Evolution of Treatments for Hereditary Angioedema in Italy: Use of Lanadelumab in Clinical Practice

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Introduction: Hereditary angioedema (HEA) is a rare genetic disorder characterised by recurrent episodes ofedema affecting various body districts. HEA therapy includes treatment of acute attacks and short- or long-termprophylaxis.Objective: To describe the main demographic and clinical characteristics of patients with HEA in real Italian clinicalpractice, as well as the use of pharmacological treatments, the use of healthcare resources and costs withparticular reference to lanadelumab.Methods: From administrative databases of healthcare institutions for about 9 million patients, between January2010 and September 2020, all-age HEA patients were identified through hospitalisation, exemption code or specificdrugs. Demographic and clinical characteristics, medications prescribed for acute attacks and prophylaxiswere described at inclusion. For patients treated with lanadelumab, the switching of dosing regimen from 2 to 4weeks was examined. Healthcare resource utilisation and costs were assessed at one-year follow-up.Results: 258 patients with HEA were identified (~0.003% of the sample). Of them, 41.1% were male, and themean age was 44.6 years; 80% of patients were treated for acute attacks, and 20% were on prophylaxis. Inlanadelumab-treated, 85% started treatment with a 2-week regimen, and of them 75.8% switched to a 4-weekregimen. For these patients, the estimated annual cost—calculated using the weighted average of patients whoswitched treatments—was €114,362.Conclusions: The data from this analysis on therapeutic management of HEA patients in the Italian clinical practiceshowed that, despite the limited number of patients treated with lanadelumab, many received one administrationevery 4 weeks, suggesting a good disease control.

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Healthcare utilization of patients with hereditary angioedema treated with lanadelumab and subcutaneous C1-inhibitor concentrate.
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Diagnosing Pediatric Patients With Hereditary C1-Inhibitor Deficiency—Experience From the Hungarian Angioedema Center of Reference and Excellence
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Hereditary Angioedema: Diagnosis, Clinical Implications, and Pathophysiology
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Characteristics and Drug Utilization of Patients with Hereditary Angioedema in Italy, a Real-World Analysis.
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Treatment of COPD: Relationships between daily dosing frequency, adherence, resource use, and costs
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Treatment of COPD: Relationships between daily dosing frequency, adherence, resource use, and costs

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  • 10.1001/jamapediatrics.2015.3446
Effect of Attribution Length on the Use and Cost of Health Care for a Pediatric Medicaid Accountable Care Organization.
  • Feb 1, 2016
  • JAMA Pediatrics
  • Eric W Christensen + 1 more

Little is known about the effect of pediatric accountable care organizations (ACOs) on the use and costs of health care resources, especially in a Medicaid population. To assess the association between the length of consistent primary care (length of attribution) as part of an ACO and the use and cost of health care resources in a pediatric Medicaid population. A retrospective study of Medicaid claims data for 28,794 unique pediatric patients covering 346,277 patient-attributed months within a single children's hospital. Data were collected for patients attributed from September 1, 2013, to May 31, 2015. The effect of the length of attribution within a single hospital system's ACO on the use and costs of health care resources were estimated using zero-inflated Poisson distribution regression models adjusted for patient characteristics, including chronic conditions and a measure of predicted patient use of resources. Receiving a plurality of primary care at an ACO clinic during the preceding 12 months (attribution to the ACO). The primary outcome measure was the length of attribution at an ACO clinic compared with subsequent inpatient hospitalization and subsequent use and cost of outpatient and ancillary health care resources. Among the 28,794 pediatric patients receiving treatment covering 346,277 patient-attributed months during the study period, continuous attribution to the ACO for more than 2 years was associated with a decrease (95% CI) of 40.6% (19.4%-61.8%) in inpatient days but an increase (95% CI) of 23.3% (2.04%-26.3%) in office visits, 5.8% (1.4%-10.2%) in emergency department visits, and 15.3% (12.5%-18.0%) in the use of pharmaceuticals. These changes in the use of health care resources combined resulted in a cost reduction of 15.7% (95% CI, 6.6%-24.8%). At the population level, the impact of consistent primary care was muted by the many patients in the ACO having shorter durations of participation. These findings suggest significant and durable reductions of inpatient use and cost of health care resources associated with longer attribution to the ACO, with attribution as a proxy for exposure to the ACO's consistent primary care. Consistent primary care among the pediatric Medicaid population is challenging, but these findings suggest substantial benefits if consistency can be improved.

