BACKGROUND: Tuberous sclerosis complex (TSC) is a multi-system genetic disorder in which renal manifestations occur in ∼50% of children and 80% of adults. Since these often present alongside other manifestations, renal TSC is likely to incur significant costs. This study aims to quantify healthcare resource use (HCRU) and costs for renal TSC patients in the UK. METHODS: TSC patients in the Clinical Practice Research Datalink (CPRD) linked to Hospital Episodes Statistics were identified from January 1987-June 2013. Clinical data were extracted over the entire history and costs were reported over the most recent 3-year period. HCRU was compared with a matched comparator cohort. Incremental costs were reported and the key cost drivers by primary manifestation category were identified by regression modeling. RESULTS: A total of 79 renal TSC patients were identified with manifestations including chronic kidney disease stage 3-5 (with prevalence increasing with age) and renal angiomyolipoma. Renal TSC patients consistently reported greater HCRU than the comparator. Inpatient hospitalizations were more frequent for renal TSC patients (3.2 vs 1.6), but length of stay was comparable; however, 70.9% of renal TSC patients recorded no kidney-related procedures ever and averaged <1 test per year in the 3-year period. Average costs for renal TSC patients were nearly 3-fold greater than the comparator (£15,162 vs £5672). Costs increased with additional manifestation categories (£3600: only renal; £27,531: renal with ≥4 additional manifestation categories [25% of patients]). Additional nervous system and dermatology/psychiatric manifestations significantly (p < 0.028) affected costs. CONCLUSIONS: Renal TSC patients have greater HCRU than the general CPRD population, likely to result from progression of renal disease and additional manifestations; however, surveillance for disease progression appears to be deficient. Inadequate monitoring may contribute to a lack of co-ordinated care and increased healthcare-associated costs. Efforts should be made to follow the TSC guidelines to effectively monitor and treat patients.
BACKGROUND:Tuberous sclerosis complex (TSC) is a multi-system genetic disorder in which renal manifestations occur in ∼50% of children and 80% of adults. Since these often present alongside other manifestations, renal TSC is likely to incur significant costs. This study aims to quantify healthcare resource use (HCRU) and costs for renal TSCpatients in the UK. METHODS: TSC patients in the Clinical Practice Research Datalink (CPRD) linked to Hospital Episodes Statistics were identified from January 1987-June 2013. Clinical data were extracted over the entire history and costs were reported over the most recent 3-year period. HCRU was compared with a matched comparator cohort. Incremental costs were reported and the key cost drivers by primary manifestation category were identified by regression modeling. RESULTS: A total of 79 renal TSCpatients were identified with manifestations including chronic kidney disease stage 3-5 (with prevalence increasing with age) and renal angiomyolipoma. Renal TSCpatients consistently reported greater HCRU than the comparator. Inpatient hospitalizations were more frequent for renal TSCpatients (3.2 vs 1.6), but length of stay was comparable; however, 70.9% of renal TSCpatients recorded no kidney-related procedures ever and averaged <1 test per year in the 3-year period. Average costs for renal TSCpatients were nearly 3-fold greater than the comparator (£15,162 vs £5672). Costs increased with additional manifestation categories (£3600: only renal; £27,531: renal with ≥4 additional manifestation categories [25% of patients]). Additional nervous system and dermatology/psychiatric manifestations significantly (p < 0.028) affected costs. CONCLUSIONS:Renal TSCpatients have greater HCRU than the general CPRD population, likely to result from progression of renal disease and additional manifestations; however, surveillance for disease progression appears to be deficient. Inadequate monitoring may contribute to a lack of co-ordinated care and increased healthcare-associated costs. Efforts should be made to follow the TSC guidelines to effectively monitor and treat patients.
Authors: Raouf M Seyam; Waleed K Alkhudair; Said A Kattan; Mohamed F Alotaibi; Hassan M Alzahrani; Waleed M Altaweel Journal: J Kidney Cancer VHL Date: 2017-10-16
Authors: Johann Philipp Zöllner; David Neal Franz; Christoph Hertzberg; Rima Nabbout; Felix Rosenow; Matthias Sauter; Susanne Schubert-Bast; Adelheid Wiemer-Kruel; Adam Strzelczyk Journal: Orphanet J Rare Dis Date: 2020-01-21 Impact factor: 4.123
Authors: Margaret Constanti; Christopher N Floyd; Mark Glover; Rebecca Boffa; Anthony S Wierzbicki; Richard J McManus Journal: Hypertension Date: 2020-12-21 Impact factor: 10.190
Authors: Johann Philipp Zöllner; Janina Grau; Felix Rosenow; Matthias Sauter; Markus Knuf; Gerhard Kurlemann; Thomas Mayer; Christoph Hertzberg; Astrid Bertsche; Ilka Immisch; Karl Martin Klein; Susanne Knake; Klaus Marquard; Sascha Meyer; Anna H Noda; Felix von Podewils; Hannah Schäfer; Charlotte Thiels; Laurent M Willems; Bianca Zukunft; Susanne Schubert-Bast; Adam Strzelczyk Journal: Orphanet J Rare Dis Date: 2021-06-02 Impact factor: 4.123
Authors: Janina Grau; Johann Philipp Zöllner; Susanne Schubert-Bast; Gerhard Kurlemann; Christoph Hertzberg; Adelheid Wiemer-Kruel; Thomas Bast; Astrid Bertsche; Ulrich Bettendorf; Barbara Fiedler; Andreas Hahn; Hans Hartmann; Frauke Hornemann; Ilka Immisch; Julia Jacobs; Matthias Kieslich; Karl Martin Klein; Kerstin A Klotz; Gerhard Kluger; Markus Knuf; Thomas Mayer; Klaus Marquard; Sascha Meyer; Hiltrud Muhle; Karen Müller-Schlüter; Anna H Noda; Susanne Ruf; Matthias Sauter; Jan-Ulrich Schlump; Steffen Syrbe; Charlotte Thiels; Regina Trollmann; Bernd Wilken; Laurent M Willems; Felix Rosenow; Adam Strzelczyk Journal: Orphanet J Rare Dis Date: 2021-06-21 Impact factor: 4.123