Adherence, treatment utilization, clinical and economic outcomes of patients with sickle cell disease with recurrent vaso-occlusive crises treated with recently approved chronic therapies in the US.

Adherence, Clinical and Economic Outcomes in Patients with Sickle Cell Disease with Recurrent Vaso-Occlusive Crises Treated with L-Glutamine, Voxelotor, or Crizanlizumab Covered By Medicaid and Commercial Insurance in the United States

Clinical and Economic Outcomes in Patients with Transfusion-Dependent β-Thalassemia and Patients with Sickle Cell Disease with Recurrent Vaso-Occlusive Crises Receiving Hematopoietic Stem Cell Transplants in the United States

Prevalence of Vaso-Occlusive Crises in Patients with Sickle Cell Disease: A Retrospective US Claims Database Analysis

IntroductionThe 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.MethodsMerative 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.ResultsIn 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.ConclusionPatients 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.Supplementary InformationThe online version contains supplementary material available at 10.1007/s12325-023-02545-7.

The purpose of this study was to describe clinical complications and healthcare resource utilization (HCRU) among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and patients with transfusion-dependent β-thalassemia (TDT) in Germany. The Betriebskrankenkasse (BKKs) Database was used to identify patients with SCD or TDT. To be eligible for inclusion, patients with SCD were required to have ≥ 2 VOCs/year in any two consecutive years and ≥ 12 months of available data before and after the index date (second VOC in the second consecutive year). Patients with TDT were required to have ≥ 8 red blood cell transfusions (RBCTs) in any 12-month period and ≥ 12 months of available data after the index date (first RBCT). Clinical and HCRU outcomes were analyzed during follow-up. Overall, 84 patients with SCD with recurrent VOCs and 68 patients with TDT were identified in the BKKs database. Among patients with SCD with recurrent VOCs, the most prevalent complications were retinopathy (45.2%), multisystem organ disease/failure (40.5%), and mental health complications (31.0%); among patients with TDT, they were endocrine (69.1%) and cardiopulmonary (55.9%) complications and malignancies (44.1%). Patients with SCD experienced a mean of 4.0 (standard deviation [SD] 3.9) VOCs and 1.9 (SD 2.5) hospitalizations per patient per year (PPPY) during follow-up. Patients with TDT had a mean (SD) of 16.4 (11.2) RBCTs and 59.4 (40.8) outpatient visits PPPY. Patients with SCD with recurrent VOCs or TDT in Germany experience significant clinical complications and HCRU.

Introduction The purpose of this study was to describe the clinical burden and healthcare resource utilization (HCRU) among patients with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs) and patients with transfusion-dependent β-thalassemia (TDT) in the Netherlands. Methods This retrospective cohort study identified two patient populations, one cohort with SCD and recurrent VOCs and the other with TDT from the PHARMO Data Network (1 January 2014–31 December 2020). Key inclusion criteria were diagnosis of SCD and ≥2 VOCs per year for 2 consecutive years following the diagnosis for SCD; or diagnosis of β-thalassemia and ≥8 red blood cell transfusions (RBCTs) per year for 1 year following the diagnosis for β-thalassemia. Complications and HCRU were evaluated for each cohort. Results A total of 383 patients with SCD and recurrent VOCs and a total of 54 patients with TDT were identified with mean ages at index of 26.9 (standard deviation [SD]: 14.4) years and 17.7 (SD: 15.2) years, respectively. Patients with SCD and recurrent VOCs experienced an average of 7.0 VOCs per patient per year (PPPY). The most common acute complication was acute chest syndrome (34.9%), and the most common chronic complications were bone and joint complications (12.0%). Patients had a mean of 2.5 inpatient hospitalizations and 7.0 outpatient visits PPPY. Patients with TDT received an average of 13.0 RBCTs PPPY. The most common acute complication was infection (5.6%), and the most common chronic complication was transfusion-induced iron overload (33.3%). Patients had a mean of 11.3 inpatient hospitalizations, mainly driven by transfusion-related hospitalizations, and 8.4 outpatient visits PPPY. Conclusion Patients with SCD and recurrent VOCs and patients with TDT in the Netherlands sustain substantial clinical complications and HCRU mainly related to VOCs and regular RBCTs, respectively.

Clinical Burden and Health Care Resource Utilization Associated With Managing Sickle Cell Disease With Recurrent Vaso-occlusive Crises in England

Title: The Impact of Recent Vaso-Occlusive Crisis on Health-Related Quality of Life in Adults with Sickle Cell Disease

Objective This retrospective, real-world claims database analysis described the clinical burden and healthcare resource utilization (HCRU) among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in France. Methods The French National Health Data System database (système national des données de santé) was used to identify eligible patients from 1 January 2012 to 1 March 2019. Inclusion criteria were a SCD diagnosis, ≥2 VOCs/year for ≥2 consecutive years following the diagnosis, and ≥1 year of follow-up data to 1 March 2020. Patients with hereditary persistence of fetal hemoglobin or hematopoietic stem cell transplant in their medical records were excluded. Clinical complications, mortality, treatment use, and HCRU were evaluated during follow-up. Results Overall, 4602 patients with SCD with recurrent VOCs were eligible; their mean (standard deviation [SD]) age was 19.8 (13.5) years, and 51.8% were female. Patients experienced a mean (SD) of 3.82 (3.57) VOCs per patient per year (PPPY). Prevalent complications were anemia or leukocytosis (44.1%), infections (42.0%), and organ failure (38.2%). In total, 101 (2.2%) patients died during follow-up (mean age of death [SD]: 39.3 [17.5] years; mortality rate: 0.64 deaths per 100 person-years). Most patients received opioids (89.1%) and hydroxycarbamide (72.8%). Patients had a mean of 5.7 inpatient hospitalizations, 6.0 emergency room visits, 6.6 outpatient visits, and 13.4 outpatient prescriptions PPPY. Conclusions Patients with SCD with recurrent VOCs in France have substantial clinical complications, mortality, and HCRU despite currently available treatment options. Innovative treatments that reduce frequency of or eliminate VOCs are needed to alleviate the burden associated with SCD.

