Healthcare utilization and costs in patients with tuberous sclerosiscomplex-related renal angiomyolipoma

Song, Xue; Liu, Zhimei; Cappell, Katherine; Gregory, Christopher; Said, Qayyim; Prestifilippo, Judith; Charles, H.; Hulbert, John C.; Bissler, John J.

doi:10.1080/13696998.2016.1272461

Cited by 7 publications

(13 citation statements)

References 14 publications

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“…We investigated the relationships between an individual’s clinical characteristics and TSC-related costs using multivariate linear regression using the BCa method with 2000 repetitions. Total direct, total indirect, and nursing care-level costs were regressed against a set of clinical variables, which we selected following univariate analysis and according to evidence presented by previous cost-of-illness studies examining TSC [ 16 , 37 , 38 ]. We tested all variables for interactions and collinearity.…”

Section: Methodsmentioning

confidence: 99%

Direct and indirect costs and cost-driving factors in adults with tuberous sclerosis complex: a multicenter cohort study and a review of the literature

Zöllner

Grau

Rosenow

et al. 2021

Orphanet J Rare Dis

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Background Tuberous sclerosis complex (TSC) is a monogenetic, multisystem disorder characterized by benign growths due to TSC1 or TSC2 mutations. This German multicenter study estimated the costs and related cost drivers associated with organ manifestations in adults with TSC. Methods A validated, three-month, retrospective questionnaire assessed the sociodemographic and clinical characteristics, organ manifestations, direct, indirect, out-of-pocket (OOP), and nursing care-level costs among adult individuals with TSC throughout Germany from a societal perspective (costing year: 2019). Results We enrolled 192 adults with TSC (mean age: 33.4 ± 12.7 years; range: 18–78 years, 51.6% [n = 99] women). Reported TSC disease manifestations included skin (94.8%) and kidney and urinary tract (74%) disorders, epilepsy (72.9%), structural brain defects (67.2%), psychiatric disorders (50.5%), heart and circulatory system disorders (50.5%), and lymphangioleiomyomatosis (11.5%). TSC1 and TSC2 mutations were reported in 16.7% and 25% of respondents, respectively. Mean direct health care costs totaled EUR 6452 (median EUR 1920; 95% confidence interval [CI] EUR 5533–7422) per patient over three months. Medication costs represented the major direct cost category (77% of total direct costs; mean EUR 4953), and mechanistic target of rapamycin (mTOR) inhibitors represented the largest share (68%, EUR 4358). Mean antiseizure drug (ASD) costs were only EUR 415 (6%). Inpatient costs (8%, EUR 518) and outpatient treatment costs (7%; EUR 467) were important further direct cost components. The mean care grade allowance as an approximator of informal nursing care costs was EUR 929 (median EUR 0; 95% CI EUR 780–1083) over three months. Mean indirect costs totaled EUR 3174 (median EUR 0; 95% CI EUR 2503–3840) among working-age individuals (< 67 years in Germany). Multiple regression analyses revealed mTOR inhibitor use and persistent seizures as independent cost-driving factors for total direct costs. Older age and disability were independent cost-driving factors for total indirect costs, whereas epilepsy, psychiatric disease, and disability were independent cost-driving factors for nursing care costs. Conclusions This three-month study revealed substantial direct healthcare, indirect healthcare, and medication costs associated with TSC in Germany. This study highlights the spectrum of organ manifestations and their associated treatment needs in the German healthcare setting. Trial registration: DRKS, DRKS00016045. Registered 01 March 2019, http://www.drks.de/DRKS00016045.

