ArticleBlood advances2020
Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases.
Erratum issuedOpen access · goldAbstract read
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Article in Blood advances, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 24 papers, 2 of them syntheses that pooled it.
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24citing papers in PubMed, 2 pooled it
1.8field-weighted citation impact, top 13% of its field
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Who cites it
24 citing papers in PubMed, 2 syntheses or guidelines pooled it, 30 citations in OpenAlex.
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- Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres.British journal of haematology · 2021Trial
- Immunosenescence and cancer predisposition in pediatric transplant recipients: An emerging paradigm.World journal of transplantation · 2026Review
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- Tracking Cytopenias inmedRxiv : the preprint server for health sciences · 2025Article
- Precision transplant and cell therapies for non-malignant disorders-The path forward.British journal of haematology · 2025Review
- Up-front alternative donor HCT in severe aplastic anemia: gaps and opportunities to translate evidence into practice.Blood advances · 2025Review
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- Challenges and Opportunities in the Care of Hematopoietic Cell Transplant Survivors in the Modern Era.Advances in experimental medicine and biology · 2025Review
- Late effects of hemopoietic stem cell transplant for sickle cell disease: monitoring and management.Expert review of hematology · 2024Review
- Update on Recommendations for Cancer Screening and Surveillance in Children with Genomic Instability Disorders.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024Review
- Late effects after allogeneic haematopoietic cell transplantation in children and adolescents with non-malignant disorders: a retrospective cohort study.The Lancet. Child & adolescent health · 2024Article
- Severity of Chronic Graft-versus-Host Disease and Late Effects Following Allogeneic Hematopoietic Cell Transplantation for Adults with Hematologic Malignancy.Transplantation and cellular therapy · 2024Article
- The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults.Frontiers in immunology · 2024Review
- Managing Survivorship after Hematopoietic Cell Transplantation.Current hematologic malignancy reports · 2023Review
- Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023Article
- Recent advances in hematopoietic cell transplantation for inherited bone marrow failure syndromes.International journal of hematology · 2022Review
- Burden of Morbidity after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A BMT Survivor Study Report.Transplantation and cellular therapy · 2022Article
- TCRαβ/CD19 depleted HSCT from an HLA-haploidentical relative to treat children with different nonmalignant disorders.Blood advances · 2022Article
Corrections and comments
- Erratum issued
Authors and funding
57 authors at 20 institutions in 8 countries.
Justine M KahnDivision of Pediatric Hematology, Oncology and Stem Cell Transplantation, Department of Pediatrics, Columbia University, New York, NY.
Ruta BrazauskasCenter for International Blood and Marrow Transplant Research, Department of Medicine, and.
Heather R TeccaCenter for International Blood and Marrow Transplant Research, Department of Medicine, and.
Stephanie Bo-SubaitCenter for International Blood and Marrow Transplant Research, Department of Medicine, and.
David BuchbinderDivision of Pediatric Hematology, Children's Hospital of Orange County, Orange, CA.
Minoo BattiwalaHematology Branch, Sarah Cannon Bone and Marrow Transplant Program, Nashville, TN.
Mary E D FlowersClinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA.
Bipin N SavaniDivision of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN.
Rachel PhelanCenter for International Blood and Marrow Transplant Research, Department of Medicine, and.
Larisa BroglieDivision of Pediatric Hematology, Oncology and Stem Cell Transplantation, Department of Pediatrics, Columbia University, New York, NY.
Allistair A AbrahamDivision of Blood and Marrow Transplantation, Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC.
Amy K KeatingChildren's Hospital Colorado and University of Colorado, Aurora, CO.
Andrew DalyTom Baker Cancer Center, Calgary, AB, Canada.
Baldeep WirkDivision of Bone Marrow Transplant, Seattle Cancer Alliance, Seattle, WA.
Biju GeorgeDepartment of Hematology, Christian Medical College, Vellore, India.
Blanche P AlterClinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Bethesda, MD.
Celalettin UstunDivision of Hematology/Oncology/Cell Therapy, Rush University, Chicago, IL.
Cesar O FreytesTexas Transplant Institute, San Antonio, TX.
