Pre-Transplant 5-Azacitidine In Patients With High-Risk Myelodysplastic Syndrome (MDS) Who Are Candidates For Allogeneic Hematopoietic Cell Transplant
Summary
The purpose of this study is to find out if treating people who have high-risk myelodysplastic syndrome (MDS) with Vidaza (also called 5-azacitidine) prior to their allogeneic hematopoietic cell transplant (HCT) is helpful in preventing their myelodysplastic syndrome from coming back.
In previous research, Vidaza appeared to help the bone marrow of a patient with MDS begin to function more normally. This means bone marrow cells can grow and do their work the way they were meant to. Vidaza is approved by the Food and Drug Administration (FDA) for the treatment of MDS. The effect of Vidaza in patients receiving hematopoietic cell transplants have not been studied.
Description
RESEARCH PLAN
- This will be a single-center prospective trial
- Patients with high risk MDS that are potentially eligible for HCT will be enrolled.
- A donor search will be initiated, and Vidaza will be given per standard practice.
- Vidaza dose is 75 mg/M2/day subcutaneously by standard practice (generally this is 7 days per monthly cycle, but alterations occur depending on clinical and laboratory parameters).
- Patients where a suitable donor is not found can continue with Vidaza per standard treatment. These patients will be followed until progression of MDS to acute myelogenous leukemia (AML) or death, for up to one year.
- If a suitable donor is obtained, the patient will proceed to HCT. The HCT conditioning regimen will be dictated by the Blood and Marrow Transplant (BMT) physician. While waiting HCT, additional cycles Vidaza may be given. Pre-HCT conditioning regimen therapy will begin no more than 8 weeks and no less than 4 weeks after the last administration of Vidaza.
- As the number of cycles of Vidaza is not standardized and the retrospective review of our patients noted above indicated a benefit to ANY exposure to Vidaza, the actual number of cycles of Vidaza delivered will not be specified. In addition, as high risk MDS patients have an average time to death of 0.4 years, any delay to HCT once it is available is to be avoided.
- A bone marrow biopsy will be performed to reassess disease response to therapy after the last cycle of Vidaza before transplant, or after the fourth cycle of Vidaza, whichever comes first. Note that both the biopsy and the timing of the biopsy is a standard evaluation procedure.
- Donor progenitor cell collection will be prescribed by the BMT Attending Physician.
HCT
- The patient will undergo HCT designated per attending BMT physician.
- Supportive care will be based on institutional guidelines, Stem cell collections, processing and laboratory studies
Stem cell collections, processing and laboratory studies
- Graft assessment, processing, and characterization will be done as per institutional guidelines
- Chimerism testing will be obtained to document post-transplant engraftment, per standard practice.
Study Design
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Conditions
Leukemia
Intervention
5-azacitidine, Allogeneic Hematopoietic cell transplantation
Location
H. Lee Moffitt Cancer Center & Research Institute
Tampa
Florida
United States
33612
Status
Active, not recruiting
Source
H. Lee Moffitt Cancer Center and Research Institute
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT00660400
- Information obtained from ClinicalTrials.gov on February 14, 2013
Medical and Biotech [MESH] Definitions
Hematopoietic Stem Cell Transplantation
Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms.
Bone Marrow Transplantation
The transference of BONE MARROW from one human or animal to another for a variety of purposes including HEMATOPOIETIC STEM CELL TRANSPLANTATION or MESENCHYMAL STEM CELL TRANSPLANTATION.
Myelodysplastic Syndromes
Clonal hematopoietic stem cell disorders characterized by dysplasia in one or more hematopoietic cell lineages. They predominantly affect patients over 60, are considered preleukemic conditions, and have high probability of transformation into ACUTE MYELOID LEUKEMIA.
Hematopoietic Stem Cell Mobilization
The release of stem cells from the bone marrow into the peripheral blood circulation for the purpose of leukapheresis, prior to stem cell transplantation. Hematopoietic growth factors or chemotherapeutic agents often are used to stimulate the mobilization.
Genes, Myb
Retrovirus-associated DNA sequences (v-myb) originally isolated from the avian myeloblastosis and E26 leukemia viruses. The proto-oncogene c-myb codes for a nuclear protein involved in transcriptional regulation and appears to be essential for hematopoietic cell proliferation. The human myb gene is located at 6q22-23 on the short arm of chromosome 6. This is the point of break in translocations involved in T-cell acute lymphatic leukemia and in some ovarian cancers and melanomas. (From Ibelgaufts, Dictionary of Cytokines, 1995).
Clinical Trials
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Randomized Allogeneic Azacitidine Study
The goal of this clinical research study is to learn if Vidaza (azacitidine) will help to control the disease in patients with acute myelogenous leukemia (AML) or myelodysplastic syndrome...
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Primary Objective: -To determine the dose and schedule combination of 5-Azacitidine, when used as maintenance treatment after allogeneic transplantation for high-risk AML / MDS. Se...
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Objectives: - To determine if post transplant treatment with 5-azacytidine will increase the rate of complete molecular remission in patients with CML following allogeneic stem c...
PubMed Articles
This study evaluated azacitidine as treatment of minimal residual disease (MRD) determined by a sensitive donor chimerism analysis of CD34(+) blood cells to pre-empt relapse in patients with CD34(+) m...
There are limited data on hematopoietic cell transplantation (HCT) in primary plasma cell leukemia (pPCL), an aggressive plasma cell disorder. We report outcomes of 147 patients with pPCL receiving au...
BACKGROUND:: Recurrence is a major cause of treatment failure after allogeneic transplantation for acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS), and treatment options are very l...
Allogeneic hematopoietic stem cell transplantation (alloHSCT) is an effective post-remission therapy in patients with acute lymphoblastic leukemia (ALL), but is associated with significant toxicity, s...
