title: The Second Pediatric Blood and Marrow Transplant Consortium International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Defining the Unique Late Effects of Children Undergoing Hematopoietic Cell Transplantation for Immune Deficiencies, Inherited Marrow Failure Disorders, and Hemoglobinopathies.
abstract: An international consensus conference sponsored by the Pediatric Blood and Marrow Transplant consortium entitled "Late Effects Screening and Recommendations Following Allogeneic Hematopoietic Cell Transplant for Immune Deficiency and Nonmalignant Hematologic Disease" was held in Minneapolis, Minnesota on May 10, 2016 and May 11, 2016. The purpose of the conference was to address the unmet need for greater understanding of and the screening for long-term complications in the growing population of survivors of transplantation for nonmalignant disorders. The conference focused on transplantation for hemoglobinopathy, immune deficiency, and inherited bone marrow syndromes. A multidisciplinary group of experts in the disease areas and transplantation late effects presented the current state of understanding of how the underlying disease, pretransplantation therapies, and transplantation-related factors uniquely interact to influence the development of late toxicities. Recommendations were put forth by the group for the late effects screening of survivors of transplantation for these nonmalignant disorders. The findings and recommendations that came from this conference will be presented in a series of 6 additional manuscripts in the upcoming months. In this manuscript, we explore the need for screening practices specific to the survivors of transplantation for nonmalignant diseases and the methodologic challenges associated with the study of these patients.
authors.affiliation: Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Los Angeles, California.
authors.forename: Andrew C
authors.initials: AC
authors.lastname: Dietz
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 Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland.
authors.forename: Blanche P
authors.initials: BP
authors.lastname: Alter
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: University of California San Francisco, Department of Pediatrics, Allergy, Immunology, and Blood and Marrow Transplant Division, San Francisco, California.
authors.forename: Morton J
authors.initials: MJ
authors.lastname: Cowan
authors.affiliation: Division of Immunology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts.
authors.forename: Luigi
authors.initials: L
authors.lastname: Notarangelo
authors.affiliation: Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland.
authors.forename: Philip S
authors.initials: PS
authors.lastname: Rosenberg
authors.affiliation: St. Louis Children's Hospital, Washington University, St. Louis, Missouri.
authors.forename: Shalini
authors.initials: S
authors.lastname: Shenoy
authors.affiliation: Great North Children's Hospital and Northern Institute of Cancer Research, Newcastle upon Tyne, United Kingdom.
authors.forename: Roderick
authors.initials: R
authors.lastname: Skinner
authors.affiliation: UCSF Benioff Children's Hospital Oakland, Oakland, California.
authors.forename: Mark C
authors.initials: MC
authors.lastname: Walters
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota.
authors.forename: John
authors.initials: J
authors.lastname: Wagner
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, Los Angeles, California. Electronic address: mpulsipher@chla.usc.edu.
authors.forename: Michael A
authors.initials: MA
authors.lastname: Pulsipher
mesh_terms: D001855:Bone Marrow Diseases / Q000235:genetics / Q000628:therapy*; D002648:Child; D006287:Health Planning Guidelines; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006453:Hemoglobinopathies / Q000628:therapy*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D000069451:Long Term Adverse Effects / Q000175:diagnosis* / Q000209:etiology*; D008403:Mass Screening / Q000379:methods; D017741:Survivors
keywords: Hemoglobinopathies; Immune deficiencies; Late effects; Marrow failure disorders; Pediatric allogeneic bone marrow transplantation