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Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Roland Meisel, Donna Wall, Selim Corbacioglu, Amanda M Li, Josu de la Fuente, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Y Mapara, Robert I. Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan A. Grupp, Hayley Lynne Merkeley, Kevin H.M. Kuo, Joachim Rupprecht, Puja Kohli, Gang Xu, Leorah Ross, Yael Bobruff, Bo Tong, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2024) 144 (Supplement 1): 512.
https://doi.org/10.1182/blood-2024-205779
Published: 2024
Journal Articles

Age Is a Crucial Determinant of GFRS with Incidence of Severe Chronic GVHD Reducing over Time in Haemopoietic Cell Transplantation for Transfusion Dependent Thalassaemia: Real World Data from 2010-2021. an Analysis of the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Working Party

Donatella Baronciani, Josu de la Fuente, Jacques-Emanuel Galimard, Arnaud Dalissier, Ashrafsadat Mousavi, Akif Yesilipek, Hasan Hashem, Gulyuz Ozturk, Franco Locatelli, Ali Alahmari, Javid Gaziev, Ali Bülent Antmen, O. Alphan Kupesiz, Tunc Fisgin, Giorgio La Nasa, Arjan C. Lankester, Eugene Goussetis, Sandrine Bremathas, Emanuele Angelucci
Journal: Blood
Blood (2024) 144 (Supplement 1): 2136.
https://doi.org/10.1182/blood-2024-208421
Published: 2024
Journal Articles

Abatacept Abrogates the Risk of Acute GvHD in Both Matched and Mismatched Transplantation for Haemoglobinopathies and Bone Marrow Failure

Daniel Dexter, Farah O'Boyle, Kirstin Lund, Toni Petterson, Adam Gassas, Leena Karnik, Josu de la Fuente, Sandrine Bremathas, Kelly Hennessy
Journal: Blood
Blood (2024) 144 (Supplement 1): 3518.
https://doi.org/10.1182/blood-2024-208717
Published: 2024
Journal Articles

Donor Trait Status Determines Haematological Outcomes Post-Haemopoietic Stem Cell Transplantation in Transfusion Dependent Thalassaemia Independent of HLA Matching and Chimerism Status

Kinza Khan, Farah O'Boyle, Kirstin Lund, Toni Petterson, Sandrine Bremathas, Kelly Hennessy, Adam Gassas, Leena Karnik, Josu de la Fuente
Journal: Blood
Blood (2024) 144 (Supplement 1): 1097.
https://doi.org/10.1182/blood-2024-211100
Published: 2024
Journal Articles

Allogeneic Transplantation for Sickle Cell Disease Offers High Rates of Cure with Low Incidence of Severe Chronic GVHD in Both Children and Adults: Real World Data from 2010-2021. An Analysis of the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Working Party

Josu de la Fuente, Jacques-Emanuel Galimard, Arnaud Dalissier, Mohsen Al Zahrani, Ali Alahmari, Fahad Almohareb, Jean-Hugues Dalle, Nathalie Dhédin, Hakan Ozdogu, Mohamed Salaheldin Bayoumy, Marco Zecca, Cristina Belendez, Javid Gaziev, Arjan C. Lankester, Philippe Lewalle, Sr., Gaurav Kharya, Sarah Lawson, Bénédicte Neven, Sandrine Bremathas, Emanuele Angelucci
Journal: Blood
Blood (2024) 144 (Supplement 1): 176.
https://doi.org/10.1182/blood-2024-207944
Published: 2024
Journal Articles

Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia

Josu de la Fuente, Franco Locatelli, Peter Lang, Roland Meisel, Donna Wall, Selim Corbacioglu, Amanda M Li, Ami J Shah, Markus Y Mapara, Robert I. Liem, Hayley Lynne Merkeley, Kevin H.M. Kuo, Joachim Rupprecht, Puja Kohli, Nanxin Li, Jaime Rubin, Siyu Zhang, Tina Liu, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2024) 144 (Supplement 1): 7454.
https://doi.org/10.1182/blood-2024-204115
Published: 2024
Journal Articles

Significant Growth in HCT Use for Haemoglobinopathies in the Last Decade Driven By Sickle Cell Disease across All Ages with Excellent Outcomes: An Analysis of EBMT Haemoglobinopathies Working Party

Josu de la Fuente, Jacques-Emanuel Galimard, Arnaud Dalissier, Mohsen Al Zahrani, Ali Alahmari, Ashrafsadat Mousavi, Javid Gaziev, Akif Yesilipek, Khalid Halahleh, Franco Locatelli, Gulyuz Ozturk, Bulent Antmen, Mahmoud Aljurf, Jean-Hugues Dalle, Arjan C. Lankester, Marco Zecca, O. Alphan Kupesiz, Sandrine Bremathas, Selim Corbacioglu, Emanuele Angelucci
Journal: Blood
Blood (2024) 144 (Supplement 1): 2184.
https://doi.org/10.1182/blood-2024-208191
Published: 2024
Journal Articles

