ITCC 020/I-BFM 2009-02

Summary

Clofarabine, cytarabine, and liposomal daunorubicin in relapsed AML

Survival for children with acute myeloid leukemia (AML) whose disease has returned (relapsed) or does not respond sufficiently to treatment (refractory) remains limited. At the Princess Máxima Center, we are therefore studying new treatment strategies to improve outcomes.

In this international study, we evaluated a modified combination of chemotherapy for children with relapsed AML. In the standard regimen, we replaced fludarabine with clofarabine, in combination with cytarabine and liposomal daunorubicin.

High response rates

The new combination regimen was well tolerated and showed high response rates. The overall response rate was 68% (n=34). This is relevant because the study included children with a second relapse, a refractory first relapse, or an early first relapse – groups with an unfavorable prognosis.

Among children treated with the recommended dose of clofarabine in combination with cytarabine and liposomal daunorubicin, the overall response rate was 80% (n=10). Several children who did not respond to the standard regimen for relapsed AML achieved a response with this schedule.

The main side effects were bone marrow suppression and infections. Given the intensity of this reinduction chemotherapy, we recommend screening for subclinical fungal infections in advance.

Based on these results, clofarabine has been incorporated into first-line treatment for newly diagnosed AML within the AML-BFM 2012 protocol. At the Máxima Center, we now use this treatment block as part of reinduction therapy for children with relapsed or refractory AML.

Would you like to read the scientific publication? Please look here: Van Eijkelenburg NKA, et al. Clofarabine, high-dose cytarabine and liposomal daunorubicin in pediatric relapsed/refractory acute myeloid leukemia: a phase IB study. Haematologica. 2018 Sep;103(9):1484-1492.