Hydroxyurea effectiveness in children and adolescents with sickle cell anemia: A large retrospective, population-based cohort.

The clinical efficacy of hydroxyurea in patients with sickle cell anemia (SCA) has been well established. However, data about its clinical effectiveness in practice is limited. We evaluated the clinical effectiveness of hydroxyurea in a large pediatric population using a retrospective cohort, pre-post treatment study design to control for disease severity selection bias. The cohort included children with SCA (SS, Sß0 thalassemia) who received care at Children's Healthcare of Atlanta (CHOA) and who initiated hydroxyurea in 2009-2011. Children on chronic transfusions, or children with inadequate follow up data and/or children who had taken hydroxyurea in the 3 years prior were excluded. For each patient healthcare utilization, laboratory values, and clinical outcomes for the 2-year period prior to hydroxyurea initiation were compared to those 2 years after initiation. Of 211 children with SCA who initiated hydroxyurea in 2009-2011, 134 met eligibility criteria. After initiation of hydroxyurea, rates of hospitalizations, pain encounters, and emergency department visits were reduced by 47% (<0.0001), 36% (P = 0.0001) and 43% (P < 0.0001), respectively. Average hemoglobin levels increased by 0.7 g/dl (P < 0.0001). Hydroxyurea effectiveness was similar across gender, insurance types and age, although there was a slightly greater reduction in hospitalizations in younger children. Am. J. Hematol. 92:77-81, 2017. © 2016 Wiley Periodicals, Inc.

Improving an Administrative Case Definition for Longitudinal Surveillance of Sickle Cell Disease.

OBJECTIVE: Several states are building infrastructure and data collection methods for longitudinal, population-based surveillance systems for selected hemoglobinopathies. The objective of our study was to improve an administrative case definition for sickle cell disease (SCD) to aid in longitudinal surveillance. METHODS: We collected data from 3 administrative data sets (2004-2008) on 1998 patients aged 0-21 in Georgia who had ≥1 encounter in which an SCD International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) code was recorded, and we compared these data with data from a laboratory and medical record review. We assessed performance (sensitivity, specificity, positive predictive value [PPV], and negative predictive value [NPV]) of case definitions that differed by number and type of SCD-coded encounters; addition of SCD-associated treatments, procedures, and complications; and length of surveillance (1 vs 5 years). We identified correct diagnoses for patients who were incorrectly coded as having SCD. RESULTS: The SCD case definition of ≥3 SCD-coded encounters in 5 years simplified and substantially improved the sensitivity (96.0% vs 85.8%) and NPV (68.2% vs 38.2%) of the original administrative case definition developed for 5-year, state-based surveillance (≥2 encounters in 5 years and ≥1 encounter for an SCD-related treatment, procedure, or complication), while maintaining a similar PPV (97.4% vs 97.4%) and specificity (76.5% vs 79.0%). CONCLUSIONS: This study supports an administrative case definition that specifies ≥3 ICD-9-CM-coded encounters to identify SCD with a high degree of accuracy in pediatric patients. This case definition can be used to help establish longitudinal SCD surveillance systems.