Seeff L B, Koff R S
Adv Intern Med. 1984;29:109-45.
As is apparent from the foregoing, there is only one etiologic variant of CAH for which specific therapy is available, namely, that associated with Wilson's disease. However, Wilson's disease is responsible for only a minute portion of the total cases of CAH. Drug-induced CAH also seems highly responsive to appropriate management--in this instance, removal of an offending agent rather than administration of a therapeutic drug. However, as stated, it too is an infrequent contributor to the CAH pool. Among the remaining forms of CAH, a reasonably consistent response to treatment can be expected only from patients with autoimmune CAH. This entity is a serious disorder with unequivocally high morbidity and mortality and thus clearly warrants treatment. Despite the considerable side effects that invariably result from the long-term use of corticosteroids--the only available, although nonspecific, form of treatment--corticosteroid use is justified and indeed recommended. Current evidence, derived from the Mayo Clinic data, suggests that the best therapeutic approach is to use both corticosteroids and azathioprine, a combination that offers the highest therapeutic index with the lowest rate of side effects. Using this regimen, complete remission is reported to result in 65% of cases within 2-3 years, although a considerable proportion of these individuals relapse and require retreatment. Much publicity has surrounded the Mayo Clinic and Royal Free Hospital treatment trials; however, it is probable that autoimmune CAH represents far less than 20% of all cases, and that severe disease requiring corticosteroid therapy comprises but a minor fraction of these. Thus, the bulk of cases of CAH in the United States occur in patients with either established or inferred viral-related disease, the group for which clearly effective therapy is not yet available. Most of these persons are asymptomatic, their disease having been detected through routine screening programs or at the time of evaluation of other disorders. Much interest is evoked, at present, by the new experimental forms of treatment, but none has proved to be consistently effective, and for some, toxicity is high. All appear to reduce levels of replicating virus, but none clearly affects HBsAg or disease activity. Research in this area continues, with highest expectations of success for the use of combination therapy.(ABSTRACT TRUNCATED AT 400 WORDS)
从上述内容可以明显看出,对于先天性肝纤维化(CAH),只有一种病因变体有可用的特异性治疗方法,即与威尔逊氏病相关的那种。然而,威尔逊氏病仅占CAH总病例的极小部分。药物性CAH似乎对适当的管理也有很高的反应性——在这种情况下,是去除致病因素而不是给予治疗药物。然而,如前所述,它在CAH病例中也是一个不常见的原因。在其余类型的CAH中,只有自身免疫性CAH患者对治疗有望出现较为一致的反应。这种疾病是一种严重的病症,发病率和死亡率明确很高,因此显然需要治疗。尽管长期使用皮质类固醇(唯一可用的、尽管是非特异性的治疗形式)总会产生相当多的副作用,但使用皮质类固醇是合理的,实际上也是推荐的。来自梅奥诊所数据的当前证据表明,最佳治疗方法是同时使用皮质类固醇和硫唑嘌呤,这种联合用药能提供最高的治疗指数且副作用发生率最低。采用这种治疗方案,据报道65%的病例在2至3年内实现完全缓解,尽管这些患者中有相当一部分会复发并需要再次治疗。梅奥诊所和皇家自由医院的治疗试验备受关注;然而,自身免疫性CAH可能占所有病例的比例远不到20%,而需要皮质类固醇治疗的严重疾病在这些病例中只占一小部分。因此,美国的大部分CAH病例发生在患有已确诊或推测与病毒相关疾病的患者中,而对于这一群体,显然还没有有效的治疗方法。这些人中大多数没有症状,他们的疾病是通过常规筛查项目或在评估其他疾病时被发现的。目前,新的实验性治疗形式引起了很大兴趣,但没有一种被证明始终有效,而且有些毒性很高。所有这些似乎都能降低复制病毒的水平,但没有一种能明显影响乙肝表面抗原(HBsAg)或疾病活动。该领域的研究仍在继续,人们对联合治疗的成功寄予厚望。(摘要截断于400字)