keyword
https://read.qxmd.com/read/38629074/donor-kir-genotype-based-outcome-prediction-after-allogeneic-stem-cell-transplantation-no-land-in-sight
#1
JOURNAL ARTICLE
Johannes Schetelig, Henning Baldauf, Falk Heidenreich, Jorinde D Hoogenboom, Stephen R Spellman, Alexander Kulagin, Thomas Schroeder, Henrik Sengeloev, Peter Dreger, Edouard Forcade, Jan Vydra, Eva Maria Wagner-Drouet, Goda Choi, Shankara Paneesha, Nuno A A Miranda, Alina Tanase, Liesbeth C de Wreede, Vinzenz Lange, Alexander H Schmidt, Jürgen Sauter, Joshua A Fein, Yung-Tsi Bolon, Meilun He, Steven G E Marsh, Shahinaz M Gadalla, Sophie Paczesny, Annalisa Ruggeri, Christian Chabannon, Katharina Fleischhauer
Optimizing natural killer (NK) cell alloreactivity could further improve outcome after allogeneic hematopoietic cell transplantation (alloHCT). The donor's Killer-cell Immunoglobulin-like Receptor (KIR) genotype may provide important information in this regard. In the past decade, different models have been proposed aiming at maximizing NK cell activation by activating KIR-ligand interactions or minimizing inhibitory KIR-ligand interactions. Alternative classifications intended predicting outcome after alloHCT by donor KIR-haplotypes...
2024: Frontiers in Immunology
https://read.qxmd.com/read/38443706/harmonizing-definitions-for-hematopoietic-recovery-graft-rejection-graft-failure-poor-graft-function-and-donor-chimerism-in-allogeneic-hematopoietic-cell-transplantation-a-report-on-behalf-of-the-ebmt-astct-cibmtr-and-apbmt
#2
JOURNAL ARTICLE
Anna Sureda, Paul A Carpenter, Andrea Bacigalupo, Vijaya Raj Bhatt, Josu de la Fuente, Aloysius Ho, Leslie Kean, Jong Wook Lee, Isabel Sánchez-Ortega, Bipin N Savani, Johannes Schetelig, Edward A Stadtmauer, Yoshiyuki Takahashi, Yoshiko Atsuta, John Koreth, Nicolaus Kröger, Per Ljungman, Shinichiro Okamoto, Uday Popat, Robert Soiffer, Heather E Stefanski, Mohamed A Kharfan-Dabaja
Despite emergence of novel therapies to treat hematologic malignancies, allogeneic hematopoietic cell transplantation (allo-HCT) remains an essential treatment modality capable of curing these diseases. Allo-HCT has been also shown to be curative in benign hematologic disorders such as aplastic anemia, sickle cell disease, and thalassemia, among others. Recently, the American Society for Transplantation and Cellular Therapy (ASTCT) published standardized definitions for hematopoietic recovery, graft rejection, graft failure, poor graft function, and donor chimerism...
March 5, 2024: Bone Marrow Transplantation
https://read.qxmd.com/read/38198511/incorporating-patient-reported-outcome-data-into-a-predictive-calculator-for-allogeneic-hematopoietic-cell-transplantation-recipients
#3
JOURNAL ARTICLE
Bronwen E Shaw, Kathryn E Flynn, Naya He, Rachel Cusatis, Anita D'Souza, Betty K Hamilton, Mary M Horowitz, Deborah Mattila, Rachel Phelan, Stephanie J Lee, Ruta Brazauskas
BACKGROUND: The Center for International Blood and Marrow Transplant Research (CIBMTR) provides a 1-year overall survival calculator to estimate outcomes for individual patients before they undergo allogeneic hematopoietic cell transplantation (HCT) to inform risk. The calculator considers pre-HCT clinical and demographic characteristics, but not patient-reported outcomes (PROs). Because pre-HCT PRO scores have been associated with post-HCT outcomes, the authors hypothesized that adding PRO scores to the calculator would enhance its predictive power...
