Survival outcomes of children with relapsed or refractory myeloid leukemia associated with Down syndrome

  • Nikhil Raghuram
    1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
  • Daisuke Hasegawa
    2Department of Pediatrics, St. Luke's International Hospital, 9-1, Akashi-cho, Chuo-ku, Tokyo, 104-8560, Japan
  • Kentaro Nakashima
    3Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan
  • Syaza Rahman
    4Division of Paediatric Haematology-Oncology and BM Transplantation, University of Malaya Medical Centre, Kuala Lumpur, Malaysia
  • Evangelia Antoniou
    5Department of Pediatric Hematology and Oncology, University Children's Hospital Essen, University of Duisburg-Essen, Essen, Germany
  • Torjus Skajaa
    6Department of Haematology, Great Ormond Street Hospital for Children, London, United Kingdom
  • Pietro Merli
    7Department of Pediatric Hematology/Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale Pediatrico Bambino Gesù, Sapienza University of Rome, Rome, Italy
  • Anupam Verma
    8Division of Hematology/Oncology, Department of Pediatrics, University of Utah and Primary Children's Hospital, Salt Lake City, UT
  • Karen R. Rabin
    10Pediatric Hematology-Oncology, Texas Children's Cancer Center, Baylor College of Medicine, Houston, TX
  • Catherine Aftandilian
    11Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA
  • Rishi S. Kotecha
    12Department of Clinical Haematology, Oncology, Blood and Marrow Transplantation, Perth Children's Hospital, Perth, WA, Australia
  • Daniel Cheuk
    15Department of Paediatrics and Adolescent Medicine, the University of Hong Kong and Hong Kong Children's Hospital, Hong Kong, China
  • Kirsi Jahnukainen
    16Children's Hospital, University of Helsinki and Helsinki University Hospital, Helsinki, Finland
  • Alexandra Kolenova
    17Comenius University Children's Hospital Limbova 1, Bratislava, Slovakia
  • Walentyna Balwierz
    18Department of Pediatric Oncology and Hematology, Institute of Pediatrics, Jagiellonian University Medical College, Krakow, Poland
  • Alice Norton
    19Department of Haematology, Birmingham Women's and Children's NHS Foundation Trust, Birmingham, United Kingdom
  • Maureen O’Brien
    20Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH
  • Sonia Cellot
    21Division of Hematology, Department of Pediatrics, Ste-Justine Hospital, Montréal, Université de Montréal, Montréal, QC, Canada
  • Ashley Chopek
    22Pediatric Blood and Marrow Transplant Program, Cancer Care Manitoba, University of Manitoba, Winnipeg, MB, Canada
  • Nira Arad-Cohen
    23Pediatric Hematology-Oncology Department, Ruth Rappaport Children's Hospital, Rambam Health Care Campus, Haifa, Israel
  • Bianca Goemans
    24Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands
  • Marta Rojas-Vasquez
    25Department of Pediatric Hematology-Oncology, Stollery Children's Hospital, University of Alberta, Edmonton, Canada
  • Hany Ariffin
    4Division of Paediatric Haematology-Oncology and BM Transplantation, University of Malaya Medical Centre, Kuala Lumpur, Malaysia
  • Jack Bartram
    6Department of Haematology, Great Ormond Street Hospital for Children, London, United Kingdom
  • E. Anders Kolb
    26Nemours Center for Cancer and Blood Disorders/Alfred I. DuPont Hospital for Children, Wilmington, DE
  • Franco Locatelli
    7Department of Pediatric Hematology/Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale Pediatrico Bambino Gesù, Sapienza University of Rome, Rome, Italy
  • Jan-Henning Klusmann
    27Goethe University Frankfurt, Frankfurt, Germany
  • Henrik Hasle
    28Department of Paediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark
  • Bryan McGuire
    1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
  • Afia Hasnain
    29Division of Genome Diagnostics, Department of Pediatric Laboratory Medicine, The Hospital for Sick Children, Toronto, ON, Canada
  • Lillian Sung
    1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
  • Johann Hitzler
    1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada

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<jats:title>Abstract</jats:title> <jats:p>Children with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population. We conducted a retrospective analysis of international study groups and pediatric oncology centers and identified 62 patients who received treatment with curative intent for r/r ML-DS between year 2000 to 2021. Median time from diagnosis to relapse was 6.8 (range, 1.1-45.5) months. Three-year event-free survival (EFS) and overall survival (OS) were 20.9 ± 5.3% and 22.1 ± 5.4%, respectively. Survival was associated with receipt of hematopoietic stem cell transplantation (HSCT) (hazard ratio [HR], 0.28), duration of first complete remission (CR1) (HR, 0.31 for &gt; 12 months) and attainment of remission after relapse (HR, 4.03). Patients who achieved complete remission (CR) before HSCT, had an improved OS and EFS of 56.0 ± 11.8% and 50.5 ± 11.9%, respectively compared to those who underwent HSCT without CR (3-year OS and EFS of 10.0 ± 9.5%). Treatment failure after HSCT was predominantly because of disease recurrence (52%) followed by treatment-related mortality (10%). The prognosis of r/r ML-DS remains dismal even in the current treatment period and serve as a reference point for current prognostication and future interventional studies. Clinical trials aimed at improving the survival of patients with r/r ML-DS are needed.</jats:p>

収録刊行物

  • Blood Advances

    Blood Advances 7 (21), 6532-6539, 2023-10-27

    American Society of Hematology

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