Multicenter phase 1/2 study of forodesine in patients with relapsed peripheral T cell lymphoma

Dai Maruyama, Kunihiro Tsukasaki, Toshiki Uchida, Yoshinobu Maeda, Hirohiko Shibayama, Hirokazu Nagai, Mitsutoshi Kurosawa, Yoko Suehiro, Kiyohiko Hatake, Kiyoshi Ando, Isao Yoshida, Michihiro Hidaka, Tohru Murayama, Yoko Okitsu, Norifumi Tsukamoto, Masafumi Taniwaki, Junji Suzumiya, Kazuo Tamura, Takahiro Yamauchi, Ryuzo UedaKensei Tobinai

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25 Citations (Scopus)


Peripheral T cell lymphomas are an aggressive group of non-Hodgkin lymphomas with poor outcomes for most subtypes and no accepted standard of care for relapsed patients. This study evaluated the efficacy and safety of forodesine, a novel purine nucleoside phosphorylase inhibitor, in patients with relapsed peripheral T cell lymphomas. Patients with histologically confirmed disease, progression after ≥ 1 prior treatment, and an objective response to last treatment received oral forodesine 300 mg twice-daily. The primary endpoint was objective response rate (ORR). Secondary endpoints included duration of response, progression-free survival (PFS), overall survival (OS), and safety. Forty-eight patients (median age, 69.5 years; median of 2 prior treatments) received forodesine. In phase 1 (n = 3 evaluable), no dose-limiting toxicity was observed during the first 28 days of forodesine treatment. In phase 2 (n = 41 evaluable), the ORR for the primary and final analyses was 22% (90% CI 12–35%) and 25% (90% CI 14–38%), respectively, including four complete responses (10%). Median PFS and OS were 1.9 and 15.6 months, respectively. The most common grade 3/4 adverse events were lymphopenia (96%), leukopenia (42%), and neutropenia (35%). Dose reduction and discontinuation due to adverse events were uncommon. Secondary B cell lymphoma developed in five patients, of whom four were positive for Epstein-Barr virus. In conclusion, forodesine has single-agent activity within the range of approved therapies in relapsed peripheral T cell lymphomas, with a manageable safety profile, and may represent a viable treatment option for this difficult-to-treat population.

Original languageEnglish
Pages (from-to)131-142
Number of pages12
JournalAnnals of Hematology
Issue number1
Publication statusPublished - Jan 30 2019


  • Forodesine
  • Overall response rate
  • Peripheral T cell lymphoma
  • Progression-free survival
  • Purine nucleoside phosphorylase inhibitor

ASJC Scopus subject areas

  • Hematology


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