Impact of extraosseous extramedullary disease on outcomes of patients with relapsed-refractory multiple myeloma receiving standard-of-care chimeric antigen receptor T-cell therapy Journal Article


Authors: Dima, D.; Abdallah, A. O.; Davis, J. A.; Awada, H.; Goel, U.; Rashid, A.; DeJarnette, S.; Anwer, F.; Shune, L.; Raza, S.; Mahmoudjafari, Z.; Williams, L.; Faiman, B.; McGuirk, J. P.; Sauter, C. S.; Ahmed, N.; Khouri, J.; Hashmi, H.
Article Title: Impact of extraosseous extramedullary disease on outcomes of patients with relapsed-refractory multiple myeloma receiving standard-of-care chimeric antigen receptor T-cell therapy
Abstract: The presence of extramedullary disease (EMD) has been associated with poor outcomes in patients with relapsed-refractory multiple myeloma (RRMM). Herein, we report the outcomes of RRMM patients who were treated with standard-of-care (SOC) chimeric antigen receptor (CAR) T-cell therapy and had active extraosseous EMD before the infusion. Data were retrospectively collected from patients at three US institutions with the intent to receive SOC CAR T. Responses were assessed per the International Myeloma Working Group criteria. A total of 152 patients proceeded with infusion, of whom 47 (31%) had EMD (EMD group) and 105 (69%) did not (non-EMD group). Baseline patient characteristics were comparable between the two groups. The EMD group had a higher incidence of high-grade CRS, steroid and anakinra use, and thrombocytopenia on day +30 compared to the non-EMD group. In addition, the EMD group had an inferior overall response rate (58% vs 96%, p < 0.00001), median progression-free survival (PFS) (5.1 vs 12.4 months; p < 0.0001), and overall survival (OS) (12.2 vs 27.5 months; p = 0.00058) compared to the non-EMD group. We further subdivided the non-EMD patients into those with paramedullary disease (PMD-only group, n = 26 [17%]) and those with neither EMD nor PMD (bone marrow-contained group or BM-only group, n = 79 [52%]). Patients with PMD-only had similar median PFS (11.2 vs 13.6 months, p = 0.3798) and OS (not reached [NR] vs 27.5 months, p = 0.6446) compared to patients with BM-only disease. However, patients with EMD exhibited inferior median PFS (5.1 vs 13.6 months, p < 0.0001) and OS (12.2 vs 27.5, p = 0.0008) compared to patients in the BM-only group. Treatment with SOC CAR T yielded meaningful clinical outcomes in real-world RRMM patients with extraosseous EMD, though responses and survival outcomes were suboptimal compared to patients without EMD. The presence of only EMD but not PMD was associated with significantly worse survival outcomes following the CAR T infusion. © The Author(s) 2024.
Keywords: adult; cancer chemotherapy; controlled study; treatment outcome; treatment response; aged; middle aged; retrospective studies; major clinical study; overall survival; fludarabine; thrombopoietin receptor; mortality; neutropenia; cancer staging; neurotoxicity; follow up; progression free survival; multiple myeloma; neoplasm recurrence, local; anemia; thrombocytopenia; incidence; relapse; bendamustine; cyclophosphamide; retrospective study; health care quality; tumor recurrence; chimeric antigen receptor; corticosteroid; recombinant granulocyte colony stimulating factor; therapy; adoptive immunotherapy; immunotherapy, adoptive; hematologic disease; standard of care; leukapheresis; clinical outcome; cytokine release syndrome; procedures; overall response rate; tocilizumab; Common Terminology Criteria for Adverse Events; refractory disease; humans; human; male; female; article; extramedullary disease; ecog performance status; chimeric antigen receptor t-cell immunotherapy; anakinra; immune effector cell associated neurotoxicity syndrome; receptors, chimeric antigen; ciltacabtagene autoleucel; idecabtagene vicleucel; cytokine receptor agonist
Journal Title: Blood Cancer Journal
Volume: 14
ISSN: 2044-5385
Publisher: Nature Publishing Group  
Date Published: 2024-05-31
Start Page: 90
Language: English
DOI: 10.1038/s41408-024-01068-w
PUBMED: 38821914
PROVIDER: scopus
PMCID: PMC11143360
DOI/URL:
Notes: Article -- Source: Scopus
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  1. Hamza Hashmi
    62 Hashmi