Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia

NCT ID: NCT00322101

Last Updated: 2014-10-31

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-01-31

Study Completion Date

2014-10-31

Brief Summary

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RATIONALE: Giving chemotherapy, such as fludarabine phosphate, busulfan, and cyclophosphamide, and total-body radiation therapy before a donor peripheral stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether low-dose chemotherapy and total-body radiation therapy is more effective than high-dose chemotherapy in treating patients with myelodysplastic syndrome or acute myeloid leukemia.

PURPOSE: This phase III trial is studying low-dose conditioning to see how well it works compared to high-dose conditioning followed by peripheral blood stem cell transplant in treating patients with myelodysplastic syndromes or acute myeloid leukemia

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Detailed Description

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OBJECTIVES:

I. Determine whether the conditioning intensity affects outcomes after HCT in patients with MDS or AML who have \< 5% marrow myeloblasts at the time of HCT.

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

Arm I (Nonmyeloablative regimen):

CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and undergo low-dose total-body irradiation on day 0.

TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell (PBSC) infusion on day 0.

GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive cyclosporine every 12 hours on days -3 to 57 with taper on days 57-177 or cyclosporine every 12 hours on days -3 to 100 with taper on days 101-177. Patients also receive oral mycophenolate mofetil every 12 hours on days 0-27 or every 8 hours on days 0-40 with taper on days 41-96.

Arm II (Myeloablative regimen):

CONDITIONING: Patients are assigned to 1 of 2 treatment groups.

Group A: Patients receive fludarabine IV once daily and oral busulfan four times daily or busulfan IV over 3 hours on days -5 to -2.

Group B: Patients receive cyclophosphamide IV over 1-2 hours on days -3 and -2 and oral busulfan four times daily or busulfan IV over 3 hours on days -7 to -4.

TRANSPLANTATION: Patients undergo PBSC infusion on day 0.

GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV continuously or orally every 12 hours on days -1 to 56 and taper on days 57-200. Patients also receive methotrexate IV on days 1, 3, 6, and 11.

Treatment in both arms continues in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up periodically.

Conditions

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Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome Acute Myeloid Leukemia/Transient Myeloproliferative Disorder Adult Acute Myeloid Leukemia in Remission Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities Adult Acute Myeloid Leukemia With Del(5q) Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) Childhood Acute Myeloid Leukemia in Remission Childhood Myelodysplastic Syndromes de Novo Myelodysplastic Syndromes Myelodysplastic Syndrome With Isolated Del(5q) Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable Previously Treated Myelodysplastic Syndromes Recurrent Adult Acute Myeloid Leukemia Recurrent Childhood Acute Myeloid Leukemia Secondary Acute Myeloid Leukemia Secondary Myelodysplastic Syndromes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm I (Nonmyeloablative regimen)

CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and undergo low-dose total-body irradiation on day 0.

TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell (PBSC) infusion on day 0.

GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive cyclosporine every 12 hours on days -3 to 57 with taper on days 57-177 or cyclosporine every 12 hours on days -3 to 100 with taper on days 101-177. Patients also receive oral mycophenolate mofetil every 12 hours on days 0-27 or every 8 hours on days 0-40 with taper on days 41-96.

Group Type EXPERIMENTAL

total-body irradiation

Intervention Type RADIATION

Radiation

mycophenolate mofetil

Intervention Type DRUG

Given orally

cyclosporine

Intervention Type DRUG

Given IV or orally

fludarabine phosphate

Intervention Type DRUG

Given IV

peripheral blood stem cell transplantation

Intervention Type PROCEDURE

Undergo transplantation

nonmyeloablative allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

Undergo allogeneic transplantation

laboratory biomarker analysis

Intervention Type OTHER

Correlative studies

cytogenetic analysis

Intervention Type GENETIC

Correlative studies

flow cytometry

Intervention Type OTHER

Correlative studies

fluorescence in situ hybridization

Intervention Type GENETIC

Correlative studies

pharmacological study

Intervention Type OTHER

Correlative studies

polymorphism analysis

Intervention Type GENETIC

Correlative studies

Arm II (Myeloablative regimen)

CONDITIONING: Patients are assigned to 1 of 2 treatment groups.

