Lymphangioleiomyomatosis Patient Research Priorities Survey (LAM-PREP): Developing a Patient-Centered Research Agenda.

BACKGROUND

Engaging patients in developing research agendas is recognized as critical in advancing scientific discovery and improving clinical care. There is a scarcity of such studies for respiratory diseases, particularly rare conditions like lymphangioleiomyomatosis (LAM).

RESEARCH QUESTION: Can a comprehensive study inform and guide future LAM research for the benefit of patients, researchers, and clinical providers?

STUDY DESIGN AND

METHODS

We used a mixed-methods analysis consisting of focus groups of patients and caregivers. Participant responses were thematically analyzed and grouped into categories and subcategories using qualitative content analysis to generate a list of health needs, quality of life issues, and research areas. A survey to rank the priorities was then developed and administered electronically to patients with LAM, caregivers, scientists, and clinicians.

RESULTS

The LAM Patient Research Priorities Survey (LAM-PREP) revealed a list of priorities centered on health, quality of life, and research needs. Areas of agreement among all respondents were prioritizing awareness of LAM among health care providers, access to comprehensive care, understanding and interpreting symptoms, mental health, managing supplemental oxygen, finding new treatments and a cure, and studying the role of hormones in LAM disease and treatment. Clinicians prioritized needs relating to travel, sexual and women's health, and childbearing and family planning. Patients prioritized questions regarding LAM and sleep quality, safe exercise, and the impact of diet. Both scientists and clinicians noted a priority in studying disease onset and progression.

INTERPRETATION: LAM-PREP integrated diverse perspectives on LAM research and clinical priorities from LAM community stakeholders. These findings should serve as a roadmap for developing and implementing projects and interventions to improve the health outcomes of women living with LAM. Importantly, LAM-PREP can serve as a model for developing patient-focused research agendas for other rare diseases, particularly those affecting women.