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Evidence Based Medicine and Level 1 Outcomes Research in Pediatric Surgery

Evidence Based Medicine and Level 1 Outcomes Research in Pediatric Surgery. George W. Holcomb, III, M.D., MBA Children’s Mercy Hospital Kansas City, Missouri Center for Prospective Clinical Trials www.cmhclinicaltrials.com. The Right People. Evidence Based Medicine.

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Evidence Based Medicine and Level 1 Outcomes Research in Pediatric Surgery

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  1. Evidence Based Medicine and Level 1 Outcomes Research in Pediatric Surgery George W. Holcomb, III, M.D., MBA Children’s Mercy Hospital Kansas City, Missouri Center for Prospective Clinical Trials www.cmhclinicaltrials.com

  2. The Right People

  3. Evidence Based Medicine • Integration of best research evidence with clinical expertise and patient values • Treating patients based on data, not “feeling” (gestalt), or one’s own experience

  4. Levels of EvidenceQuestion A clinical surgeon publishes a retrospective review of 350 patients over 20 years undergoing an endorectal pull-through (Soave procedure) for Hirschsprung’s Disease. This is felt to be a seminal paper on this disease in infants and children. What is the level of evidence for this paper? Level 1 (A) Level 2 (B) Level 3 (C) Level 4 (D) Level 5 (E)

  5. Levels Of Evidence 5– Expert opinion, or applied principles from physiology, basic science, or other conditions 4 – Case series or poor quality case control and cohort studies 3 – Case control studies 2 – Review of case control or cohort studies with agreement or poor quality randomized trial 1 – Prospective, randomized controlled trials

  6. Levels of Evidence • Expert opinion, or applied principles from physiology, basic science, or other conditions Example: • Leave patient intubated and paralyzed for 3-5 days following an esophageal resection to take tension off esophageal anastomosis No data – no study to show intubated/paralyzed patient has less esophageal tension • Transverse incision is used for abdominal exploration in baby b/c better exposure No data to support this practice

  7. Levels of Evidence • Case series or poor quality case control and cohort studies Example: • Paper reviewing results from one approach to a disease Large retrospective review of Soave operation for Hirschsprung’s Disease

  8. Levels of Evidence • Case control studies Example: • Paper showing different management strategies/operative technique for one disease process Single center (or multicenter) retrospective review of Duhamel vs Soave operation for Hirschsprung’s Disease

  9. Levels of Evidence • Review of case control or cohort (followed “long-term”) studies with agreement or a poorly performed prospective randomized trial Example: • Review of two or three large series describing one management strategy (Soave procedure) compared to two or three large series describing another management strategy (Duhamel procedure)

  10. Levels of Evidence • Prospective randomized trials

  11. Flawed Prospective Studies Are Usually Better Than Retrospective Data Collections

  12. p - Value • Commonly accepted p-value is 0.05 • 5/100 (1/20) chance that if a difference is found b/w variables, there really is no difference b/w the variables • If p=0.01 there is a 1/100 chance that if a difference is found b/w variables, there really is no difference • P=0.07 7/100 – still pretty significant, but readers will think there really is no significance b/c >0.05 (depends on what variable one is looking at)

  13. How To Set Up a Study • Alpha –Usually 0.05 is used; establishes level of significance of study • Value is used to determine # of patients needed • Retrospective fundoplication review: 12% vs 5% • 360 patients needed to show a significant difference • If α was 0.1, need fewer patients • If α was 0.01, need more patients (because significance is greater) • Set the α low to avoid a Type I error

  14. How To Set Up a Study • Power – commonly accepted is 0.8 • 20% chance that the study will fail to detect a difference when there really is one or • 80% chance that the study will detect a difference when one does exist • Underpowering a study risks a Type II error

  15. Children’s Mercy Hospital Focus on common conditions which are “controversial”: • Pyloric stenosis • Appendicitis • Pectus excavatum • Fundoplication for reflux • Empyema • Non-palpable intra-abdominal testis

  16. Remember • There is a lot more to an MIS operation than just technique • Postoperative care is also important and open for study (antibiotics?, pain management?, etc.)

  17. Landscape of Pediatric Surgery • Young field, most practicing surgeons are 2-3 generations removed from the founders of the field • Very few training centers, the opinions of a few affect the masses • Wide variety of rare cases make acquisition of objective treatment data difficult

  18. Open vs Lap Pyloromyotomy • Lap vs Open – 2003 - controversial around the world and in our hospital • Different feeding regimens used in our hospital (2 hours, 4 hours, 6 hours) • Different postoperative pain management strategies utilized • Differences between staff made it difficult for residents, NPs, floor nurses • Benefits: single protocol for feeding, pain management, discharge used in study still used currently (6 years later) • No level 1 data

