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Journal Articles

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Steven W. Pipe, MD, Michael Recht, MD PhD, Nigel S. Key, MD, Frank W.G. Leebeek, MD PhD, Giancarlo Castaman, MD, Susan U. Lattimore, R.N., Paul Van Der Valk, Kathelijne Peerlinck, MD PhD, Michiel Coppens, MD, Niamh O'Connell, MD PhD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Peter Kampmann, MD, Karina Meijer, MD PhD, Annette von Drygalski, MD Pharm D, Guy Young, MD, Cedric Hermans, MDMRCP,PhD, Jan Astermark, MD PhD, Robert Klamroth, MD PhD, Richard S. Lemons, MD, Nathan Visweshwar, MD, Shelley Crary, MD MS, Rashid Kazmi, MBBS, Emily Symington, Miguel A. Escobar, MD, Esteban Gomez, MD, Rebecca Kruse-Jarres, MD, Adam Kotowski, MD, Doris Quon, MD PhD, Michael Wang, MD, Allison P. Wheeler, MD, Eileen K Sawyer, PhD, Stephanie Verweij, BSc, Valerie Colletta, MSc, Naghmana Bajma, MD, Robert Gut, MD PhD, Wolfgang A. Miesbach, MD PhD

Blood (2020) 136 (Supplement_2): LBA-6.

https://doi.org/10.1182/blood-2020-143560

Published: 2020

Journal Articles

Mutation Profiles Identify Distinct Clusters of Lower Risk Myelodysplastic Syndromes with Unique Clinical and Biological Features and Clinical Endpoints

Blood (2020) 136 (Supplement 1): 29.

https://doi.org/10.1182/blood-2020-138549

Published: 2020

Journal Articles

Clinical Effectiveness of Combination Immunotherapy DPX-Survivac, Low Dose Cyclophosphamide, and Pembrolizumab in Recurrent/Refractory DLBCL: The Spirel Study

Neil L Berinstein, MD, Isabelle Bence-Bruckler, MD, Nicholas Allen Forward, MDMSc,FRCPC, Pierre Laneuville, MD FRCPC, Joy Mangel, MD FRCPC, Douglas A. Stewart, MD FRCPC, Irina Amitai, MD, Gail Klein, MSc, Nancy Pennell, BSc, Iran Rashedi, MD MSc, Kim Roos, BA, BSc, Yogesh Bramhecha, Rebekah Conlon, RN

Blood (2020) 136 (Supplement 1): 16.

https://doi.org/10.1182/blood-2020-136751

Published: 2020

Journal Articles

Donor-Derived T-Cells Specific for WT1 and PRAME in Combination with T-Cells Specific for Multiple Pathogens for Prevention of Relapse and Infection after Haemopoietic Stem Cell Transplant (HSCT) for Acute Myeloid Leukaemia (AML) or High-Risk Myelodysplasia (MDS) - (The INTACT Trial)

Wei Jiang, MBBS, M Clin MD FRACP, FRCPA, Emily Blyth, B. MDHons), FRACP, FRCPA, PhD, Peter J. Shaw, MA,FRACP, Leighton Edward Clancy, BSc, PhD, Kenneth P. Micklethwaite, MBBS, PhD FRACP, FRCPA, Abir Bhattacharyya, MBBS, FRACP, FRCPA, Elissa Atkins, RN, Selmir Avdic, PhD, Gaurav Sutrave, MBBS, FRACP, FRCPA, Janine Street, BSc MSc (Hons), David Gottlieb, MBBS, MD FRACP, FRCPA

Blood (2020) 136 (Supplement 1): 38.

https://doi.org/10.1182/blood-2020-143136

Published: 2020

Journal Articles

Clinical Validation of a NGS Capture Panel to Identify Mutations, Copy Number Variations and Translocations in Patients with Multiple Myeloma

Blood (2020) 136 (Supplement 1): 13–14.

https://doi.org/10.1182/blood-2020-140711

Published: 2020

Journal Articles

Matching-Adjusted Indirect Comparison (MAIC) of Lisocabtagene Maraleucel (liso-cel) Vs Axicabtagene Ciloleucel (axi-cel) and Tisagenlecleucel in Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL)

