Clinical burden and healthcare resource utilization associated with managing transfusion-dependent ß-thalassemia in France.

Objective: This retrospective, real-world claims database analysis aimed to describe the clinical burden and healthcare resource utilization associated with managing transfusion-dependent ß-thalassemia (TDT) in France.Methods: We used the French National Health Data System (système national des données de santé) to identify eligible patients from January 1, 2012, to March 1, 2019. Inclusion criteria were a diagnosis of ß-thalassemia, ≥8 red blood cell (RBC) transfusion episodes per year in ≥2 consecutive years following the diagnosis, and ≥1 year of follow-up data. Patients were excluded if their medical records showed evidence of sickle cell disease, α-thalassemia, hereditary persistence of fetal hemoglobin, or hematopoietic stem cell transplant. Clinical complications, mortality, treatment use, and healthcare resource utilization were evaluated in the follow-up period.Results: Overall, 331 eligible patients with TDT were identified. The mean age was 26.1 (standard deviation [SD]: 18.0) years, and 50.5% were male. Common clinical complications were endocrine (26.0%), hepatobiliary (22.7%), and cardiopulmonary (18.7%). Fifteen (4.5%) patients died during follow-up, with a mortality rate of 1.16 deaths per 100 person-years (mean age of death: 52.5 years [SD: 22]). Patients had a mean of 13.5 (SD: 5.2) RBC transfusion episodes and 11.2 (SD: 5.3) iron chelation therapy treatments per patient per year. healthcare resource utilization was substantial, with a mean of 14.8 inpatient hospitalizations (including 13.8 mean inpatient day cases) and 16.9 outpatient prescriptions per patient per year.Conclusions: Patients with TDT in France experience significant clinical complications, elevated mortality, and substantial healthcare resource utilization driven by frequent RBC transfusion episodes and inpatient hospitalizations. These results reinforce the need for disease-modifying therapies for this patient population.

Economic and clinical burden of managing transfusion-dependent ß-thalassemia in the United States.

AIMS: To describe clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with transfusion-dependent ß-thalassemia (TDT) in the United States. MATERIALS AND METHODS: Merative MarketScan Databases were used to identify patients with ß-thalassemia between 1 March 2010, and 1 March 2019. Patients were eligible for inclusion with ≥1 inpatient claim or ≥2 outpatient claims for ß-thalassemia and ≥8 red blood cell transfusions (RBCTs) during any 12-month period after and including the date of the first qualifying ß-thalassemia diagnosis code. Matched controls consisted of individuals without ß-thalassemia. Clinical and economic outcomes of patients were assessed during ≥12 months of follow-up, defined as the period from the index date (i.e. the first RBCT) to either the end of continuous enrollment in benefits, inpatient death, or 1 March 2020. RESULTS: Overall, 207 patients with TDT and 1035 matched controls were identified. Most patients received iron chelation therapy (ICT) (91.3%), with a mean of 12.1 (standard deviation [SD] = 10.3) ICT claims per-patient-per-year (PPPY). Many also received RBCTs, with a mean of 14.2 (SD = 4.7) RBCTs PPPY. TDT was associated with higher annual ($137,125) and lifetime ($7.1 million) healthcare costs vs. matched controls ($4183 and $235,000, respectively). Annual costs were driven by ICT (52.1%) and RBCT use (23.6%). Patients with TDT had 7-times more total outpatient visits/encounters, 3-times more prescriptions, and 33-times higher total annual costs than matched controls. LIMITATIONS: This analysis may underestimate the burden of TDT, as indirect healthcare costs (e.g. absenteeism, presenteeism, etc.) were not included. Results may not be generalizable to patients excluded from this analysis, including those with other types of insurance or without insurance. CONCLUSIONS: Patients with TDT have high HCRU and direct healthcare costs. Treatments that eliminate the need for RBCTs could reduce the clinical and economic burden of managing TDT.

Economic and Clinical Burden of Managing Sickle Cell Disease with Recurrent Vaso-Occlusive Crises in the United States.

INTRODUCTION: The aim of this study was to describe the clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in the US. METHODS: Merative MarketScan Databases were used to identify patients with SCD with recurrent VOCs from March 1, 2010, to March 1, 2019. Inclusion criteria were ≥ 1 inpatient or ≥ 2 outpatient claims for SCD and ≥ 2 VOCs per year in any 2 consecutive years after the first qualifying SCD diagnosis. Individuals without SCD in these databases were used as matched controls. Patients were followed for ≥ 12 months, from their second VOC in the 2nd year (index date) to the earliest of inpatient death, end of continuous enrollment in medical/pharmacy benefits, or March 1, 2020. Outcomes were assessed during follow-up. RESULTS: In total, 3420 patients with SCD with recurrent VOCs and 16,722 matched controls were identified. Patients with SCD with recurrent VOCs had a mean of 5.0 VOCs (standard deviation [SD] = 6.0), 2.7 inpatient admissions (SD 2.9), and 5.0 emergency department visits (SD 8.0) per patient per year during follow-up. Compared to matched controls, patients with SCD with recurrent VOCs incurred higher annual ($67,282 vs. $4134) and lifetime ($3.8 million vs. $229,000 over 50 years) healthcare costs. CONCLUSION: Patients with SCD with recurrent VOCs experience substantial clinical and economic burden driven by inpatient costs and frequent VOCs. There is a major unmet need for treatments that alleviate or eliminate clinical complications, including VOCs, and reduce healthcare costs in this patient population.