Amer M BeitinjanehSylvester Comprehensive Cancer Center, University of Miami Health System, Miami, FL.
Christine DuncanDepartment of Pediatric Oncology, Boston Children's Hospital and Dana-Farber Cancer Institute, Boston, MA.
Edward CopelanLevine Cancer Institute, Atrium Health, Carolinas HealthCare System, Charlotte, NC.
Gerhard C HildebrandtMarkey Cancer Center, University of Kentucky, Lexington, KY.
Hemant S MurthyDivision of Hematology/Oncology, College of Medicine, University of Florida, Gainesville, FL.
Hillard M LazarusDepartment of Medicine, University Hospitals Case Medical Center and Seidman Cancer Center, Case Comprehensive Cancer Center, Case Western Reserve University, Cleveland, OH.
Jeffery J AulettaBlood and Marrow Transplant Program and Host Defense Program, Division of Hematology/Oncology/Bone Marrow Transplant and Infectious Diseases, Nationwide Children's Hospital, Columbus, OH.
Kasiani C MyersDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH.
Kirsten M WilliamsChildren's Research Institute, Children's National Health Systems, Washington, DC.
Kristin M PageDivision of Pediatric Blood and Marrow Transplantation, Duke University Medical Center, Durham, NC.
Lynda M VroomanDepartment of Pediatric Oncology, Boston Children's Hospital and Dana-Farber Cancer Institute, Boston, MA.
Maxim NorkinDivision of Hematology/Oncology, College of Medicine, University of Florida, Gainesville, FL.
Michael ByrneDivision of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN.
Miguel Angel DiazDepartment of Hematology/Oncology, Hospital Infantil Universitario Nino Jesus, Madrid, Spain.
Naynesh KamaniCenter for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC.
Neel S BhattSt Jude Children's Research Hospital, Memphis, TN.
Andrew RezvaniStanford Health Care, Stanford, CA.
Nosha FarhadfarDivision of Hematology/Oncology, College of Medicine, University of Florida, Gainesville, FL.
Parinda A MehtaDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH.
Peiman HemattiDivision of Hematology/Oncology/Bone Marrow Transplantation, Department of Medicine, University of Wisconsin-Madison, Madison, WI.
Peter J ShawThe Children's Hospital at Westmead, Westmead, NSW, Australia.
Rammurti T KambleDivision of Hematology and Oncology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX.
Raquel SchearsDivision of Hematology/Bone Marrow Transplant, Mayo Clinic, Rochester, MN.
Richard F OlssonDepartment of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden.
Robert J HayashiDivision of Pediatric Hematology/Oncology, Department of Pediatrics, School of Medicine, Washington University in St. Louis, St. Louis, MO.
Robert Peter GaleHematology Research Center, Division of Experimental Medicine, Department of Medicine, Imperial College London, London, United Kingdom.
Samantha J MayoLawrence S. Bloomberg Faculty of Nursing, University of Toronto, Toronto, ON, Canada.
Saurabh ChhabraDivision of Pediatric Hematology/Oncology/BMT, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI.
Seth J RotzDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH.
Sherif M BadawyDivision of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.
Siddhartha GangulyDivision of Hematological Malignancy and Cellular Therapeutics, University of Kansas Health System, Kansas City, KS.
Steven PavleticExperimental Transplantation and Immunology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.
Taiga NishihoriDepartment of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL.
Tim PrestidgeBlood and Cancer Center, Starship Children's Hospital, Auckland, New Zealand.
Vaibhav AgrawalSimon Cancer Center, Indiana University, Indianapolis, IN; and.
William J HoganDivision of Hematology and Oncology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX.
Yoshihiro InamotoDivision of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan.
Bronwen E ShawCenter for International Blood and Marrow Transplant Research, Department of Medicine, and.
Prakash SatwaniDivision of Pediatric Hematology, Oncology and Stem Cell Transplantation, Department of Pediatrics, Columbia University, New York, NY.
