骨髓增生异常综合征患者年龄组在移植中的比较：医疗保险覆盖范围与证据发展研究。
Comparison of Patient Age Groups in Transplantation for Myelodysplastic Syndrome: The Medicare Coverage With Evidence Development Study.
机构信息
Division of Hematology & Oncology, Medical College of Wisconsin, Milwaukee.
Division of Biostatistics, Department of Institute for Health and Equity, Medical College of Wisconsin, Milwaukee.
出版信息
JAMA Oncol. 2020 Apr 1;6(4):486-493. doi: 10.1001/jamaoncol.2019.5140.
IMPORTANCE
In 2010, the US Centers for Medicare & Medicaid Services (CMS) indicated that data regarding efficacy of allogeneic hematopoietic stem cell transplantation (HCT) in the CMS beneficiary population with myelodysplastic syndrome (MDS) were currently insufficient, but that coverage would be provided for patients enrolled in a clinical study that met its criteria for Coverage with Evidence Development (CED).
OBJECTIVE
The Center for International Bone Marrow Transplant Research (CIBMTR) submitted a study concept comparing the outcomes of patients aged 55 to 64 years vs aged 65 years or older who met those criteria, effectively providing coverage by CMS for HCT for MDS.
DESIGN, SETTING, AND PARTICIPANTS: Data on patients aged 65 years or older were prospectively collected and their outcomes compared with patients aged 55 to 64 years. Patients were enrolled in the study from December 15, 2010, to May 14, 2014. The results reported herein were analyzed as of September 4, 2017, with a median follow-up of 47 months. The study was conducted by the CIBMTR. It comprises a voluntary working group of more than 420 centers worldwide that contribute detailed data on allogeneic and autologous HCT and cellular therapies.
INTERVENTIONS
Patients with MDS received HCT according to institutional guidelines and preferences.
MAIN OUTCOMES AND MEASURES
The primary outcome was overall survival (OS); secondary outcomes included nonrelapse mortality (NRM), relapse-free survival, and acute and chronic graft vs host disease.
RESULTS
During the study period, 688 patients aged 65 years or older underwent HCT for MDS and were compared with 592 patients aged 55 to 64 years. Other than age, there were no differences in patient and disease characteristics between the groups. On univariate analysis, the 3-year NRM rate was 28% vs 25% for the 65 years or older group vs those aged 55 to 64 years, respectively. The 3-year OS was 37% vs 42% for the 65 years or older group vs the 55 to 64 years age group, respectively. On multivariable analysis after adjusting for excess risk of mortality in the older group, age group had no significant association with OS (HR, 1.09; 95% CI, 0.94-1.27; P = .23) or NRM (HR, 1.19; 95% CI, 0.93-1.52; P = .16).
CONCLUSIONS AND RELEVANCE
Older patients with MDS undergoing HCT have similar OS compared with younger patients. Based on current data, we would recommend coverage of HCT for MDS by the CMS.
TRIAL REGISTRATION
ClinicalTrials.gov identifier: NCT01166009.
重要性
2010 年，美国医疗保险和医疗补助服务中心（CMS）表示，目前关于异体造血干细胞移植（HCT）在 CMS 受益人群中治疗骨髓增生异常综合征（MDS）的疗效的数据不足，但将为符合其有证据开发覆盖范围（CED）标准的临床试验中入组的患者提供覆盖。
目的
国际骨髓移植研究中心（CIBMTR）提交了一项研究方案，比较了年龄在 55 至 64 岁与 65 岁或以上符合这些标准的患者的结局，实际上为 CMS 对 MDS 的 HCT 提供了覆盖。
设计、地点和参与者：前瞻性收集了 65 岁及以上患者的数据，并将其结局与 55 至 64 岁患者进行比较。患者于 2010 年 12 月 15 日至 2014 年 5 月 14 日入组研究。本文报告的结果分析截至 2017 年 9 月 4 日，中位随访时间为 47 个月。该研究由 CIBMTR 进行。它由一个由 420 多个全球中心组成的自愿工作组组成，这些中心提供关于异体和自体 HCT 和细胞治疗的详细数据。
干预措施
MDS 患者根据机构指南和偏好接受 HCT。
主要结局和测量指标
主要结局是总生存（OS）；次要结局包括非复发死亡率（NRM）、无复发生存率以及急性和慢性移植物抗宿主病。
结果
在研究期间，688 名 65 岁及以上的 MDS 患者接受了 HCT，并与 592 名 55 至 64 岁的患者进行了比较。除了年龄之外，两组患者的特征和疾病特征无差异。单因素分析显示，65 岁及以上组的 3 年 NRM 率为 28%，55 至 64 岁组为 25%。65 岁及以上组的 3 年 OS 率为 37%，55 至 64 岁组为 42%。多变量分析后，调整老年组死亡风险增加的因素，年龄组与 OS（HR，1.09；95%CI，0.94-1.27；P=0.23）或 NRM（HR，1.19；95%CI，0.93-1.52；P=0.16）均无显著相关性。
结论和相关性
接受 HCT 的 MDS 老年患者与年轻患者的 OS 相似。根据目前的数据，我们建议 CMS 对 MDS 的 HCT 提供覆盖。
试验注册
ClinicalTrials.gov 标识符：NCT01166009。
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