Phase 3
N=89
Low-Dose Melphalan and Dexamethasone Compared With High-Dose Melphalan Followed By Autologous Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
Multiple Myeloma and Plasma Cell Neoplasm
Bottom Line
View on ClinicalTrials.gov: NCT00477971 ↗Enrolled (actual)
89
Serious AEs
10.1%
Results posted
Jul 2015
Primary outcome: Primary: Hematologic Response Rate — 55.9; 69.1 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- filgrastim (Biological); dexamethasone (Drug); melphalan (Drug); autologous hematopoietic stem cell transplantation (Procedure)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Mayo Clinic
- Primary completion
- Jul 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Hematologic Response Rate |
55.9; 69.1 | — |
| SECONDARY 3 Year Overall Survival |
58.8; 83.6 | — |
| SECONDARY Organ Response to Treatment |
26.5; 29.1 | — |
Summary
RATIONALE: Drugs used in chemotherapy, such as melphalan and dexamethasone, work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having an autologous stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly. It is not yet known whether combination chemotherapy is more effective than chemotherapy followed by an autologous stem cell transplant in treating primary systemic amyloidosis.
PURPOSE: This randomized phase III trial is studying the side effects and how well giving low-dose melphalan together with dexamethasone works compared with high-dose melphalan followed by an autologous stem cell transplant in treating patients with primary systemic amyloidosis.
Eligibility Criteria
DISEASE CHARACTERISTICS:
- Histologically confirmed primary systemic amyloidosis
- Amyloid light-chain (AL) disease
- Monoclonal protein by immunoelectrophoresis or immunofixation of the serum or urine OR abnormal free light-chain ratio
- The following amyloid syndromes* are allowed:
- Amyloid hepatomegaly
- Cardiomyopathy
- Proteinuria
- Peripheral or autonomic neuropathy
- Soft tissue involvement including the tongue, submandibular tissues, and vascular claudication
- Diffuse interstitial pulmonary AL disease allowed if pulmonary function is adequate to allow safe transplantation NOTE: *Presence of amyloid deposits in a plasmacytoma or in bone marrow vessels in an asymptomatic patient does not constitute an amyloid syndrome
- No secondary or familial amyloidosis
- No multiple myeloma with lytic or destructive bone lesions or myeloma cast nephropathy
- No multiple myeloma with > 30% plasma cells in the bone marrow
- No amyloidosis manifested only by carpal tunnel syndrome or purpura
PATIENT CHARACTERISTICS:
- ECOG performance status 0-2
- Platelet count ≥ 100,000/mm³
- Bilirubin ≤ 2.0 times upper limit of normal (ULN)
- Alkaline phosphatase ≤ 6 times ULN
- Creatinine ≤ 3.0 mg/dL
- No NYHA class IV heart disease
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- No uncontrolled infection
- No HIV positivity
PRIOR CONCURRENT THERAPY:
- Prior alkylating agents, immunosuppressive drugs, or steroids allowed provided they were given for < 1 month
- Therapeutic steroid doses of ≤ 15 mg per day (or equivalent) allowed at discretion of physician
- No concurrent participation in another clinical trial involving a pharmacologic agent
Data sourced from ClinicalTrials.gov (NCT00477971). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.