Programmatic Data Analyst Continuous Process Improvement (CPI) Tracking within the Sarah Cannon Transplant and Cellular Therapy Network (SCTCTN)
The SCTCTN is compromised of 7 blood cancer and cellular therapy programs that collectively performed over 1600 transplant and cellular therapies in 2022. Making the SCTCTN the largest network in the United States. In September 2022, the SCTCTN began tracking the CPI status of each program with the goal of strategizing programmatic form completion, aimed at maximizing resource utilization for CPI planning. The CPI report represents forms that are due and their priority. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Hector Zambrano, Tonya Cox, Rocky Billups, Navneet S. Majhail, Betsy Blunk Tags: 96 Source Type: research

Project of a Dashboard Using DATA from DATA Back to Center for Affiliated Centers with CIBMTR
Hematopoietic stem cell transplantation and other cellular therapy (HSCT/CT) researchers need to know the results of this treatment to provide greater scientific production and national benchmarking. The partnership between the Brazilian Cellular Therapy and Bone Marrow Transplant Society (SBTMO) and CIBMTR have allowed each affiliated Brazilian centers to know some results in a visual format or uploading their registered data, through Data Back to Center (DBtC) on the CIBMTR portal. However, Brazilian researches need to know other results, including survival analysis with comparative curves. (Source: Biology of Blood and ...
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Ms. Heliz Regina A. Neves, Anderson Jo ão Simioni, Cinthya Corrêa Silva, Adriana Mendes Cavilha, Bruna Letícia da Silva Santos Geraldo, Ms. Flávia Ferreira Costa, Paula Moreira da Silva Sabaini, Cristiano de Oliveira Ribeiro, Monique S Ammi, Antonio M Tags: 97 Source Type: research

Sars-Cov-2 Vaccination in the First Year after Hematopoietic Cell Transplant or Chimeric Antigen Receptor T Cell Therapy: A CIBMTR and BMT CTN Study
We previously reported the immunogenicity of SARS-CoV-2 vaccination within the first year after allogeneic hematopoietic cell transplant (HCT) from CIBMTR SC21-07/BMT CTN 2101. We herein report updated results from the full allogeneic HCT cohort in addition to the autologous HCT and CAR-T cell therapy cohorts. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Joshua A. Hill, Michael J. Martens, Jo-Anne H. Young, Kavita Bhavsar, Jianqun Kou, Min Chen, Lik Wee Lee, Aliyah Baluch, Madhav Vishnu Dhodapkar, Ryotaro Nakamura, Kristin Peyton, Zainab Shahid, Paul M. Armistead, Peter Westervelt, John M. McCarty, Joseph Tags: 98 Source Type: research

ALVR106, an Off-the-Shelf, Multivirus-Specific T-Cell Therapy, for the Treatment of Respiratory Viral Infections: Results from a Phase 1, First-in-Human, Dose-Ranging Trial
Community-acquired respiratory viruses are leading causes of morbidity and mortality after hematopoietic cell transplant (HCT) or solid organ transplant (SOT).ALVR106, an allogeneic, off-the-shelf, polyclonal, multi-respiratory virus T-cell therapy specific for influenza (Flu), human metapneumovirus (hMPV), parainfluenza virus (PIV), and respiratory syncytial virus (RSV), is generated by exposing PBMCs to viral peptides with activating cytokines. The resulting cells are Th1-polarized, polyfunctional, and selectively able to kill viral target cells. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Sarah Nikiforow, Mitchell Horowitz, John M. McCarty, Anum Abbas, James Coghill, Jenny Stanton, Julie Ma, Spyridoula Vasileiou, Sarah Gilmore, Dr. Bhagirathbhai Dholaria, Rabi Hanna, Randy Allison Taplitz, Rajat Bansal Tags: 99 Source Type: research

Single Cell Rnaseq Identifies Chemokine Responses to BK Polyomavirus Infection and Novel Contribution of MAIT Cells
In this study, we hypothesized that differences in endothelial cell and host immune responses to BKPyV viremia influence BKPyV disease. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Nathan Luebbering, Sheyar Abdullah, Krishna Roskin, Aaron Webster, Jason T Blackard, Elizabeth Odegard, Benjamin Laskin, Alix E. Seif, Sonata Jodele, Stella M. Davies, Dr. Anthony Sabulski Tags: 100 Source Type: research

