title: 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.
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.
authors.affiliation: 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.
authors.forename: Andrew C
authors.initials: AC
authors.lastname: Dietz
authors.affiliation: 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.
authors.forename: Parinda A
authors.initials: PA
authors.lastname: Mehta
authors.affiliation: 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.
authors.forename: Adrianna
authors.initials: A
authors.lastname: Vlachos
authors.affiliation: Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland.
authors.forename: Sharon A
authors.initials: SA
authors.lastname: Savage
authors.affiliation: Willem-Alexander Children's Hospital, SCT Unit, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Dorine
authors.initials: D
authors.lastname: Bresters
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota.
authors.forename: Jakub
authors.initials: J
authors.lastname: Tolar
authors.affiliation: 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.
authors.forename: Farid
authors.initials: F
authors.lastname: Boulad
authors.affiliation: Université Paris 7, Hôpital Robert-Debré, Service d'hémato-immunologie, Paris, France.
authors.forename: Jean Hugues
authors.initials: JH
authors.lastname: Dalle
authors.affiliation: Hospital de Clinicas, Federal University of Parana, Curitiba, Brazil.
authors.forename: Carmem
authors.initials: C
authors.lastname: Bonfim
authors.affiliation: Section of Paediatrics, Imperial College, London, United Kingdom; Department of Paediatric Haematology, St Mary's Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom.
authors.forename: Josu
authors.initials: J
authors.lastname: de la Fuente
authors.affiliation: Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts.
authors.forename: Christine N
authors.initials: CN
authors.lastname: Duncan
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
authors.forename: K Scott
authors.initials: KS
authors.lastname: Baker
authors.affiliation: Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, University of Southern California, Los Angeles, California.
authors.forename: Michael A
authors.initials: MA
authors.lastname: Pulsipher
authors.affiliation: 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.
authors.forename: Jeffrey M
authors.initials: JM
authors.lastname: Lipton
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota.
authors.forename: John E
authors.initials: JE
authors.lastname: Wagner
authors.affiliation: Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland.
authors.forename: Blanche P
authors.initials: BP
authors.lastname: Alter
mesh_terms: D000741:Anemia, Aplastic / Q000150:complications / Q000628:therapy*; D029503:Anemia, Diamond-Blackfan / Q000150:complications / Q000628:therapy; D035843:Biomedical Research / Q000379:methods / Q000639:trends; D001855:Bone Marrow Diseases / Q000150:complications / Q000628:therapy*; D000080983:Bone Marrow Failure Disorders; D002648:Child; D019871:Dyskeratosis Congenita / Q000150:complications / Q000628:therapy; D005199:Fanconi Anemia / Q000150:complications / Q000628:therapy; D005544:Forecasting; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications / Q000628:therapy*; D006801:Humans; D000069451:Long Term Adverse Effects
keywords: Diamond Blackfan anemia; Dyskeratosis congenita; Fanconi anemia; Inherited bone marrow failure syndromes; Late effects; Pediatric allogeneic hematopoietic cell transplantation