Children's National · USCincinnati Children's Hospital Medical Center · USColumbia University · USMedical College of Wisconsin · USUniversity of Florida · USBaylor College of Medicine · USBoston Children's Hospital · USNational Institutes of Health · USVanderbilt University Medical Center · USCarolinas Healthcare System · USChildren's Hospital at Westmead · AUChildren's Hospital Colorado · USChildren's Hospital of Orange County · USChristian Medical College, Vellore · INCHRISTUS Transplant Institute · USDuke Medical Center · USFred Hutch Cancer Center · USHospital Infantil Universitario Niño Jesús · ESImperial College London · GBIndiana University Health · US
Funding
Data Resource for Analyzing Blood &Marrow TransplantsU24CA076518 · NCI · MEDICAL COLLEGE OF WISCONSIN · PI Amy M Moskop, Bronwen Shaw · 1998 to 2026
$105.2MBlood and Marrow Transplant Clinical Trials Network DCC- The Medical College of Wisconsin, Inc.U24HL138660 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI Steven M. DeVine, Mehdi Hussain Hamadani · 2017 to 2026
$71.4MTherapeutic Potential of Adaptive NK Cells in Cancer and TransplantationP01CA111412 · NCI · UNIVERSITY OF MINNESOTA TWIN CITIES · PI MILLER, JEFFREY S. · 2005 to 2025
$38.2MHematopopietic Stem Cell TransplantationU01AI069197 · NIAID · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Effie W Petersdorf · 2005 to 2026
$18.9MRandomized study of low versus moderate dose busulfan in transplant for severe combined immunodeficiencyU01AI126612 · NIAID · CHILDREN'S HOSPITAL OF LOS ANGELES · PI KEAN, LESLIE S, PULSIPHER, MICHAEL A · 2017 to 2023
$8.5MInstitutional Career Development CoreKL2TR002547 · NCATS · CASE WESTERN RESERVE UNIVERSITY · PI DWEIK, RAED A. · 2018 to 2022
$7.7MImmuno and Epigenetics of Hematopoietic Cell TransplantationR01CA218285 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Effie W Petersdorf · 2017 to 2026
$5.6MThe role of TCOF1 in germline predisposition to and in leukemic transformation of chronic myelomonocytic leukemiaR01CA152108 · NCI · UNIVERSITY OF WISCONSIN-MADISON · PI Jing Zhang · 2011 to 2026
$5.4MIntegrated Exchange and Storage of Current- and Future-Generation Immunogenomic DataR01AI128775 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI JILL Allison HOLLENBACH, STEVEN JOHN MACK · 2017 to 2026
$4.5MNorthwestern University Patient-centered Intervention and Engagement TrainingK12HS023011 · AHRQ · NORTHWESTERN UNIVERSITY AT CHICAGO · PI CELLA, DAVID · 2014 to 2018
$3.7MIdentifying Predictors of Poor Health-Related Quality-of-life among Pediatric Hematopoietic Stem Cell DonorsR01HL131731 · NHLBI · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI PULSIPHER, MICHAEL A, SWITZER, GALEN E. · 2018 to 2022
$3.6MIntegrating health informatics in a scalable stepped care self-management program for survivors after hematopoietic cell transplantationR01CA215134 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI BAKER, KEVIN S, MAJHAIL, NAVNEET S. · 2017 to 2022
$3.6MAHRQ HHS K12 HS023011NCATS NIH HHS KL2 TR002547NCI NIH HHS P01 CA111412NCI NIH HHS R01 CA152108NCI NIH HHS R01 CA215134NCI NIH HHS R01 CA218285NCI NIH HHS R01 CA231141NCI NIH HHS U24 CA076518NHLBI NIH HHS K23 HL150232NHLBI NIH HHS R01 HL126589NHLBI NIH HHS R01 HL129472NHLBI NIH HHS R01 HL130388NHLBI NIH HHS R01 HL131731NHLBI NIH HHS R21 HL140314NHLBI NIH HHS U01 HL128568NHLBI NIH HHS U24 HL138660NIAID NIH HHS R01 AI128775NIAID NIH HHS U01 AI069197NIAID NIH HHS U01 AI126612
Abstract
We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range, <1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was <1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.
Indexed as
Anemia, AplasticHematopoietic Stem Cell TransplantationLeukemiaNeoplasmsAdolescentChildHumansTransplantation, Homologous
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