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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

Impact and Dynamics of TP53 Mutated Clones in Shwachman Diamond Syndrome in a Series of 80 Patients

Jean-Alain Martignoles, Pierre Hirsch, MD PhD, Blandine Beaupain, Thomas Longval, MD, Nawa Hachem, MsC, Mira El-Khoury, PhD, Sophie Kaltenbach, Jean Soulier, MDPhD, Thierry Leblanc, MD, Pierre Simon Rohrlich, MDPhD, Eric Jeziorski, MDPhD, Isabelle Meyts, Dalila Adjaoud, MD, Vincent Barlogis, MD, PhD, Marlène Pasquet, MD, PhD, Laetitia Largeaud, Flore Sicre de Fontbrune, Claire Fieschi, MD PhD, Virginie Gandemer, MD PhD, Nathalie Aladjidi, MD, Fanny Fouyssac, MD, Guy Leverger, MD, Wadih Abou Chahla, MD, Stephane Blanche, MD, Despina Moushous, MD PhD, Aline Moignet Autrel, MD, Yves Bertrand, MD, Cecile Renard, Pascale Flandrin-Gresta, MD, Jean Louis Stephan, MDPhD, Olivier Tournilhac, MD PhD, Liana Carausu, Frederic Millot, MD, Marielaure Couec, MD, Claude Preudhomme, MD PhD, Hélène Lapillonne, MD PhD, Vahid Asnafi, MD PhD, Patrick Revy, Christine Bellanne-Chantelot, PhD PharmD, Francois Delhommeau, MD PhD, Jean Donadieu, MDPhD

Blood (2020) 136 (Supplement 1): 22–23.

https://doi.org/10.1182/blood-2020-138540

Published: 2020

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

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

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

Germline TIM-3 Mutations Characterize Sub-Cutaneous Panniculitis T-Cell Lymphomas with Hemophagocytic Lymphohistiocytic Syndrome

Blood (2018) 132 (Supplement 1): 1569.

https://doi.org/10.1182/blood-2018-99-120297

Published: 2018

Journal Articles

A landscape of germ line mutations in a cohort of inherited bone marrow failure patients

Blood (2018) 131 (7): 717–732.

https://doi.org/10.1182/blood-2017-09-806489

Published: 2018

Includes: Supplemental data

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

Frequency and Evolution of TP53 Mutant Clones in Shwachman Diamond Syndrome. a Cohort Study from the French Severe Chronic Neutropenia (SCN) Registry

Blood (2017) 130 (Supplement 1): 780.

https://doi.org/10.1182/blood.V130.Suppl_1.780.780

Published: 2017

Journal Articles

Hematopoietic stem cell transplantation in 29 patients hemizygous for hypomorphic IKBKG /NEMO mutations

Blood (2017) 130 (12): 1456–1467.

https://doi.org/10.1182/blood-2017-03-771600

Published: 2017

Includes: Supplemental data

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

Heterozygous STAT1 gain-of-function mutations underlie an unexpectedly broad clinical phenotype

Blood (2016) 127 (25): 3154–3164.

https://doi.org/10.1182/blood-2015-11-679902

Published: 2016

Includes: Supplemental data

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

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

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1

Fabien Touzot, Despina Moshous, Rita Creidy, Bénédicte Neven, Pierre Frange, Guilhem Cros, Laure Caccavelli, Johanna Blondeau, Alessandra Magnani, Jean-Marc Luby, Brigitte Ternaux, Capucine Picard, Stéphane Blanche, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana

Blood (2015) 125 (23): 3563–3569.

https://doi.org/10.1182/blood-2014-12-616003

Published: 2015

Includes: Supplemental data

Journal Articles

Lymphoproliferative Disease in Wiskott-Aldrich Syndrome: Analysis of the French National Registry of Primary Immunodeficiencies

Morgane Cheminant, Nizar Mahlaoui, MD MSc, MPH, Danielle Canioni, MD, Celine Desconclois, PD, Olivier Hermine, MD PhD, Marlène Pasquet, MD, Marion Malphettes, MD, Yves Bertrand, Stephane Blanche, MD, Alain Fischer, MD PhD, Felipe Suarez, MD PhD

Blood (2014) 124 (21): 4129.

https://doi.org/10.1182/blood.V124.21.4129.4129

Published: 2014

Journal Articles

Defective anti-polysaccharide response and splenic marginal zone disorganization in ALPS patients

Blood (2014) 124 (10): 1597–1609.

https://doi.org/10.1182/blood-2014-02-553834

Published: 2014

Includes: Supplemental data

Journal Articles

SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID

Blood (2014) 123 (2): 281–289.

https://doi.org/10.1182/blood-2013-01-476432

Published: 2014

Includes: Supplemental data

Journal Articles

A Mendelian predisposition to B-cell lymphoma caused by IL-10R deficiency

Blood (2013) 122 (23): 3713–3722.

https://doi.org/10.1182/blood-2013-06-508267

Published: 2013

Includes: Supplemental data

Journal Articles

Circulating Endothelial Cells As a Reliable Marker Of Endothelial Damage In Children Undergoing Hematopoietic Stem Cell Transplantation

Fabien Touzot, MD, PhD, Despina Moshous, MD, PhD, Guilhem Cros, MD, Pierre Frange, MD, Sebastien Heritier, MD, Benedicte Neven, Marina Cavazzana-Calvo, MD, PhD, Alain Fischer, MD/PhD, Stephane Blanche, MD, Dominique Helley, MD, PhD

Blood (2013) 122 (21): 2049.

https://doi.org/10.1182/blood.V122.21.2049.2049

Published: 2013

NARROW

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

Impact and Dynamics of TP53 Mutated Clones in Shwachman Diamond Syndrome in a Series of 80 Patients

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

Alemtuzumab as First Line Treatment in Children with Familial Lymphohistiocytosis

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

Germline TIM-3 Mutations Characterize Sub-Cutaneous Panniculitis T-Cell Lymphomas with Hemophagocytic Lymphohistiocytic Syndrome

A landscape of germ line mutations in a cohort of inherited bone marrow failure patients

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

Frequency and Evolution of TP53 Mutant Clones in Shwachman Diamond Syndrome. a Cohort Study from the French Severe Chronic Neutropenia (SCN) Registry

Hematopoietic stem cell transplantation in 29 patients hemizygous for hypomorphic IKBKG /NEMO mutations

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

Heterozygous STAT1 gain-of-function mutations underlie an unexpectedly broad clinical phenotype

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)

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

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1

Lymphoproliferative Disease in Wiskott-Aldrich Syndrome: Analysis of the French National Registry of Primary Immunodeficiencies

Defective anti-polysaccharide response and splenic marginal zone disorganization in ALPS patients

SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID

A Mendelian predisposition to B-cell lymphoma caused by IL-10R deficiency

Circulating Endothelial Cells As a Reliable Marker Of Endothelial Damage In Children Undergoing Hematopoietic Stem Cell Transplantation

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American Society of Hematology

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ASH Publications

American Society of Hematology

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