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Randomized Controlled Trials What do they have to do with you?

Randomized Controlled Trials What do they have to do with you?. Joseph H Piatt, Jr, MD, FAAP Neurological Surgery and Pediatrics Doernbecher Children’s Hospital Oregon Health Sciences University Portland, Oregon, USA. EXPERIMENTAL. EXPERIMENTAL. PATIENTS. STANDARD. STANDARD. TREATMENT.

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Randomized Controlled Trials What do they have to do with you?

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  1. Randomized Controlled TrialsWhat do they have to do with you? Joseph H Piatt, Jr, MD, FAAP Neurological Surgery and Pediatrics Doernbecher Children’s Hospital Oregon Health Sciences University Portland, Oregon, USA RCTs - Hydrocephalus 2000 / Sydney

  2. EXPERIMENTAL EXPERIMENTAL PATIENTS STANDARD STANDARD TREATMENT OUTCOMES RCTs - Hydrocephalus 2000 / Sydney

  3. The patients in the RCT must be like your patient. • Similar with respect to known prognostic factors • Successfully randomized (or stratified before randomization) • Enrolled in the trial without bias RCTs - Hydrocephalus 2000 / Sydney

  4. Medulloblastoma • Evans AE, Jenkin RD, Sposto R, et al.: The treatment of medulloblastoma. Results of a prospective randomized trial of radiation therapy with and without CCNU, vincristine, and prednisone. J Neurosurg 72:572-582, 1990 • Krischer JP, Ragab AH, Kun L, et al.: Nitrogen mustard, vincristine, procarbazine, and prednisone as adjuvant chemotherapy in the treatment of medulloblastoma. A Pediatric Oncology Group study. J Neurosurg 74:905-909, 1991 • Mulhern RK, Kepner JL, Thomas PR, et al.: Neuropsychologic functioning of survivors of childhood medulloblastoma randomized to receive conventional or reduced-dose craniospinal irradiation: a Pediatric Oncology Group study. J Clin Oncol 16:1723-1728, 1998 RCTs - Hydrocephalus 2000 / Sydney

  5. EC / IC Bypass • Failure of extracranial-intracranial arterial bypass to reduce the risk of ischemic stroke. Results of an international randomized trial. The EC/IC Bypass Study Group. N Engl J Med 313:1191-1200, 1985 • Medicare program; withdrawal of coverage of extracranial-intracranial arterial bypass surgery for the treatment or prevention of stroke-- HCFA. Final notice. Fed Regist 56:8206-8208, 1991 • Grubb RL Jr, Derdeyn CP, Fritsch SM, et al.: Importance of hemodynamic factors in the prognosis of symptomatic carotid occlusion. Jama 280:1055-1060, 1998 RCTs - Hydrocephalus 2000 / Sydney

  6. Nimodipine for Head Injury • Teasdale G, Bailey I, Bell A, et al.: A randomized trial of nimodipine in severe head injury: HIT I. British/Finnish Co-operative Head Injury Trial Group. J Neurotrauma 9 Suppl 2:S545-550, 1992 • Murray GD, Teasdale GM, Schmitz H: Nimodipine in traumatic subarachnoid haemorrhage: a re-analysis of the HIT I and HIT II trials. Acta Neurochir (Wien) 138:1163-1167, 1996 • Harders A, Kakarieka A, Braakman R: Traumatic subarachnoid hemorrhage and its treatment with nimodipine. German tSAH Study Group. J Neurosurg 85:82-89, 1996 RCTs - Hydrocephalus 2000 / Sydney

  7. Other treatment measures must be similar to your practice. • Institutional practice variation • surgical technique • operating room practices • medications • nursing policies • New technology RCTs - Hydrocephalus 2000 / Sydney

  8. Antifibrinolytics • Fodstad H, Liliequist B, Schannong M, et al.: Tranexamic acid in the preoperative management of ruptured intracranial aneurysms. Surg Neurol 10:9-15, 1978 RCTs - Hydrocephalus 2000 / Sydney

  9. The experimental treatment must be relevant to your practice. • Institutional practice variation • surgical technique • operating room practices • medications • nursing policies • New technology • “Efficacy” vs “effectiveness” RCTs - Hydrocephalus 2000 / Sydney

  10. Barbiturate Coma • Ward JD, Becker DP, Miller JD, et al.: Failure of prophylactic barbiturate coma in the treatment of severe head injury. J Neurosurg 62:383-388, 1985 • Eisenberg HM, Frankowski RF, Contant CF, et al.: High-dose barbiturate control of elevated intracranial pressure in patients with severe head injury. J Neurosurg 69:15-23, 1988 • Schwartz ML, Tator CH, Rowed DW, et al.: The University of Toronto head injury treatment study: a prospective, randomized comparison of pentobarbital and mannitol. Can J Neurol Sci 11:434-440, 1984 RCTs - Hydrocephalus 2000 / Sydney

  11. The outcomes reported for the standard treatment must make sense. • Institutional practice variation • surgical technique • operating room practices • medications • nursing policies RCTs - Hydrocephalus 2000 / Sydney

  12. Antibiotic prophylaxis • Walters BC, Goumnerova L, Hoffman HJ, et al.: A randomized controlled trial of perioperative rifampin/trimethoprim in cerebrospinal fluid shunt surgery [see comments]. Childs Nerv Syst 8:253-257, 1992 • Zentner J, Gilsbach J, Felder T: Antibiotic prophylaxis in cerebrospinal fluid shunting: a prospective randomized trial in 129 patients. Neurosurg Rev 18:169-172, 1995 • Djindjian M, Fevrier MJ, Otterbein G, et al.: Oxacillin prophylaxis in cerebrospinal fluid shunt procedures: results of a randomized open study in 60 hydrocephalic patients. Surg Neurol 25:178-180, 1986 RCTs - Hydrocephalus 2000 / Sydney

  13. The outcomes measured must be important clinically. • Patient-centered end-points • functional measures • QALYs • Hard end-points • death • device failure • “Surrogate“ outcomes • biochemical data • physiological data RCTs - Hydrocephalus 2000 / Sydney

  14. Hyperbaric Oxygen • Rockswold GL, Ford SE, Anderson DC, et al.: Results of a prospective randomized trial for treatment of severely brain-injured patients with hyperbaric oxygen. J Neurosurg 76:929-934, 1992 RCTs - Hydrocephalus 2000 / Sydney

  15. “Surrogate” End-points - examples • van Ek B, Dijkmans BA, van Dulken H, et al.: Effect of cloxacillin prophylaxis on the bacterial flora of craniotomy wounds. Scand J Infect Dis 22:345-352, 1990 • Niemi T, Tanskanen P, Taxell C, et al.: Effects of nonsteroidal anti-inflammatory drugs on hemostasis in patients with aneurysmal subarachnoid hemorrhage. J Neurosurg Anesthesiol 11:188-194, 1999 • Simma B, Burger R, Falk M, et al.: A prospective, randomized, and controlled study of fluid management in children with severe head injury: lactated Ringer's solution versus hypertonic saline. Crit Care Med 26:1265-1270, 1998 • Eisenberg HM, Frankowski RF, Contant CF, et al.: High-dose barbiturate control of elevated intracranial pressure in patients with severe head injury. J Neurosurg 69:15-23, 1988 RCTs - Hydrocephalus 2000 / Sydney

  16. Surrogate End-points • Because • that’s • where • the light • is! RCTs - Hydrocephalus 2000 / Sydney

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