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A Pilot Study Of Pre-Transplant 5-Azacitidine (Vidaza) In Patients With High-Risk Myelodysplastic Syndrome Who Are Candidates For Allogeneic Hematopoietic Cell Transplantation

18 Years
68 Years
Open (Enrolling)

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Trial Information

A Pilot Study Of Pre-Transplant 5-Azacitidine (Vidaza) In Patients With High-Risk Myelodysplastic Syndrome Who Are Candidates For Allogeneic Hematopoietic Cell Transplantation


- 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

- 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.


- 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

- Chimerism testing will be obtained to document post-transplant engraftment, per
standard practice.

Inclusion Criteria:

- Potential candidate for HCT.

- Histologically confirmed diagnosis by pathologic review of previous diagnosis of
high-risk myelodysplastic syndrome (MDS): International Prognostic Scoring System
(IPSS) > 1 or AML-MDS or treatment related MDS.

- Serum bilirubin levels ≤1.5 times the upper limit of the normal (ULN) range for the
laboratory. Higher levels are acceptable if these can be attributed to active
hemolysis or ineffective erythropoiesis; Serum glutamic-oxaloacetic transaminase
(SGOT) (aspartate aminotransferase [AST]) or serum glutamicpyruvic transaminase
(SGPT) (alanine aminotransferase [ALT]) levels ≤2 x ULN.

- Serum creatinine levels ≤1.5 x ULN

- Karnofsky performance status greater or equal to 70%

- Signed informed consent form in accordance with institutional policies

Exclusion Criteria:

- Known or suspected hypersensitivity to Vidaza or mannitol

- Pregnant or lactating women

- Human immunodeficiency virus (HIV) or seropositive, confirmed by nucleic acid
amplification testing (NAT)

- Active central nervous system (CNS) malignancy

- Active infection

- History or presence of primary hepatoma

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants With Progression Free Survival (PFS)

Outcome Description:

Evaluate progression free survival at one year after allogeneic hematopoietic cell transplantation (HCT) in patients receiving at least one complete cycle of Vidaza in the pretransplant setting. Progression-free survival time will be calculated by the method of Kaplan-Meier with standard errors computed using Greenwood's formula.

Outcome Time Frame:

One year

Safety Issue:


Principal Investigator

Teresa Field, M.D., Ph.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

H. Lee Moffitt Cancer Center and Research Institute


United States: Institutional Review Board

Study ID:




Start Date:

March 2008

Completion Date:

April 2013

Related Keywords:

  • Leukemia
  • Myeloid
  • Monocytic
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia



H. Lee Moffitt Cancer Center & Research Institute Tampa, Florida  33612