G-CSF+Decitabine+BUCY vs BUCY Conditioning Regimen for RAEB-1, REAB-2 and AML Secondary to MDS Undergoing Allo-HSCT

Granulocyte Colony-stimulating Factor+Decitabine+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for Patients With RAEB-1, RAEB-2 and AML Secondary to MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) appears to be an efficient tool to cure refractory anemia with excess blasts-1 (RAEB-1), refractory anemia with excess blasts-2 (RAEB-2) and acute myeloid leukemia (AML) secondary to myelodysplastic syndrome (MDS). At present, the best conditioning regimen for RAEB-1, RAEB-2 and AML secondary to MDS undergoing allo-HSCT remains in discussion. In this prospective randomized controlled study, the safety and efficacy of G-CSF+ Decitabine + BUCY and BUCY myeloablative conditioning regimens in patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT are evaluated.

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndrome; Allogeneic Hematopoietic Stem Cell Transplantation; Conditioning
• Intervention / Treatment: Drug: Busulfan (BU); Drug: Cyclophosphamide (CY); Drug: Decitabine; Drug: Granulocyte Colony-Stimulating Factor(G-CSF)

Detailed Description

Allo-HSCT appears to be an efficient tool to cure patients with MDS and AML secondary to MDS. At present, the best conditioning regimen for MDS and AML secondary to MDS undergoing allo-HSCT remains in discussion. BUCY conditioning regimen is the standard myeloablative regimen for MDS and AML secondary to MDS undergoing allo-HSCT. However, it appears to have higher relapse rate. To reduce the relapse rate, decitabine is added in the conditioning regimen. In this prospective randomized controlled study, the safety and efficacy of G-CSF + Decitabine + BUCY and BUCY myeloablative conditioning regimens in RAEB-1, REAB-2 and AML secondary to MDS undergoing allo-HSCT are evaluated.

• Study Type: Interventional
• Enrollment (Actual): 202
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510515
      • Department of Hematology,Nanfang Hospital, Southern Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT
• 14-65 years

Exclusion Criteria:

• Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
• Patients with any conditions not suitable for the trial (investigators' decision)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: G-CSF + Decitabine + BUCY; For patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT ，Granulocyte Colony-Stimulating Factor(G-CSF)+Decitabine+BUCY conditioning regimen was G-CSF 5-10ug/kg/day on days -17 and -10 (when white blood cell is more than 20G/L, stop using G-CSF)；Decitabine 20mg/m2/day on days -14 and -10； Busulfan (BU) 3.2 mg/kg/day on days -7 and -4；Cyclophosphamide (CY) 60 mg/kg/day on days -3 and -2.	Drug: Busulfan (BU); Busulfan was administered at 3.2 mg/kg/day on days -7 to -4.; Drug: Cyclophosphamide (CY); Cyclophosphamide was administered at 60 mg/kg/day on days -3 to -2.; Drug: Decitabine; Decitabine was administered at 20mg/m2/day on days -14 and -10.; Drug: Granulocyte Colony-Stimulating Factor(G-CSF); G-CSF was administered at 5-10 ug/kg/day on days -17 and -10. When white blood cell is more than 20G/L, stop using G-CSF.
Active Comparator: BUCY; For patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT ，BUCY conditioning regimen was Busulfan (BU) 3.2 mg/kg/day on days -7 and -4；Cyclophosphamide (CY) 60 mg/kg/day on days -3 and -2.	Drug: Busulfan (BU); Busulfan was administered at 3.2 mg/kg/day on days -7 to -4.; Drug: Cyclophosphamide (CY); Cyclophosphamide was administered at 60 mg/kg/day on days -3 to -2.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
relapse rate	2 year

Secondary Outcome Measures

Outcome Measure	Time Frame
overall survival (OS)	2 year
disease-free survival (DFS)	2 year
transplant-related mortality (TRM)	2 year

Collaborators and Investigators

• Sponsor: Nanfang Hospital of Southern Medical University
• Collaborators: Liuzhou Workers Hospital; Peking University People's Hospital; Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University; Institute of Hematology & Blood Diseases Hospital; First People's Hospital of Chenzhou

Study record dates

Study Major Dates

• Study Start (Actual): April 18, 2016
• Primary Completion (Actual): September 30, 2019
• Study Completion (Actual): September 30, 2021

Study Registration Dates

• First Submitted: April 18, 2016
• First Submitted That Met QC Criteria: April 18, 2016
• First Posted (Estimate): April 20, 2016

Study Record Updates

• Last Update Posted (Actual): February 14, 2022
• Last Update Submitted That Met QC Criteria: January 30, 2022
• Last Verified: April 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Myelodysplastic Syndromes; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antirheumatic Agents; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Adjuvants, Immunologic; Cyclophosphamide; Decitabine; Lenograstim; Busulfan
• Other Study ID Numbers: G-CSF+Dec+BUCYvsBUCY-MDS-2016

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes