Al-Shaghana May, Bentall Andrew, Jesky Mark D, Lester William, Lipkin Graham
Department of Nephrology, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
School of Immunity and Infection, University of Birmingham, Birmingham, UK.
Oxf Med Case Reports. 2017 Jun 15;2017(6):omx025. doi: 10.1093/omcr/omx025. eCollection 2017 Jun.
Thrombotic microangiopathy (TMA) is characterized by microscopic angiopathic haemolytic anaemia, thrombocytopenia and organ injury. Supportive therapies include the use of blood products. Recently the terminal complement inhibitor eculizumab has been approved in atypical haemolytic uraemic syndrome (aHUS) in some countries. We report the case of a 23-year-old female Jehovah's Witness presenting with vaginal haemorrhage from thrombocytopaenia, severe anaemia (nadir Hb 28 g/L) and anuric acute kidney injury with TMA secondary to aHUS. Despite a life threatening illness, the patient declined the use of blood components and plasma exchange. Eculizumab was administered early with subsequent improvement and resolution of haemolysis, return to baseline renal function whilst avoiding use of blood products. This case demonstrates the effective use of eculizumab for life saving therapy in a patient refusing blood products. It highlights the importance of accessibility for high cost therapies, but the disparity in access between healthcare systems.
血栓性微血管病（TMA）的特征是微血管性溶血性贫血、血小板减少和器官损伤。支持性治疗包括使用血液制品。最近，终末补体抑制剂依库珠单抗在一些国家已被批准用于非典型溶血性尿毒症综合征（aHUS）。我们报告了一例23岁女性耶和华见证会成员的病例，该患者因aHUS继发TMA，出现血小板减少导致的阴道出血、严重贫血（最低血红蛋白28 g/L）和无尿性急性肾损伤。尽管患有危及生命的疾病，但患者拒绝使用血液成分和进行血浆置换。早期给予依库珠单抗后，溶血得到改善并消退，肾功能恢复至基线水平，同时避免了使用血液制品。该病例证明了依库珠单抗在拒绝血液制品的患者中用于挽救生命治疗的有效性。它凸显了获得高成本治疗方法的重要性，但也揭示了不同医疗系统之间在获得治疗方面的差距。
