Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A

التفاصيل البيبلوغرافية
العنوان: Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A
المؤلفون: José Luis Poveda, Georg Goldmann, Ramiro Núñez, Björn Vandewalle, Valeska Andreozzi, Karin Kurnik, Sandra Santos, Santiago Bonanad
المصدر: ADVANCES IN THERAPY
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
instname
Advances in Therapy
بيانات النشر: SPRINGER, 2021.
سنة النشر: 2021
مصطلحات موضوعية: Adult, medicine.medical_specialty, Matching (statistics), MAIC, Adolescent, Haemophilia A, Octocog alfa, Hemorrhage, Hemophilia A, Immunotherapy, Adoptive, Efmoroctocog alfa, Annualized bleeding rate, law.invention, law, rVIII-SingleChain, Internal medicine, medicine, Humans, Pharmacology (medical), Propensity Score, FVIII consumption, Original Research, Factor VIII, business.industry, General Medicine, Odds ratio, Matching-adjusted indirect comparison, medicine.disease, Recombinant Proteins, Lonoctocog alfa, Indirect comparison, Clinical trial, Relative risk, Recombinant DNA, business, Prophylactic treatment
الوصف: Introduction Given the relatively small number of patients with haemophilia A, head-to-head comparisons between recombinant FVIII (rFVIII) products are difficult to conduct. This study compared the efficacy and consumption of rVIII-SingleChain (lonoctocog alfa, AFSTYLA®) with rAHF-PFM (octocog alfa, Advate®) and rFVIIIFc (efmoroctocog alfa, Elocta®), for the prophylaxis and treatment of bleeding episodes in previously treated adolescents/adults with severe haemophilia A, through a matching-adjusted indirect comparison (MAIC). Methods A systematic literature review identified published clinical trials for rAHF-PFM and rFVIIIFc. Individual patient data for rVIII-SingleChain were used to match baseline patient characteristics to those from published trials, using an approach similar to propensity score weighting. After matching, annualized bleeding rates (ABR), percentage of patients with zero bleeds, and rFVIII consumption were compared across trial populations. Results Published data were identified from two rAHF-PFM trials and one rFVIIIFc trial. rVIII-SingleChain had similar ABR (risk ratio [RR]: 0.74 [0.16; 3.48]; RR: 1.18 [0.85; 1.65]) and percentage of patients with zero bleeds (odds ratio [OR]: 1.34 [0.56; 3.22]; OR: 0.78 [0.47; 1.31]) versus rAHF-PFM and rFVIIIFc, respectively. Annual rVIII-SingleChain consumption was significantly lower than rAHF-PFM (mean difference: − 1507.66 IU/kg/year [− 2011.71; − 1003.61]) and equivalent to rFVIIIFc (RR: 0.96 [0.62; 1.49]). Conclusion Although limited to published information for comparator trials, these results suggest that with an annualized rFVIII consumption comparable to rFVIIIFc, but significantly lower than rAHF-PFM, routine prophylaxis with rVIII-SingleChain is able to maintain a similar ABR and percentage of patients with zero bleeds, attesting to the long-acting nature of rVIII-SingleChain. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-021-01853-0.
Plain Language Summary It is difficult to directly compare different recombinant FVIII products in head-to-head studies because there are few patients with haemophilia A. This study aimed to indirectly compare the efficacy and consumption of different recombinant FVIII products in the prophylactic treatment of haemophilia A using published clinical data. A proven method for performing indirect comparisons of products is referred to as a matching-adjusted indirect comparison. Using this approach, we were able to compare rVIII-SingleChain with two other recombinant FVIII products (rAHF-PFM and rFVIIIFc). Our results suggest that annual FVIII consumption with rVIII-SingleChain is comparable to rFVIIIFc, but is significantly lower than rAHF-PFM, while maintaining a similar bleeding rate. These results highlight the long-acting nature of the product. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-021-01853-0.
وصف الملف: application/pdf
تدمد: 0741-238X
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ce20f9c20127f9c9857dcc4094b03f40Test
https://fundanet.iislafe.san.gva.es/publicaciones/ProdCientif/PublicacionFrw.aspx?id=14560Test
حقوق: OPEN
رقم الانضمام: edsair.doi.dedup.....ce20f9c20127f9c9857dcc4094b03f40
قاعدة البيانات: OpenAIRE