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Allogeneic hematopoietic stem cell transplantation in ERCC6L2 disease.

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Title: Allogeneic hematopoietic stem cell transplantation in ERCC6L2 disease.
Authors: Hakkarainen M; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland., Sicre de Fontbrune F; Hematology Transplant Unit, National French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Centre de Référence des Aplasies Médullaires Acquises et Constitutionnelles, Hôpital Saint-Louis, Paris, France., Kaaja I; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland., Douglas S; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland., Dalle JH; Pediatric Immunology and Hematology Department and Centre de Référence des Maladies Rares Aplasies Médullaires, Robert Debré Hospital, Groupe Hospitalier Universitaire Assistance Publique-Hôpitaux de Paris Nord Université de Paris Cité, Paris, France., Risitano AM; Hematology, Hospital 'San Giuseppe Moscati,' Avellino, Italy.; Severe Aplastic Anemia Working Party, European Bone Marrow Transplant, Leiden, The Netherlands., Kulasekararaj A; Severe Aplastic Anemia Working Party, European Bone Marrow Transplant, Leiden, The Netherlands.; Department of Haematological Medicine, King's College Hospital NHS Foundation Trust, London, United Kingdom., Alsultan A; Oncology Center, King Saud University Medical City, Riyadh, Saudi Arabia.; Department of Pediatrics, College of Medicine, King Saud University, Riyadh, Saudi Arabia., Cutler C; Division of Transplantation and Cellular Therapy, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA., Ho VT; Division of Transplantation and Cellular Therapy, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA., Hellström-Lindberg E; Center for Hematology and Regenerative Medicine, Department of Medicine, Karolinska Institutet, Stockholm, Sweden., Mielke S; Center for Hematology and Regenerative Medicine, Department of Medicine, Karolinska Institutet, Stockholm, Sweden., Myhre AE; Department of Haematology, Oslo University Hospital, Oslo, Norway., Rihani R; Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation, King Hussein Cancer Center, Amman, Jordan., Shanap MA; Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation, King Hussein Cancer Center, Amman, Jordan., Hashem H; Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation, King Hussein Cancer Center, Amman, Jordan., Shimamura A; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA., Rowe RG; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA., Auer F; Department of Pediatrics, Technical University of Munich, Munich, Germany., Beier F; Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, Rheinisch-Westfälische Technische Hochschule Aachen University, Aachen, Germany.; Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen, Germany., Desnica L; Division of Hematology, Department of Internal Medicine, University Hospital Centre Zagreb, Zagreb, Croatia., Hough R; Children and Young People's Cancer Service, University College London Hospitals NHS Foundation Trust, London, United Kingdom., Ali Jafri SR; Section of Stem Cell Transplant & Car T Cell Therapy, Department of Pediatrics Hematology & Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia., Ayas M; Section of Stem Cell Transplant & Car T Cell Therapy, Department of Pediatrics Hematology & Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia., Jardine L; Northern Centre for Cancer Care, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.; Biosciences Institute, Newcastle University, Newcastle upon Tyne, United Kingdom., Mellid EF; Complexo Hospitalario Universitario de Santiago de Compostela, A Coruña, Spain., Corrales I; Laboratori de Coagulopaties Congènites, Banc de Sang i Teixits, Barcelona, Spain.; Medicina Transfusional, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Barcelona, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Cardiovasculares, Instituto de Salud Carlos III, Madrid, Spain., Richardson D; Department of Haematology, University Hospital Southampton, Southampton, United Kingdom., Özbek NY; Department of Pediatric Hematology and Oncology, Ankara City Hospital, University of Health Sciences, Turkey University of Health Sciences, Ankara, Turkey., Zaniewska-Tekieli A; Department of Transplantation, University Children's Hospital in Krakow, Krakow, Poland., Gozdzik J; Department of Clinical Immunology and Transplantation, Medical College, Jagiellonian University, Transplantation Center, University Children's Hospital in Krakow, Krakow, Poland., Ryhänen S; Division of Hematology, Oncology, and Stem Cell Transplantation, Children's Hospital, and Pediatric Research Center, University of Helsinki and Helsinki University Hospital, Helsinki, Finland., Niinimäki R; Department of Pediatrics, Oulu University Hospital and PEDEGO Research Unit, University of Oulu, Oulu, Finland., Jahnukainen K; Division of Hematology, Oncology, and Stem Cell Transplantation, Children's Hospital, and Pediatric Research Center, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.; Department of Women's and Children's Health, Nordic Center for Fertility Preservation Research Lab Stockholm, Karolinska Institutet and University Hospital, Stockholm, Sweden., Salmenniemi U; Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland., Kilpivaara O; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland.; iCAN Digital Precision Cancer Medicine Flagship, University of Helsinki, Helsinki, Finland.; K. Albin Johansson Cancer Researcher Fellow, Foundation for the Finnish Cancer Institute, Helsinki, Finland., Wartiovaara-Kautto U; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland.; iCAN Digital Precision Cancer Medicine Flagship, University of Helsinki, Helsinki, Finland.
