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Journal Articles

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Richard J. Jones, Michael R. DeBaun

Blood (2021) 138 (11): 942–947.

https://doi.org/10.1182/blood.2021011488

Published: 2021

Journal Articles

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Lauri M. Burroughs, Aleksandra Petrovic, Ruta Brazauskas, Xuerong Liu, Linda M. Griffith, Hans D. Ochs, Jack J. Bleesing, Stephanie Edwards, Christopher C. Dvorak, Sonali Chaudhury, Susan E. Prockop, Ralph Quinones, Frederick D. Goldman, Troy C. Quigg, Shanmuganathan Chandrakasan, Angela R. Smith, Suhag Parikh, Blachy J. Dávila Saldaña, Monica S. Thakar, Rachel Phelan, Shalini Shenoy, Lisa R. Forbes, Caridad Martinez, Deepak Chellapandian, Evan Shereck, Holly K. Miller, Neena Kapoor, Jessie L. Barnum, Hey Chong, David C. Shyr, Karin Chen, Rolla Abu-Arja, Ami J. Shah, Katja G. Weinacht, Theodore B. Moore, Avni Joshi, Kenneth B. DeSantes, Alfred P. Gillio, Geoffrey D. E. Cuvelier, Michael D. Keller, Jacob Rozmus, Troy Torgerson, Michael A. Pulsipher, Elie Haddad, Kathleen E. Sullivan, Brent R. Logan, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Sung-Yun Pai, David J. Rawlings, Morton J. Cowan

Blood (2020) 135 (23): 2094–2105.

https://doi.org/10.1182/blood.2019002939

Published: 2020

Journal Articles

Is there an optimal conditioning for older patients with AML receiving allogeneic hematopoietic cell transplantation?

Stefan O. Ciurea, Piyanuch Kongtim, Ankur Varma, Gabriela Rondon, Julianne Chen, Samer Srour, Qaiser Bashir, Amin Alousi, Rohtesh Mehta, Betul Oran, Uday Popat, Chitra Hosing, Amanda Olson, Naval Daver, Marina Konopleva, Richard E. Champlin

Blood (2020) 135 (6): 449–452.

https://doi.org/10.1182/blood.2019003662

Published: 2020

Includes: Supplemental data

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Immune Reconstitution in 107 Children with Sickle Cell Anemia Transplanted with Bone Marrow or Cord Blood from a Matched-Sibling Donor after Myeloablative Conditioning Regimen Including 20mg/Kg ATG

Francoise Bernaudin, MD, Azadeh Djavidi, MD, Cécile Arnaud, MD, Annie Kamdem, MD, Isabelle Hau, MD, Corinne Pondarré, MD PhD, Jean-Paul Vernant, MD, Mathieu Kuentz, MD, Nathalie Dhedin, MD, Jean-Hugues Dalle, MD PhD, Eliane Gluckman, Gerard Socie, MD PhD

Blood (2019) 134 (Supplement_1): 2253.

https://doi.org/10.1182/blood-2019-125316

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Risk Factors for Central Nervous System (CNS) Relapse after Autologous Stem Cell Transplant (ASCT) in Diffuse Large B-Cell Lymphoma (DLBCL)

Yukie Terasaki, MD, Hiroaki Shimizu, MD PhD, Yuri Miyazawa, MD PhD, Kohtaro Toyama, MD PhD, Hiromi Koiso, MD PhD, Akihiko Yokohama, MD PhD, Morio Matsumoto, MD, Hidemi Ogura, Chiaki Naito, MD, Norifumi Tsukamoto, MD PhD, Hiroshi Handa, MD PhD

Blood (2019) 134 (Supplement_1): 5344.

https://doi.org/10.1182/blood-2019-127990

Published: 2019

Journal Articles

Low-Dose Methotrexate Could Reduce Severe Early Immune Reactions but Probably Increase Garft Failure in Umbilical Cord Blood Transplantation

Liu Huilan, MD PhD, Xiaoyu Zhu, Kaidi Song, Xiang Wan, Guangyu Sun, Baolin Tang, Juan Tong, Wen Yao, Xuhan Zhang, Liangquan Geng, Zimin Sun

Blood (2019) 134 (Supplement_1): 5679.

https://doi.org/10.1182/blood-2019-131524

Published: 2019

Journal Articles

Pentotstatin/TBI Conditioning Is Well-Tolerated and Permits Engraftment of a Second Allogeneic Stem Cell Transplant Following Primary or Secondary Rejection of an Allogeneic Hematopoietic Stem Graft

Ani Gvajaia, MD, Amelia Langston, MD, Natia Esiashvil, MD, Michael Graiser, PhD, Kelly Valla, PharmD, Edmund K. Waller, PhDMD

Blood (2019) 134 (Supplement_1): 5657.

