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. 2022 May;53(4):934-944.
doi: 10.1007/s11239-021-02599-w. Epub 2021 Nov 13.

Italian experience with rVIII-single chain: a survey of patients with haemophilia A and their physicians

Alessandra Borchiellini  1 , Giancarlo Castaman  2 , Giulio Feola  3 , Antonietta Ferretti  4 , Paola Giordano  5 , Matteo Luciani  6 , Giuseppe Malcangi  7 , Maurizio Margaglione  8 , Angelo Claudio Molinari  9 , Berardino Pollio  10 , Angiola Rocino  11 , Cristina Santoro  12 , Michele Schiavulli  13 , Ezio Zanon  14
Affiliations

Italian experience with rVIII-single chain: a survey of patients with haemophilia A and their physicians

Alessandra Borchiellini et al. J Thromb Thrombolysis. 2022 May.

Abstract

rVIII-SingleChain is indicated for treatment and prophylaxis of bleeding in patients with haemophilia A (HA). The safety and efficacy of rVIII-SingleChain have previously been shown in the AFFINITY clinical trial programme. This survey evaluated clinical experience following a switch to rVIII-SingleChain from the perspective of both physicians and patients. A web-based survey (July-September 2019) involving 14 Haemophilia Treatment Centres (HTCs) collected data about HA patients who were under treatment with rVIII-SingleChain for ≥ 12 months, as reported by their physicians. In addition, about half of these patients were separately interviewed. Out of 91 patients receiving rVIII-SingleChain in the 14 participating HTCs, 48 had been treated for ≥ 12 months; among those 48, 38% were ≤ 18 years, 37% 19-40 years and 25 % ≥ 41 years; 73% of them had severe HA and 85% were being treated with prophylactic therapy. Twenty-six patients accepted to be separately interviewed: mean age was 30 years; 62% had severe HA and 85% were receiving prophylaxis. Focusing on those patients who were already in prophylaxis with prior FVIII (all but one with recombinant factors), infusion frequency was significantly reduced from 3-2 per week following the switch to rVIII-SingleChain (mean, 2.74 vs. 2.44, respectively; p=0.013), as reported by physicians; the rate of patients needing 3 infusions per week dropped from 74% with previous products to 44% with rFVIII-SingleChain. The annual mean factor consumption was 4740 IU/Kg (median, 4500 IU/Kg; min, 2.215 IU/Kg; max, 7.200 IU/Kg) with prior product and 4320 IU/Kg (median, 4320 IU/Kg; min, 2.215 IU/Kg; max, 6.646 IU/Kg) with rVIII-SingleChain. Both physicians and patients reported a significant reduction in annual total bleeding rates with rVIII-SingleChain compared with prior product (mean 2.15-0.96 and 2.46-0.71 events/year, p = 0.031 and p = 0.018, respectively). Mean satisfaction ratings (from 1; dissatisfied, to 5; very satisfied) for rVIII-SingleChain were quite high for both physicians (4.14, 86% satisfied/very satisfied) and patients (4.18, 86% satisfied/very satisfied). This survey suggested that switching to rVIII-SingleChain allowed patients to reduce their injection frequency without increasing factor consumption or compromising clinical results. Both physicians and patients reported a positive experience with rVIII-SingleChain after 1 year of treatment.

Keywords: Haemophilia A; Injection frequency; rVIII-SingleChain.

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Conflict of interest statement

AB received honoraria for speaker’s bureau and/or participation in Advisory Boards from Bayer, CSL Behring, Kedrion, Novo Nordisk, Roche, Sobi, Takeda. GC received honoraria for speaker’s bureau and/or participation in Advisory Boards for Alexion. Bayer, Biomarin, CSL Behring, Grifols, Kedrion, LFB, NovoNordisk, Roche, Sanofi, Sobi, Takeda, Uniqure, Werfen. AR acted as paid consultant/member of Advisory Board/speaker for Bayer, CSL Behring, Kedrion, NovoNordisk, Roche, Sobi, Takeda. CS received honoraria for speaker’s bureau and/or participation in Advisory Boards from Bayer, Takeda, CSL Behring, Sobi, Roche, NovoNordisk.

Figures

Fig. 1
Fig. 1
Survey design and patients’ disposition
Fig. 2
Fig. 2
Number of weekly infusions before and after treatment with rVIII-SingleChain
Fig. 3
Fig. 3
Impact of haemophilia on everyday life from the patients’ perspective
See this image and copyright information in PMC

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