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Temozolomide in resistant or relapsed pediatric solid tumors.

L De Sio1, G M Milano, A Castellano

  • 1Division and Laboratory of Oncology, Department of Pediatric of Pediatric Oncology Oncology-Haematology and Blood Bank Service, Bambino Gesù Children's Hospital, Rome, Italy.

Pediatric Blood & Cancer
|July 28, 2005
PubMed
Summary

Temozolomide (TMZ) showed activity in pediatric solid tumors that relapsed or were resistant to treatment. This oral chemotherapy was well-tolerated, with notable responses in neuroblastoma and CNS tumors.

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Area of Science:

  • Pediatric Oncology
  • Medical Chemotherapy
  • Clinical Trial Research

Background:

  • Relapsed or refractory solid tumors in children present significant treatment challenges.
  • Standard chemotherapy regimens often have limited efficacy in recurrent or resistant pediatric cancers.
  • Investigating novel therapeutic agents is crucial for improving outcomes in pediatric oncology.

Purpose of the Study:

  • To evaluate the efficacy and safety of temozolomide (TMZ) as a single agent in pediatric patients with relapsed or resistant solid tumors.
  • To determine response rates, survival, and toxicity profiles of TMZ in this patient population.
  • To explore potential correlations between treatment outcomes and patient/tumor characteristics.

Main Methods:

  • A cohort of 52 pediatric patients with various relapsed/resistant solid tumors received temozolomide (TMZ) at specific doses.

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  • Patients received prior treatments including craniospinal irradiation (CSI) or autologous bone marrow transplantation (ABMT).
  • Treatment involved two outpatient courses of TMZ, with a median of 4.8 courses administered per patient.

    • Main Results:

    • An objective response rate (CR+PR+MR) of 13.4% was observed, with 38.4% achieving stable disease (SD) and 48% progressing (PD).
    • Median survival was 7.8 months, and median time to progression was 3.4 months, influenced by histology and prior nitrosourea exposure.
    • Grade 3-4 hematological toxicity (primarily thrombocytopenia) occurred in 21.4% of courses; nausea and respiratory distress were less common.

    Conclusions:

    • Oral temozolomide (TMZ) is well-tolerated in pediatric patients with relapsed or resistant solid tumors.
    • TMZ demonstrated clinical activity, particularly in neuroblastoma (NB) and central nervous system (CNS) tumors refractory to standard chemotherapy.
    • These findings support the potential role of TMZ in specific pediatric solid tumor settings.