University of Colorado Anschutz Medical Campus, Aurora, CO, USA.
Lund University and Skåne University Hospital, Lund, Sweden.
J Thromb Haemost. 2017 Nov;15(11):2115-2124. doi: 10.1111/jth.13811. Epub 2017 Oct 10.
Essentials High-quality data are lacking on use of prophylaxis in adults with hemophilia and arthropathy. SPINART was a 3-year randomized clinical trial of late/tertiary prophylaxis vs on-demand therapy. Prophylaxis improved function, quality of life, activity and pain but not joint structure by MRI. Prophylaxis improves function but must start before joint bleeding onset to prevent arthropathy.
Background Limited data exist on the impact of prophylaxis on adults with severe hemophilia A and pre-existing joint disease. Objectives To describe 3-year bleeding, joint health and structure, health-related quality-of-life (HRQoL) and other outcomes from the open-label, randomized, multinational SPINART study. Patients/Methods Males aged 12-50 years with severe hemophilia A, ≥ 150 factor VIII exposure days, no inhibitors and no prophylaxis for > 12 consecutive months in the past 5 years were randomized to sucrose-formulated recombinant FVIII prophylaxis or on-demand therapy (OD). Data collected included total and joint bleeding events (BEs), joint structure (magnetic resonance imaging [MRI]), joint health (Colorado Adult Joint Assessment Scale [CAJAS]), HRQoL, pain, healthcare resource utilization (HRU), activity, and treatment satisfaction. Results Following 3 years of prophylaxis, adults maintained excellent adherence, with a 94% reduction in BEs despite severe pre-existing arthropathy; 35.7% and 76.2% of prophylaxis participants were bleed-free or had fewer than two BEs per year, respectively. As compared with OD, prophylaxis was associated with improved CAJAS scores (least squares [LS] mean, - 0.31 [n = 42] versus + 0.63 [n = 42]) and HAEMO-QoL-A scores (LS mean, + 3.98 [n = 41] versus - 6.00 [n = 42]), less chronic pain (50% decrease), and approximately two-fold less HRU; activity, Euro QoL-5D-3L (EQ-5D-3L) scores and satisfaction scores also favored prophylaxis. However, MRI score changes were not different for prophylaxis versus OD (LS mean, + 0.79 [n = 41] versus + 0.96 [n = 38]). Conclusions Over a period of 3 years, prophylaxis versus OD in adults with severe hemophilia A and arthropathy led to decreased bleeding, pain, and HRU, better joint health, activity, satisfaction, and HRQoL, but no reduction in structural arthropathy progression, suggesting that pre-existing joint arthropathy may be irreversible.
关于预防治疗对患有严重血友病 A 和先前存在关节疾病的成年人的影响，数据有限。
描述为期 3 年的出血、关节健康和结构、健康相关生活质量 (HRQoL) 以及来自开放标签、随机、多国 SPINART 研究的其他结果。
患者/方法：年龄在 12-50 岁之间、患有严重血友病 A、因子 VIII 暴露天数≥150 天、过去 5 年内无抑制剂且过去 12 个月内无预防治疗>12 个月的男性，随机接受蔗糖配方重组 FVIII 预防治疗或按需治疗 (OD)。收集的数据包括总出血事件和关节出血事件 (BEs)、关节结构 (磁共振成像 [MRI])、关节健康 (科罗拉多成人关节评估量表 [CAJAS])、HRQoL、疼痛、医疗保健资源利用 (HRU)、活动和治疗满意度。
在接受 3 年预防治疗后，尽管存在严重的先前存在的关节病，但成年人仍保持了极好的依从性，BE 减少了 94%；35.7%和 76.2%的预防治疗参与者分别无出血或每年出血少于两次。与 OD 相比，预防治疗与 CAJAS 评分改善相关（最小二乘[LS]均值，-0.31[42 名参与者]与+0.63[42 名参与者]）和 HAEMO-QoL-A 评分改善相关（LS 均值，+3.98[41 名参与者]与-6.00[42 名参与者]）、慢性疼痛减少（减少 50%）、HRU 减少约两倍；活动、Euro QoL-5D-3L（EQ-5D-3L）评分和满意度评分也有利于预防治疗。然而，预防治疗与 OD 相比，MRI 评分变化无差异（LS 均值，+0.79[41 名参与者]与+0.96[38 名参与者]）。
在 3 年期间，与 OD 相比，严重血友病 A 和关节病成人中接受预防治疗导致出血、疼痛和 HRU 减少，关节健康、活动、满意度和 HRQoL 改善，但结构关节病进展无减少，表明先前存在的关节病可能是不可逆转的。
