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Hematopoietic stem cell transplantation for purine nucleoside phosphorylase deficiency: an EBMT-IEWP retrospective study.

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Title: Hematopoietic stem cell transplantation for purine nucleoside phosphorylase deficiency: an EBMT-IEWP retrospective study.
Authors: Herrmann US; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.; Division of Pediatric Hematology and Oncology, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.; Department for BioMedical Research, University of Bern, Bern, Switzerland., Felber M; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland., Worth A; Department of Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom., Haskologlu S; Department of Pediatric Immunology and Allergy, Ankara University Medical School, Ankara, Turkey., Dogu F; Department of Pediatric Immunology and Allergy, Ankara University Medical School, Ankara, Turkey., Lewis VA; Department of Pediatric Oncology and Blood and Marrow Transplant, Alberta Children's Hospital, University of Calgary Cumming School of Medicine, Calgary, Canada., Strahm B; Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, Children's Hospital, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany., Groll A; Department of Hematology/Oncology/Bone Marrow Transplantation, University Children's Hospital, Münster, Germany., Gennery AR; Institute of Cellular Medicine, Child Health, University of Newcastle upon Tyne, Newcastle Upon Tyne, United Kingdom., Hauck F; Department of Pediatric Hematology and Oncology, Dr. von Hauner Children's University Hospital, Ludwig Maximilian University, Munich, Germany., Wynn R; Department of Blood and Marrow Transplant, Royal Manchester Children's Hospital, Manchester, United Kingdom., Coussons M; Department of Blood and Marrow Transplant, Royal Manchester Children's Hospital, Manchester, United Kingdom., Meyts I; Department of Microbiology, Immunology and Transplantation, Katholieke Universiteit, Leuven, Belgium.; Department of Pediatrics, University Hospital, Leuven, Belgium., Lindemans C; Pediatric Blood and Marrow Transplantation Program, Princess Máxima Centre for Pediatric Oncology, Utrecht, The Netherlands.; Department of Pediatrics, University Medical Centre, Utrecht, The Netherlands., Bordon V; Department of Pediatric Hemato-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium., Bredius RGM; Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands., Kühl JS; Department of Pediatric Hematology/Oncology/Hemostaseology, Leipzig University Hospital, Leipzig, Germany., Völler M; Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Berlin, Germany., Zirngibl F; Department of Pediatric Oncology and Hematology, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany., Zaidman I; Department of Pediatric Hematology Oncology, Hadassah Medical Center, Jerusalem, Israel., Laberko A; Department of Immunology, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia., Zeilhofer U; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland., Hauri-Hohl M; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland., Lankester A; Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands., Ikinciogullari A; Department of Pediatric Immunology and Allergy, Ankara University Medical School, Ankara, Turkey., Guilcher GMT; Department of Pediatric Oncology and Blood and Marrow Transplant, Alberta Children's Hospital, University of Calgary Cumming School of Medicine, Calgary, Canada., Hackenberg A; Department of Pediatric Neurology, University Children`s Hospital, Zurich, Switzerland., Yeşilipek A; Department of Pediatric Hematology and Oncology, Clinic Medical Park Antalya Hospital, Antalya, Turkey., Davies G; Department of Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom., Rao K; Department of Blood and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom., Hershfield MS; Division of Pediatric Blood and Marrow Transplantation, Duke University Medical Center, Durham, NC., Parikh SH; Department of Pediatrics, Emory University School of Medicine, Atlanta, GA.; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA., Gilbert P; European Society of Blood and Marrow Transplantation, Leiden Study Unit, Leiden, The Netherlands., Bettoni da Cunha Riehm C; Department of Pediatric Hematology and Oncology, Medical School Hannover, Hannover, Germany., Albert MH; Department of Pediatric Hematology and Oncology, Dr. von Hauner Children's University Hospital, Ludwig Maximilian University, Munich, Germany., Schulz AS; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany., Hönig M; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany., Neven B; Department of Immunology and Stem Cell Transplantation, Hôpital Necker Enfants Malades, Paris, France., Güngör T; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.
Source: Blood [Blood] 2026 Jan 08; Vol. 147 (2), pp. 138-163.
Publication Type: Journal Article; Multicenter Study
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Hematopoietic stem cell transplantation for purine nucleoside phosphorylase deficiency: an EBMT-IEWP retrospective study.
