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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

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

Blood (2019) 134 (Supplement_1): 610.

https://doi.org/10.1182/blood-2019-125968

Published: 2019

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

The Sequential Flamsa-Bu Conditioning Regimen Does Not Improve Outcome in Patients Allografted for High Risk Acute Myeloid and Myelodysplasia Irrespective of Pre-Transplant MRD Status: Results of the UK NCRI Figaro Trial

Blood (2019) 134 (Supplement_1): 2031.

https://doi.org/10.1182/blood-2019-125646

Published: 2019

Journal Articles

Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents with GATA2-Related Myelodysplastic Syndrome

Blood (2019) 134 (Supplement_1): 2033.

https://doi.org/10.1182/blood-2019-122403

Published: 2019

Journal Articles

A Phase II Clinical Trial of Post-Transplant Cyclophosphamide for Graft Versus Host Disease Prevention Following Myeloablative Peripheral Blood Stem Cell Matched Unrelated Donor Hematopoietic Stem Cell Transplantation

Omer Jamy, MD, Racquel Innis-Shelton, MD, Susan Bal, MDMBBS, Ravi K. Paluri, MD, Samina Karim, Joseph H. Chewning, MD, Donna Salzman, MD, Luciano J. Costa, MD PhD, Antonio Di Stasi, MD, Lawrence S. Lamb, Jr., PhD, Shin Mineishi, Ayman Saad, MD

Blood (2019) 134 (Supplement_1): 2003.

https://doi.org/10.1182/blood-2019-129536

Published: 2019

Journal Articles

Optimal Conditioning for Older Patients with Acute Myeloid Leukemia (AML) Receiving Allogeneic Hematopoietic Stem Cell Transplantation: A Propensity Score Analysis

Stefan O. Ciurea, MD, Ankur Varma, MD, Piyanuch Kongtim, MD, Samer Srour, Qaiser Bashir, MBBS, Amin M. Alousi, MD, Rohtesh S. Mehta, MD, Betul Oran, MDMS, Uday Popat, MD, Chitra Hosing, MD, Gabriela Rondon, MD, Julianne Chen, Amanda L. Olson, MD, Marina Y Konopleva, MD PhD, Richard E Champlin

Blood (2019) 134 (Supplement_1): 42.

https://doi.org/10.1182/blood-2019-123699

Published: 2019

Journal Articles

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Mark C. Walters, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Julie Kanter, MD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 2061.

https://doi.org/10.1182/blood-2019-128814

Published: 2019

Journal Articles

Treosulfan Metabolite (S, S-EBDM) Pharmacokinetics Influences Regimen Related Toxicity in Patients with Beta Thalassaemia Major Undergoing HSCT

Aswin Anand PAI, MSc, John C Panetta, PhD, Ezhil Pavai Mohanan, PhD, Raveen Stephen Stallon Illangeswaran, MSc, Balaji Balakrishnan, PhD, Eunice Sindhuvi Edison, PhD, Kavitha M Lakshmi, MSc, Uday Prakash Kulkarni, MD DM, Anup Joseph Devasia, MD DM, Fouzia N., Anu Korula, MD DM, Aby Abraham, MD DM, Biju George, MDDM, Vikram Mathews, MD DM, Alok Srivastava, MD,FRACP,FRCPA, Poonkuzhali Balasubramanian, PhD

Blood (2019) 134 (Supplement_1): 1977.

https://doi.org/10.1182/blood-2019-125604

Published: 2019

Journal Articles

Potential Influence of Hematological or Immunological Hereditary Predisposition Genes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Hematological Malignancies

Zhihui Li, Qinlong Zheng, Yongqiang Zhao, Yanzhi Song, Fan Yang, Min Jiang, Chunrong Tong, Tong Wu, MD

Blood (2019) 134 (Supplement_1): 2037.

https://doi.org/10.1182/blood-2019-126339

Published: 2019

Journal Articles

Methodology for the Start-up of a Pediatric Hematopoietic Stem Cell Transplant Unit (pHSCTU) in Middle-Income Countries (MICs): An Exportable Model?

Marta Verna, MD, Attilio Rovelli, MD, Valentino Conter, MD, Marta Canesi, Nurse, Ignazio Majolino, MD, Dosti Othman, MD, Vian Faeq, MD, Jabibi Noguera, MD, Maria Liz Benitez, MD, Othman Dastan, MD, Andrea Biondi, MD PhD

Blood (2019) 134 (Supplement_1): 2044.

https://doi.org/10.1182/blood-2019-121922

Published: 2019

Journal Articles

Profound Lymphopenia at the Time of ATG Administration Is Not Predictive of Survivals after Allotransplant Using Purine Analogue/Busulfan-Based Conditioning Regimen

Blood (2019) 134 (Supplement_1): 1985.

https://doi.org/10.1182/blood-2019-122013

Published: 2019

Journal Articles

Combination of a Cyclophosphamide-Total Body Irradiation Conditioning Regimen and Tacrolimus Plus Mycophenolate Mofetil for Gvhd Prophylaxis Is Associated with Higher HHV-6 Encephalitis Incidence after Cord Blood Transplantation

