Explanipedia

Effect of isatuximab use in combination with autologous stem cell transplant on mobilized stem cell product and post-transplant immune recovery - Results of a randomized prospective Phase 2 trial Open

Divaya Bhutani, Shami Chakrabarti, Michael S. Hughes, Markus Y. Mapara, Ran Reshef · 2025

Background: Autologous stem cell transplantation (ASCT) in hematological malignancies remains an important therapy for eligible patients with multiple myeloma (MM) and non-Hodgkin and Hodgkin lymphomas (NHL and HD). Favorable clinical outc…

Fludarabine pharmacokinetics and dynamic immune monitoring as predictive biomarkers for CD19 and BCMA CAR-T Open

Hei Ton Chan, Pawel Muranski, Tamna Wangjam, Markus Y. Mapara · 2025

Introduction: The optimal use of fludarabine-based lymphodepletion (LD) and the requisite degree of lymphopenia for chimeric antigen receptor (CAR) T-cell efficacy and toxicity are not well-defined across different targets. We analyzed the…

Improvements in Health-Related Quality of Life in Patients with Transfusion-Dependent β-Thalassemia After Exagamglogene Autotemcel Open

Josu de la Fuente, Haydar Frangoul, Peter Lang, Donna A. Wall, Roland Meisel , et al. · 2025

Transfusion-dependent β-thalassemia (TDT) can have negative impacts on a patient's health-related quality of life (HRQoL). Exagamglogene autotemcel (exa-cel) is a one-time, ex vivo CRISPR-Cas9 gene-edited cell therapy for TDT shown in a Ph…

Improvements in Health-Related Quality of Life in Patients with Severe Sickle Cell Disease After Exagamglogene Autotemcel Open

Akshay Sharma, Franco Locatelli, Monica Bhatia, Lyndsay Molinari, Markus Y. Mapara , et al. · 2025

Severe sickle cell disease (SCD), an inherited blood disorder characterized by recurrent vaso-occlusive crises (VOCs), has substantial negative impact on health-related quality of life (HRQoL). Exagamglogene autotemcel (exa-cel), a one-tim…

Life‐Threatening Cytopenias in a Jehovah's Witness Following CD19‐Directed Chimeric Antigen Receptor T Cell Therapy Open

Corinne Shamehdi, Christian Gordillo, Ahmed Sawas, Rodney Macedo, Markus Y. Mapara , et al. · 2025

Here we report on a Jehovah's Witness (JW) patient who experienced profound and prolonged life‐threatening cytopenias following CD19‐targeted CAR T cell therapy, successfully managed with bloodless medicine strategies. This case highlights…

Long-term follow-up of patients with AL amyloidosis treated on a phase 1 trial of CAEL-101 Open

Michael Sang Hughes, Samuel S Pan, Rajshekhar Chakraborty, J. Raikhelkar, Matthew J. Maurer , et al. · 2025

GCK inhibition enhances iberdomide antimyeloma effects by promoting IKZF1 degradation via a CRBN-independent mechanism Open

Shirong Li, Josefine Krüger, Guifen Liu, Huihui Ma, Michael Sang Hughes , et al. · 2025

Our recent study identifies germinal center kinase (GCK) as a novel therapeutic target in RAS-mutated multiple myeloma (MM). Inhibiting GCK downregulates critical transcriptional factors, notably IKZF1/3, BCL-6, and c-MYC proteins, leading…

Spatiotemporal Single-Cell Analysis Reveals T Cell Clonal Dynamics and Phenotypic Plasticity in Human Graft-versus-Host Disease Open

Lingting Shi, Ajna Uzuni, Ximi K. Wang, Michael Pressler, David W Harle , et al. · 2025

SUMMARY Allogeneic hematopoietic cell transplantation (alloHCT) is curative for various hematologic diseases but often leads to acute graft-versus-host disease (GVHD), a potentially life-threatening complication. We leverage GVHD as a uniq…

Emapalumab for severe cytokine release syndrome in solid tumor CAR-T: a case report Open

Tyler Ruemmele, Rodney Macedo, Mark N. Stein, Hei Ton Chan, Markus Y. Mapara , et al. · 2025

Chimeric Antigen Receptor T (CAR-T) cell therapy significantly and rapidly changed the treatment paradigm for lymphoma, myeloma and leukemia, and the recent approvals of the first cellular immunotherapies in melanoma and synovial sarcoma d…

Diffuse lung involvement as a rare presentation of systemic AL amyloidosis: a retrospective cohort study Open

