Neuropsychological, Behavioral, and Quality of Life Outcomes in Children
and Adolescents with Sickle Cell Disease Treated with Nonmyeloablative
Matched Sibling Donor Hematopoietic Cell Transplantation: A case series.
Abstract
Background/Objectives. Despite advances in the treatment of sickle cell
disease (SCD), cerebrovascular and cognitive consequences can be
lifelong. Hematopoietic cell transplantation (HCT) is an established
curative therapy and recent studies have demonstrated efficacy with
reduced toxicity nonmyeloblative (NMA) regimens, but little is known
about neuropsychological outcomes. The objective of this study was to
describe neuropsychological, behavioral, and quality of life outcomes
with medical correlates in children with SCD who received an NMA matched
sibling donor (MSD) HCT. Design/Methods. This retrospective cohort
analysis of nine patients with hemoglobin SS SCD underwent MSD HCT using
the National Institutes of Health (NIH) NMA protocol. Results. Mean full
scale intellectual functioning (FSIQ) was average pre-HCT (FSIQ=92.1, SD
9.0; n=8) and 2 years post-HCT (mean FSIQ=96.6; SD 11.1; N=9).
Neuropsychological functioning was largely average across all cognitive
domains. Moderate improvements were seen in processing speed and verbal
memory (Cohen’s d=0.50-0.57) post-HCT, and declines occurred in measures
of attention and fine motor speed and dexterity (Cohen’s d=0.70-0.81).
Parents reported improved quality of life (Cohen’s d=0.91), less impact
of SCD on their family, and less worry about their child’s future
(Cohen’s d=1.44). Exploratory analysis showed relationships between
pre-HCT hemoglobin (r=0.74, p<0.05) and creatinine (r=-0.75,
p<0.01) with cognitive functioning, and a positive
relationship between processing speed and time post-HCT (r=0.73).
Conclusion. Neuropsychological functioning in a sample of children and
adolescents treated identically with NMA MSD HCT remained stable or
improved in most cognitive domains, and improvements in quality of life
and family functioning were observed.