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  Data: <searchLink fieldCode="AU" term="%22Herrmann+US%22">Herrmann US</searchLink>; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.; Division of Pediatric Hematology and Oncology, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.; Department for BioMedical Research, University of Bern, Bern, Switzerland.<br /><searchLink fieldCode="AU" term="%22Felber+M%22">Felber M</searchLink>; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Worth+A%22">Worth A</searchLink>; Department of Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Haskologlu+S%22">Haskologlu S</searchLink>; Department of Pediatric Immunology and Allergy, Ankara University Medical School, Ankara, Turkey.<br /><searchLink fieldCode="AU" term="%22Dogu+F%22">Dogu F</searchLink>; Department of Pediatric Immunology and Allergy, Ankara University Medical School, Ankara, Turkey.<br /><searchLink fieldCode="AU" term="%22Lewis+VA%22">Lewis VA</searchLink>; Department of Pediatric Oncology and Blood and Marrow Transplant, Alberta Children's Hospital, University of Calgary Cumming School of Medicine, Calgary, Canada.<br /><searchLink fieldCode="AU" term="%22Strahm+B%22">Strahm B</searchLink>; Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, Children's Hospital, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.<br /><searchLink fieldCode="AU" term="%22Groll+A%22">Groll A</searchLink>; Department of Hematology/Oncology/Bone Marrow Transplantation, University Children's Hospital, Münster, Germany.<br /><searchLink fieldCode="AU" term="%22Gennery+AR%22">Gennery AR</searchLink>; Institute of Cellular Medicine, Child Health, University of Newcastle upon Tyne, Newcastle Upon Tyne, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Hauck+F%22">Hauck F</searchLink>; Department of Pediatric Hematology and Oncology, Dr. von Hauner Children's University Hospital, Ludwig Maximilian University, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Wynn+R%22">Wynn R</searchLink>; Department of Blood and Marrow Transplant, Royal Manchester Children's Hospital, Manchester, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Coussons+M%22">Coussons M</searchLink>; Department of Blood and Marrow Transplant, Royal Manchester Children's Hospital, Manchester, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Meyts+I%22">Meyts I</searchLink>; Department of Microbiology, Immunology and Transplantation, Katholieke Universiteit, Leuven, Belgium.; Department of Pediatrics, University Hospital, Leuven, Belgium.<br /><searchLink fieldCode="AU" term="%22Lindemans+C%22">Lindemans C</searchLink>; Pediatric Blood and Marrow Transplantation Program, Princess Máxima Centre for Pediatric Oncology, Utrecht, The Netherlands.; Department of Pediatrics, University Medical Centre, Utrecht, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Bordon+V%22">Bordon V</searchLink>; Department of Pediatric Hemato-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22Bredius+RGM%22">Bredius RGM</searchLink>; Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Kühl+JS%22">Kühl JS</searchLink>; Department of Pediatric Hematology/Oncology/Hemostaseology, Leipzig University Hospital, Leipzig, Germany.<br /><searchLink fieldCode="AU" term="%22Völler+M%22">Völler M</searchLink>; Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Berlin, Germany.<br /><searchLink fieldCode="AU" term="%22Zirngibl+F%22">Zirngibl F</searchLink>; Department of Pediatric Oncology and Hematology, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.<br /><searchLink fieldCode="AU" term="%22Zaidman+I%22">Zaidman I</searchLink>; Department of Pediatric Hematology Oncology, Hadassah Medical Center, Jerusalem, Israel.<br /><searchLink fieldCode="AU" term="%22Laberko+A%22">Laberko A</searchLink>; Department of Immunology, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Zeilhofer+U%22">Zeilhofer U</searchLink>; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Hauri-Hohl+M%22">Hauri-Hohl M</searchLink>; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Lankester+A%22">Lankester A</searchLink>; Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Ikinciogullari+A%22">Ikinciogullari A</searchLink>; Department of Pediatric Immunology and Allergy, Ankara University Medical School, Ankara, Turkey.<br /><searchLink fieldCode="AU" term="%22Guilcher+GMT%22">Guilcher GMT</searchLink>; Department of Pediatric Oncology and Blood and Marrow Transplant, Alberta Children's Hospital, University of Calgary Cumming School of Medicine, Calgary, Canada.<br /><searchLink fieldCode="AU" term="%22Hackenberg+A%22">Hackenberg A</searchLink>; Department of Pediatric Neurology, University Children`s Hospital, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Yeşilipek+A%22">Yeşilipek A</searchLink>; Department of Pediatric Hematology and Oncology, Clinic Medical Park Antalya Hospital, Antalya, Turkey.<br /><searchLink fieldCode="AU" term="%22Davies+G%22">Davies G</searchLink>; Department of Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Rao+K%22">Rao K</searchLink>; Department of Blood and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Hershfield+MS%22">Hershfield MS</searchLink>; Division of Pediatric Blood and Marrow Transplantation, Duke University Medical Center, Durham, NC.<br /><searchLink fieldCode="AU" term="%22Parikh+SH%22">Parikh SH</searchLink>; Department of Pediatrics, Emory University School of Medicine, Atlanta, GA.; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA.<br /><searchLink fieldCode="AU" term="%22Gilbert+P%22">Gilbert P</searchLink>; European Society of Blood and Marrow Transplantation, Leiden Study Unit, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Bettoni+da+Cunha+Riehm+C%22">Bettoni da Cunha Riehm C</searchLink>; Department of Pediatric Hematology and Oncology, Medical School Hannover, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Albert+MH%22">Albert MH</searchLink>; Department of Pediatric Hematology and Oncology, Dr. von Hauner Children's University Hospital, Ludwig Maximilian University, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Schulz+AS%22">Schulz AS</searchLink>; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.<br /><searchLink fieldCode="AU" term="%22Hönig+M%22">Hönig M</searchLink>; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.<br /><searchLink fieldCode="AU" term="%22Neven+B%22">Neven B</searchLink>; Department of Immunology and Stem Cell Transplantation, Hôpital Necker Enfants Malades, Paris, France.<br /><searchLink fieldCode="AU" term="%22Güngör+T%22">Güngör T</searchLink>; Department of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.
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