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Journal Articles

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Haydar Frangoul, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Lyndsay Molinari, Donna Wall, Robert I. Liem, Paul Telfer, Ami J. Shah, Marina Cavazzana, Selim Corbacioglu, Damiano Rondelli, Roland Meisel, Laurence Dedeken, Stephan Lobitz, Mariane de Montalembert, Martin H. Steinberg, Mark C. Walters, Laura Bower, Suzan Imren, Christopher Simard, Fengjuan Xuan, Weiyu Zhou, Phuong Khanh Morrow, William Hobbs, Stephan Grupp
Journal: Blood
Blood (2023) 142 (Supplement 1): 1052.
https://doi.org/10.1182/blood-2023-190139
Published: 2023
Journal Articles

Availability and Cost of Basic Drugs for Sickle Cell Disease in 13 African Countries

Gladys Kelly Keza, Dapa Aly Diallo, Ibrahima Diagne, Mariane de Montalembert, Anne Corbasson, Françoise Bernaudin, Brigitte Ranque, Binta Coulibaly, Galacteros Frederic, Saliou Diop, Constant Vodouhé, Olivier Hermine, Agnès Lainé, Eléonor Kafando, Ahoefa Vovor, Sébastien Jan, Leon Tshilolo, Robert Hue, Jean-Benoit Arlet
Journal: Blood
Blood (2023) 142 (Supplement 1): 1152.
https://doi.org/10.1182/blood-2023-188652
Published: 2023
Journal Articles

Safety Profile of Crizanlizumab 5.0 Mg/Kg in Sickle Cell Disease: Pooled Data from Clinical Trials

Abdullah Kutlar, Vikas Joshi, Andreas Brueckner, Julie Kanter, Darla K. Liles, Jeremie Lincy, Rahul Manjare, Yvonne Dei Adomakoh, Matthew M. Heeney, Mariane de Montalembert, Deborah Keefe, Kwaku Marfo, Kenneth I. Ataga
Journal: Blood
Blood (2023) 142 (Supplement 1): 1139.
https://doi.org/10.1182/blood-2023-188639
Published: 2023
Journal Articles

The Cs a and Cs b Red Cell Antigens of the Cost Blood Group Collection Correspond to the HNA-3a and HNA-3b Neutrophil Antigens: Unexpected Twins with Implications for Sickle Cell Anemia

Jonathan De Oliveira Rios, Alisa Soudry, Romain Duval, Alexandre Raneri, Thomas Poyot, Jérome Babinet, Mariane De Montalembert, Claudia Regina Bonini Domingos, Caroline Le Van Kim, Marc Romana, Thierry Peyrard, Slim Azouzi
Journal: Blood
Blood (2023) 142 (Supplement 1): 698.
https://doi.org/10.1182/blood-2023-178250
Published: 2023
Journal Articles

Efficacy, Safety, and Biomarker Analysis of 5 Mg and 7.5 Mg Doses of Crizanlizumab in Patients with Sickle Cell Disease: Primary Analyses from the Phase III STAND Study

Miguel R Abboud, Rodolfo D Cançado, Mariane De Montalembert, Wally R Smith, Hala Rimawi, Ersi Voskaridou, Birol Guvenc, Deborah Keefe, Kai Grosch, Michele L Nassin, Jimmy Watson, Justin Yssel, Evgeniya Reshetnyak, Yvonne Dei Adomakoh
Journal: Blood
Blood (2023) 142 (Supplement 1): 272.
https://doi.org/10.1182/blood-2023-185429
Published: 2023
Journal Articles

Sickle Cell Health Awareness, Perspectives, and Experiences (SHAPE) Survey: Findings on the Burden of Sickle Cell Disease and Impact on the Quality of Life of Patients and Caregivers in Gulf Cooperation Council Countries

Wasil Jastaniah, Regina Hartfield, John James, Biba Tinga, Elvie Ingoli, Mariane de Montalembert, Fernando F. Costa, Alan Randall Anderson, Joachim B. Kunz, Isaac Odame, Belinda Lartey, Baba P.D. Inusa
Journal: Blood
Blood (2023) 142 (Supplement 1): 2516.
https://doi.org/10.1182/blood-2023-177655
Published: 2023
Journal Articles

Sickle Cell Health Awareness, Perspectives, and Experiences (SHAPE) Survey: Perspectives of Physicians in Gulf Cooperation Council Countries on Sickle Cell Disease

Wasil Jastaniah, Mariane de Montalembert, Fernando F. Costa, Alan Randall Anderson, Joachim B. Kunz, Isaac Odame, Belinda Lartey, Baba P.D. Inusa
Journal: Blood
Blood (2023) 142 (Supplement 1): 5309.
https://doi.org/10.1182/blood-2023-177685
Published: 2023
Journal Articles

Puberty Onset and Preservation of Fertility in Male Patients with Sickle Cell Disease Treated with Hydroxyurea: Data from the European Sickle Cell Disease Cohort - Hydroxyurea Extension (ESCORT-HU Extension) Study

