{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T23:57:21Z","timestamp":1789516641390,"version":"build-2803163510"},"reference-count":29,"publisher":"MDPI AG","issue":"18","license":[{"start":{"date-parts":[[2023,9,21]],"date-time":"2023-09-21T00:00:00Z","timestamp":1695254400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"National Research, Development and Innovation Office (NKFIH)","award":["K-142728"],"award-info":[{"award-number":["K-142728"]}]},{"name":"National Research, Development and Innovation Office (NKFIH)","award":["K-125470"],"award-info":[{"award-number":["K-125470"]}]},{"name":"National Research, Development and Innovation Office (NKFIH)","award":["TKP2021-EGA-24"],"award-info":[{"award-number":["TKP2021-EGA-24"]}]},{"name":"National Research, Development and Innovation Office (NKFIH)","award":["STIA-KFI-2021 (AJSz)"],"award-info":[{"award-number":["STIA-KFI-2021 (AJSz)"]}]},{"name":"Semmelweis University","award":["K-142728"],"award-info":[{"award-number":["K-142728"]}]},{"name":"Semmelweis University","award":["K-125470"],"award-info":[{"award-number":["K-125470"]}]},{"name":"Semmelweis University","award":["TKP2021-EGA-24"],"award-info":[{"award-number":["TKP2021-EGA-24"]}]},{"name":"Semmelweis University","award":["STIA-KFI-2021 (AJSz)"],"award-info":[{"award-number":["STIA-KFI-2021 (AJSz)"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>The recent classification of pediatric thrombotic microangiopathies (TMA) takes into consideration mechanisms of disease for guidance to targeted therapies. We present our experience with seven patients with antibody mediated atypical hemolytic uremic syndrome (aHUS) and thrombotic thrombocytopenic purpura (TTP). Five children had aHUS with antibodies against complement factor H (CFH-ab) and two with TTP with antibodies against metalloproteinase ADAMTS13. In the aHUS cases diagnosed and treated before the eculizumab era, CFH-ab was detected using the ELISA assay. Mutational analysis of selected complement genes was performed. TTP was diagnosed if, in addition to microangiopathic hemolytic anemia and thrombocytopenia, ischemic organ involvement and severe deficiency in ADAMTS13 activity were present. Treatment protocol consisted of plasma exchanges (PE) and steroid pulses, followed by the combination of cyclophosphamide and rituximab to achieve long-term immunosuppression. Four patients with CFH-ab and the TTP patients with ADAMTS13 antibodies came into sustained remission. After a median follow-up of 11.7 (range 7.7\u201312.9) years without maintenance therapy, no disease recurrence was observed; nevertheless, six patients, two had hypertension and two had proteinuria as a late consequence. One patient, with late diagnosis of CFH-ab and additional genetic risk factors who was treated only with PE and plasma substitution, reached end-stage renal disease and was later successfully transplanted using eculizumab prophylaxis. In the cases of antibody-mediated TMAs, PE and early immunosuppressive treatment may result in sustained remission with preserved kidney function. Further data are needed to establish optimal treatment of anti-FH antibody-associated HUS.<\/jats:p>","DOI":"10.3390\/ijms241814389","type":"journal-article","created":{"date-parts":[[2023,9,21]],"date-time":"2023-09-21T21:08:36Z","timestamp":1695330516000},"page":"14389","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Immunosuppressive Therapy of Antibody-Mediated aHUS and TTP"],"prefix":"10.3390","volume":"24","author":[{"given":"Kata","family":"Kelen","sequence":"first","affiliation":[{"name":"B\u00f3kay Street Unit, Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3812-2711","authenticated-orcid":false,"given":"Orsolya","family":"Horv\u00e1th","sequence":"additional","affiliation":[{"name":"B\u00f3kay Street Unit, Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"\u00c9va","family":"Kis","sequence":"additional","affiliation":[{"name":"Department of Pediatric Cardiology, Gottsegen Gy\u00f6rgy Hungarian Institute of Cardiology, 1096 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B\u00e1lint","family":"Mikes","sequence":"additional","affiliation":[{"name":"B\u00f3kay Street Unit, Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P\u00e9ter","family":"Sallay","sequence":"additional","affiliation":[{"name":"B\u00f3kay Street Unit, Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zolt\u00e1n","family":"Proh\u00e1szka","sequence":"additional","affiliation":[{"name":"Research Laboratory, Department of Medicine and Hematology, Semmelweis University, 1083 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Attila J\u00f3zsef","family":"Szab\u00f3","sequence":"additional","affiliation":[{"name":"B\u00f3kay Street Unit, Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary"},{"name":"Pediatric Center, MTA Center of Excellence, Semmelweis University, 1083 Budapest, Hungary"},{"name":"ELKH-SE Pediatrics and Nephrology Research Group, 1052 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gy\u00f6rgy S.","family":"Reusz","sequence":"additional","affiliation":[{"name":"B\u00f3kay Street Unit, Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,9,21]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1038\/sj.ki.5001581","article-title":"A classification of hemolytic uremic syndrome and thrombotic thrombocytopenic purpura and related disorders","volume":"70","author":"Besbas","year":"2006","journal-title":"Kidney Int."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1007\/s00467-008-0964-1","article-title":"Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome","volume":"24","author":"Ariceta","year":"2009","journal-title":"Pediatr. 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