Clinical experience with Optivate^®, high‐purity factor VIII (FVIII) product with von Willebrand factor (VWF) in young children with haemophilia A

Abstract

Optivate® is a high-purity FVIII/VWF product. Its safety, tolerability and efficacy in subjects ≥ 12 years have been demonstrated. This study was undertaken to assess Optivate® in children with haemophilia A. Twenty-five children, including one PUP (previously untreated patient), aged 1-6 years (mean 4.67 years) were treated with Optivate® for 26 weeks. Inhibitors were assessed every 3 months and viral status at the study start and end. Prophylaxis was used by five boys and on demand by twenty. The mean number of bleeds in the study was lower compared to the same period pre-study (12.0/child vs. 16.2/child), with fewer bleeds (P < 0.05) in the prophylactic subgroup (8.0/child) compared with the on-demand sub-group (13.4/child). Fourteen major bleeds were reported, all by the on-demand sub-group. Children on prophylaxis were administered a mean of 59.4 infusions; on-demand group 35.1 infusions. A total of 998 infusions were used with a mean dose of 29.1 IU kg⁻¹, and a mean of 38.6 exposure days (ED). Children < 4 years used higher doses, and reported fewer bleeds than older children. Children's Parents/Guardians rated Optivate® as helpful or very helpful in controlling 97.5% of bleeds by the prophylactic group, and in 98.5% of the bleeds in the on-demand group. Only 5 of 101 ADRs were treatment-related events (5%), all were mild and non-serious. There were no clinically significant changes in vital signs, viral transmissions or inhibitors. In young children Optivate® was well tolerated, safe and efficacious.

Thrombotic adverse events to coagulation factor concentrates for treatment of patients with haemophilia and von Willebrand disease: a systematic review of prospective studies

“…Types and brands of factor concentrates administered in the selected studies are shown in Table 1. Forty‐five studies [20–64] reported patients with HA receiving FVIII concentrates (Table 2), 15 studies [65–79] patients with HB treated with FIX concentrates (Table 3) and 11 studies [80–90] VWD patients treated with VWF containing products (Table 4). Owing to the peculiar pathophysiologic situation, six studies [91–96] reporting haemophilia patients who had central venous access devices (CVAD) in order to facilitate long‐term regular concentrate administration (prophylaxis, immune tolerance induction) were analyzed separately (Table 5).…”

Searching for the role of primary prophylaxis in preventing inhibitor development in hemophilia A

“…Of them, 24 were further excluded because the relevant data were unavailable or because analyzed patients were included in other published studies or because the study design did not permit a pooled analysis of the data. Finally, 27 studies with usable information (15 prospective, 11 retrospective and one retrospective/prospective), published between 1996 and 2011, were included in this systematic review [8,10,12,14–37], as reported in Table 1. Statistical analysis was performed using the chi‐square test.…”

Pharmacokinetic profile of Optivate® (high‐purity factor VIII/von Willebrand factor concentrate) in treating von Willebrand disease

“…Overall Optivate was well tolerated. There have been no reports of inhibitor development to FVIII and/or VWF with Optivate in this study or any of the other clinical studies [2,[6][7][8][9] or from postmarketing experience to date.…”

“…Optivate is licensed in several countries worldwide for the treatment of Haemophilia A in both adults and children [6][7][8][9]. This letter reviews data from a BPL sponsored single centre, randomized pharmacokinetic (PK) study comparing Optivate and Haemate P (also known as Humate P) in VWD patients.…”

“…Therefore, maintaining lower postinfusion FVIII levels could possibly lower the risk of thrombosis. To date, there have been no adverse reactions with a thrombotic aetiology from either formal clinical trials or reporting of spontaneous adverse drug reactions with Optivate [2,[6][7][8][9]. In addition, thrombogenicity data gathered from a separate Optivate study in 11 VWD patients (unpublished data) concluded there was no clinical evidence of thrombosis.…”