Stalker Margaret, Garfall Alfred, Cohen Adam, Vogl Dan T, Djulbegovic Mia, Susanibar-Adaniya Sandra, Stadtmauer Edward, Megherea Oxana, Waxman Adam J
Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Division of Hematology and Oncology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Eur J Haematol. 2025 Mar;114(3):443-447. doi: 10.1111/ejh.14348. Epub 2024 Nov 17.
Teclistamab has demonstrated deep responses in patients with multiple myeloma in the MajesTEC-1 study. However, the safety and efficacy of teclistamab in patients with AL amyloidosis are unknown.
We retrospectively analyzed patients with biopsy-proven relapsed/refractory AL amyloidosis who were treated with teclistamab from December 2022 to February 2024 at the University of Pennsylvania. The data cutoff was 2/29/24. Adverse events (AE) were extracted from the electronic medical record. Patients were assessed for hematologic and organ response per consensus guidelines.
Eight patients were included in this case series: median age 63 (range 59-67), 75% female, 88% White. All eight patients achieved at least very good partial response (VGPR) and had normalization of free light chains (FLC), and six (75%) patients achieved undetectable FLC levels. Of the six patients with immunofixation completed, all six (100%) achieved hematologic complete response (hCR). The median time to hematologic VGPR and hCR was 13 days (range 12-18 days) and 88 days (range 32-150 days), respectively. The median duration of follow-up was 8.5 months (range 1-14 months). Of the five patients with cardiac involvement, four (80%) achieved a cardiac response. Of the seven patients with renal involvement, two patients already achieved renal response prior to teclistamab, and of the remaining five, three (60%) achieved renal response. Six patients (75%) developed low-grade cytokine release syndrome (CRS). No patients developed ICANS. Neutropenia and AKI both occurred in 25% of patients, respectively.
In this series of patients, teclistamab showed outstanding depth of response and was well-tolerated. Teclistamab shows promise in treating patients with relapsed AL amyloidosis.
在MajesTEC-1研究中，替雷利珠单抗已在多发性骨髓瘤患者中显示出深度缓解。然而，替雷利珠单抗在AL淀粉样变性患者中的安全性和有效性尚不清楚。
我们回顾性分析了2022年12月至2024年2月在宾夕法尼亚大学接受替雷利珠单抗治疗的经活检证实为复发/难治性AL淀粉样变性患者。数据截止日期为2024年2月29日。不良事件（AE）从电子病历中提取。根据共识指南对患者的血液学和器官反应进行评估。
本病例系列纳入了8名患者：中位年龄63岁（范围59-67岁），75%为女性，88%为白人。所有8名患者均至少达到非常好的部分缓解（VGPR），游离轻链（FLC）正常化，6名（75%）患者的FLC水平检测不到。在完成免疫固定的6名患者中，所有6名（100%）均达到血液学完全缓解（hCR）。达到血液学VGPR和hCR的中位时间分别为13天（范围12-18天）和88天（范围32-150天）。中位随访时间为8.5个月（范围1-14个月）。在5名有心脏受累的患者中，4名（80%）实现了心脏反应。在7名有肾脏受累的患者中，2名患者在接受替雷利珠单抗治疗前已实现肾脏反应，其余5名患者中，3名（60%）实现了肾脏反应。6名患者（75%）发生了低级别细胞因子释放综合征（CRS）。没有患者发生免疫效应细胞相关神经毒性综合征（ICANS）。中性粒细胞减少症和急性肾损伤的发生率均为25%。
在这一系列患者中，替雷利珠单抗显示出出色的缓解深度且耐受性良好。替雷利珠单抗在治疗复发的AL淀粉样变性患者方面显示出前景。
