The depot formulation of liposomal cytarabine (DepoCyte) has proven to be useful as intrathecal (IT) treatment of neoplastic and lymphomatous meningitis. We report the results of compassionate use of DepoCyte in 6 patients diagnosed with acute leukemia (AL) and CNS involvement. Two patients had CNS involvement at diagnosis and the remaining 4 had CNS relapse. Three patients received DepoCyte as adjuvant therapy and achieved complete response whereas 2 out of the remaining 3 cases treated with DepoCyte as the only drug showed sustained response. The side effects were mild and manageable in all patients. These findings justify the development of clinical trials to evaluate the efficacy and safety of IT depot cytarabine in meningeal involvement of AL.
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Zhonghua Xue Ye Xue Za Zhi
December 2024
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin 300020, China Tianjin Institutes of Health Science, Tianjin 301600, China.
This case report presents a patient with pediatric acute myeloid leukemia (AML) with RUNX1∷MTG16, admitted to the Blood Disease Hospital of the Chinese Academy of Medical Sciences in October 2023. He was 13 years old, with a chief complaint of fatigue for 20 days. Bone marrow smear revealed 17.
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Drug Metabolism and Pharmacokinetics Research Department, Discovery Research Laboratories, Nippon Shinyaku Co., Ltd, Japan.
CPX-351 (NS-87; Vyxeos®) has a characteristic liposomal formulation and contains cytarabine and daunorubicin at a 5:1 molar ratio, which demonstrates synergistic activity in both in vitro and in vivo animal models. It has been approved in several countries for the treatment of newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Since there are very few Asian patients, especially Japanese adult and pediatric patients, only a small clinical study has been conducted in Japanese adult patients and no study in Japanese pediatric patients.
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Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
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Centre for Pharmacy, Department of Clinical Science, University of Bergen, Norway. Electronic address:
The backbone of induction therapy in acute myeloid leukaemia (AML) is to use an anthracycline in combination with cytarabine. Despite recent advances in AML therapy, this treatment remains the standard, and it has been largely unchanged for decades. There are few curative options for patients unfit for this treatment.
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Arkansas Laboratory, Office of Regulatory Science, Office of Regulatory Affairs, U.S. Food and Drug Administration, Jefferson, AR 72079, United States. Electronic address:
VYXEOS® is the first FDA-approved dual-API liposomal formulation containing two different chemotherapeutics, daunorubicin and cytarabine at a 1:5 molar ratio. Analysis of bulk formulation does not provide insight to size-based distribution of APIs and excipients, therefore asymmetrical flow field-flow fractionation (AF4) was utilized for the size-based separation of VYXEOS® liposomes and collected size fractions were further analyzed for the concentrations of APIs, lipid excipients, and copper. Analysis results revealed a significant variation in API distribution across the size fractions, with the larger liposomes encapsulating a higher ratio of cytarabine to daunorubicin compared to the smaller liposomes, while lipid excipient composition was held constant across the size range.
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