Multicenter phase II trial of enzastaurin in patients with relapsed or refractory advanced cutaneous T-cell lymphoma LEUKEMIA & LYMPHOMA Querfeld, C., Kuzel, T. M., Kim, Y. H., Porcu, P., Duvic, M., Musiek, A., Rook, A. H., Mark, L. A., Pinter-Brown, L., Hamid, O., Lin, B., Bian, Y., Boye, M., Day, J. M., Rosen, S. T. 2011; 52 (8): 1474-1480


This multicenter, single-arm, open-label non-randomized phase II trial (NCT00744991) was conducted in patients with recurrent/refractory mycosis fungoides (MF), stage IB-IVB, or Sézary syndrome (SS). A Simon two-stage design required 25 patients enrolled in stage 1 with =7 confirmed objective responses for expansion into stage 2. Patients were treated with oral enzastaurin (250?mg twice daily) until disease progression or intolerable toxicity. The primary endpoint was investigator-assessed response rate; secondary endpoints were time to objective response, response duration, time-to-progression, patient-reported pruritus, and safety/tolerability. Twenty-five patients were enrolled. A partial response was observed in one patient with MF. Median time-to-progression was 78 and 44 days in MF and SS, respectively. Self-reported pruritus relief and improved composite pruritus-specific symptom scores were documented in six and four patients, respectively. Enzastaurin was well tolerated with mostly grade 1-2 adverse events, mainly diarrhea and fatigue. There were two adverse event-related drug discontinuations with one possibly treatment-related.

View details for DOI 10.3109/10428194.2011.572265

View details for Web of Science ID 000292747300011

View details for PubMedID 21649541