title: Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group.
abstract: Diagnosis and management of immune thrombocytopenic purpura (ITP) remain largely dependent on clinical expertise and observations more than on evidence derived from clinical trials of high scientific quality. One major obstacle to the implementation of such studies and in producing reliable meta-analyses of existing data is a lack of consensus on standardized critical definitions, outcome criteria, and terminology. Moreover, the demand for comparative clinical trials has dramatically increased since the introduction of new classes of therapeutic agents, such as thrombopoietin receptor agonists, and innovative treatment modalities, such as anti-CD 20 antibodies. To overcome the present heterogeneity, an International Working Group of recognized expert clinicians convened a 2-day structured meeting (the Vicenza Consensus Conference) to define standard terminology and definitions for primary ITP and its different phases and criteria for the grading of severity, and clinically meaningful outcomes and response. These consensus criteria and definitions could be used by investigational clinical trials or cohort studies. Adoption of these recommendations would serve to improve communication among investigators, to enhance comparability among clinical trials, to facilitate meta-analyses and development of therapeutic guidelines, and to provide a standardized framework for regulatory agencies.
authors.affiliation: Department of Hematology, San Bortolo Hospital, Vicenza, Italy. rodeghiero@hemato.ven.it
authors.forename: Francesco
authors.initials: F
authors.lastname: Rodeghiero
authors.forename: Roberto
authors.initials: R
authors.lastname: Stasi
authors.forename: Terry
authors.initials: T
authors.lastname: Gernsheimer
authors.forename: Marc
authors.initials: M
authors.lastname: Michel
authors.forename: Drew
authors.initials: D
authors.lastname: Provan
authors.forename: Donald M
authors.initials: DM
authors.lastname: Arnold
authors.forename: James B
authors.initials: JB
authors.lastname: Bussel
authors.forename: Douglas B
authors.initials: DB
authors.lastname: Cines
authors.forename: Beng H
authors.initials: BH
authors.lastname: Chong
authors.forename: Nichola
authors.initials: N
authors.lastname: Cooper
authors.forename: Bertrand
authors.initials: B
authors.lastname: Godeau
authors.forename: Klaus
authors.initials: K
authors.lastname: Lechner
authors.forename: Maria Gabriella
authors.initials: MG
authors.lastname: Mazzucconi
authors.forename: Robert
authors.initials: R
authors.lastname: McMillan
authors.forename: Miguel A
authors.initials: MA
authors.lastname: Sanz
authors.forename: Paul
authors.initials: P
authors.lastname: Imbach
authors.forename: Victor
authors.initials: V
authors.lastname: Blanchette
authors.forename: Thomas
authors.initials: T
authors.lastname: Kühne
authors.forename: Marco
authors.initials: M
authors.lastname: Ruggeri
authors.forename: James N
authors.initials: JN
authors.lastname: George
mesh_terms: D006287:Health Planning Guidelines; D006801:Humans; D007391:International Cooperation; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000145:classification* / Q000175:diagnosis* / Q000628:therapy*; D012015:Reference Standards; D009626:Terminology as Topic; D016896:Treatment Outcome