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治疗性红细胞单采术与放血疗法用于遗传性血色素沉着症初始治疗的比较——一项试点研究

Therapeutic erythrocytapheresis versus phlebotomy in the initial treatment of hereditary hemochromatosis - A pilot study.

作者信息

Rombout-Sestrienkova Eva, van Noord Paulus A H, van Deursen Cees Th B M, Sybesma Bob J P H, Nillesen-Meertens Ans E L, Koek Ger H

机构信息

Sanquin Blood Bank Southeast Region, Geatano Martinolaan 95, 6229 GS Maastricht, The Netherlands.

出版信息

Transfus Apher Sci. 2007 Jun;36(3):261-7. doi: 10.1016/j.transci.2007.03.005. Epub 2007 Jun 13.

Abstract

Hereditary Hemochromatosis (HH) is a genetic disorder of iron metabolism, resulting in excessive iron overload. Currently, phlebotomy is the standard effective treatment that prevents progression of tissue damage. Aim of the therapy is to reach ferritin levels between 20 and 50mugl(-1). In patients with total iron stores of more than 30g, intensive treatment by means of weekly phlebotomies during 2-3 years is required to reach this aim. More recently mechanical removal of erythrocytes through therapeutic erythrocytapheresis (TE) has become a new therapeutic modality. By means of TE, up to 1000ml erythrocytes per session can be removed, depending on patient characteristics, compared to 250ml erythrocytes per phlebotomy. Thus, TE potentially offers a more efficient method of removing iron overload with less procedures in a shorter treatment period. In a pilot study between 2002 and 2005, results from a group of HH patients treated with TE (N=6) were compared to the results of a historical control group of HH patients (N=6) treated with phlebotomy. The results showed a reduction of almost 70% in both the total number and the duration of treatments in the TE group. Although, the procedure costs compared on the basis of a single TE session were higher, the total costs for the whole treatment were comparable or cheaper with the use of TE. Future prospective studies are needed to compare both therapies in a randomized setting.

摘要

遗传性血色素沉着症(HH)是一种铁代谢的遗传性疾病,会导致铁过度负荷。目前,放血疗法是预防组织损伤进展的标准有效治疗方法。该疗法的目标是使铁蛋白水平达到20至50μg/L。对于总铁储存量超过30g的患者,需要在2至3年内通过每周放血进行强化治疗才能达到这一目标。最近,通过治疗性红细胞单采术(TE)机械去除红细胞已成为一种新的治疗方式。通过TE,根据患者的具体情况,每次可去除多达1000ml红细胞,而每次放血只能去除250ml红细胞。因此,TE可能提供一种更有效的方法,在更短的治疗期内以更少的步骤去除铁过载。在2002年至2005年的一项试点研究中,将一组接受TE治疗的HH患者(N = 6)的结果与一组接受放血治疗的HH患者历史对照组(N = 6)的结果进行了比较。结果显示,TE组的治疗总数和治疗持续时间均减少了近70%。虽然基于单次TE治疗的程序成本较高,但使用TE进行整个治疗的总成本相当或更低。未来需要进行前瞻性研究,在随机环境中比较这两种疗法。

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