Haemophilia Centre, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt/Main, Germany.
Biotest AG, Dreieich, Germany.
Haemophilia. 2020 May;26(3):467-477. doi: 10.1111/hae.13988. Epub 2020 Apr 15.
Prophylaxis with factor VIII (FVIII) concentrates in children with haemophilia A (HA) is current standard of care. The benefit of prophylactic treatment for adult HA patients is not commonly accepted.
To investigate the benefit of prophylaxis over on-demand treatment in adult and elderly patients with severe or non-severe HA in a real-life setting.
Data from 163 patients comprising 1202 patient-years were evaluated for 7.5 (±5.3) years. The effects on the annual bleeding rate (ABR, including spontaneous and traumatic bleeds) of treatment with a plasma-derived FVIII concentrate, the patient's age and disease severity were investigated. The effect of changing the treatment from on demand to continuous prophylaxis on the patients' ABRs was further analysed.
Prophylaxis had the greatest effect on the ABRs of patients of any age with severe or non-severe HA. The difference in ABR of all patients treated on demand (median 31.4; interquartile range (IQR) 27.6; N = 83) compared with those treated prophylactically (median 1.3; IQR 3.6; N = 122) was statistically significant (P < .05), even for patients with non-severe HA (median 8.4; IQR 15.5; N = 11) vs median 1.5; IQR 4.2 (N = 17), P < .05). Patients, aged up to 88 years, switching from on demand to continuous prophylaxis showed the lowest median ABR (1.1; N = 51) after their regimen change.
Any (even low-frequency) prophylaxis results in lower ABR than on-demand treatment. Patients switching to prophylaxis benefitted the most, irrespective of age or HA severity. Prophylactic treatment-even tertiary-is the regimen of choice for patients of any age, including elderly patients, with severe or non-severe HA.
在甲型血友病（HA）患儿中，使用因子 VIII（FVIII）浓缩物进行预防治疗是目前的标准治疗方法。但预防性治疗对成年 HA 患者的益处尚未得到普遍认可。
在真实环境中，研究预防治疗对成年和老年重度或非重度 HA 患者相对于按需治疗的益处。
共纳入 163 例患者（1202 患者年）的数据，平均随访 7.5（±5.3）年。评估了血浆源性 FVIII 浓缩物治疗对年出血率（ABR，包括自发性和创伤性出血）的影响，以及患者年龄和疾病严重程度的影响。进一步分析了从按需治疗转为连续预防治疗对患者 ABR 的影响。
预防治疗对任何年龄的重度或非重度 HA 患者的 ABR 影响最大。与按需治疗（中位数 31.4；四分位距（IQR）27.6；N=83）相比，接受预防治疗（中位数 1.3；IQR 3.6；N=122）的所有患者的 ABR 差异具有统计学意义（P<.05），甚至对非重度 HA 患者（中位数 8.4；IQR 15.5；N=11）与中位数 1.5；IQR 4.2（N=17）相比也具有统计学意义（P<.05）。在方案改变后，年龄在 88 岁以下的患者从按需治疗转为连续预防治疗后，其 ABR 的中位数最低（1.1；N=51）。
任何（即使是低频率）的预防治疗都会导致 ABR 低于按需治疗。无论年龄或 HA 严重程度如何，转为预防治疗的患者获益最大。对于任何年龄的患者，包括老年患者，无论疾病严重程度如何，预防治疗-即使是三级预防-都是首选方案。
