A new modeling approach allowing prediction and comparison of the long-term outcomes of treatments for hemophilia B. (January 2018)

• Record Type: Journal Article
• Authors: Keep, Marjolijn van; Hoxer, Christina Stentoft; Hemstock, Matthew; Groth, Andreas Velsing; Knight, Christopher
• Is Part Of: Journal of comparative effectiveness research. Volume 7:Number 1(2018)
• Page Start: 39
• Page End: 48
• Publication Date: 2018-01
• Subjects: alternative indirect treatment comparison -- factor IX -- hemophilia B -- modeling -- nonacog beta pegol -- pharmacokinetics -- prophylaxis; Medical care -- Research -- Periodicals; Medical care -- Research -- Evaluation -- Periodicals; Medical care -- Decision making -- Periodicals; Evidence-based medicine -- Periodicals; Medicine, Comparative -- Periodicals; 362.1072
• DOI: 10.2217/cer-2017-0028
• Languages: English
• ISSNs: 2042-6305

Abstract

Aim: To develop a modeling approach to compare clinical outcomes of nonacog beta pegol to a standard-acting factor IX (FIX) product.Methods: Regression analysis linked FIX activity to bleed rates. Pharmacokinetic parameters were used to estimate FIX activity over time. The probability of bleeds was estimated for both treatment arms. A Markov model estimated the presence of target joints and annualized bleed rates (ABRs).Results: Higher FIX activity showed reduced ABRs (p < 0.001). Target joints resulted in higher bleed rates (p < 0.001). When FIX activity levels and bleed risks were applied to the Markov model, ABRs for nonacog beta pegol and its comparator were 2.40 and 6.36, respectively.Conclusion: This model provides a starting point for assessing the added value of new FIX products.