Background: Nearly all patients with end-stage renal disease (ESRD) who are undergoing dialysis are anemic. Severe anemia can cause disabling fatigue, decreased quality of life, and increased mortality. To mitigate anemia, dialysis patients often receive Erythropoiesis-Stimulating Agents (ESAs) therapy to reduce the need for blood transfusions and increase hemoglobin (Hb) level. Due to the lack of evidence surrounding the safe and effective use of ESA therapy, currently there’re several important unanswered questions—whether and when to start ESA therapy, what Hb level to target treatment to, and who can receive ESAs safely. Therefore, it is vital that we identify which aspects of ESA treatment strategies are most important to patients’ goals, adopt these outcome measures/metrics in future study designs, and ultimately translate findings into a better quality of life. However, the current patient-centered outcomes research base in this regard is limited because patient perspectives on the value and relevance of optimal anemia management strategies have been absent.
Objectives: The specific purpose of this conference project is incorporating the patient voice and perspective into all aspects of the consensus building regarding optimal anemia management strategies. The first year will focus on creating a Patient-Centered Research Partnerships (PCRP) between patients, care practitioners, and other stakeholders to build community and connections. These partnerships will serve as an infrastructure for identifying potential candidate metrics for optimal anemia management strategies. Year 2 will focus on challenging this community to build action plan that’s necessary to ensure use of optimal treatment metrics identified in Year 1 and to share knowledge of effective dissemination activities with all renal community stakeholders to enhance implementation. Participants of these conferences will include a broad spectrum of dialysis patient and stakeholder partners including patients’ families, caregivers, physicians, nurses, researchers, pharmaceutical companies, FDA and CMS regulators, renal data experts, NIDDK, and AHRQ.
Activities: PCRP will develop conference theme and topics, agendas, and matrices for use in two conferences. Some of the main activities are: 1) Two full day conferences and workshops will be held at the National Institute of Heath Natcher Conference Center, featuring formal presentations by patients, patient advocates, physicians, payers, industry, and regulators. To facilitate participation/discussions, a lay data dictionary will be developed to assist non-medical personnel in all aspects of planning, participation, and dissemination of findings/recommendations. The conference will also involve plenary panels, and interactive breakout sessions within which new relationships can be formed; 2) Two planning meetings will take place prior to each conference, and three additional teleconference meetings to finalize each year’s symposium; 3) workshops with curriculum materials for researchers and communities, build and launch the conference website, track its use, promoting the conference, videotape the conference as a resource for sharing the conference findings. Speakers will share their knowledge about what has already been accomplished in this area and will identify successful case examples and/or ESA treatment protocols in improving anemia management. Knowledge gaps will be identified and methodologies to address unmet needs will be discussed. The summit is designed with panels dedicated to critical areas of interest. Panels will be moderated by topic experts and followed by discussions to engage all stakeholders in thought-provoking dialogue. These activities will foster bidirectional research partnerships between patients and other stakeholders.
Projected Outcomes and Outputs: Three primary outcomes of this project are: 1) the establishment active engagement of PCRP in which for patients are placed at the center of all aspects of research to identify anemia management strategies, from the prioritization and generation of conference topics to the development of appropriate conference format, design of facilitated discussions, procedures, outreach, promotion, and eventual dissemination of results to achieve ultimate impact; 2) identification of prioritized ESA treatment strategies regarding initiation, dose, titration, patient responsiveness, and Hb target; and 3) the conference will provide lessons learned and develop an action plan for addressing gaps in anemia management and recommend future studies. Deliverables will focus on informing future research, policy, and clinical practice. The conference proceedings will be summarized and distributed to all attendees and through communication channels of all steering committee member organizations (see below).
Patient and Stakeholder Engagement Plan: The project team’s patient and stakeholder engagement plan is to utilize a Planning and Steering Committee (PSC) with the specific purpose of ensuring patient stakeholders play an active role in all phases of conference design and planning. The PSC includes a broad stakeholder representation, each contributing to the shaping of the program and the dissemination of the summit proceedings. Specifically, the planning committee has included two patient and patient advocacy representatives, American Association of Kidney Patients (patient advocacy/education), and Dialysis Patient Citizens (patient advocacy); three dialysis practitioner groups including two physician groups (American Society of Nephrology and Renal Physicians Association), one nurse group (American Nephrology Nurses Association), and one educational practitioner (National Kidney Foundation), and three renal research groups (National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) /Kidney Research National Dialogue (KRND), NIDDK/ Kidney Interagency Coordinating Committee (KICC), VHA National Kidney Program) who will participate in the event steering committee. All event topics and conference content will be reviewed by this PSC to ensure they meet the needs of a wide spectrum of patients.