Group A: Patients receive fludarabine IV once daily and oral busulfan four times daily or busulfan IV over 3 hours on days -5 to -2.

Group B: Patients receive cyclophosphamide IV over 1-2 hours on days -3 and -2 and oral busulfan four times daily or busulfan IV over 3 hours on days -7 to -4.

TRANSPLANTATION: Patients undergo PBSC infusion on day 0.

GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV continuously or orally every 12 hours on days -1 to 56 and taper on days 57-200. Patients also receive methotrexate IV on days 1, 3, 6, and 11.

Group Type EXPERIMENTAL

allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

Undergo allogeneic transplantation

cyclophosphamide

Intervention Type DRUG

Given IV

busulfan

Intervention Type DRUG

Given IV or orally

fludarabine phosphate

Intervention Type DRUG

Given IV

peripheral blood stem cell transplantation

Intervention Type PROCEDURE

Undergo transplantation

laboratory biomarker analysis

Intervention Type OTHER

Correlative studies

flow cytometry

Intervention Type OTHER

Correlative studies

fluorescence in situ hybridization

Intervention Type GENETIC

Correlative studies

pharmacological study

Intervention Type OTHER

Correlative studies

polymorphism analysis

Intervention Type GENETIC

Correlative studies

tacrolimus

Intervention Type DRUG

Given IV or orally

methotrexate

Intervention Type DRUG

Given IV

Interventions

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total-body irradiation

Radiation

Intervention Type RADIATION

allogeneic hematopoietic stem cell transplantation

Undergo allogeneic transplantation

Intervention Type PROCEDURE

cyclophosphamide

Given IV

Intervention Type DRUG

mycophenolate mofetil

Given orally

Intervention Type DRUG

busulfan

Given IV or orally

Intervention Type DRUG

cyclosporine

Given IV or orally

Intervention Type DRUG

fludarabine phosphate

Given IV

Intervention Type DRUG

peripheral blood stem cell transplantation

Undergo transplantation

Intervention Type PROCEDURE

nonmyeloablative allogeneic hematopoietic stem cell transplantation

Undergo allogeneic transplantation

Intervention Type PROCEDURE

laboratory biomarker analysis

Correlative studies

Intervention Type OTHER

cytogenetic analysis

Correlative studies

Intervention Type GENETIC

flow cytometry

Correlative studies

Intervention Type OTHER

fluorescence in situ hybridization

Correlative studies

Intervention Type GENETIC

pharmacological study

Correlative studies

Intervention Type OTHER

polymorphism analysis

Correlative studies

Intervention Type GENETIC

tacrolimus

Given IV or orally

Intervention Type DRUG

methotrexate

Given IV

Intervention Type DRUG

Other Intervention Names

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TBI CPM CTX Cytoxan Endoxan Endoxana Cellcept MMF BSF BU Misulfan Mitosan Myeloleukon ciclosporin cyclosporin cyclosporin A CYSP Sandimmune 2-F-ara-AMP Beneflur Fludara PBPC transplantation PBSC transplantation peripheral blood progenitor cell transplantation transplantation, peripheral blood stem cell fluorescence in situ hybridization (FISH) pharmacological studies FK 506 Prograf amethopterin Folex methylaminopterin Mexate MTX

