Sensitivity and Specificity of a Signaling Question for Surveillance of Cognitive Functioning in Pediatric Sickle Cell Disease.

Background: Sickle cell disease (SCD) confers neurological risks that contribute to cognitive and academic difficulties. Clinical guidelines state that cognition should be monitored using signaling questions. However, evidence is lacking regarding the extent to which signaling questions accurately identify children with cognitive issues.

Adding hydroxyurea to chronic transfusion therapy for sickle cell anemia reduces transfusion burden.

Background: Chronic red blood cell (RBC) transfusion is an established therapy to prevent stroke in patients with sickle cell anemia (SCA). It is unclear if adding daily hydroxyurea treatment to chronic transfusion is beneficial.

Study Design And Methods: We conducted a phase 2 clinical trial (NCT03644953) investigating the addition of dose-escalated hydroxyurea to chronic transfusion for patients with SCA receiving simple chronic transfusion for stroke prevention.

Disparity in HLA typing rates among hospitalized pediatric patients with sickle cell disease.

Hematopoietic stem cell transplantation (HSCT) using an HLA-identical sibling donor is a well-established cure for sickle cell disease (SCD). The proportion of patients with SCD who have completed HLA typing, a key first step in considering HSCT, is unknown. We sought to determine the prevalence of HLA typing among patients with SCD hospitalized at our institution, identify characteristics associated with having had typing, and describe the acceptability of this testing.

Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease.

Disease-modifying therapies are standard of care (SOC) for sickle cell disease (SCD), but hematopoietic cell transplantation (HCT) has curative potential. We compared outcomes prospectively through 2-years after biologic assignment to a Donor or No Donor (SOC) Arm based on the availability of an HLA-matched sibling or unrelated donor (BMTCTN 1503; NCT02766465). A donor search was commenced after eligibility confirmation.