University of East London, Stratford, E15 4LZ, UK.
National Hospital for Neurology and Neurosurgery, UCLH NHS Foundation Trust, Queen Square, London, WC1N 3BG, UK.
J Neurol. 2024 Aug;271(8):5671-5686. doi: 10.1007/s00415-024-12436-8. Epub 2024 Jun 19.
Valproate is the most effective medication for generalised epilepsies, and several specific epilepsy syndromes. For some people, it will be the only medication to establish seizure remission, and withdrawing it carries risks of seizure recurrence and Sudden Unexpected Death in Epilepsy (SUDEP). It is also of proven efficacy for bipolar disorder and migraine prevention. Guidelines based on observational and epidemiological studies stress that maternal valproate related teratogenicity and neurodevelopmental effects are significantly higher than for other antiseizure medications (ASMs). It should, therefore, only be used if other medications are ineffective and after balancing the teratogenicity risk. Regulatory restrictions have changed prescribing practices and reduced valproate use. The number of other medications that must be trialled in the different conditions for which valproate has effectiveness and the consequences of the lack of efficacy of those drugs leading to significant harm including death remains unexplored. Risk minimisation measures (RMMs) for valproate, chiefly Pregnancy Prevention practices (PPP), consider foetal risk and not risk to people living with epilepsy. In the United Kingdom (UK), limitations relating to valproate use in all people < 55 years commenced in January 2024. While the evidence in child-bearing women is not disputed, the data in males are based on animal models, case reports, and one commissioned, unpublished, non-peer reviewed report unavailable to the UK public, stakeholder charities or professionals. Evidence suggests that 30-40% of people switching from valproate have breakthrough seizures. Thus, an estimated 21,000-28000 people in the UK will imminently be exposed to the potential hazards of breakthrough seizures, including death. There is little government investment in monitoring the effects of these changes to valproate prescribing on patient health and quality of life. This review summarises the history of valproate regulation, evidence underpinning it and argues how the latest regulations in the UK do not align with the country's medical regulatory bodies ethical principles nor with the Montgomery principles of informed patient choice and autonomy. It dissects how such regulations infringe Common Law principles, nor give due regard for patient outcomes beyond reproduction. The paper looks to provide recommendations to redress these concerns while appreciating the core need for such governance to emerge in the first place.
丙戊酸盐是治疗全身性癫痫和几种特定癫痫综合征最有效的药物。对于某些人来说,它将是唯一能控制癫痫发作的药物,而停药会增加癫痫发作和癫痫猝死(SUDEP)的风险。它也已被证明对双相情感障碍和偏头痛预防有效。基于观察性和流行病学研究的指南强调,丙戊酸盐相关的致畸性和神经发育影响明显高于其他抗癫痫药物(ASMs)。因此,只有在其他药物无效且权衡了致畸风险后,才应使用丙戊酸盐。监管限制改变了处方实践并减少了丙戊酸盐的使用。在丙戊酸盐有效的不同情况下,必须试验其他药物的数量,以及这些药物缺乏疗效导致的严重伤害(包括死亡)的后果仍未得到探索。丙戊酸盐的风险最小化措施(RMMs),主要是妊娠预防措施(PPP),考虑胎儿风险,而不是癫痫患者的风险。在英国,2024 年 1 月开始限制所有年龄<55 岁的人使用丙戊酸盐。虽然关于生育期妇女的证据没有争议,但关于男性的数据基于动物模型、病例报告和一份委托的、未发表的、非同行评审的报告,该报告在英国公众、利益相关者慈善机构或专业人员中不可用。有证据表明,30%-40%的从丙戊酸盐转换的患者会出现突破性发作。因此,英国估计有 21000-28000 人将立即面临突破性发作的潜在危险,包括死亡。政府对监测丙戊酸盐处方改变对患者健康和生活质量的影响几乎没有投资。本文综述了丙戊酸盐监管的历史、支撑它的证据,并认为英国最新的监管规定既不符合该国医疗监管机构的伦理原则,也不符合蒙哥马利关于知情患者选择和自主权的原则。它剖析了这些规定如何侵犯普通法原则,以及如何不适当考虑生育以外的患者结果。本文旨在提供建议,以纠正这些担忧,同时认识到首先需要这种治理的核心必要性。