Clinical burden and healthcare resource utilization of patients with sickle cell disease and recurrent vaso-occlusive crises or transfusion-dependent beta-thalassemia in the Netherlands.

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 (January 1, 2014-December 31, 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 (n = 54) 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.