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: Busulfan/Fludarabine- or Treosulfan/Fludarabine-Based Conditioning Regimen in Patients with Wiskott-Aldrich Syndrome Given Allogeneic Hematopoietic Cell Transplantation — an EBMT Inborn Errors Working Party and Scetide Retrospective Analysis

Researchers

Presenter

  • Michael H. Albert

Principal Investigators

  • Andrew Gennery

  • Tayfun Guengoer

  • Henric-Jan Blok

  • Junfeng Wang

  • Virginie Courteille

  • Maria Ester Bernardo

  • Ivana Bodova

  • Benedicte Bruno

  • Tatjana A Bykova

  • Robert Chiesa

  • Alain Fischer

  • Renata Formankova

  • Krzysztof Kalwak

  • Christoph Klein

  • Svetlana Kozlovskaya

  • O. Alphan Kupesiz

  • Franco Locatelli

  • Nizar Mahlaoui

  • Despina Moshous

  • Benedicte Neven

  • Fulvio Porta

  • Ansgar Schulz

  • Karl-Walter Sykora

  • Ekrem Unal

  • Jacek H. Winiarski

  • Marco Zecca

  • Paul Veys

  • Arjan Lankester

Medical Centers

  • University Children`s Hospital, Ludwig Maximilians University, Munich, Germany

  • Great North Children's Hospital, Newcastle, United Kingdom

  • Great North Children's Hospital, Newcastle, United Kingdom

  • University Childrens Hospital, Zurich, Switzerland

  • Leiden University Medical Centre, Leiden, Netherlands

  • SCETIDE, Paris, France

  • IRCCS Ospedale San Raffaele, Milan, Italy

  • University Childrens´Hospital, Bratislava, Slovakia

  • Hopital Jeanne de Flandre, Lille, France

  • Raisa Gorbacheva Memorial Institute of Children Oncology, Hematology and Transplantation, First Pavlov State Medical University of Saint-Petersburg, Saint-Petersburg, Russian Federation,

  • Bone Marrow Transplantation Department, Great Ormond Street hospital NHS Trust, London, United Kingdom

  • Hopital Necker-Enfants Malades, Paris, France

  • University Hospital Motol, Prague, Czech Republic

  • Pediatric Hematology/Oncology and BMT,Wroclaw Medical University,Wroclaw,Poland

  • Dr. von Hauner Children's Hospital, Ludwig-Maximilians-Universitat (LMU), Munich, Germany

  • Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation

  • Dumlupinar Bulvari, Antalya, Turkey

  • Department of Pediatric Hematology and Oncology, Cell and Gene Therapy,IRCCS Bambino Gesù Children's Hospital,Roma,Italy

  • Hopital Necker-Enfants Malades, Paris, France

  • Necker Hospital, Assistance Publique-Hopitaux de Paris, Paris, France

  • Ospedale dei Bambini, Brescia, Italy

  • Pediatrics, University Medical Center, Ulm, Germany

  • University Children's Hospital, Tubingen, Germany

  • Department of Pediatrics, Division of Pediatric Hematology and Oncology, Erciyes University, Kayseri, Turkey

  • Karolinska Institutet, Stockholm, Sweden

  • Medical Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

  • Bone Marrow Transplantation Department, Great Ormond Street hospital NHS Trust, London, United Kingdom

Locations

  • Germany

  • United Kingdom

  • Switzerland

  • Netherlands

  • France

  • Italy

  • Slovakia

  • Russia

  • Czech Republic

  • Poland

  • Turkey

  • Sweden

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Autoimmune (AI)

Disease

  • Post-transplant lymphoproliferative disorder

  • Wiskott-aldrich syndrome

  • Graft-versus-host disease

  • Macrocephaly macrosomia facial dysmorphism syndrome

Biomarkers

  • N/A

Drug/Treatment

  • Cyclophosphamide

  • Cyclophosphamide Injection

  • fludarabine tablet

  • Ovastat

  • Busulfan

  • Thiotepa

  • Insulin tregopil

Outcome

  • N/A

Study Design

  • Cohort

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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