Clinical trial NCT00003961: Sargramostim After Bone Marrow Transplantation in Treating Patients With Myelodysplastic Syndrome
Official title: Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) After T-Lymphocyte Depleted Allogeneic BMT for Myelodysplastic Syndromes
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Leukemia
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Keywords: refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
chronic myelomonocytic leukemia
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
refractory cytopenia with multilineage dysplasia
atypical chronic myeloid leukemia
myelodysplastic/myeloproliferative disease, unclassifiable
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2003-01-27
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: RATIONALE: Colony-stimulating factors, such as sargramostim, may increase the number of immune cells found in bone marrow or peripheral blood.

PURPOSE: Phase II trial to study the effectiveness of sargramostim after bone marrow transplantation in treating patients who have myelodysplastic syndrome.

Detailed description: OBJECTIVES:

* Determine the effect of sargramostim (GM-CSF) on the progression-free 1-year survival of patients with myelodysplastic syndrome who have undergone T-cell-depleted CD34+ augmented allogeneic bone marrow transplantation.

OUTLINE: All patients receive elutriated, CD34+ stem cell augmented donor bone marrow according to another protocol on day 0.

Patients receive sargramostim (GM-CSF) subcutaneously on days 5-60.

Patients are followed on days 120, 180, 360 and periodically thereafter.

PROJECTED ACCRUAL: A total of 45 patients will be accrued for this study within 3-4 years.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Cytologically proven myelodysplastic syndrome (MDS) of one of the following types:

  * Refractory anemia with excess blasts (RAEB)
  * RAEB in transformation
  * Chronic myelomonocytic leukemia
  * MDS with multiple chromosomal abnormalities
  * MDS with life threatening cytopenias in at least 2 cell lines

    * Platelet count \< 30,000/mm\^3 OR
    * Absolute neutrophil count no greater than 1,000/mm\^3 OR
    * Anemia requiring transfusion support
  * Leukemia out of MDS (meet any of above requirements, but greater than 30% blasts in marrow)
* No acute leukemia
* Medically eligible for bone marrow transplant according to standard operating procedure of the Sidney Kimmel Cancer Center at Johns Hopkins Blood and Bone Marrow Transplant

PATIENT CHARACTERISTICS:

Age:

* 18 to 65

Performance status:

* ECOG 0-1

Life expectancy:

* Not specified

Hematopoietic:

* See Disease Characteristics

Hepatic:

* Not specified

Renal:

* Not specified

Other:

* No prior acute allergic reactions to sargramostim (GM-CSF)
* Not pregnant
* Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

Biologic therapy:

* Not specified

Chemotherapy:

* Not specified

Endocrine therapy:

* Not specified

Radiotherapy:

* Not specified

Surgery:

* Not specified

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
state: Maryland
Study officials:
affiliation: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
name: B. Douglas Smith, MD
role: PRINCIPAL_INVESTIGATOR
References: