Oka Gaëlle, Roussel-Robert Valérie, Levivien Clara, Lopez Isabelle, Pieragostini Rémi
Pharmacy Department, Cochin Hospital (Assistance Publique - Hôpitaux de Paris), Paris, France.
Haemophilia Treatment Centre, Cochin Hospital (Assistance Publique - Hôpitaux de Paris), Paris, France.
Haemophilia. 2023 May;29(3):709-715. doi: 10.1111/hae.14755. Epub 2023 Feb 7.
Since the approval by the EMA of emicizumab for the care of severe haemophilia A without inhibitor, most of the patients of our haemophilia treatment centre started this new treatment. Thanks to the setting of a therapeutic patient education program including three pharmaceutical consultations (PC), we could follow patients' lifestyle evolution.
The study aimed to assess the perceived clinical evolution, quality of life and treatment satisfaction of patients after 1 year of emicizumab therapy in real-life settings.
The study was observational, retrospective and monocentric. Every patient over 18 years old receiving emicizumab from June 2020 and who underwent the 3 PC until March 2022 were included. The clinical evolution was self-estimated by patients with zero-to-six scales before versus 1 year after emicizumab, according to the following parameters: general health state, pain and bleedings (spontaneous or post-traumatic, and patients' identification ability). Patients' quality of life was also estimated with the EQ-5D-3L survey. Their satisfaction, graduated with a zero-to-ten scale, and treatment management were reported during the third PC.
Thirty-eight patients were enrolled. Their general health state improved significantly (p = .0023) with an EQ-5D-3L score at 69.6 (±19.4) out of 100. Although chronic pains remained a persistent issue for 33 (86.8%) patients, their intensity was significantly decreasing after 1 year. Perceived frequency of bleedings was significantly reduced too. On average, the satisfaction of emicizumab therapy was 9.1 (± 1.02) out of 10.
After 1 year of emicizumab therapy, the general health state estimated by patients improved, the pain and the perceived frequency of bleedings diminished. Overall, this treatment received a high patients' satisfaction rate.
自欧洲药品管理局（EMA）批准emicizumab用于治疗无抑制剂的重度A型血友病以来，我们血友病治疗中心的大多数患者都开始了这种新疗法。通过设立一个包括三次药物咨询（PC）的治疗患者教育项目，我们能够跟踪患者的生活方式变化。
本研究旨在评估在现实生活环境中，emicizumab治疗1年后患者的临床感知进展、生活质量和治疗满意度。
本研究为观察性、回顾性和单中心研究。纳入了2020年6月起接受emicizumab治疗且在2022年3月前接受了3次药物咨询的所有18岁以上患者。临床进展由患者在emicizumab治疗前与治疗1年后，根据以下参数用0至6分进行自我评估：总体健康状况、疼痛和出血情况（自发性或创伤后出血，以及患者的识别能力）。患者的生活质量也通过EQ-5D-3L调查问卷进行评估。在第三次药物咨询期间报告了他们的满意度（用0至10分进行评分）和治疗管理情况。
共纳入38例患者。他们的总体健康状况显著改善（p = 0.0023），EQ-5D-3L评分为69.6（±19.4）（满分100分）。尽管慢性疼痛仍然是33例（86.8%）患者持续存在的问题，但1年后疼痛强度显著降低。出血的感知频率也显著降低。平均而言，emicizumab治疗的满意度为9.1（±1.02）（满分10分）。
emicizumab治疗1年后，患者自我评估的总体健康状况有所改善，疼痛和出血的感知频率降低。总体而言，这种治疗获得了较高的患者满意度。
