Alobaidi Sami, AlDabbagh Ammar, Alamoudi Amany, Almowarey Murad, Akl Ahmed
Department of Medicine, Dr.Soliman Fakeeh Hospital, Jeddah, Saudi Arabia.
Department of Medicine, University of Jeddah, Jeddah, Saudi Arabia.
CEN Case Rep. 2019 May;8(2):139-143. doi: 10.1007/s13730-019-00383-7. Epub 2019 Feb 4.
Pregnancy-associated atypical hemolytic uremic syndrome (P-aHUS) is a rare condition. It is characterized by very high maternal mortality and morbidity. Most cases of P-aHUS (79%) manifest in the postpartum period; this is probably due to the complement's involvement in aHUS pathogenesis. Eculizumab is approved for aHUS treatment, but its use is limited due to cost, unknown duration of treatment, and vague dose intervals to keep patients in remission. In this case report, we present a 26-year-old female with P-aHUS with hybrid CFHR1/CFH gene. Eculizumab was initiated after 5 weeks of being on hemodialysis and plasmapheresis sessions. Full remission successfully achieved after 6th dose of Eculizumab, within 13 weeks of onset of aHUS. Due to financial issues and inability to financially cover the cost, Eculizumab was set in hold. Within 6 months, she suffered recurrence of the disease and Eculizumab was re-instated. After re-inducing full remission, the patient was switched to Eculizumab every 3 months instead of the recommended manufacture dose interval of every 2 weeks. We followed this patient for 3 years and she continued to be in remission based on clinical and laboratory data. In conclusion, achievement of successful and maintenance of remission of P-aHUS in this patient who had limited access to Eculizumab raise the attention of the efficacy of Eculizumab at longer time intervals. However, it is time to consider conducting a long-term study to learn about the safety and efficacy of this approach, which may have a major financial advantage for patients.
妊娠相关非典型溶血性尿毒症综合征(P-aHUS)是一种罕见病。其特征是孕产妇死亡率和发病率极高。大多数P-aHUS病例(79%)在产后发病;这可能是由于补体参与了非典型溶血性尿毒症综合征(aHUS)的发病机制。依库珠单抗被批准用于aHUS的治疗,但由于成本、未知的治疗持续时间以及维持患者缓解的剂量间隔不明确,其使用受到限制。在本病例报告中,我们介绍了一名患有CFHR1/CFH杂交基因的26岁P-aHUS女性患者。在进行血液透析和血浆置换5周后开始使用依库珠单抗。在aHUS发病13周内,第6剂依库珠单抗后成功实现完全缓解。由于经济问题且无法负担费用,依库珠单抗停用。6个月内,她疾病复发,依库珠单抗重新启用。再次诱导完全缓解后,患者改为每3个月使用一次依库珠单抗,而非推荐的每2周一次的生产厂家规定剂量间隔。我们对该患者随访了3年,根据临床和实验室数据,她持续处于缓解状态。总之,在这名难以获得依库珠单抗的患者中成功实现并维持P-aHUS缓解,引发了对依库珠单抗在更长时间间隔下疗效的关注。然而,现在是时候考虑开展一项长期研究,以了解这种方法的安全性和有效性了,这可能对患者具有重大的经济优势。