Significant Improvement of Child Physical and Emotional Functioning After Familial Haploidentical Stem Cell Transplant

Authors

Susan K. Parsons, Department of Medicine and Pediatrics, Tufts Medical Center, Boston, MA, USA.
Angie Mae Rodday, Department of Medicine and Pediatrics, Tufts Medical Center, Boston, MA, USA.
Ruth Ann Weidner, Department of Medicine and Pediatrics, Tufts Medical Center, Boston, MA, USA.
Erin Morris, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Suzanne Braniecki, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Shalini Shenoy, Department of Pediatrics, Washington University, St Louis, MO, USA.
Julie-An Talano, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA.
Theodore B. Moore, Department of Pediatrics, University of California Los Angeles, Los Angeles, CA, USA.
Anne Panarella, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Allyson Flower, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Jordan Milner, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Sandra Fabricatore, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Harshini Mahanti, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Carmella van de Ven, Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
Qiuhu Shi, Department of Biostatistics, New York Medical College, Valhalla, NY, USA.
Mitchell S. Cairo, Department of Pediatrics, New York Medical College, Valhalla, NY, USA. Mitchell_Cairo@nymc.edu.Follow

Author Type(s)

Faculty

DOI

10.1038/s41409-022-01584-y

Journal Title

Bone Marrow Transplantation

First Page

586

Last Page

592

Document Type

Article

Publication Date

4-1-2022

Department

Pediatrics

Second Department

Public Health

Abstract

Allogeneic stem cell transplantation (AlloSCT) represents the only curative therapy for sickle cell disease (SCD). However, limited availability of matched related donors and suboptimal outcomes following AlloSCT with unrelated donors has led to investigation of alternative donors. Among children with high-risk SCD, we evaluated health-related quality of life (HRQoL) impact in the two years following familial haploidentical SCT. HRQoL was collected from parent and child raters, using the Child Health Ratings Inventories Generic measure and haploidentical SCT-specific module. Repeated measures models were fit to assess HRQoL changes over time and by rater. Nineteen children (mean age 12.9 yrs [standard deviation, 5.3]; 63% male) and their parents were included. There were no differences in the 2-yr trajectories of child physical or emotional functioning (EF) by rater. Child physical functioning and EF scores were significantly lower at day +45 than baseline, but scores recovered by day +180. There was significant improvement in EF (p = 0.03) at 2 yrs vs baseline. A similar pattern of scores over time was seen for parent ratings of child's global HRQoL. Despite treatment intensity in the initial months following AlloSCT, patient scores recovered or exceeded baseline scores at two years. This trial is registered at clinicaltrials.gov (NCT01461837).

Recommended Citation

Parsons, S. K., Rodday, A. M., Weidner, R. A., Morris, E., Braniecki, S., Shenoy, S., Talano, J., Moore, T. B., Panarella, A., Flower, A., Milner, J., Fabricatore, S., Mahanti, H., van de Ven, C., Shi, Q., & Cairo, M. S. (2022). Significant Improvement of Child Physical and Emotional Functioning After Familial Haploidentical Stem Cell Transplant. Bone Marrow Transplantation, 57 (4), 586-592. https://doi.org/10.1038/s41409-022-01584-y