January 10, 2024: Cancer
https://read.qxmd.com/read/38176404/busulfan-melphalan-and-carfilzomib-high-dose-chemotherapy-and-autologous-haematopoietic-stem-cell-transplantation-in-multiple-myeloma
#4
JOURNAL ARTICLE
Patrick Hagen, Joseph Norton, Stephanie Tsai, Loredana Campo, Mary Lee, Kayeromi Gomez, Patrick Stiff
The standard of care for fit, newly diagnosed multiple myeloma patients includes induction therapy followed by consolidative high-dose chemotherapy with melphalan and autologous stem cell transplant (AHSCT). Intensified preparative regimens, such as busulfan and melphalan (BuMel), have shown promise to lengthen progression-free survival (PFS). We previously reported that the addition of bortezomib to BuMel improved PFS compared to melphalan alone in CIBMTR-matched controls. We now integrate the second-generation protease inhibitor, carfilzomib, before and after BuMel (BuMelCar) in a phase I/II trial with carfilzomib...
January 4, 2024: British Journal of Haematology
https://read.qxmd.com/read/38127270/expanding-donor-options-haploidentical-transplant-recipients-are-also-highly-likely-to-have-a-7-8-matched-unrelated-donor
#5
JOURNAL ARTICLE
Stephanie Fingerson, Martin Maiers, Yung-Tsi Bolon, Steven M Devine, Stephen R Spellman
The use of haploidentical related donor (HRD) hematopoietic cell transplants (HCTs) in the United States grew by more than fourfold in the last decade, driven mainly by use of posttransplant cyclophosphamide (PTCy)-based graft-versus-host-disease prophylaxis. However, not all patients have a suitable HRD available. In this study, we explored the existence of unrelated donors (URDs) on the National Marrow Donor Program (NMDP) registry at the 8/8- or 7/8-match level for patients receiving HRD HCT in the United States and reporting to the Center for International Blood and Marrow Transplant Research between 2013 and 2020...
February 13, 2024: Blood Advances
https://read.qxmd.com/read/37981238/comparison-of-vital-status-cause-of-death-and-follow-up-after-hct-in-linked-cibmtr-and-california-cancer-registry-data-1991-2018
#6
JOURNAL ARTICLE
Bryan Valcarcel, Sara J Schonfeld, Christa L Meyer, Ann Brunson, Julianne J P Cooley, Renata Abrahão, Ted Wun, Jeffery J Auletta, Shahinaz M Gadalla, Eric Engels, Paul S Albert, Stephen R Spellman, J Douglas Rizzo, Bronwen E Shaw, Lori Muffly, Theresa H M Keegan, Lindsay M Morton
BACKGROUND: Assessing outcomes following hematopoietic cell transplantation (HCT) poses challenges due to the necessity for systematic and often prolonged patient follow-up. Linking the HCT database of the Center for International Blood and Marrow Transplant Research (CIBMTR) with cancer registry data may improve long-term outcome ascertainment, but the reliability of mortality data in death certificates from cancer registries among HCT recipients remains unknown. OBJECTIVES: We compared the classification of vital status and primary cause of death (COD), as well as the length of follow-up between the CIBMTR and California Cancer Registry (CCR) to assess the feasibility of supplementing the CIBMTR with cancer registry data...
November 17, 2023: Transplantation and cellular therapy
https://read.qxmd.com/read/37940716/myeloablative-or-reduced-intensity-non-myeloablative-hematopoietic-cell-transplantation-for-philadelphia-positive-acute-lymphoblastic-leukemia-in-adults-older-than-40%C3%A2-years-old-a-secondary-analysis-of-a-cibmtr-database
#7
JOURNAL ARTICLE
Irtis de Oliveira Fernandes Junior, Leonardo Javier Arcuri
Few studies have addressed the role of reduced-intensity conditioning (RIC) and non-myeloablative (NMA) regimens in older adults with Philadelphia acute lymphoblastic leukemia (Ph + ALL). The objective of this current study was to compare the outcomes of RIC/NMA versus TBI-based myeloablative (MAC) regimens in Ph + ALL patients older than 40 years old who underwent hematopoietic cell transplantation (HCT) in CR1. We used a freely available database from the CIBMTR. Transplants were performed between 2013 and 2017...
