Current Knowledge and Priorities for Future Research in Late Effects 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 Hematopoietic Cell Transplantation
Year: 2017
Type: article
Abstract: Fanconi anemia (FA), dyskeratosis congenita (DC), and Diamond Blackfan anemia (DBA) are 3 of the most common inherited bone marrow failure syndromes (IBMFS), in which the hematologic manifestations can be cured with hematopoietic cell transplantation (HCT). Later in life, these patients face a variety of medical conditions, which may be a manifestation of underlying disease or due to pre-HCT therapy, the HCT, or a combination of all these elements. Very limited long-term follow-up data exist in these populations, with FA the only IBMFS that has specific published data. During the international consensus conference sponsored by the Pediatric Blood and Marrow Transplant Consortium entitled "Late Effects Screening and Recommendations following Allogeneic Hematopoietic Cell Transplant (HCT) for Immune Deficiency and Nonmalignant Hematologic Disease" held in Minneapolis, Minnesota in May of 2016, a half-day session was focused specifically on the unmet needs for these patients with IBMFS. A multidisciplinary group of experts discussed what is currently known, outlined an agenda for future research, and laid out long-term follow-up guidelines based on a combination of evidence in the literature as well as expert opinion. This article addresses the state of science in that area as well as consensus regarding the agenda for future research, with specific screening guidelines to follow in the next article from this group.
Institutions: University of Southern California, Children's Hospital Los Angeles, Cincinnati Children's Hospital Medical Center, University of Cincinnati, University of Cincinnati Medical Center, Feinstein Institute for Medical Research, Hofstra University, Cohen Children's Medical Center, Schneider Children's Hospital, National Cancer Institute, Division of Cancer Epidemiology and Genetics, Leiden University Medical Center, Willem-Alexander Kinderziekenhuis, University of Minnesota, Memorial Sloan Kettering Cancer Center, NewYork–Presbyterian Hospital, Weill Cornell Medicine, Hôpital Robert-Debré, Université Paris Diderot, Hospital de Clínicas Universidade Federal do Paraná, Universidade Federal do Paraná, Imperial College Healthcare NHS Trust, St Mary's Hospital, Imperial College London, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Fred Hutch Cancer Center
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health 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, G5630405805, G6425276207, Blood and Marrow Transplant Clinical Trials Network, G7132514506, G7863853056, Cancer Center Support Grant
OpenAlex ID: W2577516487