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Background: Post‐transplant health‐related quality of life (HRQOL) is associated with health outcomes for kidney transplant (KT) recipients. However, pretransplant predictors of improvements in post‐transplant HRQOL remain incompletely understood. Namely, important pretransplant cultural factors, such as experience of discrimination, perceived racism in healthcare, or mistrust of the healthcare system, have not been examined as potential HRQOL predictors. Also, few have examined predictors of decline in HRQOL post‐transplant. Methods: Using data from a prospective cohort study, we examined HRQOL change pre‐ to post‐transplant, and novel cultural predictors of the change. We measured physical, mental, and kidney‐specific HRQOL as outcomes, and used cultural factors as predictors, controlling for demographic, clinical, psychosocial, and transplant knowledge covariates. Results: Among 166 KT recipients (57% male; mean age 50.6 years; 61.4% > high school graduates; 80% non‐Hispanic White), we found mental and physical, but not kidney‐specific, HRQOL significantly improved post‐transplant. No culturally related factors outside of medical mistrust significantly predicted change in any HRQOL outcome. Instead, demographic, knowledge, and clinical factors significantly predicted decline in each HRQOL domain: physical HRQOL—older age, more post‐KT complications, higher pre‐KT physical HRQOL; mental HRQOL—having less information pre‐KT, greater pre‐KT mental HRQOL; and, kidney‐specific HRQOL—poorer kidney functioning post‐KT, lower expectations for physical condition to improve, and higher pre‐KT kidney‐specific HRQOL. Conclusions: Instead of cultural factors, predictors of HRQOL decline included demographic, knowledge, and clinical factors. These findings are useful for identifying patient groups that may be at greater risk of poorer post‐transplant outcomes, in order to target individualized support to patients. [ABSTRACT FROM AUTHOR]

John Devin Peipert,1,* Sherif M Badawy,2,* Sharon H Baik,1 Laura B Oswald,1 Fabio Efficace,3 Sofia F Garcia,1 Daniel K Mroczek,1 Michael Wolf,4 Karen Kaiser ,1 Betina Yanez,1 David Cella1 1Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; 2Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA; 3Italian Group for Adult Hematologic Disease (GIMEMA), Health Outcomes Research Unit, Rome, Italy; 4Department of Medicine, Division of General Internal Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA*These authors contributed equally to this workCorrespondence: John Devin Peipert Email john.peipert@northwestern.eduAbstract: Poor medication adherence is associated with reduced drug effectiveness, poor health-related quality of life, increased morbidity and mortality, and increased healthcare utilization and cost. Including the patient’s voice is essential in understanding barriers to adherence. Useful patient-reported adherence measures are brief, inexpensive, non-invasive; can indicate barriers to adherence; and can be incorporated in electronic health records. The NIH Patient-Reported Outcomes Measurement Information System (PROMIS®) includes high-quality, freely available patient-reported measures covering many important constructs in patient-centered research but does not include a medication adherence measure. To fill this gap, we developed the PROMIS Medication Adherence Scale (PMAS) using the rigorous PROMIS instrument development guidelines. To develop the PMAS, we first conducted a review of the reviews, which enabled us to identify content areas relevant to medication adherence behavior. Then, we conducted qualitative research to elicit patients’ views of and experiences with medication adherence. This process identified the following important content areas to guide item writing: extent medication is taken, knowledge of medication regimen, beliefs about medication, remembering to take medication, skipping due to side effects, skipping due to feeling better, and cost of medications. Based on the results of these activities, we wrote items and aimed to retain 1– 2 items per content area. The final item set included 9 total adherence items, which were then refined through intensive comprehension and translatability review, as well as cognitive interviews. Future steps include testing the PMAS’s validity.Keywords: medication adherence, patient-reported outcome, PROMIS

Karen Kaiser,1 Susan E Yount,1 Christa E Martens,1 Kimberly A Webster,1 Sara Shaunfield,1 Amy Sparling,1 John Devin Peipert,1 David Cella,1 Scott T Rottinghaus,2 Bonnie MK Donato,2 Richard Wells,3 Ioannis Tomazos2 1Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; 2Alexion Pharmaceuticals Inc, Boston, MA, USA; 3Sunnybrook Health Sciences Centre, Toronto, ON, CanadaCorrespondence: Karen KaiserDepartment of Medical Social Sciences, Northwestern University Feinberg School of Medicine, 625 N. Michigan Ave. Suite 2700, Chicago, IL 60611, USATel +1 312-503-3521Fax +1 312-503-4800Email k-kaiser@northwestern.eduPurpose: To develop a patient preference questionnaire (PPQ) assessing eculizumab and ravulizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH).Patients and Methods: The development of the PNH-PPQ© was consistent with Food and Drug Administration guidelines for patient-reported outcome measure development, and included 1) a targeted literature review; 2) PNH expert clinician input on treatment preferences; 3) review of existing qualitative data on the PNH treatment and disease experience; 4) concept elicitation interviews with 8 PNH patients who received eculizumab and/or ravulizumab; 5) translatability review; and 6) cognitive debriefing with 5 patients. Interview participants were recruited through a United Kingdom PNH patient advocacy group and a Canadian clinical site involved in clinical trial ALXN1210-PNH-302.Results: Six themes were identified as most relevant to the PNH treatment experience from the concept elicitation interviews: disease symptoms (n=8/8); treatment frequency (n=7/8); quality of life impact of treatment/disease (n=7/8); treatment burden (n=7/8); treatment efficacy (n=5/8); and treatment side effects (n=5/8). An initial list of 88 preference questions was reduced to 11 highly relevant and non-redundant questions reflecting the 6 themes. Cognitive interview participants unanimously agreed that the PNH-PPQ instructions were clear; response options were understandable, easy to use, and provided enough choices; and the questions captured the factors that inform treatment preferences.Discussion: When new drugs have similar efficacy to existing medications, documenting patient preferences is important for confirming patient benefit from the new medication. Understanding what matters most to patients is essential for delivering patient-centered care and may play a particularly significant role in treatment decision making. The availability of such a tool may be especially important as new orphan drugs are developed and patients with rare diseases have more than one treatment option to consider.Conclusion: The PNH-PPQ provides a patient-centered approach for evaluating preferences for the treatment of PNH. The PNH-PPQ has subsequently assessed patient preference in the clinical trial sub-study ALXN1210-PNH-302s.Keywords: paroxysmal nocturnal hemoglobinuria, questionnaire development, ravulizumab, eculizumab, treatment experience