Real-World Evidence on Disease Burden and Economic Impact of Sickle Cell Disease in Italy

Abstract

A real-world analysis was conducted in Italy among sickle cell disease (SCD) patients to evaluate the epidemiology of SCD, describe patients' characteristics and the therapeutic and economic burden. A retrospective analysis of administrative databases of various Italian entities was carried out. All patients with ≥1 hospitalization with SCD diagnosis were included from 01/2010-12/2017 (up to 12/2018 for epidemiologic analysis). The index date corresponded to the first SCD diagnosis. In 2018, SCD incidence rate was 0.93/100,000, the prevalence was estimated at 13.1/100,000. Overall, 1816 patients were included. During the 1st year of follow-up, 50.7% of patients had one all-cause hospitalization, 27.8% had 2, 10.4% had 3, and 11.1% had ≥4. Over follow-up, 6.1-7.2% of patients were treated with SCD-specific, 58.4-69.4% with SCD-related, 60.7-71.3% with SCD-complications-related drugs. Mean annual number per patient of overall treatments was 14.9 ± 13.9, hospitalizations 1.1 ± 1.1, and out-patient services 5.3 ± 7.6. The total mean direct cost per patient was EUR 7918/year (EUR 2201 drugs, EUR 3320 hospitalizations, and EUR 2397 out-patient services). The results from this real-world analysis showed a high disease burden for SCD patients with multiple hospitalizations during the follow-up. High healthcare resource utilization and costs were associated with patient' management and were most likely underestimated since indirect costs and Emergency Room admissions were not included.

Keywords: SCD epidemiology; administrative databases; real-life; transfusions; vaso-occlusive crisis.

Figure 1

(Upper plots A–D) Epidemiology of SCD in the sample population (N = 15,300,00). (A) Incidence rate overall and by gender per year included in the study (2010–2018). (B) Prevalence by gender and age (<18 years and ≥18 years) for year 2018. (C) Prevalence by age classes in adult SCD patients with and without VOC at diagnosis for year 2018. (D) Prevalence by geographic area and presence of crisis for year 2018. (Lower plots E–G) Projections to Italian population for year 2018. (E) Number of SCD patients estimated in Italy by age and gender. (F) Number of SCD patients estimated in Italy by age ranges. (G) Number of SCD patients estimated in Italy by presence of VOCs at diagnosis.

Figure 2

Study flow-chart of patients in analysis. The inclusion period was from 1st January 2010 to 31st December 2017. Patients were identified by SCD diagnosis retrieved from the hospitalization database (ICD-9-CM codes 282.41, 282.42, 282.60, 282.61, 282.62, 282.63, 282.64, 282.68, and 282.69; code 282.50 related to the Sickle cell trait was not considered for inclusion).

Figure 3

Hospitalization of patients with SCD. (A) More frequent primary diagnosis in SCD patients without crisis with secondary SCD diagnosis at inclusion (n = 1129). (B) Percentages of patients with all-cause hospitalizations during follow-up period. Hospitalizations comprised ordinary admissions and day hospitals; Emergency Room access not requiring hospitalization was not included. Hospitalization corresponding to the index date was counted in the number of hospitalizations. (C) Percentages of SCD patients with VOC episodes or complications during follow-up.

Figure 4

Therapeutic pathways and healthcare resource costs. (A) Patients treated with at least one SCD-specific drug, SCD-related drug, and SCD complication-related drug during the follow-up period (1–4 years). (B) Patients with crisis treated with at least one SCD-specific drug, SCD-related drug, and SCD complication-related drug during the follow-up period (1–4 years). (C) Mean annual healthcare resource consumption and related costs considering all available follow-up. List of SCD treatments in analysis and the related ATC code is reported in Supplementary Table S2. Abbreviation: OSS, outpatient services.