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Developing a Risk-Based Composite Neurologic Outcome for a Trial of Hydroxyurea in Young Children with Sickle Cell Disease

James F. Casella,Robert J. Adams,Donald J. Brambilla,John J. Strouse, Pia Maier,Rachel Dlugash,Radhika Avadhani,Krista Vermillion,James Tonascia,Jenifer H. Voeks,Daniel F. Hanley,Richard E. Thompson,Harold P. Lehmann,Neil A. Goldenberg,Gregory A. Hale, Connie M. Piccone Case,Deepa G. Manwani, Michael U. Callaghan,Sharada A. Sarnaik,Janet L. Kwiatkowski,Jan P. Boswinkel,Rebecca N. Ichord,Ram V. Kalpatthi,Robert S. Nickel,Eugenie S. Heitmiller,Michael F. Guerrera,Banu Aygun, Hyde Park,Nancy S. Green,Beng R. Fuh, Robert C. Brown,Baba P. D. Inusa,David C. Rees,Alexis A. Thompson,Sherron M. Jackson,Julie Kanter,Anthony D. Villella,Manisha M. Bansal,Paul T. Telfer,Jason M. Fixler,Melanie Kirby-Allen,Elliott P. Vichinsky,Lynne D. Neumayr,Jeffrey D. Lebensburger, Lucien V. Black,Lewis L. Hsu,Ofelia A. Alvarez,Rupa C. Redding-Lallinger,Allison A. King,Robert C. McKinstry,Desiree A. White,James J. Fehr,Michael J. Noetzel,Lori C. Jordan,Julie A. Panepinto,Dennis W. Shaw, John P. Whittle,Suvankar Majumdar,Lydia H. Pecker, Donna A. Whyte-Stewart, Cynthia A. Smith,Joanne E. Shay,Michael A. Kraut,Emily A. Barron-Casella,Kimberly Jones, Andre W. Hackman, Diane C. Weiss,W. Andrew Mould,Steven W. Mayo,Noeleen Ostapkovich

Clinical Trials（2018）

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关键词
Orphan disease,composite outcomes,translating research into practice,clinical trial,sickle cell disease,neurological sequelae
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