Reliability and efficacy of palifermin in prevention and management of oral mucositis in patients with acute lymphoblastic leukemia: a randomized, double-blind controlled clinical trial.
Reliability and efficacy of palifermin in prevention and management of oral mucositis in patients with acute lymphoblastic leukemia: a randomized, double-blind controlled clinical trial.
复制标题
帕利弗明预防和治疗急性淋巴细胞白血病患者口腔粘膜炎的可靠性和有效性：一项随机、双盲对照临床试验。
作者：
A. Lucchese;G. Matarese;M. Manuelli;C. Ciuffreda;L. Bassani;G. Isola;G. Cordasco;E. Gherlone
背景
清髓和造血干细胞移植治疗(HSCT)是口腔黏膜炎(OM)的一种副作用，目前尚无有效的治疗方法。这项随机对照试验分析了帕利福明在HSCT治疗期间作为一级预防药物对儿童急性淋巴细胞白血病(ALL)患者的疗效。
方法
本文分析了46例9~15岁B细胞急性淋巴细胞白血病(B-ALL)患者的临床资料。患者接受清髓方案条件下的同种异基因造血干细胞移植。随后，随机将帕利福明组患者分配给帕利福明，60 mg/kg，在移植预适应方案周期前3天和后3天单次静脉注射。对照组患者仅接受安慰剂治疗。评估OM的最大严重程度、溃疡性OM的发生率和持续时间、严重OM限制的发生率和持续时间。
结果
研究发现，与对照组相比，帕利福明组3级以上的OM发生率有统计学意义的降低。在60天后，帕利福明组的粘膜炎严重程度也得到了证实，平均为1.54分，对照组和阿片类止痛药的使用情况为2.16分。
结论
本研究表明，在HSTC治疗中单次应用派利福明可预防儿童急性淋巴细胞白血病患者的严重OM，作为二级预防可预防既往有黏膜损伤的高危患者的严重OM的复发，并改善儿童ALL患者的生活质量。
BACKGROUND
Myeloablative and hematopoietic stem cells transplantation therapy (HSCT) often acts as side-effect to oral mucositis (OM) with no effective treatment. This randomized-controlled trial analyzed the efficacy of palifermin, administered as a dose during HSCT therapy, as primary prophylaxis on pediatric patients with acute lymphoblastic leukemia (ALL).
METHODS
In this study forty-six patients (9-15 years) with B-cell acute lymphoblastic leukemia (B-ALL) were analyzed. The patients underwent allogenic HSCT conditioned by myeloablative regimen. Subsequently to randomization, patients in the palifermin group were assigned to receive palifermin, 60 mg/kg, intravenously as a single dose 3 days before and after transplant conditioning regimen cycle. The patients in the Control group received only a placebo treatment. Maximum severity of OM, incidence and duration of ulcerative OM, incidence and duration of severe OM limitations were evaluated.
RESULTS
A statistically significant reduction in the incidence of OM up to grade 3 in the palifermin group compared to the control group was discovered. There was also a reduction, confirmed at 60 days, in the degree of severity of mucositis in the palifermin group, with an average of 1.54 grade in the palifermin group, and of 2.16 in the Control group and in the use of opioid analgesics.
CONCLUSIONS
This study indicates that a single dose of palifermin used as primary prophylaxis during HSTC therapy can prevent severe OM in pediatric patients with ALL and used as secondary prophylaxis can prevent the recurrence of severe OM in high-risk patients with previous mucosal injury and improves the quality of life in pediatric patients with ALL.