Pembrolizumab in relapsed and refractory mycosis fungoides and Sézary syndrome: A multicenter phase II study

Michael S. Khodadoust, Alain H. Rook, Pierluigi Porcu, Francine Foss, Alison J. Moskowitz, Andrei Shustov, Satish Shanbhag, Lubomir Sokol, Steven P. Fling, Nirasha Ramchurren, Robert Pierce, Asa Davis, Richard Shine, Shufeng Li, Sophia Fong, Jinah Kim, Yi Yang, Wendy M. Blumenschein, Jennifer H. Yearley, Biswajit DasRajesh Patidar, Vivekananda Datta, Erin Cantu, Justine N. McCutcheon, Chris Karlovich, P. Mickey Williams, Priyanka B. Subrahmanyam, Holden T. Maecker, Steven M. Horwitz, Elad Sharon, Holbrook E. Kohrt, Martin A. Cheever, Youn H. Kim

Research output: Contribution to journalArticlepeer-review

49 Scopus citations


PURPOSE To assess the efficacy of pembrolizumab in patients with advanced relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS). PATIENTS AND METHODS CITN-10 is a single-arm, multicenter phase II trial of 24 patients with advanced MF or SS. Patients were treated with pembrolizumab 2 mg/kg every 3 weeks for up to 24 months. The primary end point was overall response rate by consensus global response criteria. RESULTS Patients had advanced-stage disease (23 of 24 with stage IIB to IV MF/SS) and were heavily pretreated with a median of four prior systemic therapies. The overall response rate was 38% with two complete responses and seven partial responses. Of the nine responding patients, six had 90% or more improvement in skin disease by modified Severity Weighted Assessment Tool, and eight had ongoing responses at last follow-up. The median duration of response was not reached, with a median response follow-up time of 58 weeks. Immune-related adverse events led to treatment discontinuation in four patients. A transient worsening of erythroderma and pruritus occurred in 53% of patients with SS. This cutaneous flare reaction did not result in treatment discontinuation for any patient. The flare reaction correlated with high PD-1 expression on Sézary cells but did not associate with subsequent clinical responses or lack of response. Treatment responses did not correlate with expression of PD-L1, total mutation burden, or an interferon-g gene expression signature. CONCLUSION Pembrolizumab demonstrated significant antitumor activity with durable responses and a favorable safety profile in patients with advanced MF/SS.

Original languageEnglish (US)
Pages (from-to)20-28
Number of pages9
JournalJournal of Clinical Oncology
Issue number1
StatePublished - 2020

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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