David G. Maloney, MD PhD, John Kuruvilla, MD, Christopher P Fox, MD PhD, Guillaume Cartron, MD PhD, Daniel Li, PhD, Jens Hasskarl, MD, Ashley Bonner, PhD, Yixie Zhang, PhD, Fei Fei Liu, BSc, MBA

Blood (2020) 136 (Supplement 1): 18–19.

https://doi.org/10.1182/blood-2020-137719

Published: 2020

Journal Articles

A Randomized Study of Bortezomib, Cyclophosphamide and Dexamethasone Induction (VCD) Versus VCD and Daratumumab Induction Followed By Daratumumab Maintenance (VCDD) for the Initial Treatment of Transplant-Ineligible Patients with Multiple Myeloma (AMaRC 03-16)

Peter Mollee, FRACP,MBBS,MSc,FRCPA, John Reynolds, PhD, Wojt Janowski, FRCPA,FRACP, Hang Quach, MDFRACP,FRCPA,MBBS, Philip Campbell, MBBS, Simon D. Gibbs, FRACP, FRCPA, MBBS, Sophie Lee, FRACP, FRCPA, PhD, James D'Rozario, MBBS, FRACP, FRCPA, Kerry Taylor, FRACP,FRCPA,MBBS, Tara Cochrane, MBBS,FRCPA,FRACP, Craig Thomas Wallington-Beddoe, BSc,MBBS,PhDFRACP,FRCPA, Fiona Kwok, FRACP,FRCPA,MBBS, Nicholas Weber, MBBS, Ian H. Kerridge, FRACP FRCPA MPhil BA, Helen Weston, MBBS, FRACP, FRCPA, Phoebe Joy Ho, MBBS,DPhil,FRACP,FRCPA, Noemi Horvath, MB ChB FRACP FRCPA, Flora Yuen, BBiomedSc, Andrew Spencer

Blood (2020) 136 (Supplement 1): 4–5.

https://doi.org/10.1182/blood-2020-138394

Published: 2020

Journal Articles

Molecular Characterization and Clinical Outcomes of Young Adult Patients (≤45 years old) with Philadelphia-Negative Myeloproliferative Neoplasms

Marcio M Andrade-Campos, MD PhD, Lierni Fernández-Ibarrondo, Joan Gibert, PhD, Concepción Fernández, Brayan Merchan, MD, Nieves García Gisbert, BSc, Laura Camacho, Leonor Arenillas, MD PhD, Sara García Ávila, MD, Raquel Longarón, Antonio Salar, MD PhD, Carles Besses, MD PhD, Beatriz Bellosillo, PhD

Blood (2020) 136 (Supplement 1): 13–14.

https://doi.org/10.1182/blood-2020-142705

Published: 2020

Journal Articles

High Transfusion Dependence and Serum Ferritin but Not Transferrin Saturation Predict Inferior Clinical Outcomes in Patients with MDS

Blood (2020) 136 (Supplement 1): 47–48.

https://doi.org/10.1182/blood-2020-140065

Published: 2020

Journal Articles

Implementation of Flow Cytometric Monocyte Repartition Assay for Assessment of Chronic Myelomonocytic Leukaemia (CMML) in Bone Marrow

Sharon A Allen, MBBS, B.MDci(Path), Eugene Ng, BSc, Suzanne Edwards, GDipMStat, GDipMa, BN, Uwe H Hahn, MBBS, MD FRCPA, Tatjana Banovic, MBBS, MD PhD FRCPA, David M Ross, MBBS, PhD FRACP, FRCPA

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-140932

Published: 2020

Journal Articles

A Phase 1b Study of Eltrombopag and Azacitidine in Patients with High Risk Myelodysplastic Syndromes and Related Disorders

Alex Sternberg, MRCP,FRCPath, Rebecca H. Boucher, BSc, MSc, PhD, Helen C Coulthard, BSc, Manoj Raghavan, MBBS, PhD MRCP, FRCPath, Dominic J. Culligan, MD, Aimee Jackson, BSc, MSc, Catherine Cargo, MB, PhD, Mike Dennis, MD, Marlen Metzner, MSc, Rachel Moore, BA, David Bowen, MD, Paresh Vyas, MRCP, FRCP, FRCPath

Blood (2020) 136 (Supplement 1): 10.