  19. OR time (mins) 19:28 +/- 0.60 19:34 +/- 0.78 Emesis (#) 2.61 +/- 0.27 1.84 +/- 0.23 Full Feeds (hrs) 21:01 +/- 2.16 19:30 +/- 1.46 LOS (hrs) 33:10 +/- 1.63 29:38 +/- 1.69 Tylenol (doses) 2.23 +/- 0.18 1.59 +/- 0.16 ResultsOutcomes OPEN (n = 100) LAP (n = 100) P Value (Mean +/- S.E.) (Mean +/- S.E.) 0.93 0.05 0.43 0.12 0.01 Ann Surg 244:363-370, 2006

  20. Conclusions • Operative approach for pyloromyotomy has no significant influence on operating time or length of recovery • Laparoscopic pyloromyotomy results in significantly less post-operative discomfort • Fewer episodes of emesis and doses of Tylenol • Laparoscopic pyloromyotomy results in obvious cosmetic benefits ASA, 2006

  21. 2. Thoracoscopy vs Fibrinolysis for Empyema

  22. Treatment Of Empyema • Fibrinolysis had been shown to be better than chest tube drainage alone • Primary thoracoscopic debridement had been shown to better than tube drainage alone in several retrospective studies • At the initiation of this study, there were no comparative data between primary thoracoscopic debridement and fibrinolysis as initial treatment for empyema in children

  23. Sample Size • Using our own institution’s retrospective data on length of hospitalization after intervention between thoracoscopic debridement and fibrinolysis with an alpha 0.05 and power of 0.8 • Sample size of 36 with 18 in each arm

  24. Empyema Study ProtocolFibrinolysis • 12 Fr tube placed by IR or surgery in procedure room • 4mg tPA in 40ml NS given into tube on insertion and each day for 3 doses Thoracoscopy • Thoracoscopic debridement with chest tube left behind on – 20 cm H20 suction J Pediatr Surg 44:106-111, 2008

  25. Empyema Study ProtocolPrimary Outcome Measure • Length of hospitalization after intervention (tPA or thoracoscopic debridement) until discharge criteria met (chest tube removed, afebrile & oral analgesics) J Pediatr Surg 44:106-111, 2008

  26. Empyema Study ProtocolSecondary Outcome Measure • Days of Tmax > 38CDays of tube drainage • Doses of analgesia • Days of oxygen requirement • Hospital charges after intervention • Procedure charges J Pediatr Surg 44:106-111, 2008

  27. Study Results Patient Variables at Consultation VATS tPA P Value Age (Years) 4.8 5.2 0.77 Weight (kg) 24.6 20.7 0.52 WBC 20.8 19.7 0.71 O2 support (L/min) 0.81 0.79 0.96 Days of Symptoms 9.0 10.6 0.32 ER/PCP visits 2.9 2.7 0.69 J Pediatr Surg 44:106-111, 2008

  28. LOS (Days) 6.89 6.83 0.96 O2 tx (Days) 2.25 2.33 0.89 PO Fever (Days) 3.1 3.8 0.46 Analgesic doses 22.3 21.4 0.90 Patient Charges $11,660 $7,575 0.01 Study Results Outcomes VATS tPA P Value 16.6% failure rate for fibrinolysis J PediatrSurg 44:106-111, 2008

  29. London Prospective Trial VATS v Fibrinolysis w/Urokinase • No difference in LOS (6 v 6 days) • No difference in 6 month CXR • VATS more expensive ($11.3K v $9.1K) • 16 % failure rate for fibrinolysis Am J Respir Crit Care Med 174:221-227, 2006

  30. CONCLUSIONS • There appears to be no therapeutic or recovery advantages to thoracoscopic debridement compared to fibrinolysis as the primary treatment for empyema • Thoracoscopy results in significantly higher patient charges J PediatrSurg 44:106-111, 2008

  31. PRCTs Now Enrolling • Burn study – SSD vs collagenase (100/150) – Stopped early at interim analysis • One stage vs 2 stage laparoscopic orchiopexy for intra-abdominal testis (28/30) • Standardized feeding protocol vs ad lib feedings following laparoscopic pyloromyotomy (100/150) • Esophago-crural sutures vs no sutures at laparoscopic fundoplication (both groups receive minimal esophageal dissection), APSA 2010 • Irrigation/suction vs suction alone in patients with perforated appendicitis American Surgical 2012? • Use of US vs landmark guided CVL placement (with Stanford) (40/80) • IR drainage of appendiceal abscess with vs w/o instillation of fibrinolytic agent (tPA) (39/62) • SSULS cholecystectomy vs 4 port lap cholecystectomy, APSA 2012? • SSULS splenectomy vs 4 port lap splenectomy (6/30)

  32. Summary • Evidence based decision making will continue to have a stronger presence in medical training • Patient management will continue to become more influenced by evidence over opinion • Hurdles to performing prospective trials in surgery and medicine are mostly surmountable

  33. QUESTIONS www.cmhclinicaltrials.com

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