Healthcare utilization, quality of life, and work productivity associated with primary hyperoxaluria: a cross-sectional web-based US survey

Abstract

Primary hyperoxaluria (PH) is a family of ultra-rare, autosomal recessive, metabolic disorders associated with frequent kidney stones, chronic kidney disease and kidney failure, and serious complications due to systemic oxalosis, resulting in significant morbidity. We investigated the burden of PH among affected patients and caregivers. This cross-sectional, web-based survey was used to quantify the burden of PH, in terms of healthcare resource utilization, health-related quality of life, and work productivity and activity impairment among adults (≥ 18 years) with PH and caregivers of children (≤ 17 years) with PH in the US. Among the 20 respondents, there were 7 adults with PH and 13 caregivers of children with PH. Adherence to hyperhydration was noted as the most, or one of the most, difficult aspects of PH by 56% of respondents. Most patients (95%) had experienced painful kidney stone events, one-third had visited the emergency room, and 29% were hospitalized for complications due to PH. Of the 24% of patients on dialysis, all found the procedure burdensome. Adult patients’ quality of life was negatively affected across several domains. Most respondents (81%) reported that PH had a negative effect on their finances. Employed adult patients and caregivers, and children with PH, had moderate impairment in work productivity, school attendance, and activity. Anxiety about future PH-related sequelae was moderate to high. These findings highlight the need for improvements in PH medical management. A plain language summary is available in the supplementary information.

Nedosiran in primary hyperoxaluria subtype 3: results from a phase I, single-dose study (PHYOX4)

“…It is not unusual for patients with PH3 to have symptomatic stone disease throughout their life [ 4 , 5 ]. Multiple occurrences of painful kidney stones requiring urologic intervention represent one of the most troubling aspect of PH for both patients and their caregivers [ 8 – 10 ]. Currently, PH3 is managed based solely on supportive options [ 11 ].…”

Nedosiran Safety and Efficacy in PH1: Interim Analysis of PHYOX3

“…This comparison was calculated using clinical data during the PHYOX3 and PHYOX2 on-treatment periods, and as such, is a more informative comparison than using the PHYOX3 baseline event rate, which is estimated from patient recall. In 2 other studies of patients with PH1 who were RNAi treatment-naive, 85% of participants 31 or 95% of participants 32 reported a history of kidney stone events at baseline; the annualized event rate was 3.19 per person-year over the 12 months prior to RNAi treatment. 31 Like preservation of kidney function, the reduction of kidney stone events reflects the clinical importance of nedosiran for patients with PH1 in reducing the burden of disease and helping to improve their quality of life.…”

“…Hyperhydration regimens are intended to reduce kidney stone events but are extremely difficult to adhere to by patients with PH1 32 ; both time consuming and difficult to maintain, especially for children. Patients with PH report that hyperhydration is one of the most burdensome aspects of PH treatment and declines in quality-of-life scores were attributed in part to the difficulty of maintaining hyperhydration.…”

“…Patients with PH report that hyperhydration is one of the most burdensome aspects of PH treatment and declines in quality-of-life scores were attributed in part to the difficulty of maintaining hyperhydration. 32 Similarly, citrate medications and other treatment options represent a quality-of-life burden for patients with PH. 30 In the current study, 11 of the 13 participants were eligible for reduction of hyperhydration regimens due to satisfactory Uox control at normal levels.…”