November 9, 2023: Annals of Hematology
https://read.qxmd.com/read/37844687/severity-of-chronic-graft-versus-host-disease-and-late-effects-following-allogeneic-hematopoietic-cell-transplantation-for-adults-with-hematologic-malignancy
#8
JOURNAL ARTICLE
Catherine J Lee, Tao Wang, Karen Chen, Mukta Arora, Ruta Brazauskas, Stephen R Spellman, Carrie Kitko, Margaret L MacMillan, Joseph A Pidala, Sherif M Badawy, Neel Bhatt, Vijaya R Bhatt, Zachariah DeFilipp, Miguel A Diaz, Nosha Farhadfar, Shahinaz Gadalla, Shahrukh Hashmi, Peiman Hematti, Nasheed M Hossain, Yoshihiro Inamoto, Lazaros J Lekakis, Akshay Sharma, Scott Solomon, Stephanie J Lee, Daniel R Couriel
PURPOSE: Our aim was to determine the association of chronic graft-versus-host disease (cGVHD) diagnosis and severity with the development of subsequent neoplasms (SN) and non-malignant late effects (NM-LE) in 2-year disease-free adult survivors following hematopoietic cell transplantation (HCT) for a hematologic malignancy. METHODS: We conducted a retrospective analysis of 3884 survivors of HCT for hematologic malignancy in the Center of International Blood and Marrow Transplant Research (CIBMTR) database...
October 14, 2023: Transplantation and cellular therapy
https://read.qxmd.com/read/37792849/allogeneic-hematopoietic-cell-transplantation-for-blastic-plasmacytoid-dendritic-cell-neoplasm-a-cibmtr-analysis
#9
JOURNAL ARTICLE
Hemant S Murthy, Mei-Jie Zhang, Karen Chen, Sairah Ahmed, Uday Deotare, Siddhartha Ganguly, Ankit Kansagra, Fotios V Michelis, Taiga Nishihori, Mrinal M Patnaik, Muhammad Bilal Abid, Mahmoud Aljurf, Yasuyuki Arai, Ulrike Bacher, Talha Badar, Sherif M Badawy, Karen Ballen, Minoo Battiwalla, Amer Beitinjaneh, Nelli Bejanyan, Vijaya R Bhatt, Valerie Inez Brown, Rodrigo Martino, Jean-Yves Cahn, Paul Castillo, Jan Cerny, Saurabh Chhabra, Edward A Copelan, Andrew Daly, Bhagirathbhai Dholaria, Miguel Angel Diaz, César O Freytes, Michael R Grunwald, Shahrukh Hashmi, Gerhard C Hildebrandt, Omer Jamy, Jacinth Joseph, Christopher G Kanakry, Nandita Khera, Maxwell M Krem, Yachiyo Kuwatsuka, Hillard M Lazarus, Lazaros J Lekakis, Hongtao Liu, Dipenkumar Modi, Pashna Munshi, Alberto Mussetti, Neil Palmisiano, Sagar S Patel, David A Rizzieri, Sachiko Seo, Mithun Vinod Shah, Akshay Sharma, Melhem M Solh, Scott R Solomon, Matthew L Ulrickson, Celalettin Ustun, Marjolein W M van der Poel, Leo F Verdonck, John L Wagner, Trent P Wang, Baldeep Wirk, Amer M Zeidan, Mark R Litzow, Partow Kebriaei, Christopher S Hourigan, Daniel J Weisdorf, Wael Saber, Mohamed A Kharfan-Dabaja
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy with a poor prognosis and considered incurable with standard conventional chemotherapy. Small observational studies have shown that allogeneic hematopoietic cell transplantation (allo-HCT) offers durable remissions in patients with BPDCN. We conducted an analysis of 164 patients with BPDCN from 78 centers who underwent allo-HCT between 2007-2018 using data reported to the Center for International Blood and Marrow Transplant Research (CIBMTR)...
October 4, 2023: Blood Advances
https://read.qxmd.com/read/37785563/real-world-utilization-of-cranial-or-craniospinal-radiation-in-adult-acute-lymphoid-leukemia-patients-data-from-the-center-for-international-blood-and-marrow-transplant-research
#10
JOURNAL ARTICLE
S C Zhang, G Schmidt, R L Paquette, B Hakimian, L K Ballas
PURPOSE/OBJECTIVE(S): Though CNS involvement at diagnosis in adult acute lymphoid leukemia (ALL) is relatively rare (3-7%), >50 % of patients will develop CNS relapse without CNS-directed therapy. Radiation therapy is commonly used as part of stem cell transplant (SCT) conditioning. Historically, a cranial boost was given to reduce risk of CNS relapse. While a CNS boost is not a universal part of prophylaxis, CNS directed RT is used in patients at highest risk for CNS relapse following transplantation, typically those with CNS disease at diagnosis or after induction...