Sickle cell disease (SCD) is characterized by vaso-occlusive crises (VOCs) that can lead to clinical complications, higher mortality, and healthcare resource use (HCRU). Lack of Canadian data poses challenges to health system planning, a gap this study intends to close. We analyzed clinical complications, mortality, and HCRU in patients with SCD with recurrent VOCs in Canada. This retrospective cohort study identified patients with SCD with recurrent VOCs from the Institute for Clinical Evaluative Sciences (ICES) databases in Ontario, Canada from January 1, 2010, to December 31, 2021. VOCs were defined as SCD with crisis, priapism, or acute chest syndrome. Patients were matched (1:3) by age, sex, and geographic area with non-SCD individuals in the general population. Clinical complications, mortality, and HCRU were analyzed. Eight hundred fifty-nine patients with SCD with recurrent VOCs were identified and matched to 2577 controls. Mean (standard deviation [SD]) age was 22.1 (14.4) years; 50.9% were female. Mean (SD) rate of VOCs per person per year was 3.2 (4.4) in patients with recurrent VOCs. Acute and chronic complication rates were higher in patients than controls. Compared with controls, mortality was substantially higher for patients with recurrent VOCs. Mean (SD) age at death for patients with recurrent VOCs was 39.2 (17.2) years. There was significantly higher HCRU than matched controls (P < 0.0001). Patients with SCD with recurrent VOCs had substantially higher rates of complications, mortality, and HCRU than controls, further highlighting the need for novel therapies to reduce VOCs and the associated burden.

Clinical Complications and Healthcare Resource Utilization in Medicaid and Commercially Insured Patients with Sickle Cell Disease Receiving Frequent Red Blood Cell Transfusions

To examine the clinical burden and healthcare resource utilization (HCRU) among patients with transfusion-dependent β-thalassemia (TDT) and patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in Italy. Eligible patients were identified from an administrative claims database from 1 January 2010 and 1 February 2019. Patients with TDT had ≥ 1 iron chelation treatment, ≥ 8 red blood cell transfusions (RBCTs) during any 12-month period, and ≥ 12 months of available data pre- and post-index (i.e., first RBCT claim). Patients with SCD with recurrent VOCs had ≥ 2 VOCs/year in ≥ 2 consecutive years and ≥ 12 months of available data pre- and post-index (second VOC claim in the second of 2 consecutive years). Patients were propensity score matched to five controls by age, sex, geographic area, and index year. Clinical and HCRU outcomes were evaluated post-index. In total, 214 patients with TDT and 111 patients with SCD with recurrent VOCs were matched to 1070 and 555 controls, respectively. Both patient groups had substantially higher mortality rates than controls (TDT: 4.8 versus 0.8 deaths per 100 person-years; SCD: 1.6 versus 0.4 deaths per 100 person-years). Clinical complications were prevalent in both patient groups. Compared with controls, both patient groups had significantly higher mean rates of all-cause hospitalizations (TDT: 1.4 versus 0.1; SCD: 2.0 versus 0.1) and outpatient services (TDT: 21.9 versus 1.6; SCD: 6.2 versus 1.0) per patient per year (all: p < 0.05). Management of TDT and SCD in Italy is associated with significant clinical and health system burden, highlighting the need for new treatments that eliminate RBCTs and VOCs.

Standard of care for patients with sickle cell disease (SCD) includes red blood cell transfusions (RBCTs). Data on clinical and economic outcomes of patients with SCD receiving frequent RBCTs are limited. This longitudinal, retrospective, claims-based analysis used the Merative™ MarketScan® Commercial, Medicare, and Multi-State Medicaid databases. Patients with SCD (identified using ICD-9/10 codes) receiving frequent RBCTs (≥6 RBCTs during any 12-month period) between January 1, 2015, and March 1, 2019, were included. The index date was the date of the sixth RBCT. Eligible patients were required to have ≥12 months of continuous enrollment pre- and post-index. Patients were followed from index to end of enrollment, death, or end of the study period (February 29, 2020), whichever came first. Clinical complications, all-cause healthcare resource utilization (HCRU), and healthcare costs were descriptively summarized during follow-up. A total of 919 patients with SCD receiving frequent RBCTs met the eligibility criteria for inclusion. Patients experienced a mean of 4.0 vaso-occlusive crises (VOCs) per patient per year (PPPY) and received a mean of 8.3 RBCTs PPPY during follow-up. The most common clinical complications were iron overload (77%), infections (66%), and cerebrovascular disease (48%). Patients had a mean of 2.3 inpatient admissions, 83.5 outpatient visits, and 37.4 outpatient prescriptions PPPY during follow-up. Mean total annual healthcare costs were $106,123 PPPY, including mean inpatient, outpatient medical, and outpatient pharmacy costs of $48,463, $28,307, and $29,353, respectively. Compared to those with <2 baseline VOCs, patients with ≥2 baseline VOCs had more HCRU and higher annual healthcare costs. Despite utilizing available care with frequent RBCTs, patients with SCD experienced a variety of disease and transfusion-related complications, including frequent VOCs and iron overload, which led to substantial HCRU and costs. These findings highlight the need for novel therapies for this patient group.

Vaso-Occlusive Crises and Costs of Sickle Cell Disease from a Commercial Payer's Perspective

28-Year-Old Man With Joint Pain