Results from a multicenter, open-label, pivotal phase II study of chidamide in relapsed or refractory peripheral T-cell lymphoma

Ann Oncol. 2015 Aug;26(8):1766-71. doi: 10.1093/annonc/mdv237. Epub 2015 Jun 23.


Background: Chidamide is a novel benzamide type of subtype-selective histone deacetylase (HDAC) inhibitor with unique mechanisms of action compared with marketed HDAC inhibitors. This phase II study was to evaluate the efficacy and safety of chidamide in relapsed or refractory peripheral T-cell lymphoma (PTCL) in Chinese population.

Patients and methods: Patients with relapsed or refractory PTCL of different subtypes received chidamide of 30 mg orally twice per week. The primary end point was overall response rate (ORR). Responding patients should be confirmed at least 4 weeks after the criteria of the response were first met, and were reviewed by an independent review committee.

Results: Eighty-three patients were enrolled and 79 patients with eligible PTCL histology were for efficacy assessments. Patients enrolled over 10% were with subtypes of PTCL not otherwise specified (34%), anaplastic large-cell lymphoma (22%), extranodal natural killer (NK)/T-cell lymphoma, nasal type (20%), or angioimmunoblastic T-cell lymphoma (AITL, 13%). The ORR was 28% (22 of 79) including 14% (11 of 79) with complete response/unconfirmed complete response (CR/CRu). Median progression-free survival and overall survival were 2.1 and 21.4 months, respectively. AITL patients tended to have higher ORR (50%) and CR/CRu rate (40%), as well as more durable responses, to chidamide treatment. Most adverse events (AEs) were grade 1 or 2, and AEs ≥grade 3 that occurred in ≥10% patients were thrombocytopenia (22%), leucopenia (13%) and neutropenia (11%), respectively.

Conclusion: Chidamide represents a novel oral benzamide class of HDAC inhibitor with significant single-agent activity and manageable toxicity in relapsed or refractory PTCL, and provides a much needed treatment option in this indication in China. Results led to China Food and Drug Administration approval of chidamide in this indication.

Keywords: HDAC inhibitor; T-cell lymphoma; chidamide; epigenetic; pivotal trial.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aminopyridines / therapeutic use*
  • Antineoplastic Agents / therapeutic use*
  • Benzamides / therapeutic use*
  • Disease-Free Survival
  • Enteropathy-Associated T-Cell Lymphoma / drug therapy
  • Female
  • Histone Deacetylase Inhibitors / therapeutic use*
  • Humans
  • Leukopenia / chemically induced
  • Lymphoma, Extranodal NK-T-Cell / drug therapy
  • Lymphoma, Large-Cell, Anaplastic / drug therapy
  • Lymphoma, T-Cell, Cutaneous / drug therapy
  • Lymphoma, T-Cell, Peripheral / drug therapy*
  • Male
  • Middle Aged
  • Mycosis Fungoides / drug therapy
  • Neoplasm Recurrence, Local / drug therapy*
  • Neutropenia / chemically induced
  • Skin Neoplasms / drug therapy
  • Thrombocytopenia / chemically induced
  • Treatment Outcome
  • Young Adult


  • Aminopyridines
  • Antineoplastic Agents
  • Benzamides
  • Histone Deacetylase Inhibitors
  • N-(2-amino-5-fluorobenzyl)-4-(N-(pyridine-3-acrylyl)aminomethyl)benzamide