Carol Oliveira1, Nicole T de Silva2, Sanja Stanojevic3, Yaron Avitzur4, Ahmed M Bayoumi5, Wendy J Ungar6, Jeffrey S Hoch7, Paul W Wales8. 1. Group for Improvement of Intestinal Function and Treatment Program (GIFT), The Hospital for Sick Children, Toronto, Canada; Division of General and Thoracic Surgery, The Hospital for Sick Children, Toronto, Canada; Child Health Evaluative Sciences (CHES) Program, The Hospital for Sick Children, Toronto, Canada; The Institute of Health Policy, Management and Evaluation (IHPME), University of Toronto, Toronto, Canada. 2. Group for Improvement of Intestinal Function and Treatment Program (GIFT), The Hospital for Sick Children, Toronto, Canada; Division of General and Thoracic Surgery, The Hospital for Sick Children, Toronto, Canada. 3. Division of Respiratory Medicine, The Hospital for Sick Children, Toronto, Canada; Child Health Evaluative Sciences (CHES) Program, The Hospital for Sick Children, Toronto, Canada; The Institute of Health Policy, Management and Evaluation (IHPME), University of Toronto, Toronto, Canada. 4. Group for Improvement of Intestinal Function and Treatment Program (GIFT), The Hospital for Sick Children, Toronto, Canada; Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children, Toronto, Canada. 5. The Institute of Health Policy, Management and Evaluation (IHPME), University of Toronto, Toronto, Canada; Centre for Research on Inner City Health, Li Ka Shing Knowledge Institute and Division of General Internal Medicine, St Michael's Hospital, Toronto, Canada. 6. Child Health Evaluative Sciences (CHES) Program, The Hospital for Sick Children, Toronto, Canada; The Institute of Health Policy, Management and Evaluation (IHPME), University of Toronto, Toronto, Canada. 7. The Institute of Health Policy, Management and Evaluation (IHPME), University of Toronto, Toronto, Canada; Centre for Excellence in Economic Analysis Research (CLEAR), St Michael's Hospital, Toronto, Canada. 8. Group for Improvement of Intestinal Function and Treatment Program (GIFT), The Hospital for Sick Children, Toronto, Canada; Division of General and Thoracic Surgery, The Hospital for Sick Children, Toronto, Canada; Child Health Evaluative Sciences (CHES) Program, The Hospital for Sick Children, Toronto, Canada; The Institute of Health Policy, Management and Evaluation (IHPME), University of Toronto, Toronto, Canada. Electronic address: paul.wales@sickkids.ca.
Abstract
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.
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.
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