https://doi.org/10.1182/blood-2020-134227

Published: 2020

Journal Articles

Ibrutinib and Obinutuzumab in CLL: MRD Responses Sustained for Several Years with Deepest MRD Depletion in Patients with >1 Year Prior Ibrutinib Exposure

Andy C. Rawstron, PhD, Peter Hillmen, MBChB, FRCP, FRCPath, PhD, Shanna Maycock, BSc, MSc, Nichola Webster, Kristian Brock, PhD, Rebecca H. Boucher, BSc, MSc, PhD, Francesca Yates, PhD, Rebecca Jarvis, BSc, Surita Dalal, PhD, Ruth M. de Tute, PhD FRCPath, Paul Moss, MBBS, PhD FRCP, Donald Macdonald, MBChB, MD FRCP, PhD FCRPath, Piers Patten, MD PhD, Christopher Fegan, Andrew Pettitt, MA, MB BChir, PhD MRCP, MRCPath, Christopher P. Fox, MD PhD, Adrian Bloor, PhD FRCP, FRCPath, Oonagh Sheehy, MB BCh BAO, MRCP, FRCPath, Peter Hillmen, MBChB, PhD

Blood (2020) 136 (Supplement 1): 27–28.

https://doi.org/10.1182/blood-2020-136990

Published: 2020

Journal Articles

COVID-19 and Stem Cell Transplantation; Results from the Prospective Survey By the Infectious Diseases Working Party of the European Society for Blood and Marrow Transplantation (EBMT) and the Spanish Hematopoietic Stem Cell Transplantation and Cell Therapy Group (GETH)

Per Ljungman, MD PhD, Rafael De La Camara, MD, Malgorzata Mikulska, Gloria Tridello, Beatriz Aguado, MD, Yves Beguin, MD PhD, Rodrigo Martino Bufarull, Fabio Ciceri, MD, Lucía López Corral, MD, Claudia Crippa, MD, Dries Deeren, MD PhD, Rafael F. Duarte, MD PhD FRCP, María Laura Fox, Anna Grassi, MD, María-José Jimenez, MD, Safiye Koculu, MD, Mi Kwon, MD PhD, Juan Carlos Vallejo Llamas, Jose Luiz Lopez Lorenzo, MD, Stephan Mielke, MD, Javier López Jiménez, MD PhD, Zoraida Mesa Morales, Kim H. Orchard, MBBS,PhDFRCP,FRCPath, Rocio Parody Porras, Victoria Potter, MBBS, BSc, FRACP, FRCPA, María Suárez-Lledó, MD, Nicolaas Schaap, MD PhD, Celestine Simand, MD, Luisa Sisinni, MD, John A Snowden, MD PhD, Daniele Vallisa, MD, Ipek Yonalhindilerden, Alienor Xhaard, MD, Nina Simone Knelange, Angel Cedillo, Nicolaus Kröger, MD, Jose Luis Piñana Sanchez, Jan Styczynski, MD PhD

Blood (2020) 136 (Supplement 1): 32–33.

https://doi.org/10.1182/blood-2020-138732

Published: 2020

Journal Articles

Early Response Predicts Long-Term Improvement or Stabilization of Persons with Multiple Sclerosis Treated with Hematopoietic Stem Cell Transplantation

Iván Murrieta-Álvarez, MD, Montserrat Rivera-Álvarez, MD, Gilberto David Elias-de-la-Cruz, MD, Luisa Fernanda Sánchez-Valledor, MD, Carmina Alejandra Córdova-Ramírez, Yahveth Cantero-Fortiz, MD, Andrés A. León-Peña, MD, Martin Ojeda-Laguna, BSc, José Manuel Priesca-Marin, MD, Guillermo J. Ruiz-Delgado, MD FACP, Guillermo J. Ruiz-Arguelles, FRCP, MACP

Blood (2020) 136 (Supplement 1): 13–14.

https://doi.org/10.1182/blood-2020-134056

Published: 2020

Journal Articles

Multicenter, Retrospective Evaluation of Therapeutic Efficacy of Ruxolitinib for Chronic Gvhd Treatment

Jennifer White, Nada Hamad, MBBS,BSc,MSc,FRACP FRCPA, Swe Mar Linn, MBBS, Igor Nicolas Novitzky-Basso, MDPhDMRCP,FRCPath, Omar Abduljalil, Arjun Law, MD, Dennis Dong Hwan Kim, MD

