Extended interval between enzyme therapy infusions for adult patients with Gaucher's disease type 1.
Extended interval between enzyme therapy infusions for adult patients with Gaucher's disease type 1.
复制标题
1 型戈谢病成年患者的酶治疗输注间隔时间延长。
DOI：
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发表时间：
2003
影响因子：
1.6
通讯作者：
M. Pocovi
中科院分区：
文献类型：
--
作者：
J. I. Pérez;Pilar Giraldo;Gregory M. Pastores;M. Fernandez;G. Martín‐Nuñez;M. Pocovi
背景
酶替代疗法(ERT)是一种有效、耐受性好、安全的治疗高雪氏病的方法。然而，费用方面的考虑、对医疗设施的访问、可能的生命周期等都是一部分接受治疗的患者所关心的主要问题，在某些情况下，这些问题已导致停止治疗。
目标
为了阐明将两次酶注射之间的间隔延长到每三周一次是否同样有效地维持了双月方案的临床反应。
材料和方法
对ERT有最佳反应的4名患者(平均27个月，每两周30个单位/公斤)接受了酶剂量/频率的改变，基本上构成了治疗期间累积剂量的减少。每6个月评估一次患者的血液学参数、血浆几丁糖苷酶水平、肝和脾大小以及骨骼症状的变化。
结果
所有患者都必须恢复之前每两周一次的输液计划；一名患者因新的骨髓浸润性病变，两名患者因内脏增大，第四名患者因进行性贫血。
结论
这一有限的经验表明，随着两次输液间隔的延长，酶剂量的减少可能会导致不同的疾病控制，并强调了酶疗法个体化的必要性。
BACKGROUND
Enzyme replacement therapy (ERT) for Gaucher's disease with alglucerase or imiglucerase is efficacious, well-tolerated and safe. However, cost considerations, visits to medical facilities, potentially duration of theray for life, are issues of major concern to a proportion of treated patients and has, in some cases, led to the withdrawal of therapy.
AIMS
To elucidate whether an extension of the interval between enzyme infusions to once every three weeks is as effective in maintaining the clinical responses achieved with the bi-monthly regimen.
MATERIALS AND METHODS
Four patients with an optimal response to ERT (at 30 units/kg every two weeks for an average of 27 months), were subjected to enzyme dose/frequency changes that essentially constituted a reduction in cumulative dose over the treatment period. Patients were assessed every 6 months for alterations in haematological parameters, plasma chitotriosidase levels, liver and spleen size, and bone symptoms.
RESULTS
All patients had to resume the previous infusion schedule of once every two weeks; one because of new bone marrow infiltrates, two because of visceral enlargement, and the fourth due to progressive anaemia.
CONCLUSIONS
This limited experience suggests that a reduction in enzyme dose associated with an extended interval between infusions may lead to variable disease control, and underscores the need for individualization of enzyme therapy.
戈谢病 1 型酶疗法：33 名患者治疗 6 至 24 个月的剂量疗效和不良反应。
影响因子：
20.3
作者：
Pastores,GM;Sibille,AR;Grabowski,GA
通讯作者：
Grabowski,GA