Hematological toxicity of mTOR inhibitors is mild and dose-dependent in patients with tuberous sclerosis and subependymal giant cell astrocytoma

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Introduction: Everolimus is the primary therapeutic option for patients with tuberous sclerosis complex (TSC) and subependymal giant astrocytoma (SEGA). This study aimed to assess the effect of everolimus, a mammalian-target-of-rapamycin (mTOR) inhibitor, on complete blood count (CBC) parameters in patients with TSC and SEGA. Material and methods: The study included 18 pediatric patients with TSC-associated SEGA tumors as indications for everolimus treatment. During the standard dose therapy and maintenance phase (<50% of standard dose), CBC results and everolimus whole blood concentrations were collected at five study time points. Results: Everolimus contributed to decreasing almost all blood cell values. The most severe toxicity occurred six months after therapy commenced. Dose reduction resulted in slightly normalizing parameters, but they did not return to the initial ones in all cases. During the study, only a few instances of cytopenia were reported. The most common abnormality was granulocytopenia, observed in one in three patients. Almost all cytopenias occurred during the standard treatment phase, and none were classified as severe. Hematological toxicity was not the reason for therapy interruption or withdrawal. Conclusions: A decrease in almost all hematological parameters is widespread during everolimus therapy. Hematological toxicities are rare and mild. The unique abnormality for mTOR inhibitors is microcytosis. Reduction of mTOR inhibitor dose results in a lower frequency of hematological side effects.

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