NCI, NHLBI First International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Etiology and Pathogenesis of Late Effects after HCT Performed in Childhood-Methodologic Challenges.

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume 17, Issue 10, p.1428-35 (2011)


2011, Adolescent, Center-Authored Paper, Child, Child, Preschool, Chronic Disease, Clinical Research Division, Consensus Development Conferences as Topic, Endocrine System Diseases, Female, Genomics Core Facility, Graft vs Host Disease, Heart Diseases, Hematologic Neoplasms, hematopoietic stem cell transplantation, HOMOLOGOUS, Humans, Infant, Lung Diseases, Male, Metabolic Diseases, Musculoskeletal Diseases, National Cancer Institute (U.S.), Neoplasms, Second Primary', Survival Rate, Time Factors,, Research Trials Office Core Facility - Biostatistics Service, September 2011, Shared Resources, United States


Hematopoietic cell transplantation (HCT) is now a curative option for certain categories of patients with hematologic malignancies and other life-threatening illnesses. Technical and supportive care has resulted in survival rates that exceed 70% for those who survive the first 2 years after HCT. However, long-term survivors carry a high burden of morbidity, including endocrinopathies, musculoskeletal disorders, cardiopulmonary compromise, and subsequent malignancies. Understanding the etiologic pathways that lead to specific post-HCT morbidities is critical to developing targeted prevention and intervention strategies. Understanding the molecular underpinnings associated with graft-versus-host disease (GVHD), organ toxicity, relapse, opportunistic infection, and other long-term complications now recognized as health care concerns will have significant impact on translational research aimed at developing novel targeted therapies for controlling chronic GVHD, facilitating tolerance and immune reconstitution, reducing risk of relapse and secondary malignancies, minimizing chronic metabolic disorders, and improving quality of life. However, several methodological challenges exist in achieving these goals; these issues are discussed in detail in this paper.