Lack of treatment-related mortality definitions in clinical trials of children, adolescents and young adults with lymphomas, solid tumors and brain tumors: a systematic review.

Thai Hoa Tran,Michelle Lee,Jason D Pole,Sarah Alexander,Marianna Silva,Ute Bartels,Paul Gibson,Carol Portwine,Donna L Johnston,Lillian Sung

doi:10.1186/1471-2407-14-612

Abstract

BackgroundThere is a lack of standardized definition for treatment-related mortality (TRM), which represents an important endpoint in cancer. Our objective was to describe TRM definitions used in studies of children, adolescents and young adults with lymphomas, solid tumors and brain tumors.MethodsWe conducted a systematic review of studies enrolling children, adolescents and young adults with lymphomas, solid tumors and brain tumors in which an anti-cancer intervention was randomized, or all study designs in which TRM was a primary or secondary outcome. We searched Ovid MEDLINE, EMBASE and Evidence-Based Medicine Reviews from 1980 to June 2013. Two reviewers evaluated study eligibility and abstracted data.ResultsIn total, 67 studies were included and consisted of 62 randomized therapeutic trials and 5 TRM studies. None of the studies (0/67) provided a definition for TRM. Only one randomized trial of rhabdomyosarcoma provided a definition of early death.ConclusionsWe were unable to identify any TRM definitions used in studies of children, adolescents and young adults with lymphomas, solid tumors and brain tumors. Given that a proportion of this patient population may receive intensive treatment, there is an urgent need for consensus-based definitions of TRM for use across clinical trials.Electronic supplementary materialThe online version of this article (doi:10.1186/1471-2407-14-612) contains supplementary material, which is available to authorized users.

Highlights

There is a lack of standardized definition for treatment-related mortality (TRM), which represents an important endpoint in cancer
If TRM is the primary cause of failure for a specific cancer, the strategy must focus on enhancing supportive care and/or using less toxic therapies [1]
We focused on two types of studies, namely: (1) trials in lymphoma, solid tumor or brain tumor patients in which an anti-cancer intervention was applied in a randomized fashion; and (2) any type of study with TRM as a primary or secondary outcome

Summary

Introduction

There is a lack of standardized definition for treatment-related mortality (TRM), which represents an important endpoint in cancer. Our objective was to describe TRM definitions used in studies of children, adolescents and young adults with lymphomas, solid tumors and brain tumors. Treatment-related mortality (TRM) is essential information for physicians involved in the care of children, adolescents and young adults with cancer. Outcomes for pediatric cancer have improved remarkably over time and the 5-year overall survival rate for childhood cancers currently exceeds 80% [1]. Appreciating TRM versus disease-related death is fundamental to understanding the best strategy to improve overall survival. If TRM is the primary cause of failure for a specific cancer, the strategy must focus on enhancing supportive care and/or using less toxic therapies [1]. Correct identification of TRM and diseaserelated mortality allows for appropriate monitoring of outcomes between trials and over time

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