{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T19:48:30Z","timestamp":1789069710000,"version":"build-2803163510"},"reference-count":20,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,1,27]],"date-time":"2025-01-27T00:00:00Z","timestamp":1737936000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Nephrology"],"published-print":{"date-parts":[[2025,2]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:p>Complement 3 glomerulonephritis (C3GN) is a rare glomerular disease involving dysregulation of the complement system. We describe our experience using pegcetacoplan, an inhibitor of C3 and its activation fragment, C3b, for treatment\u2010resistant C3GN in a 9\u2010year\u2010old boy referred for evaluation of refractory membranoproliferative glomerulonephritis. Despite treatment with intense immunosuppression (high\u2010dose steroids, mycophenolate mofetil and calcineurin inhibitor), he continued to have high disease activity with low C3 levels (35\u2009mg\/dL), hypertension, symptomatic oedema, anaemia, and nephrotic\u2010range proteinuria (e.g., urine protein\u2010to\u2010creatinine ratio [uPCR], 10\u2009g\/g; serum creatinine, 0.4\u2009mg\/dL). Given the concern for refractory C3GN following a steroid taper and tacrolimus trial with modest response (reduced proteinuria), we initiated pegcetacoplan 540\u2009mg twice weekly for 1\u2009week, followed by 648\u2009mg twice weekly. Laboratory values before pegcetacoplan initiation included uPCR, 1.1\u2009g\/g, serum creatinine, 0.87\u2009mg\/dL, serum albumin, 4.7\u2009g\/dL, and serum C3, 30\u2009mg\/dL. Clinically significant improvements in serum C3 (142\u2009mg\/dL) and uPCR (422\u2009mg\/g) were observed within 1\u2009week of pegcetacoplan initiation; within 3\u2009months (uPCR, 322\u2009mg\/g; serum creatinine, 0.69\u2009mg\/dL; serum C3, 297\u2009mg\/dL), all immunosuppressive and antihypertensive medications were discontinued. No adverse effects of pegcetacoplan were reported. A kidney biopsy after 6\u2009months of pegcetacoplan treatment showed mesangial and focal endocapillary proliferative glomerulonephritis with isolated C3c deposition by immunofluorescence, consistent with previous C3GN diagnosis. In this paediatric patient, compassionate use of pegcetacoplan was associated with rapid clinical improvement without adverse effects, and clinical effectiveness was confirmed by laboratory and histologic results within 6\u2009months of treatment initiation.<\/jats:p>","DOI":"10.1111\/nep.70001","type":"journal-article","created":{"date-parts":[[2025,1,28]],"date-time":"2025-01-28T06:10:28Z","timestamp":1738044628000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Pegcetacoplan for the Treatment of Paediatric <scp>C3<\/scp> Glomerulonephritis: A Case Report"],"prefix":"10.1111","volume":"30","author":[{"given":"German Lozano","family":"Guzman","sequence":"first","affiliation":[{"name":"Phoenix Children's Hospital\u2014Thomas Campus  Phoenix Arizona USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine W.","family":"Perry","sequence":"additional","affiliation":[{"name":"Phoenix Children's Hospital\u2014Thomas Campus  Phoenix Arizona USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,1,27]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrneph.2010.85"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms21020525"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.2215\/CJN.04700513"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41581-018-0107-2"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.kint.2017.10.022"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.kint.2021.05.015"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ekir.2020.09.019"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1053\/j.ajkd.2017.11.019"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1053\/j.ajkd.2018.12.046"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.2215\/CJN.12901211"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00467-023-06120-8"},{"key":"e_1_2_8_13_1","volume-title":"Prescribing Information","author":"Fabhalta","year":"2023"},{"key":"e_1_2_8_14_1","volume-title":"Prescribing Information","author":"Empaveli","year":"2023"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-021-01560-8"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ekir.2023.08.033"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/path.5512"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrneph.2017.156"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1681\/ASN.20233411S1987b"},{"key":"e_1_2_8_20_1","unstructured":"ClinicalTrials.gov. NCT05067127 \u201cPhase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune\u2010Complex Membranoproliferative Glomerulonephritis (VALIANT) \u201daccessed July 31 2024 https:\/\/clinicaltrials.gov\/study\/NCT05067127?term=VALIANT%20AND%20C3G&intr=pegcetacoplan&rank=1."},{"key":"e_1_2_8_21_1","unstructured":"ClinicalTrials.gov. NCT04817618 \u201cStudy of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy (APPEAR\u2010C3G) \u201daccessed July 31 2024 https:\/\/clinicaltrials.gov\/study\/NCT04817618?cond=C3%20Glomerulopathy&term=iptacopan&rank=3."}],"container-title":["Nephrology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/nep.70001","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,3,5]],"date-time":"2025-03-05T03:42:31Z","timestamp":1741146151000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/nep.70001"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1,27]]},"references-count":20,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2025,2]]}},"alternative-id":["10.1111\/nep.70001"],"URL":"https:\/\/doi.org\/10.1111\/nep.70001","archive":["Portico"],"relation":{},"ISSN":["1320-5358","1440-1797"],"issn-type":[{"value":"1320-5358","type":"print"},{"value":"1440-1797","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1,27]]},"assertion":[{"value":"2024-08-29","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-01-17","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-01-27","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70001"}}