Improvement of sickle cell disease care mitigates the healthcare utilization induced by increased prevalence: Experience of a tertiary pediatric center Free

Introduction:Sickle cell disease (SCD) is one of the most frequent monogenic diseases worldwide. SCD has undergone two major changes in the last decades. First, its prevalence is steadily increasing, with recent data showing a 41.4% increase globally from 2000 to 2021 (Lancet Haematology, 2023). Most patients with SCD are living in Sub-Saharan Africa, but the population of children affected is likely growing in North America as well. In particular, epidemiological data are extremely limited in Canada. Second, numerous progresses have been made in the management of the disease and the prevention of the complications. However, the effect in real-life setting of these improvements combined with the increased prevalence of SCD is unknown. We aimed to describe the evolution in the healthcare utilization of children with SCD in a tertiary center of North America in the last 15 years.

Methods:Retrospective review of healthcare utilization markers for pediatric patients (< 18 years old) with SCD from 2009 to 2024 at a pediatric tertiary care center in Canada. Patients followed in obstetrics were excluded. Significant practice changes were made in the emergency department (ED) and hematology department at our institution over the study period, following quality improvement projects of a multidisciplinary group. This included the set-up of a structured SCD program for patient follow-up and screening of complications, increased use of hydroxyurea, and standardization of pain and fever management to favor out-patient management. A provincial newborn screening program was initiated in 2013.

Results:The number of children with SCD followed at our center more than doubled between 2009 and 2024 (221 to 499 patients). Similarly, the absolute number of annual outpatient visits and of ED visits increased significantly: from 1207 to 1769 and from 192 to 526, respectively (p<0.0001). However, this was not reflected in the number of hospitalisations, which remained stable (p=0.89), nor in the number of intensive care admissions (p=0.97). In effect, the mean annual number of outpatient visits, of hospitalisations and of transfusions per patients per year decreased: 5.46 to 3.55 (p=0.03), 1.18 to 0.58 (p<0.0001) and 2.4 to 1.0 (p<0.0001), respectively. There was also a trend towards decreased intensive care admissions by patients per year; 4.98% to 2.40% (p=0.10). Finally, the mean number of ED visits per number of patients followed remained stable (0.87 to 1.05, p=0.42), but the percentage of admissions per visits decreased from 62.5% to 42.6% (p<0.0001). Of note, no patient has been admitted for acute stroke at our institution since 2019. The proportion of hematopoietic stem cell transplantation per patients followed remained stable: 0.45% to 1.20% (p = 0.90). The mean fetal hemoglobin percentage per year increased from 9.14% to 18.39% over the study period (p<0.0001). Interestingly, the highest average fetal hemoglobin percentage was reached in 2017 (19.78%), 3 years after the implementation of systematic introduction of hydroxyurea (HU) around 9-12 months of age in patients with SS or S/Β⁰ phenotypes. Values have remained relatively stable since, which might reflect that the maximal effect of the systematic and earlier introduction of HU has been reached.

Conclusion:

The number of patients living with SCD followed at our institution drastically increased in 15 years. This study provides real-world data about healthcare utilization trends in recent years, showing that the improvement of patient care, the introduction of universal newborn screening and earlier introduction of hydroxyurea likely mitigated the impact on healthcare utilization. It also reflects the positive impact of the practice changes implemented in the ED, which directly led to a decrease in admissions per visit. However, there was an important increase in the resources utilization overall. Increased allocated resources should reflect these changes to allow for continuous optimal care of this vulnerable population.