Purpura trombocitopenica

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Heterogeneity of terminology and clinical definitions in adult idiopathic thrombocytopenic purpura: a critical appraisal from a systematic review of the literature
Marco Ruggeri, Stefania Fortuna, and Francesco Rodeghiero
Division of Hematology, S. Bortolo Hospital, Vicenza, Italy


Key words:idiopathic thrombocytopenic purpura, ITP,systematic review, diagnosis, therapy, terminology.

Citation: Ruggeri M, Fortuna S, Rodeghiero F. Heterogeneity of terminology and clinical definitions in adult idiopathic thrombocytopenic purpura: a critical appraisal from a systematic review of the literature. Haematologica. 2008 Jan; 93:(1)98-103. DOI: 10.3324/haematol.11582
©2008 Ferrata Storti Foundation. This is an open-access paper.Introduction

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Idiopathic (or immune) thrombocytopenic purpura (ITP) is an acquired disease characterized by a decrease of the platelet count due to platelet auto-antibodies and, in its more severe forms, by bleeding symptoms.1-4 Although ITP has been known for many years, there are still many unresolved issues about pathogenetic mechanisms, epidemiology, diagnosis and management.Notably, for the most part, the treatment of ITP is not based on evidence, due to the lack of clinical trials. Consequently, the few published guidelines are inevitably based only on expert opinions.5-7 Recently, systematic reviews and meta-analysis have been produced8-10 to obtain more accurate and consistent evaluations on the short and long-term outcomes after splenectomy, therapy for refractorycases or anti CD 20 antibody treatment. Unfortunately, all of these reviews had intrinsic weaknesses in the analysis due to the great vari-

Acknowledgments: we are grateful to the members of the Scientific Working Group on Thrombocytopenias of the European Hematology Association (www.tcpeha.org) for their helpful suggestions and criticisms. Funding: this work was supported in part by FondazioneProgetto Ematologia (Hematology Project Foundation). Manuscript received April 2, 2007. Manuscript accepted August 8, 2007. Correspondence: Francesco Rodeghiero, M.D., Division of Hematology, S. Bortolo Hospital Vicenza, 36100 Italy. E-mail: rodeghiero@hemato.ven.it The online version of this article contains a supplemental appendix.
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Clinical definitions and terminology vary greatly in clinical studies on idiopathic thrombocytopenic purpura (ITP). An objective assessment of this heterogeneity may be of interest, providing a basis for standardizing ITP terminology. A systematic review of the recent literature on ITP in adults was carried out. The following items were extracted from the articles forcomparison: platelet count cut-off values to decide treatment and type of response; timing for evaluating the response to treatment; evaluation of bleeding symptoms; criteria to define initial, chronic and refractory forms. A total of 79 papers, among those published or referenced from 2000 to 2006, were considered eligible. No consensus among the different authors was found on several issues,including:platelet count for definition of ITP; grading of severity; definition of chronic ITP; platelet threshold to start treatment; platelet count to define response to treatment and timing for evaluating the response to therapy. There was only major consensus for the length of disease duration required to diagnose chronic ITP the criteria for splenectomy and the definition of refractory ITP. Confusingterminology and an unacceptable het, erogeneity of clinical definitions used for management decisions and to describe outcomes were evident in recent ITP literature. This makes it very difficult to compare different studies and to share data and clinical experiences. A standardization of terminology and definitions used in ITP is urgently needed.

ability of clinical definitions used by the...