G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for High-risk MDS Undergoing Allo-HSCT

Nanfang Hospital, Southern Medical University

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Allogeneic Hematopoietic Stem Cell Transplantation; Conditioning; Myelodysplastic Syndrome

Treatments: Drug: Decitabine (DAC); Drug: Granulocyte Colony-Stimulating Factor(G-CSF); Drug: Busulfan; Drug: Busulfan (BU); Drug: Cyclophosphamide (CY); Drug: Fludarabine (FLU)

Study type: Interventional

Funder types: Other

Identifiers

NCT05453552

NFEC-2022-233

Details and patient eligibility

About

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.

Full 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.

Enrollment: 242 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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242 participants in 2 patient groups

G-CSF+DAC+BF

Experimental group

Description:

For patients with high-risk MDS undergoing allo- HSCT, Granulocyte Colony-Stimulating Factor (G-CSF)+Decitabine+BF conditioning regimen was G-CSF 5ug/kg/day on days -17 to -10 (when white blood cell is more than 20G/L, stop using G-CSF), Decitabine 20mg/m2/day on days -14 to -10, Busulfan (BU) 3.2 mg/kg/day on days -6 to -3, Fludarabine (FLU) 30mg/m2/ day on days -7 to -3.

Treatment:

Drug: Fludarabine (FLU)

Drug: Busulfan (BU)

Drug: Decitabine (DAC)

Drug: Granulocyte Colony-Stimulating Factor(G-CSF)

G-CSF+DAC+BUCY

Active Comparator group

Description:

For patients with high-risk MDS undergoing allo- HSCT, Granulocyte Colony -Stimulating Factor (G-CSF)+Decitabine+BUCY conditioning regimen was G-CSF 5ug/kg/day on days -17 to -10 (when white blood cell is more than 20G/L, stop using G-CSF), Decitabine 20mg/m2/day on days -14 to -10, Busulfan (BU) 3.2 mg/kg/day on days -7 to -4, Cyclophosphamide (CY) 60 mg/kg/day on days -3, -2.

Treatment:

Drug: Cyclophosphamide (CY)

Drug: Busulfan

Drug: Decitabine (DAC)

Drug: Granulocyte Colony-Stimulating Factor(G-CSF)