Mariane De Montalembert, Pablo Bartolucci, Gonzalo De Luna, Anoosha Habibi, Scylia Alexis-Fardini, Maryse Etienne-Julan, Gylna Loko, Giovanna Cannas, Jean-Benoit Arlet, Latifatou Boukari, Sylvain Le Jeune, Laure Joseph
Journal: Blood
Blood (2023) 142 (Supplement 1): 145.
https://doi.org/10.1182/blood-2023-178219
Published: 2023
Journal Articles

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease

Haydar Frangoul, Franco Locatelli, Monica Bhatia, Markus Y. Mapara, Lyndsay Molinari, Akshay Sharma, Stephan Lobitz, Mariane de Montalembert, Damiano Rondelli, Martin Steinberg, Mark C. Walters, Suzan Imren, Lanju Zhang, Anjali Sharma, Yang Song, Christopher Simard, William Hobbs, Stephen Grupp
Journal: Blood
Blood (2022) 140 (Supplement 1): 29–31.
https://doi.org/10.1182/blood-2022-162353
Published: 2022
Journal Articles

Hydroxyurea Is Associated with Later Onset of Occurrence of Acute Splenic Sequestration Episodes in Sickle Cell Disease: Lessons from the European Sickle Cell Disease Cohort - Hydroxyurea (ESCORT-HU) Study

Mariane de Montalembert, Frédéric Galactéros, Lena Oevermann, Giovanna Cannas, Laure Joseph, Gylna Loko, Narcisse Elenga, Malika Benkerrou, Maryse Etienne-Julan, Marie-Pierre Castex, Regine Grosse, Valentine Brousse
Journal: Blood
Blood (2022) 140 (Supplement 1): 449–450.
https://doi.org/10.1182/blood-2022-165946
Published: 2022
Journal Articles

Sputum IL-6 Level Is a Potential Predictor and Severity Marker of Acute Chest Syndrome in Sickle Cell Disease

Slimane Allali, Juliette Elie, Mariane de Montalembert, Melissa Taylor, Joséphine Brice, Stéphanie Chhun, Rachel Rignault-Bricard, Claire Heilbronner, Thiago Trovati Maciel, Olivier Hermine
Journal: Blood
Blood (2022) 140 (Supplement 1): 8265.
https://doi.org/10.1182/blood-2022-166823
Published: 2022
Journal Articles

HbS promotes TLR4-mediated monocyte activation and proinflammatory cytokine production in sickle cell disease

Slimane Allali, Rachel Rignault-Bricard, Mariane de Montalembert, Melissa Taylor, Tahar Bouceba, Olivier Hermine, Thiago Trovati Maciel
Journal: Blood
Blood (2022) 140 (18): 1972–1982.
https://doi.org/10.1182/blood.2021014894
Published: 2022
Includes: Supplemental data
Journal Articles

Limited Access to Transcranial Doppler Screening and Stroke Prevention for Children with Sickle Cell Disease in Europe: Results of a Multinational Eurobloodnet Survey

Daniela Cuzzubbo, Victoria Gutierrez-Valle, Maddalena Casale, Vincenzo Voi, Corrina McMahon, Maria Del Mar Mañú Pereira, Mariane de Montalembert, Baba PD Inusa, Raffaella Colombatti
Journal: Blood
Blood (2021) 138 (Supplement 1): 915.
https://doi.org/10.1182/blood-2021-147298
Published: 2021
Journal Articles

Implementation of Escort-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study : Interests and Methodology

Mariane de Montalembert, Ersi Voskaridou, Lena Oevermann, Giovanna Cannas, Anoosha Habibi, Gylna Loko, Laure Joseph, Raffaella Colombatti, Pablo Bartolucci, Valentine Brousse, Frédéric Galactéros
Journal: Blood
Blood (2021) 138 (Supplement 1): 3098.
https://doi.org/10.1182/blood-2021-152525
Published: 2021
Journal Articles

Experiences of Sickle Cell Disease (SCD) Reported By Healthcare Professionals (HCPs) across Different Regions: International Sickle Cell World Assessment Survey (SWAY)

Ifeyinwa Osunkwo, Biree Andemariam, Caterina P Minniti, Fuad El Rassi, Erfan Nur, Alecia C Nero, Raffaella Colombatti, Mariane de Montalembert, Miguel R Abboud, Jean-Benoît Arlet, Suman Jain, Wasil Jastaniah, Marimilia Pita, Beverley Francis-Gibson, Cassandra Trimnell, Laurie DeBonnett, Tom Bailey, Olivera Rajkovic-Hooley, Baba PD Inusa, John James
Journal: Blood
Blood (2021) 138 (Supplement 1): 3026.
https://doi.org/10.1182/blood-2021-145409
Published: 2021
Journal Articles

ERN-EuroBloodNet European Registry of Patients Affected by Red Blood Cell Disorders and COVID-19

