Highlights from

ASH 2020

62nd Annual Meeting & Exposition of the American Society of Hematology

Virtual 5 - 8 December 2020

Additional work needed to standardise outcome measures in immune thrombocytopenia

To investigate the impact of the immune thrombocytopenia (ITP) international working group (IWG)’s report on the reporting of studies conducted on ITP, researchers conducted a systematic review on literature published in that field in the decade following the release of the IWG report [1]. What they found was disappointing.

Despite the release of standardised platelet count response definitions by the ITP IWG in 2009, studies reporting treatment responses continue to use variable outcome measures. This lack of uniform reporting standards renders comparisons between treatment approaches meaningless.

Researchers included in this review 168 peer-reviewed manuscripts published between January 2010 and December 2019, that made reference to a platelet count, bleeding, and/or a health-related quality of life inventory as part of assessing response to treatment of ITP. Comparing across publications, they discovered variable definitions of platelet count response outcomes, and a lack of a widely accepted bleeding scale, health-related quality of life questionnaire, or definition of remission for use in ITP patients. These outcome measures were all overlooked by the IWG report.

Of the 168 publications, 141 reported platelet count response outcomes; of these, only 61 (43%) used the definitions as proposed by the IWG. In the 80 (57%) studies that did not use IWG definitions, no fewer than 21 different platelet response outcome measures were utilised.

Only 67 of the 168 manuscripts included references to bleeding outcomes; among these reports, 21 different bleeding outcome schemes were employed.

Only a small number (15) of the studies used a quality-of-life questionnaire as part of their evaluation of treatment effectiveness; among these 15 studies, 8 distinct questionnaires were used.

These findings illustrate the need to further standardise platelet count response, bleeding, and health-related quality of life outcome measures in ITP. Only with a more systematic universal approach can meaningful comparisons of response to treatment be made across studies.

  1. Al-Samkari H et al. Extensive Variability in Platelet Count, Bleeding, and Quality of Life Outcome Measures in Adult and Pediatric Immune Thrombocytopenia: An Appraisal from a Critical Review of the Literature. 62nd ASH Annual Meeting, 5-8 December 2020. Abstract 149.

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