October 1, 2023: International Journal of Radiation Oncology, Biology, Physics
https://read.qxmd.com/read/37775070/incidence-and-impact-of-fungal-infections-in-post-transplantation-cyclophosphamide-based-graft-versus-host-disease-prophylaxis-and-haploidentical-hematopoietic-cell-transplantation-a-center-for-international-blood-and-marrow-transplant-research-analysis
#11
JOURNAL ARTICLE
Genovefa A Papanicolaou, Min Chen, Naya He, Michael J Martens, Soyoung Kim, Marjorie V Batista, Neel S Bhatt, Peiman Hematti, Joshua A Hill, Hongtao Liu, Sunita Nathan, Matthew D Seftel, Akshay Sharma, Edmund K Waller, John R Wingard, Jo-Anne H Young, Christopher E Dandoy, Miguel-Angel Perales, Roy F Chemaly, Marcie Riches, Celalettin Ustun
UNLABELLED: Fungal infection (FI) after allogeneic hematopoietic cell transplantation (HCT) is associated with increased morbidity and mortality. Neutropenia, HLA mismatch, graft-versus-host disease (GVHD), and viral infections are risk factors for FI. The objectives of this Center for International Blood and Marrow Transplant Research registry study were to compare the incidence and density of FI occurring within 180 days after HCT in matched sibling (Sib) transplants with either calcineurin inhibitor (CNI)-based or post-transplantation cyclophosphamide (PTCy)-based GVHD prophylaxis and related haploidentical transplants receiving PTCy, and to examine the impact of FI by day 180 on transplantation outcomes...
January 2024: Transplantation and cellular therapy
https://read.qxmd.com/read/37773924/unfavorable-transcriptome-profiles-and-social-disadvantage-in-hematopoietic-cell-transplantation-a-cibmtr-analysis
#12
JOURNAL ARTICLE
Mallory Taylor, Steve W Cole, Joelle Strom, Ruta Brazauskas, K Scott Baker, Rachel Phelan, David K Buchbinder, Betty K Hamilton, Hélène M Schoemans, Bronwen E Shaw, Akshay Sharma, Neel S Bhatt, Sherif M Badawy, Lena E Winestone, Jaime M Preussler, Samantha J Mayo, Kareem Jamani, Taiga Nishihori, Michelle A Lee, Jennifer M Knight
Patient-reported outcomes (PROs) capture subjective social determinants of health (SDOH), which can affect health outcomes through the stress response pathway. The Conserved Transcriptional Response to Adversity (CTRA) is a stress-mediated pro-inflammatory transcriptomic pattern that has been linked to adverse hematopoietic cell transplant (HCT) outcomes. This study examined the association of pre-transplant CTRA with patient-reported SDOH in allogeneic HCT recipients. In this cross-sectional study, pre-HCT SDOH-related PROs included the 36-Item Short Form Health Survey (SF-36) and the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT)...
September 29, 2023: Blood Advances
https://read.qxmd.com/read/37748539/haplo-cord-transplantation-with-8mg-kg-anti-thymocyte-globulin-as-graft-versus-host-disease-prophylaxis-compared-to-haploidentical-transplantation-with-10mg-kg-anti-thymocyte-globulin-in-the-treatment-of-acute-leukemia
#13
JOURNAL ARTICLE
Fangfang Yuan, Gangping Li, Minghui Li, Xudong Wei, Yuewen Fu
BACKGROUND: Clinical outcomes of the transplantation strategy combined with a haploidentical stem cell graft and an unrelated umbilical cord blood unit (haplo-cord HSCT) with low-dose anti-thymocyte globulin as graft-versus-host disease prophylaxis for the treatment of acute leukemia (AL) remains unclear. OBJECTIVE: This study aimed to explore the clinical outcomes of haplo-cord HSCT in acute leukemia patients with the GVHD prevention strategy of 8 mg/kg ATG compared with haplo- transplantation with 10 mg/kg ATG...
