Triple intrathecal therapy without cranial irradiation for central nervous system preventive therapy in childhood

Wei-Ying Lin1, Hsi-Che Liu, Ting-Chi Yeh

  • 1Division of Pediatric Hematology and Oncology, Mackay Memorial Hospital, Taipei, Taiwan.

Insights

Triple intrathecal therapy (TIT) alone effectively prevents central nervous system (CNS) relapse in children with acute lymphoblastic leukemia (ALL). This approach avoids severe complications and offers high survival rates, making it a viable CNS preventive therapy (CNSP) option.

Area of Science:

  • Pediatric Oncology
  • Hematology
  • Neuro-oncology

Background:

  • Acute lymphoblastic leukemia (ALL) requires effective central nervous system (CNS) prophylaxis.
  • Traditional CNS preventive therapy (CNSP) methods can cause significant morbidities.
  • Evaluating alternative CNSP strategies is crucial for improving outcomes in pediatric ALL.

Purpose of the Study:

  • To assess the efficacy and safety of triple intrathecal therapy (TIT) as a standalone CNSP in children with ALL.
  • To determine the rate of CNS relapse in ALL patients treated with TIT alone.
  • To evaluate treatment-related complications associated with TIT.

Main Methods:

  • Retrospective cohort study of 59 pediatric ALL patients.
  • Patients classified into standard-risk (SR), high-risk (HR), and very-high-risk (VHR) groups.
  • Triple intrathecal therapy (TIT) comprising methotrexate, hydrocortisone, and cytarabine used for CNSP.

Main Results:

  • High 3-year event-free survival (89.4%) and overall survival (93.1%) rates observed.
  • No instances of CNS relapse occurred in patients treated with TIT alone.
  • TIT was well-tolerated, with no reported seizures, encephalitis, infections, or irradiation-like morbidities.

Conclusions:

  • Triple intrathecal therapy (TIT) alone is an effective and safe CNSP for most pediatric ALL patients.
  • TIT offers a promising alternative to cranial irradiation, minimizing treatment-related toxicities.
  • This approach supports favorable survival outcomes in pediatric ALL management.
Abstract