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Low concentrations of STI571 in the cerebrospinal fluid: a case report
Andreas L Petzer1, Eberhard Gunsilius, Michael Hayes
1Abteilung für Hämatologie & Onkologie, Universitätsklinik Innsbruck, Anichstrasse 35, A-6020 Innsbruck, Austria. andreas.petzer@uibk.ac.at
Insights
This study shows that STI571, a BCR-ABL inhibitor, achieved remission in chronic myeloid leukemia. However, low drug levels in cerebrospinal fluid led to central nervous system relapse.
Area of Science:
- Hematology
- Pharmacology
- Oncology
Background:
- Chronic myeloid leukemia (CML) is a myeloproliferative neoplasm.
- Philadelphia chromosome-positive (Ph+) CML is characterized by the BCR-ABL fusion gene.
- Tyrosine kinase inhibitors (TKIs) like STI571 target BCR-ABL activity.
Observation:
- A 53-year-old male patient with Ph+ CML in lymphoid blast crisis was treated with STI571.
- The patient achieved complete cytogenetic remission with STI571 at 600 mg/d.
- Despite sustained bone marrow remission, the patient experienced an isolated central nervous system (CNS) relapse.
Findings:
- Cerebrospinal fluid (CSF) analysis revealed significantly lower STI571 concentrations compared to plasma levels (2-log lower).
- This indicates poor penetration of orally administered STI571 into the human CNS.
- This is the first reported data on STI571 CSF penetration in humans.
Implications:
- Low CNS penetration of STI571 may contribute to isolated CNS relapses in CML patients.
- This finding highlights the need for CNS-directed therapies or alternative strategies in TKI-treated CML.
- Further research is warranted to optimize TKI delivery to the CNS for effective CML management.
Abstract:
We report a 53-year-old man with lymphoid blast crisis of Ph+ chronic myeloid leukaemia who was treated with STI571, a selective inhibitor of the enzymatic activity of BCR-ABL. He responded excellently to STI571 (600 mg/d), obtaining a complete cytogenetic remission after 3 months of therapy. Although remission in the bone marrow was sustained, the patient developed an isolated central nervous system relapse. Subsequent analyses of STI571 concentrations in the cerebrospinal fluid (CSF) revealed 2-log lower CSF levels of STI571 than corresponding plasma levels. These are the first data demonstrating a low penetration of orally administered STI571 into the CSF in humans.
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