Amy Song, George Mellgard, Claudia Bellofiore, Marco Basset, Mario Nuvolone , et al. · 2025

Timing and outcomes of second‐line therapy in the era of daratumumab‐based frontline therapy in AL amyloidosis Open

Abdul Hamid Bazarbachi, Divaya Bhutani, Jai Radhakrishnan, Markus Y. Mapara, Mathew S. Maurer , et al. · 2024

The therapeutic goal in immunoglobulin light-chain amyloidosis (AL) is to eradicate the plasma cell (PC) clone producing misfolded amyloid fibrils. Therapies for AL often draw inspiration from successful treatments in multiple myeloma (MM)…

Lovotibeglogene Autotemcel Gene Therapy for Sickle Cell Disease: 60 Months Follow-up Open

Julie Kanter, Anjulika Chawla, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh , et al. · 2024

Presentation Date: 6/8/2024 Presentation Start Time: 8:45:00 AM Background Lovotibeglogene autotemcel (lovo-cel) gene therapy uses autologous transplantation of hematopoietic stem and progenitor cells (HSPC) transduced with the BB305 lenti…

Absolute lymphocyte count after BCMA CAR-T therapy is a predictor of response and outcomes in relapsed multiple myeloma Open

Mateo Mejía Saldarriaga, Darren Pan, Caitlin Unkenholz, Tarek H. Mouhieddine, Juan Esteban Velez-Hernandez , et al. · 2024

B-cell maturation antigen (BCMA)–targeting chimeric antigen receptor T cells (CAR-Ts) used in multiple myeloma (MM) are rapidly becoming a mainstay in the treatment of relapsed/refractory (R/R) disease, and CAR-T expansion after infusion h…

Absence of Stat1 in donor CD4+ T cells promotes the expansion of Tregs and reduces graft-versus-host disease in mice Open

Huihui Ma, Caisheng Lu, Judith Ziegler, Ailing Liu, Antonia R. Sepulveda , et al. · 2024

Amplification of Signal on Cell Surfaces in Molecular Cascades Open

Sergei Rudchenko, Steven K. Taylor, Nenad Milosavić, M. N. Rudchenko, Betina Wedderhoff Tissi , et al. · 2023

We can formulate mixtures of oligonucleotide–antibody conjugates to act as molecular cascade-based automata that analyze pairs of cell surface markers (CD markers) on individual cells in a manner consistent with the implementation of Boole…

Reduced early mortality with <span>Daratumumab‐based</span> frontline therapy in <span>AL</span> amyloidosis: A retrospective cohort study Open

Rajshekhar Chakraborty, Divaya Bhutani, Markus Y. Mapara, Ran Reshef, Matthew J. Maurer , et al. · 2023

In the past decade, significant advancements in therapies targeting plasma cell clone have brought about a transformative shift in the treatment landscape for immunoglobulin light chain (AL) amyloidosis. Despite these strides, early mortal…

The prospect of universal coronavirus immunity: characterization of reciprocal and non-reciprocal T cell responses against SARS-CoV2 and common human coronaviruses Open

Mithil Soni, Edoardo Migliori, Jianing Fu, Amer Assal, Hei Ton Chan , et al. · 2023

T cell immunity plays a central role in clinical outcomes of Coronavirus Infectious Disease 2019 (COVID-19) and T cell-focused vaccination or cellular immunotherapy might provide enhanced protection for some immunocompromised patients. Pre…

S270: TRANSFUSION INDEPENDENCE AFTER EXAGAMGLOGENE AUTOTEMCEL IN PATIENTS WITH TRANSFUSION-DEPENDENT ΒETA-THALASSEMIA Open

Franco Locatelli, Peter Lang, Selim Corbacioglu, Amanda M Li, Josu de la Fuente , et al. · 2023

Topic: 27. Thalassemias Background: Exagamglogene autotemcel (exa-cel) is a one-time, non-viral cell therapy designed to reactivate fetal hemoglobin (HbF) via ex vivo CRISPR/Cas9 gene-editing at the erythroid enhancer region of BCL11A in a…

PB2151: CLINICAL FEATURES AND OUTCOMES OF DIFFUSE PULMONARY LIGHT CHAIN AMYLOIDOSIS: A RARE PRESENTATION OF SYSTEMIC AL AMYLOIDOSIS Open

Rajshekhar Chakraborty, Claudia Bellofiore, Marco Basset, Mario Nuvolone, Andrea Foli , et al. · 2023

