背景:健康研究机构建议患者参与研究和医疗保健计划,尽管它们的实施还不广泛。这种缺陷延伸到进行性肺纤维化(PPF),关键方面仍然未知,包括因果机制,治愈性治疗和最佳症状管理。本研究通过寻求利益相关者的观点来指导研究和治疗方向,从而解决了这些差距。
方法:建立了优先级设定伙伴关系,以探索利益相关者的诊断优先级,治疗,PPF的管理和护理,包括特发性肺纤维化,这是典型的PPF。利益相关者包括生活在PPF中的人,他们的照顾者,亲戚和医疗保健专业人员参与他们的管理。
结果:通过在线开放式调查,从638个利益相关方收集了2542份回复。专题分析确定了48个具体的研究问题,然后与学术文献交叉引用,以查明研究差距。在证据检查之后,在第二次在线调查中,834个利益相关者入围了44个未回答的问题。最终,通过协商一致确定了十大优先事项清单。优先考虑的研究问题包括(1)提高诊断准确性和时机,(2)开发新的治疗方法,(3)提高初级保健的准确性,(4)药物和非药物干预的最佳时机,(5)有效治疗咳嗽,(6)PPF的早期干预,(7)提高生存率,(8)症状减轻,(9)干预措施对预期寿命的影响和(10)减少副作用的新疗法。
结论:利益相关者的优先事项可以概括为五个方面:早期诊断,药物和非药物治疗,生存和症状管理。理想情况下,这些主题应指导资助机构和卫生政策。
BACKGROUND: Health research bodies recommend patient involvement and engagement in research and healthcare planning, although their implementation is not yet widespread. This deficiency extends to progressive pulmonary fibrosis (PPF), where crucial aspects remain unknown, including causal mechanisms, curative treatments and optimal symptom management. This study addresses these gaps by seeking stakeholders\' perspectives to guide research and treatment directions.
METHODS: A priority-setting partnership was established to explore stakeholders\' priorities in the diagnosis, treatment, management and care of PPF, including idiopathic pulmonary fibrosis which is the archetypal PPF. Stakeholders included people living with PPF, their carers, relatives and healthcare professionals involved in their management.
RESULTS: Through an online open-ended survey, 2542 responses were collected from 638 stakeholders. Thematic analysis identified 48 specific research questions, which were then cross-referenced with academic literature to pinpoint research gaps. Following the evidence check, 44 unanswered questions were shortlisted by 834 stakeholders in a second online survey. Ultimately, a top 10 priority list was established through consensus.The prioritised research questions include (1) improved diagnosis accuracy and timing, (2) development of new treatments, (3) enhanced accuracy in primary care, (4) optimal timing for drug and non-drug interventions, (5) effective cough treatment, (6) early intervention for PPF, (7) improved survival rates, (8) symptom reduction, (9) impact of interventions on life expectancy and (10) new treatments with reduced side effects.
CONCLUSIONS: Stakeholders\' priorities can be summarised into five areas: early diagnosis, drug and non-drug treatments, survival and symptom management. Ideally, these topics should guide funding bodies and health policies.