PRO Gap Analysis in Antibody Mediated Rejection, Delayed Graft Function, Lupus Nephritis, and Membranous Nephropathy

OBJECTIVES: Antibody Mediated Rejection (AMR), Delayed Graft Function (DGF), Lupus Nephritis (LN), and Membranous Nephropathy (MN) have a known impact on patients’ quality of life that should be considered when measuring treatment benefit from patients’ perspectives.

Our aim was to develop Patient-Reported Outcome (PRO) strategies for AMR, DGF, LN, and MN clinical trial programs.

METHODS: A targeted literature review was performed in Medline and PsycInfo databases to ascertain qualitative concepts of importance to AMR, DGF, LN and MN patients. Additional searches were performed in Medline, Clinicaltrials.gov, and ePROVIDE^TM databases to identify PROs used in these indications.

A gap analysis compared the qualitative concepts to the item content of a selection of the identified PROs, and appraised the selected instruments’ development and validation data.

RESULTS: Two conceptual models were developed in LN and kidney graft failure after a review of 7 qualitative studies. A list of concepts was developed in MN after one clinician interview. Generic and disease-specific PROs were identified from 41 published and 21 registered clinical studies, and the ePROVIDE^TM databases.

The disease-specific LupusPRO was selected to assess the concepts of importance to LN patients. No disease-specific PROs were found in AMR, DGF, or MN. Therefore, PROMIS instruments and the Kidney Disease Quality of Life Short Form (KD-QOL-SF) were considered the most applicable for use in AMR and DGF clinical trials, while the Incontinence Quality of Life (I-QoL) scale was considered most relevant for MN.

CONCLUSIONS: Although generic PROs are available, the poor symptomatology of AMR, DGF, and MN may have prevented the development of disease-specific instruments for these indications. Disease-specific PROs should be developed for these patients to allow for granularity in measuring impact of disease and treatment from a patient-centered perspective in clinical trials. Impacts related to anxiety/depression and social functioning could be areas for further investigation.