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Prior elicitation of the efficacy and tolerability of Methotrexate and Mycophenolate Mofetil in Juvenile Localised Scleroderma.

Yasin DesaiThomas JakiMichael W BeresfordThomas BurnettDespina EleftheriouHeidi JacobeValentina LeoneSuzanne LiPavel MozgunovAthimalaipet V RamananKathryn S TorokMarina E AndersonJordi AntonTadej AvcinJessie FeltonIvan FoeldvariBisola LagudaFlora McErlaneLindsay ShawFrancesco ZulianClare E Pain
Published in: AMRC open research (2021)
Key factors have been defined regarding the design of a future Bayesian approach clinical trial including elicitation of prior opinion of the efficacy and tolerability of MTX and MMF in JLS. Combining further efficacy data on MTX and MMF with prior opinion could potentially reduce the pre-trial uncertainty so that, when combined with smaller trial sample sizes a compelling evidence base is available.
Keyphrases
  • clinical trial
  • open label
  • phase ii
  • phase iii
  • study protocol
  • double blind
  • systemic sclerosis
  • electronic health record
  • high dose
  • current status