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Journal Articles

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

Jean-Sebastien Diana, Despina Moshous, Martin Castelle, Boris Bessot, Elisa Magrin, Marwa Chbihi, Paul Bastard, Alice Girardot, Cecile Roudaut, Chloe Mollet, Clotilde Aussel, Caroline Tuchmann-Durand, Eden Schwartz, Olaf Neth, Peter Olbrich, Irene d'Alba, Jacques Riviere, Medhi Oualha, Laure Joseph, Benedicte Neven, Jean-Pierre De Villartay, Anne Galy, Chantal Lagresle-Peyrou, Marina Cavazzana
Journal: Blood
Blood (2024) 144 (Supplement 1): 2201.
https://doi.org/10.1182/blood-2024-208350
Published: 2024
Journal Articles

Childhood Langerhans cell histiocytosis hematological involvement: severity associated with BRAFV600E loads

Julian Thalhammer, Eric Jeziorski, Perrrine Marec-Berard, Mohamed-Aziz Barkaoui, Anne Pagnier, Pierre-Simon Rohrlich, Aurore Chevallier, Liana Carausu, Nathalie Aladjidi, Charlotte Rigaud, Amaury Leruste, Saba Azarnoush, Thomas Lauvray, Solenne Le Louet, Virginie Gandemer, Pauline Treguier, Ludovic Mansuy, Marlène Pasquet, Laura Olivier, Angélique Rome, Paul Saultier, Florentina Isfan, Cecile Renard, Valérie Li Thiao Te, Alexandra Salmon, Laurence Blanc, Wadih Abou Chahla, Anne Lambilliotte, Jean-Louis Louis Stephan, Frederic Geissmann, Julien Lejeune, Coralie Mallebranche, Yves Reguerre, Audrey Grain, Caroline Thomas, Zofia Hélias-Rodzewicz, Despina Moshous, Odile Fenneteau, Aurore Coulomb-L'hermine, Hélène Lapillonne, Geneviève de Saint Basile, Jean-Francois Emile, Sébastien Héritier, Jean Donadieu
Journal: Blood
Blood blood.2024025625.
https://doi.org/10.1182/blood.2024025625
Published: 2024
Includes: Supplemental data
Journal Articles

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

Clinical Trials & Observations
Su Han Lum, Michael H. Albert, Patrick Gilbert, Tiarlan Sirait, Mattia Algeri, Rafaella Muratori, Benjamin Fournier, Alexandra Laberko, Musa Karakukcu, Elrem Unal, Mouhab Ayas, Satya Prakash Yadav, Tunc Fisgin, Reem Elfeky, Juliana Fernandes, Maura Faraci, Theresa Cole, Ansgar Schulz, Roland Meisel, Marco Zecca, Marianne Ifversen, Alessandra Biffi, Jean-Sebastien Diana, Tanja Vallée, Stefano Giardino, Gizem Zengin Ersoy, Despina Moshous, Andrew R. Gennery, Dmitry Balashov, Carmem Bonfim, Franco Locatelli, Arjan Lankester, Bénédicte Neven, Mary Slatter
Journal: Blood
Blood (2024) 144 (5): 565–580.
https://doi.org/10.1182/blood.2024024038
Published: 2024
Includes: Supplemental data
Journal Articles

Clinical and functional spectrum of RAC2-related immunodeficiency

Clinical Trials & Observations
Ágnes Donkó, Svetlana O. Sharapova, Juraj Kabat, Sundar Ganesan, Fabian H. Hauck, Jenna R. E. Bergerson, Louis Marois, Jordan Abbott, Despina Moshous, Kelli W. Williams, Nicholas Campbell, Paul L. Martin, Chantal Lagresle-Peyrou, Timothy Trojan, Natalia B. Kuzmenko, Ekaterina A. Deordieva, Elena V. Raykina, Michael S. Abers, Hassan Abolhassani, Vincent Barlogis, Carlos Milla, Geoffrey Hall, Talal Mousallem, Joseph Church, Neena Kapoor, Guilhem Cros, Hugo Chapdelaine, Clara Franco-Jarava, Ingrid Lopez-Lerma, Maurizio Miano, Jennifer W. Leiding, Christoph Klein, Marie José Stasia, Alain Fischer, Kuang-Chih Hsiao, Timi Martelius, Mikko R. J. Sepännen, Sara Barmettler, Jolan Walter, Tania N. Masmas, Anna A. Mukhina, Emilia Liana Falcone, Sven Kracker, Anna Shcherbina, Steven M. Holland, Thomas L. Leto, Amy P. Hsu
Journal: Blood
Blood (2024) 143 (15): 1476–1487.
https://doi.org/10.1182/blood.2023022098
Published: 2024
Includes: Supplemental data
Journal Articles

