Hawkins Lauren M, Sinha Cynthia B, Ross Diana, Yee Marianne E M, Quarmyne Maa-Ohui, Krishnamurti Lakshmanan, Bakshi Nitya
Emory University School of Medicine, Atlanta, GA, USA.
Division of Pediatric Hematology-Oncology-BMT, Emory University School of Medicine, Atlanta, GA, USA.
BMC Pediatr. 2020 Apr 18;20(1):172. doi: 10.1186/s12887-020-02078-w.
There is a limited understanding of the patient and family experience of Chronic Transfusion Therapy (CTT) for prevention of complications of Sickle Cell Disease (SCD). We sought to understand patient and family experience with CTT using qualitative methods.
Fifteen parents of children < 18 years old and nine children 12-18 years old with SCD who were receiving CTT for > 1 year were interviewed using a semi-structured interview format, and interviews were analyzed using open coding methods.
Four themes created a narrative of the patient and family experience of CTT: 1) Burden of CTT, 2) Coping with CTT, 3) Perceived benefits and risks of CTT, and 4) Decision making regarding CTT. Participants reported substantial burden of CTT, including the impact of CTT on daily life and family, distress about venous access, burden of chelation therapy, and anxiety about CTT complications. Participants described how they coped with CTT. Participants reported increased energy, decreased pain, fewer hospitalizations, and stroke prevention with CTT, but also recognized complications of CTT, though awareness was limited in adolescents. Parents described sharing in the informed decision-making process with their healthcare provider about CTT, but adolescent patient participants reported that they were not involved in this process.
CTT is associated with significant patient and family burden. Support from family, healthcare providers and school may help individuals cope with some of this burden. These findings provide the basis for future studies to identify strategies to mitigate the burden of CTT and improve the patient experience with this therapy. Future studies should also systematically assess patient knowledge about the key components of CTT and chelation using quantitative assessments.
对于慢性输血疗法（CTT）预防镰状细胞病（SCD）并发症的患者及家庭体验，人们了解有限。我们试图采用定性方法了解患者及家庭对CTT的体验。
对15名18岁以下镰状细胞病患儿的父母以及9名12至18岁正在接受CTT治疗超过1年的镰状细胞病患儿进行了半结构式访谈，并使用开放编码方法对访谈进行分析。
四个主题构成了患者及家庭对CTT体验的叙述：1）CTT的负担，2）应对CTT，3）CTT的感知益处和风险，4）关于CTT的决策。参与者报告了CTT带来的巨大负担，包括CTT对日常生活和家庭的影响、静脉通路的困扰、螯合疗法的负担以及对CTT并发症的焦虑。参与者描述了他们应对CTT的方式。参与者报告CTT使精力增加、疼痛减轻、住院次数减少以及预防了中风，但也认识到CTT的并发症，不过青少年对此的认知有限。父母描述了他们与医疗服务提供者共同参与关于CTT的知情决策过程，但青少年患者参与者报告称他们未参与该过程。
CTT给患者及家庭带来了巨大负担。来自家庭、医疗服务提供者和学校的支持可能有助于个人应对部分此类负担。这些发现为未来研究确定减轻CTT负担和改善患者治疗体验的策略提供了基础。未来研究还应使用定量评估系统地评估患者对CTT和螯合疗法关键组成部分的了解情况。