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First Salvage Therapy for Relapsed or Refractory Acute Myeloid Leukemia: Associated Health Care Resource Use and Costs
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  • Lori Muffly + 5 more

First Salvage Therapy for Relapsed or Refractory Acute Myeloid Leukemia: Associated Health Care Resource Use and Costs

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  • 10.1016/j.clinthera.2013.10.005
Treatment Patterns, HealthCare Resource Utilization and Costs in Patients with Bipolar Disorder, Newly Treated with Extended Release or Immediate Release Quetiapine Fumarate using US Healthcare Administrative Claims Data
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Treatment Patterns, HealthCare Resource Utilization and Costs in Patients with Bipolar Disorder, Newly Treated with Extended Release or Immediate Release Quetiapine Fumarate using US Healthcare Administrative Claims Data

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Healthcare resource utilization and costs of major atherothrombotic vascular events among patients with peripheral artery disease after revascularization
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  • Journal of Medical Economics
  • Urvi Desai + 9 more

Aims Peripheral artery disease (PAD), often treated with lower extremity revascularization, is associated with risk of major atherothrombotic vascular events (acute limb ischemia [ALI], major non-traumatic lower-limb amputation, myocardial infarction [MI], ischemic stroke, cardiovascular death). This study aims to assess healthcare resource utilization and costs of such events among patients with PAD after revascularization. Materials and methods Patients aged ≥50 years with PAD who were treated with lower-extremity revascularization were identified from Optum Clinformatics Data Mart claims database (01/2014–06/2019). The first lower extremity revascularization after PAD diagnosis was defined as the index date. Patients had ≥6 months of health plan enrollment before the index date. Patients were followed until the earliest of 1) end of enrollment or data; 2) diagnosis of atrial fibrillation or venous thromboembolism; or 3) oral anticoagulant use. All-cause healthcare resource use per-patient-year was compared before and after a major atherothrombotic vascular event post-revascularization among those with an event. Additionally, event-related healthcare costs per-patient-year were reported for each event type. Results Of the 38,439 PAD patients meeting the study criteria, 6,675 (17.4%) had a major atherothrombotic vascular event. On average, patients were observed for 7.3 months before an event and 6.2 months after an event. Patients with an event had significantly higher all-cause healthcare resource use versus similar metrics pre-event (e.g. inpatient visits among those with ALI: 3.5 ± 5.8 post-event vs. 2.0 ± 8.1 pre-event, p < .05). Event-related costs ranged from $57,825±$131,810 per-patient-year for ischemic stroke to $108,302±$150,168 for major non-traumatic lower-limb amputation. Limitations Data do not contain clinical information. Additionally, results are limited to commercially insured and Medicare Advantage beneficiaries. Conclusion Patients with PAD who experience major atherothrombotic vascular events post-revascularization have considerably higher healthcare resource use and costs compared with similar metrics pre-event. Therefore, reducing the rate of such events could reduce overall healthcare costs for this population.

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Healthcare utilization and costs among patients with psoriasis and psoriatic arthritis in the USA-a retrospective study of claims data from 2009 to 2020.
  • May 2, 2021
  • Clinical Rheumatology
  • J F Merola + 10 more

