Evaluation of in vivo antineoplastic effects of rapamycin in patients with chemotherapy-refractory AML.
case_series · Level IV
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- Record sourced from PubMed, PMID 19892307.
- Also identified by DOI 10.1016/j.ejim.2009.09.007.
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Abstract
BACKGROUND: The mammalian target of rapamycin (mTOR) has recently been identified as a potential target in acute myeloid leukemia (AML). METHODS: We treated 5 patients with chemotherapy-refractory AML with the mTOR-inhibitor rapamycin at 2mg per os daily for 14 days, with dose adjustment allowed to reach a target serum rapamycin concentration of 10-20 ng/mL. Four of five patients received additional hydroxyurea at constant dose during treatment with rapamycin. RESULTS: Two patients achieved a leukocyte response, in one of them, a prolonged response was seen. In the other patients, blast counts remained stable or increased during rapamycin therapy. We did not observe severe hematologic or non-hematologic side effects of rapamycin. CONCLUSION: Rapamycin at 2mg per day acts mildly cytoreductive in a subgroup of patients with refractory AML. Higher doses and drug combinations may be required to obtain long lasting anti-leukemic effects in these patients.