Survival outcomes of children with relapsed or refractory myeloid leukemia associated with Down syndrome
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- Nikhil Raghuram
- 1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
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- Daisuke Hasegawa
- 2Department of Pediatrics, St. Luke's International Hospital, 9-1, Akashi-cho, Chuo-ku, Tokyo, 104-8560, Japan
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- Kentaro Nakashima
- 3Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan
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- Syaza Rahman
- 4Division of Paediatric Haematology-Oncology and BM Transplantation, University of Malaya Medical Centre, Kuala Lumpur, Malaysia
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- Evangelia Antoniou
- 5Department of Pediatric Hematology and Oncology, University Children's Hospital Essen, University of Duisburg-Essen, Essen, Germany
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- Torjus Skajaa
- 6Department of Haematology, Great Ormond Street Hospital for Children, London, United Kingdom
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- 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
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- Anupam Verma
- 8Division of Hematology/Oncology, Department of Pediatrics, University of Utah and Primary Children's Hospital, Salt Lake City, UT
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- Karen R. Rabin
- 10Pediatric Hematology-Oncology, Texas Children's Cancer Center, Baylor College of Medicine, Houston, TX
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- Catherine Aftandilian
- 11Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA
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- Rishi S. Kotecha
- 12Department of Clinical Haematology, Oncology, Blood and Marrow Transplantation, Perth Children's Hospital, Perth, WA, Australia
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- Daniel Cheuk
- 15Department of Paediatrics and Adolescent Medicine, the University of Hong Kong and Hong Kong Children's Hospital, Hong Kong, China
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- Kirsi Jahnukainen
- 16Children's Hospital, University of Helsinki and Helsinki University Hospital, Helsinki, Finland
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- Alexandra Kolenova
- 17Comenius University Children's Hospital Limbova 1, Bratislava, Slovakia
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- Walentyna Balwierz
- 18Department of Pediatric Oncology and Hematology, Institute of Pediatrics, Jagiellonian University Medical College, Krakow, Poland
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- Alice Norton
- 19Department of Haematology, Birmingham Women's and Children's NHS Foundation Trust, Birmingham, United Kingdom
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- Maureen O’Brien
- 20Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH
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- Sonia Cellot
- 21Division of Hematology, Department of Pediatrics, Ste-Justine Hospital, Montréal, Université de Montréal, Montréal, QC, Canada
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- Ashley Chopek
- 22Pediatric Blood and Marrow Transplant Program, Cancer Care Manitoba, University of Manitoba, Winnipeg, MB, Canada
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- Nira Arad-Cohen
- 23Pediatric Hematology-Oncology Department, Ruth Rappaport Children's Hospital, Rambam Health Care Campus, Haifa, Israel
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- Bianca Goemans
- 24Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands
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- Marta Rojas-Vasquez
- 25Department of Pediatric Hematology-Oncology, Stollery Children's Hospital, University of Alberta, Edmonton, Canada
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- Hany Ariffin
- 4Division of Paediatric Haematology-Oncology and BM Transplantation, University of Malaya Medical Centre, Kuala Lumpur, Malaysia
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- Jack Bartram
- 6Department of Haematology, Great Ormond Street Hospital for Children, London, United Kingdom
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- E. Anders Kolb
- 26Nemours Center for Cancer and Blood Disorders/Alfred I. DuPont Hospital for Children, Wilmington, DE
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- 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
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- Jan-Henning Klusmann
- 27Goethe University Frankfurt, Frankfurt, Germany
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- Henrik Hasle
- 28Department of Paediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark
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- Bryan McGuire
- 1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
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- Afia Hasnain
- 29Division of Genome Diagnostics, Department of Pediatric Laboratory Medicine, The Hospital for Sick Children, Toronto, ON, Canada
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- Lillian Sung
- 1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
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- Johann Hitzler
- 1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
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説明
<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 > 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
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- Blood Advances
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Blood Advances 7 (21), 6532-6539, 2023-10-27
American Society of Hematology
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キーワード
- Risk
- Acute megakaryoblastic leukemia
- Clinical Trials and Observations
- Down Syndrome/complications
- Gata1
- Transient leukemia
- Medizin
- Hematopoietic Stem Cell Transplantation
- Trial
- Leukemia, Myeloid, Acute
- Recurrence
- Chemotherapy
- Humans
- Therapy
- Down Syndrome
- Child
- Mutations
- Gynaecology and paediatrics
- Reduction
- Retrospective Studies
詳細情報 詳細情報について
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- CRID
- 1360021393796666752
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- ISSN
- 24739537
- 24739529
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- HANDLE
- 10138/568800
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- PubMed
- 36735769
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- データソース種別
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- Crossref
- OpenAIRE