ICON Plc, Lyon, France.
Advicenne S.A., 21 Allée Boissy d'Anglas, 30000, Nîmes, France.
Orphanet J Rare Dis. 2022 Mar 28;17(1):141. doi: 10.1186/s13023-022-02294-w.
Consequences of distal renal tubular acidosis (dRTA) on growth, bone and kidney, sometimes associated with hearing loss, may significantly affect quality of life (QoL). This descriptive qualitative study explores QoL linked to dRTA and gathers the impressions of patients with this rare disease (and caregivers) 5 years after enrolment in a clinical study, during which patients were treated with ADV7103, a prolonged-release granule formulation combining potassium citrate and potassium bicarbonate. Semi-structured, one-hour interviews with 6 adult and 13 paediatric patients with a confirmed diagnosis of dRTA and with parents of paediatric patients were performed using an interview guide. Qualitative analysis of anonymized interview transcripts based on grounded theory was conducted.
The main QoL domains impacted by dRTA and its treatment were education/work, social/family life, and emotional and physical well-being. ADV7103 (administered twice daily) was compared with the standard of care (SoC) taken before study entry (more than twice daily). Patients/parents reported that switching from previous SoC to ADV7103 had changed their lives: Difficulties at school due to burdensome administrative issues and need to explain disease and treatment affecting all families of paediatric patients (n = 13) disappeared, facilitating parents who had stopped working (to deal with their child's treatment) to return to work, Family functioning was improved (n = 18), as travel and holidays became easier to organise and patients/parents stopped thinking about managing treatment daily/nightly, reducing tension in the family or couple, The emotional burden of disease perceived was relieved (n = 12) in the absence of treatment-related invasive questions from others, Gastro-intestinal adverse events and taste problems improved with ADV7103 (n = 18) and better compliance led to milder physical impacts and less need to be hospitalised. The mean satisfaction score with ADV7103 compared to SoC was 9 out of 10 (10 = very satisfied). ADV7103 exceeded or met the expectations of 14 out of 17 patients that commented on that.
Qualitative interviews show that dRTA and its treatment have a significant impact on QoL of patients and parents and that ADV7103 helps improve daily-life and reduces treatment burden, resulting in greater overall satisfaction of the patients and their families. Trial registration EU Clinical Trials Register, EudraCT 2013-003828-36 on the 3rd of September 2013.
远端肾小管性酸中毒（dRTA）可导致生长、骨骼和肾脏受损，有时还伴有听力损失，这可能显著影响生活质量（QoL）。本描述性定性研究探讨了与 dRTA 相关的生活质量，并收集了在临床研究中接受 ADV7103（一种结合枸橼酸钾和碳酸氢钾的缓释颗粒制剂）治疗的 5 年后，患有这种罕见疾病的患者（及其照护者）的印象。使用访谈指南对 6 名成年患者和 13 名儿科患者及其儿科患者的父母进行了长达 1 小时的半结构化访谈。对基于扎根理论的匿名访谈记录进行了定性分析。
受 dRTA 及其治疗影响的主要生活质量领域包括教育/工作、社会/家庭生活以及情绪和身体健康。ADV7103（每日两次给药）与研究入组前的标准治疗（每日两次以上）进行了比较。患者/父母报告说，从之前的标准治疗（每日两次以上）转换为 ADV7103 改变了他们的生活：由于繁琐的行政问题和需要解释疾病和治疗而导致的学业困难（儿科患者 13 例）以及所有家庭都消失了，使已停止工作（处理孩子的治疗）的父母能够重返工作岗位，家庭功能得到改善（18 例），旅行和度假变得更容易安排，患者/父母不再每天/每晚考虑治疗，减轻了家庭或夫妻之间的紧张感，疾病带来的情绪负担（12 例）减轻了，因为不再需要与治疗相关的侵入性问题来询问他人，ADV7103 改善了胃肠道不良事件和味觉问题（18 例），更好的依从性导致身体影响更温和，住院需求减少。与标准治疗相比，患者对 ADV7103 的满意度平均评分为 10 分中的 9 分（10 分表示非常满意）。对此发表评论的 17 名患者中有 14 名表示，ADV7103 超过或满足了他们的期望。
定性访谈表明，dRTA 及其治疗对患者和父母的生活质量有重大影响，ADV7103 有助于改善日常生活并减轻治疗负担，从而使患者及其家属总体上更加满意。试验注册欧盟临床试验注册处，EudraCT 2013-003828-36 于 2013 年 9 月 3 日。