To compare healthcare resource utilization and costs among patients with psoriasis, psoriatic arthritis (PsA), and a control group of patients without psoriasis and PsA in the USA. The IBM® MarketScan® Commercial Database was used to identify three adult patient groups from 1/1/2009 through 4/30/2020: (1) Psoriasis: ≥ 2 diagnoses ≥ 30 days apart for psoriasis (no PsA diagnoses); (2) PsA: ≥ 2 diagnoses for PsA; (3) Control: no psoriasis or PsA diagnoses in their entire claims records. Patients with comorbid rheumatoid arthritis, ankylosing spondylitis, Crohn's disease, or ulcerative colitis were excluded from the analyses. Controls were matched 1:1 to psoriasis and PsA patients based on age, gender, index year, and number of non-rheumatological comorbidities. Healthcare resource utilization and costs (in 2019 USD) were evaluated descriptively and through mixed models for five years of follow-up. A total of 142,531 psoriasis and 21,428 PsA patients were matched to the control group (N = 163,959). Annual all-cause healthcare costs per patient were $7,470, $11,062, and $29,742 for the control, psoriasis, and PsA groups, respectively. All-cause healthcare costs increased over time and were significantly greater among PsA vs. psoriasis (p < 0.0001) and the control groups (p < 0.0001). Across all categories of healthcare resources, utilization was greatest among patients with PsA and lowest in the control group. Annual healthcare costs and resource utilization were significantly higher with PsA compared with psoriasis and the control group, confirming the substantial economic burden of PsA. The cost disparity between these patient groups highlights a continued unmet medical need. Key Points • Patients with PsA incurred significantly greater healthcare resource utilization and costs than patients with psoriasis and patients without psoriasis and PsA. • Significantly greater costs and healthcare resource utilization were also observed among patients with psoriasis compared with patients without psoriasis and PsA.

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  • Cite Count Icon 3
  • 10.1111/head.14822
Healthcare resource use and costs associated with the misdiagnosis of migraine.
  • Aug 28, 2024
  • Headache
  • Jae Rok Kim + 6 more

To compare healthcare resource utilization and healthcare costs in patients with migraine with or without a history of misdiagnosis. Despite the high prevalence of migraine, migraine is commonly misdiagnosed. The healthcare resource use and cost burden of a misdiagnosis is unknown. This retrospective cohort study identified adults with an incident migraine diagnosis from the Merative™ Marketscan® Commercial and Medicare Supplemental Databases between June 2018 and 2019. Patients with a diagnosis of commonly considered misdiagnoses (headache, sinusitis, or cervical pain) before their migraine diagnosis were classified as the "misdiagnosed cohort." Patients in the misdiagnosed cohort were potentially misdiagnosed, then eventually received a correct diagnosis. Patients without a history of commonly considered misdiagnoses prior to their migraine diagnosis were classified as the "correctly diagnosed cohort." Healthcare resource utilization and healthcare costs were assessed in the period before migraine diagnosis and compared between the cohorts. Outcomes were reported as per patient per month and compared with incidence rate ratios. A total of 29,147 patients comprised the correctly diagnosed cohort and 3841 patients comprised the misdiagnosed cohort and met the inclusion criteria. Patients in the misdiagnosed cohort had statistically significantly higher rates of inpatient admissions (0.02 vs. 0.01, incidence rate ratio [IRR] 1.61, 95% confidence interval [CI] 1.47-1.74), emergency department visits (0.10 vs. 0.05; IRR 1.89, 95% CI 1.79-1.99), neurologist visits (0.12 vs. 0.02; IRR 5.95, 95% CI 5.40-6.57), non-neurologist outpatient visits (2.64 vs. 1.58; IRR 1.67, 95% CI 1.62-1.72) and prescription fills (2.82 vs. 1.84; IRR 1.53, 95% CI 1.48-1.58) compared to correctly diagnosed patients. Misdiagnosed patients had statistically significantly higher rates of healthcare cost accrual for inpatient admissions ($1362 vs. $518; IRR 2.62, 95% CI 2.50-2.75), emergency department visits ($222 vs. $98; IRR 2.27, 95% CI 2.18-2.36), neurologist visits ($42 vs. $9; IRR 4.39, 95% CI 4.00-4.79), non-neurologist outpatient visits ($1327 vs. $641; IRR 2.07, 95% CI 1.91-2.24), and prescription fills ($305 vs. $215; IRR 1.41, 95% CI 1.18-1.70) compared to correctly diagnosed patients. Patients with migraine who have a history of misdiagnoses have higher rates of healthcare resource utilization and cost accrual versus those without such history.

