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Journal Articles

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

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

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

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

Blood (2023) 142 (Supplement 1): 4997.

https://doi.org/10.1182/blood-2023-179916

Published: 2023

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 )

Blood (2022) 140 (Supplement 1): 2383–2385.

https://doi.org/10.1182/blood-2022-165229

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 )

Blood (2022) 140 (Supplement 1): 10462–10464.

https://doi.org/10.1182/blood-2022-170552

Published: 2022

Journal Articles

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

Blood (2022) 140 (Supplement 1): 4899–4901.

https://doi.org/10.1182/blood-2022-166881

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

Blood (2022) 140 (Supplement 1): 7613–7615.

https://doi.org/10.1182/blood-2022-162825

Published: 2022

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

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

Blood (2021) 138 (Supplement 1): 2906.

https://doi.org/10.1182/blood-2021-153519

Published: 2021

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

Blood (2021) 138 (Supplement 1): 424.

https://doi.org/10.1182/blood-2021-147809

Published: 2021

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

Blood (2020) 136 (Supplement 1): 45–47.

https://doi.org/10.1182/blood-2020-142397

Published: 2020

Journal Articles

Single-Cell Transcriptional Landscapes of Human Bone Marrow Reveal Distinct Erythroid Phenotypes Underpinned By Genotype in Diamond-Blackfan Anemia

Blood (2020) 136 (Supplement 1): 1–2.

https://doi.org/10.1182/blood-2020-141535

Published: 2020

Journal Articles

Upfront Alternative Donor Transplant Versus Immunosuppressive Therapy in Patients with Severe Aplastic Anemia Who Lack Fully HLA Matched Related Donor: Systematic Review and Meta-Analysis of Retrospective Studies. on Behalf of the Severe Aplastic Anemia Working Party of European Group for Blood and Marrow Transplantation (SAAWP of EBMT)

Hind Alotaibi, MBBS, Mahmoud Aljurf, Regis Peffault De Latour, MD PhD, Shahid Iqbal, Andrea Bacigalupo, Judith Marsh, Hubert Schrezenmeier, Eliane Gluckman, Mansour Alfayez, Britta Hoechsmann, Constantijn Halkes, Josu de la Fuente, Nawal AlShehry, Simone Cesaro, Jakob Passweg, Carlo Dufour, Antonio Risitano, John F. DiPersio, MD PhD, Ibraheem Motabi

Blood (2020) 136 (Supplement 1): 6–7.

https://doi.org/10.1182/blood-2020-140846

Published: 2020

Journal Articles

Increased Incidence of New-Onset Diabetes Mellitus Type II Following Haploidentical Bone Marrow Transplant with Post-Transplant Cyclophosphamide for Sickle Cell Disease

Dilan A Patel, MD, Josu de la Fuente, Michael Matheney, MD, Leena Karnik, FRCPath, MRCP, Heidi Chen, PhD, Katie S. Gatwood, PharmD, Kathryn A. Culos, PharmD, Karina L Wilkerson, NP, MSN, Michael T. Byrne, DO, Salyka M. Sengsayadeth, MD, Stacey Ann Goodman, MD, Bhagirathbhai Dholaria, MBBS, Bipin N. Savani, MD, Wichai Chinratanalab, MD, Reena Jayani, MD, Jim Connelly, MD, Carrie Kitko, MD, Brian G. Engelhardt, MD, Adetola A. Kassim, MDMS

Blood (2020) 136 (Supplement 1): 20.

https://doi.org/10.1182/blood-2020-143039

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

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-139575

Published: 2020

Journal Articles

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

Adetola A. Kassim, MDMS, Josu de la Fuente, Ali Debsan Alahmari, MBBS, MD MNAMS, Michael J. Eckrich, MD MPH, Rabi Hanna, MD, Nathalie Dhedin, MD, Biljana N. Horn, MD, Vandy Black, MDMSc, Roseane V Gouveia, Adriana Seber, MDMS, Belinda Pinto Simões, MD PhD, Thalita Cristina de Mello Costa, MD, Renato Guerino-Cunha, Carmem M. S. Bonfim, MDPhD, Roberto Luiz da Silva, Nelson Hamerschlak, MD PhD, Juliana Folloni Fernandes, MD, Michael Kent, MDBS, Satya Prakash Yadav, MBBS, MD, Erfan Nur, MDPhD, Mohsen Alzahrani, Ben Carpenter, PhD, William Tse, MD, Maria Isabel, Hemalatha G Rangarajan, MD, Ibrahim Ahmed, MD, Adeseye Michael Akinsete, MBBS, Courtney D. Fitzhugh, MD, Kathryn A. Culos, PharmD, Leena Karnik, FRCPath, MRCP, James Connelly, MD, Carrie Kitko, MD, Jennifer Andrews, MDMSc, Karina L Wilkerson, NP, MSN, Robert Brodsky, Tatsuki Koyama, Michael R. DeBaun, MD MPH

Blood (2020) 136 (Supplement 1): 8–9.

https://doi.org/10.1182/blood-2020-141771

Published: 2020

Journal Articles

Single-cell analysis of bone marrow–derived CD34 + cells from children with sickle cell disease and thalassemia

Peng Hua, Noemi Roy, Josu de la Fuente, Guanlin Wang, Supat Thongjuea, Kevin Clark, Anindita Roy, Bethan Psaila, Neil Ashley, Yvonne Harrington, Claus Nerlov, Suzanne M. Watt, Irene Roberts, James O. J. Davies

Blood (2019) 134 (23): 2111–2115.

https://doi.org/10.1182/blood.2019002301

Published: 2019

Includes: Supplemental data

Journal Articles

Stem Cell Transplantation for Diamond-Blackfan Anemia. a Retrospective Study on Behalf of Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)

Blood (2019) 134 (Supplement_1): 44.

https://doi.org/10.1182/blood-2019-124644

Published: 2019

Journal Articles

Results of Unrelated Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease in Europe on Behalf of Paediatric Diseases (PDWP) and Inborn Errors Working Parties (IEWP) of the EBMT

Blood (2019) 134 (Supplement_1): 4583.

https://doi.org/10.1182/blood-2019-121952

Published: 2019

NARROW

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

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

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

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

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 )

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 )

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

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

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

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

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

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

Single-Cell Transcriptional Landscapes of Human Bone Marrow Reveal Distinct Erythroid Phenotypes Underpinned By Genotype in Diamond-Blackfan Anemia

Increased Incidence of New-Onset Diabetes Mellitus Type II Following Haploidentical Bone Marrow Transplant with Post-Transplant Cyclophosphamide for Sickle Cell Disease

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

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

Single-cell analysis of bone marrow–derived CD34 + cells from children with sickle cell disease and thalassemia

Stem Cell Transplantation for Diamond-Blackfan Anemia. a Retrospective Study on Behalf of Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)

Results of Unrelated Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease in Europe on Behalf of Paediatric Diseases (PDWP) and Inborn Errors Working Parties (IEWP) of the EBMT

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American Society of Hematology

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American Society of Hematology

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