SUN-393 DIALYSIS FREE ONE YEAR AFTER RECEIVING ECULIZUMAB

Abstract

Thrombotic microangiopathy (TMA) is characterized by thrombocytopenia, microangiopathic hemolytic anemia and end organ damage from microvascular thrombosis. Atypical hemolytic uremic syndrome (aHUS) is one form of TMA that occurs because of pathologic complement activation. Mutations in the genes coding for complement components predispose to hyperactivation of the alternative complement pathway. Therapeutic complement inhibition is effective in aHUS. Eculizumab, a monoclonal humanized anti-C5 antibody, has shown success in patients with aHUS. We report a case of a patient with aHUS that recovered renal function one year after being in treatment with eculizumab. 37-year-old female with past medical history significant for discoid lupus, Raynaud’s phenomenon and hypertension, was transferred from and outside hospital for further management of sepsis, acute respiratory failure, anemia, thrombocytopenia and acute renal failure. RPGN due to lupus nephritis was suspected therefore kidney biopsy was obtained prior to transfer. She was also given empiric IV steroids, oral cyclophosphamide and received 3 treatments of plasma exchange. Her hospital course was complicated by hemoptysis, worsening respiratory status and worsening renal failure to the point of needing initiation of hemodialysis. Workup showed elevated LDH (222 U/L), low Haptoglobin (<10 mg/dL), slightly low C3 (84 mg/dL) and normal C4 (23 mg/dL). Workup was negative for ADAMTS13, antiphospholipid antibody syndrome, scleroderma and lupus. The pattern of renal injuryin the biopsy was that of a thrombotic microangiopathy. Both glomeruli and small arterioles appear to be disproportionately affected. chronic scarring was mild suggesting an acute/subacute process. There was no evidence of an immune complex mediated lupus-related process. Patient was diagnosed with atypical hemolytic uremic syndrome and started on eculizumab on 8/29/2016. She remained on dialysis and Eculizumab was continued long term mostly for her hematologic disturbances. To our surprise, she recovered renal function almost one year after initiating her treatment. Her last dialysis treatment was July 25, 2017. Atypical hemolytic uremic syndrome (aHUS) is a rare, chronic, life-threatening disease due to complement dysregulation. Early treatment with eculizumab, a humanized monoclonal antibody against complement, has shown to be effective in controlling symptoms and reversing organ damage. Several cases have been described of patients coming off renal replacement therapy after treatment with eculizumab. Prospective studies of patients with renal failure due to aHUS treated with eculizumab are required to validate the applicability of the positive experience described in this case study.