Improving methodology to go beyond histology in rare cancers

摘要

In The Lancet Oncology, Gary Schwartz and colleagues1 report the findings from a phase 2 trial assessing the combination of an mTOR inhibitor (temsirolimus) and a monoclonal antibody that inhibits insulin-like growth factor 1 receptor (IGF-1R; cixutumumab) in patients with advanced, previously treated soft-tissue and bone sarcomas of any type. 162 patients were allocated on the basis of IGF-1R expression by immunohistochemistry to one of three groups: IGF-lR-positive soft-tissue sarcoma (n=54), IGF-lR-positive bone sarcoma (n=54), or IGF-1R-negative bone and soft-tissue sarcoma (n=54). The proportion of patients who were progression free at 12 weeks (primary endpoint) was between 31% and 39% in all groups, so that the study was positive as per protocol. Should clinicians thus conclude that this combination is promising in all soft-tissue and bone sarcomas?