https://doi.org/10.1182/blood-2019-122467

Published: 2019

Journal Articles

Total Body Irradiation Free Haploidentical Stem Cell Transplant with Post-Transplant Cyclophosphamide for Children with Sickle Cell Disease

Satya Prakash Yadav, MBBS, DCH, DNB, Anil Sharma, MBBS, MD, Rohit Kapoor, MBBS, MD, Goutomi Chatterjee, MBBS, MD, Dhwanee Thakkar, MBBS, MDFNB, Neha Rastogi, MBBS,DCH,DNB

Blood (2019) 134 (Supplement_1): 5661.

https://doi.org/10.1182/blood-2019-130988

Published: 2019

Journal Articles

Dual T-Cell Depletion with a Very Low Dose of ATG and Ptcy Provides an Effective Control of Acute Gvhd in PBSC RIC Allo-HSCT

Maria Queralt Salas, MD, Arjun Law, MDDM, Wilson Lam, MD, David Loach, MD, Zeyad Al-Shaibani, Fotios V. Michelis, MD PhD, Dennis Dong Hwan Kim, MD PhD, Jeffrey H. Lipton, MD PhD, Rajat Kumar, MD, Jonas Mattsson, MD, Auro Viswabandya, MDDM

Blood (2019) 134 (Supplement_1): 5669.

https://doi.org/10.1182/blood-2019-128532

Published: 2019

Journal Articles

Impact of Absolute Lymphocyte Count at Time of Antithymocyte Globulin on Outcomes of Allogeneic Unrelated Hematopoietic Cell Transplantation with Busulfan and Fludarabine Myeloablative Conditioning

Adithya Chennamadhavuni, MBBS, Kalyan Nadiminti, Lindsay Dozeman, Umar Farooq, MD, Yogesh Jethava, MBBS, MDFRCPath, MRCP, Sarah Mott, MS, Margarida Magalhaes Silverman, MD

Blood (2019) 134 (Supplement_1): 5671.

https://doi.org/10.1182/blood-2019-128186

Published: 2019

Journal Articles

Comparison of Fixed Dose, Reduced-Intensity Conditioning with Busulfan and Fludarabine to Reduced PK-Guided Busulfan AUC Conditioning in Patients Undergoing Hematopoietic Stem Cell Transplant for AML/MDS

Brendan Rasor, PharmD, Tyler Dickerson, PharmD, Qiuhong Zhao, MS, Jonathan E Brammer, MD, Karilyn Larkin, MD, Samantha Jaglowski, MDMPH, Alice S. Mims, MD, Sam Penza, MD, Sumithira Vasu, MDMBBS, Sarah A Wall, MD, Basem M. William, MRCP,MD, Ayman Saad, MD, Julianna Roddy, PharmD, Hannah K. Choe, MD, Marcin Puto, PharmD

Blood (2019) 134 (Supplement_1): 5715.

https://doi.org/10.1182/blood-2019-123657

Published: 2019

Journal Articles

Expanding the Indications for Ibrutinib: Complete Remission Obtained By Induction with Ibrutinib Followed By Consolidative Ahsct in Refractory Primary CNS Lymphoma

Cassidy Brothers, MD MSC

Blood (2019) 134 (Supplement_1): 5631.

https://doi.org/10.1182/blood-2019-125378

Published: 2019

Journal Articles

Liposomal Daunorubicin/Cytarabine As a Bridge to Donor Lymphocyte Infusion or Allogeneic Stem Cell Transplantation for High-Risk Acute Myelogenous Leukemia

Daulath Singh, MD, Patrick Hagen, MD, Nasheed Hossain, MD, Scott E. Smith, MD PhD, Patrick J. Stiff, MD, Stephanie B. Tsai, MD MS

Blood (2019) 134 (Supplement_1): 5726.

https://doi.org/10.1182/blood-2019-129380

Published: 2019

Journal Articles

Impact of Comorbidities and Body Mass Index in Patients with Polycythemia Vera: A PV-NET Real World Study

Blood (2019) 134 (Supplement_1): 4184.

https://doi.org/10.1182/blood-2019-125387

Published: 2019

Journal Articles

Complementary Transplantation with Unrelated Cord Blood Following Haploidentical Stem Cells for Juvenile Myelomonocytic Leukemia:Double-Centers Study from China

Zhiyong Peng, MD, Xiaoqin Feng, Huaying Liu, MD, Jianyun Liao, MD, Chaoke Bu, Yuelin He, MD, Jianmin Zhang, MD, Jingyu Gao, Chunfu Li, MD

Blood (2019) 134 (Supplement_1): 4181.

https://doi.org/10.1182/blood-2019-128331

Published: 2019

Journal Articles

High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Hematopoietic and Microglia Recovery and Disease Correction in a Mouse Model of Hurler Syndrome

