Myelodysplastic Syndromes
Conditions
Brief summary
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).
Detailed description
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.
Interventions
OTHERImmediate HSCT Group
Patients undergo direct allogeneic HSCT.
OTHERBridging Therapy Group
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.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥18 years 2. High relapse risk MDS, defined by: * IPSS-R score ≥3.5. * IPSS-M stratification as intermediate-high, high, or very high risk. 3. Eligible for allogeneic HSCT (including matched or mismatched related/unrelated donor transplantations). 4. Karnofsky Performance Status (KPS) ≥60. 5. Signed informed consent.
Exclusion criteria
1. Severe organ dysfunction: * Left ventricular ejection fraction \<50%. * Oxygen supplementation requirement. * Serum bilirubin \>1.5x upper limit of normal (unless due to Gilbert syndrome) or AST/ALT \>5x upper limit of normal. * Estimated glomerular filtration rate (eGFR) \<50 mL/min. 2. History of prior allogeneic HSCT. 3. Any condition deemed unsuitable by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 2-year Disease-Free Survival (DFS) post-HSCT | 2-year | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete Remission (CR) or CR Equivalent Rate from Randomization | 2-year | The percentage of patients achieving CR or CR equivalent, defined as the first documented occurrence. |
| 2-year Overall Survival (OS) post-HSCT | 2-year | Defined as the time from HSCT to death from any cause within two years. |
| Cumulative Incidence of Allogeneic HSCT | The proportion of patients who undergo HSCT at 4, 8, 16, and 24 weeks post-randomization. | The proportion of patients who undergo HSCT at 4, 8, 16, and 24 weeks post-randomization. |
| 2-year Quality of Life (QoL) Assessment: 2-year Quality of Life (QoL) Assessment from Randomization | 2-year | Defined as the assessment of patient-reported QoL starting from randomization over a 2-year period, evaluated using the EORTC QLQ-C30 questionnaire. |
| Molecular Clearance Rate | 2-year | 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. |
| 2-year Leukemia-Free Survival (LFS) from Randomization | 2-year | The time from randomization to the occurrence of disease progression, relapse, or death from any cause within two years. |
Countries
China
Contacts
Primary ContactJiang Erlie, doctor
Backup ContactXiao Zhijian, doctor
Outcome results
None listed