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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

Emapalumab Therapy Prior to Hematopoietic Stem Cell Transplant Is Associated with Improved Long-Term Chimerism and Event-Free Survival in Pediatric Patients with Hemophagocytic Lymphohistiocytosis
Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening, hyperinflammatory disorder driven by interferon-gamma (IFN γ) mediated immunopathology. Emapalumab, an anti-IFNγ human monoclonal antibody, was approved by the FDA in 2018 for refractory/recurrent HLH. Hematopoietic stem cell transplant (HSCT) is required for cure in primary (familial) HLH and other select cases. Use of reduced intensity conditioning (RIC ) for HSCT in HLH has led to improved survival but higher incidences of mixed chimerism and graft failure. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Bethany Verkamp, Sonata Jodele, Rebecca A. Marsh, Dr. Anthony Sabulski, Stella M. Davies, Michael B. Jordan Tags: 111 Source Type: research

Real World Experience on the Use of Vedolizumab for Treatment of Steroid Refractory Lower Gastrointestinal Acute Graft Versus Host Disease in Pediatric Allogeneic Stem Cell Transplant Recipients: A Multicenter Study
Data on the use of vedolizumab (a monoclonal antibody blocking α4β7 integrin) for treatment of lower gastrointestinal (LGI) acute graft versus host disease (aGVHD) post allogeneic hematopoietic stem cell transplantation (HCT) in pediatrics is restricted to case reports or single center studies with limited subject number and variable response rates. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Sara Bowman, Tami John, Erin A Morales, Rachel Phelan, Kristine Schmeling, Hannah Lust, Gregory A. Yanik, Rolla Abu-Arja, Joseph Stanek, Jordan Milner, Biljana Horn, Hemalatha G. Rangarajan Tags: 112 Source Type: research

Elevated C3 Is Predictive, Diagnostic and Prognostic in Pediatric Transplant Associated Thrombotic Microangiopathy- a Prospective Study
Transplant associated thrombotic microangiopathy (TA-TMA) is a common and severe complication of hematopoietic cell transplantation (HCT). Diagnostic biomarkers of TA-TMA are lacking. While elevated terminal complement (C5b9) has been linked to poor prognosis, responses to eculizumab vary and biomarkers have not predicted response to eculizumab therapy. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Michelle Long Schoettler, Elyse Bryson, Laura Deeb, Kathleen Spencer, Muna Qayed, Katherine Silvis, Jayre A Jones, Seema Patel, William M Briones, Adrianna Lynn Westbrook, Kirsten Williams, Satheesh Chonat Tags: 113 Source Type: research

Lower PG-Free Mel (Evomela) Clearance and Higher Exposure Are Associated with Better Treatment Response in Newly Diagnosed Multiple Myeloma Patients Undergoing Autologous Hematopoietic Cell Transplantation
Melphalan (Alkeran) is unstable after reconstitution, preventing prolonged infusions or pharmacokinetic (PK) analysis. Propylene glycol-free melphalan (PG-free Mel; Evomela) is stable at room temperature for ∼24 hours. We studied the PK analysis of Evomela and its relationship with the disease response in patients with multiple myeloma (MM) undergoing autologous hematopoietic cell transplantation. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Jitesh D. Kawedia, Xiaoqian Liu, Dawen Sui, Alison M. Gulbis, Jeremy Ramdial, Samer A. Srour, Uday R. Popat, Yago Nieto, Partow Kebriaei, Neeraj Y. Saini, Paul Lin, Yosra Aljawai, Oren Pasvolsky, Hans C. Lee, Robert Z. Orlowski, Sheeba K. Thomas, Krina K. Tags: 114 Source Type: research

Patterns of CMV Infection after Letermovir Withdrawal in Recipients of Post-Transplant Cyclophosphamide Based Transplant
Reactivation of latent cytomegalovirus (CMV) is increased in CMV seropositive (CMV+) recipients of allogeneic hematopoietic cell transplantation (allo HCT) using post-transplant cyclophosphamide (PT-Cy) based graft versus host disease prophylaxis. Letermovir, a novel DNA terminase complex inhibitor, reduces the incidence of clinically significant CMV infection (csCMVi) in this population. We and others have demonstrated that extended use of letermovir is effective in recipients of PT-Cy; however, late csCMVi is a frequent complication that can occur after letermovir withdrawal. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Andrew Lin, Samantha Brown, Ms. Stephanie Chinapen, Yeon Joo Lee, Susan K. Seo, Doris M. Ponce, Zainab Shahid, Sergio A. Giralt, Genovefa A Papanicolaou, Miguel-Angel Perales, Brian C. Shaffer Tags: 115 Source Type: research

Defibrotide Prophylaxis after Second Hematopoietic Cell Transplant in Pediatric Patients at Very High Risk for Veno-Occlusive Disease
Veno-occlusive disease (VOD) is a serious complication after hematopoietic cell transplant (HCT). Incidence of VOD in children is ∼15-20% and particular risk factors portend some patients to be very high risk (VHR). VOD is potentially fatal in up to 85% of patients who experience severe grades of VOD. Use of defibrotide for prophylaxis can be considered for those at VHR for developing VOD, but it is expensive. The cost of tr eating VOD is significant and the disease processes may result in long-term health effects. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Susie Long, Alex Hoover, Christen L. Ebens, Ashish O. Gupta Tags: 116 Source Type: research

Preclinical Evaluation of an Anti-CD41 CAR T Cell in Acute Megakaryoblastic Leukemia on in Vitro and In Vivo Models
Acute Megakaryoblastic Leukemia (AMkL) is a rare disease that represents 5% of all reported AML cases and is diagnosed with high frequency in children with Down Syndrome and elderly people. M7-AMkL is characterized by a low overall survival and the patients have poor outcome to treatment, thus alternative treatments, such as CAR T cell therapies, are a need for better patient management.Herein, we present an evaluation of a new CAR T cell which targets CD41 marker, a specific surface antigen for M7-AMkL, in a preclinical model for AMkL using DAMI Luc2 cell line for in vitro and in vivo experiments. (Source: Biology of Bloo...
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Adrian Bogdan Tigu, Catalin Constantinescu, Patric Teodorescu, David Kegyes, Raluca Munteanu, Richard Feder, Mareike Peters, Ioana Pralea, Cristina Iuga, Diana Cenariu, Andra Marcu, Alina Daniela Tanase, Anca Colita, Rares Drula, Jon Thor Bergthorsson, Vi Tags: 117 Source Type: research