Kahr Rasmussen Niklas, Berg Birgitte, Christiansen Anne Sia Louise, Neergaard Jesper Skov, Ter-Borch Gitte, Hildebrand Emily A, Gonczi Maya, Sparre Thomas
Medical & Science Innovation, Novo Nordisk A/S, Søborg, Denmark.
Clinical Operations, Novo Nordisk A/S, Søborg, Denmark.
Patient Prefer Adherence. 2024 Aug 14;18:1713-1727. doi: 10.2147/PPA.S470091. eCollection 2024.
Concizumab is a once-daily prophylactic treatment developed for patients with hemophilia A or B (HA/HB) with or without inhibitors. It is the first treatment for hemophilia patients to be delivered subcutaneously using a pre-filled, multi-dose pen-injector with a 4 mm, 32 G needle.
To investigate patient and caregiver handling and preference for the concizumab pen-injector compared with current injection systems used to treat hemophilia.
This preference and handling study was conducted in accordance with authority guidelines for approval of new devices and included adults and adolescents with HA/HB with or without inhibitors and caregivers currently administering factor replacement therapy or factor VIII mimetic (emicizumab) therapy. All participants underwent a training session, followed by a test session during which participants independently administered a single pen-injection into an injection pad or manikin. Time to train, time to prepare and inject, and number of complete independent injections handling the pen were assessed. Participants evaluated handling and preference via the Hemophilia Device Handling and Preference Assessment Questionnaire.
80 participants (44 adults, 21 adolescents, 15 caregivers) currently using factor replacement therapy (n=41, 51%) or emicizumab (n=39, 49%) participated. Average training time and time to complete an injection were 7 min 49s and 1 min 21s. In total, 98% of independent complete injections were achieved at first attempt. 98% (n=78; 95% confidence interval [CI] 91-100%) of participants assessed the pen-injector as either "easy" or "very easy" to use. 88% of participants preferred the pen-injector (n=70; 95% CI 78-94%) over their current injection system, and 9% (n=7) reported "no preference".
Participants found the concizumab pen-injector easy to learn and easy to use and preferred it over their current injection systems.
康西珠单抗是一种为患有或不患有抑制剂的甲型或乙型血友病（HA/HB）患者开发的每日一次预防性治疗药物。它是第一种使用带有4毫米、32G针头的预填充多剂量笔式注射器皮下给药的血友病患者治疗药物。
与目前用于治疗血友病的注射系统相比，调查患者和护理人员对康西珠单抗笔式注射器的操作情况和偏好。
这项偏好和操作研究是按照新设备批准的权威指南进行的，纳入了患有或不患有抑制剂的HA/HB成人和青少年以及目前正在进行因子替代治疗或因子VIII模拟物（依美珠单抗）治疗的护理人员。所有参与者都参加了一次培训课程，随后进行了一次测试课程，在此期间参与者独立地将一次笔式注射剂注射到注射垫或人体模型中。评估了培训时间、准备和注射时间以及操作笔式注射器进行的完全独立注射次数。参与者通过血友病设备操作和偏好评估问卷对操作情况和偏好进行评估。
80名目前正在使用因子替代治疗（n=41，51%）或依美珠单抗（n=39，49%）的参与者（44名成人、21名青少年、15名护理人员）参与了研究。平均培训时间和完成一次注射的时间分别为7分49秒和1分21秒。总共98%的独立完全注射在首次尝试时完成。98%（n=78；95%置信区间[CI]91-100%）的参与者将笔式注射器评估为使用“容易”或“非常容易”。88%的参与者（n=70；95%CI 78-94%）比他们目前的注射系统更喜欢笔式注射器，9%（n=7）报告“无偏好”。
参与者发现康西珠单抗笔式注射器易于学习和使用，并且比他们目前的注射系统更喜欢它。
