https://m2.mtmt.hu/ hun 2026-09-22 20:17 JournalArticle 34309657 VALIDATED true 0 false 2026-08-05T08:53:07.662+0000 2024-07-29T17:41:58.920+0000 2023-11-12T18:09:37.963+0000 true 10015544 Csajbók Edit /api/admin/10015544 Admin true 10015544 2024-07-29T17:41:27.403+0000 2024-07-29T17:41:27.403+0000 2024-07-29T17:41:58.932+0000 true 10015544 Csajbók Edit /api/admin/10015544 Admin true 10015544 true false true 24 24 /api/publicationtype/24 PublicationType 1 true 24 JournalArticle true 10000359 Study Group true 24 24 /api/publicationtype/24 PublicationType 1 true 24 JournalArticle /api/subtype/10000359 Sokszerzős vagy csoportos szerzőségű szakcikk SubType 104 true 10000359 true 1 /api/category/1 Category true 1 Wallace, Eric L Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function results from the 2-year randomised phase III BALANCE study. true true /api/journal/10007110 REVIEWED true false 10007110 false 10007110 true 0022-2593 1468-6244 Journal FOREIGN 61 6 520 530 520 11 2024 Pegunigalsidase alfa is a PEGylated α-galactosidase A enzyme replacement therapy. BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease with an annualised estimated glomerular filtration rate (eGFR) slope more negative than -2 mL/min/1.73 m2/year who had received agalsidase beta for ≥1 year.Patients were randomly assigned 2:1 to receive 1 mg/kg pegunigalsidase alfa or agalsidase beta every 2 weeks for 2 years. The primary efficacy analysis assessed non-inferiority based on median annualised eGFR slope differences between treatment arms.Seventy-seven patients received either pegunigalsidase alfa (n=52) or agalsidase beta (n=25). At baseline, mean (range) age was 44 (18-60) years, 47 (61%) patients were male, median eGFR was 74.5 mL/min/1.73 m2 and median (range) eGFR slope was -7.3 (-30.5, 6.3) mL/min/1.73 m2/year. At 2 years, the difference between median eGFR slopes was -0.36 mL/min/1.73 m2/year, meeting the prespecified non-inferiority margin. Minimal changes were observed in lyso-Gb3 concentrations in both treatment arms at 2 years. Proportions of patients experiencing treatment-related adverse events and mild or moderate infusion-related reactions were similar in both groups, yet exposure-adjusted rates were 3.6-fold and 7.8-fold higher, respectively, with agalsidase beta than pegunigalsidase alfa. At the end of the study, neutralising antibodies were detected in 7 out of 47 (15%) pegunigalsidase alfa-treated patients and 6 out of 23 (26%) agalsidase beta-treated patients. There were no deaths.Based on rate of eGFR decline over 2 years, pegunigalsidase alfa was non-inferior to agalsidase beta. 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http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=37940383&dopt=Abstract false true Keyword 3114 /api/keyword/3114 true 3114 true Keyword 17889 /api/keyword/17889 true 17889 true Keyword 1052782 /api/keyword/1052782 true 1052782 true Keyword 1342923 /api/keyword/1342923 true 1342923 true Keyword 1486912 /api/keyword/1486912 true 1486912 true SjrRating 11466289 /api/sjrrating/11466289 17 0.24 journal RatingType 10002 /api/ratingtype/10002 sjr true true ClassificationExternal 2716 /api/classificationexternal/2716 true 2716 true Q1 DIRECT true true /api/publication/34309657 <div class="JournalArticle Publication long-list"> <div class="authors"> <img title="Forrásközlemény" style="float: left" src="/frontend/resources/grid/publication-core-icon.png"> <div class="autype autype0"> <span class="author-name" >Wallace Eric L </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" mtid="10002781"><a href="/gui2/?type=authors&mode=browse&sel=10002781" 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(36) </div> <div><a target="_blank" href="/api/publication/34309657?labelLang=hun">Összes szerző</a></div> </div> <div class="title"><a href="/gui2/?mode=browse&params=publication;34309657" target="_blank">Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function : results from the 2-year randomised phase III BALANCE study..</a></div> <div> <span class="journal-title">JOURNAL OF MEDICAL GENETICS</span> <span class="journal-issn">(<a target="_blank" href="https://portal.issn.org/resource/ISSN/0022-2593">0022-2593</a> <a target="_blank" href="https://portal.issn.org/resource/ISSN/1468-6244">1468-6244</a>)</span>: <span class="journal-volume">61</span> <span class="journal-issue">6</span> <span class="page"> pp 520-530 </span> <span class="year">(2024)</span> </div> <div class="pub-footer"> <span class="language" xmlns="http://www.w3.org/1999/html">Nyelv: Angol | </span> <span class="identifiers"> <span class="id identifier oa_none" title="none"> <a style="color:black" title="10.1136/jmg-2023-109445" target="_blank" href="https://doi.org/10.1136/jmg-2023-109445"> DOI </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:black" title="001102281300001" target="_blank" href="https://www.webofscience.com/wos/woscc/full-record/001102281300001"> WoS </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:black" title="85176409620" target="_blank" href="http://www.scopus.com/record/display.url?origin=inward&eid=2-s2.0-85176409620"> Scopus </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:black" title="37940383" target="_blank" href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=37940383&dopt=Abstract"> PubMed </a> </span> </span> <OnlyViewableByAuthor><div class="ratings"> <div class="journal-subject">Folyóirat szakterülete: Scopus - Genetics (clinical)&nbsp;&nbsp;&nbsp;SJR indikátor:&nbsp;Q1</div> <div class="journal-subject">Folyóirat szakterülete: Scopus - Genetics&nbsp;&nbsp;&nbsp;SJR indikátor:&nbsp;Q1</div> </div></OnlyViewableByAuthor> <div class="publication-citation" style="margin-left: 0.5cm;"> <span title="Nyilvános idézőközlemények összesen, említések nélkül" class="citingPub-count">Nyilvános idéző összesen: 46</span> | Független: 34 | Függő: 12 | Nem jelölt: 0 | WoS jelölt: 44 | Scopus jelölt:&nbsp;44 | WoS/Scopus jelölt:&nbsp;46 | DOI jelölt:&nbsp;46 </div> <div class="mtid"><span class="long-pub-mtid">Közlemény: 34309657</span> | <span class="status-data status-VALIDATED"> Egyeztetett </span> Forrás | <span class="type-subtype">Folyóiratcikk ( Sokszerzős vagy csoportos szerzőségű szakcikk ) </span> | <span class="pub-category">Tudományos</span> | <span class="publication-sourceOfData">PubMed XML</span> </div> <div class="lastModified">Utolsó módosítás: 2024.07.29. 19:41 Csajbók Edit (SE_KK_Admin5_CSE, admin) </div> </div></div>