Kevin A. Goncalves, PhD, Sharon L. Hyzy, MS, Melissa L. Brooks, BS, Hans J. Hertzler, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 4424.

https://doi.org/10.1182/blood-2019-131189

Published: 2019

Journal Articles

Comparison of Mycophenolate Mofetil and a Calcineurin Inhibitor Versus Calcineurin Inhibitor Based Graft-Versus-Host-Disease Prophylaxis for Matched Unrelated Donor Transplant for Acute Myeloid Leukemia. a Study from the ALWP of the EBMT

Annalisa Paviglianiti, MD, Myriam Labopin, MD, Didier Blaise, MD PhD, Gerard Socie, MD PhD, Claude-Eric Bulabois, MD, Celestine Simand, MD, Nathalie Fegueux, MD, Armin Gerbitz, MD, Gaëlle Guillerm, MD, Johan Maertens, Patrice Chevallier, MD, Anne Huynh, MD, Pascal Turlure, MD, Eric Deconinck, MD PhD, Edouard Forcade, MD, Arnon Nagler, MD MSc, Mohamad Mohty, MD PhD

Blood (2019) 134 (Supplement_1): 4548.

https://doi.org/10.1182/blood-2019-122251

Published: 2019

Journal Articles

Full Donor Chimerism after Allografts for Myelofibrosis: The Role of Conditioning Regimen

Patrizia Chiusolo, Sabrina Giammarco, MD PhD, Teresa Lamparelli, Lucia Casarino, Monica Rossi, Alida Dominietto, MD, Anna Maria Raiola, MD, Francesca Gualandi, Carmen Di Grazia, MD, Silvia Betti, MD PhD, Federica Sorà, MD, Elena Rossi, MD PhD, Luciana Teofili, MD, Simona Sica, MD, Andrea Bacigalupo, MD, Emanuele Angelucci, MD

Blood (2019) 134 (Supplement_1): 4490.

https://doi.org/10.1182/blood-2019-126711

Published: 2019

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Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Is there an optimal conditioning for older patients with AML receiving allogeneic hematopoietic cell transplantation?

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Immune Reconstitution in 107 Children with Sickle Cell Anemia Transplanted with Bone Marrow or Cord Blood from a Matched-Sibling Donor after Myeloablative Conditioning Regimen Including 20mg/Kg ATG

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Risk Factors for Central Nervous System (CNS) Relapse after Autologous Stem Cell Transplant (ASCT) in Diffuse Large B-Cell Lymphoma (DLBCL)

Low-Dose Methotrexate Could Reduce Severe Early Immune Reactions but Probably Increase Garft Failure in Umbilical Cord Blood Transplantation

Pentotstatin/TBI Conditioning Is Well-Tolerated and Permits Engraftment of a Second Allogeneic Stem Cell Transplant Following Primary or Secondary Rejection of an Allogeneic Hematopoietic Stem Graft

Total Body Irradiation Free Haploidentical Stem Cell Transplant with Post-Transplant Cyclophosphamide for Children with Sickle Cell Disease

Dual T-Cell Depletion with a Very Low Dose of ATG and Ptcy Provides an Effective Control of Acute Gvhd in PBSC RIC Allo-HSCT

Impact of Absolute Lymphocyte Count at Time of Antithymocyte Globulin on Outcomes of Allogeneic Unrelated Hematopoietic Cell Transplantation with Busulfan and Fludarabine Myeloablative Conditioning

Comparison of Fixed Dose, Reduced-Intensity Conditioning with Busulfan and Fludarabine to Reduced PK-Guided Busulfan AUC Conditioning in Patients Undergoing Hematopoietic Stem Cell Transplant for AML/MDS

Expanding the Indications for Ibrutinib: Complete Remission Obtained By Induction with Ibrutinib Followed By Consolidative Ahsct in Refractory Primary CNS Lymphoma

Liposomal Daunorubicin/Cytarabine As a Bridge to Donor Lymphocyte Infusion or Allogeneic Stem Cell Transplantation for High-Risk Acute Myelogenous Leukemia

Impact of Comorbidities and Body Mass Index in Patients with Polycythemia Vera: A PV-NET Real World Study

Complementary Transplantation with Unrelated Cord Blood Following Haploidentical Stem Cells for Juvenile Myelomonocytic Leukemia:Double-Centers Study from China

High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Hematopoietic and Microglia Recovery and Disease Correction in a Mouse Model of Hurler Syndrome

Comparison of Mycophenolate Mofetil and a Calcineurin Inhibitor Versus Calcineurin Inhibitor Based Graft-Versus-Host-Disease Prophylaxis for Matched Unrelated Donor Transplant for Acute Myeloid Leukemia. a Study from the ALWP of the EBMT

Full Donor Chimerism after Allografts for Myelofibrosis: The Role of Conditioning Regimen

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