PRO23 An Observational Cohort Analysis on the Economic IMPACT of Chronic Kidney Disease in Patients with Fabry Disease

Wallace, Eric; Sacks, Naomi; Cyr, Philip L.; Baker-Wagner, M.

doi:10.1016/j.jval.2021.04.1008

Cited by 1 publication

(5 citation statements)

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“…Wallace and colleagues did not specifically consider the contribution of ERT to healthcare costs, but looked at overall FD-related expenditure according to presence or absence of CKD [ 66 ]. The study reported a 3.5-times higher mean annual cost for patients with CKD compared with patients without CKD ( p < 0.01).…”

Section: Resultsmentioning

confidence: 99%

“…Seven of the identified studies reported data relating to resource use by patients with FD (Table 5 ) [ 40 , 61 , 63 , 64 , 66 , 69 , 70 ].…”

Section: Resultsmentioning

confidence: 99%

“…A 2021 study by Wallace and colleagues demonstrated significantly higher healthcare resource utilization (including emergency department visits, physician office visits, outpatient hospital visits, and prescription medications) in patients with FD with versus without CKD [ 66 ].…”

Section: Resultsmentioning

confidence: 99%

“…In terms of the economic burden, FD was associated with a high cost and healthcare resource use burden [ 40 , 61 , 64 , 66 , 67 ]. All studies that included patients who were receiving ERT reported that it made a substantial contribution to the cost of FD management [ 40 , 61 , 63 – 65 ].…”

Section: Discussionmentioning

confidence: 99%

“…It should be noted, however, that migalastat is only indicated in patients with an amenable GLA variant [ 13 ]. High healthcare resource utilization was apparent across all studies [ 40 , 61 , 63 , 64 , 66 , 69 , 70 ], with higher resource use among patients presenting with renal complications than in those with uncomplicated disease [ 66 ].…”

Section: Discussionmentioning

confidence: 99%

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A systematic literature review on the health-related quality of life and economic burden of Fabry disease

Jovanovic,

Miller-Hodges,

Castriota

et al. 2024

Orphanet J Rare Dis

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Background Fabry disease (FD) is a rare lysosomal storage disease associated with glycolipid accumulation that impacts multiple physiological systems. We conducted a systematic literature review (SLR) to characterize the humanistic (quality of life [QoL]) and economic burden of FD. Methods Searches were conducted in the Embase, MEDLINE®, and MEDLINE® In-Process databases from inception to January 19, 2022. Conference abstracts of specified congresses were manually searched. Additional searches were performed in the Cochrane and ProQuest databases for the humanistic SLR and the National Health Service Economic Evaluations Database for the economic SLR. Studies of patients with FD of any sex, race, and age, and published in the English language were included. There was no restriction on intervention or comparator. For the humanistic SLR, studies that reported utility data, database/registry-based studies, questionnaires/surveys, and cohort studies were included. For the economic SLR, studies reporting economic evaluations or assessing the cost of illness and resource use were included. Results Of the 1363 records identified in the humanistic search, 36 studies were included. The most commonly used QoL assessments were the 36-item Short-Form Health Survey (n = 16), EQ-5D questionnaire descriptive system or visual analog scale (n = 9), and the Brief Pain Inventory (n = 8). Reduced QoL was reported in patients with FD compared with healthy populations across multiple domains, including pain, physical functioning, and depressive symptoms. Multiple variables—including sex, age, disease severity, and treatment status—impacted QoL. Of the 711 records identified in the economic burden search, 18 studies were included. FD was associated with high cost and healthcare resource use. Contributors to the cost burden included enzyme replacement therapy, healthcare, and social care. In the seven studies that reported health utility values, lower utility scores were generally associated with more complications (including cardiac, renal, and cerebrovascular morbidities) and with classical disease in males. Conclusion FD remains associated with a high cost and healthcare resource use burden, and reduced QoL compared with healthy populations. Integrating information from QoL and economic assessments may help to identify interventions that are likely to be of most value to patients with FD.

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Section: Resultsmentioning

confidence: 99%

“…Seven of the identified studies reported data relating to resource use by patients with FD (Table 5 ) [ 40 , 61 , 63 , 64 , 66 , 69 , 70 ].…”

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%