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异基因造血细胞移植后使用奥米杜昔单抗与脐带血治疗的健康相关生活质量。

Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood.

机构信息

Division of Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, North Carolina.

Analysis Group, Inc, Boston, Massachusetts.

出版信息

Transplant Cell Ther. 2023 Jan;29(1):52.e1-52.e9. doi: 10.1016/j.jtct.2022.09.018. Epub 2022 Sep 28.

Abstract

Omidubicel is an advanced cell therapy derived from umbilical cord blood (UCB) for use in allogeneic hematopoietic cell transplantation (HCT). A recent randomized phase 3 clinical trial demonstrated faster engraftment, shorter length of hospital stays, and lower rates of infection with omidubicel compared with standard UCB transplantation in patients with high-risk hematologic malignancies. Despite the proven clinical benefits of omidubicel, its impact on health-related quality of life (HRQL) from the patient's perspective has not been described. This study analyzed patient-reported HRQL measures collected prospectively in the randomized phase 3 trial comparing omidubicel to standard UCB transplantation. A total of 108 patients at 33 international stem cell transplantation centers underwent myeloablative allogeneic HCT with either omidubicel or standard UCB. Patients completed serial HRQL questionnaires at screening and on days 42, 100, 180, and 365 post-transplantation. The HRQL surveys included the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT), a 50-item cancer-specific questionnaire assessing physical, functional, emotional, social/family, and HCT-specific well-being, and the EuroQol 5-Dimension 3-Level, a 5-item generic HRQL survey. A mixed model with repeated measures was used to compare changes in HRQL from baseline in the 2 treatment arms. The average change in HRQL scores over time was compared by estimating the difference in the area under the curve (AUC) in each treatment group. Seventy-five patients (omidubicel arm, n = 37; standard UCB arm, n = 38) who completed the FACT-BMT at baseline and on 1 or more follow-up visits were included in this study. Baseline characteristics were similar in the 2 treatment arms. Over the first year post-transplantation, the AUCs of mean changes in physical, functional, and total FACT-BMT scores indicated significantly better HRQL with omidubicel (P < .05), with mean differences across time points ranging from 1.4 to 3.1 points, 1.6 to 3.2 points, and 7.2 to 11.0 points, respectively. The minimal clinically important difference was exceeded at 1 or more time points for each of these measures. The HRQL improvements with omidubicel were observed as early as 42 days post-transplantation and persisted at 1 year, indicating the potential long-term benefits of omidubicel on HRQL. Across all patients, adverse clinical outcomes, such as grade 3 viral infections and lower rates of neutrophil engraftment, were associated with worse HRQL scores. The observed improvements in HRQL measures may reflect the known clinical benefits of omidubicel. Compared with standard UCB, allogeneic HCT with omidubicel resulted in significant and clinically meaningful improvements in patient-reported HRQL measures.

摘要

Omidubicel 是一种源自脐带血(UCB)的先进细胞疗法,可用于异基因造血细胞移植(HCT)。最近的一项随机 3 期临床试验表明,与标准 UCB 移植相比,omidubicel 可更快地植入、缩短住院时间、降低感染率,适用于患有高危血液恶性肿瘤的患者。尽管 omidubicel 具有已证实的临床益处,但尚未从患者角度描述其对健康相关生活质量(HRQL)的影响。本研究分析了在比较 omidubicel 与标准 UCB 移植的随机 3 期试验中前瞻性收集的患者报告的 HRQL 测量结果。在 33 个国际干细胞移植中心,共有 108 例患者接受了清髓性异基因 HCT,分别使用 omidubicel 或标准 UCB。患者在筛选和移植后第 42、100、180 和 365 天完成了系列 HRQL 问卷。HRQL 调查包括功能性评估癌症治疗-骨髓移植(FACT-BMT),这是一种 50 项癌症特异性问卷,评估身体、功能、情感、社会/家庭和 HCT 特定的幸福感,以及欧洲五维健康量表 3 级(EQ-5D-3L),这是一种 5 项通用 HRQL 调查。采用具有重复测量的混合模型来比较 2 种治疗组基线 HRQL 的变化。通过估计每个治疗组中曲线下面积(AUC)的差异来比较随时间变化的 HRQL 评分的平均变化。本研究纳入了在基线和 1 次或多次随访时完成 FACT-BMT 的 75 例患者(omidubicel 组,n=37;标准 UCB 组,n=38)。2 种治疗组的基线特征相似。在移植后第一年,物理、功能和总 FACT-BMT 评分的平均变化 AUC 表明 omidubicel 具有更好的 HRQL(P<0.05),各时间点的平均差异范围为 1.4 至 3.1 分、1.6 至 3.2 分和 7.2 至 11.0 分。这些措施中,每个措施在 1 个或多个时间点都超过了最小临床重要差异。在移植后 42 天即可观察到 omidubicel 的 HRQL 改善,并持续至 1 年,表明 omidubicel 对 HRQL 具有潜在的长期益处。在所有患者中,严重病毒感染等不良临床结局和较低的中性粒细胞植入率与较差的 HRQL 评分相关。观察到的 HRQL 测量改善可能反映了 omidubicel 的已知临床益处。与标准 UCB 相比,异基因 HCT 使用 omidubicel 可显著改善患者报告的 HRQL 测量。

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