Homoharringtonine-Based Induction in Pediatric AML

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In a Chinese study (CCLG-AML 2015) reported in the Journal of Clinical Oncology, Li et al found that homoharringtonine (HHT)-based induction therapy was associated with promising outcomes in pediatric acute myeloid leukemia (AML).

As stated by the investigators, “HHT is commonly used for the treatment of Chinese adult AML, and all-trans retinoic acid (ATRA) has been verified in acute promyelocytic leukemia (APL). However, the efficacy and safety of HHT-based induction therapy have not been confirmed for childhood AML, and ATRA-based treatment has not been evaluated among patients with non-APL AML.”

Study Details

In the open-label multicenter trial, 1,253 newly diagnosed pediatric patients with AML were randomly assigned to receive a HHT-based induction regimen (n = 594) or an etoposide-based induction regimen (n = 659). Patients receiving maintenance therapy in the HHT group and etoposide group were randomly assigned to receive cytarabine-based (n = 165, n = 161) or ATRA-based (n =114, n = 124) maintenance therapy. The primary endpoint was complete remission rate after induction therapy.

Key Findings

Complete remission after induction was achieved in 79.9% of the HHT group vs 73.9% of the etoposide group (P = .014). In an intention-to-treat analysis, 3-year overall survival was 69.2% (95% confidence interval [CI] = 65.1%–72.9%) in the HHT group vs 62.8% (95% CI = 58.7%–66.6%) in the etoposide group (P = .025); 3-year event-free survival was 61.1% (95% CI = 56.8%–65.0%) vs 53.4% (95% CI = 49.2%–57.3%), respectively (P = .022).

In the per-protocol population of patients who received maintenance therapy, 3-year event-free survival did not significantly differ between groups: 70.7% with HHT plus ATRA-based maintenance vs 74.8% with HHT plus cytarabine-based maintenance (P = .933); and 66.2% with etoposide plus ATRA-based maintenance vs 72.9% with etoposide plus cytarabine-based maintenance (P = .336).

The investigators concluded, “HHT is an alternative combination regimen for childhood AML. The effects of ATRA-based maintenance are comparable with those of cytarabine-based maintenance therapy.”

Huyong Zheng, MD, PhD, of the Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology, Beijing Children’s Hospital, is the corresponding author for the Journal of Clinical Oncology article.

Disclosure: The study was supported by the National Natural Science Foundation of China, Beijing Natural Science Foundation, and others. For full disclosures of the study authors, visit

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