Takuto Mori, MD, Nobuhiro Hiramoto, MD, Ryosuke Ueda, MD PhD, Yoshimitsu Shimomura, Satoshi Yoshioka, MD PhD, Takayuki Ishikawa, MD PhD

Blood (2019) 134 (Supplement_1): 1986.

https://doi.org/10.1182/blood-2019-125209

Published: 2019

Journal Articles

Alternative Donor and Disease-Specific Conditioning Regimen Hematopoietic Stem Cell Transplantation for Inherited Bone Marrow Failure Syndromes

Fang Xu, Min Xiong, Ruijuan Sun, Yanli Zhao, Jianping Zhang, Xingyu Cao, Deyan Liu, Zhijie Wei, Jiarui Zhou, Yue Lu, Peihua Lu, Daopei Lu

Blood (2019) 134 (Supplement_1): 2047.

https://doi.org/10.1182/blood-2019-125695

Published: 2019

Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) with Different Intensity Conditioning Regimens in Children with Very High Risk Acute Lymphoblastic Leukemia (VHR ALL): A Single-Center Experience

Elena Vladimirovna Semenova, MD PhD, Olesia V Paina, PhD, Zhemal Zarifovna Rakhmanova, Dr., Polina Valerievna Kozhokar, Dr., Anastasia S Frolova, Dr, Kirill Alexandrovich Ekushov, Dr., Tatiana A Bykova, MD PhD, Anna Osipova, Inna V Markova, Dr., Sergey N. Bondarenko, PhDMD, Ildar Barhatov, Aleksandr Alyanski, Elena Babenko, Ivan S Moiseev, MD PhD, Ludmila S Zubarovskaya, MD PhD professor, Axel Zander, Boris V Afanasyev, MD PhD professor

Blood (2019) 134 (Supplement_1): 2050.

https://doi.org/10.1182/blood-2019-128163

Published: 2019

Journal Articles

Sustained Donor Chimerism and Rapid Immune Cell Reconstitution Following Familial Haploidentical (FHI) CD34 Enriched Stem Cell Transplantation with Pbmnc Addback in Patients with High Risk Sickle Cell Disease (SCD) (IND 14359)

Yaya Chu, PhD, Julie-An Talano, MD, Lee Ann Baxter-Lowe, PhD, Carolyn A. Keever-Taylor, PhD, Erin Morris, RN, BSN, Harshini Mahanti, BS, MSCR, Janet Ayello, MS, MT(ASCP), Rona Singer Weinberg, PhD, Qiuhu Shi, PhD, Theodore B. Moore, MD, Sandra Fabricatore, RN, PNP, MSN, Brenda J. Grossman, MD, Carmella van de Ven, MA, Shalini Shenoy, MD, Mitchell S Cairo, MD

Blood (2019) 134 (Supplement_1): 1990.

https://doi.org/10.1182/blood-2019-126757

Published: 2019

NARROW

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

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

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)

The Sequential Flamsa-Bu Conditioning Regimen Does Not Improve Outcome in Patients Allografted for High Risk Acute Myeloid and Myelodysplasia Irrespective of Pre-Transplant MRD Status: Results of the UK NCRI Figaro Trial

Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents with GATA2-Related Myelodysplastic Syndrome

A Phase II Clinical Trial of Post-Transplant Cyclophosphamide for Graft Versus Host Disease Prevention Following Myeloablative Peripheral Blood Stem Cell Matched Unrelated Donor Hematopoietic Stem Cell Transplantation

Optimal Conditioning for Older Patients with Acute Myeloid Leukemia (AML) Receiving Allogeneic Hematopoietic Stem Cell Transplantation: A Propensity Score Analysis

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Treosulfan Metabolite (S, S-EBDM) Pharmacokinetics Influences Regimen Related Toxicity in Patients with Beta Thalassaemia Major Undergoing HSCT

Potential Influence of Hematological or Immunological Hereditary Predisposition Genes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Hematological Malignancies

Methodology for the Start-up of a Pediatric Hematopoietic Stem Cell Transplant Unit (pHSCTU) in Middle-Income Countries (MICs): An Exportable Model?

Profound Lymphopenia at the Time of ATG Administration Is Not Predictive of Survivals after Allotransplant Using Purine Analogue/Busulfan-Based Conditioning Regimen

Combination of a Cyclophosphamide-Total Body Irradiation Conditioning Regimen and Tacrolimus Plus Mycophenolate Mofetil for Gvhd Prophylaxis Is Associated with Higher HHV-6 Encephalitis Incidence after Cord Blood Transplantation

Alternative Donor and Disease-Specific Conditioning Regimen Hematopoietic Stem Cell Transplantation for Inherited Bone Marrow Failure Syndromes

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) with Different Intensity Conditioning Regimens in Children with Very High Risk Acute Lymphoblastic Leukemia (VHR ALL): A Single-Center Experience

Sustained Donor Chimerism and Rapid Immune Cell Reconstitution Following Familial Haploidentical (FHI) CD34 Enriched Stem Cell Transplantation with Pbmnc Addback in Patients with High Risk Sickle Cell Disease (SCD) (IND 14359)

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American Society of Hematology

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American Society of Hematology

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