Development and testing of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection and results from the Phase 3 HAVEN 3 study of emicizumab prophylaxis in persons with haemophilia A without FVIII inhibitors.
Development and testing of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection and results from the Phase 3 HAVEN 3 study of emicizumab prophylaxis in persons with haemophilia A without FVIII inhibitors.
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DOI：
10.1111/hae.14222
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发表时间：
2021-03
期刊：
影响因子：
--
通讯作者：
Mahlangu JN
中科院分区：
文献类型：
--
作者：
Kempton C;Trask P;Parnes A;Niggli M;Campinha-Bacote A;U Callaghan M;O'Connell N;Paz-Priel I;Mahlangu JN
Emicizumab是一种皮下（SC）给药的A型血友病（PwHA）预防药物。作为其临床开发的一部分，需要一种新的仪器来测量治疗满意度。描述静脉或皮下血友病注射满意度问卷（SQ‐ISHI）的开发及其随后在HAVEN 3研究受试者中的测试，以测量患者对emicizumab的满意度。为了开发SQ-ISHI，我们在五个定性研究机构进行了四轮面对面访谈。年龄≥12岁的中度或重度血友病A受试者，接受静脉注射因子VIII（FVIII）预防治疗，提供反馈以优化内容理解、易于完成和项目相关性。最终SQ‐ISHI由既往接受FVIII预防性治疗的HAVEN 3受试者完成;将基线评分与emicizumab预防性治疗第21或25周时的评分进行比较。63名HAVEN 3参与者有资格完成问卷，并在0（“完全不满意”）至10（“非常满意”）的量表上对其满意度进行评分。基线时对既往FVIII预防治疗的平均“总体满意度”为6. 9（95%置信区间[CI]：6. 2 - 7. 7），随访时（emicizumab治疗第21/25周）增至8. 8（95% CI：8. 4 - 9. 3）。在治疗半衰期满意度方面观察到最大改善（基线平均评分：5.8 [95% CI：4.9 - 6.6] vs随访时8.6 [95% CI：8.0 - 9.2]）。这些结果表明，与FVIII预防相比，emicizumab预防导致更高的治疗满意度，部分反映了emicizumab治疗负担较低，与其不频繁的SC给药相关。
Emicizumab is a subcutaneously (SC) administered prophylactic agent for persons with haemophilia A (PwHA). As part of its clinical development, a new instrument was required to measure treatment satisfaction. Describe development of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection (SQ‐ISHI) and its subsequent testing with HAVEN 3 study participants to measure patient satisfaction with emicizumab. To develop the SQ‐ISHI, we conducted four rounds of in‐person interviews at five qualitative research facilities. Participants aged ≥12 years with moderate or severe haemophilia A, receiving intravenous factor VIII (FVIII) prophylaxis, provided feedback to optimize content understanding, ease of completion and item relevance. The final SQ‐ISHI was completed by HAVEN 3 participants who previously received FVIII prophylaxis; baseline scores were compared with those at Week 21 or 25 of emicizumab prophylaxis. Sixty‐three HAVEN 3 participants were eligible to complete the questionnaire and rate their satisfaction on a scale of 0 (‘not at all satisfied’) to 10 (‘extremely satisfied’). Mean ‘overall satisfaction’ with previous FVIII prophylaxis at baseline was 6.9 (95% confidence interval [CI]: 6.2 to 7.7) increasing to 8.8 (95% CI: 8.4 to 9.3) at follow‐up (Week 21/25 of treatment with emicizumab). The greatest improvement was observed in satisfaction with treatment half‐life (mean score at baseline: 5.8 [95% CI: 4.9 to 6.6] vs 8.6 [95% CI: 8.0 to 9.2] at follow‐up). These results demonstrate that emicizumab prophylaxis leads to greater treatment satisfaction compared with FVIII prophylaxis, reflecting in part the low treatment burden of emicizumab associated with its infrequent, SC administration.
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影响因子：
158.5
作者：
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通讯作者：
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影响因子：
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作者：
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作者：
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作者：
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