TY - JOUR
T1 - Neurocognitive functioning in preschool children with sickle cell disease
AU - Heitzer, Andrew M.
AU - Cohen, Diana L.
AU - Okhomina, Victoria I.
AU - Trpchevska, Ana
AU - Potter, Brian
AU - Longoria, Jennifer
AU - Porter, Jerlym S.
AU - Estepp, Jeremie H.
AU - King, Allison
AU - Henley, Misham
AU - Kang, Guolian
AU - Hankins, Jane S.
N1 - Funding Information:
Jane S. Hankins, Jeremie H. Estepp, and Allison King receive research funding from Global Blood Therapeutics. Jeremie H. Estepp receives consultancy fees from Global Blood Therapeutics, Forma Therapeutics, and bluebird bio. Jane S. Hankins receives consultancy fees from Global Blood Therapeutics, Vindico Medical Education, UpToDate, and bluebird bio. There are no other conflicts of interest to report.
Funding Information:
Jane S. Hankins received funding from U01HL133996 during the conduct of this study. Allison King received funding from R01HL129241, K24HL148305, K12HL137942, U01HL143477, and 5U01HL133994 during the time of his study. Jerlym S. Porter was supported by K01HL125495 at the time of this project. This research was supported by the American Lebanese Syrian Associated Charities (ALSAC). The authors would like to thank Jason Hodges, PhD, Pei‐Lin Chen, MPH, Courtney Mays, Erin MacArthur, MS, Madelene Wilson, Tiana Thomas, Ruth Johnson, and Michelle Brignac for support with data collection and regulatory matters.
Publisher Copyright:
© 2021 Wiley Periodicals LLC
PY - 2022/3
Y1 - 2022/3
N2 - Background: Children with sickle cell disease (SCD) experience neurodevelopmental delays; however, there is limited research with preschool-age children. This study examined neurocognitive risk and protective factors in preschoolers with SCD. Procedure: Sixty-two patients with SCD (60% HbSS/HbSβ0-thalassemia; 40% HbSC/HbSβ+-thalassemia) between the ages of 3 and 6 years (mean = 4.77 years) received a neuropsychological evaluation as routine systematic surveillance. Patients were not selected for disease severity, prior central nervous system findings, or existing cognitive concerns. Thirty-four patients (82% HbSS/HbSβ0-thalassemia) were prescribed hydroxyurea (HU) at the time of their neuropsychological evaluation. On average, these patients had been prescribed HU at 2.15 (standard deviation = 1.45) years of age. The average dose was 28.8 mg/kg/day. Besides genotype, there were no group differences in medical or demographic factors based on HU treatment status. Results: Patients with HbSS/HbSβ0-thalassemia scored below normative expectations on measures of intelligence, verbal comprehension, and school readiness (false discovery rate-adjusted p-value [pFDR] <.05). Age, sickle genotype, and HU treatment exposure were not associated with measured neurocognitive outcomes (pFDR >.05). Greater social vulnerability at the community level was associated with poorer performance on measures of intellectual functioning, verbal comprehension, visuomotor control, and school readiness, as well as parent report of executive dysfunction (pFDR <.05). Greater household socioeconomic status was positively associated with academic readiness. Conclusions: Preschoolers with severe SCD (HbSS/HbSβ0-thalassemia) perform below age expectations on measures of intelligence and academic readiness. Sociodemographic factors were stronger drivers of neurocognitive performance than disease severity or disease-modifying treatment. Neurodevelopmental interventions targeting the home and broader community environment are needed.
AB - Background: Children with sickle cell disease (SCD) experience neurodevelopmental delays; however, there is limited research with preschool-age children. This study examined neurocognitive risk and protective factors in preschoolers with SCD. Procedure: Sixty-two patients with SCD (60% HbSS/HbSβ0-thalassemia; 40% HbSC/HbSβ+-thalassemia) between the ages of 3 and 6 years (mean = 4.77 years) received a neuropsychological evaluation as routine systematic surveillance. Patients were not selected for disease severity, prior central nervous system findings, or existing cognitive concerns. Thirty-four patients (82% HbSS/HbSβ0-thalassemia) were prescribed hydroxyurea (HU) at the time of their neuropsychological evaluation. On average, these patients had been prescribed HU at 2.15 (standard deviation = 1.45) years of age. The average dose was 28.8 mg/kg/day. Besides genotype, there were no group differences in medical or demographic factors based on HU treatment status. Results: Patients with HbSS/HbSβ0-thalassemia scored below normative expectations on measures of intelligence, verbal comprehension, and school readiness (false discovery rate-adjusted p-value [pFDR] <.05). Age, sickle genotype, and HU treatment exposure were not associated with measured neurocognitive outcomes (pFDR >.05). Greater social vulnerability at the community level was associated with poorer performance on measures of intellectual functioning, verbal comprehension, visuomotor control, and school readiness, as well as parent report of executive dysfunction (pFDR <.05). Greater household socioeconomic status was positively associated with academic readiness. Conclusions: Preschoolers with severe SCD (HbSS/HbSβ0-thalassemia) perform below age expectations on measures of intelligence and academic readiness. Sociodemographic factors were stronger drivers of neurocognitive performance than disease severity or disease-modifying treatment. Neurodevelopmental interventions targeting the home and broader community environment are needed.
UR - http://www.scopus.com/inward/record.url?scp=85122613814&partnerID=8YFLogxK
U2 - 10.1002/pbc.29531
DO - 10.1002/pbc.29531
M3 - Article
C2 - 34971013
AN - SCOPUS:85122613814
VL - 69
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
SN - 1545-5009
IS - 3
M1 - e29531
ER -