Source: Blood advances [Blood Adv] 2026 Apr 14; Vol. 10 (7), pp. 2525-2537.
Publication Type: Journal Article; Multicenter Study
Journal Info: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Allogeneic hematopoietic stem cell transplantation in ERCC6L2 disease.
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  Data: <searchLink fieldCode="AU" term="%22Hakkarainen+M%22">Hakkarainen M</searchLink>; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland.<br /><searchLink fieldCode="AU" term="%22Sicre+de+Fontbrune+F%22">Sicre de Fontbrune F</searchLink>; Hematology Transplant Unit, National French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Centre de Référence des Aplasies Médullaires Acquises et Constitutionnelles, Hôpital Saint-Louis, Paris, France.<br /><searchLink fieldCode="AU" term="%22Kaaja+I%22">Kaaja I</searchLink>; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland.<br /><searchLink fieldCode="AU" term="%22Douglas+S%22">Douglas S</searchLink>; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland.<br /><searchLink fieldCode="AU" term="%22Dalle+JH%22">Dalle JH</searchLink>; Pediatric Immunology and Hematology Department and Centre de Référence des Maladies Rares Aplasies Médullaires, Robert Debré Hospital, Groupe Hospitalier Universitaire Assistance Publique-Hôpitaux de Paris Nord Université de Paris Cité, Paris, France.<br /><searchLink fieldCode="AU" term="%22Risitano+AM%22">Risitano AM</searchLink>; Hematology, Hospital 'San Giuseppe Moscati,' Avellino, Italy.; Severe Aplastic Anemia Working Party, European Bone Marrow Transplant, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Kulasekararaj+A%22">Kulasekararaj A</searchLink>; Severe Aplastic Anemia Working Party, European Bone Marrow Transplant, Leiden, The Netherlands.; Department of Haematological Medicine, King's College Hospital NHS Foundation Trust, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Alsultan+A%22">Alsultan A</searchLink>; Oncology Center, King Saud University Medical City, Riyadh, Saudi Arabia.; Department of Pediatrics, College of Medicine, King Saud University, Riyadh, Saudi Arabia.<br /><searchLink fieldCode="AU" term="%22Cutler+C%22">Cutler C</searchLink>; Division of Transplantation and Cellular Therapy, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA.<br /><searchLink fieldCode="AU" term="%22Ho+VT%22">Ho VT</searchLink>; Division of Transplantation and Cellular Therapy, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA.<br /><searchLink fieldCode="AU" term="%22Hellström-Lindberg+E%22">Hellström-Lindberg E</searchLink>; Center for Hematology and Regenerative Medicine, Department of Medicine, Karolinska Institutet, Stockholm, Sweden.<br /><searchLink fieldCode="AU" term="%22Mielke+S%22">Mielke S</searchLink>; Center for Hematology and Regenerative Medicine, Department of Medicine, Karolinska Institutet, Stockholm, Sweden.<br /><searchLink fieldCode="AU" term="%22Myhre+AE%22">Myhre AE</searchLink>; Department of Haematology, Oslo University Hospital, Oslo, Norway.<br /><searchLink fieldCode="AU" term="%22Rihani+R%22">Rihani R</searchLink>; Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation, King Hussein Cancer Center, Amman, Jordan.<br /><searchLink fieldCode="AU" term="%22Shanap+MA%22">Shanap MA</searchLink>; Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation, King Hussein Cancer Center, Amman, Jordan.<br /><searchLink fieldCode="AU" term="%22Hashem+H%22">Hashem H</searchLink>; Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation, King Hussein Cancer Center, Amman, Jordan.<br /><searchLink fieldCode="AU" term="%22Shimamura+A%22">Shimamura A</searchLink>; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.<br /><searchLink fieldCode="AU" term="%22Rowe+RG%22">Rowe RG</searchLink>; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.<br /><searchLink fieldCode="AU" term="%22Auer+F%22">Auer F</searchLink>; Department of Pediatrics, Technical University of Munich, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Beier+F%22">Beier F</searchLink>; Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, Rheinisch-Westfälische Technische Hochschule Aachen University, Aachen, Germany.; Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen, Germany.