Steinhardt Maximilian J, Schaefers Christoph, Leypoldt Lisa B, Blau Igor-Wolfgang, Harzer Marie, Zhou Xiang, Riedhammer Christine, Kamili Abdulaziz, Kosch Ricardo, Topp Laura S, Molwitz Isabel, Gross-Fengels Nils-Ole, Melzer Yasmin Fede, Artzenroth Jule, Al-Bazaz Maximilian, Alsdorf Winfried, Topp Max S, Duell Johannes, Mersi Julia, Waldschmidt Johannes, Bokemeyer Carsten, Einsele Hermann, Kortüm K Martin, Weisel Katja, Rasche Leo
Department of Internal Medicine II, University Hospital of Würzburg, Würzburg, Germany.
Department of Hematology, Oncology and Bone Marrow Transplantation with Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Blood Cancer J. 2025 Jul 30;15(1):126. doi: 10.1038/s41408-025-01330-9.
Extramedullary multiple myeloma (EMD) is associated with low response rates, short progression-free survival, and poor prognosis. CAR T cells and bispecific antibodies (bsABs) have shown efficacy in relapsed myeloma, but it remains uncertain whether one T cell redirection strategy should be preferred. We retrospectively analyzed 80 patients with EMD not adjacent to the bone treated with ide-cel, cilta-cel, teclistamab, or talquetamab at three academic centers in Germany. All patients were heavily pretreated, and a high-risk cytogenetic profile was prevalent in >41% of patients. All cohorts had a median of 5 to 7 prior lines of therapy. The vast majority of patients receiving cilta-cel, ide-cel, or teclistamab were BCMA-naive ( >88%). Response rates after CAR T cell infusion were significantly higher (100% with cilta-cel, 82% with ide-cel) than with bsABs (29% for talquetamab, 36% for teclistamab). Complete resolution of EMD was more frequent after CAR T cell therapies (50% and 41%) than after bsABs (16% and 14%). With a median follow-up of 12.2 months, median (m)PFS was not reached in patients that had received cilta-cel; mPFS was 7.3 months after ide-cel and significantly longer for both CAR T products compared to talquetamab or teclistamab (mPFS 4.0 and 2.6 months). Effective debulking therapy prolonged remissions after CAR T cell infusion compared to no debulking or no response to debulking. Visceral and soft tissue manifestations responded significantly less frequently than EMD in other locations. With significantly higher response rates, deeper remissions, and longer mPFS, our retrospective data suggest CAR T cells may provide a meaningful benefit in EMD.
髓外多发性骨髓瘤(EMD)与低缓解率、短无进展生存期和不良预后相关。嵌合抗原受体(CAR)T细胞和双特异性抗体(bsABs)已在复发骨髓瘤中显示出疗效,但尚不确定哪种T细胞重定向策略更具优势。我们回顾性分析了德国三个学术中心80例接受ide-cel、cilta-cel、teclistamab或talquetamab治疗的非骨邻近性EMD患者。所有患者均经过大量预处理,超过41%的患者具有高危细胞遗传学特征。所有队列的患者既往治疗中位数为5至7线。接受cilta-cel、ide-cel或teclistamab治疗的绝大多数患者既往未接触过B细胞成熟抗原(BCMA)(>88%)。CAR T细胞输注后的缓解率显著高于bsABs(cilta-cel为100%,ide-cel为82%)(talquetamab为29%,teclistamab为36%)。CAR T细胞治疗后EMD完全缓解的情况比bsABs更常见(分别为50%和41%对比16%和14%)。中位随访12.2个月,接受cilta-cel治疗的患者未达到中位无进展生存期(mPFS);ide-cel治疗后的mPFS为7.3个月,两种CAR T产品的mPFS均显著长于talquetamab或teclistamab(mPFS分别为4.0个月和2.6个月)。与未进行减瘤治疗或减瘤治疗无效相比,有效的减瘤治疗可延长CAR T细胞输注后的缓解期。与其他部位的EMD相比,内脏和软组织表现的缓解频率显著更低。我们的回顾性数据显示,CAR T细胞的缓解率显著更高、缓解程度更深且mPFS更长,提示其可能为EMD带来显著获益。