The primary endpoint of this study is to estimate morphologic complete remission rate. Estimation of response rate is also a secondary objection.
Myelodysplastic syndrome (MDS) is a hematological disorder characterized by ineffective hematopoiesis. The only known curative treatment for patients with MDS is allogeneic stem cell transplantation. However, only a minority of patients are candidates for this aggressive therapy. DNA hypomethylation agents have been shown to have activity in this disorder and are postulated to work by reversing this epigenetic mechanism of gene-silencing. Recently, 5-azacitidine, administered subcutaneously for seven days, received approval by the FDA for the therapy of MDS based on a randomized trial which demonstrated a diminished risk of leukemic transformation and improved survival when compared to best supportive care. The subcutaneous route of administration can present challenges to implementing this therapy. In the CALGB studies 8921 and 9221, approximately 23% of patients had significant injection site pain. Moreover, 35 % of patients had injection site bruising which can be extensive in thrombocytopenic patients. Due to limitations on drug concentration and administration volumes for subcutaneous dosing, patients often need to have two or three injections at separate sites each day to meet target dosing. In addition, the schedule of administration is inconvenient in an outpatient setting secondary to the need to schedule administrations over weekends. Therefore, there is great interest in pursuing an abbreviated intravenous route for administration of the drug.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Washington University School of Medicine
St Louis, Missouri, United States
Rate of Complete Remission (CR) and Partial Remission (PR)
Defined according to the modified International Working Group (IWG) (2006) response criteria for myelodysplasia: CR=bone marrow with \<5% myeloblasts and 0% peripheral blasts, hemoglobin ≥11g/dL, platelets ≥ 100 x 10\^9/L, and neutrophils ≥1.0 x 10\^9/L. Residual dysplasia was allowed. PR= All of the CR criteria if abnormal before treatment except: bone marrow blasts decreased by ≥50% over pretreatment but still \>5%.
Time frame: After 4 cycles of therapy (up to 112 days after start of treatment)
Rate of Hematologic Improvement
International Working Group (IWG) for Myelodysplasia (MDS).
Time frame: 4 weeks following last azacitidine dose [median number of cycles 4.5 (1-20)]
Rate of Transfusion Independence
Time frame: 4 weeks following last azacitidine dose [median number of cycles 4.5 (1-20)]
Rate of Cytogenetic Response
Time frame: 2 years after first dose of study drug or until participant is lost to follow-up or dies
Rate of Overall Survival
Overall survival is defined as the date of first dose of study drug to the date of death from any cause.
Time frame: 2 years after first dose of study drug or until participant is lost to follow-up or dies
Rate of Relapse After Hematopoietic Stem Cell Transplant in Individuals Treated With 5-azacitidine Prior to Transplant.
Time frame: 2 years after first dose of study drug or until participant is lost to follow-up or dies
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.