Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland.
Genesis Research, Newcastle, United Kingdom.
Clin Ther. 2021 Nov;43(11):1983-1996.e3. doi: 10.1016/j.clinthera.2021.09.013. Epub 2021 Nov 2.
Patients with relapsed and refractory multiple myeloma (RRMM) have a poor prognosis and limited treatment options after exposure to an immunomodulatory drug, proteasome inhibitor (PI), and anti-CD38 antibody (triple-class exposure [TCE]). However, current understanding about the management of these patients and associated health care resource use (HCRU) is limited outside the United States. The objective of the International Treatment pattern and resource use Evaluation for Multiple myeloma In a Study of triple-class Exposed patients (ITEMISE) study was to use a physician-developed survey fielded to hematologists across Europe and Canada to assess the treatment, management, HCRU, and end-of-life care for patients with RRMM after TCE.
The ITEMISE study used a 3-phase Delphi-like approach that consisted of in-depth interviews with 7 hematology experts; the development of a cross-sectional survey fielded to hematologists across Belgium, Canada, France, Germany, Italy, the Netherlands, Spain, Sweden, Switzerland, and the United Kingdom from August to October 2020; and a final workshop of hematology experts to validate the pooled findings. Hematologists were asked to consider the management of patients in the first 3 treatment lines after TCE, including treatment options, treatment duration and outcomes, and frequency of outpatient visits and hospitalizations.
The survey was completed by 202 hematologists (60% from academic hospitals, 38% from other public hospitals, and 2% from private hospitals). Hematologists estimated that 55% of patients would receive active treatment after TCE, the equivalent of fourth-line treatment onward since diagnosis of multiple myeloma. Immunomodulatory drug, anti-CD38 antibody plus immunomodulatory drug, and PI-based regimens (received by 22.5%, 17.8%, and 15.1% of patients, respectively) were reported for first treatment strategy after TCE. Pomalidomide, daratumumab, lenalidomide, bortezomib, and carfilzomib were the most frequently selected antimyeloma agents. Associated outcomes of median overall survival, progression-free survival, and objective response rate for first treatment after TCE were estimated as 12 months, 4 months, and 40%, respectively. HCRU included outpatient visits and unplanned hospitalizations that were commonly reported during treatment after TCE.
Findings indicate an intent to actively treat patients after TCE with a range of combination regimens frequently consisting of immunomodulatory drugs, PIs, and anti-CD38 antibodies, highlighting the lack of standard of care and suggesting a large clinical unmet need. Estimated clinical outcomes are consistent with data from US studies and indicate the poor prognosis for patients after TCE. Substantial HCRU is associated with management of patients after TCE across Europe and Canada, signifying a high patient and societal impact and a need for better treatment options to reduce this burden.
接受免疫调节剂、蛋白酶体抑制剂(PI)和抗 CD38 抗体(三药联合暴露 [TCE])治疗后,复发/难治性多发性骨髓瘤(RRMM)患者预后较差,治疗选择有限。然而,目前对这些患者的管理以及相关医疗资源使用(HCRU)的了解在美国以外的地区有限。国际 TCE 暴露后多发性骨髓瘤治疗模式和资源使用评估研究(ITEMISE)旨在使用医生开发的问卷调查,在欧洲和加拿大的血液学家中进行评估,以评估 TCE 后 RRMM 患者的治疗、管理、HCRU 和临终关怀。
ITEMISE 研究采用了 3 个阶段的德尔菲式方法,包括对 7 名血液学专家进行深入访谈;从 2020 年 8 月至 10 月,对来自比利时、加拿大、法国、德国、意大利、荷兰、西班牙、瑞典、瑞士和英国的血液学家进行横断面调查;以及血液学专家的最终研讨会,以验证汇总结果。血液学家被要求考虑 TCE 后前 3 线治疗中患者的管理,包括治疗选择、治疗持续时间和结果,以及门诊就诊和住院的频率。
共有 202 名血液学家完成了调查(60%来自学术医院,38%来自其他公立医院,2%来自私立医院)。血液学家估计,55%的患者在 TCE 后会接受积极治疗,这相当于多发性骨髓瘤确诊后的四线治疗。TCE 后一线治疗策略报告的免疫调节剂、抗 CD38 抗体联合免疫调节剂和 PI 为基础的方案分别占 22.5%、17.8%和 15.1%。替莫唑胺、达雷妥尤单抗、来那度胺、硼替佐米和卡非佐米是最常选择的抗骨髓瘤药物。TCE 后一线治疗的中位总生存期、无进展生存期和客观缓解率的相关结局估计分别为 12 个月、4 个月和 40%。HCRU 包括 TCE 后治疗期间经常发生的门诊就诊和非计划性住院。
研究结果表明,TCE 后患者意图积极治疗,采用多种联合方案,其中常包括免疫调节剂、PI 和抗 CD38 抗体,这突显了缺乏标准治疗方法的现状,并表明存在大量的临床需求未得到满足。估计的临床结果与美国研究的数据一致,表明 TCE 后患者预后较差。在欧洲和加拿大,TCE 后患者的 HCRU 较大,这表明患者和社会受到了重大影响,需要更好的治疗选择来减轻这种负担。