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Improved outcome for Chinese children with acute promyelocytic leukemia: a comparison of two protocols
Xue-Qun Luo1, Zhi-Yong Ke, Li-Bin Huang
1Department of Pediatric, The First Affiliated Hospital of Sun Yat-Sen University, Zhongshan Er Lu, Guangzhou, China. l-xuequn@126.com
Insights
A modified PETHEMA protocol significantly improved event-free survival for childhood acute promyelocytic leukemia (APL) in developing countries. This less intensive treatment reduced toxicity and costs, improving patient outcomes.
Area of Science:
- Pediatric Oncology
- Hematology
- Clinical Research
Background:
- Acute promyelocytic leukemia (APL) is a highly curable malignancy.
- Treatment accessibility and outcomes for APL in developing countries remain suboptimal.
- Current treatment strategies may enhance outcomes for pediatric APL patients in resource-limited settings.
Purpose of the Study:
- To evaluate the effectiveness of a modified PETHEMA protocol for childhood APL in a developing country.
- To compare outcomes between an intensive in-house protocol and a less intensive modified PETHEMA protocol.
- To assess treatment-related toxicity, relapse rates, and hospitalization costs.
Main Methods:
- Diagnosis based on FAB classification and PML-RAR alpha rearrangement detection.
- Two treatment protocols were used: an intensive in-house protocol (high-dose AraC, anthracycline) and a modified PETHEMA LPA99 protocol.
- Patient data, including event-free survival (EFS), treatment failures, and costs, were analyzed.
Main Results:
- The modified PETHEMA protocol demonstrated a significantly higher 3.5-year EFS (79.6%) compared to the in-house protocol (37.5%).
- Treatment failures, including death from intracranial hemorrhage and sepsis, and relapses, were lower with the modified PETHEMA protocol.
- The modified PETHEMA protocol resulted in significantly lower sepsis rates (7.7% vs. 77.8%) and hospitalization costs (median US$4,700 vs. US$20,000).
Conclusions:
- A less intensive, modified PETHEMA LPA99-based protocol effectively treats childhood APL in developing countries.
- This approach reduces chemotherapy toxicity, sepsis, and hospitalization costs without increasing relapse rates.
- Improved outcomes, decreased treatment abandonment, and reduced morbidity are achievable with this modified protocol.
Objective:
Acute promyelocytic leukemia (APL) is now highly curable, except in many developing countries. Introduction of current treatment strategies may improve the outcome for children with APL in these countries.
Methods:
The diagnosis was based on the FAB classification and detection of PML-RAR alpha rearrangement. From December 1999 to September 2004, 16 eligible children were treated with an intensive in-house protocol including high-dose AraC and anthracycline. Subsequently, 14 cases were treated with a less intensive protocol modified from the PETHEMA LPA99.
Results:
The 3.5 years event-free survival (EFS) was 37.5% (95% CI, 13.8-61.2%) for patients treated on initial protocol. The treatment failures were: six patients abandoned treatment (37.5%), two who died of intracranial hemorrhage at diagnosis (6.3%) and sepsis in remission (6.3%) respectively, and two who relapsed (12.5%). Those treated on modified PETHEMA had a 3.5 years EFS of 79.6% (95% CI, 52.9-106.3%). Treatment failures included: one who died of intracranial hemorrhage at diagnosis (7.1%) and one who relapsed (7.1%). The patients on modified PETHEMA had a significantly higher EFS (P = 0.012), lower frequency of sepsis during treatment (7.7% vs. 77.8%; P = 0.0015), and lower hospitalization cost (median US$ 4,700 vs. US$ 20,000; P < 0.0001) than those on in-house protocol.
Conclusion:
Treatment with the less intensive protocol based on the PETHEMA LPA99 study of childhood APL successfully reduced chemotherapy toxicity and lowered hospitalization costs without increasing relapses. This led to decreases in treatment-related morbidity and the treatment abandonment rate, thus improving overall outcome.
