Bortezomib in Treating Patients With Myelodysplastic Syndromes

Conditions:Blood Cancer
Therapuetic Areas:Oncology
Age Range:18 - Any
Start Date:May 2005

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A Phase II Pilot Study of VELCADE in Patients With MDS

RATIONALE: Bortezomib may stop the growth of cancer cells by blocking some of the enzymes
needed for cell growth.

PURPOSE: This phase II trial is studying how well bortezomib works in treating patients with
myelodysplastic syndromes.



- Determine the efficacy of bortezomib, in terms of reduced cytopenia, in patients with
myelodysplastic syndromes.

- Determine the safety and toxic effects of this drug in these patients.


- Determine changes in marrow blast percentage or karyotypic profile in patients treated
with this drug.

OUTLINE: This is an open-label study.

Patients receive bortezomib IV on days 1, 4, 8, and 11. Treatment repeats every 21 days for
up to 12 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed periodically for 1 year.

PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study.


- Diagnosis of myelodysplastic syndromes (MDS)

- Requires treatment or transfusion support for MDS, as indicated by 1 of the

- Demonstrates transfusion or epoetin alfa dependence

- Transfusion dependence is defined as requiring ≥ 2 units of packed RBCs
within an 8-week period prior to study entry

- Hemoglobin < 11g/dL on 2 separate occasions 2 weeks apart

- No iron, cyanocobalamin (vitamin B_12), or folic acid deficiency or other
causes of anemia

- Must have 1 of the following FAB subtypes:

- Refractory anemia

- Refractory anemia with ringed sideroblasts

- Refractory anemia with excess blasts

- Secondary MDS (if ≥ 3 years since active primary cancer)

- No chronic myelomonocytic leukemia

- Not refractory to platelet transfusion support (i.e., inability to maintain platelet
count > 20,000/mm^3 with transfusion)

- No current acute myelogenous leukemia (e.g., > 30% blasts)


Performance status

- Karnofsky 50-100%

Life expectancy

- At least 6 months


- See Disease Characteristics


- Bilirubin ≤ 2 mg/dL

- AST and ALT < 2 times upper limit of normal


- Creatinine clearance ≥ 30 mL/min


- No significant cardiovascular condition that would preclude study participation

- No uncontrolled hypertension


- No significant pulmonary condition that would preclude study participation


- No serious concurrent infection

- Active infections must be adequately treated with antibiotics prior to study

- No hypersensitivity to bortezomib, boron, or mannitol


- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception during and for up to 4 weeks after
completion of study treatment

- No peripheral neuropathy ≥ grade 2

- No uncontrolled seizure activity, as defined by no activity within the past year on
stable anticonvulsant medications

- No other malignancy within the past 3 years except adequately treated basal cell skin
cancer or carcinoma in situ of the cervix

- No endocrine, neurologic, or other systemic disease that would preclude study entry


Biologic therapy

- See Disease Characteristics

- No prior allogeneic bone marrow transplantation

- Concurrent transfusion support allowed

- Concurrent epoetin alfa or darbepoetin alfa allowed if initiated before start of
study therapy, dose is stable for ≥ 4 weeks, and dose is stable during study

- No concurrent platelet growth factor support

- No concurrent thalidomide


- No concurrent chemotherapy

- No concurrent hydroxyurea

Endocrine therapy

- Concurrent corticosteroids for chronic autoimmune or inflammatory condition allowed
if initiated before start of study therapy and maintained on a stable or decreasing


- Recovered from all prior therapies

- At least 4 weeks since prior MDS therapy, except epoetin alfa, darbepoetin alfa,
filgrastim (G-CSF), pegfilgrastim (G-CSF), or transfusion support

- At least 30 days since prior investigational agents

- No prior bortezomib

- No other concurrent investigational agents

- No other concurrent therapy for MDS
We found this trial at
601 Elmwood Avenue
Rochester, New York 14642
(585) 275-5830
James P. Wilmot Cancer Center at University of Rochester Medical Center The Wilmot Cancer Center...
Rochester, NY
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