PMID- 16281429
OWN - NLM
STAT- MEDLINE
DCOM- 20051129
LR  - 20170214
IS  - 1740-7745 (Print)
IS  - 1740-7745 (Linking)
VI  - 2
IP  - 4
DP  - 2005
TI  - A Bayesian approach to randomized controlled trials in children utilizing 
      information from adults: the case of Guillain-Barre syndrome.
PG  - 305-10; discussion 364-78
AB  - BACKGROUND: Guillain-Barre syndrome (GBS) is a rare neurologic disease that 
      occurs at all ages, causing a progressive, ascending paralysis that usually 
      resolves over weeks or months. The disease appears to be identical in children 
      and adults, except that children recover more quickly, with fewer residua. For 
      patients who lose the ability to walk independently, the main treatment options 
      are plasmapheresis or intravenous immune globulin (IVIg), treatments that have 
      shown to have identical effectiveness in adults in two large RCTs involving 388 
      patients. The effectiveness of the treatments in children has only been studied 
      in small, poorly controlled studies. If one could capture all eligible patients 
      in the United States, only about 100-300 children would be available for a trial 
      annually. METHODS: The goal of this case was to demonstrate how Bayesian methods 
      could be used to incorporate prior information on treatment efficacy from adults 
      to design a randomized noninferiority trial of IVIg versus plasmapheresis in 
      children. A Bayesian normal-normal model on the hazard ratio of time to 
      independent walking was implemented. RESULTS: An evidence-based prior was 
      constructed that was equivalent to 72 children showing exact equivalence between 
      the therapies. A design was constructed based on a Bayesian normal-normal model 
      on the hazard ratio, yielding a sample size of 160 children, with a preposterior 
      analysis demonstrating a "Type I" error rate of 5% and a power of 77%. 
      CONCLUSIONS: This case study illustrates a rational approach to constructing an 
      evidence-based prior that would allow information from adults to formally augment 
      data from children to minimize unnecessary pediatric experimentation. The 
      frequentist properties of a Bayesian design can be evaluated and reported as they 
      would be for a standard design. Discussion of the appropriate prior for such 
      designs is both a necessary and desirable feature of Bayesian trials.
FAU - Goodman, Steven N
AU  - Goodman SN
AD  - Department of Oncology, Johns Hopkins School of Medicine and Public Health, USA.
FAU - Sladky, John T
AU  - Sladky JT
LA  - eng
PT  - Journal Article
PL  - England
TA  - Clin Trials
JT  - Clinical trials (London, England)
JID - 101197451
RN  - 0 (Immunoglobulins, Intravenous)
SB  - IM
CIN - Clin Trials. 2005;2(4):311-8; discussion 319-24, 364-78. PMID: 16281430
MH  - Adult
MH  - *Bayes Theorem
MH  - Child
MH  - Evidence-Based Medicine
MH  - Guillain-Barre Syndrome/classification/*drug therapy/therapy
MH  - Humans
MH  - Immunoglobulins, Intravenous/*therapeutic use
MH  - *Plasmapheresis
MH  - Randomized Controlled Trials as Topic/*methods
MH  - Treatment Outcome
MH  - Walking
EDAT- 2005/11/12 09:00
MHDA- 2005/12/13 09:00
CRDT- 2005/11/12 09:00
PHST- 2005/11/12 09:00 [pubmed]
PHST- 2005/12/13 09:00 [medline]
PHST- 2005/11/12 09:00 [entrez]
AID - 10.1191/1740774505cn102oa [doi]
PST - ppublish
SO  - Clin Trials. 2005;2(4):305-10; discussion 364-78. doi: 10.1191/1740774505cn102oa.