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.

Impact of Intestinal Rehabilitation Program and Its Innovative Therapies on the Outcome of Intestinal Transplant Candidates.

OBJECTIVES: The outcome of children with intestinal failure has improved during the past decade following the introduction of novel therapies by dedicated intestinal rehabilitation programs (IRP). The aim of the present study was to assess the impact of IRP on the outcome of intestinal transplant (IT) candidates and the transplant waiting list. METHODS: A retrospective cohort study of children assessed for IT (n = 84) during a 10-year period. Comparisons were made among the following 3 time periods: before the establishment of our center's IRP (1999-2002; n = 33), early IRP (2003-2005; n = 18), and late IRP (2006-2009; n = 33). The following endpoints were used: patient outcome following IT assessment (not listed, listed and removed from the list, received transplant, died while on the list), patient characteristics at IT assessment, and patient status at the end of the study. RESULTS: The late-IRP era was associated with an increase in patients who were not listed (42% vs 28% at other periods, P = NS) and patients who were removed from the IT waiting list because of clinical improvement (P < 0.0005), and a decrease in those who died before transplant (15% vs >60% at other periods, P < 0.0005). The cause of death shifted from traditional causes such as liver failure or sepsis to other comorbid conditions (P < 0.005). Improved liver function at listing was also observed during late IRP (P < 0.005). CONCLUSIONS: Treatment by IRP, coupled with recent advances in the medical management of intestinal failure, is associated with improved survival and outcome of patients waiting for IT, and may lead to overall reduction in the number of IT in the future.

Peripherally inserted central catheters for long-term parenteral nutrition in infants with intestinal failure.

BACKGROUND AND AIM: Infants with intestinal failure often require long-term central access for delivery of parenteral nutrition (PN). Traditionally, surgically placed central venous catheters (CVCs) have been used; however, the complications associated with these catheters can lead to significant morbidity. Peripherally inserted central catheters (PICCs) are potentially superior to CVCs because they tend to be smaller, and can be placed without general anesthesia. The purpose of the study is to report the use of PICCs for long-term administration of PN in infants with intestinal failure and compare with previously published catheter infection and venous thrombosis rates. METHODS: A 4-year review of infants younger than 12 months with intestinal failure and a PICC for PN delivery was performed to determine the incidence of catheter-related bloodstream infections (CRBSIs) and PICC-associated venous thrombosis. The complication rates were compared with those reported for CVCs and PICCs in the pediatric literature. RESULTS: A total of 45 infants with intestinal failure, receiving PN through a PICC were included in the study. Data from 95 PICCs accounting for 10,189 catheter days were collected. The overall incidence of CRBSI was 5.3/1000 catheter days and the incidence of venous thrombosis was 2.0/1000 catheter days. CONCLUSIONS: PICCs offer an advantage over CVCs in that they can often be inserted without a general anesthesia and do not require manipulation of the vein. Given the low rate of CRBSI and venous thrombosis, we recommend PICCs for infants with intestinal failure requiring PN.

Change of Outcomes in Pediatric Intestinal Failure: Use of Time-Series Analysis to Assess the Evolution of an Intestinal Rehabilitation Program.

BACKGROUND: The clinical picture of pediatric intestinal failure has changed over the past 15 years, while effectiveness evolving treatment options remains unclear. This study explored evolution in care and quantified independent effects of new treatment options. STUDY DESIGN: Consecutive patients (n = 196) with neonatal or infantile intestinal failure, born between July 1996 and December 2011, were derived from an intestinal rehabilitation program (IRP) patient registry. Change over time was analyzed using multivariable Box-Jenkins method-based autoregressive integrative moving average models (ARIMA), robust linear regression, and nonparametric trend analysis. Four systematically introduced treatment options (IRP, serial transverse enteroplasty, omega-3 lipid emulsions, and ethanol locks) were evaluated. Analyses were adjusted for patient characteristics and disease severity. The primary outcome was disease-specific mortality from liver failure and sepsis. Secondary outcomes included parenteral nutrition weaning, transplantations, catheter complications, and liver disease. RESULTS: Patient characteristics remained unchanged over time, except for decreasing small bowel length (-0.5%/quarter; 95% CI -0.85, -0.16) and ICU admission time (-0.6 days/quarter; 95% CI -1.03, -0.18). Disease-specific mortality diminished significantly over time (-0.02 deaths/quarter; 95% CI -0.03, -0.01) by IRP and omega-3 lipids introduction (-0.6 deaths/quarter each, 95% CI -1.23, -0.02 and -0.77, -0.45, respectively). Serial transverse enteroplasty and ethanol locks had no significant impact. Parenteral nutrition weaning and transplantations remained unchanged, while catheter sepsis and complication rates decreased by 0.3 episodes/1,000 catheter-days each (95% CI -0.43, -0.2 and -0.45, -0.24, respectively). CONCLUSIONS: Introduction of IRP and omega-3 lipids independently decreased disease-specific mortality. For the first time, time series analysis was applied to evaluate effectiveness of treatment options in intestinal rehabilitation.

Delayed primary serial transverse enteroplasty as a novel management strategy for infants with congenital ultra-short bowel syndrome.

