes strategies to preserve available bowel and maximize its function. Utilization of novel surgical techniques and autologous bowel reconstruction can have a significant impact on children with IF secondary to short bowel syndrome related to the function of their bowel and ability to achieve enteral autonomy. It is also important to understand the potential long-term complications to ensure strategies are put in place to mitigate risk with early detection to improve long-term outcomes.

Carrying the Burden: Informal Care Requirements by Caregivers of Children with Intestinal Failure Receiving Home Parenteral Nutrition.

OBJECTIVE: To measure the time that caregivers spend on tasks related to providing care to their child with intestinal failure receiving home parenteral nutrition (PN). STUDY DESIGN: We conducted an exploratory cross-sectional study of caregivers of children with intestinal failure receiving long-term PN followed by our intestinal rehabilitation program. Caregivers completed a daily diary of care-related tasks. Data were analyzed using descriptive statistics. Exploratory models were completed to evaluate factors that influenced the amount of time that caregivers spent providing care. SAS University Edition 2018 (SAS Institute, Cary, NC) was used for data analysis with a P value of less than .05 considered significant. RESULTS: Thirty-four caregivers of children with intestinal failure consented with response rates of 85%. The mean age of the primary caregiver was 37 ± 7.9 years of age with 97% being the child's mother. The median PN exposure was 1239 days (IQR, 432-3012). Caregivers reported a median of 29.2 hours per week (IQR, 20.8-45.7 hours per week) of direct medical care. The majority of time was spent on providing PN and care of the central venous catheter (6.1 hours; IQR, 5.2-8.8). CONCLUSIONS: Caregivers of children with intestinal failure receiving long-term PN provide a significant amount of care to ensure their child remains healthy at home. The most significant amounts of time were spent on the administration of the PN and care of the central venous catheter.

"Line care governs our entire world": Understanding the experience of caregivers of children with intestinal failure receiving long-term parenteral nutrition.

BACKGROUND: Children with intestinal failure (IF) receiving long-term parenteral nutrition (PN) require significant medical care, including high-risk procedures such as accessing a central venous catheter, with the majority provided by family caregivers in the home. This study sought to understand the experiences of family caregivers. METHODS: This was a qualitative study of family caregivers of children with IF. Participants were recruited from an intestinal rehabilitation program to participate in virtual focus groups, which were recorded and transcribed. Thematic analysis was used to capture and describe experiences. RESULTS: Thirteen caregivers providing care to 11 children participated in three virtual focus groups held between May and June 2020. Data analysis revealed five primary themes: (1) caregiving as a 24/7 commitment, (2) facing constant risk of death, (3) chronic illness creates difficult feelings and emotions, (4) effects on all aspects of family life, and (5) adapting and functioning as a family. The findings of this study pair well with the biopsychosocial model highlighting the need for multifaceted assessment and supports. CONCLUSION: The impact of caring for a child with IF receiving long-term PN is significant for caregivers and their families and has implications for all aspects of their lives. A better understanding of the impact on caregivers and their coping strategies lays the groundwork for the optimization of quality of life for caregivers and their family.

Normal anthropometry does not equal normal body composition in pediatric intestinal failure.

BACKGROUND: Published reports on abnormal body composition in pediatric patients with intestinal failure have been in patients with poor growth. The goal of the current study is to report the body composition of normally growing patients with intestinal failure. METHODS: Children 8-18 years old with a dual-energy x-ray absorptiometry (DXA) between January 1, 2013, and July 15, 2018, were included in the study. Data were retrospectively collected from the medical charts and included demographics, residual bowel anatomy, nutrition support, height, and weight. DXA data, including total body less head bone mineral density (BMD), fat mass (FM), and fat-free mass (FFM), were collected and compared with published literature controls matched for age and sex. RESULTS: Thirty-four children met inclusion criteria. Mean age at the time of DXA was 9.6 ± 1.8 years. Weight- and height-for-age z-scores were -0.4 ± 0.9 and -0.5 ± 1.0, respectively. Mean BMD z-score was -1.0 ± 1.3. Twenty-six percent of patients (n = 9) had reduced BMD. Patients with intestinal failure had higher FM (P = .02) and lower FFM (P = .02) compared with controls. CONCLUSIONS: These data show that, despite reference range z-scores for height and weight, children with intestinal failure are at risk for abnormal body composition. Body composition should be routinely measured in children with intestinal failure to direct nutrition interventions.

Trends in Pediatric Intestinal Failure: A Multicenter, Multinational Study.

OBJECTIVES: To assess the natural history and outcomes of children with intestinal failure in a large, multicenter, geographically diverse contemporary cohort (2010-2015) from 6 pediatric intestinal failure programs. STUDY DESIGN: Retrospective analysis of a multicenter intestinal failure cohort (n = 443). Competing-risk analysis was used to obtain cumulative incidence rates for the primary outcome (enteral autonomy, transplantation, or death). The χ2 test and Cox proportional hazard regression were used for bivariate and multivariable analyses. RESULTS: The study cohort comprised 443 patients (61.2% male). Primary etiologies included short bowel syndrome (SBS), 84.9%; dysmotility disorder, 7.2%; and mucosal enteropathy, 7.9%. Cumulative incidences for enteral autonomy, transplantation, and death at 6 years of follow-up were 53.0%, 16.7%, and 10.5%, respectively. Enteral autonomy was associated with SBS, ≥50% of small bowel length, presence of an ileocecal valve (ICV), absence of portal hypertension, and follow-up in a non-high-volume transplantation center. The composite outcome of transplantation/death was associated with persistent advanced cholestasis and hypoalbuminemia; age <1 year at diagnosis, ICV, and intact colon were protective. CONCLUSIONS: The rates of death and transplantation in children with intestinal failure have decreased; however, the number of children achieving enteral autonomy has not changed significantly, and a larger proportion of patients remain parenteral nutrition dependent. New strategies to achieve enteral autonomy are needed to improve patient outcomes.