Clinical trial NCT05453552: G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for High-risk MDS Undergoing Allo-HSCT
Official title: G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With High-risk MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Myelodysplastic Syndrome
Allogeneic Hematopoietic Stem Cell Transplantation
Conditioning
Lead sponsor: Nanfang Hospital, Southern Medical University
Sponsor class: OTHER
Collaborators: name: Peking University People's Hospital
name: Institute of Hematology & Blood Diseases Hospital, China
name: Guangzhou First People's Hospital
name: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
name: First People's Hospital of Chenzhou
name: The Seventh Affiliated Hospital of Sun Yat-sen University
First posted: 2022-07-12
Enrollment: 242
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Allo-HSCT is the most effective way to cure high-risk MDS patients. At present, the best conditioning regimen for high-risk MDS patients undergoing allo-HSCT remains in discussion. In this prospective study, the safety and efficacy of G-CSF+DAC+BUCY and G-CSF+DAC+BF conditioning regimens in high-risk MDS patients undergoing allo-HSCT are evaluated.

Detailed description: Allo-HSCT is the most effective way to cure high-risk MDS patients. At present, the best conditioning regimen for high-risk MDS patients undergoing allo-HSCT remains in discussion. A previous study by the investigators has showed that G-CSF +DAC+BUCY conditioning regimen could reduce the relapse and improve the survival compared with BUCY conditioning regimen, while the two conditioning regimens both have high non-relapse mortality (NRM). Several retrospective and prospective studies have demonstrated that BF conditioning regimen has a lower NRM compared with BUCY conditioning regimen, while the relapse and survival are similar in patients undergoing BF and BUCY conditioning regimens. Based on the above, the investigators design the prospective randomized controlled study to evaluate the safety and efficacy of G-CSF+DAC+BUCY and G-CSF+DAC+BF conditioning regimens in high-risk MDS patients undergoing allo-HSCT.

Eligibility criteria: Inclusion Criteria:

* Had a diagnosis of RAEB-1 or RAEB-2 with IPSS-R \>3
* Age 18 to 65 years old
* ECOG performance status of 0-2
* HCT-CI of 0-2
* Were willing to undergo allo-HSCT

Exclusion Criteria:

* Therapy-related MDS
* Previous allo-HSCT
* Uncontrolled infections
* Liver or renal dysfunction
* Severe concomitant conditions not suitable for the trial

Primary outcomes:
measure: Non-relapse mortality (NRM)
timeFrame: 1 year
Secondary outcomes:
measure: Overall survival (OS)
timeFrame: 1 year
measure: Disease-free survival (DFS)
timeFrame: 1 year
measure: Cumulative incidence of relapse
timeFrame: 1 year
measure: Adverse effects
timeFrame: within 100 days post-transplantation
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Department of Hematology,Nanfang Hospital, Southern Medical University
state: Guangdong
Study officials:
affiliation: Nanfang Hospital, Southern Medical University
name: Qifa Liu
role: PRINCIPAL_INVESTIGATOR
References: