A Phase II Trial of Tandem Transplantation in AL Amyloidosis
18 Years
65 Years
Both
Multiple Myeloma and Plasma Cell Neoplasm
Trial Information
A Phase II Trial of Tandem Transplantation in AL Amyloidosis
OBJECTIVES:
- Determine the tolerability of tandem autologous stem cell transplantation in patients
with AL amyloidosis.
- Determine whether this regimen can convert a hematologic non-complete response (CR) to
CR in these patients.
- Determine the overall survival of patients treated with this regimen.
OUTLINE:
- First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily
beginning 3 days before the initiation of stem cell collection and continuing until the
day before the completion of stem cell collection. Patients may undergo bone marrow
harvest if an inadequate number of peripheral blood stem cells are collected.
Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo
autologous stem cell transplantation (ASCT) on day 0.
- Second transplantation: Within 6-12 months after the first ASCT, patients not achieving
a complete response receive high-dose melphalan IV over 20 minutes on days -3 and -2
and a second ASCT on day 0.
Treatment continues in the absence of unacceptable toxicity.
Patients are followed at 3 and 6 months, 1 year, and then annually thereafter.
PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 years.
Inclusion Criteria
DISEASE CHARACTERISTICS:
- Histologically confirmed AL amyloidosis, meeting 1 of the following criteria:
- Plasma cell dyscrasia, evidenced by 1 of the following:
- Monoclonal protein in the serum or urine by immunofixation electrophoresis
- Plasmacytosis of the bone marrow with monoclonal staining for kappa or
lambda light chain isotype
- Macroglossia with at least 1 other site having biopsy proven amyloidosis and
absence of a mutant transthyretin is ruled out
- No senile, secondary, localized, dialysis-related, or familial amyloidosis
- No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis,
extensive [more than 2] lytic lesions, hypercalcemia)
PATIENT CHARACTERISTICS:
Age
- 18 to 65
Performance status
- SWOG 0-2
Life expectancy
- At least 1 year
Hematopoietic
- Not specified
Hepatic
- Not specified
Renal
- Not specified
Cardiovascular
- LVEF ≥ 45% by MUGA or echocardiogram
- No myocardial infarction within the past 6 months
- No congestive heart failure
- No arrhythmia refractory to therapy
- No evidence of symptomatic transient ischemic attacks or strokes
Pulmonary
- DLCO ≥ 50%
Other
- Not pregnant or nursing
- Fertile patients must use effective contraception
- Able to tolerate 2 courses of high-dose therapy
- HIV negative
- No other malignancy within the past 5 years except adequately treated basal cell or
squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated
stage I or II cancer currently in complete remission
PRIOR CONCURRENT THERAPY:
Biologic therapy
- Not specified
Chemotherapy
- Prior alkylating agent chemotherapy allowed provided there is no morphologic or
cytogenetic evidence of myelodysplastic syndromes
- Prior total cumulative oral melphalan dose < 300 mg
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Other
- At least 4 weeks since prior cytotoxic therapy and recovered
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
safety
Outcome Time Frame:
100 days, 6 months, and annual
Safety Issue:
Yes
Principal Investigator
Vaishali Sanchorawala, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Boston Medical Center
Authority:
United States: Federal Government
Study ID:
CDR0000347381
NCT ID:
NCT00075621
Start Date:
August 2000
Completion Date:
May 2015
Related Keywords:
- Multiple Myeloma and Plasma Cell Neoplasm
- primary systemic amyloidosis
- Amyloidosis
- Neoplasms
- Multiple Myeloma
- Neoplasms, Plasma Cell
- Plasmacytoma
Name | Location |
|---|---|
| Cancer Research Center at Boston Medical Center | Boston, Massachusetts 02118 |