DT Why: Learning Health Systems Demonstrate Rich Data Benefits
The CIBMTR Data Transformation Initiative (DTI) has been a frequent presentation topic at Tandem, often in the context of increasing the ef{ciency of satisfying a center's reporting requirements to the CIBMTR.Appropriate, as the burden of data collection is a concern for every center, and any initiative with the goal of increasing the volume or frequency of reported data must address this barrier if it has any chance of being widely adopted. DTI has never just been about ef{ciency but also about increasing the richness and frequency of reporting, enabling powerful new use cases for research, and opening up new possibilitie...
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: John Huber Tags: 101 Source Type: research

Cellular Therapy Data Exchange: Bridging the Gaps to Accelerate Innovation
Cellular therapy is a rapidly evolving field with the potential to revolutionize the treatment of a wide range of diseases. However, the development and implementation of cellular therapies is complex and challenging. One of the key challenges is the need to exchange data efficiently and securely between the different stakeholders involved in cellular therapy, including clinicians, cell and gene therapy laboratories, manufacturers, and regulators.In this presentation, we will discuss the challenges, successes, and lessons learned from implementing cellular therapy data exchange solutions. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Mike Lujan Tags: 102 Source Type: research

Abstract Title: Cell & Gene Therapy Information Systems and Analytics Frameworkabstract Description: Implementing a Strong and Sustainable Information Systems and Analytics Framework for Cell & Gene Therapy
• Abstract Title: Cell& Gene Therapy Information Systems and Analytics Framework (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Dinesh Keerthi Tags: 103 Source Type: research

Characterization of Extramedullary Disease (EMD) Response to CD19 Targeted Chimeric Antigen Receptor T-Cells (CART) in Patients with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (B-ALL): A Multi-Site, Retrospective Cohort Review
CD19 CART are highly effective in eradicating bone marrow (BM) B-ALL, but efficacy in EMD is less clear. Recent data suggests that response in central nervous system (CNS) disease may be comparable to BM disease without increased toxicity. Data using CART in non-CNS EMD is limited to small retrospective analyses with demonstration of mixed BM/EMD responses. An improved understanding of CART efficacy in EMD is needed to help inform management decisions. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Alexander W. Rankin, Regina M. Myers, Adam Lamble, Emily M. Hsieh, Sara Silbert, Seth M. Steinberg, Colleen Annesley, Bonnie Yates, Samuel John, Jennifer Sheppard, Lee Chen, Daniel W. Lee, Amanda DiNofia, Stephan A. Grupp, Lia Gore, Mike A. Pulsipher, Nir Tags: 104 Source Type: research

Optimal Rabbit Antithymocyte Pharmacokinetics (PK) Is Associated with Improved Disease-Free Survival (DFS) in TCR- Αβ/CD19-Depleted Pediatric Haploidentical Hematopoietic Cell Transplantation for Hematologic Malignancies
Rabbit antithymocyte globulin (rATG; Thymoglobulin ®) is often included as part of conditioning prior to TCR-αβ/CD19-T-cell depleted (AB-TCD) haploidentical hematopoietic cell transplantation (haplo-HCT) for pediatric patients with hematologic malignancies. PK models demonstrate that weight-based dosing of rATG generally results in higher exposur e pre- and post-HCT for older patients. We hypothesized that the inferior DFS noted in patients ≥10 years of age undergoing AB-TCD haploidentical HCT in our earlier manuscript (Pulsipher, Blood 2022) was due to excessive rATG exposure. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Christopher C. Dvorak, Lucia Holbrook-Brown, Janel Renee Long-Boyle, Hisham Abdel-Azim, Alice Bertaina, Anant Vatsayan, Julie A Talano, Nancy J. Bunin, Eric J Anderson, Allyson Flower, Nahal Lalefar, Christine S. Higham, Neena Kapoor, Maryanne A Odinakach Tags: 105 Source Type: research

Preliminary Results of a Phase 2a Clinical Trial to Evaluate Safety, Tolerability and Antiviral Activity of Intravenous Brincidofovir (BCV IV) in Immunocompromised Patients with Adenovirus Infection
Adenovirus (AdV) may cause fatal infections in immunosuppressed patients including recipients of allogeneic hematopoietic cell transplant. Brincidofovir (BCV) is a lipid conjugate of cidofovir, that crosses cell membranes and has a long intracellular half-life. The safety, tolerability and antiviral activity of intravenous BCV (BCV IV) were evaluated in a dose ascending Phase 2a ATHENA study (NCT04706923). (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Michael Grimley, Gabriela M. Mar ón, Carlos A Gomez, Vinod K Prasad, Jasmeen Dara, Thomas Lion, Genovefa A Papanicolaou, Koji Fukushima, Robert F Wynn, Michael J. Boeckh Tags: 106 Source Type: research

