Individualised therapeutic approach to the patient with atypical haemolytic-uraemic syndrome.

IF 3.6 4区 医学 Q1 MEDICINE, GENERAL & INTERNAL Clinical Medicine Pub Date : 2024-10-04 DOI:10.1016/j.clinme.2024.100250
Ivana Mikačić, Nikolina Marić
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Abstract

Atypical haemolytic-uraemic syndrome (aHUS) is a rare disease associated with uncontrolled activation of the alternative complement pathway, leading to thrombotic microangiopathy (TMA). Early diagnosis and treatment with eculizumab, a monoclonal antibody targeting the complement component C5, are crucial to improve outcomes and prevent renal failure and mortality. Current recommendations include lifelong eculizumab therapy, yet this practice presents challenges including high treatment costs and increased infection risks from prolonged complement inhibition. We hypothesise that a personalised eculizumab dosing strategy tailored to individual patient responses could optimise therapy, reduce costs and improve safety. This hypothesis was evaluated through a presentation of a patient who was managed with a specific eculizumab treatment approach. The patient's condition improved significantly, allowing for a gradual reduction in eculizumab dosage based on clinical response and drug level monitoring. Throughout treatment, the patient's complement activity and eculizumab levels were closely monitored, showing that lower doses maintained therapeutic efficacy without evident TMA recurrence. This case supports the feasibility of transitioning from fixed regimens to personalised dosing strategies in managing aHUS. Such approaches could mitigate the risks and costs associated with lifelong therapy while maintaining disease control, especially considering the variability in relapse risk among different genetic mutations. This personalised treatment model might significantly impact the management of aHUS, aligning clinical care with individual patient needs and economic considerations. Further research should relate drug pharmacokinetics/pharmacodynamics to clinical/genetic setting to identify milestones of individual patient treatment approach.

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针对非典型溶血尿毒综合征患者的个性化治疗方法。
非典型溶血性尿毒症综合征(aHUS)是一种罕见疾病,与不受控制的替代补体途径激活有关,会导致血栓性微血管病(TMA)。早期诊断和使用针对补体成分 C5 的单克隆抗体依库珠单抗(eculizumab)治疗对于改善预后、预防肾衰竭和死亡至关重要。目前的建议包括终生使用依库珠单抗治疗,但这种做法面临着高昂的治疗费用和因长期抑制补体而增加的感染风险等挑战。我们假设,根据患者个体反应定制个性化的依库珠单抗给药策略可以优化治疗、降低成本并提高安全性。我们通过介绍一位采用特定依库珠单抗治疗方法的患者来评估这一假设。患者的病情得到了明显改善,因此可以根据临床反应和药物水平监测情况逐步减少依库珠单抗的用量。在整个治疗过程中,对患者的补体活性和依库珠单抗水平进行了密切监测,结果显示,较低剂量的依库珠单抗仍能维持疗效,且无明显的 TMA 复发。该病例证明了在治疗 aHUS 时从固定治疗方案过渡到个性化用药策略的可行性。特别是考虑到不同基因突变之间复发风险的差异性,这种方法可以在维持疾病控制的同时减轻终身治疗带来的风险和成本。这种个性化治疗模式可能会对 aHUS 的治疗产生重大影响,使临床治疗符合患者的个体需求和经济考虑。进一步的研究应将药物药代动力学/药效学与临床/遗传学环境联系起来,以确定个体化患者治疗方法的里程碑。
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来源期刊
Clinical Medicine
Clinical Medicine 医学-医学:内科
CiteScore
7.20
自引率
0.00%
发文量
0
审稿时长
6-12 weeks
期刊介绍: Clinical Medicine is aimed at practising physicians in the UK and overseas and has relevance to all those managing or working within the healthcare sector. Available in print and online, the journal seeks to encourage high standards of medical care by promoting good clinical practice through original research, review and comment. The journal also includes a dedicated continuing medical education (CME) section in each issue. This presents the latest advances in a chosen specialty, with self-assessment questions at the end of each topic enabling CPD accreditation to be acquired. ISSN: 1470-2118 E-ISSN: 1473-4893 Frequency: 6 issues per year
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