Human Pro-T-Cell Manufacturing in Vitro Is a Safe Procedure for Hematopoietic Stem Cell Transplantation with Delayed T-Cell Reconstitution: Interim Results from 2 Different Clinical Trials

Elisa Magrin, Jean-Sebastien Diana, Martin Castelle, Olivier Negre, Laura Simons, Clotilde Aussel, Eden Schwartz, Alessandra Magnani, Jimni Baek, Caroline Elie, Axelle Maulet, Chloe Mollet, Alice Girardot, Jeremie Rosain, Capucine Picard, Aurore Touzart, Vahid Asnafi, Edouard Forcade, Anne Huynh, jean-Marc Treluyer, Isabelle Andre, Despina Moshous, Olivier Hermine, Marina Cavazzana
Journal: Blood
Blood (2023) 142 (Supplement 1): 6868.
https://doi.org/10.1182/blood-2023-187607
Published: 2023
Journal Articles

Hematological Involvement in Childhood Langerhans Cell Histiocytosis: A Cohort Study of 303 Patients

Julian Thalhammer, Perrine Marec-Bérard, Mohamed Barkaoui, Eric Jeziorski, Angelique Rome, Anne Pagnier, Pierre Simon Rohrlich, Nathalie Aladjidi, Charlotte Rigaud, Amaury Leruste, Saba Azarnoush, Thomas Lauvray, Solenne Le Louet, Virginie Gandemer, Pauline Treguier, Ludovic Mansuy, Marlène Pasquet, Laura Olivier, Florentina Isfan, Cécile Renard, Alexandra Salmon, Laurence Blanc, Wadih Abou Chahla, Anne Lambilliotte, Coralie Mallebranche, Caroline Thomas, Despina Moshous, Genevieve de Saint-Basile, Jean-Francois Emile, Sébastien Héritier, Jean Donadieu
Journal: Blood
Blood (2023) 142 (Supplement 1): 4573.
https://doi.org/10.1182/blood-2023-184588
Published: 2023
Journal Articles

Preclinical Assessment for Gene Addition Strategy in Familial Hemophagocytic Lymphohistiocytosis Related to Munc 13-4 Deficiency

Chantal Lagresle-Peyrou, Jean-Sebastien Diana, Elisa Magrin, Clotilde Aussel, Natael Sorel, Yetki Aslan, Cécile Roudaut, Eden Schwartz, Aurélie Gabrion, Caroline Tuchmann-Durand, Francois Lefrere, Anne Galy, Despina Moshous, Marina Cavazzana
Journal: Blood
Blood (2023) 142 (Supplement 1): 3633.
https://doi.org/10.1182/blood-2023-186133
Published: 2023
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 6949.
https://doi.org/10.1182/blood-2023-187478
Published: 2023
Journal Articles

Fertility and Pregnancy in Patients with Congenital Neutropenia: Experience of the French Severe Chronic Neutropenia Registry in 565 Patients Above Age of 15 Years

Aurelia Alimi, Evrard Nika, Blandine Beaupain, Claire Fieschi, Flore Sicre De Fontbrune, Nathalie Aladjidi, Despina Moshous, Aline Moignet.Autrel, Coralie Mallebranche, Martin Biosse Duplan, Christine Bellanne-Chantelot, Jean Donadieu
Journal: Blood
Blood (2023) 142 (Supplement 1): 2545.
https://doi.org/10.1182/blood-2023-184559
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
Journal: Blood
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

Clinical Trials & Observations
Morgane Cheminant, Thomas A. Fox, Mickael Alligon, Olivier Bouaziz, Bénédicte Neven, Despina Moshous, Stéphane Blanche, Aurélien Guffroy, Claire Fieschi, Marion Malphettes, Nicolas Schleinitz, Antoinette Perlat, Jean-François Viallard, Nathalie Dhedin, Françoise Sarrot-Reynauld, Isabelle Durieu, Sébastien Humbert, Fanny Fouyssac, Vincent Barlogis, Benjamin Carpenter, Rachael Hough, Arian Laurence, Ambroise Marçais, Ronjon Chakraverty, Olivier Hermine, Alain Fischer, Siobhan O. Burns, Nizar Mahlaoui, Emma C. Morris, Felipe Suarez
Journal: Blood
Blood (2023) 141 (1): 60–71.
https://doi.org/10.1182/blood.2022015482
Published: 2023
Includes: Supplemental data
Journal Articles

