Pui C H, Mahmoud H H, Wiley J M, Woods G M, Leverger G, Camitta B, Hastings C, Blaney S M, Relling M V, Reaman G H
St Jude Children's Research Hospital, Memphis, TN 38105, USA.
J Clin Oncol. 2001 Feb 1;19(3):697-704. doi: 10.1200/JCO.2001.19.3.697.
To improve the control of hyperuricemia in patients with leukemia or lymphoma, we tested a newly developed uricolytic agent, recombinant urate oxidase (SR29142; Rasburicase; Sanofi-Synthelabo, Inc, Paris, France), which catalyzes the oxidation of uric acid to allantoin, a highly water-soluble metabolite readily excreted by the kidneys.
We administered Rasburicase intravenously, at 0.15 or 0.20 mg/kg, for 5 to 7 consecutive days to 131 children, adolescents, and young adults with newly diagnosed leukemia or lymphoma, who either presented with abnormally high plasma uric acid concentrations or had large tumor cell burdens. Blood levels of uric acid, creatinine, phosphorus, and potassium were measured daily. The pharmacokinetics of Rasburicase, the urinary excretion rate of allantoin, and antibodies to Rasburicase were also studied.
At either dosage, the recombinant enzyme produced a rapid and sharp decrease in plasma uric acid concentrations in all patients. The median level decreased by 4 hours after treatment, from 9.7 to 1 mg/dL (P =.0001), in the 65 patients who presented with hyperuricemia, and from 4.3 to 0.5 mg/dL (P =.0001) in the remaining 66 patients. Despite cytoreductive chemotherapy, plasma uric acid concentrations remained low throughout the treatment (daily median level, 0.5 mg/dL). The urinary excretion rate of allantoin increased during Rasburicase treatment, peaking on day 3. Serum phosphorus concentrations did not change significantly during the first 3 days of treatment, decreased significantly by day 4 in patients presenting with hyperuricemia (P =.0003), and fell within the normal range in all patients by 48 hours after treatment. Serum creatinine levels decreased significantly after 1 day of treatment in patients with or without hyperuricemia at diagnosis (P =.0003 and P =.02, respectively) and returned to normal range in all patients by day 6 of treatment. Toxicity was negligible, and none of the patients required dialysis. The mean plasma half-lives of the agent were 16.0 +/- 6.3 (SD) hours and 21.1 +/- 12.0 hours, respectively, in patients treated at dosages of 0.15 or 0.20 mg/kg. Seventeen of the 121 assessable patients developed antibodies to the enzyme.
Rasburicase is safe and highly effective for the prophylaxis or treatment of hyperuricemia in patients with leukemia or lymphoma.
为改善白血病或淋巴瘤患者高尿酸血症的控制情况,我们对一种新研发的尿酸分解剂重组尿酸氧化酶(SR29142;拉布立酶;赛诺菲-圣德拉堡公司,法国巴黎)进行了测试,该酶可催化尿酸氧化为尿囊素,尿囊素是一种极易溶于水的代谢产物,可通过肾脏轻易排出。
我们对131名新诊断的白血病、淋巴瘤儿童、青少年及年轻成人静脉注射拉布立酶,剂量为0.15或0.20mg/kg,连续给药5至7天,这些患者要么血浆尿酸浓度异常升高,要么肿瘤细胞负荷量大。每天测量血液中的尿酸、肌酐、磷和钾水平。还研究了拉布立酶的药代动力学、尿囊素的尿排泄率以及针对拉布立酶的抗体。
两种剂量下,重组酶均使所有患者的血浆尿酸浓度迅速大幅下降。在65名高尿酸血症患者中,治疗后4小时中位数水平从9.7降至1mg/dL(P = 0.0001);在其余66名患者中,从4.3降至0.5mg/dL(P = 0.0001)。尽管进行了细胞减灭化疗,但整个治疗期间血浆尿酸浓度一直保持在低水平(每日中位数水平为0.5mg/dL)。拉布立酶治疗期间尿囊素的尿排泄率增加,在第3天达到峰值。治疗的前3天血清磷浓度无显著变化,高尿酸血症患者在第4天显著下降(P = 0.0003),所有患者在治疗后48小时内降至正常范围。诊断时无论有无高尿酸血症的患者,治疗1天后血清肌酐水平均显著下降(分别为P = 0.0003和P = 0.02),所有患者在治疗第6天恢复至正常范围。毒性可忽略不计,无一例患者需要透析。在接受0.15或0.20mg/kg剂量治疗的患者中,该药物的平均血浆半衰期分别为16.0±6.3(标准差)小时和21.1±12.0小时。121名可评估患者中有17名产生了针对该酶的抗体。
拉布立酶对于白血病或淋巴瘤患者预防或治疗高尿酸血症安全且高效。