Eligibility Criteria

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Inclusion Criteria

* Myelodysplastic syndrome (MDS) or transformed acute myelogenous leukemia (transformed from MDS)
* De novo acute myelogenous leukemia (AML) beyond first remission
* Intermediate or high risk de novo AML in first complete response (at FHCRC unrelated donor recipients only)
* Chemotherapy required prior to HCT for all patients:
* A) Interval between start of a cycle of cytoreductive chemotherapy and infusion of donor stem cells must be at least 30 days; chemotherapy received for disease maintenance will be allowed during this time period
* B) All patients must have \< 5% myeloblasts based on marrow morphology performed within 21 days prior to start of conditioning regimen and at least 3-4 weeks after the start of pre-transplant cytoreductive chemotherapy
* C) All patients must have no circulating peripheral blood myeloblasts present based on morphologic analysis
* Age 65 years or under for patients with related donors; age 60 years or under for patients with unrelated donors
* HCT-Specific Comorbidity Index Score (HCT-CI) \< 3
* Related donor (age \> 12 years, nonsyngeneic) or unrelated donor, HLA phenotypically or genotypically identical at the allele level at A,B,C,DRQ1, and CBQ1
* DONOR: Related or unrelated donors who are genotypically or phenotypically matched by high resolution HLA typing (HLA-A, B, C, DRB1, and DQB1); class 1 single allele mismatch allowed
* DONOR: Patient and donor pairs homozygous at a mismatched allele in the graft rejection vector are considered a two-allele mismatch, i.e., the patient is A\*0101 and the donor is A\*0201, and this type of mismatch is not allowed
* DONOR: A positive anti-donor cytotoxic crossmatch or flow cytometric assay is an absolute donor exclusion at FHCRC/SCCA
* DONOR: Age \>= 12 years
* DONOR: Donors must consent to PBSC mobilization with G-CSF and leukaphereses; bone marrow as a source of stem cells will not be allowed
* DONOR: Donor must have adequate veins for leukaphereses or agree to placement of central venous catheter (femoral, subclavian)

Exclusion Criteria

* HIV seropositivity
* Fungal infections with radiographic progression after appropriate therapy for greater than one month
* Organ dysfunction
* Symptomatic coronary artery disease or ejection fraction \< 35%
* DLCO \< 65%, FEV1 \< 65% or receiving supplementary continuous oxygen
* Liver function abnormalities: Patients with clinical or laboratory evidence of liver disease will be evaluated for the cause of liver disease, its clinical severity in terms of liver function, histology, and the degree of portal hypertension; patients with fulminant liver failure, cirrhosis with evidence of portal hypertension or bridging fibrosis, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin \> 3 mg/dL, and symptomatic biliary disease will be excluded
* Karnofsky Performance Score \< 70
* Lansky-Play Performance Score \< 70 for pediatric patients
* Life expectancy severely limited (\< 2 years) by disease other than MDS/AML
* Fertile men and women unwilling to use contraceptive techniques during and for 12 months following treatment
* Patients with active non-hematological malignancies except:
* A) Patients with follicular or low grade lymphoma will be eligible as long as they have not and do not require active treatment for control of their disease
* B) Patients with localized non-melanoma skin malignancies
* Patients with poorly controlled hypertension who are unable to have blood pressure stabilized below 150/90 mm Hg on standard medication
* Females who are pregnant or breastfeeding
* Patients with systemic, uncontrolled infections
* Active CNS disease as identified by positive CSF cytospin
* DONOR: Identical twin
* DONOR: Age \< 12 years
* DONOR: Pregnancy
* DONOR: HIV seropositivity
* DONOR: Inability to achieve adequate venous access
* DONOR: Known adverse reaction to G-CSF
Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

Fred Hutchinson Cancer Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bart Scott

Role: PRINCIPAL_INVESTIGATOR

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Locations

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HealthOne Presbyterian St. Lukes Medical Center

Denver, Colorado, United States

Site Status

Emory University

Altanta, Georgia, United States

Site Status

Weill Cornell University

New York, New York, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Veterans Administration Center-Seattle

Seattle, Washington, United States

Site Status

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Seattle, Washington, United States

Site Status

Medical College Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Technical University Dresden

Dresden, Saxony, Germany

Site Status

Countries

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United States Germany

Other Identifiers

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NCI-2010-00737

Identifier Type: REGISTRY

Identifier Source: secondary_id

P01CA078902

Identifier Type: NIH

Identifier Source: secondary_id

View Link

K23HL084054

Identifier Type: NIH

Identifier Source: secondary_id

View Link

P01CA018029

Identifier Type: NIH

Identifier Source: secondary_id

View Link

P01HL036444

Identifier Type: NIH

Identifier Source: secondary_id

View Link

1992.00

Identifier Type: -

Identifier Source: org_study_id

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