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

NCT05453552 — Phase 2 / Phase 3, Unknown, 242 participants, Nanfang Hospital, Southern Medical University

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G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With High-risk MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Registry	NCT05453552
Status	Unknown
Phase	Phase 2 / Phase 3
Study type	Interventional; randomized, parallel, masking: none, treatment
Enrollment	242 (Estimated)
Sponsor	Nanfang Hospital, Southern Medical University (Other); collaborators: Peking University People's Hospital, Institute of Hematology & Blood Diseases Hospital, China, Guangzhou First People's Hospital, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, First People's Hospital of Chenzhou, The Seventh Affiliated Hospital of Sun Yat-sen University
Conditions	Myelodysplastic Syndrome, Allogeneic Hematopoietic Stem Cell Transplantation, Conditioning
Interventions	Granulocyte Colony-Stimulating Factor(G-CSF), Decitabine (DAC), Busulfan, Fludarabine (FLU), Cyclophosphamide (CY), Busulfan (BU) (Drug)
Dates	start 2022-07-01, primary completion 2023-12-01 (estimated), completion 2024-12-01 (estimated), first posted 2022-07-12, last updated 2023-03-21
Locations	1 site in China

-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.

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+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.

+Arms and interventions

Arm	Type	Interventions	Description
G-CSF+DAC+BF	Experimental	Drug: Granulocyte Colony-Stimulating Factor(G-CSF); Drug: Decitabine (DAC); Drug: Fludarabine (FLU); Drug: Busulfan (BU)	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.
G-CSF+DAC+BUCY	Active comparator	Drug: Granulocyte Colony-Stimulating Factor(G-CSF); Drug: Decitabine (DAC); Drug: Busulfan; Drug: Cyclophosphamide (CY)	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.

Granulocyte Colony-Stimulating Factor(G-CSF) (Drug): G-CSF was administered at 5 ug/kg/day on days-17 to -10. When white blood cell is more than 20G/L, stop using G-CSF.
Decitabine (DAC) (Drug): Decitabine was administered at 20mg/m2/day on days -14 to -10.
Busulfan (Drug): Busulfan was administered at 3.2 mg/kg/day on days -7 to -4 in G-CSF+DAC+BUCY group.
Fludarabine (FLU) (Drug): Fludarabine was administered at 30 mg/m2/day on days -7 to -3.
Cyclophosphamide (CY) (Drug): Cyclophosphamide was administered at 60 mg/kg/day on days -3,-2.
Busulfan (BU) (Drug): Busulfan was administered at 3.2 mg/kg/day on days -6 to -3 in G-CSF+DAC +BF group.

+Outcome measures

Primary outcomes

Non-relapse mortality (NRM) [1 year]

Secondary outcomes

Overall survival (OS) [1 year]
Disease-free survival (DFS) [1 year]
Cumulative incidence of relapse [1 year]
Adverse effects [within 100 days post-transplantation]

+Eligibility

Sex: All, ages: 18 Years to 65 Years

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

+Locations (1)

Facility	City	Country	Status
Department of Hematology,Nanfang Hospital, Southern Medical University	Guangzhou, Guangdong	China	Recruiting

+Investigators

Qifa Liu, Principal investigator, Nanfang Hospital, Southern Medical University