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

Chen Wang, Bijun Sun, Kevin Wu, Jocelyn Farmer, Boglarka Ujhazi, Christoph B. Geier, Sumai Gordon, Emma Westermann-Clark, Sinisa Savic, Sargur Ravishankar, Karin Chen, Cullen M Dutmer, Maria G Kanariou, Mehdi Adeli, Paolo Palma, Carmem Bonfim, Evangelia Lycopoulou, Beata Wolska-Kusnierz, Dbaibo Ghassan, Jack Bleesing, Despina Moshous, Benedicte Neven, Catharina Schuetz, Geha Raif, Luigi D. Notarangelo, Maurizio Miano, David K. Buchbinder, Krisztian Csomos, Wenjie Wang, Ji-Yang Wang, Xiaochuan Wang, Jolan E. Walter
Journal: Blood
Blood (2022) 140 (Supplement 1): 2448–2450.
https://doi.org/10.1182/blood-2022-162845
Published: 2022
Journal Articles

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

Clinical Trials & Observations
Michael H. Albert, Tiarlan Sirait, Dirk-Jan Eikema, Katerina Bakunina, Claudia Wehr, Felipe Suarez, Maria Laura Fox, Nizar Mahlaoui, Andrew R. Gennery, Arjan C. Lankester, Rita Beier, Maria Ester Bernardo, Venetia Bigley, Caroline A. Lindemans, Siobhan O. Burns, Ben Carpenter, Jaroslaw Dybko, Tayfun Güngör, Fabian Hauck, Su Han Lum, Dmitry Balashov, Roland Meisel, Despina Moshous, Ansgar Schulz, Carsten Speckmann, Mary A. Slatter, Brigitte Strahm, Duygu Uckan-Cetinkaya, Isabelle Meyts, Tanja C. Vallée, Robert Wynn, Bénédicte Neven, Emma C. Morris
Journal: Blood
Blood (2022) 140 (14): 1635–1649.
https://doi.org/10.1182/blood.2022015506
Published: 2022
Includes: Supplemental data
Journal Articles

Determinants of long-term outcomes of splenectomy in pediatric autoimmune cytopenias

Clinical Trials & Observations
Thomas Pincez, Nathalie Aladjidi, Sébastien Héritier, Nathalie Garnier, Mony Fahd, Wadih Abou Chahla, Helder Fernandes, Claire Dichamp, Stéphane Ducassou, Marlène Pasquet, Sophie Bayart, Despina Moshous, Nathalie Cheikh, Catherine Paillard, Dominique Plantaz, Eric Jeziorski, Caroline Thomas, Corinne Guitton, Marianna Deparis, Aude Marie Cardine, Jean-Louis Stephan, Isabelle Pellier, Eric Doré, Joy Benadiba, Claire Pluchart, Claire Briandet, Vincent Barlogis, Guy Leverger, Thierry Leblanc
Journal: Blood
Blood (2022) 140 (3): 253–261.
https://doi.org/10.1182/blood.2022015508
Published: 2022
Includes: Supplemental data
Journal Articles

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

Clinical Trials & Observations
Michael H. Albert, Mary A. Slatter, Andrew R. Gennery, Tayfun Güngör, Katerina Bakunina, Benyamin Markovitch, Sheree Hazelaar, Tiarlan Sirait, Virginie Courteille, Alessandro Aiuti, Olga V. Aleinikova, Dmitry Balashov, Maria Ester Bernardo, Ivana Bodova, Benedicte Bruno, Marina Cavazzana, Robert Chiesa, Alain Fischer, Fabian Hauck, Marianne Ifversen, Krzysztof Kałwak, Christoph Klein, Alexander Kulagin, Alphan Kupesiz, Baris Kuskonmaz, Caroline A. Lindemans, Franco Locatelli, Su Han Lum, Alexey Maschan, Roland Meisel, Despina Moshous, Fulvio Porta, Martin G. Sauer, Petr Sedlacek, Ansgar Schulz, Felipe Suarez, Tanja C. Vallée, Jacek H. Winiarski, Marco Zecca, Bénédicte Neven, Paul Veys, Arjan C. Lankester
Journal: Blood
Blood (2022) 139 (13): 2066–2079.
https://doi.org/10.1182/blood.2021014687
Published: 2022
Includes: Supplemental data
Journal Articles

