Rotz Seth J, Wiener Lori, Baker K Scott, Choi Sung Won, Phelan Rachel, Cuvelier Geoffrey D E, Duncan Christine, Williams Kirsten M, Qayed Muna
Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Cleveland Clinic, Cleveland, Ohio.
Pediatric Oncology Branch, National Cancer Institute, Bethesda, Maryland.
Transplant Cell Ther. 2025 Apr;31(4):224.e1-224.e13. doi: 10.1016/j.jtct.2024.12.019. Epub 2024 Dec 27.
Chronic graft-versus-host disease (cGVHD) occurs in approximately 1 in 5 pediatric allogeneic HCT patients and is a leading cause of late morbidity and mortality. Late effects of hematopoietic cell transplantation (HCT) may lead to long-term chronic health conditions and shortened life expectancy. In addition to direct physiologic 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 4 key areas for focus: (1) What are the key mental health and QOL concerns of survivors of pediatric cGVHD? (2) What is the impact of cGVHD on cognitive performance and social development? (3) What multilevel 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, developed recommendations for clinical practice, and highlighted areas to prioritize for future research. Eleven recommendations were adapted and approved. Substantial overlap exists between the role of cGVHD and late effects on the QOL and mental health of childhood HCT survivors. Recommendations based on available data and consensus opinion may be helpful to improve outcomes for these patients. However, several gaps remain that need further study.
慢性移植物抗宿主病（cGVHD）发生在约五分之一的儿童异基因造血细胞移植（HCT）患者中，是晚期发病和死亡的主要原因。造血细胞移植（HCT）的晚期效应可能导致长期慢性健康问题和预期寿命缩短。除了cGVHD和其他晚期效应带来的直接生理挑战外，众多对患者重要的结果会影响患者及其家庭的生活质量（QOL）。召开GVHD后效应解决方案研究与教育以创新、卓越和培育（RESILIENT）共识会议，以更好地了解cGVHD与儿童HCT幸存者晚期效应的重叠情况。第四工作委员会：患者重要结果确定了4个重点关注领域：（1）儿童cGVHD幸存者关键的心理健康和生活质量问题有哪些？（2）cGVHD对认知表现和社会发展有何影响？（3）哪些多层次的健康社会决定因素会影响cGVHD幸存者、家庭和社区？（4）种族、民族和社会经济因素在cGVHD发生发展以及与生存相关的不良后果风险中起什么作用？对于每个重点领域，工作委员会审查了该领域的现状，制定了临床实践建议，并突出了未来研究需优先考虑的领域。11项建议得到了调整和批准。cGVHD的作用与晚期效应在儿童HCT幸存者的生活质量和心理健康方面存在大量重叠。基于现有数据和共识意见的建议可能有助于改善这些患者的预后。然而，仍有几个差距需要进一步研究。