Topic: 14. Myeloma and other monoclonal gammopathies - Clinical Background: Systemic light chain (AL) amyloidosis can involve lungs in 3 different ways: nodular, tracheobronchial, or diffuse. While the natural history of localized amyloid …

S264: EDIT-301 SHOWS PROMISING PRELIMINARY SAFETY AND EFFICACY RESULTS IN THE PHASE I/II CLINICAL TRIAL (RUBY) OF PATIENTS WITH SEVERE SICKLE CELL DISEASE USING HIGHLY SPECIFIC AND EFFICIENT ASCAS12A ENZYME Open

Rabi Hanna, Haydar Frangoul, Christopher Mckinney, Luis Piñeiro, Markus Y. Mapara , et al. · 2023

Background: EDIT-301, an investigational gene-edited autologous hematopoietic stem cell medicine, has a unique genomic modification at the γ-globin gene (HBG1/HBG2) promoters where multiple naturally occurring mutations for hereditary pers…

The checkpoint inhibitor PD-1H/VISTA controls osteoclast-mediated multiple myeloma bone disease Open

Jing Fu, Shirong Li, Huihui Ma, Jun Yang, Gabriel M. Pagnotti , et al. · 2023

Psychiatric comorbidities in adults with sickle cell disease: A narrative review Open

Adrienne D. Mishkin, Elizabeth Prince, Elizabeth J. Leimbach, Markus Y. Mapara, C. Patrick Carroll · 2023

Summary Although descriptions of quality of life and patient reports of mood in sickle cell disease (SCD) have become more common in the literature, less is known about psychiatric illness prevalence, presentation, and treatment, particula…

Supplementary Figure 1 from Targeting the Microtubular Network as a New Antimyeloma Strategy Open

Rentian Feng, Shirong Li, Caisheng Lu, Carrie Andreas, Donna B. Stolz , et al. · 2023

PDF file - 52K, Confirmation of drug resistant phenotype of RPMI-multiple myeloma cell lines.

Data from Elevated Translation Initiation Factor eIF4E Is an Attractive Therapeutic Target in Multiple Myeloma Open

Shirong Li, Jing Fu, Caisheng Lu, Markus Y. Mapara, Shahzad Raza , et al. · 2023

eIF4E is the key regulator of protein translation and critical for translation. The oncogenic potential of tumorigenesis, which is highly contingent on cap-dependent eIF4E, also arises from the critical role in the nuclear export and cytos…

Supplementary Figures 1 and 2 from Elevated Translation Initiation Factor eIF4E Is an Attractive Therapeutic Target in Multiple Myeloma Open

Shirong Li, Jing Fu, Caisheng Lu, Markus Y. Mapara, Shahzad Raza , et al. · 2023

Supplementary Figures 1. Analysis of eIF4E protein levels by western blotting of whole cell extracts from primary stromal cells, CD138- cells, primary myeloma cells (CD138+) and normal plasma cells. Supplementary Figures 2. CT shRNA, eIF4E…

Supplementary Figure Legends 1-3 from Targeting the Microtubular Network as a New Antimyeloma Strategy Open

Rentian Feng, Shirong Li, Caisheng Lu, Carrie Andreas, Donna B. Stolz , et al. · 2023

PDF file - 87K

Data from Targeting the Microtubular Network as a New Antimyeloma Strategy Open

Rentian Feng, Shirong Li, Caisheng Lu, Carrie Andreas, Donna B. Stolz , et al. · 2023

We identified nocodazole as a potent antimyeloma drug from a drug screening library provided by the Multiple Myeloma Research Foundation. Nocodazole is a benzimidazole that was originally categorized as a broad-spectrum anthelmintic drug w…

Supplementary Figure Legends 1-3 from Targeting the Microtubular Network as a New Antimyeloma Strategy Open

Rentian Feng, Shirong Li, Caisheng Lu, Carrie Andreas, Donna B. Stolz , et al. · 2023

PDF file - 87K

Supplementary Figure 2 from Targeting the Microtubular Network as a New Antimyeloma Strategy Open

Rentian Feng, Shirong Li, Caisheng Lu, Carrie Andreas, Donna B. Stolz , et al. · 2023

PDF file - 199K, Nocodazole induces microtubular network disarray and morphologic elongation in multiple myeloma cells.

Supplementary Figure 1 from Targeting the Microtubular Network as a New Antimyeloma Strategy Open

Rentian Feng, Shirong Li, Caisheng Lu, Carrie Andreas, Donna B. Stolz , et al. · 2023

PDF file - 52K, Confirmation of drug resistant phenotype of RPMI-multiple myeloma cell lines.

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