Late Effects Screening Guidelines after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects After Pediatric HCT
Year: 2017
Type: article
Abstract: Patients with inherited bone marrow failure syndromes (IBMFS), such as Fanconi anemia (FA), dyskeratosis congenita (DC), or Diamond Blackfan anemia (DBA), can have hematologic manifestations cured through hematopoietic cell transplantation (HCT). Subsequent late effects seen in these patients arise from a combination of the underlying disease, the pre-HCT therapy, and the HCT process. During the international consensus conference sponsored by the Pediatric Blood and Marrow Transplant Consortium on late effects screening and recommendations following allogeneic hematopoietic cell transplantation for immune deficiency and nonmalignant hematologic diseases held in Minneapolis, Minnesota in May 2016, a half-day session was focused specifically on the unmet needs for these patients with IBMFS. This multidisciplinary group of experts in rare diseases and transplantation late effects has already published on the state of the science in this area, along with discussion of an agenda for future research. This companion article outlines consensus disease-specific long-term follow-up screening guidelines for patients with IMBFS.
Authors: Andrew C. Dietz, Sharon A. Savage, Adrianna Vlachos, Parinda A. Mehta, Dorine Bresters, Jakub Tolar, Carmem Bonfim, Jean Hugues Dalle, Josu de la Fuente, Roderick Skinner, Farid Boulad, Christine N. Duncan, K. Scott Baker, Michael A. Pulsipher, Jeffrey M. Lipton, John E. Wagner, Blanche P. Alter
Institutions: University of Southern California, Children's Hospital Los Angeles, National Cancer Institute, Division of Cancer Epidemiology and Genetics, Feinstein Institute for Medical Research, Hofstra University, Cohen Children's Medical Center, Schneider Children's Hospital, Cincinnati Children's Hospital Medical Center, University of Cincinnati, University of Cincinnati Medical Center, Leiden University Medical Center, Willem-Alexander Kinderziekenhuis, University of Minnesota, Hospital de Clínicas Universidade Federal do Paraná, Universidade Federal do Paraná, Hôpital Robert-Debré, Université Paris Diderot, Imperial College Healthcare NHS Trust, St Mary's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Great North Children's Hospital, Newcastle University, Memorial Sloan Kettering Cancer Center, NewYork–Presbyterian Hospital, Weill Cornell Medicine, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Fred Hutch Cancer Center
Language: English
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
Awards: Blood and Marrow Transplant Clinical Trials Network, Late Effects after Pediatric HSCT: State of the Science, Future Directions, Unrelated Donor BMT vs. Immune Suppression for Newly Diagnosed Severe Aplastic Anemia in Children and Young Adults: BMT CTN Core Center Renewal for the Pediatric Blood and Marrow Transplant Consortium, BONE MARROW TRANSPLANT (BMT) SURVIVORS STUDY, A Vital tool for the Study of DBA: The Diamond Blackfan Anemia Registry, Blood and Marrow Transplant Clinical Trials Network, G7132514506
OpenAlex ID: W2616400973