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Journal Articles

Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities

Blood (2024) 144 (Supplement 2): LBA-5.

https://doi.org/10.1182/blood-2024-212967

Published: 2024

Journal Articles

Allogeneic Transplantation for Sickle Cell Disease Offers High Rates of Cure with Low Incidence of Severe Chronic GVHD in Both Children and Adults: Real World Data from 2010-2021. An Analysis of the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Working Party

Blood (2024) 144 (Supplement 1): 176.

https://doi.org/10.1182/blood-2024-207944

Published: 2024

Journal Articles

Gene Therapy for Artemis-SCID and Artemis-Leaky-SCID Patients: Preliminary Results of the French Artegene Phase I/II Clinical Trial

Blood (2024) 144 (Supplement 1): 2201.

https://doi.org/10.1182/blood-2024-208350

Published: 2024

Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) Outcomes in Leukocyte Adhesion Deficiency Type -I

Blood (2024) 144 (Supplement 1): 5079.

https://doi.org/10.1182/blood-2024-203643

Published: 2024

Journal Articles

Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

Blood (2024) 144 (5): 565–580.

https://doi.org/10.1182/blood.2024024038

Published: 2024

Includes: Supplemental data

Journal Articles

Antinuclear antibody–associated autoimmune cytopenia in childhood is a risk factor for systemic lupus erythematosus

Blood (2024) 143 (16): 1576–1585.

https://doi.org/10.1182/blood.2023021884

Published: 2024

Includes: Supplemental data

Journal Articles

Cryopreservation Impacts Allogenic Stem Cell Engraftment: Relation between Graft Quality Control and Post-Transplant Clinical Outcome

Clotilde Aussel, Jeromine Beaufort, Elisa Magrin, Olivia Leblanc, Laure Joseph, Francois Lefrere, Clemence Loiseau, Martin Castelle, Benjamin Fournier, Morseny Gueye, Despina Moshous, Alessandra Magnani, Marina Cavazzana, Felipe Suarez, Benedicte Neven, Ambroise Marçais, Jean-Sebastien Diana

Blood (2023) 142 (Supplement 1): 6949.

https://doi.org/10.1182/blood-2023-187478

Published: 2023

Journal Articles

DOCK11 deficiency in patients with X-linked actinopathy and autoimmunity

Charlotte Boussard, Laure Delage, Tania Gajardo, Alexandre Kauskot, Maxime Batignes, Nicolas Goudin, Marie-Claude Stolzenberg, Camille Brunaud, Patricia Panikulam, Quentin Riller, Maryse Moya-Nilges, Jean Solarz, Christelle Repérant, Béatrice Durel, Jean-Claude Bordet, Olivier Pellé, Corinne Lebreton, Aude Magérus, Vithura Pirabakaran, Pablo Vargas, Sébastien Dupichaud, Marie Jeanpierre, Angélique Vinit, Mohammed Zarhrate, Cécile Masson, Nathalie Aladjidi, Peter D. Arkwright, Brigitte Bader-Meunier, Sandrine Baron Joly, Joy Benadiba, Elise Bernard, Dominique Berrebi, Christine Bodemer, Martin Castelle, Fabienne Charbit-Henrion, Marwa Chbihi, Agathe Debray, Philippe Drabent, Sylvie Fraitag, Miguel Hié, Judith Landman-Parker, Ludovic Lhermitte, Despina Moshous, Pierre Rohrlich, Frank Ruemmele, Anne Welfringer-Morin, Maud Tusseau, Alexandre Belot, Nadine Cerf-Bensussan, Marie Roelens, Capucine Picard, Bénédicte Neven, Alain Fischer, Isabelle Callebaut, Mickaël Ménager, Fernando E. Sepulveda, Frédéric Adam, Frédéric Rieux-Laucat

Blood (2023) 141 (22): 2713–2726.

https://doi.org/10.1182/blood.2022018486

Published: 2023

Includes: Supplemental data

Journal Articles

Allogeneic stem cell transplantation compared to conservative management in adults with inborn errors of immunity

Blood (2023) 141 (1): 60–71.