September 23, 2023: Transplantation and cellular therapy
https://read.qxmd.com/read/37741763/long-term-outcomes-after-hematopoietic-cell-transplant-in-peripheral-t-cell-lymphoma-the-oregon-health-and-science-university-experience
#14
JOURNAL ARTICLE
Derek Galligan, Staci Williamson, Jessie Myers, Andy I Chen, Brandon Hayes-Lattin, Craig Okada, Stephen Spurgeon, Richard Maziarz, Levanto Schachter
BACKGROUND: Peripheral T-cell lymphomas (PTCL) are a group of aggressive malignancies with inferior outcomes compared to B-cell non-Hodgkin lymphoma (NHL). Both allogeneic and autologous hematopoietic cell transplantation (HCT) are commonly employed for consolidation and salvage. MATERIALS AND METHODS: We conducted a single-center review of all adult PTCL patients at OHSU who received HCT from 1991 to 2020 with responses assed by CIBMTR criteria. RESULTS: 88 patients (autoHCT = 52, alloHCT = 36) were identified from the internal registry of ∼3800 autoHCT & alloHCT recipients in that time period...
September 12, 2023: Clinical Lymphoma, Myeloma & Leukemia
https://read.qxmd.com/read/37577706/critical-illness-risk-and-long-term-outcomes-following-intensive-care-in-pediatric-hematopoietic-cell-transplant-recipients
#15
Matt S Zinter, Ruta Brazauskas, Joelle Strom, Stella Chen, Stephanie Bo-Subait, Akshay Sharma, Amer Beitinjaneh, Dimana Dimitrova, Greg Guilcher, Jaime Preussler, Kasiani Myers, Neel S Bhatt, Olle Ringden, Peiman Hematti, Robert J Hayashi, Sagar Patel, Satiro Nakamura De Oliveira, Seth Rotz, Sherif M Badawy, Taiga Nishihori, David Buchbinder, Betty Hamilton, Bipin Savani, Hélène Schoemans, Mohamed Sorror, Lena Winestone, Christine Duncan, Rachel Phelan, Christopher C Dvorak
BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) can be complicated by the development of organ toxicity and infection necessitating intensive care. Risk factors for intensive care admission are unclear due to heterogeneity across centers, and long-term outcome data after intensive care are sparse due to a historical paucity of survivors. METHODS: The Center for International Blood and Marrow Transplant Research (CIBMTR) was queried to identify patients age ≤21 years who underwent a 1 st allogeneic HCT between 2008-2014 in the United States or Canada...
August 5, 2023: medRxiv
https://read.qxmd.com/read/37577695/measurable-residual-idh1-before-allogeneic-transplant-for-acute-myeloid-leukemia
#16
Gege Gui, Laura W Dillon, Niveditha Ravindra, Pranay S Hegde, Georgia Andrew, Devdeep Mukherjee, Zoë Wong, Jeffery Auletta, Firas El Chaer, Evan Chen, Yi-Bin Chen, Adam Corner, Steven M Devine, Sunil Iyer, Antonio Martin Jimenez Jimenez, Marcos J G De Lima, Mark R Litzow, Partow Kebriaei, Stephen R Spellman, Scott L Zeger, Kristin M Page, Christopher S Hourigan
Measurable residual disease (MRD) in adults with acute myeloid leukemia (AML) in complete remission is an important prognostic marker, but detection methodology requires optimization. The persistence of mutated NPM1 or FLT3 -ITD in the blood of adult patients with AML in first complete remission (CR1) prior to allogeneic hematopoetic cell transplant (alloHCT) has been established as associated with increased relapse and death after transplant. The prognostic implications of persistence of other common AML-associated mutations, such as IDH1 , at this treatment landmark however remains incompletely defined...
August 1, 2023: medRxiv
https://read.qxmd.com/read/37517612/a-pilot-study-of-donor-engrafted-clonal-hematopoiesis-evolution-and-clinical-outcomes-in-allogeneic-hematopoietic-cell-transplant-recipients-using-a-national-registry
#17
JOURNAL ARTICLE
Nancy Gillis, Eric Padron, Tao Wang, Karen Chen, Jakob D DeVos, Stephen R Spellman, Stephanie J Lee, Carrie L Kitko, Margaret L MacMillan, Jeffrey West, Yi-Han Tang, Mingxiang Teng, Samantha McNulty, Todd E Druley, Joseph A Pidala, Aleksandr Lazaryan
BACKGROUND: Improved treatment options, such as reduced-intensity conditioning, enable older patients to receive potentially curative allogeneic hematopoietic cell transplantation (HCT). This progress has increased use of older HLA-matched sibling donors. An unintended potential risk associated with older donors is transplantation of donor cells with clonal hematopoiesis (CH) into patients. OBJECTIVE: We aimed to determine the prevalence of CH in older HLA-matched sibling donors pre-transplant and to assess the clinical impact of donor-engrafted CH on HCT outcomes...
