Despite improvement in the long-term survival rate following pediatric acute myeloid leukemia (AML), the rate remains low, even with optimal treatment. The present study reports the long-term outcome of a small patient group treated with a single drug, high-dose chemotherapy (HDCT) with cytarabine, including consolidation and maintenance therapy. RT-PCR was conducted to assess 43 fusion genes, and after treatment, all cases have been followed up for 20 years (June 2002-December 2020). With an 80% 5-year survival rate, the results of this study highlight the possibility that pediatric AML can be reasonably effectively treated with relatively simple chemotherapy when necessary. HDCT is clinically safe, effective and relatively inexpensive. We propose that in the context of limited resources, HDCT should be considered as an alternative therapy for pediatric AML.

In spite of advancements being made in technology and treatment strategies, which have markedly improved the survival rate of patients, the cost of cancer care worldwide has increased over the past decades. The presence of several cost-effectiveness ratios has provided significant indexes to assess the balance between cost and effectiveness. However, the currently available indexes still fail to provide a comprehensive and objective evaluation of cancer treatment. Therefore, the present study developed a novel approach, namely a quantitative cost-effectiveness index of cancer treatment, based on the calculation of the hospitalization expense index and efficacy evaluation index. The present study used the data of 16 patients with childhood acute myeloid leukemia who received the high-dose chemotherapy as an example, and the quantitative cost-effectiveness index was used to evaluate the value of this approach. As the increasing prevalence of cancer and the rising cost of pharmaceuticals have contributed to the expenditure, the development of this index may help to solve the current dilemma of cancer treatment and may prove to be essential for the development of an effective approach which may be accessible to and affordable by common persons; thus would then lead to a higher cure rate.