Matching-Adjusted Indirect Comparison of Recombinant Factor IX Albumin Fusion Protein Versus Recombinant Factor IX Fc Fusion Protein for Weekly Prophylactic Treatment of Hemophilia B.
Efficacy
Hemophilia B
Matching-adjusted indirect comparisons
Prophylactic treatment
Recombinant factor IX Fc fusion protein
Recombinant factor IX albumin fusion protein
Journal
Advances in therapy
ISSN: 1865-8652
Titre abrégé: Adv Ther
Pays: United States
ID NLM: 8611864
Informations de publication
Date de publication:
09 Dec 2023
09 Dec 2023
Historique:
received:
13
09
2023
accepted:
17
11
2023
medline:
10
12
2023
pubmed:
10
12
2023
entrez:
9
12
2023
Statut:
aheadofprint
Résumé
Prophylactic treatment of hemophilia B with recombinant factor IX (rFIX) molecules with enhanced pharmacokinetics including rIX-FP (albutrepenonacog alfa; Idelvion MAIC analyses leveraged individual patient data from the PROLONG-9FP trial and published summary-level data from the B-LONG trial for subjects who received weekly prophylaxis regimens. Individual patient data were used to assign weights and balance subjects from PROLONG-9FP with subjects from B-LONG on baseline disease severity, age, prior FIX regimen, and body mass index (BMI). Six efficacy outcomes were analyzed including annualized bleeding rate (ABR), annualized spontaneous bleeding rate (AsBR), annualized joint bleeding rate (AjBR), and the proportion of subjects without bleeding events (for total, spontaneous, and joint bleeding events). After adjustment for baseline disease severity, age, prior FIX regimen, and BMI, rIX-FP was associated with a statistically significant decrease in AsBR (rate ratio [RR] 0.42; 95% confidence interval [CI] 0.22, 0.82; P = 0.0107), and the proportion of patients without bleeding events (odds ratio [OR] 3.24; 95% CI 1.41, 7.45; P = 0.0057), spontaneous bleeding events (OR 3.47; 95% CI 1.56, 7.73; P = 0.0023), and joint bleeding events (OR 2.41; 95% CI 1.10, 5.26; P = 0.0274) compared with rFIXFc. Prophylactic treatment with rIX-FP was also associated with a numerically lower ABR (RR 0.75; 95% CI 0.32, 1.75; P = 0.5095) and AjBR (RR 0.82; 95% CI 0.37, 1.82; P = 0.6178). The MAICs demonstrated that weekly prophylaxis treatment of severe hemophilia B with rIX-FP resulted in favorable efficacy outcomes as compared to rFIXFc. These findings suggest rIX-FP may offer improved clinical benefits over rFIXFc.
Identifiants
pubmed: 38070040
doi: 10.1007/s12325-023-02745-1
pii: 10.1007/s12325-023-02745-1
doi:
Types de publication
Journal Article
Langues
eng
Informations de copyright
© 2023. The Author(s).
Références
Berntorp E, Fischer K, Hart DP, et al. Haemophilia. Nat Rev Dis Primers. 2021;7(1):45.
doi: 10.1038/s41572-021-00278-x
pubmed: 34168126
Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015;125(13):2038–44.
doi: 10.1182/blood-2015-01-528414
pubmed: 25712992
Castaman G. The benefits of prophylaxis in patients with hemophilia B. Expert Rev Hematol. 2018;11(8):673–83.
doi: 10.1080/17474086.2018.1489719
pubmed: 29909699
von Mackensen S, Eldar-Lissai A, Auguste P, von Maltzahn R, Yu R, Wyrwich KW. Measurement properties of the Haem-A-QoL in haemophilia clinical trials. Haemophilia. 2017;23(3):383–91.
doi: 10.1111/hae.13140
Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia. 2001;7(4):392–6.
doi: 10.1111/j.1365-2516.2001.00534.x
pubmed: 11442644
Preijers T, Bukkems L, van Spengler M, Leebeek F, Cnossen M, Mathot R. In silico comparison of pharmacokinetic properties of three extended half-life factor IX concentrates. Eur J Clin Pharmacol. 2021;77(8):1193–200.
doi: 10.1007/s00228-021-03111-2
pubmed: 33624121
pmcid: 8275546
Powell JS, Pasi KJ, Ragni MV, et al. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013;369(24):2313–23.
doi: 10.1056/NEJMoa1305074
pubmed: 24304002
Santagostino E, Martinowitz U, Lissitchkov T, et al. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood. 2016;127(14):1761–9.
doi: 10.1182/blood-2015-09-669234
pubmed: 26755710
pmcid: 4825413
Signorovitch JE, Sikirica V, Erder MH, et al. Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research. Value Health. 2012;15(6):940–7.
doi: 10.1016/j.jval.2012.05.004
pubmed: 22999145
Signorovitch JE, Wu EQ, Yu AP, et al. Comparative effectiveness without head-to-head trials: a method for matching-adjusted indirect comparisons applied to psoriasis treatment with adalimumab or etanercept. Pharmacoeconomics. 2010;28(10):935–45.
doi: 10.2165/11538370-000000000-00000
pubmed: 20831302
Phillippo DM, Ades AE, Dias S, Palmer S, Abrams KR, Welton NJ. Methods for population-adjusted indirect comparisons in health technology appraisal. Med Decis Making. 2018;38(2):200–11.
doi: 10.1177/0272989X17725740
pubmed: 28823204
BiogenIdec. Clinical Study Report: B-LONG: an open-label, multi-center evaluation of the safety, pharmacokinetics, and efficacy of recombinant, long-acting coagulation factor IX Fc fusion protein (rFIXFc) in the prevention and treatment of bleeding in previously treated subjects with severe hemophilia B. 2012. Accessed from ema.europa.eu .
Zeileis A, Köll S, Graham N. Various versatile variances: an object-oriented implementation of clustered covariances in R. J Stat Softw. 2020;95(1):1–36.
doi: 10.18637/jss.v095.i01
Astermark J, Wojciechowski P, Aballea S, Hakimi Z, Nazir J, Klamroth R. Efficacy of rFIXFc versus rIX-FP for the treatment of patients with hemophilia B: matching-adjusted indirect comparison of B-LONG and PROLONG-9FP trials. J Blood Med. 2021;12:613–21.
doi: 10.2147/JBM.S312885
pubmed: 34285621
pmcid: 8286734