ArticleHaemophilia : the official journal of the World Federation of Hemophilia2021
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.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 16 papers, 2 of them syntheses that pooled it.
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Who cites it
16 citing papers in PubMed, 2 syntheses or guidelines pooled it, 23 citations in OpenAlex.
- Patient-reported outcome measures for medication treatment satisfaction: a systematic review of measure development and measurement properties.BMC medicine · 2024Pooled it
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- Health-Related Quality of Life, Physical Activity and Joint Health in People With Severe Haemophilia A Receiving Emicizumab: Results From the Phase IV HemiNorth 2 Study.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Hemophilia in Mexico: Updated Consensus Recommendations on Diagnosis, Treatment and Gene Therapy.Diseases (Basel, Switzerland) · 2026Review
- Content Validity of the Hemophilia Device Handling and Preference Assessment Questionnaire - A Cognitive Debrief Study in Patients with Hemophilia and Their Caregivers.Patient preference and adherence · 2025Article
- Article
- Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review.Research and practice in thrombosis and haemostasis · 2024Article
- The Concizumab Pen-Injector is Easy to Use and Preferred by Hemophilia Patients and Caregivers: A Usability Study Assessing Pen-Injector Handling and Preference.Patient preference and adherence · 2024Article
- Serious bleeds in pediatric persons with hemophilia A on emicizumab prophylaxis.Research and practice in thrombosis and haemostasis · 2023Article
- Review
- Radiosynovectomy for the Treatment of Chronic Hemophilic Synovitis: An Old Technique, but Still Very Effective.Journal of clinical medicine · 2022Review
- Patient preference for emicizumab versus prior factor therapy in people with haemophilia A: Results from the HAVEN 3 and HAVEN 4 studies.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Corrigendum.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Patients' Perception of the Impact of Innovation on Hemophilia Care Management Organization: A Qualitative Study Protocol (INNOVHEMO Study).Patient preference and adherence · 2021Article
- Progress in the Development of Anti-tissue Factor Pathway Inhibitors for Haemophilia Management.Frontiers in medicine · 2021Review
- Real-World Unmet Treatment Needs for Patients With Haemophilia: Results From the Global Adelphi Disease Specific Programme Database.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
- Erratum issuedCorrigendum.2021
Authors and funding
9 authors at 6 institutions in 4 countries.
Funding
Abstract
introductionEmicizumab 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.
aimDescribe 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.
methodsTo 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.
resultsSixty-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).
conclusionThese 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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