Minarik Jiri, Pour Ludek, Maisnar Vladimir, Spicka Ivan, Jungova Alexandra, Jelinek Tomas, Brozova Lucie, Krhovska Petra, Scudla Vlastimil, Hajek Roman
Department of Hemato-oncology, University Hospital Olomouc and Faculty of Medicine, Palacky University Olomouc, Czech Republic.
Department of Internal Medicine, Hematology and Oncology, University Hospital Brno, Czech Republic.
Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub. 2019 Sep;163(3):279-283. doi: 10.5507/bp.2018.064. Epub 2018 Nov 6.
The treatment of relapsed and refractory multiple myeloma (RRMM) remains challenging. The outcomes in highly pretreated populations are unsatisfactory and there is urgent need for novel and safe therapeutic approaches. Recently, daratumumab has been approved for RRMM with promising results even in monotherapy. The aim of this study was to assess the efficacy of single agent daratumumab outside a clinical trial.
14 patients with RRMM and significant pretreatment (median 4.5 previous lines) entered a specific healthcare program and received treatment with single agent daratumumab. They were followed for therapeutic response based on IMWG criteria, and incidence of adverse events. The data were collected using the Registry of Monoclonal Gammopathies.
The overall response rate was 38.5%. 23.1% of patients reached very good partial response, 15.4% reached partial remission, 15.4% had minimal response, 38.5% had stable disease and 7.7% had progressive disease. The median progression free survival was 4.6 months and median overall survival was not achieved. The toxicities were mostly mild, only infectious complications and hematological toxicity reached grade III.
We conclude that daratumumab has significant activity in highly pretreated RRMM even as a single agent, with an acceptable toxicity profile and survival impact.
复发难治性多发性骨髓瘤（RRMM）的治疗仍然具有挑战性。经过高度预处理的患者群体的治疗结果并不理想，迫切需要新的安全治疗方法。最近，达雷妥尤单抗已被批准用于RRMM，即使单药治疗也有令人鼓舞的结果。本研究的目的是评估达雷妥尤单抗单药在临床试验之外的疗效。
14例RRMM且有显著预处理史（既往中位数4.5线治疗）的患者进入特定医疗保健项目，接受达雷妥尤单抗单药治疗。根据国际骨髓瘤工作组（IMWG）标准对他们进行治疗反应随访以及不良事件发生率的监测。数据通过单克隆丙种球蛋白病登记处收集。
总缓解率为38.5%。23.1%的患者达到非常好的部分缓解，15.4%达到部分缓解，15.4%有微小缓解，38.5%疾病稳定，7.7%疾病进展。无进展生存期的中位数为4.6个月，总生存期的中位数未达到。毒性大多为轻度，只有感染性并发症和血液学毒性达到3级。
我们得出结论，达雷妥尤单抗即使作为单药在经过高度预处理的RRMM中也具有显著活性，具有可接受的毒性特征和生存影响。
