Comparison of the overall treatment outcome of two protocols for childhood acute promyelocytic leukemia / 白血病·淋巴瘤

ABSTRACT

Objective For further improving the prognosis of childhood acute promyelocytic leukemia (APL) in China,the treatment efficacies, outcomes and costs of protocols for childhood APL between in developed countries and in our hospital were compared. Methods 30 cases aged ＜15 years were diagnosed according to the FAB classification and detection of PML-RARα rearrangement. From December 1999 to September 2004,sixteen eligible children were treated with an intensive in-house protocol including high-dose Ara-C and anthracycline for post remission treatment. From September 2004 to January 2008,14 cases enrolled were treated with a less intensive protocol modified from the PETHEMA LPA99. Results The 3.5 years EFS was 37.5 % (s-x=0.121) for total 16 patients on in-house protocol. Six patients (37.5 %) abandoned treatment,2 died of intracranial hemorrhage at diagnosis (6.3 %) and sepsis in remission (6.3 %),respectively,and 2 relapsed (12.5 %). The 14 cases treated with modified PETHEMA had a 3.5 years EFS of 79.6 % (s-x=0.136). One died of intracranial hemorrhage at diagnosis (7.1 %) and 1 relapsed (7.1 %). 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,RMB 35 200 vs 150 000; P ＜0.0001) than those on in-house protocol. Conclusion Treatment with the less intensive protocol based on the PETHEMA LPA99 study for childhood APL successively reduced complication of chemotherapy and reduced hospitalization cost without increasing relapses, which led to decreases in treatment-related toxicity and treatment abandonment rate,thus improving overall outcome.