Pharmacokinetic and clinical improvements after PK-guided switch from standard half-life to extended half-life factor VIII products

Megías‐Vericat, Juan Eduardo; Bonanad, Santiago; Berrueco, Rubén; Mingot‐Castellano, María Eva; López, Manuel Rodríguez; Hirnyk, M. Canaro; Mateo, José; Villas, J. M. Calvo; Guaita, S. Haya; Meda, M; Jaime, F. López; Albo-López, C.; Palomero-Massanet, Antonio; Seto, Noelia Vilalta; Leciñena, I. Larrodé; Haro, Ana Rosa Cid; Andres, Jennifer

doi:10.1016/j.thromres.2022.06.001

Cited by 8 publications

(25 citation statements)

References 37 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Among the 46 identified publications, 38 were only available as abstracts, 11–48 and eight were published as full articles 49–56 . The two publications by Van der Sluijs et al 36,55 .…”

Section: Resultsmentioning

confidence: 99%

“…Study populations ranged from 3 to 290 patients. Thirty three of the identified publications did not specify whether the study population included children, adolescents or adults 11–13,15–22,25,26,30,31,33–37,39,41–48,53–56 . Of the remaining, three publications only included children, 32,40,51 one publication only included adolescents, 14 three publications only included adults, 38,49,52 and six publications included a combination of children, adolescents and adults 23,24,27–29,50 .…”

Section: Resultsmentioning

confidence: 99%

“…Of the remaining, three publications only included children, 32,40,51 one publication only included adolescents, 14 three publications only included adults, 38,49,52 and six publications included a combination of children, adolescents and adults 23,24,27–29,50 . Most publications included previously treated patients 11–14,17–19,22–25,27,28,30–37,41–43,45,46,48–55 and patients with severe haemophilia A 11,14,15,17,18,20–23,25–27,29,31,33–35,40,41,43,45–47,49–54,56 . If most of the patient population had severe haemophilia A (set to ≥80%), the study was considered a subgroup analysis of severe haemophilia A patients in the current literature review.…”

Section: Resultsmentioning

confidence: 99%

“…Sixteen of the 26 studies with results on ABR reported results on a subgroup of severe haemophilia A patients, 11,16,18,20,22,24,25,27,29,35,41,49,50,52,54,56 and 11 studies reported ABR results from a patient population with mixed disease severity 16,20,30,36–39,50,53,55,56 …”

Section: Resultsmentioning

confidence: 99%

“…Sources : a. Holmström et al., 30 b. Wall et al., 37 c. Goldmann et al., 38 d. Scott et al., 39 e. Olivieri et al., 56 f. Delmotte et al., 12 g. Chhabra et al., 53 h. Van er Sluijs et al, 36,55 i. Mancuso et al., 16 j. Scott et al., 24 k. Ay et al., 49 l. Giraud et al., 50 m. Megias‐Vericat et al., 18 n. Heller et al., 29 o. Holmström et al., 54 p. Olivieri et al, 56 RCT phase 2; Mahlangu et al 4 …”

Section: Resultsunclassified

See 4 more Smart Citations

Real‐world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe

Blatný

Nielsen²,

Reitzel³

et al. 2023

Haemophilia

View full text Add to dashboard Cite

Introduction:The real-world effectiveness of the efmoroctocog alfa (recombinant FVIII Fc fusion protein, a rFVIIIFc) has been investigated in numerous studies, however, currently, there exists no comprehensive collection of the existing real-world evidence (RWE) on the performance of prophylactic use of rFVIIIFc. Aim:The aims of this systematic literature study were to identify, review, evaluate and collate the RWE of prophylactic rFVIIIFc for patients with haemophilia A reported in Europe. Methods: We searched Medline and Embase from 2014 to February 2022 to identify publications reporting the effectiveness of rFVIIIFc in patients with haemophilia A. The outcomes of interest were annualised bleeding rates (ABR, AjBR, AsBR), injection frequency, factor consumption, adherence, development of inhibitors and quality-of-life measures.Results: 46 eligible publications (eight full-text articles) were included. rFVIIIFc showed a low ABR in patients with haemophilia A. Studies assessing treatment switching from a standard half-life (SHL) treatment to rFVIIIFc found that the ABR and consumption were reduced in most patients. Studies assessing rFVIIIFc effectiveness reported a median ABR between 0.0 and 2.0 with median injections per week ranging between 1.8 and 2.4 and median doses between 60 and 105 IU/kg/week. Of the studies assessing inhibitor development, only one study reported an incidence of a low titre inhibitor, and no patients developed clinically significant inhibitors. Conclusion: rFVIIIFc prophylaxis treatment results in a low ABR across studiesin patients with haemophilia A in a European real-world setting, which correlates with findings from clinical trials assessing the efficacy of rFVIIIFc in patients with haemophilia A.

show abstract

“…Among the 46 identified publications, 38 were only available as abstracts, 11–48 and eight were published as full articles 49–56 . The two publications by Van der Sluijs et al 36,55 .…”

Section: Resultsmentioning

confidence: 99%