High-dose thiotepa, in conjunction with melphalan, followed by autologous hematopoietic stem cell transplantation in patients with pediatric solid tumors, including brain tumors - Takeaways - MDSpire

High-dose thiotepa, in conjunction with melphalan, followed by autologous hematopoietic stem cell transplantation in patients with pediatric solid tumors, including brain tumors

  • By

  • Junichi Hara

  • Kimikazu Matsumoto

  • Naoko Maeda

  • Mariko Takahara-Matsubara

  • Saori Sugimoto

  • Hiroaki Goto

  • November 3, 2022

  • 0 min

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  • 1

    Thiotepa is an antitumor alkylating agent used as high-dose chemotherapy before autologous HSCT for pediatric solid tumors.

  • 2

    The drug effectively crosses the blood-brain barrier, achieving high concentrations in the cerebrospinal fluid.

  • 3

    A recent study showed high survival rates of 77.8% for pediatric solid tumors and 100% for malignant lymphoma with thiotepa.

  • 4

    Thiotepa was approved in Japan for use as HDT before autologous HSCT for pediatric malignant solid tumors in 2019.

  • 5

    The expanded access program evaluated thiotepa's safety and efficacy in pediatric patients with solid tumors or brain tumors.

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