(016) Assessment of Evidence-Based Pharmacological Therapy for Pediatric Patients with Sickle Cell Disease

Poster Abstract:

Background:
In the United States, approximately 100,000 people currently living with sickle cell disease (SCD). There are multiple subtypes of SCD, including hemoglobin SS (HbSS), which is generally considered to be a more severe phenotype. SCD is known to have a significant number of complications, such as vaso-occlusive crises, acute chest syndrome, infections, splenic sequestration, stroke, and early death. Despite these complications, there has been growth in the understanding and treatment of SCD in recent decades. The use of hydroxyurea has improved SCD outcomes, showing a decrease in vaso-occlusive crises, acute chest syndrome, need for blood transfusions, and mortality. Other strides in management include penicillin prophylaxis for those less than 5 years of age, optimization of immunizations, and novel agents.

Objectives:
The purpose of this study is to holistically evaluate pediatric patients with HbSS disease by assessing adherence to evidence-based pharmacological therapy. The data collected will be utilized to create a dashboard within the electronic medical record (EMR) to assist in SCD directed therapy assessments.

Methods:
This study is a retrospective, single-center chart review assessing current compliance with guideline directed therapy for patients with HbSS. Patients will be reviewed based on health-care encounters with the hematology team in the inpatient or outpatient setting between April 1st, 2023 – December 31st, 2023. Regarding treatment of patients with HbSS disease, data collected includes an active prescription for hydroxyurea or were clinically managed with erythrocytapheresis, supportive care with antibiotic prophylaxis for patient less than 5 years old, and vaccinations assessed per current guideline recommendations.

Results:
An interim analysis comprising 83 patients was performed. Of those reviewed, 78.3% were on hydroxyurea and 20.5% were receiving erythrocytapheresis, with only 1.2% of patients not receiving any SCD-directed therapy. Supportive care results showed 85% of patients less than 5 were receiving penicillin prophylaxis and, upon comparison, those up to date on vaccinations were 59% vs 9.4% in the previous study (p < 0.05).
Discussion/conclusions:
Interim results demonstrated that most patients were receiving either hydroxyurea or erythrocytapheresis for SCD. However, penicillin prophylaxis can be improved for those less than 5 years of age. Comparing vaccination rates, there has been significant progress in vaccination status at this facility. These results highlight strength in therapy with hydroxyurea or erythrocytapheresis and opportunity for growth in penicillin prophylaxis and vaccinations.

References (must also be included in final poster): 1. Riley TR, Boss A, McClain D, et al. Review of Medication Therapy for the Prevention of Sickle Cell Crisis. P T. July 2018. 43(7): 417-421, 437.
2. Kavanagh PL, Fasipe TA, Wun T. Sickle Cell Disease: A Review. JAMA. July 2022. 328(1): 57-68.
3. Quantifying the Life Expectancy Gap for People Living with Sickle Cell Disease.
4. Retrieved from https://www.hematology.org/newsroom/press-releases/2023/quantifying-the-life-expectancy-gap-for-people-living-with-sickle-cell-disease.
5. Infanti LM, Elder JJ, Franco K, et al. Immunization Adherence in Children With Sickle Cell Disease: A Single-Institution Experience. J Pediatr Pharmacol Ther. 2020. 225(1):39-46.