https://doi.org/10.1182/blood.2022015482

Published: 2023

Includes: Supplemental data

Journal Articles

HLA-Haploidentical Stem Cell Transplantation in Children with Inherited Bone Marrow Failure Syndromes: A Retrospective Analysis on Behalf of EBMT Severe Aplastic Anemia Working Party

Blood (2022) 140 (Supplement 1): 5813–5815.

https://doi.org/10.1182/blood-2022-163219

Published: 2022

Journal Articles

Clinical, Immunological Features, Treatments, and Outcomes of Autoimmune Hemolytic Anemia in Patients with RAG Deficiency

Blood (2022) 140 (Supplement 1): 2448–2450.

https://doi.org/10.1182/blood-2022-162845

Published: 2022

Journal Articles

Impact of Hydroxyurea and Vaso-Occlusive Crisis on Ovarian Follicle Density in Girls and Young Females with Sickle Cell Disease

Tamara Diesch, Catherine Poirot, Carlos Sanchez, Andrew Atkinson, Corinne Pondarré, Nathalie Dhedin, Benedicte Neven, France Pirenne, Gilles Leanour, Isabelle Broncheriou, Françoise Bernaudin, Jean-Hugues Dalle

Blood (2022) 140 (Supplement 1): 453–454.

https://doi.org/10.1182/blood-2022-164679

Published: 2022

Journal Articles

Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Blood (2022) 140 (14): 1635–1649.

https://doi.org/10.1182/blood.2022015506

Published: 2022

Includes: Supplemental data

Journal Articles

Defects in mucosal immunity and nasopharyngeal dysbiosis in HSC-transplanted SCID patients with IL2RG/JAK3 deficiency

Pedro Goncalves, Jean-Marc Doisne, Toshiki Eri, Bruno Charbit, Vincent Bondet, Celine Posseme, Alba Llibre, Armanda Casrouge, Christelle Lenoir, Bénédicte Neven, Darragh Duffy, Alain Fischer, James P. Di Santo

Blood (2022) 139 (17): 2585–2600.

https://doi.org/10.1182/blood.2021014654

Published: 2022

Includes: Supplemental data

Journal Articles

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Blood (2022) 139 (13): 2066–2079.

https://doi.org/10.1182/blood.2021014687

Published: 2022

Includes: Supplemental data

Journal Articles

Rapid and Safe T Cell Immune Reconstitution By T Cell Progenitor Injection Following Haploidentical Transplantation for Severe Combined Immunodeficiency (SCID)

Despina Moshous, Elisa Magrin, Sarah Winter, Benjamin Fournier, Martin Castelle, Laurence Vendrame, Lucienne Chatenoud, Capucine Picard, Bénédicte Neven, Alessandra Magnani, Pierre Gaudeaux, Tayebeh-Shabi Soheili, Isabelle Andre, Laura Simons, Marina Cavazzana

Blood (2021) 138 (Supplement 1): 1752.

https://doi.org/10.1182/blood-2021-152281

Published: 2021

Journal Articles

CALR mutant protein rescues the response of MPL p.R464G variant associated with CAMT to eltrombopag

Francesca Basso-Valentina, Gabriel Levy, Leila N. Varghese, Myriam Oufadem, Benedicte Neven, Charlotte Boussard, Nathalie Balayn, Caroline Marty, William Vainchenker, Isabelle Plo, Paola Ballerini, Stefan N. Constantinescu, Remi Favier, Hana Raslova

Blood (2021) 138 (6): 480–485.

https://doi.org/10.1182/blood.2020010567

Published: 2021

Includes: Supplemental data

Journal Articles

Hydroxyurea does not affect the spermatogonial pool in prepubertal patients with sickle cell disease

Blood (2021) 137 (6): 856–859.

https://doi.org/10.1182/blood.2020008146

Published: 2021

Includes: Supplemental data

Journal Articles

Extended clinical and immunological phenotype and transplant outcome in CD27 and CD70 deficiency

