A pilot trial testing the feasibility of administering D-penicillamine to extremely low birth weight neonates
A pilot trial testing the feasibility of administering D-penicillamine to extremely low birth weight neonates
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DOI：
10.1038/sj.jp.7211440
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发表时间：
2006-02-01
影响因子：
2.9
通讯作者：
Baer, V. L.
中科院分区：
文献类型：
--
作者：
Christensen, R. D.;Alder, S. C.;Baer, V. L.
目的：我们肠内给予3-巯基-D-缬氨酸14天的疗程（D-青霉胺）治疗5名极低出生体重（ELBW）新生儿，作为评估该疗法作为降低早产儿视网膜病变（ROP）发病率或严重程度的一种手段的一步。通过鼻胃管给予研究药物（100 mg/ml），剂量为100 mg/k，每8小时一次，持续3天，然后每天一次，50 mg/k，持续11天。由床边护士保存日志，以记录可能的即刻不耐受体征。实验室检查评估肝、肾和血液学毒性。根据ICROP指南对ROP进行评分。比较与一个队列的139个连续最近的新生儿相同的出生体重和胎龄range.Results：5名新生儿参加了这项研究，并按计划接受研究药物的全过程。在所有受试者中均未观察到研究药物即刻不耐受的体征。研究患者的肌酐升高、血小板减少、中性粒细胞减少、高胆红素血症或肝功能检查异常的发生率并不高于队列组。五个没有ROP和一个开发短暂的阶段1，相比ROP的发生率为54%的cohol.Conclusions：肠内给药14天的3-巯基-D-缬氨酸ELBW新生儿和悬浮液似乎耐受性良好，是可行的。这些结果表明，可以进行II期安全性和初步疗效试验。
Objective: We enterally administered a 14-day course of 3-mercapto-D-valine (D-penicillamine) to five extremely low birth weight (ELBW) neonates, as a step toward assessing this therapy as a means of reducing the incidence or severity of retinopathy of prematurity (ROP).Methods: The study drug ( 100 mg/ml) was given by nasogastric tube at a dose of 100 mg/k every 8 h for three days, and then 50 mg/k once per day for 11 additional days. Logbooks were maintained by the bedside nurses to record signs of possible immediate intolerance. Laboratory tests assessed hepatic, renal, and hematologic toxicity. ROP was scored according to the ICROP guidelines. Comparisons were with a cohort of 139 consecutive recent neonates of the same birth weight and gestational age range.Results: Five neonates were enrolled in the study, and all received the full course of study drug as planned. Signs of immediate intolerance of the study drug were not observed in any. The study patients did not have a higher incidence, than that of the cohort group, in creatinine elevation, thrombocytopenia, neutropenia, hyperbilirubinemia, or abnormal liver function test. Four of the five had no ROP and one developed transient stage 1, compared with a 54% occurrence of ROP in the cohort.Conclusions: It is feasible to enterally administer a 14-day course of 3-mercapto-D-valine to ELBW neonates and the suspension appears to be well tolerated. These results suggest that phase II safety and preliminary efficacy trials can be undertaken.