Baseline tumor size is an independent prognostic factor for overall survival in patients with melanoma treated with pembrolizumab

Richard W. Joseph, Jeroen Elassaiss-Schaap, Richard Kefford, Wen Jen Hwu, Jedd D. Wolchok, Anthony M. Joshua, Antoni Ribas, F. Stephen Hodi, Omid Hamid, Caroline Robert, Adil Daud, Roxana Dronca, Peter Hersey, Jeffrey S. Weber, Amita Patnaik, Dinesh P. De Alwis, Andrea Perrone, Jin Zhang, S. Peter Kang, Scot EbbinghausKeaven M. Anderson, Tara C. Gangadhar

Research output: Contribution to journalArticlepeer-review

110 Scopus citations


Purpose: The purpose of this study was to assess the association of baseline tumor size (BTS) with other baseline clinical factors and outcomes in pembrolizumab-treated patients with advanced melanoma in KEYNOTE-001 (NCT01295827). Experimental Design: BTS was quantified by adding the sum of the longest dimensions of all measurable baseline target lesions. BTS as a dichotomous and continuous variable was evaluated with other baseline factors using logistic regression for objective response rate (ORR) and Cox regression for overall survival (OS). Nominal P values with no multiplicity adjustment describe the strength of observed associations. Results: Per central review by RECIST v1.1, 583 of 655 patients had baseline measurable disease and were included in this post hoc analysis. Median BTS was 10.2 cm (range, 1-89.5). Larger median BTS was associated with Eastern Cooperative Oncology Group performance status 1, elevated lactate dehydrogenase (LDH), stage M1c disease, and liver metastases (with or without any other sites; all P ≤ 0.001). In univariate analyses, BTS below the median was associated with higher ORR (44% vs. 23%; P < 0.001) and improved OS (HR, 0.38; P < 0.001). In multivariate analyses, BTS below the median remained an independent prognostic marker of OS (P < 0.001) but not ORR. In 459 patients with available tumor programmed death ligand 1 (PD-L1) expression, BTS below the median and PD-L1-positive tumors were independently associated with higher ORR and longer OS. Conclusions: BTS is associated with many other baseline clinical factors but is also independently prognostic of survival in pembrolizumab-treated patients with advanced melanoma.

Original languageEnglish (US)
Pages (from-to)4960-4967
Number of pages8
JournalClinical Cancer Research
Issue number20
StatePublished - Oct 15 2018

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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