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Biol Blood Marrow Transplant. 2012 Feb;18(2):162-71. doi: 10.1016/j.bbmt.2011.12.501. Epub 2011 Dec 10.

NCI, NHLBI/PBMTC First International Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: health-related quality of life, functional, and neurocognitive outcomes.

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  • 1Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, Massachusetts, USA.


The purpose of this manuscript is to summarize issues relevant to health-related quality of life (HRQL), physical function, and neurocognitive function for survivors of pediatric hematopoietic stem cell transplantation (HCT). The physiologic and psychological demands of HCT and its sequelae have the potential to substantially alter HRQL. When compared with research on adult HCT recipients, research in pediatric HRQL following HCT has lagged considerably. Initially, this lag was because of limited validated questionnaires, small numbers of affected patients, and a general lack of salience for the topic relative to traditional endpoints, such as transplant-related toxicity and potential mortality. The percentage of childhood HCT survivors with physical disability ranges from 7% to 17% in studies where the outcome was based on clinician or self-report measures, to over 40% in studies where the outcome was based on a directly measured physical performance task. Direct and comprehensive measures of physical performance may help further clarify the proportion of individuals who have subclinical problems amenable to intervention before apparent functional loss becomes a problem. There is a need to include longer term survivors in such an assessment. In terms of neurocognitive function, the majority of reports demonstrate relatively good function in survivors. However, it is clear that little or no data on outcomes beyond 5 years posttransplant have been obtained, and clinicians working with this population remain concerned regarding the cognitive functions of these survivors. Research focused on these domains should attempt to better understand the prevalence of the problem using child self-report and direct measurements of function, standardize measurement methods, and tools across trials, obtain longer term evaluations and begin to consider interventional trials.

Copyright © 2012 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

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