Please use this identifier to cite or link to this item: https://dora.health.qld.gov.au/qldresearchjspui/handle/1/3020
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dc.contributor.authorSchiavulli, M.en
dc.contributor.authorBrown, S. A.en
dc.contributor.authorLiesner, R.en
dc.contributor.authorGunawardena, S.en
dc.contributor.authorCarcao, M.en
dc.contributor.authorWinding, B.en
dc.contributor.authorJayawardene, D.en
dc.contributor.authorMukhopadhyay, S.en
dc.contributor.authorKönigs, C.en
dc.contributor.authorOzelo, M. C.en
dc.contributor.authorDunn, A.en
dc.contributor.authorKulkarni, R.en
dc.contributor.authorNolan, B.en
dc.date.accessioned2022-11-07T23:37:45Z-
dc.date.available2022-11-07T23:37:45Z-
dc.date.issued2020en
dc.identifier.citation4, (SUPPL 1), 2020, p. 8en
dc.identifier.otherRISen
dc.identifier.urihttp://dora.health.qld.gov.au/qldresearchjspui/handle/1/3020-
dc.description.abstractBackground: PUPs A-LONG is the first study evaluating an extended half-life (EHL), recombinant factor VIII Fc fusion protein (rFVIIIFc), in previously untreated patients (PUPs) with haemophilia A. Aims: To evaluate safety, including inhibitor development, and efficacy of rFVIIIFc in PUPs. Methods: This open-label, multicentre, Phase 3 study (NCT02234323) enrolled male PUPs aged <6 years with haemophilia A (<1 IU/dL endogenous FVIII) to receive rFVIIIFc. Primary endpoint was inhibitor development (incidence rate=number of patients with inhibitor/number of patients reaching ≥ 10 exposure days (ED) milestone or with inhibitor). A secondary endpoint was annualized bleeding rate (ABR). Results: Of 103 patients receiving ≥ 1 dose; 80 (77.7%) were <1-year old; 20 (19.4%) had a family history of inhibitors, and 82 (79.6%) had a high-risk haemophilia genotype. Eighty-one patients started on episodic treatment; of these, 69 switched to prophylaxis. Twenty patients started on prophylaxis and 2 were not assigned a regimen. Eighty-seven (84.5%) patients completed the study. Eighty-seven (84.5%), 85 (82.5%), and 81 (78.6%), patients had ≥ 10, ≥20, ≥50 EDs to rFVIIIFc, respectively. Total and high-titre (≥5.00 BU/mL) inhibitor rate was 31.1% (28/90) and 15.6% (14/90), respectively, for patients with ≥ 10 EDs (3 inhibitor patients with <10 EDs included). Median time to inhibitor development was 9 EDs (range: 1-53). rFVIIIFc dosing and efficacy data in Table 1. Twenty-eight (27.2%) had rFVIIIFc treatment-related serious adverse events (TESAEs); FVIII inhibition, n = 28; soft tissue haemorrhage, n = 1; deep vein/CVAD-associated thromboses, n = 2). There was 1 non-treatment- related death due to intracranial haemorrhage (onset during screening period prior to first rFVIIIFc dose). Conclusions: This was the first prospective study of an EHL rFVIIIFc treatment for PUPs with severe haemophilia A. Overall inhibitor development was in the range that can be expected, although high-titre incidence was lower than that reported in the literature. The data demonstrate that rFVIIIFc was well tolerated and effective in this paediatric patient population.L6335466442020-12-07 <br />en
dc.language.isoenen
dc.relation.ispartofResearch and Practice in Thrombosis and Haemostasisen
dc.titleFinal results of pups a-long study: Evaluating safety and efficacy of rFVIIIFc in previously untreated patients with haemophilia Aen
dc.typeArticleen
dc.identifier.doi10.1002/rth2.12401en
dc.subject.keywordschilden
dc.subject.keywordsclinical trialen
dc.subject.keywordsconference abstracten
dc.subject.keywordscontrolled studyen
dc.subject.keywordsdrug safetyen
dc.subject.keywordsdrug therapyen
dc.subject.keywordsfamily historyen
dc.subject.keywordsgenotypeen
dc.subject.keywordshalf life timeen
dc.subject.keywordshemophilia Aen
dc.subject.keywordshumanen
dc.subject.keywordsincidenceen
dc.subject.keywordsmajor clinical studyen
dc.subject.keywordsmaleen
dc.subject.keywordsrecombinant blood clotting factor 8en
dc.subject.keywordspediatric patienten
dc.subject.keywordsphase 3 clinical trialen
dc.subject.keywordsprophylaxisen
dc.subject.keywordsprospective studyen
dc.subject.keywordsside effecten
dc.subject.keywordssoft tissueen
dc.subject.keywordsthrombosisen
dc.subject.keywordsveinen
dc.subject.keywordsfusion proteinen
dc.subject.keywordsblood clotting factor 8endogenous compounden
dc.subject.keywordsmulticenter studyen
dc.subject.keywordsadverse drug reactionen
dc.subject.keywordsbrain hemorrhageen
dc.relation.urlhttps://www.embase.com/search/results?subaction=viewrecord&id=L633546644&from=exporthttp://dx.doi.org/10.1002/rth2.12401 |en
dc.identifier.risid2244en
dc.description.pages8en
item.cerifentitytypePublications-
item.languageiso639-1en-
item.grantfulltextnone-
item.fulltextNo Fulltext-
item.openairetypeArticle-
item.openairecristypehttp://purl.org/coar/resource_type/c_18cf-
Appears in Sites:Children's Health Queensland Publications
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