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DC Field | Value | Language |
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dc.contributor.author | Schiavulli, M. | en |
dc.contributor.author | Brown, S. A. | en |
dc.contributor.author | Liesner, R. | en |
dc.contributor.author | Gunawardena, S. | en |
dc.contributor.author | Carcao, M. | en |
dc.contributor.author | Winding, B. | en |
dc.contributor.author | Jayawardene, D. | en |
dc.contributor.author | Mukhopadhyay, S. | en |
dc.contributor.author | Königs, C. | en |
dc.contributor.author | Ozelo, M. C. | en |
dc.contributor.author | Dunn, A. | en |
dc.contributor.author | Kulkarni, R. | en |
dc.contributor.author | Nolan, B. | en |
dc.date.accessioned | 2022-11-07T23:37:45Z | - |
dc.date.available | 2022-11-07T23:37:45Z | - |
dc.date.issued | 2020 | en |
dc.identifier.citation | 4, (SUPPL 1), 2020, p. 8 | en |
dc.identifier.other | RIS | en |
dc.identifier.uri | http://dora.health.qld.gov.au/qldresearchjspui/handle/1/3020 | - |
dc.description.abstract | Background: 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.iso | en | en |
dc.relation.ispartof | Research and Practice in Thrombosis and Haemostasis | en |
dc.title | Final results of pups a-long study: Evaluating safety and efficacy of rFVIIIFc in previously untreated patients with haemophilia A | en |
dc.type | Article | en |
dc.identifier.doi | 10.1002/rth2.12401 | en |
dc.subject.keywords | child | en |
dc.subject.keywords | clinical trial | en |
dc.subject.keywords | conference abstract | en |
dc.subject.keywords | controlled study | en |
dc.subject.keywords | drug safety | en |
dc.subject.keywords | drug therapy | en |
dc.subject.keywords | family history | en |
dc.subject.keywords | genotype | en |
dc.subject.keywords | half life time | en |
dc.subject.keywords | hemophilia A | en |
dc.subject.keywords | human | en |
dc.subject.keywords | incidence | en |
dc.subject.keywords | major clinical study | en |
dc.subject.keywords | male | en |
dc.subject.keywords | recombinant blood clotting factor 8 | en |
dc.subject.keywords | pediatric patient | en |
dc.subject.keywords | phase 3 clinical trial | en |
dc.subject.keywords | prophylaxis | en |
dc.subject.keywords | prospective study | en |
dc.subject.keywords | side effect | en |
dc.subject.keywords | soft tissue | en |
dc.subject.keywords | thrombosis | en |
dc.subject.keywords | vein | en |
dc.subject.keywords | fusion protein | en |
dc.subject.keywords | blood clotting factor 8endogenous compound | en |
dc.subject.keywords | multicenter study | en |
dc.subject.keywords | adverse drug reaction | en |
dc.subject.keywords | brain hemorrhage | en |
dc.relation.url | https://www.embase.com/search/results?subaction=viewrecord&id=L633546644&from=exporthttp://dx.doi.org/10.1002/rth2.12401 | | en |
dc.identifier.risid | 2244 | en |
dc.description.pages | 8 | en |
item.cerifentitytype | Publications | - |
item.languageiso639-1 | en | - |
item.grantfulltext | none | - |
item.fulltext | No Fulltext | - |
item.openairetype | Article | - |
item.openairecristype | http://purl.org/coar/resource_type/c_18cf | - |
Appears in Sites: | Children's Health Queensland Publications |
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