Switch rates and total cost associated with apremilast and biologics in biologic-naive patients with psoriatic arthritis.

Aim: Real-world treatment data for psoriatic arthritis are limited. We evaluated switch rates, adherence, and costs for patients initiating apremilast versus tumor necrosis factor inhibitor (TNFi) and interleukin inhibitor (ILi) among biologic-naive psoriatic arthritis patients. Materials & methods: This retrospective analysis used IBM MarketScan claims data to assess treatment switches, adherence and costs. Results: Twelve-month switch rates were significantly lower for apremilast versus TNFi (15.5% vs 26.6%; p < 0.0001) and similar to ILi (15.5% vs 14.0%; p = 0.71). Apremilast initiators had lower total costs versus TNFi and ILi (US$39,854 vs US$57,243 and US$65,687; p < 0.05) and adherence was slightly lower versus TNFi and higher versus ILi. Conclusion: Biologic-naive apremilast initiators had lower switch rates versus TNFi initiators and lower total costs versus TNFi or ILi initiators.

Switch Rates and Total Cost of Care Associated with Apremilast and Biologic Therapies in Biologic-Naive Patients with Plaque Psoriasis.

PURPOSE: Compare treatment switching rates and costs among biologic-naive psoriasis patients initiating apremilast or biologics. METHODS: This retrospective claims analysis used IBM MarketScan Commercial and Medicare Supplemental databases to identify patients who initiated apremilast or a biologic (ie, tumor necrosis factor [TNF] or interleukin [IL] inhibitor) for psoriasis treatment between January 1, 2015, and December 31, 2016. A 1:1 propensity score matching was used to adjust for possible selection bias and maximize the number of patients available for analysis. Treatment switching, days to switch, and healthcare costs were assessed at 12 months. T-test and chi-square test were used to evaluate differences between cohorts for continuous and categorical variables as appropriate; Wilcoxon rank-sum tests were used to assess cost differences. RESULTS: In total, 88,025 patients newly initiated apremilast, a TNF inhibitor, or an IL inhibitor. After inclusion/exclusion criteria were applied and patients were propensity score matched, 1645 (apremilast), 1207 (TNF inhibitor), and 438 (IL inhibitor) patients were included in this analysis. Twelve-month switch rates were significantly lower for apremilast initiators compared with TNF inhibitor initiators (14% vs 25%; p<0.01) and comparable to IL inhibitors (14% vs 11%; p>0.05). No statistical difference was observed in days to switch at 12 months for any treatment group. Total healthcare costs were lower for apremilast initiators compared with TNF and IL inhibitor initiators ($34,028 vs $55,973 and $64,430; p<0.0001). Per-patient per-month (PPPM) costs were significantly lower for apremilast initiators compared with TNF inhibitor and IL inhibitor initiators ($2834 vs $4662 and $5366; p<0.0001). CONCLUSION: Over a 12-month follow-up, biologic-naive psoriasis patients initiating apremilast had significantly lower switching rates compared with patients on TNF inhibitors and similar rates as patients on IL inhibitors. PPPM and total healthcare costs were significantly lower for patients initiating apremilast vs TNF or IL inhibitors, primarily due to lower pharmacy costs.

Impact of Outpatient vs Inpatient ABSSSI Treatment on Outcomes: A Retrospective Observational Analysis of Medical Charts Across US Emergency Departments.

BACKGROUND: The objective of this study was to characterize treatment of patients with acute bacterial skin and skin structure infections (ABSSSIs) and describe the association between hospital admission and emergency department (ED) visits or readmissions within 30 days after initial episode of care (IEC). METHODS: This was a retrospective, observational, cohort study of adults with ABSSSI who presented to an ED between July 1, 2012, and June 30, 2013. Patient, health care facility, and treatment characteristics, including unplanned ED visits or readmissions, were obtained through manual chart review and abstraction. Adjusted logistic regression analysis examined likelihood of all-cause unplanned ED visits or readmissions between admitted and nonadmitted patients. RESULTS: Records from 1527 ED visits for ABSSSI from 40 centers were reviewed (admitted, n = 578 [38%]; nonadmitted, n = 949 [62%]). Admitted patients were typically older (mean age, 52.2 years vs 43.0 years), more likely to be morbidly obese (body mass index > 40 kg/m2; 17.3% vs 9.1%), and had more comorbidities (Charlson Comorbidity Index ≥ 4; 24.4% vs 6.8%) compared with those not admitted. In the primary analysis, adjusted logistic regression, controlling for comorbidities and severity of illness, demonstrated that there was a similar likelihood of all-cause unplanned ED visits or readmissions between admitted and nonadmitted patients (odds ratio, 1.03; 95% confidence interval, 0.74-1.43; P = .87). CONCLUSIONS: ABSSSI treatment pathways leveraging outpatient treatment vs hospital admission support similar likelihood of unplanned 30-day ED visits or readmissions, an important clinical outcome and quality metric at US hospitals. Further research regarding the decision criteria around hospital admission to avoid potentially unnecessary hospitalizations is warranted.