An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease

CME Clinical Trials & Observations
Adetola A. Kassim, Josu de la Fuente, Erfan Nur, Karina L. Wilkerson, Ali D. Alahmari, Adriana Seber, Carmem Bonfim, Belinda Pinto Simões, Mohsen Alzahrani, Michael J. Eckrich, Biljana Horn, Rabi Hanna, Nathalie Dhedin, Hemalatha G. Rangarajan, Roseane Vasconcelos Gouveia, Fahad Almohareb, Mahmoud Aljurf, Mohammed Essa, Bader Alahmari, Katie Gatwood, James A. Connelly, Elisabeth Dovern, Mark Rodeghier, Michael R. DeBaun
Journal: Blood
Blood (2024) 143 (25): 2654–2665.
https://doi.org/10.1182/blood.2023023301
Published: 2024
Includes: Supplemental data
Journal Articles

Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507

Adetola A. Kassim, Mark C. Walters, Mary Eapen, Nicole Ritzau, Madoc Smith, Melhem M. Solh, Christopher McKinney, Michael Nieder, Maureen Ross, Michael Kent, Ghada Abusin, Kanwaldeep K. Mallhi, Jorge Galvez Silva, Paul Shaughnessy, Julie Kanter, Hilary Haines, Rafic J Farah, Yasser Khaled, Allistair Abraham, Catherine M. Bollard, Kenneth R. Cooke, Josu de La Fuente, Rabi Hanna, Mary M. Horowitz, Lori C Jordan, Lakshmanan Krishnamurti, Eric Leifere, Kris Michael Mahadeo, Shalini Shenoy, Nicole M. Ritzau, Michael R. DeBaun, Robert A. Brodsky
Journal: Blood
Blood (2023) 142 (Supplement 2): LBA-4.
https://doi.org/10.1182/blood-2023-192022
Published: 2023
Journal Articles

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Selim Corbacioglu, Donna Wall, Roland Meisel, Amanda M Li, Josu de La Fuente, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Mapara, Robert I. Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan Grupp, Puja Kohli, Daoyuan Shi, Leorah Ross, Yael Bobruff, Christopher Simard, Lanju Zhang, Phuong Khanh Morrow, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2023) 142 (Supplement 1): 1053.
https://doi.org/10.1182/blood-2023-190534
Published: 2023
Journal Articles

Improvements in Health-Related Quality of Life after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia

Franco Locatelli, Peter Lang, Selim Corbacioglu, Donna Wall, Amanda M Li, Josu de La Fuente, Puja Kohli, Nanxin Li, Jaime Rubin, Siyu Zhang, Lanju Zhang, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2023) 142 (Supplement 1): 4997.
https://doi.org/10.1182/blood-2023-179916
Published: 2023
Journal Articles

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Amanda Li, Selim Corbacioglu, Josu de la Fuente, Donna A. Wall, Robert Liem, Roland Meisel, Markus Y. Mapara, Ami J. Shah, Maria Domenica Domenica Cappellini, Antonis Kattamis, Sujit Sheth, Yael Bobruff, Laura Bower, Lanju Zhang, Anjali Sharma, Yang Song, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2022) 140 (Supplement 1): 4899–4901.
https://doi.org/10.1182/blood-2022-166881
Published: 2022
Journal Articles

Early Donor T-Cell Engraftment Is Critical for Immune Tolerance in Reduced Intensity Haploidentical Bone Marrow Transplant with Post-Transplant Cyclophosphamide for Sickle Cell Disease: Vanderbilt Global Haploidentical Transplant Learning Collaborative (VGC 2 )

Jim A. Connelly, Eden Biltibo, Karina Wilkerson, Adriana Seber, Roseane V Gouveia, Carmem Bonfim, Belinda Pinto Simões, Erfan Nur, Biljana N. Horn, Michael Eckrich, Rabi Hanna, Nathalie Dhedin, Fahed Almhareb, Mahmoud Aljurf, Hemalatha G. Rangarajan, Katie S. Gatwood, Lindsay Orton, Carrie Lynn Kitko, Mohsen Alzahrani, Ali Debsan Alahmari, Josu De La Fuente, Adetola A. Kassim
Journal: Blood
Blood (2022) 140 (Supplement 1): 10462–10464.
https://doi.org/10.1182/blood-2022-170552
Published: 2022
Journal Articles