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Comparison of real-world healthcare resource utilization and costs among patients with hereditary angioedema on lanadelumab or berotralstat long-term prophylaxis.
  • Apr 1, 2025
  • Journal of comparative effectiveness research
  • Nicole Princic + 5 more

Aim: Hereditary angioedema (HAE) is a rare and chronic genetic condition. Lanadelumab and berotralstat, two plasma kallikrein inhibitors, have both been approved for long-term prophylaxis in patients with HAE; however, real-world data comparing costs and healthcare resource utilization (HCRU) are lacking. Materials & methods: This retrospective study used administrative healthcare insurance claims data (Merative™ MarketScan® Commercial, Medicare and Early View Research Databases; 1 July 2017-31 July 2023) to identify patients with HAE who initiated lanadelumab or berotralstat and were persistent for ≥18months or 6months, respectively. Sex, baseline healthcare costs and baseline number of on-demand treatment/short-term prophylaxis medication claims were used to calculate covariate balancing propensity scores for inverse probability of treatment weighting. Following weighting, outcomes during the 6-month follow-up period in patients receiving berotralstat were compared with those during months 0-6, 7-12 and 13-18 in lanadelumab-treated patients. Results: Fifty-seven lanadelumab- and 32 berotralstat-treated patients were included. After weighting, more berotralstat-treated patients had an all-cause inpatient admission (berotralstat, 9.4%; lanadelumab, months 0-6, 4.0%, 7-12, 1.8%, months 13-18, 2.0%) and emergency room visit (berotralstat, 21.9%; lanadelumab, months 0-6, 14.0%, 7-12, 8.0%, months 13-18, 17.9%). Total HAE treatment costs were similar during months 0-6 (lanadelumab, $377,326 vs berotralstat, $373,010), but decreased in months 7-12 ($319,967) and 13-18 ($283,241) of lanadelumab. On-demand treatment/short-term prophylaxis costs were lower for lanadelumab across the three follow-up periods than for berotralstat during months 0-6 (berotralstat, $60,451; lanadelumab, months 0-6, $46,336, months 7-12, $37,578, months 13-18, $23,968). The proportion of lanadelumab-treated patients who reduced dosing frequency was 24.8% during months 7-12 and 21.6% during months 13-18. Conclusion: Patients with HAE initiating lanadelumab versus berotralstat may require less on-demand and supportive HAE treatments and incur lower treatment-related and total healthcare costs. The ability to reduce lanadelumab dosing frequency after an attack-free period may be key in treatment selection, given the combination of cost savings and lower healthcare resource utilization.

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Healthcare Resource Utilization During a Multimodal Nutritional Program with Oral Nutritional Supplements in Malnourished Outpatients.
  • Sep 3, 2025
  • Nutrients
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Background/Objective: We compared healthcare utilization and cost outcomes for patients before and after they underwent a nutrition care intervention that included daily consumption of specialized or standard oral nutritional supplements (ONS) as part of a multimodal program. We sought to determine whether this nutritional intervention was associated with reduced use of healthcare resources and lowered costs. Methods: This retrospective analysis included adult patients who were referred to the medical nutrition office with malnutrition or its risk. We followed outcomes for patients who had received nutritional interventions (3-6 months) that included diet and exercise recommendations as well as a specialized ONS enriched with β-hydroxy-β-methylbutyrate (HMB-ONS) or a standard ONS (S-ONS). We reviewed hospital records for resource utilization data-hospital (re)admissions, number of days in the hospital, patient visits to the emergency department (ED), and visits to general practitioners (GP) and specialty physician clinics. We compared healthcare utilization for intervals up to 12 months before and after the initiation of the nutrition care intervention. We also examined healthcare utilization and costs as well as changes in oncology treatment following initiations of the nutrition care intervention for a subgroup of cancer patients. Results: Over a 12-month period following the start of nutritional intervention with ONS, the patients had lower healthcare resource use and costs. With daily use of S-ONS or HMB-ONS for up to 6 months, the patients' healthcare utilization was significantly reduced at 3-, 6-, and 12-month time points for hospital admissions, ED visits, GP visits, and specialty visits (all p < 0.001). Overall, ONS use was associated with reduced patient healthcare costs by half (EUR 27,024.80 to EUR 13,349.60, p < 0.001). Significant clinical predictors of higher use of healthcare resources and costs were older age, having positive indications of malnutrition, exhibiting a greater Charlson Comorbidity Index (CCI), and having been hospitalized for oncology surgery. Regression analysis revealed that ONS intervention was associated with lower total healthcare costs (βPost-ONS = -0.504), and that patients receiving HMB-ONS (βONS-HMB = -0.583) had a greater reduction in costs than did patients receiving S-ONS in the 12 months after nutritional intervention. Patients with cancer who received HMB-ONS were less likely than those receiving S-ONS to need suspension or dose reduction in oncology drug treatment due to failure or intolerance of the treatment drug. Conclusions: multimodal programs including ONS improve health economic outcomes for patients with poor nutritional status due to disease. Health economic outcome improvements included lower healthcare resource use, lower healthcare costs, and, for patients being treated for cancer, a reduced likelihood of treatment failure. The use of ONS enriched with HMB provided advantages over standard ONS, both in health outcomes and cost-of-care reductions.

