Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group.
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.
Rodeghiero
Francesco
F
Department of Hematology, San Bortolo Hospital, Vicenza, Italy. rodeghiero@hemato.ven.it
Stasi
Roberto
R
Gernsheimer
Terry
T
Michel
Marc
M
Provan
Drew
D
Arnold
Donald M
DM
Bussel
James B
JB
Cines
Douglas B
DB
Chong
Beng H
BH
Cooper
Nichola
N
Godeau
Bertrand
B
Lechner
Klaus
K
Mazzucconi
Maria Gabriella
MG
McMillan
Robert
R
Sanz
Miguel A
MA
Imbach
Paul
P
Blanchette
Victor
V
Kühne
Thomas
T
Ruggeri
Marco
M
George
James N
JN
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