<br /><searchLink fieldCode="AU" term="%22Desnica+L%22">Desnica L</searchLink>; Division of Hematology, Department of Internal Medicine, University Hospital Centre Zagreb, Zagreb, Croatia.<br /><searchLink fieldCode="AU" term="%22Hough+R%22">Hough R</searchLink>; Children and Young People's Cancer Service, University College London Hospitals NHS Foundation Trust, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Ali+Jafri+SR%22">Ali Jafri SR</searchLink>; Section of Stem Cell Transplant & Car T Cell Therapy, Department of Pediatrics Hematology & Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.<br /><searchLink fieldCode="AU" term="%22Ayas+M%22">Ayas M</searchLink>; Section of Stem Cell Transplant & Car T Cell Therapy, Department of Pediatrics Hematology & Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.<br /><searchLink fieldCode="AU" term="%22Jardine+L%22">Jardine L</searchLink>; Northern Centre for Cancer Care, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.; Biosciences Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Mellid+EF%22">Mellid EF</searchLink>; Complexo Hospitalario Universitario de Santiago de Compostela, A Coruña, Spain.<br /><searchLink fieldCode="AU" term="%22Corrales+I%22">Corrales I</searchLink>; Laboratori de Coagulopaties Congènites, Banc de Sang i Teixits, Barcelona, Spain.; Medicina Transfusional, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Barcelona, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Cardiovasculares, Instituto de Salud Carlos III, Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Richardson+D%22">Richardson D</searchLink>; Department of Haematology, University Hospital Southampton, Southampton, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Özbek+NY%22">Özbek NY</searchLink>; Department of Pediatric Hematology and Oncology, Ankara City Hospital, University of Health Sciences, Turkey University of Health Sciences, Ankara, Turkey.<br /><searchLink fieldCode="AU" term="%22Zaniewska-Tekieli+A%22">Zaniewska-Tekieli A</searchLink>; Department of Transplantation, University Children's Hospital in Krakow, Krakow, Poland.<br /><searchLink fieldCode="AU" term="%22Gozdzik+J%22">Gozdzik J</searchLink>; Department of Clinical Immunology and Transplantation, Medical College, Jagiellonian University, Transplantation Center, University Children's Hospital in Krakow, Krakow, Poland.<br /><searchLink fieldCode="AU" term="%22Ryhänen+S%22">Ryhänen S</searchLink>; Division of Hematology, Oncology, and Stem Cell Transplantation, Children's Hospital, and Pediatric Research Center, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.<br /><searchLink fieldCode="AU" term="%22Niinimäki+R%22">Niinimäki R</searchLink>; Department of Pediatrics, Oulu University Hospital and PEDEGO Research Unit, University of Oulu, Oulu, Finland.<br /><searchLink fieldCode="AU" term="%22Jahnukainen+K%22">Jahnukainen K</searchLink>; Division of Hematology, Oncology, and Stem Cell Transplantation, Children's Hospital, and Pediatric Research Center, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.; Department of Women's and Children's Health, Nordic Center for Fertility Preservation Research Lab Stockholm, Karolinska Institutet and University Hospital, Stockholm, Sweden.<br /><searchLink fieldCode="AU" term="%22Salmenniemi+U%22">Salmenniemi U</searchLink>; Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland.<br /><searchLink fieldCode="AU" term="%22Kilpivaara+O%22">Kilpivaara O</searchLink>; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Medical and Clinical Genetics/Medicum, Faculty of Medicine, University of Helsinki, Helsinki, Finland.; iCAN Digital Precision Cancer Medicine Flagship, University of Helsinki, Helsinki, Finland.; K. Albin Johansson Cancer Researcher Fellow, Foundation for the Finnish Cancer Institute, Helsinki, Finland.<br /><searchLink fieldCode="AU" term="%22Wartiovaara-Kautto+U%22">Wartiovaara-Kautto U</searchLink>; Applied Tumor Genomics and Research Program, Research Programs Unit, University of Helsinki, Helsinki, Finland.; Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland.; iCAN Digital Precision Cancer Medicine Flagship, University of Helsinki, Helsinki, Finland.
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