This study aims to evaluate whether immediate allogeneic hematopoietic stem cell transplantation (HSCT) is non-inferior to HSCT following bridging therapy in patients with higher-risk myelodysplastic syndrome (HR-MDS).
A total of 236 patients will be randomized in a 1:1 ratio into the immediate transplantation group (n=118) and the disease control group (n=118). The study will continue until at least 124 events occur.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
236
Patients undergo direct allogeneic HSCT.
Patients receive one to two cycles of bridging therapy before undergoing allogeneic HSCT. o Bridging Therapy Regimen: Hypomethylating agents (HMA) alone or HMA-based combination chemotherapy, e.g., azacitidine (AZA) 100 mg/day + venetoclax (VEN) 400 mg/day for 7 days. Targeted therapies (e.g., IDH1 inhibitors) will be used for eligible patients.
Peking University People's Hospital
Beijin, Beijing Municipality, China
RECRUITINGZhengzhou University First Affiliated Hospital
Zhengzhou, Henan, China
RECRUITINGThe 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation Army
Jinan, Shandong, China
2-year Disease-Free Survival (DFS) post-HSCT
Defined as the time from transplantation to two years post-HSCT, with primary events including death or failure to achieve CR or CR equivalent at the time of assessment
Time frame: 2-year
Cumulative Incidence of Allogeneic HSCT
The proportion of patients who undergo HSCT at 4, 8, 16, and 24 weeks post-randomization.
Time frame: The proportion of patients who undergo HSCT at 4, 8, 16, and 24 weeks post-randomization.
Complete Remission (CR) or CR Equivalent Rate from Randomization
The percentage of patients achieving CR or CR equivalent, defined as the first documented occurrence.
Time frame: 2-year
2-year Overall Survival (OS) post-HSCT
Defined as the time from HSCT to death from any cause within two years.
Time frame: 2-year
2-year Leukemia-Free Survival (LFS) from Randomization
The time from randomization to the occurrence of disease progression, relapse, or death from any cause within two years.
Time frame: 2-year
2-year Quality of Life (QoL) Assessment: 2-year Quality of Life (QoL) Assessment from Randomization
Defined as the assessment of patient-reported QoL starting from randomization over a 2-year period, evaluated using the EORTC QLQ-C30 questionnaire.
Time frame: 2-year
Molecular Clearance Rate
Molecular clearance was defined by two consecutive blood samples obtained at least 4 weeks apart after that were negative for driver mutations in a patient who had been positive before transplantation.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
People's Liberation Army The General Hospital of Western Theater Command
Chengdu, Sichuan, China
RECRUITINGThe Second Hospital of Hebei Medical University
Shijia Zhuang, China
RECRUITINGTime frame: 2-year