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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.

Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, University of Southern California, Los Angeles, California. Electronic address: adietz@chla.usc.edu. Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio. Hofstra Northwell School of Medicine, Feinstein Institute for Medical Research, Cohen Children's Medical Center, Division of Hematology/Oncology and Stem Cell Transplantation, New Hyde Park, New York. Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland. Willem-Alexander Children's Hospital, SCT Unit, Leiden University Medical Center, Leiden, The Netherlands. Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota. Bone Marrow Transplant Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, Division of Pediatric Hematology/Oncology, New York Presbyterian Hospital, Weill Cornell Medical College, New York, New York. Université Paris 7, Hôpital Robert-Debré, Service d'hémato-immunologie, Paris, France. Hospital de Clinicas, Federal University of Parana, Curitiba, Brazil. Section of Paediatrics, Imperial College, London, United Kingdom; Department of Paediatric Haematology, St Mary's Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom. Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington. Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, University of Southern California, Los Angeles, California.

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2017;(5):726-735
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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.

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