Flood Emuella, Pocoski Jennifer, Michaels Lisa A, McCoy Anne, Beusterien Kathleen, Sasanè Rahul
Oxford Outcomes Inc., An ICON plc. Company, Bethesda, Maryland, USA.
Eur J Haematol. 2014 Jun;93 Suppl 75:19-28. doi: 10.1111/ejh.12329.
Patients with haemophilia experience bleeds because of absent or reduced clotting factor. The study objective was to understand the bleeding experience from the patients' perspective.
Individuals with moderate/severe haemophilia participated in interviews and were asked to describe their most recent bleeding experience, including symptoms, signs of onset, impacts, when bleeding stopped and treatment effectiveness. Interview transcripts were analysed using a thematic analysis involving the coding of transcripts to identify key concepts and themes.
Twenty males [10 adults, mean age = 41 (19-52); 10 adolescents, mean age = 13 (12-17)] with moderate (n = 5) or severe (n = 15) haemophilia participated. Symptoms signalling bleed onset included pain, swelling, stiffness, tingling/numbness and/or warmth. Participants reported feeling anger and frustration due to the unpredictable nature, pain and inconvenience of the episode. Adults sometimes reported delaying treatment due to inconvenience or cost; adolescents generally treated right away. Reported bleed severity was influenced by pain level, speed of symptom progression, location, continued use of the affected area, recurrence in same location of recent bleed and treatment delay. Participants reported that it was 'easy' to know when the bleed had stopped. Participants reported that symptoms might linger for days before they returned back to 'normal'.
This qualitative study details the substantial impact of an acute bleed from the patient perspective. Given that treatment was reported to be delayed in part due to inconvenience, more convenient treatment options could help reduce delays in treating bleeds and thereby minimise bleed-related impacts. Clinical studies in haemophilia should include validated patient-reported measurements of acute symptoms and bleed severity to comprehensively assess the bleeding event.
血友病患者因凝血因子缺乏或减少而出血。本研究的目的是从患者的角度了解出血经历。
中重度血友病患者参与访谈，被要求描述其最近的出血经历，包括症状、出血开始的迹象、影响、出血停止的时间以及治疗效果。访谈记录采用主题分析法进行分析，包括对记录进行编码以识别关键概念和主题。
20名男性[10名成年人，平均年龄=41岁（19 - 52岁）；10名青少年，平均年龄=13岁（12 - 17岁）]患有中度（n = 5）或重度（n = 15）血友病参与了研究。提示出血开始的症状包括疼痛、肿胀、僵硬、刺痛/麻木和/或发热。参与者报告称，由于出血事件的不可预测性、疼痛和不便，他们感到愤怒和沮丧。成年人有时报告因不便或费用而延迟治疗；青少年一般立即进行治疗。报告的出血严重程度受疼痛程度、症状进展速度、部位、受影响部位的持续使用情况、近期出血同一部位的复发以及治疗延迟的影响。参与者报告说很“容易”知道出血何时停止。参与者报告说症状可能会持续数天，然后才恢复“正常”。
这项定性研究从患者角度详细描述了急性出血的重大影响。鉴于据报告治疗部分因不便而延迟，更方便的治疗选择可能有助于减少出血治疗的延迟，从而将与出血相关的影响降至最低。血友病的临床研究应包括经过验证的患者报告的急性症状和出血严重程度测量，以全面评估出血事件。