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Section: Methodsmentioning

confidence: 99%

Direct and indirect costs and cost-driving factors in adults with tuberous sclerosis complex: a multicenter cohort study and a review of the literature

Zöllner

Grau

Rosenow

et al. 2021

Orphanet J Rare Dis

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“…Patients aged 60 years or older had lower costs as compared with patients aged younger than 20 years, maybe owing to less frequent testing. In a US study based on commercial and governmental claims data [114] adult and pediatric TSC patients with AML utilized more resources than did the general population. Direct health care costs (in 2013 USD) in commercial claims were between USD 29,240 (PPP-$ 31,605) and USD 48,499 (PPP-$ 52,422) for TSC patients, or 14 to 22 times higher than that in the general population.…”

Section: Direct Costsmentioning

confidence: 99%

A systematic review on the burden of illness in individuals with tuberous sclerosis complex (TSC)

Zöllner

Franz

Hertzberg

et al. 2020

Orphanet J Rare Dis

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Objective: This review will summarize current knowledge on the burden of illness (BOI) in tuberous sclerosis complex (TSC), a multisystem genetic disorder manifesting with hamartomas throughout the body, including mainly the kidneys, brain, skin, eyes, heart, and lungs. Methods: We performed a systematic analysis of the available literature on BOI in TSC according to the PRISMA guidelines. All studies irrespective of participant age that reported on individual and societal measures of disease burden (e.g. health care resource use, costs, quality of life) were included. Results: We identified 33 studies reporting BOI in TSC patients. Most studies (21) reported health care resource use, while 14 studies reported quality of life and 10 studies mentioned costs associated with TSC. Only eight research papers reported caregiver BOI. Substantial BOI occurs from most manifestations of the disorder, particularly from pharmacoresistant epilepsy, neuropsychiatric, renal and skin manifestations. While less frequent, pulmonary complications also lead to a high individual BOI. The range for the mean annual direct costs varied widely between 424 and 98,008 International Dollar purchasing power parities (PPP-$). Brain surgery, end-stage renal disease with dialysis, and pulmonary complications all incur particularly high costs. There is a dearth of information regarding indirect costs in TSC. Mortality overall is increased compared to general population; and most TSC related deaths occur as a result of complications from seizures as well as renal complications. Long term studies report mortality between 4.8 and 8.3% for a follow-up of 8 to 17.4 years. Conclusions: TSC patients and their caregivers have a high burden of illness, and TSC patients incur high costs in health care systems. At the same time, the provision of inadequate treatment that does not adhere to published guidelines is common and centralized TSC care is received by no more than half of individuals who need it, especially adults. Further studies focusing on the cost effectiveness and BOI outcomes of coordinated TSC care as well as of new treatment options such as mTOR inhibitors are necessary.

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“…Exhaustive search of commercial and Medicaid databases in the United States indicates that TSC‐renal disease patients utilized more healthcare services than their matched controls, with an annual average total cost that is 10–20 times higher than that of control patients ( p < .05) (Song et al, ). Work from the United Kingdom confirms the high economic burden of TSC (Kingswood, Crawford, et al, ) and that renal complications are a major contributor to this cost (Kingswood, Nasuti, et al, ).…”

Section: Introductionmentioning

confidence: 99%

Renal manifestation of tuberous sclerosis complex

Bissler

Kingswood

2018

American J of Med Genetics Pt C

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Tuberous sclerosis complex (TSC) is a tumor predisposition syndrome with significant renal cystic and solid tumor disease. It commonly causes several types of cystic disease and benign tumors (angiomyolipomata) in the kidneys that can both lead to significant premature loss of glomerular filtration rate. The main risks of angiomyolipomata, severe bleeding, loss of renal function, and pulmonary lymphangioleiomyomatosis, can be ameliorated by active surveillance and preemptive therapy with mTOR inhibitors. The cystogenic mechanism may involve primary cilia, but also appears to also involve a majority of normal tubular cells and may be driven by a minority of cells with mutations inactivating both their TSC1 or TSC2 genes. Malignant tumors are rare.

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Healthcare utilization and costs in patients with tuberous sclerosiscomplex-related renal angiomyolipoma

Abstract: Compared to controls, TSC-renal AML patients incurred substantially higher annual healthcare utilization and costs.

Cited by 7 publications

References 14 publications

Direct and indirect costs and cost-driving factors in adults with tuberous sclerosis complex: a multicenter cohort study and a review of the literature

Direct and indirect costs and cost-driving factors in adults with tuberous sclerosis complex: a multicenter cohort study and a review of the literature

A systematic review on the burden of illness in individuals with tuberous sclerosis complex (TSC)

Renal manifestation of tuberous sclerosis complex

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