Pablo Velasco, Filomena Longo, Andrea Piolatto, Eduardo J. Bardón-Cancho, Beatriz Ponce-Salas, Pagona Flevari, Ersi Voskaridou, Bart J. Biemond, Erfan Nur, Polyxeni Delaporta, Tatiana Besse-Hammer, Anna Ruiz-Llobet, Simona Raso, Anna Spasiano, Maria Elena Guerzoni, David Beneitez-Pastor, Laurence Dedeken, Alessia Pepe, Rosamaria Rosso, Joachim B. Kunz, Mariane de Montalembert, Saveria Campisi, Andreas Glenthøj, Paula Gonzalez Urdiales, Fleur Samantha Benghiat, Marie-Agnès Azerad, Christopher J. Saunders, Teresa Ferreira Faria, Tommaso Casini, Sabrina Bagnato, Ann Van de Velde, Veerle Labarque, Elisa Bertoni, An Van Damme, Michael D. Diamantidis, Roberta Russo, Eftichia Stiakaki, Alessandra Quota, Soteroula Christou, Maria Jose Teles, Ioannis Lafiatis, Jean-Louis Kerkhoffs, María Argüello Marina, Mikael Lorite, Alexis Rodriguez, Achille Iolascon, Ali T. Taher, Raffaella Colombatti, Noemi Roy, Maria Del Mar Mañú Pereira
Journal: Blood
Blood (2021) 138 (Supplement 1): 4058.
https://doi.org/10.1182/blood-2021-147236
Published: 2021
Journal Articles

Initial Safety and Efficacy Results from the Phase II, Multicenter, Open-Label Solace-Kids Trial of Crizanlizumab in Adolescents with Sickle Cell Disease (SCD)

Matthew M. Heeney, David C. Rees, Mariane De Montalembert, Isaac Odame, R. Clark Clark Brown, Yasser Wali, Thu Thuy Nguyen, Du Lam, Nadege Pfender, Julie Kanter
Journal: Blood
Blood (2021) 138 (Supplement 1): 12.
https://doi.org/10.1182/blood-2021-144730
Published: 2021
Journal Articles

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

Clinical Trials & Observations
Anne-Sophie Gille, Corinne Pondarré, Jean-Hugues Dalle, Françoise Bernaudin, Céline Chalas, Mony Fahd, Camille Jean, Harry Lezeau, Lydia Riou, Véronique Drouineaud, Annabel Paye-Jaouen, Annie Kamdem, Bénédicte Neven, Cécile Arnaud, Saba Azarnoush, Karima Yakouben, Sabine Sarnacki, Mariane de Montalembert, Eva Maria Comperat, Gilles Lenaour, Mathilde Sibony, Nathalie Dhédin, Daniel Vaiman, Jean-Philippe Wolf, Catherine Patrat, Pierre Fouchet, Catherine Poirot, Virginie Barraud-Lange
Journal: Blood
Blood (2021) 137 (6): 856–859.
https://doi.org/10.1182/blood.2020008146
Published: 2021
Includes: Supplemental data
Journal Articles

Global Treatment Satisfaction Levels and Treatment Patterns from the International Sickle Cell World Assessment Survey (SWAY): Hydroxyurea (HU) Versus No HU

Fuad A El Rassi, MD, John James, MSc, Biree Andemariam, MD, Baba PD Inusa, MDMBBS, Beverley Francis-Gibson, MA, Alecia C Nero, MD, Caterina P Minniti, MD, Cassandra Trimnell, BA, Miguel R Abboud, MD, Jean-Benoît Arlet, MD, Raffaella Colombatti, MDPhD, Mariane de Montalembert, MD PhD, Suman Jain, MD, Wasil Jastaniah, MD, Erfan Nur, MD PhD, Marimilia Pita, MD, Nicholas Ramscar, MBBS, Tom Bailey, MSci, Olivera Rajkovic-Hooley, PhD, Ifeyinwa Osunkwo, MD MPH
Journal: Blood
Blood (2020) 136 (Supplement 1): 8–10.
https://doi.org/10.1182/blood-2020-137103
Published: 2020
Journal Articles

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Haydar Frangoul, MD, Yael Bobruff, PhD, Maria Domenica Cappellini, Selim Corbacioglu, MD, Christine Marie Fernandez, RN,MSN, Josu de la Fuente, Stephan A Grupp, MD PhD, Rupert Handgretinger, MD, Tony W. Ho, MD, Suzan Imren, MD, Antonis Kattamis, Julie Lekstrom-Himes, MD, Franco Locatelli, MD PhD, Yimeng Lu, PhD, Markus Y Mapara, MD PhD, Mariane de Montalembert, MD PhD, Sarah D. Mulcahey, MS, Damiano Rondelli, MD, Niraj Shanbhag, MD PhD, Sujit Sheth, MD, Sandeep Soni, MBBS, Martin H. Steinberg, MD, Michael J. Weinstein, John K. Wu, MBBS, FRCPC, MS, Donna Wall, MDCCPE
Journal: Blood
Blood (2020) 136 (Supplement 1): 3–4.
https://doi.org/10.1182/blood-2020-139575
Published: 2020