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: Prospective and Retrospective Observational Study in Adult and Pediatric Patients with GATA2 Variants (GATA2 Deficiency)

Researchers

Presenter

  • Samuele Roncareggi

Principal Investigators

  • Francesca Fioredda

  • Francesca Conti

  • Erika Borlenghi

  • Concetta Micalizzi

  • Gaia Mancuso

  • Fabiola Guerra

  • Emilia Cirillo

  • Antonio Marzollo

  • Piero Farruggia

  • Daniela Onofrillo

  • Riccardo Masetti

  • Raffaele Badolato

  • Luca Arcuri

  • Tiziana Angela Coliva

  • Francesca Vendemini

  • Claudio Pignata

  • Sonia Bonanomi

  • Andrea Biondi

  • Francesco Saettini

Medical Centers

  • Hematology Unit, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS)-Istituto Giannina Gaslini, Genova, Italy

  • Centro Ricerca Tettamanti, Pediatric Department, University of Milano-Bicocca, Fondazione MBBM, Monza, Italy

  • Department of Hematology, ASST Spedali Civili di Brescia, Brescia, Italy

  • Federico II University, Naples, ITA

  • Tettamanti Research Centre, Department of Paediatrics, University of Milano-Bicocca, Monza, Italy., Monza, ITA

  • Haematology Unit, Istituto Giannina Gaslini, Genoa, Italy., Genoa, Italy

  • University of Milano Bicocca, Monza, Italy, Monza, Italy

  • Haematology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy

  • Ospedale Civile, Pescara, Italy

  • Pediatric Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, ITA

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

  • Ospedale San Gerardo, Monza, ITA

  • Department of Paediatrics, Paediatric Haematology-Oncology Unit, Fondazione MBBM/San Gerardo Hospital, Monza, Italy., Monza, ITA

  • Pediatric Hematology and Oncology Unit, A.R.N.A.S. Ospedale Civico, Palermo, Italy

  • Unit of Immunology, Rheumatology, Allergy and Rare Diseases (UnIRAR), IRCCS San Raffaele Scientific Institute, Milan, Italy., Milan, Italy

  • U.O.C. Oncoematologia Pediatrica, Padova, ITA

  • Milano Bicocca University, Monza, Italy

Locations

  • Italy

  • United States

Companies

  • N/A

Study Components

Therapeutic Area

  • Autoimmune (AI)

  • Pulmonary/Respiratory Diseases

  • Cardiovascular (CVS)

  • Oncology (ONC)

  • Genetic Disorder

  • Blood/haematological Diseases

Disease

  • Pulmonary alveolar proteinosis

  • Hematological malignancies

  • Acute myelocytic leukemia

  • Hypogammaglobulinaemia

  • Advanced biliary tract carcinoma

  • Myeloid neoplasms

  • Lymphocytopenia

  • Myelodysplastic Syndrome

  • Myelofibrosis

  • Venous thrombosis

  • Wiskott-aldrich syndrome

Biomarkers

  • Cluster of Differentiation 19

  • GATA-binding protein 2

  • Enhancer of zeste homolog 2

  • MDS1 And EVI1 Complex Locus

  • X-Ray Repair Cross Complementing 1

Drug/Treatment

  • EZH2

Outcome

  • N/A

Study Design

  • Observational

  • Prospective

  • Cohort

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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