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Biol Blood Marrow Transplant. 2017 Sep;23(9):1422-1428. doi: 10.1016/j.bbmt.2017.05.022. Epub 2017 May 19.

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.

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Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, University of Southern California, Los Angeles, California. Electronic address:
Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland.
Division of Hematology/Oncology and Stem Cell Transplantation, Hofstra Northwell School of Medicine, Feinstein Institute for Medical Research, Cohen Children's Medical Center, New Hyde Park, New York.
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.
Willem-Alexander Children's Hospital, SCT Unit, Leiden University Medical Center, Leiden, The Netherlands.
Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota.
Hospital de Clinicas, Federal University of Parana, Curitiba, Brazil.
Service d'hémato-immunologie,Université Paris 7, Hôpital Robert-Debré, Paris, France.
Section of Paediatrics, Department of Paediatric Haematology, St Mary's Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom.
Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust and Northern Institute of Cancer Research, Newcastle University, Newcastle upon Tyne, United Kingdom.
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.
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.


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.


Diamond Blackfan anemia; Dyskeratosis congenita; Fanconi anemia; Inherited bone marrow failure syndromes; Late effects; Pediatric allogeneic hematopoietic cell transplant

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