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 (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.