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  • Title: Successful Treatment of Transplantation-associated Atypical Hemolytic Uremic Syndrome With Eculizumab.
    Author: Hasegawa D, Saito A, Nino N, Uemura S, Takafuji S, Yokoi T, Kozaki A, Ishida T, Kawasaki K, Yasumi T, Sakata N, Ohtsuka Y, Hirase S, Mori T, Nishimura N, Kusumoto M, Ogawa Y, Tominaga K, Nakagawa T, Kanda K, Tanaka R, Kosaka Y.
    Journal: J Pediatr Hematol Oncol; 2018 Jan; 40(1):e41-e44. PubMed ID: 28538511.
    Abstract:
    We herein reported a 4-month-old boy with transplantation-associated atypical hemolytic uremic syndrome (TA-aHUS) who was successfully treated with eculizumab. The patient diagnosed with type 3 of familial hemophagocytic lymphohistiocytosis underwent cord blood transplantation. After transplantation, he developed TA-aHUS, but plasma exchanges were unsuccessful. We identified deletions in CFH-related gene 1 (del-CFHR1) by the multiplex ligation-dependent probe amplification testing procedure and CFH autoantibodies. Eculizumab has been administered to the patient, with a marked improvement being achieved in thrombocytopenia. He has been well except for the persistent microhematuria for a year after transplantation. Uncontrolled complement activation might be involved in the pathophysiology of TA-aHUS.
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