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  • Cite Count Icon 25
  • 10.3111/13696998.2015.1132225
Frequency of skeletal-related events and associated healthcare resource use and costs in US patients with multiple myeloma
  • Jan 22, 2016
  • Journal of Medical Economics
  • Emily Nash Smyth + 6 more

Objective:A potential complication for all new multiple myeloma (MM) patients is the clinical presentation of osteolytic lesions which increase the risk for skeletal-related events (SREs). However, the contribution of SREs to the overall economic impact of MM is unclear. The impact of SREs on healthcare resource utilization (HCRU) and costs for US patients with MM was analyzed in Truven Health Marketscan Commercial Claims and Medicare Supplemental Databases.Methods:Adults diagnosed with MM between January 1, 2005 and December 31, 2010 with ≥2 claims ≥30 days apart (first claim = index date) were included. SREs included: hypercalcemia, pathologic fracture, surgery for the prevention and treatment of pathologic fractures or spinal cord compression, and radiation for bone pain. Rates of HCRU (outpatient [OP], inpatient [IP], emergency room [ER], orthopedic consultation [OC], and ancillary) and healthcare costs were compared between MM patients with and without SREs. Inverse propensity weighting was applied to adjust for potential bias.Results:Of 1028 MM patients (mean age = 67, standard deviation = 13.2), 596 patients with ≥1 SRE and 432 without SREs were assessed. HCRU rates in IP, ER, and ancillary (p < 0.01) and mean total costs of OP, IP, and ER were significantly higher (p < 0.05) for patients with vs without SREs during follow-up. HCRU rates also increased with SRE frequency (p < 0.05 in OP, IP, ER, OC, and ancillary), as did mean total healthcare costs, except for OC (p < 0.001).Limitations:A broad assessment of pharmacotherapy for the treatment of MM was not an objective of the current study. Bisphosphonate use was evaluated; however, results were descriptively focused on frequency of utilization only and were not included in the broader cost and HCRU analysis.Conclusions:Among US patients with MM, higher SRE frequency was associated with a significant trend of higher HCRU and total healthcare costs in several settings.

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Health Care Resource Use and Economic Burden in Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy and Atrial Fibrillation
  • Aug 22, 2025
  • Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
  • John C Stendahl + 7 more

