Bortezomib-bendamustine-melphalan vs High-dose Melphalan in Autologous Hematopoietic Stem Cell Transplantation for Relapsed Multiple Myeloma - a Single Center Retrospective Cohort Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Mean difference in time to next treatment (TNT) after ASCT1 and ASCT2 for each individual patient
研究概览
简要总结
This project will evaluate the efficacy and safety of the conditioning regimen bortezomib-bendamustine-melphalan (BBM) in combination with autologous hematopoietic stem cell transplantation (ASCT) in relapsed multiple myeloma given from 2011 to 2018 at Uppsala University Hospital. This approach will be retrospectively compared to high dose melphalan (HDM) in the same setting in the years prior to, and following the BBM-period. Data on efficacy and safety data will be collected through systematic analysis of electronic medical records and from the Swedish Cancer Registry.
详细描述
Study design This is a retrospective single center cohort study comparing the new conditioning regimen bortezomib-bendamustine-melphalan to standard high-dose melphalan. The data sources will be electronic medical records and prospectively collected data from the Swedish Cancer Registry. The comparison will be analyzed in two parts. First, each patient will be its own control, comparing time to next treatment (TNT) for the first ASCT (always HDM, referred to as ASCT1) and second ASCT (BBM or HDM, referred to as ASCT2), and compare the mean difference between the two cohorts. Secondly, the difference in efficacy and severe adverse events between BBM and HDM at ASCT2 will be compared.
Study population Fifty consecutive patients, who were referred to Uppsala University Hospital (UUH) for a second ASCT after relapse in multiple myeloma following HDM and ASCT between 1 Nov 2011 and 30 Oct 2018 and who received conditioning with bortezomib-bendamustine-melphalan will be included in this study. As a control group, 25 consecutive patients who were treated with HDM prior to 1 Nov 2011 and 25 consecutive patients following 30 Oct 2018. The patients will be identified through the local European Society for Blood and Marrow Transplantation (EBMT) registry at UUH.
UUH is the referral hospital for seven Swedish regions with a total population of 2 151 353 at Dec 31 2022, which constitutes roughly one fifth of the population of Sweden.
Data collection Study data will be collected through systematic analysis of medical records from UUH and all the hospitals referring patients to UUH and from the Swedish Cancer Registry. All severe adverse events (AEs) will be collected until day 100 after ASCT2 according to National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
Primary endpoints
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of first relapse after previous ASCT for multiple myeloma according to the International Myeloma Working Group.
- •Treated with a second ASCT (ASCT2) as part of second line treatment at UUH.
- •Conditioning at ASCT2 with bortezomib-bendamustine-melphalan or high-dose melphalan only.
排除标准
- •Double (tandem) ASCT in first or second line treatment
- •Allogenic haematopoietic stem cell transplantation as part of first or second line therapy
- •Failure to meet the minimal dataset, defined as: (date of ASCT1 and ASCT2, date of start of induction treatment for relapsed myeloma prior to ASCT2, medical records from hospitalization for ASCT2, at least one follow-up visit (unless early death before first follow-up visit), date of progression and first treatment of relapsed multiple myeloma after ASCT2.
研究组 & 干预措施
Bortezomib-bendamustine-melphalan
Myeloma patients receiving bortezomib-bendamustine-melphalan at autologous hematopoietic stem cell transplantation first relapse.
干预措施: Bortezomib-bendamustine-melphalan (Drug)
结局指标
主要结局
Mean difference in time to next treatment (TNT) after ASCT1 and ASCT2 for each individual patient
时间窗: 0.2-18 years
Average time to next myeloma treatment within each individual patient making each patient as its' own control
次要结局
- Median time to next treatment after ASCT2(0-18 years)
- Median progression free survival (PFS) after ASCT2(0-18 years)
- Median overall survival after ASCT2(0-18 years)
- Depth of best response after ASCT2(0-24 months)
- Treatment related mortality(0-12 months)
- Mean duration of neutropenia at ASCT2(7-50 days)
- Mean time until engraftment at ASCT2(5-50 days)
- Mean duration of hospitalization at ASCT2(7-50 days)
- Frequency of severe adverse events at ASCT2(100 days)