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

https://doi.org/10.1182/blood-2020-136164

Published: 2020

Journal Articles

Immune Cell Profiles in Patients Treated with Lenalidomide and Alternate Day Prednisolone Maintenance Post Upfront ASCT for Multiple Myeloma (LEOPARD Trial)

Anna Kalff, MBBS (Hons) FRACP FRCPA, Sam Norton, PhD, Tiffany Khong, BSc, PhD, Malarmathy Ramachandran, Mary H. Young, PhD, William Pierceall, Anjan Thakurta, Udo Oppermann, PhD, Manman Guo, John Reynolds, PhD, Andrew Spencer

Blood (2020) 136 (Supplement 1): 34–35.

https://doi.org/10.1182/blood-2020-141746

Published: 2020

Journal Articles

Evaluation of Maternal Safety Following Prenatal Cell and Gene Therapy for Hemophilia a in a Large Animal Model Demonstrates Absence of Maternal Exposure to the Cells or Gene Products Infused into the Fetus

Quan DM Pham, Martin Rodriguez, BSc, Ritu M Ramamurthy, Sunil George, PhD, Jorge Figueroa, MD, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD, M. Graca Almeida-Porada, MD PhD

Blood (2020) 136 (Supplement 1): 32.

https://doi.org/10.1182/blood-2020-140883

Published: 2020

Journal Articles

Real World Experience of Etanercept in Steroid Refractory Acute Graft Versus Host Disease from a Tertiary Cancer Centre in India

Akanksha Chichra, DNB, Sachin Punatar, Anant Gokarn, MDDM, Sumeet Prakash Mirgh, MDDM, Vijay Shirure, MD DM, Vinodhini Murugaiyan, Avinash Bonda, MD DM, Lingaraj Nayak, MD DM, Bhausaheb Bagal, MD DM, Sadhana Kannan, MSc, Libin J Mathew, BSc, Tapan Saikia, MD, Navin Khattry, MD DM

Blood (2020) 136 (Supplement 1): 2–3.

https://doi.org/10.1182/blood-2020-141920

Published: 2020

Journal Articles

An Australasian Bone Marrow Transplant Registry (ABMTR) Study of the Trends and Outcomes of Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Hodgkin Lymphoma between 2009-2019: Relapse Remains the Most Common Cause of Death Post Transplantation

Kirsty M Sharplin, MBBS, David Ritchie, MB ChB, PhD FRACP, FRCPA, David Gottlieb, MBBS, MD FRACP, FRCPA, Ian Bilmon, BMBS, FRACP, FRCPA, Shyam Panicker, MBBS,MDMRCP,FRACP,FRCPA, Glen A Kennedy, MBBS FRACP FRCPA, Cameron Curley, MBBS, FRACP, FRCPA, Duncan Purtill, MBBS,FRCPA,FRACP, Julian Cooney, FRACP,FRCPA,MBBS,, Richard Doocey, MB ChB, FRACP, FRCPA, Leanne Berkahn, MD, Sam Milliken, MBBS, FRACP, FRCPA, Pietro R Di Ciaccio, BA/LLB MBBS FRACP FRCPA, Peter G Bardy, MBBS, FRACP, FRCPA, FRCPath, Andrew Spencer, David J. Curtis, MBBS,PhD, Sushrut S. Patil, Stephen R Larsen, MBBS PhDFRACP FRCPA, Travis Perera, MB ChB, FRACP, FRCPA, Andrew Butler, MB ChB(Edin), MRCP, MRCPath, Matthew Greenwood, MBBS, FRACP, FRCPA, Anne-Marie Watson, FRACP, FRCPA, MBBS, Hock Choong Lai, MBBS, FRACP, FRCPA, Eric Wong, MDPhD FRACP, FRCPA, Steven Tran, BEng MBiomedE, Nada Hamad, MBBS,BSc,MSc,FRACP FRCPA

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-138684

Published: 2020

Journal Articles

Immunocluster: A Computational Tool to Explore the Immune Profile and Cellular Heterogeneity of Hematological Diseases Using Liquid and Imaging Mass, and Flow Cytometry Data

