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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.
  • +12
  • Taryn Fay-McClymont,
  • Dania Monagel,
  • GURPREET SINGH,
  • Fiona Schulte,
  • Brian Brooks,
  • William MacAllister,
  • Naddley Desire,
  • Aleksandra Mineyko,
  • Marsha Vasserman,
  • Michael Leaker,
  • Tony Truong,
  • Ravi Shah,
  • Victor Lewis,
  • Keith Yeates,
  • Gregory Guilcher
Taryn Fay-McClymont
Alberta Children's Hospital

Corresponding Author:[email protected]

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Dania Monagel
King Saud bin Abdulaziz University for Health Sciences
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GURPREET SINGH
Alberta Children's Hospital
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Fiona Schulte
University of Calgary Cumming School of Medicine
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Brian Brooks
Alberta Children's Hospital
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William MacAllister
Alberta Children's Hospital
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Naddley Desire
The Hospital for Sick Children
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Aleksandra Mineyko
University of Calgary Cumming School of Medicine
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Marsha Vasserman
Alberta Children's Hospital
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Michael Leaker
Alberta Children's Hospital
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Tony Truong
Alberta Children's Hospital
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Ravi Shah
Alberta Children's Hospital
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Victor Lewis
Alberta Childrens Hosptial
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Keith Yeates
Alberta Children's Hospital Research Institute for Child and Maternal Health
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Gregory Guilcher
Alberta Children's Hospital
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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.
26 Jul 2022Published in Pediatric Blood & Cancer. 10.1002/pbc.29893