BackgroundAtrial fibrillation (AF) is common among patients with obstructive hypertrophic cardiomyopathy (oHCM), although the impact of AF on health care resource use and costs is not well defined.MethodsWe performed a retrospective analysis of claims data from 2016 to 2021 and used International Classification of Diseases, Tenth Revision (ICD‐10) codes to identify adult patients with symptomatic oHCM and classify their status with respect to AF as follows: (1) prevalent AF, (2) incident AF, and (3) no AF. Health care resource use and costs for each cohort were analyzed and expressed as per person per year (PPPY).ResultsOf 22 216 patients with symptomatic oHCM, 6677 had prevalent AF (30.1%), 2879 had incident AF (13.0%), and 12 660 were without AF (57.0%). Patients with incident AF incurred mean total health care costs that were similar to those with prevalent AF but substantially greater than those without AF (mean, $66 619 [95% CI, $59 702–$74 336] versus $63 937 [95% CI, $59 803–$68 356] versus $46 686 [95% CI, $43 901–$49 648] per person per year, P<0.0001). After adjusting for age, sex, major comorbidities, and septal reduction therapy, mean total health care costs remained greater in the groups with incident and prevalent AF than the group without AF, with trends toward even greater relative costs in the group with incident AF. Similar trends were present in adjusted costs related to hospitalizations, surgeries, and urgent care.ConclusionsThe diagnosis of AF in the setting of symptomatic oHCM not only has important implications for patient management but also substantial economic impacts, as it is associated with significantly greater health care costs and resource use relative to patients with symptomatic oHCM and no AF.

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  • Cite Count Icon 1
  • 10.1016/j.anai.2021.07.015
Demographic and clinical characteristics of patients with hereditary angioedema in Canada
  • Jul 20, 2021
  • Annals of Allergy, Asthma &amp; Immunology
  • Erika Yue Lee + 8 more

Demographic and clinical characteristics of patients with hereditary angioedema in Canada

  • Abstract
  • 10.1182/blood-2019-128368
Healthcare Resource Utilization and Costs in Patients with Relapsed and Refractory Diffuse Large B-Cell Lymphoma: A U.S. Real-World Observational Study
  • Nov 13, 2019
  • Blood
  • Xiaoqin Yang + 8 more

Healthcare Resource Utilization and Costs in Patients with Relapsed and Refractory Diffuse Large B-Cell Lymphoma: A U.S. Real-World Observational Study

  • Abstract
  • 10.1182/blood-2022-167262
Healthcare Resource Utilization and Costs of Relapsed or Refractory Patients with Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma Treated with Ibrutinib
  • Nov 15, 2022
  • Blood
  • Xiaoqin Yang + 6 more

Healthcare Resource Utilization and Costs of Relapsed or Refractory Patients with Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma Treated with Ibrutinib

  • Research Article
  • Cite Count Icon 36
  • 10.3111/13696998.2015.1075995
Kidney involvement in tuberous sclerosis complex: the impact on healthcare resource use and costs
  • Aug 26, 2015
  • Journal of Medical Economics
  • Francis Vekeman + 6 more

Objective:Tuberous sclerosis complex (TSC) is associated with non-malignant kidney lesions—angiomyolipomata—that may be associated with chronic kidney disease (CKD). This study investigated the relationship between renal angiomyolipomata and CKD in TSC, including the impact on healthcare resource utilization (HCRU) and costs.Methods:This was a retrospective, longitudinal cohort study based on medical record data spanning January 1990–April 2012 for 369 TSC patients treated at a specialty center in the Netherlands. Cohorts were established based on CKD stage and angiomyolipoma size. Rates of HCRU (physician visits, monitoring, and interventions) were compared across cohorts using rate ratios. Healthcare costs were compared across cohorts using cost differences. Regression models were used to identify predictive factors for HCRU and healthcare costs.Results:Sixteen per cent of patients reached CKD stage 3 or higher during follow-up. Patients at more advanced stages of CKD more frequently had either large or multiple small angiomyolipomata and higher HCRU rates and healthcare costs. In the multivariate analyses, male gender, CKD stage >1, angiomyolipoma size ≥3.5 cm, embolization, and the presence of moderate or severe lymphangioleiomyomatosis (LAM) were associated with greater HCRU (p ≤ 0.002 for all comparisons). Definite (vs suspected) TSC diagnosis, CKD stage 5 (vs CKD stage 1), angiomyolipoma size ≥3.5 cm, and moderate or severe LAM were associated with higher costs (p = 0.050 for TSC diagnosis, p ≤ 0.002 for other comparisons). Costs in CKD stage 5 were driven primarily by dialysis.Conclusions:A substantial proportion of patients with TSC developed moderate-to-severe CKD, which was associated with renal angiomyolipomata and increased HCRU and costs.

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