Jessica A. Timms, James W Opzoomer, Kevin Blighe, PhD, Benedetta Apollonio, PhD, Thanos Mourikis, Nicolas Chapuis, PharmD, PhD, Richard Bekoe, Alan G. Ramsay, BSc,MSc,PhD, Donal P. McLornan, MRCP, PhD FRCPath, Claire Harrison, DM, FRCP, FRCPath, Paul R. Barber, Peter Parker, Michaela Fontenay, MD PhD, James N. Arnold, Shahram Kordasti, MD PhD

Blood (2020) 136 (Supplement 1): 9–10.

https://doi.org/10.1182/blood-2020-140258

Published: 2020

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First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Mutation Profiles Identify Distinct Clusters of Lower Risk Myelodysplastic Syndromes with Unique Clinical and Biological Features and Clinical Endpoints

Clinical Effectiveness of Combination Immunotherapy DPX-Survivac, Low Dose Cyclophosphamide, and Pembrolizumab in Recurrent/Refractory DLBCL: The Spirel Study

Donor-Derived T-Cells Specific for WT1 and PRAME in Combination with T-Cells Specific for Multiple Pathogens for Prevention of Relapse and Infection after Haemopoietic Stem Cell Transplant (HSCT) for Acute Myeloid Leukaemia (AML) or High-Risk Myelodysplasia (MDS) - (The INTACT Trial)

Clinical Validation of a NGS Capture Panel to Identify Mutations, Copy Number Variations and Translocations in Patients with Multiple Myeloma

Matching-Adjusted Indirect Comparison (MAIC) of Lisocabtagene Maraleucel (liso-cel) Vs Axicabtagene Ciloleucel (axi-cel) and Tisagenlecleucel in Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL)

A Randomized Study of Bortezomib, Cyclophosphamide and Dexamethasone Induction (VCD) Versus VCD and Daratumumab Induction Followed By Daratumumab Maintenance (VCDD) for the Initial Treatment of Transplant-Ineligible Patients with Multiple Myeloma (AMaRC 03-16)

Molecular Characterization and Clinical Outcomes of Young Adult Patients (≤45 years old) with Philadelphia-Negative Myeloproliferative Neoplasms

High Transfusion Dependence and Serum Ferritin but Not Transferrin Saturation Predict Inferior Clinical Outcomes in Patients with MDS

Implementation of Flow Cytometric Monocyte Repartition Assay for Assessment of Chronic Myelomonocytic Leukaemia (CMML) in Bone Marrow

A Phase 1b Study of Eltrombopag and Azacitidine in Patients with High Risk Myelodysplastic Syndromes and Related Disorders

Ibrutinib and Obinutuzumab in CLL: MRD Responses Sustained for Several Years with Deepest MRD Depletion in Patients with >1 Year Prior Ibrutinib Exposure

COVID-19 and Stem Cell Transplantation; Results from the Prospective Survey By the Infectious Diseases Working Party of the European Society for Blood and Marrow Transplantation (EBMT) and the Spanish Hematopoietic Stem Cell Transplantation and Cell Therapy Group (GETH)

Early Response Predicts Long-Term Improvement or Stabilization of Persons with Multiple Sclerosis Treated with Hematopoietic Stem Cell Transplantation

Multicenter, Retrospective Evaluation of Therapeutic Efficacy of Ruxolitinib for Chronic Gvhd Treatment

Immune Cell Profiles in Patients Treated with Lenalidomide and Alternate Day Prednisolone Maintenance Post Upfront ASCT for Multiple Myeloma (LEOPARD Trial)

Evaluation of Maternal Safety Following Prenatal Cell and Gene Therapy for Hemophilia a in a Large Animal Model Demonstrates Absence of Maternal Exposure to the Cells or Gene Products Infused into the Fetus

Real World Experience of Etanercept in Steroid Refractory Acute Graft Versus Host Disease from a Tertiary Cancer Centre in India

An Australasian Bone Marrow Transplant Registry (ABMTR) Study of the Trends and Outcomes of Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Hodgkin Lymphoma between 2009-2019: Relapse Remains the Most Common Cause of Death Post Transplantation

Immunocluster: A Computational Tool to Explore the Immune Profile and Cellular Heterogeneity of Hematological Diseases Using Liquid and Imaging Mass, and Flow Cytometry Data

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