Allogeneic HSCT in 17p- CLL in First or Second Partial or Complete Remission at Transplant: a Non-interventional Prospective Study.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 41
- 试验地点
- 9
- 主要终点
- Progression-free survival (PFS) rate
研究概览
简要总结
17p-/p53-mutated chronic lymphocytic leukemia (CLL) is an orphan disease, accounting for approximately 5% of newly diagnosed CLL. This subgroup of patients has a very poor outcome after chemoimmunotherapy. Allogeneic HCT may change the poor prognosis. In a retrospective EBMT-analysis on 44 patients with advanced 17p-CLL 2-year progression-free survival was 45% (95% CI, 30% to 60%) after allogeneic HCT (Allogeneic hematopoietic stem-cell transplantation for chronic lymphocytic leukemia with 17p deletion: a retrospective European Group for Blood and Marrow Transplantation analysis. J Clin Oncol, 2008, 26, 5094-5100).
Referring to these favorable results and small additional series, patients with 17p-CLL requiring therapy are considered to have an indication for allogeneic transplantation by many CLL study groups. Several CLL study groups recommend allogeneic HCT in 17p-CLL as part of the first- or second line treatment.
The aim is to collect additional evidence on allogeneic HCT in 17p-/p53-mutated CLL in first or second remission by a non-interventional prospective study. Patients shall be registered prior to HCT at the Leiden Office in order to rule out a reporting bias after transplantation.
详细描述
Objective:
The aim is to determine early PFS after allogeneic HCT in first or second remission of 17p-/p53-mutated CLL within an epidemiologic study.
Methods:
Neither the decision for allogeneic transplantation nor specific treatment recommendations for patients with 17p-/p53-mutated CLL are part of the study. Instead, the study protocol refers to EBMT guidelines. Indications for allogeneic stem cell transplantation in chronic lymphocytic leukemia: the EBMT transplant consensus. Leukemia 21, 2007, 12-17). Minimal essential data (MED) A and B, defined by the EBMT, will be collected (www.ebmt.org).
The rate of progression-free survival (PFS) at 1 year after HSCT was selected as primary endpoint. Death, clinical relapse or progression but not immune manipulations (taper of immunosuppression, DLI, rituximab) are considered as treatment failure for PFS. Patients without information on one-year follow up will be considered as having experienced treatment failure. The rate of PFS at 1 year will be calculated by dividing the number of patients without treatment failure by the number of patients who met all selection criteria.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 69 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •17p-/p53-mutated CLL by FISH or sequencing, confirmed by review by an experienced laboratory
- •first or second partial remission or complete remission at HCT according to the updated NCI-criteria (Hallek 2008)
- •MRD diagnostic as part of the local standard follow up
- •allogeneic HCT from a matched related or unrelated donor with up to one mismatch refering to HLA-A, -B, -C and DRB1
排除标准
- •ex vivo T-cell depletion
- •in vivo T-cell depletion with alemtuzumab
结局指标
主要结局
Progression-free survival (PFS) rate
时间窗: 1 year after HSCT
次要结局
- Overall survival, cumulative incidence of relapse and non-relapse mortality(1 year after HSCT)
- Rate of MRD-negative complete remissions(1 year after HSCT)