Toxicity and efficacy of intrathecal liposomal cytarabine in children with leukemia/lymphoma relapsing in the central nervous system: a retrospective multicenter study

Abstract

The toxicity and efficacy of intrathecal liposomal cytarabine (LC) were evaluated in children with central nervous system (CNS) relapsed/refractory acute leukemia/lymphoma. Thirty patients (male:female ratio 21:9; median age 9.4 years) with CNS relapsed/resistant disease were treated with intrathecal LC at dosages adjusted for age. Twenty-seven (90%) patients simultaneously received systemic chemotherapy, including concurrent high-dose cytarabine or methotrexate in 21 (70%) cases. Of 28 patients evaluable for response, 25 patients (89%) achieved CNS complete remission and three (11%) partial remission. The median number of intrathecal LC administrations per patient was 4. The cerebrospinal fluid was cleared after a median of 3 intrathecal LC administrations. Neurological toxicity ≥ grade 3 occurred in four (13%) patients. No permanent sequelae were observed. The median overall survival was 20.9 months and the 5-year probability of survival was 46%. These encouraging data suggest that intrathecal LC is well tolerated and effective in children with relapsed/refractory CNS leukemia/lymphoma.

Keywords::

• Intrathecal liposomal cytarabine
• pediatric acute leukemia
• childhood hematological malignancies
• CNS relapse
• neurological adverse events

Acknowledgements

The authors are grateful to Dr. Massimo Di Maio, MD, Clinical Trial Unit, National Cancer Institute of Naples, Italy, for the statistical analysis.

We thank Gaia Sepe, Data Manager of the Pediatric Hemato-Oncology Department, Pausilipon Hospital, Naples, for data collection and Dr. Alessandra Trocino, Librarian of the National Cancer Institute of Naples, Italy, for bibliographic assistance.

The study was presented in part at the 17th Congress of the European Hematology Association (EHA), 15–18 June 2012, Amsterdam, The Netherlands.

Potential conflict of interest:

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