Sujal Ghosh, Sevgi Köstel Bal, Emily S. J. Edwards, Bethany Pillay, Raúl Jiménez Heredia, Funda Erol Cipe, Geetha Rao, Elisabeth Salzer, Samaneh Zoghi, Hassan Abolhassani, Tooba Momen, Emma Gostick, David A. Price, Yu Zhang, Andrew J. Oler, Claudia Gonzaga-Jauregui, Baran Erman, Ayse Metin, Inci Ilhan, Sule Haskologlu, Candan Islamoglu, Kubra Baskin, Serdar Ceylaner, Ebru Yilmaz, Ekrem Unal, Musa Karakukcu, Dagmar Berghuis, Theresa Cole, Aditya K. Gupta, Fabian Hauck, Hubert Kogler, Andy I. M. Hoepelman, Safa Baris, Elif Karakoc-Aydiner, Ahmet Ozen, Leo Kager, Dirk Holzinger, Michael Paulussen, Renate Krüger, Roland Meisel, Prasad T. Oommen, Emma Morris, Benedicte Neven, Austen Worth, Joris van Montfrans, Pieter L. A. Fraaij, Sharon Choo, Figen Dogu, E. Graham Davies, Siobhan Burns, Gregor Dückers, Ruy Perez Becker, Horst von Bernuth, Sylvain Latour, Maura Faraci, Marco Gattorno, Helen C. Su, Qiang Pan-Hammarström, Lennart Hammarström, Michael J. Lenardo, Cindy S. Ma, Tim Niehues, Asghar Aghamohammadi, Nima Rezaei, Aydan Ikinciogullari, Stuart G. Tangye, Arjan C. Lankester, Kaan Boztug

Blood (2020) 136 (23): 2638–2655.

https://doi.org/10.1182/blood.2020006738

Published: 2020

Includes: Supplemental data

Journal Articles

Pediatric-Onset Evans Syndrome Is Associated with Broad Immunopathological Manifestations, High Treatment Burden and Mortality in Long-Term Follow-up

Blood (2020) 136 (Supplement 1): 20–22.

https://doi.org/10.1182/blood-2020-133778

Published: 2020

NARROW

Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities

Allogeneic Transplantation for Sickle Cell Disease Offers High Rates of Cure with Low Incidence of Severe Chronic GVHD in Both Children and Adults: Real World Data from 2010-2021. An Analysis of the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Working Party

Gene Therapy for Artemis-SCID and Artemis-Leaky-SCID Patients: Preliminary Results of the French Artegene Phase I/II Clinical Trial

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) Outcomes in Leukocyte Adhesion Deficiency Type -I

Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

Antinuclear antibody–associated autoimmune cytopenia in childhood is a risk factor for systemic lupus erythematosus

Cryopreservation Impacts Allogenic Stem Cell Engraftment: Relation between Graft Quality Control and Post-Transplant Clinical Outcome

DOCK11 deficiency in patients with X-linked actinopathy and autoimmunity

Allogeneic stem cell transplantation compared to conservative management in adults with inborn errors of immunity

HLA-Haploidentical Stem Cell Transplantation in Children with Inherited Bone Marrow Failure Syndromes: A Retrospective Analysis on Behalf of EBMT Severe Aplastic Anemia Working Party

Clinical, Immunological Features, Treatments, and Outcomes of Autoimmune Hemolytic Anemia in Patients with RAG Deficiency

Impact of Hydroxyurea and Vaso-Occlusive Crisis on Ovarian Follicle Density in Girls and Young Females with Sickle Cell Disease

Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Defects in mucosal immunity and nasopharyngeal dysbiosis in HSC-transplanted SCID patients with IL2RG/JAK3 deficiency

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Rapid and Safe T Cell Immune Reconstitution By T Cell Progenitor Injection Following Haploidentical Transplantation for Severe Combined Immunodeficiency (SCID)

CALR mutant protein rescues the response of MPL p.R464G variant associated with CAMT to eltrombopag

Hydroxyurea does not affect the spermatogonial pool in prepubertal patients with sickle cell disease

Extended clinical and immunological phenotype and transplant outcome in CD27 and CD70 deficiency

Pediatric-Onset Evans Syndrome Is Associated with Broad Immunopathological Manifestations, High Treatment Burden and Mortality in Long-Term Follow-up

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American Society of Hematology

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American Society of Hematology

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