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Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant

Bone Marrow Transplantation volume 57, pages 586–592 (2022)Cite this article

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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).

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Fig. 1: Generic HRQoL trajectories based on least square means from repeated measures models.
Fig. 2: HSCT-specific trajectories based on least square means from repeated measures models.

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Acknowledgements

We would like to thank Virginia Davenport, RN for her editorial assistance. We would also like to thank the patients and families who participated in this clinical trial and all the members of the external data safety monitoring committee and external advisory committee.

Funding

This study was supported in large part by FDA R01FD004090 and in small part by the Pediatric Cancer Research Foundation. However, the funding entities had no role in the design of the study or data collection, analysis or interpretation.

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Authors and Affiliations

  1. Department of Medicine and Pediatrics, Tufts Medical Center, Boston, MA, USA

    Susan K. Parsons, Angie Mae Rodday & Ruth Ann Weidner

  2. Department of Pediatrics, New York Medical College, Valhalla, NY, USA

    Erin Morris, Suzanne Braniecki, Anne Panarella, Allyson Flower, Jordan Milner, Sandra Fabricatore, Harshini Mahanti, Carmella van de Ven & Mitchell S. Cairo

  3. Department of Pediatrics, Washington University, St Louis, MO, USA

    Shalini Shenoy

  4. Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA

    Julie-An Talano

  5. Department of Pediatrics, University of California Los Angeles, Los Angeles, CA, USA

    Theodore B. Moore

  6. Department of Biostatistics, New York Medical College, Valhalla, NY, USA

    Qiuhu Shi

  7. Department of Medicine, New York Medical College, Valhalla, NY, USA

    Mitchell S. Cairo

  8. Department of Pathology, New York Medical College, Valhalla, NY, USA

    Mitchell S. Cairo

  9. Microbiology & Immunology, New York Medical College, Valhalla, NY, USA

    Mitchell S. Cairo

  10. Cell Biology & Anatomy, New York Medical College, Valhalla, NY, USA

    Mitchell S. Cairo

Authors
  1. Susan K. Parsons
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  2. Angie Mae Rodday
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  15. Qiuhu Shi
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  16. Mitchell S. Cairo
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Contributions

SKP, SS, JAT, TBM, and MSC conceived and designed the study. SKP, AMR, RAW, EM, SB, SS, JAT, TBM, AP, AF, JM, SF, HM, CVV, QS, and MSC contributed to the data acquisition, data analysis and data interpretation. SKP, AMR, RAW, CVV, and QS performed the statistical analysis. All authors critically reviewed the manuscript for important intellectual content. The corresponding author designed the study, had full access to all the data in the study and had final responsibility for the decision to submit for publication.

Corresponding author

Correspondence to Mitchell S. Cairo.

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Competing interests

MSC has received grant support from the FDA (R01FD004090), Otsuka Pharmaceutical, the Pediatric Cancer Research Foundation and non-financial support from Miltenyi Biotech during the conduct of this study. SS reports consulting for Graphite Bio. All other authors declare no conflicts of interest. This research has been presented in part at the American Society of Hematology (ASH), December 2018, San Diego, CA.

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Parsons, S.K., Rodday, A.M., Weidner, R.A. et al. Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant. Bone Marrow Transplant 57, 586–592 (2022). https://doi.org/10.1038/s41409-022-01584-y

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