A Patient Preferences-Controlled Study of Allogeneic Hematopoietic Cell Transplantation Versus Best Available Non-Transplant Therapies in Patients With High-Risk Myelofibrosis (ALLO-BAT Study)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 90
- 试验地点
- 10
- 主要终点
- Number of patients allocated to best available non-transplant therapies (BAT)
研究概览
简要总结
The purpose of this research study is to see how effective hematopoietic stem cell transplantation (HCT) is compared to best available non-transplant therapies (BAT) in patients with high risk myelofibrosis. This will be done by asking participants to choose the treatment that they prefer to receive (HCT or BAT) and then comparing the outcomes of the participants in both treatment groups.
详细描述
There is currently little information regarding which treatments are best for patients with myelofibrosis. On one hand, hematopoietic stem cell transplantation (HCT) is potentially curative treatment but is associated with significant risk of complications related to graft failure (the new donor cells does not grow properly after the transplant), side effects such as graft versus host disease (the patient's cells attack the new donor cells), and risk of infections. Non-transplant therapies such as ruxolitinib provide effective symptom control for few months to few years, but are not curative in nature. As such, this study will compare the effectiveness of HCT versus best available non-transplant therapies (BAT) in patients with high risk myelofibrosis.
This is an observational study, meaning that participants will be followed to assess the effects of their treatment, but no intervention (treatments) will be given as a part of this study.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Recruitment Part:
- •Documented diagnosis of pre-fibrotic primary myelofibrosis (pre-fibrotic PMF), overt PMF, post-polycythemia MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF) confirmed by bone marrow biopsy
- •Have been tested or have results available for phenotypic driver mutations (JAK2/CALR/MPL) and high molecular risk (HMR) mutations using a broad myeloid malignancies targeted gene panel.
- •Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- •Able to provide informed consent
- •Adequate organ function
- •Donor search initiated or patient is agreeable to donor search
- •Meet the definition/criteria for high-risk myelofibrosis
- •Study Arm Allocation:
- •Grade of fibrosis on bone marrow biopsy available according to World Health Organization (WHO) criteria
- •Results available for phenotypic driver mutations (JAK2/CALR/MPL) and targeted sequencing results using a broad myeloid malignancy panel with a minimal requirement to include results on High molecular risk (HMR) mutations such as ASXL1/EZH2/IDH1/IDH2/SRSF2/U2AF1/TP53
- •ECOG performance status 0-2
- •Adequate organ function
- •Information on donor search and donor type available
排除标准
- •Recruitment Part:
- •Blasts in peripheral blood or bone marrow ≥10%
- •For patients already on ruxolitinib at study entry, and meet the criteria of ruxolitinib failure
- •Previous history of transformation to blast phase or acute myeloid leukemia
- •Received allogeneic stem cell transplant for myeloproliferative neoplasm
- •Presence of an active uncontrolled infection
- •Myocardial infarction in the preceding 3 months
- •Active hepatitis A, B or C
- •Known human immunodeficiency virus (HIV) positive
- •History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer
- •Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation.
- •Pregnant or breastfeeding women
- •Study Arm Allocation:
- •Blasts in peripheral blood or bone marrow ≥10%
- •Meet the criteria of ruxolitinib failure
- •Presence of an active uncontrolled infection
- •Myocardial infarction in the preceding 3 months
- •Active hepatitis A, B or C
- •Known HIV positive
- •History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer
- •Pregnant or breastfeeding women
- •Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation.
- •Time between registration and allocation of study arm >24 weeks
研究组 & 干预措施
Hematopoietic stem cell transplant (HCT)
Standard of care hematopoietic stem cell transplant with a matched donor.
干预措施: Hematopoietic stem cell transplant (Biological)
Best available non-transplant therapies (BAT)
Standard of care treatment with a janus kinase (JAK) inhibitor drug called ruxolitinib or treatment with an antimetabolite drug called hydroxyurea.
干预措施: Ruxolitinib (Drug)
Best available non-transplant therapies (BAT)
Standard of care treatment with a janus kinase (JAK) inhibitor drug called ruxolitinib or treatment with an antimetabolite drug called hydroxyurea.
干预措施: Hydroxyurea (Drug)
结局指标
主要结局
Number of patients allocated to best available non-transplant therapies (BAT)
时间窗: 5 years
Overall survival rate of patients who receive hematopoietic stem cell transplantation (HCT)
时间窗: 5 years
Time from study allocation to death or last follow up.
Overall survival rate of patients who receive best available non-transplant therapies (BAT)
时间窗: 5 years
Time from study allocation to death or last follow up.
Number of patients allocated to hematopoietic stem cell transplantation (HCT)
时间窗: 5 years
次要结局
- Median change in Patient Global Impression of Change (PGIC) score(0 and 36 months)
- Number of patients who receive hematopoietic stem cell transplantation (HCT) in remission (complete and partial)(3 years)
- Median change in MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)(0 and 36 months)
- Number of patients who receive best available non-transplant therapies (BAT) in remission (complete and partial)(3 years)
- Disease-free survival of patients who receive hematopoietic stem cell transplantation (HCT)(5 years)
- Median change in FACT-BMT Questionnaire(0 and 36 months)
- Disease-free survival of patients who receive best available non-transplant therapies (BAT)(5 years)