Outcomes of Non-Myeloablative HLA-Haploidentical Bone Marrow Transplant with Thiotepa and Post-Transplant Cyclophosphamide in Children and Adults with Sickle Cell Disease, a Phase II Trial: Vanderbilt Global Haploidentical Transplant Learning Collaborative (VGC 2 )

Adetola A. Kassim, Karina Wilkerson, Josu de la Fuente, Adriana Seber, Roseane V Gouveia, Carmem Bonfim, Belinda Pinto Simões, Erfan Nur, Biljana N. Horn, Michael Eckrich, Rabi Hanna, Nathalie Dhedin, Fahed Almhareb, Mahmoud Aljurf, Hemalatha G. Rangarajan, Katie S. Gatwood, James P Connelly, Mohsen Alzahrani, Ali Alahmari, Mark Rodeghier, Michael R. DeBaun
Journal: Blood
Blood (2022) 140 (Supplement 1): 2383–2385.
https://doi.org/10.1182/blood-2022-165229
Published: 2022
Journal Articles

Effective Desensitization Strategy for Patients with Sickle Cell Disease with High Donor Specific Antibodies Undergoing Haploidentical Bone Marrow Transplant with Posttransplant Cyclophosphamide

Eden Biltibo, Reena Jayani, Leena Karnik, Adam Gassas, Farah O'Boyle, Daniel Dexter, Karina Wilkerson, Katie S. Gatwood, Lindsay Orton, Bhagirathbhai Dholaria, Bipin N. Savani, Brian G. Engelhardt, Salyka M. Sengsayadeth, Carrie Lynn Kitko, James P Connelly, Adetola A. Kassim, Josu de la Fuente
Journal: Blood
Blood (2022) 140 (Supplement 1): 7613–7615.
https://doi.org/10.1182/blood-2022-162825
Published: 2022
Journal Articles

Haploidentical Allogeneic Stem Cell Transplantation in Sickle Cell Disease: A Systematic Review and Meta-Analysis

Mesire Aydin, Elisabeth Dovern, Mariska M.G. Leeflang, Josu de la Fuente, Adetola A. Kassim, Bart J. Biemond, Erfan Nur
Journal: Blood
Blood (2021) 138 (Supplement 1): 424.
https://doi.org/10.1182/blood-2021-147809
Published: 2021
Journal Articles

Nonmyeloablative Transplant for Sickle Cell Disease Does Not Lead to Kidney Dysfunction Post-HSCT

Emily Limerick, Santosh L. Saraf, Neal Jeffries, Farah O'Boyle, Clarissa Diamantidis, Yuliya Lokhnygina, Matthew M. Hsieh, Josu de la Fuente, Courtney D. Fitzhugh
Journal: Blood
Blood (2021) 138 (Supplement 1): 984.
https://doi.org/10.1182/blood-2021-153230
Published: 2021
Journal Articles

Complex Clonal Evolution Can Occur Following Transplantation for Transfusion Dependent Thalassaemia in the Context of Mixed Myeloid Chimerism and Reduced Conditioning Regimens

Adam Gassas, Farah O'Boyle, Shahzya Chaudhury, Toni Petterson, Kirstin Lund, Leena Karnik, Josu de la Fuente
Journal: Blood
Blood (2021) 138 (Supplement 1): 2906.
https://doi.org/10.1182/blood-2021-153519
Published: 2021
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
Journal Articles

Transplantation for Congenital Sideroblastic Anaemia Is Feasible and Offers Outcomes Comparable to Other Transfusion Dependent Anaemias. a Joint Retrospective Study of the Paediatric Diseases and Severe Aplastic Anaemia Working Parties (PDWP/SAAWP) of EBMT

Josu de la Fuente, Dirk-Jan Eikema, Paul Bosman, Robert F Wynn, MRCP,MDFRCPath, Miguel Díaz, Peter J Shaw, MD, Amal Al-Seraihy, MD, Muhlis Cem Ar, MD PhD, Mohamed Salaheldin Mohamed, Dominique Bron, MDPhD, Cristina Díaz de Heredia, MD PhD, Tobias Gedde-Dahl, Brenda Gibson, MD PhD, Rupert Handgretinger, MD, Professor John G. Gribben, MD DSc, Tessa Kerre, MD, Vassiliki Kitra-Roussou, MD, Stig Lenhoff, Jose Maria Moraleda, MD PhD, Antonio Perez Martinez, MD PhD, John Moppett, FRCPath, Henrik Sengeloev, Abdelghani Tbakhi, MD, Hendrik Veelken, MD PhD, Estelle Verburgh, MBChB, M MDInt, FCPSA, PhD, Giuseppe Visani, Antonio Risitano, Carlo Dufour, Selim Corbacioglu, MD, Regis Peffault De Latour, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 45–47.
https://doi.org/10.1182/blood-2020-142397
Published: 2020