The need to reduce the burden of injections, and improve adherence and clinical outcomes in haemophilia A led to the development of recombinant FVIII products endowed with an extended plasma half-life (EHL-rFVIII) in comparison with standard half-life products (SHL-rFVIII). Lack of head-to-head studies makes difficult to grasp the relative value of each treatment option. We conducted a combined evaluation of the individual pivotal trials in order to assess between-product differences regarding the reported efficacy results and FVIII consumption. We evaluated 4 EHL-rFVIII products available to treat patients with haemophilia A without inhibitors and also a SHL-rFVIII as a comparator. In the frame of these clinical studies, all the EHL-rFVIII products showed a decrease in the injection burden coupled with good clinical efficacy, even though there were between-product differences in terms of reduction in injection frequencies. Further, between-product differences in terms of weekly/yearly consumption of rFVIII expressed in IU/Kg were identified, suggesting a different economic impact for the different EHL-rFVIII products in the context of comparable clinical efficacy. The present findings based upon the review of pivotal studies done in the frame of a highly selected clinical scenario should be integrated with real-life data.

Comparative analysis of the pivotal studies of extended half-life recombinant FVIII products for treatment of haemophilia A / Mannucci, P. M.; Cortesi, P. A.; Di Minno, M. N. D.; Sano, M.; Mantovani, L. G.; Di Minno, G.. - In: HAEMOPHILIA. - ISSN 1351-8216. - 27:4(2021), pp. e422-e433. [10.1111/hae.14313]

Comparative analysis of the pivotal studies of extended half-life recombinant FVIII products for treatment of haemophilia A

Mannucci P. M.;Di Minno M. N. D.;Mantovani L. G.;Di Minno G.
2021

Abstract

The need to reduce the burden of injections, and improve adherence and clinical outcomes in haemophilia A led to the development of recombinant FVIII products endowed with an extended plasma half-life (EHL-rFVIII) in comparison with standard half-life products (SHL-rFVIII). Lack of head-to-head studies makes difficult to grasp the relative value of each treatment option. We conducted a combined evaluation of the individual pivotal trials in order to assess between-product differences regarding the reported efficacy results and FVIII consumption. We evaluated 4 EHL-rFVIII products available to treat patients with haemophilia A without inhibitors and also a SHL-rFVIII as a comparator. In the frame of these clinical studies, all the EHL-rFVIII products showed a decrease in the injection burden coupled with good clinical efficacy, even though there were between-product differences in terms of reduction in injection frequencies. Further, between-product differences in terms of weekly/yearly consumption of rFVIII expressed in IU/Kg were identified, suggesting a different economic impact for the different EHL-rFVIII products in the context of comparable clinical efficacy. The present findings based upon the review of pivotal studies done in the frame of a highly selected clinical scenario should be integrated with real-life data.
2021
Comparative analysis of the pivotal studies of extended half-life recombinant FVIII products for treatment of haemophilia A / Mannucci, P. M.; Cortesi, P. A.; Di Minno, M. N. D.; Sano, M.; Mantovani, L. G.; Di Minno, G.. - In: HAEMOPHILIA. - ISSN 1351-8216. - 27:4(2021), pp. e422-e433. [10.1111/hae.14313]
Comparative analysis of the pivotal studies of extended half-life recombinant FVIII products for treatment of haemophilia A / Mannucci, P. M.; Cortesi, P. A.; Di Minno, M. N. D.; Sano, M.; Mantovani, L. G.; Di Minno, G.. - In: HAEMOPHILIA. - ISSN 1351-8216. - 27:4(2021), pp. e422-e433. [10.1111/hae.14313]
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/864193
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 13
  • ???jsp.display-item.citation.isi??? 14
social impact