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Journal Articles

Expanding access to haploidentical transplantation : Clinical feasibility of a centralized nationwide distribution model for fresh TCRα/β-depleted grafts in France

Blood (2025) 146 (Supplement 1): 5959.

https://doi.org/10.1182/blood-2025-5959

Published: 2025

Journal Articles

GMP-manufactured allogeneic human t lymphoid progenitors (HTLP) promote early and sustained T-cell recovery after CD34+-selected haploidentical HSCT in SCID patients : Final results of the htlp-SCID study

Blood (2025) 146 (Supplement 1): 271.

https://doi.org/10.1182/blood-2025-271

Published: 2025

Journal Articles

Hematopoietic stem cell transplantation in 746 children with osteopetrosis: A study of the inborn errors working party of the EBMT

Robert Chiesa, Michael H. Albert, Patrick Gilbert, Jeroen Knippenberg, Irina Zaidman, Hawazen Alsaedi, Fulvio Porta, R.F. Wynn, Rakefet Sidlik Muskatel, Serap Aksoylar, Barış Kuşkonmaz, Zohreh Nademi, Sarah Lawson, Akif Yesilipek, Kerstin Felgentreff, Marta Gonzalez Vicent, Juliana Silva, Tatyana Bykova, O. Alphan Kupesiz, Marco Zecca, Maura Faraci, Amir Hamidieh, Mayada Abu Shanap, Etai Adam, Tariq Ghafoor, Musa Karakükcü, Natalia Maximova, Sophie van Lancker, Catherine Paillard, Marleen Renard, Franco Locatelli, Martin Castelle, Persis Amrolia, Srividhya Senthil, Cécile Renard, Mohammed Essa, Benedicte Bruno, Cristina Díaz-de-Heredia, Ingrid Furlan, Renata Formankova, Matthias Felber, Nuno Miranda, Kanchan Rao, Baris Malbora, Jacek Winiarski, Robbert Bredius, Beki James, Jean-Sebastien Diana, Melek Erdem, Amal Alseraihy, Melissa Gabriel, Bernd Gruhn, Tania Masmas, Alessandra Biffi, Katarzyna Drabko, Krzysztof Kalwak, David Bueno, Manfred Hoenig, Carlos Vaz, Katharina Wustrau, Benedicte Brichard, Samppa Ryhänen, Anna Villa, Benedicte Neven, Mehtap Sirin, Despina Moshous, Ansgar Schulz

Blood (2025) 146 (Supplement 1): 165.

https://doi.org/10.1182/blood-2025-165

Published: 2025

Journal Articles

Long-term outcome and risk factors for relapse in pediatric warm autoimmune hemolytic anemia.

Blood (2025) 146 (Supplement 1): 4657.

https://doi.org/10.1182/blood-2025-4657

Published: 2025

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

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

Blood (2023) 142 (Supplement 1): 6868.

https://doi.org/10.1182/blood-2023-187607

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

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

Oral SGLT2 Inhibitors in Glycogen Storage Disease Type Ib and G6PC3-Deficiency. Preliminary Results from an Off-Label Study of 21 Patients

Blood (2022) 140 (Supplement 1): 8320–8322.

https://doi.org/10.1182/blood-2022-169243

Published: 2022

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

HLA-Matched Related Hematopoietic Stem Cell Transplantation in Adolescents and Adults with Sickle Cell Disease: Comparison of Myeloablative Versus Non Myeloablative Approaches. Report from the Société Francophone De Greffe De Moelle Et De Thérapie Cellulaire

Nathalie Dhedin, Florian Chevillon, Martin Castelle, Virginie Lavoipierre, Jean-Hugues Dalle, Loïc Vasseur, Laure Joseph, Florence Beckerich, Nimrod Buchbinder, Tereza Coman, Frederic Garban, Alina Ferster, Nicolas Boissel, Stephanie Nguyen Quoc, Jean-Benoit Arlet, Corinne Pondarré

Blood (2021) 138 (Supplement 1): 909.

https://doi.org/10.1182/blood-2021-147052

Published: 2021

Journal Articles

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Jean-Sebastien Diana, MD, Naim Bouazza, PhD, Chloé Couzin, MD, Martin Castelle, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Joseph, MD, Marianne Delville, MD, Francois Lefrere, MD, jean-Marc Treluyer, MD PhD, Capucine Picard, MD PhD, Despina Moshous, MD PhD, Isabelle André, PhD, Stephane Blanche, MD, Benedicte Neven, MD PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 5668.

https://doi.org/10.1182/blood-2019-127974

Published: 2019

Journal Articles

Alemtuzumab as First Line Treatment in Children with Familial Lymphohistiocytosis

Blood (2019) 134 (Supplement_1): 80.

https://doi.org/10.1182/blood-2019-124477

Published: 2019

Journal Articles

Reiterated Therapeutic Drug Monitoring (TDM) Dosing to Significantly Improve the Control of Exposure to IV Busulfan in Infants and Older Children Undergoing Hematopoietic Stem-Cell Transplantation (HSCT)

Alexandre Amin, PharmD, Philippe Bourget, PharmD, PhD, Benedicte Neven, MD PhD, Marie-Louise Fremond, MD, Martin Castelle, MD, Elodie Elkaim, MD, Guilhem Cros, MD, Despina Moshous, MD, Fabien Touzot, MD, Alexandre Lalli, PharmD, Aurélie Petain, Laurent Nguyen, Stephane Blanche

Blood (2015) 126 (23): 4326.

https://doi.org/10.1182/blood.V126.23.4326.4326

Published: 2015

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Expanding access to haploidentical transplantation : Clinical feasibility of a centralized nationwide distribution model for fresh TCRα/β-depleted grafts in France

GMP-manufactured allogeneic human t lymphoid progenitors (HTLP) promote early and sustained T-cell recovery after CD34+-selected haploidentical HSCT in SCID patients : Final results of the htlp-SCID study

Hematopoietic stem cell transplantation in 746 children with osteopetrosis: A study of the inborn errors working party of the EBMT

Long-term outcome and risk factors for relapse in pediatric warm autoimmune hemolytic anemia.

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

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

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

Oral SGLT2 Inhibitors in Glycogen Storage Disease Type Ib and G6PC3-Deficiency. Preliminary Results from an Off-Label Study of 21 Patients

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

HLA-Matched Related Hematopoietic Stem Cell Transplantation in Adolescents and Adults with Sickle Cell Disease: Comparison of Myeloablative Versus Non Myeloablative Approaches. Report from the Société Francophone De Greffe De Moelle Et De Thérapie Cellulaire

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Alemtuzumab as First Line Treatment in Children with Familial Lymphohistiocytosis

Reiterated Therapeutic Drug Monitoring (TDM) Dosing to Significantly Improve the Control of Exposure to IV Busulfan in Infants and Older Children Undergoing Hematopoietic Stem-Cell Transplantation (HSCT)

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