July 28, 2023: Transplantation and cellular therapy
https://read.qxmd.com/read/37482244/racial-and-socioeconomic-disparities-in-long-term-outcomes-in-%C3%A2-1-year-allogeneic-hematopoietic-cell-transplantation-survivors-a-cibmtr-analysis
#18
JOURNAL ARTICLE
Brandon J Blue, Ruta Brazauskas, Karen Chen, Jinalben Patel, Amer M Zeidan, Amir Steinberg, Karen Ballen, Janette Kwok, Seth J Rotz, Miguel Angel Diaz Perez, Amar H Kelkar, Siddhartha Ganguly, John R Wingard, Deepesh Lad, Akshay Sharma, Sherif M Badawy, Hillard M Lazarus, Hasan Hashem, David Szwajcer, Jennifer M Knight, Neel S Bhatt, Kristin Page, Sara Beattie, Yasuyuki Arai, Hongtao Liu, Staci D Arnold, César O Freytes, Muhammad Bilal Abid, Amer Beitinjaneh, Nosha Farhadfar, Baldeep Wirk, Lena E Winestone, Vaibhav Agrawal, Jaime M Preussler, Sachiko Seo, Shahrukh Hashmi, Leslie Lehmann, William A Wood, Hemalatha G Rangarajan, Wael Saber, Navneet S Majhail
Racial/ethnic minorities have demonstrated worse survival after allogeneic hematopoietic cell transplantation (HCT) compared to whites. Whether the racial disparity in HCT outcomes persists in long-term survivors and possibly may be even exacerbated in this population, which frequently transitions back from the transplant center to their local healthcare providers, is unknown. In the current study, we compared long-term outcomes among 1-year allogeneic HCT survivors by race/ethnicity and socioeconomic status (SES)...
November 2023: Transplantation and cellular therapy
https://read.qxmd.com/read/37481243/younger-matched-unrelated-donors-confer-decreased-relapse-compared-to-older-sibling-donors-in-older-b-cell-all-patients-undergoing%C3%A2-allogeneic-hematopoietic-cell-transplantation
#19
JOURNAL ARTICLE
Muhammad Bilal Abid, Noel Estrada Merly, Mei-Jie Zhang, Karen Chen, Christopher Bredeson, David Allan, Mitchell Sabloff, David I Marks, Mark Litzow, Christopher Hourigan, Partow Kebriaei, Wael Saber
INTRODUCTION: While allogeneic hematopoietic cell transplant (alloHCT) offers cures for older patients with acute lymphoblastic leukemia (ALL), disease relapse remains a major issue. Whether matched sibling donors (MSD) are still the preferred donor choice compared to younger matched unrelated donors (MUD), in the contemporary era of improved transplant practices, remains unknown. METHODS: This retrospective cohort registry study queried the Center for International Blood and Marrow Transplant Research database (CIBMTR) data in B-cell ALL patients 50 years or older, undergoing alloHCT from older MSDs (donor age ≥ 50) or younger MUDs (donor age ≤ 35) between 2011 and 2018...
July 20, 2023: Transplantation and cellular therapy
https://read.qxmd.com/read/37481241/inspired-symposium-part-1-clinical-variables-associated-with-improved-outcomes-for-children-and-young-adults-treated-with-chimeric-antigen-receptor-t-cells-for-b-cell-acute-lymphoblastic-leukemia
#20
REVIEW
Regina M Myers, Elad Jacoby, Michael A Pulsipher, Marcelo C Pasquini, Stephan A Grupp, Nirali N Shah, Theodore W Laetsch, Kevin J Curran, Liora M Schultz
Chimeric antigen receptor (CAR) T cell therapy (CAR-T) targeting the CD19 antigen on B cell acute lymphoblastic leukemia (B-ALL) has transitioned from a highly investigational therapy with limited access to a commercial therapy with established toxicities, response and survival rates, and access in numerous countries. With more than a decade of clinical study and 5 years of commercial access, data showing associations with success and failure have emerged. To address functional limitations of CAR-T and overcome constrained sample sizes when studying single-trial or single-center data, collaborative groups, including the Pediatric Real World CAR Consortium, the CAR-Multicenter Analysis, the Center for International Blood and Marrow Transplant Research, and the International BFM Study Group, among others, have been retrospectively interrogating the amassed clinical experience...
October 2023: Transplantation and cellular therapy
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