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.
Highlights
Haemophilia A (HA) is an X-linked congenital bleeding disorder resulting from mutations in the gene encoding the coagulation factor VIII (FVIII) [1]
The most frequent previous medication used for prophylaxis by the 35 patients was a full length 2nd generation recombinant FVIII (rFVIII) from baby hamster kidney (BHK) cells (Helixate NG) (66 ); 17% were treated with 3rd generation rFVIII; 14% with other 1st/2nd generation rFVIII, and one patients (3%) with a plasma–derived FVIII product (3 %)
The results reinforced the good clinical performances of rVIII-SingleChain as previously reported in the pivotal clinical trials, namely the significant proportion of patients able to follow a twice per week prophylaxis regimen, without an increase in annual total FVIII
Summary
Haemophilia A (HA) is an X-linked congenital bleeding disorder resulting from mutations in the gene encoding the coagulation factor VIII (FVIII) [1]. Regular prophylactic factor VIII replacement is currently the standard treatment for patients with severe haemophilia A (defined as FVIII levels < 1 IU/dL), and its introduction has greatly improved the prognosis, life expectancy, and quality of life (QoL) of subjects with haemophilia, preventing spontaneous bleeding episodes into the joints or the muscles, and the developing of haemophilic arthropathy [2, 3]. Issues related to the burden of treatment, due to the need for frequent injections, and to the serious complication of inhibitor development, still calls for the need of effective new agents with improved stability and reduced immunogenicity. Thanks to its innovative features, rVIII-SingleChain allows for less frequent prophylaxis regimes (i.e. 2–3 times per week) compared to standard half-life (SHL) rFVIII products, which typically require 3–4 infusions per week, with major advantages in terms of burden of care and compliance to treatment for patients [6]. Since even most effective current prophylactic regimens may not completely prevent joint disease, in a long-term perspective adherence to therapy has important implications for the functional outcome of haemophilic patients
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