BACKGROUND: Congenital ultra-short bowel syndrome (USBS) is a challenging problem with a poor outcome. We report a new management approach for USBS infants that attempts to optimize gut growth potential. METHODS: We report five neonates with USBS in whom no correction was performed at primary surgery except placement of a gastrostomy (G) tube. Sham feeds were started with intermittent G-tube clamping to induce bowel dilatation/growth. Serial fluoroscopy was done until bowel caliber reached 5 cm. STEP was performed and continuity established to the colonic remnant. Small bowel length (SBL) and enteral caloric intake were tabulated. RESULTS: Patients were born with a mean residual SBL of 19 ± 7.6 cm (14.8% of expected). Median duration of sham feeds prior to STEP was 108 (range 27-232)days. Mean SBL at STEP was 47 ± 12.1cm, which increased post-STEP to 70 ± 12.7 cm (a mean increase of 296% from birth, representing 36.4% ± 13.1% of expected gut length). With a median follow-up time of 20 months (range 8-28), 4/5 achieved >50% enteral calories and have normal liver function. One has undergone liver transplantation. CONCLUSIONS: In USBS patients, delayed surgical correction with sham feeds accelerates gut growth, optimizing potential for autologous reconstruction. This approach may offer greater opportunity for intestinal adaptation than traditional options.

The role of parenteral lipids in the development of advanced intestinal failure-associated liver disease in infants: a multiple-variable analysis.

BACKGROUND: Given the recent interest in the role of ω-6 lipids in the development of intestinal failure-associated liver disease (IFALD), the authors sought to examine the role of parenteral lipids in the development of a serum conjugated bilirubin >100 µmol/L (5.9 mg/dL; CB100) in infants. METHOD: Between 2003 and 2004, data were collected prospectively on infants undergoing an abdominal surgical procedure. Univariate logistic regression models for the prediction of CB100 by 1 year postoperatively were developed. Predictors significant at the 0.2 level on univariate analysis were entered into a backward stepwise multiple variable logistic regression. RESULTS: Of 152 infants who received parenteral nutrition (PN) postoperatively, 22 developed CB100. Predictors that met criteria for consideration in the multiple-variable model were age, weight, small bowel length, presence of a stoma, proportion of enteral feeds postoperatively, septic episodes, days of maximal PN amino acid (>2.5 g/kg/d), days of maximal lipid (>2.5 g/kg/d), and PN duration. The final model included septic episodes (odds ratio, 3.23; 95% confidence interval, 1.8-5.9) and days of lipid >2.5 g/kg/d (1.04; 1.003-1.06). At 60 days of maximal lipid, the odds of advanced IFALD were increased 10-fold. CONCLUSIONS: This model suggests a key role of parenteral lipids and septic events in the development of CB100 from IFALD. These data may provide targets, such as careful line care, reduction in maximal lipid dose, or alternate lipids such as ω-3 fatty acids, to prevent CB100, an identified marker of subsequent liver failure from IFALD.

Is the use of parenteral omega-3 lipid emulsions justified in surgical neonates with mild parenteral nutrition-associated liver dysfunction?

PURPOSE: Although evidence suggests that parenteral omega-3 lipid emulsions (O-3LEs) may be beneficial in treating advanced parenteral nutrition (PN)-associated liver disease, our objective was to determine if O-3LEs are justified in those with early liver disease. METHODS: This is a retrospective analysis of prospectively collected data on all surgical neonates, who received more than 1 day of PN postoperatively between 2001 and 2004 with observation through 2005 (era before O-3LE introduction). We examined the proportion of those who developed mild and advanced liver dysfunction. RESULTS: Of the 292 infants in the cohort, 104 (36%) developed mild liver dysfunction (conjugated bilirubin, 34 micromol/L [cBili34]) after a mean of 22 days. Thirty-one (30%) of the cBili34 patients reached a serum conjugated bilirubin of 100 micromol/L, and 13 (13%) developed liver failure. Of these, 4 underwent transplantation, and 5 died of hepatic disease. Overall, 86 of the cBili34 patients (83%) were weaned off PN. CONCLUSION: With more than 80% of cBili34 patients being weaned from PN without adverse hepatic sequelae, it is difficult, in the absence of definitive evidence of efficacy and safety for O-3LEs together with increased costs, to justify the routine use of O-3LEs in this low-risk population outside formal research protocols.

Does the colon play a role in intestinal adaptation in infants with short bowel syndrome? A multiple variable analysis.

PURPOSE: We sought to examine in a multiple variable model the impact of residual colonic length on time to intestinal adaptation in a cohort of infants with short bowel syndrome. METHOD: Infants with a surgical diagnosis of short bowel syndrome who underwent operation 90 days or younger were included in this analysis. Univariate Cox proportional hazards models for time to full-enteral feeds were developed. Predictors significant at the .2 level were entered into a stepwise multiple variable Cox proportional hazards model. RESULTS: A total of 106 infants were included in the cohort (70 adapted). Predictors meeting the criteria for the multiple variable model were as follows: multidisciplinary management (P = .045), Serial Transverse Enteroplasty Procedure (P = .057), percent small bowel (P < .001), percent large bowel (P < .001), preserved ileocecal valve (P = .001), number of septic (P < .001), and central line complications (P < .001). The final model included the following: multidisciplinary management (hazard ratio [HR], 1.932; 95% confidence interval [CI], 1.137-3.281), percent small bowel (HR, 1.028; 95% CI, 1.02-1.04), and septic events (HR, 0.695; 95% CI, 0.6-0.805). CONCLUSIONS: The colon does not seem to play a significant role in intestinal adaptation. However, in addition to highlighting the importance of residual small bowel length, our model highlights the benefit of multidisciplinary intestinal rehabilitation and reduction of septic complications in achieving intestinal adaptation.

Understanding neonatal bowel obstruction: building knowledge to advance practice.

Providing care to neonates with bowel obstruction requires a basic understanding of gastrointestinal (GI) anatomy and functional landmarks as well as knowledge of the pathophysiology associated with intestinal blockage. Early recognition and prompt diagnosis necessitate astute assessment of common presenting symptoms and accurate interpretation of diagnostic investigations. Initial medical management is focused primarily on gastric decompression and maintenance of fluid and electrolyte balance. This article describes features of the neonatal GI tract and discusses common causes of neonatal bowel obstruction.