Efficacy And Cost-Effectiveness Of Clofarabine With High-Dose Cytarabine For Treatment Of Relapsed/Refractory Acute Myeloid Leukemia (R/R Aml).

JOURNAL OF CLINICAL ONCOLOGY(2018)

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Abstract
e19009 Background: There is no standard reinduction regimen for adults with R/R AML. Clofarabine with high-dose cytarabine (Clo-HiDAC) is commonly used, but limited data exists to support its efficacy. Its cost-effectiveness over other non-clofarabine based regimens is also controversial. Methods: We performed a single-center, retrospective cohort study evaluating 71 adult patients with R/R AML treated with Clo-HiDAC (cytarabine dose ≥ 1 g/m2 x5 days) between July 2012 and June 2017. The primary outcome was complete remission (CR). Secondary outcomes included overall response rate (ORR; defined as CR + CR with incomplete neutrophil and/or platelet recovery), event free survival (EFS) and overall survival (OS). We analyzed the cost-effectiveness of this regimen based on wholesale drug acquisition costs. Results: Median age in this cohort was 54 years (range 21-75). Median number of prior lines of therapy was 1 (range 1-5), and 24% had prior allogeneic transplant. The CR rate was 30% and ORR was 42%. In responders, median duration of response was 18.8 months. For the overall cohort, median EFS was 1.4 months, and median OS was 12.2 months. There was no difference in CR, ORR, EFS, or OS by age ( < 55 vs ≥55 years), primary refractory vs relapsed disease, prior lines of therapy (1 vs > 1) or prior allogeneic transplant. Using wholesale drug acquisition costs, the cost per patient of Clo-HiDAC is $25,083 using generic clofarabine and $57,724 using brand name clofarabine. We compared the efficacy (based on literature review), cost per patient and cost per CR of common AML reinduction regimens and found Clo-HiDAC not to be cost-effective (Table). Conclusions: Our analysis found rates of CR and ORR (30% and 42% respectively) to be comparable to prior report. The poor cost effectiveness of Clo-HiDAC may limit its utility to patients who cannot receive anthracycline. Future work will include a retrospective review of patients receiving anthracycline-based reinduction. Regimen CR Rate Cost Cost/CR Clo-HiDAC (generic) 30% $25,083 $83,610 Clo-HiDAC (brand) 30% $57,724 $192,413 MEC 18-65%* $924 $1,422 – $5,133 FLAG-Ida 63%* $2,679 $4,252 CLAG-M 53%* $3,094 $5,838 *Based on literature review
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Key words
relapsed/refractory acute myeloid leukemia,acute myeloid leukemia,clofarabine,cost-effectiveness,high-dose
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