Gastroesophageal Reflux Disease in Children with Neurological Impairment

1. Gastroesophageal Reflux Disease in Children with Neurological Impairment Raj Srivastava, MD, FRCP(C), MPH Center for Pediatric Clinical Effectiveness, Children’s Hospital of Philadelphia, Dec 5th, 2008

2. Overview • Children with neurologic impairment • Why this population? • High resource utilization • Nutritional and respiratory conditions • Current Studies • Future Steps • Care process model • Multi-center studies

3. Why This Population? • Children with NI are living longer and use increasing resources of the health care system • NI results from many different conditions but they share several common clinical issues (e.g. nutritional and respiratory conditions) • Lack of sufficient evidence base due to small numbers • Idiosyncratic practices within institutions • Lack of a clear medical group that studies outcomes in this population • Opportunity for further study (generalists and specialists, multidisciplinary)

4. High Resource Utilization

5. High Resource Utilization • Agency for Healthcare Research Quality • Kid’s Inpatient Database • 1997, 2000, 2003 • National estimates of hospitalizations • Children ages 0-18 years • Clustered, stratified, weighted sample • 2.9 million hospital discharges • 3,400 hospitals • 38 states

6. Total Number of Hospitalizations Children with Neurologic Impairment 33% increase, p <.01 Year

7. Percentage of All Hospitalizations Children with Neurologic Impairment *p < 0.01

8. Nutritional and Respiratory Conditions • Children with neurological impairment (NI) and severe functional limitations have dysfunctional swallowing and gastroesophageal reflux disease (GERD) placing them at risk for aspiration pneumonia (AP) • AP leads to repeated hospitalization, respiratory failure, compromised quality of life, and death. • Initial GERD management consists of medications

9. Nutritional and Respiratory Conditions • Some children with NI and GERD fail medical management • There are competing management approaches for treating the GERD in order to prevent AP and subsequent respiratory failure • Few published studies comparing treatment efficacy and quality of life outcomes with long-term follow-up for treating these conditions in this population

10. Fundoplication • Fundoplication is an anti-reflux procedure used to treat GERD in children who have failed medical management. • Fundoplication is the third most common procedure performed by pediatric surgeons in the U.S. • Half of these procedures are performed on children with NI

11. Gastrojejunal feeding tubes • GJ tubes are an anti-reflux procedure used to treat GERD in children who have failed medical management. • GJ tubes are frequently used in children with NI

12. Background and Rationale • Multi-center randomized control trial (RCT) is the best approach to answering questions about GERD management in this population: • Equipoise • Outcomes • Sufficient number of patients • Expertise to conduct study

13. Clinical Question In children with neurodevelopmental disabilities who have GERD and have failed medical management, what is the next best management option? Compare time to develop AP and survival Treatment with first fundoplication compared to first gastrojejunal feeding tube (change from gastrostomy to GJ feeding tube in radiology)

14. Study Design and Timeline Post-Enrollment and F/U Period Pre-Enrollment Period Jan 1997 Dec 2005 Oct 2006 Enrollment Period for Cohort Outcomes Inclusion criteria Born NI GERD First Fundo or First GJT AP Death • Retrospective cohort followed for outcomes • Excluded medical management only patients

15. Integrated Reporting and Analysis EDW Financial Data Claims& Eligibility Clinical Data A single source for complex data analysis and reporting Enterprise Data Warehouse Slide Courtesy of Brent James, Intermountain Healthcare

16. Study Groups

17. SurvivalFundoplication vs. Gastrojejunal Tube

18. Aspiration Pneumonia FreeFundoplication vs. Gastrojejunal Tube

19. Comparative Studies

20. Objectives • To examine child and caregiver quality of life for children with NI who received a first fundoplication for treatment of GERD

21. Prospective Study • Eligible: •  NI or at risk •  GERD • 0 – 21 years old • At time of procedure Fundoplication Screening + Entry Repeat all assessments Repeat all assessments Gastrojejunal Feeding Tube 1) Baseline Functional Status – (WeeFIM®) 2) Child QoL – PedQL®/CHQ 3) Caregiver QoL – PSI/SF36 4) Nutrition Outcomes Primary Outcome = Child QoL, Caregiver QoL (1 year post-procedure) Secondary Outcome = Nutrition, Mortality, Adverse Events, Costs, Long-Term Outcomes

22. Study Enrollment

23. Reason for NI

24. Characteristics

25. 6 month Outcomes *Visits related to a complication, the gastrostomy tube, AP or GERD symptoms

26. Child HRQoL

27. Bodily Pain

28. Caregiver Quality of Life

29. Caregiver Quality of Life

30. Caregiver Quality of Life

31. Caregiver Quality of Life • Total Stress remains high during study period • Significantly higher stress than parental norms • 1 in 3 parents expressed clinically significant levels of stress (scores > 90, 90th percentile)

32. Caregiver Quality of Life Total Stress remains high during study period Significantly higher stress than parental norms 1 in 3 parents expressed clinically significant levels of stress (scores > 90, 90th percentile) 1 in 3 parents of children with TBI, 1 in 5 parents of children with CHD

33. Goals of Caregivers • Symptom Reduction • Stop vomiting • Decrease gagging • No more aspiration • Nutrition • Gain weight • Eat by mouth • Medical • Stay healthy • Keep out of hospital

34. Cohort Identification

37. Median Time to RRH

38. Next Steps Evidence-based best practice care process model for GERD and dysphagia evaluation, medical management and when to refer

39. Medical Managementn=60 all with NI who received a Fundoplication • Various medications had been tried and were considered to have failed in these patients • 39% had been treated with acid suppressive agents • 80% with acid blocking agents • 61% with prokinetic agents • Duration? • Dose? • Impact on Symptoms?

40. Diagnosis of GERD

42. Next Steps • R03 for Oct 2009 – Two hospital study • Pilot data for GJ tubes • Equipoise • Two hospitals (feasibility of multi-center study) • Pilot study • Using evidence-based best practices guideline

44. Next Steps • R01 for June 2009 – Multi-center study • Which first procedure? • Feasibility and Inclusion criteria • Outcomes • Number of sites, patients, power • DCC • Protocol • Investigator Meeting or at APSA/other meetings

45. Child Health Research Center, Primary Children’s Medical Center Foundation Eunice Kennedy Shriver National Institute for Child Health and Human Development K23 HD 052553 Acknowledgements

46. Future Steps • Prospective, multidisciplinary • Specifically defined patient population • Clear cut diagnostic criteria for GERD • Uniform implementation of medical therapy • Stringent documentation throughout the course of diagnosis and therapy with objective data • Feedback to providers regarding outcomes of children • Study comparing surgical therapies: fundoplication/GT vs GJ tube placement