An expanded-access clinical study of thiotepa (DSP-1958) high-dose chemotherapy before autologous hematopoietic stem cell transplantation in patients with malignant lymphoma

Momoko Nishikori, Yasufumi Masaki, Nobuharu Fujii, Takashi Ikeda, Mariko Takahara-Matsubara, Saori Sugimoto, Eisei Kondo

Research output: Contribution to journalArticlepeer-review

Abstract

Thiotepa, an antineoplastic ethylenimine alkylating agent that can penetrate the central nervous system, was recently approved in Japan as high-dose chemotherapy prior to autologous hematopoietic stem cell transplantation (HSCT) for patients with malignant lymphoma. To further evaluate the safety and efficacy of thiotepa, a multicenter, open-label, non-comparative, expanded access program was undertaken in Japan, including a larger population of Asian patients with malignant lymphoma. Intravenous thiotepa (200 mg/m2/day) was administered over 2 h on days -4 and -3 before scheduled HSCT, plus intravenous busulfan (0.8 mg/kg) over 2 h every 6 h on days -8, -7, -6 and -5. In the safety analysis population (N = 51), 25 patients (49.0%) had primary central nervous system lymphomas. The most common treatment-emergent adverse event was febrile neutropenia (49/51 [96.1%]). No unexpected safety events were observed, and no event resulted in death or treatment modification. Forty-seven patients (92.2%) had engraftment (neutrophil count ≥ 500/mm3 for three consecutive days after bone-marrow suppression and HSCT). The survival rate at day 100 post-transplantation was 100%. These data confirm the safety of thiotepa prior to autologous HSCT for patients with malignant lymphoma. Trial registration: JapicCTI-173654.

Original languageEnglish
JournalInternational journal of hematology
DOIs
Publication statusAccepted/In press - 2021
Externally publishedYes

Keywords

  • Autologous
  • Hematopoietic stem cell transplantation
  • Lymphoma
  • Thiotepa
  • Transplantation

ASJC Scopus subject areas

  • Hematology

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