Primary Hyperoxaluria: The Patient and Caregiver Perspective

Primary Hyperoxaluria: The Patient and Caregiver Perspective

Living with primary hyperoxaluria—a rare genetic disease with excess oxalate production leading to frequent kidney stones, kidney impairment, and oxalosis—presents many challenges to patients, caregivers, and their families. Although the progression and severity of primary hyperoxaluria is variable, care for any child or adult with primary hyperoxaluria is an unusual strain to the family due to intensity of required medical care and associated financial hardship. To identify the concerns of the primary hyperoxaluria community, multiple in-person meetings were convened and a web-based survey was developed by families, the Oxalosis and Hyperoxaluria Foundation (OHF), the Kidney Health Initiative (KHI), and the American Institutes for Research. The patient and caregiver perspectives listed in this paper as well as the survey findings (Figure 1) were derived from the respondents from the survey and meetings and highlight the daily challenges and the great need for new therapies. The companion paper, “Endpoints for Clinical Trials in Primary Hyperoxaluria” by Milliner et al. presents a systematic analysis of available data to identify appropriate end points to assess the efficacy of potential therapies in clinical trials.


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