Targeting Ewing Sarcoma By Genetically and Epigenetically Modified Ex-Vivo Expanded NK Cells in Combination with NKTR-255 and Dinutuximab
Metastatic Ewing sarcoma (ES) has a dismal prognosis, largely due to therapy resistance within the tumor microenvironment (TME). NK cell resistance in solid tumors is mainly due to the small number and lack of specific targeting of NK cells, poor NK cell function and persistence, TGF β-mediated immune suppression, and inability of NK cells to infiltrate the tumor [1]. IL1RAP is a novel immunotherapy target highly expressed on ES cells [2]. Addition of TGFβ during NK expansion epigenetically reprograms NK cells (TGFβi-NK) and enhances NK-specific lysis of tumor cells [3]. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Wen Luo, Hai-Feng Zhang, Wei Li, Hai Hoang, Yaya Chu, Ronan Gandhi, Wiebke Chemnitz, Janet Ayello, Renata Scopim-Ribeiro, Michael M Lizardo, Melanie Rouleau, Dimiter S Dimitrov, Dean Anthony Lee, Poul H Sorensen, Mitchell S. Cairo Tags: 107 Source Type: research

Expression of a Novel Chimeric Inhibitory Receptor Attenuates CD19 CAR T Cells: A Promising Strategy for Toxicity Mitigation
CAR T cells targeting CD19 are highly effective in patients with refractory/relapsed acute lymphoblastic leukemia and lymphoma but often trigger severe cytokine release syndrome (sCRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). sCRS occurs when hyperactivated CAR T cells undergo exponential expansion accompanied by the release of excessive amounts of inflammatory cytokines. To mitigate these toxicities, we sought to develop a novel auto-regulatory element in the form of a chimeric inhibitory receptor (CIR), that when engaged by a specific CRS-associated pro-inflammatory molecule will trigger inhibi...
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Dustin Cobb, Philip Mollica, Lixia Liu, Daniel W. Lee Tags: 108 Source Type: research

Model-Based Dosing of Anti-Thymocyte Globulin in Pediatric Allogeneic Hematopoietic Cell Transplantation Improves Survival Chances: Updated Results from the Single Arm Phase II Parachute-Trial Combined with Real-World Data
Anti-thymocyte globulin (ATG) is used in the conditioning for pediatric hematopoietic cell transplantation (HCT) to prevent graft-versus-host-disease (GvHD) and graft failure (GF). Toxicity includes poor T-cell recovery, associated with viral reactivations and higher mortality. The PARACHUTE-trial1 (non-randomized prospective trial investigating model-based dosing [MBD] compared to historical fixed dose of ATG Thymoglobulin) showed that MBD of ATG improves T-cell recovery compared to fixed dosing, while maintaining an anti-GvHD effect. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Rick Admiraal, Stefan Nierkens, Marc Bierings, Mirjam Belderbos, Alwin D.R. Huitema, Robbert Bredius, Yilin Jiang, Kevin J. Curran, Andrew C. Harris, Andromachi Scaradavou, Maria I. Cancio, Elizabeth Klein, Wouter Kollen, Dorine Bresters, Friso Calkoen, A Tags: 109 Source Type: research

Outcomes Following Matched Sibling Donor Transplant for Severe Combined Immunodeficiency: A Report from the Pidtc
Hematopoietic cell transplant (HCT) for SCID using an HLA-matched sibling donor (MSD) is associated with excellent survival and low risk of morbidity and GVHD. In the largest cohort of MSD outcomes in SCID patients to date, we examined the impact on survival and immune recovery of 1) use of a conditioning regimen and 2) GVHD prophylaxis approach. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Ahmad Rayes, Brent Logan, Xuerong Liu, Jasmeen Dara, Rebecca H. Buckley, Joseph H. Oved, Neena Kapoor, Malika Kapadia, Sharat Chandra, Caridad Martinez, Nancy J. Bunin, Shanmuganathan Chandrakasan, Lauri M. Burroughs, Jeffrey J Bednarski, Hilary Haines, G Tags: 110 Source Type: research