5-Azacytidine Prior to Allogeneic Stem Cell Transplant in High Risk Myelodysplastic Syndrome

Virginia Commonwealth University16 enrolled

Overview

The purpose of this study is to examine the feasibility and efficacy of using the demethylating agent 5-Azacytidine prior to allogeneic stem cell transplantation in patients with high risk myelodysplastic syndrome (MDS).

The study drug, 5-azacytidine, is given daily intravenously for 7 days. After every 2 cycles study participants will have a bone marrow test to evaluate the effect of the 5-azacytidine on the Myelodysplastic Syndrome (MDS). Participants continue to get cycles of 5-Azacytidine until 2 bone marrow tests show the MDS has stopped responding to the treatment. At that time they will undergo a transplant if a donor is available.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Myelodysplastic Syndrome

Interventions

5-azacytidineDRUG

The recommended starting dose for the first treatment cycle, for all patients regardless of baseline hematology laboratory values, is 75 mg/m2 subcutaneously or intravenously, daily for 7 days.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients fulfilling the following criteria will be eligible for study entry: 1. Diagnosis of MDS according to WHO criteria 2. Intermediate-2 or high risk by IPSS score 3. Clinically able to receive 5-Azacytidine 4. Serum bilirubin levels \</=1.5 times the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed to active hemolysis or ineffective erythropoiesis 5. Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) levels \</=2 x ULN 6. Serum creatinine levels \</=1.5 x ULN 7. Negative serum pregnancy test prior to 5-Azacytidine treatment for women of childbearing potential 8. Women and men of childbearing potential agree to use contraception while receiving treatment with 5-Azacytidine 9. Potentially eligible for allogeneic transplantation 10. No prior allogeneic transplant 11. Age 18 to 70, inclusive. Exclusion Criteria: 1. Known or suspected hypersensitivity to 5-azacytidine or mannitol 2. Patients previously treated with 5-azacytidine or deoxyazacytidine 3. Pregnant or breast feeding 4. Patients with advanced malignant hepatic tumors

Locations (1)

Massey Cancer Center / Virginia Commonwealth University

Richmond, Virginia, United States

Outcomes

Primary Outcomes

One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation

Percentage of patients alive one year after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated one year survival rate from this curve is 50%, while the estimated two year survival rate is 50%.

Time frame: 1 year

Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation

Percentage of patients alive two years after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated two year survival rate is 50%, the same as one year survival rate.

Time frame: 2 years

One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation

Percentage of participants that received allogeneic transplant and had event free survival, as estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated one-year event-free survival rate is the same as overall survival, 50%.

Time frame: 1 year

Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation

Percentage of participants that received allogeneic transplant and had event free survival. The percentage of patients was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated two-year event-free survival rate is the same as overall survival, 50%.

Time frame: 2 years

Secondary Outcomes

One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts

Percentage of participants alive one year after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored. The estimated one year overall survival rate from this curve is 47%. The one year overall survival is the same as one year event free survival rate.

Data from ClinicalTrials.gov

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