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Journal Articles

Long term follow-up of the STOPAGO study

Blood blood.2024025707.

https://doi.org/10.1182/blood.2024025707

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

Efficacy and Safety of Dapsone in Primary Immune Thrombocytopenia. Results of a Randomized, Placebo-Controlled Multicenter Trial and of a Single-Arm, Emulated Trial in the Prospective, Multicenter Carmen-France Registry

Marion Larue, Guillaume Moulis, Manuela Rueter, Sylvain Audia, Louis Terriou, Jean-Francois Viallard, Brigitte Pan Petesch, Bruno Royer, Bernard Bonnotte, Lionel Galicier, Olivier Lambotte, Francois Lefrere, Stephane Cheze, Mikael Ebbo, Orianne Wagner-Ballon, Matthieu Mahevas, Bertrand Godeau, Marc Michel

Blood (2023) 142 (Supplement 1): 3983.

https://doi.org/10.1182/blood-2023-181606

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

Blood (2023) 142 (Supplement 1): 3633.

https://doi.org/10.1182/blood-2023-186133

Published: 2023

Journal Articles

Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study

Blood (2023) 141 (23): 2867–2877.

https://doi.org/10.1182/blood.2022018665

Published: 2023

Includes: Supplemental data

Journal Articles

Rate of Prolonged Response after Stopping Thrombopoietin-Receptor Agonists Treatment in Primary Immune Thrombocytopenia (ITP): Results from a Nationwide Prospective Multicenter Interventional Study (STOPAGO)

Blood (2021) 138 (Supplement 1): 583.

https://doi.org/10.1182/blood-2021-152767

Published: 2021

Journal Articles

Therapeutic Plasma Exchange in Pediatrics for Immunologic Disorders; Tolerated and Safe Process for Pediatric Life-Threatening Conditions

Blood (2019) 134 (Supplement_1): 4986.

https://doi.org/10.1182/blood-2019-122106

Published: 2019

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

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

Blood (2019) 134 (Supplement_1): 3358.

https://doi.org/10.1182/blood-2019-127393

Published: 2019

Journal Articles

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Jean-Sebastien Diana, MD, Sandra Manceau, PhD, Thierry Leblanc, MD, Chloé Couzin, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Matthieux Bendavid, MD, Laure Joseph, MD, Marianne Delville, MD, Stephane Blanche, MD, Jean Soulier, MD PhD, Marina Cavazzana, MD PhD, Francois Lefrere, MD

Blood (2019) 134 (Supplement_1): 1970.

https://doi.org/10.1182/blood-2019-128051

Published: 2019

Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Blood (2019) 134 (Supplement_1): 4627.

https://doi.org/10.1182/blood-2019-129686

Published: 2019

Journal Articles

The Impact of Cytogenetic Clonality on Outcomes in Younger Acute Myeloid Leukemia Patients: Results on 1290 Patients Included in the EORTC/Gimema AML-10 and AML-12 Trials

Blood (2017) 130 (Supplement 1): 1385.

https://doi.org/10.1182/blood.V130.Suppl_1.1385.1385

Published: 2017

Journal Articles

Impact of the Type of Anthracycline and of Allogeneic Stem Cell Transplantation in the Treatment of Younger Acute Myeloid Leukemia Patients: Long-Term Follow-up of the EORTC-Gimema AML-10 Trial

Blood (2017) 130 (Supplement 1): 3850.

https://doi.org/10.1182/blood.V130.Suppl_1.3850.3850

Published: 2017

Journal Articles

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

Blood (2017) 130 (Supplement 1): 4609.

https://doi.org/10.1182/blood.V130.Suppl_1.4609.4609

Published: 2017

Journal Articles

Impact of Induction Regimen and of Allogeneic Hematopoietic Cell Transplantation on the Outcome in Younger Adults Patients with Acute Myeloid Leukemia with a Monosomal Karyotype: Results from the EORTC/Gimema AML-10 and AML-12 Trials

Frederic Baron, MD PhD, Marian Stevens-Kroef, PhD, Giovanna Meloni, MD, Petra Muus, MD PhD, Jean-Pierre Marie, MD, Constantijn J.M. Halkes, MD PhD, Xavier Thomas, MD PhD, Radovan Vrhovac, MD PhD, Giorgina Specchia, MD, Francois Lefrere, Sr., MD, Simona Sica, Marco Mancini, MD, Adriano Venditti, MD, Anne Hagemeijer, MD, Joop H. Jansen, PhD, Sergio Amadori, MD, Theo de Witte, MD, Roelof Willemze, MD PhD, Stefan Suciu, PhD

Blood (2016) 128 (22): 2847.

https://doi.org/10.1182/blood.V128.22.2847.2847

Published: 2016

Journal Articles

Efficacy and Safety of Erythropoietic-Stimulating Agents with Ruxolitinib in Myelofibrosis Patients : A Retrospective Analysis on 45 Patients. on Behalf of the French Intergroup of Myeloproliferative Disorders (FIM)

Sandra Malak, MD, Pascale Cony-Makhoul, MD, Jean-Christophe Ianotto, MD, Dana Ranta, MD, Philippe Rodon, MD, Fabienne Vacheret, MD, Barraco Fiorenza, MD, Francoise Boyer, MD, Katell Le Du, MD, Loic Garcon, MD PhD, Francois Lefrere, MD, Mathieu Wemeau, MD, Valerie Ugo, MD PhD, Jean-Jacques Kiladjian, MD PhD, Lydia Roy, MD

Blood (2016) 128 (22): 3123.

https://doi.org/10.1182/blood.V128.22.3123.3123

Published: 2016

Journal Articles

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Blood (2016) 128 (22): 2311.

https://doi.org/10.1182/blood.V128.22.2311.2311

Published: 2016

Journal Articles

Arterio-Venous Fistula Is an Applicable Vascular Access for Erythracytapheresis in Patients with Sickle Cell Disease

Blood (2016) 128 (22): 4856.

https://doi.org/10.1182/blood.V128.22.4856.4856

Published: 2016

Journal Articles

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Blood (2015) 126 (23): 202.

https://doi.org/10.1182/blood.V126.23.202.202

Published: 2015

Journal Articles

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

Blood (2014) 124 (21): 4797.

https://doi.org/10.1182/blood.V124.21.4797.4797

Published: 2014

NARROW

Long term follow-up of the STOPAGO study

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

Efficacy and Safety of Dapsone in Primary Immune Thrombocytopenia. Results of a Randomized, Placebo-Controlled Multicenter Trial and of a Single-Arm, Emulated Trial in the Prospective, Multicenter Carmen-France Registry

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

Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study

Rate of Prolonged Response after Stopping Thrombopoietin-Receptor Agonists Treatment in Primary Immune Thrombocytopenia (ITP): Results from a Nationwide Prospective Multicenter Interventional Study (STOPAGO)

Therapeutic Plasma Exchange in Pediatrics for Immunologic Disorders; Tolerated and Safe Process for Pediatric Life-Threatening Conditions

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

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

The Impact of Cytogenetic Clonality on Outcomes in Younger Acute Myeloid Leukemia Patients: Results on 1290 Patients Included in the EORTC/Gimema AML-10 and AML-12 Trials

Impact of the Type of Anthracycline and of Allogeneic Stem Cell Transplantation in the Treatment of Younger Acute Myeloid Leukemia Patients: Long-Term Follow-up of the EORTC-Gimema AML-10 Trial

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

Impact of Induction Regimen and of Allogeneic Hematopoietic Cell Transplantation on the Outcome in Younger Adults Patients with Acute Myeloid Leukemia with a Monosomal Karyotype: Results from the EORTC/Gimema AML-10 and AML-12 Trials

Efficacy and Safety of Erythropoietic-Stimulating Agents with Ruxolitinib in Myelofibrosis Patients : A Retrospective Analysis on 45 Patients. on Behalf of the French Intergroup of Myeloproliferative Disorders (FIM)

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Arterio-Venous Fistula Is an Applicable Vascular Access for Erythracytapheresis in Patients with Sickle Cell Disease

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

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American Society of Hematology

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American Society of Hematology

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