Physical activity and bleeding outcomes among people with severe hemophilia on extended half‐life or conventional recombinant factors

Abstract

Background Few have assessed physical activity (PA) and annual bleed rates (ABRs) among people with hemophilia on extended half‐life (EHL) factors (recombinant factor VIII Fc [rFVIIIFc]/recombinant factor IX Fc [rFIXFc]) and conventional factors (recombinant factor VIII [rFVIII]/recombinant factor IX [rFIX]). Objective To assess changes in PA and ABR at consecutive annual visits in individuals with severe hemophilia A and B (HA/HB) on prophylactic treatment with rFVIIIFc/rFIXFc versus rFVIII/rFIX. Patients/Methods We conducted a retrospective chart review of 344 people with severe HA/HB (ages 6‐35) receiving prophylaxis with rFVIIIFc/rFIXFc (EHL factors) or rFVIII/rFIX (conventional factors) for ≥6 months in 2014‐2015. Differences in changes in outcomes from 2014 to 2015 were compared across the treatment groups. Results Baseline characteristics and adherence to the prophylactic regimen were similar across the treatment groups. Greater increase in weekly PA frequency and duration were observed among all EHL groups, except for children treated with rFIXFc. The increase in PA frequency was greater among the children on rFVIIIFc group, adults on rFVIIIFc group, and adults on rFIXFc group by 1.2, 1.2, and 1.4 events/week, respectively, compared to their rFVIII/rFIX counterparts. The increases in PA duration were 44, 60, and 80 min/wk greater among the children on rFVIIIFc, adults on rFVIIIFc, and adults on rFIXFc groups, respectively. Larger reductions in total ABR were observed in children and adults treated with rFVIIIFc compared to rFVIII (0.4 and 0.7 fewer bleeds). Larger reductions were also observed in spontaneous ABR in adult rFVIIIFc and rFIXFc groups (0.8 and 0.3 fewer bleeds, respectively). Conclusions This study suggests that rFVIIIFc/FIXFc agents can positively impact PA while maintaining low ABRs.

Efmoroctocog Alfa: A Review in Haemophilia A

“…A large number of retrospective real-world studies have confirmed the efficacy of efmoroctocog alfa in the prophylaxis of bleeding observed in phase III clinical trials. The following discussion, however, only focuses on those involving > 30 PTPs from one or more European countries [31,[36][37][38][39][40][41][42][43][44][47][48][49] or the USA [50][51][52].…”

“…Table 5 Real-world effectiveness of prophylaxis with efmoroctocog alfa versus other recombinant FVIII products in patients with haemophilia A: results of multicentre, retrospective, chart-review studies from Europe and the USA Pts had been treated for ≥ 8 wks [36,38,51] or ≥ 6 months [52] at the time of data collection ABR annualized bleeding rate, BL baseline, CFC clotting factor consumption, IF injection frequency, IU international unit, NR not reported, pts patients, rFVIII recombinant FVIII, rFVIII-FS sucrose-formulated rFVIII, wk(s) week(s) *p = 0.001 vs rFVIII a Median values, except for Shrestha et al [52] The efficacy and safety of efmoroctocog alfa for the longterm prophylaxis, acute treatment and perioperative management of bleeding in male PTPs of all ages with severe haemophilia A was initially demonstrated in phase III clinical trials, including two parent studies (A-LONG and Kids A-LONG) and their extension study (ASPIRE) (Sects. 3.1, 4).…”

Aerobic exercise in patients with haemophilia: A systematic review on safety, feasibility and health effects

“…Further, well supervised sport therapies do not cause higher bleeding rates, emphasizing that regular PA may even reduce the frequency of bleeding events 11,16,17 . Prior literature showed that recent replacement strategies with extended half‐life factors positively impact PA as the trough level is substantially increased 16 . Hence, an expert consensus exists that planned and professionally supervised PA should be recommended for PwH 10–13,18 …”

“…These changes ultimately improve the patients’ quality of life. Further, well supervised sport therapies do not cause higher bleeding rates, emphasizing that regular PA may even reduce the frequency of bleeding events 11,16,17 . Prior literature showed that recent replacement strategies with extended half‐life factors positively impact PA as the trough level is substantially increased 16 .…”

“…11,16,17 Prior literature showed that recent replacement strategies with extended half-life factors positively impact PA as the trough level is substantially increased. 16 Hence, an expert consensus exists that planned and professionally supervised PA should be recommended for PwH. [10][11][12][13]18 However, there is a gap of knowledge regarding health-related effects of aerobic exercise (AE) in PwH.…”