An Autosomal Dominant Form of Ras-Related C3 Botulinum Toxin Substrate 2 (RAC2) Is Associated with Haematopoiesis Failure

Chantal Lagresle-Peyrou, Aurélien Olichon, Hanem Sadek, Philippe Roche, Claudine Tardy, Natael Sorel, Boris Bessot, Cindy Da Silva, Alain Fischer, Despina Moshous, Yves Collette, Capucine Picard, Jean-Laurent Casanova, Isabelle André, Marina Cavazzana
Journal: Blood
Blood (2021) 138 (Supplement 1): 4306.
https://doi.org/10.1182/blood-2021-150114
Published: 2021
Journal Articles

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Alessandra Magnani, Michaela Semeraro, Frédéric ADAM, Claire Booth, Loic Dupre, Emma C Morris, Aurélie Gabrion, Cecile Roudaut, Delphine Borgel, Antoine Toubert, Emmanuel Clave, Chrystelle Abdo, Guy Gorochov, Rachel Petermann, Mélanie Guiot, Makoto Miyara, Despina Moshous, Elisa Magrin, Adeline Denis, Felipe Suarez, Chantal Lagresle-Peyrou, Aoife Doto, John K. Everett, Amélie Trinquand, Marianne Guisset, Jin Hua Xu-Bayford, Salima Hacein-Bey-Abina, Alexandre Kauskot, Reem Elfeky, Christine Rivat, Sarah Abbas, Bobby H Gaspar, Elizabeth A. Macintyre, Capucine Picard, Frederic D. Bushman, Anne Galy, Alain Fischer, Emmanuelle Six, Adrian J. Thrasher, Marina Cavazzana
Journal: Blood
Blood (2021) 138 (Supplement 1): 2934.
https://doi.org/10.1182/blood-2021-147697
Published: 2021
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
Journal: Blood
Blood (2021) 138 (Supplement 1): 1752.
https://doi.org/10.1182/blood-2021-152281
Published: 2021
Journal Articles

How Many Patients Have Congenital Neutropenia? a Population-Based Estimation from the Nationwide French Severe Chronic Neutropenia Registry

Jean Donadieu, MDPhD, Blandine Beaupain, MsC, Hélène Lapillonne, MD PhD, Odile Fenneteau, PharmD, Flore Sicre de Fontbrune, Yves Bertrand, MD, Nathalie Aladjidi, MD, Vincent Barlogis, MD, PhD, Wadih Abou Chahla, MD, Thierry Lamy, PhD MD, Aline Moignet Autrel, MD, Claire Fieschi, MD PhD, Thierry Leblanc, MD, Despina Moshous, MD PhD, Jean Soulier, MDPhD, Sarah cohen Beaussant, Francois Delhommeau, MD PhD, Helene Cavé, PharmD, PhD, Catherine Paillard, MD PhD, Elodie Gouache, MD, Fares bou Mitri, Damien Bonnet, Laurence Olivier Faivre, Philippe Labrune, MD PhD, Marlène Pasquet, MD, PhD, Virginie Gandemer, MD PhD, Nizar Mahlaoui, MD, Karl Balabanian, Francoise Bachelerie, PhD, Christine Bellanne-Chantelot, PhD PharmD
Journal: Blood
Blood (2020) 136 (Supplement 1): 40–41.
https://doi.org/10.1182/blood-2020-135912
Published: 2020
Journal Articles

Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults

Robert Chiesa, Junfeng Wang, Henric-Jan Blok, Sheree Hazelaar, Benedicte Neven, Despina Moshous, Ansgar Schulz, Manfred Hoenig, Fabian Hauck, Amal Al Seraihy, Jolanta Gozdzik, Per Ljungman, Caroline A. Lindemans, Juliana F. Fernandes, Krzysztof Kalwak, Brigitte Strahm, Urs Schanz, Petr Sedlacek, Karl-Walter Sykora, Serap Aksoylar, Franco Locatelli, Polina Stepensky, Robert Wynn, Su Han Lum, Marco Zecca, Fulvio Porta, Mervi Taskinen, Brenda Gibson, Susanne Matthes, Musa Karakukcu, Mathias Hauri-Hohl, Paul Veys, Andrew R. Gennery, Giovanna Lucchini, Matthias Felber, Michael H. Albert, Dmitry Balashov, Arjan Lankester, Tayfun Güngör, Mary A. Slatter
Journal: Blood
Blood (2020) 136 (10): 1201–1211.
https://doi.org/10.1182/blood.2020005590
Published: 2020
Includes: Supplemental data