Trends in blood transfusion, hydroxyurea use, and iron overload among children with sickle cell disease enrolled in Medicaid, 2004–2019

Abstract

Background: There have been significant changes in clinical guidelines for sickle cell disease (SCD) over the past two decades, including updated indications for hydroxyurea, transfusions, and iron overload management. In practice however, there are few studies that examine SCD care utilization over time. Methods:We conducted a serial cross-sectional cohort study of pediatric SCD patients from 2004 to 2019 using Georgia Medicaid claims data. For each year, we reported receipt of any transfusion, chronic transfusion, or three or more filled hydroxyurea prescriptions. For children receiving chronic transfusion (six or more annual transfusions), we evaluated iron overload diagnosis, monitoring, and chelation use. Among children with sickle cell anemia (SCA), we examined rates of transfusions and hydroxyurea use.The Cochran-Armitage test was used to assess trend.Results: There were 5316 unique children 2-18 years old with SCD enrolled in Georgia Medicaid from 2004 to 2019. Children receiving any transfusion increased from 2004 to 2010, then stabilized. In SCA patients, chronic transfusions initially increased from 2004 to 2010, then stabilized from 2010 to 2019. For chronically transfused children, monitoring of iron burden and filled chelator prescriptions both increased significantly. Hydroxyurea use in SCA patients increased from 12% to 37%, with increases noted within each age group, most notably from 21% to 60% in the 13-18-year-old cohort. Conclusion:We demonstrated changes in SCD care utilization over time, including increased hydroxyurea use, changes in transfusion rates, and increased attention to iron overload management. While trends in clinical management do follow updates in treatment guidelines, there is still delayed and suboptimal uptake of guideline recommendations in pediatric SCD patients.

Iron Load Toxicity in Medicine: From Molecular and Cellular Aspects to Clinical Implications

“…Another inherited hemoglobinopathy affecting millions of people is SCD, which is prevalent in Africa and many other countries with descendants from Africa. In this abnormal hemoglobin condition, a single amino acid change in the beta globin chain can cause hemoglobin polymerization, sickling of RBC, anemia, and painful side effects as a result of the sickling crisis [ 3 , 29 , 30 ]. Treatment with RBC transfusions improves anemia and alleviates some categories of the SCD patients from the painful side effects of the sickling crisis.…”

“…Millions of patients with hemoglobinopathies and other refractory anemias worldwide, including thalassemia, sickle cell disease (SCD), myelodyspasia, and hematopoietic stem cell transplantation (HCT), receive regular red blood cell (RBC) transfusions for the treatment of their anemia [ 1 , 2 , 3 , 4 , 5 ].…”

Emerging Therapeutic Options for Sickle Cell Anemia: Enhancing the Current Predominant Treatment

“…We have read the article entitled "Trends in blood transfusion, hydroxyurea use, and iron overload among children with sickle cell disease enrolled in Medicaid, 2004-2019" by Amy et al [1] with great interest and appreciate the effort put into the research. However, upon reviewing the manuscript, we have identified a few ideas that we believe are worth discussing to enhance the overall quality of the article.…”

Comment on: Trends in blood transfusion, hydroxyurea use, and iron overload among children with sickle cell disease enrolled in Medicaid, 2004–2019. Emerging therapeutic options for sickle cell anemia: Enhancing the current predominant treatment

“…The American Society of Pediatric Hematology/Oncology 1 with great interest and appreciation for the effort put into the research. However, upon reviewing the manuscript, we have identified a few ideas that we believe are worth discussing to enhance the overall quality of the article.…”