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)
概览
- 阶段
- 不适用
- 干预措施
- Hematopoietic stem cell transplant
- 疾病 / 适应症
- Myelofibrosis
- 发起方
- University Health Network, Toronto
- 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)
- 状态
- 进行中（未招募）
- 最后更新
- 3个月前
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.
研究者
入排标准
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
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
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
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
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
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
- Disease-free survival of patients who receive hematopoietic stem cell transplantation (HCT)(5 years)
- 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 FACT-BMT Questionnaire(0 and 36 months)
- Disease-free survival of patients who receive best available non-transplant therapies (BAT)(5 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)