Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-Versus-Host Disease Survivorship After Hematopoietic Cell Transplantation: Part IV. Patient Important Outcomes

Seth J Rotz; Lori Wiener; K Scott Baker; Sung Won Choi; Rachel Phelan; Geoffrey D E Cuvelier; Christine Duncan; Kirsten M Williams; Muna Qayed

doi:10.1016/j.jtct.2024.12.019

Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-Versus-Host Disease Survivorship After Hematopoietic Cell Transplantation: Part IV. Patient Important Outcomes

Transplant Cell Ther. 2024 Dec 27:S2666-6367(24)00838-8. doi: 10.1016/j.jtct.2024.12.019. Online ahead of print.

Authors

Seth J Rotz¹, Lori Wiener², K Scott Baker³, Sung Won Choi⁴, Rachel Phelan⁵, Geoffrey D E Cuvelier⁶, Christine Duncan⁷, Kirsten M Williams⁸, Muna Qayed⁸

Affiliations

¹ Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Cleveland Clinic, Cleveland, OH, USA. Electronic address: rotzs@ccf.org.
² Pediatric Oncology Branch, National Cancer Institute, Bethesda, MD, USA.
³ Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
⁴ Pediatric Hematology/Oncology, University of Michigan, Ann Arbor, MI, USA.
⁵ Division of Pediatric Hematology/Oncology/Blood and Marrow Transplant, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI.
⁶ Department of Pediatric Oncology and Transplantation, Alberta Children's Hospital, University of Calgary, Canada.
⁷ Dana Farber Cancer Institute, Boston, MA, US.
⁸ Aflac Blood and Cancer Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA, USA.

PMID: 39733839
DOI: 10.1016/j.jtct.2024.12.019

Abstract

Chronic graft versus host disease (cGVHD), occurs in approximately one in five pediatric allogeneic HCT patients and is a leading cause of late morbidity and mortality. Late effects of HCT may lead to long-term chronic health conditions and shortened life expectancy. In addition to direct physiological challenges from cGVHD and other late-effects, numerous patient-important outcomes impact the quality of life (QOL) of patients and their families. The Research and Education towards Solutions for Late Effects to Innovate, Excel, and Nurture (RESILIENT) after GVHD Consensus Conference was convened to better understand the overlap of cGVHD and late effects in pediatric HCT survivors. Working Committee IV: Patient Important Outcomes identified four key areas for focus: 1) What are the key mental health and quality of life (QOL) concerns of survivors of pediatric cGVHD? 2) What is the impact of cGVHD on cognitive performance and social development? 3) What multi-level social determinants of health impact cGVHD survivors, families, and communities? 4) What is the role of racial, ethnic, and socioeconomic factors on the development of cGVHD and the risk for adverse outcomes related to survivorship? For each focus area the working committee reviewed the current state of the field, develop recommendations for clinical practice, and highlighted areas to prioritize for future research. Eleven total recommendations were adapted and approved. Substantial overlap exists between the role of cGVHD and late effects on the quality of life and mental health of childhood HCT survivors. Recommendations based on available data and consensus opinion may be helpful to improve outcomes for these patients, but several gaps remain which need further study.

Keywords: Patient reported outcomes; allo; cGVHD; late-effects; patient important outcomes; psychosocial; survivorship.