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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

Targeted Mass Spectrometry-Based Serum M-Protein Monitoring for Early Relapse Detection

Zac McDonald, PhD, Mariya Liyasova, PhD, Paul Taylor, Xin Xu, Kathleen Gorospe, Chenyu Yao, Qixin Liu, Liqiang Yang, PhD, Bin Ma, PhD, Jesus Piza, MD, Donna E Reece, MD, Suzanne Trudel, MD

Blood (2019) 134 (Supplement_1): 4347.

https://doi.org/10.1182/blood-2019-130251

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

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

Hematopoietic Stem Cell Transplantation in Βthalassemia Major: The Egyptian Outcome

Amal El-Beshlawy, MD, Amina AbdelSalam, MD, Gamal Fathy, MD, Khaled M. Salamaa, MD, Mona El ghamrawy, MD, Samar S. Ramadan, M.Sc., Alaa M. Elhaddad, MD

Blood (2019) 134 (Supplement_1): 5732.

https://doi.org/10.1182/blood-2019-124398

Published: 2019

Journal Articles

Outcomes of Hematopoietic Cell Transplantation in Acute Promyelocytic Leukemia - a Single Institution Experience

Hamza Hashmi, MD, Jeffrey Lancet, MD, Asmita Mishra, MD, Kendra L. Sweet, MD, Rami S. Komrokji, MD, Hugo F. Fernandez, MD, Lia Perez, MD, Farhad Khimani, MD, Michael Nieder, MD, Hany Elmariah, MD MS, Joseph Pidala, MD, Nelli Bejanyan, MD, Alan F. List, MD, Claudio Anasetti, MD, Taiga Nishihori, MD

Blood (2019) 134 (Supplement_1): 5739.

https://doi.org/10.1182/blood-2019-128985

Published: 2019

Journal Articles

Poor Outcomes of HLA-Mismatched Allogeneic Hematopoietic Cell Transplantation and High Ferritin Levels after a Reduced-Intensity Conditioning Regimen in Patients with Advanced Myelofibrosis

Han Bi Lee, Jae-Ho Yoon, MD PhD, Gi June Min, MD, Sung-Soo Park, Silvia Park, Sung-Eun Lee, MD, Yoo-Jin Kim, MD PhD, Seok Lee, MD PhD, Hee-Je Kim, MD PhD, Chang-Ki Min, MD PhD, Seok-Goo Cho, MD PhD, Jong Wook Lee, MD PhD, Ki-Seong Eom, MD PhD

Blood (2019) 134 (Supplement_1): 5742.

https://doi.org/10.1182/blood-2019-130338

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

Journal Articles

Cord Blood Transplantation Is an Effective Treatment Option in Patients with Myelodysplastic and Myeloproliferative Syndromes

Laura Roberts, Rachel B. Salit, MD, Lauren Longo, Elisabetta Xue, MD, Corinne Summers, MD, Colleen Delaney, MD MSc, Ann Dahlberg, MD, Filippo Milano, MD PhD

Blood (2019) 134 (Supplement_1): 2048.

https://doi.org/10.1182/blood-2019-126216

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

Journal Articles

Use of Thymoglobulin with Lower Dose of Busulfan Results in Less Acute Gvhd Following Unrelated Donor Allogeneic Stem Cell Transplantation for AML without Affecting Relapse-Free and Overall Survival

Dipenkumar Modi, MD, Vijendra Singh, MD, Seongho Kim, PhD, Abhinav Deol, MD, Asif Alavi, MD, Lois Ayash, MD, Andrew Kin, MD, Voravit Ratanatharathorn, MD, Joseph P. Uberti, MD, PhD

Blood (2019) 134 (Supplement_1): 4488.

https://doi.org/10.1182/blood-2019-125418

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

Targeted Mass Spectrometry-Based Serum M-Protein Monitoring for Early Relapse Detection

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

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

Hematopoietic Stem Cell Transplantation in Βthalassemia Major: The Egyptian Outcome

Outcomes of Hematopoietic Cell Transplantation in Acute Promyelocytic Leukemia - a Single Institution Experience

Poor Outcomes of HLA-Mismatched Allogeneic Hematopoietic Cell Transplantation and High Ferritin Levels after a Reduced-Intensity Conditioning Regimen in Patients with Advanced Myelofibrosis

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)

Cord Blood Transplantation Is an Effective Treatment Option in Patients with Myelodysplastic and Myeloproliferative Syndromes

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

Use of Thymoglobulin with Lower Dose of Busulfan Results in Less Acute Gvhd Following Unrelated Donor Allogeneic Stem Cell Transplantation for AML without Affecting Relapse-Free and Overall Survival

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