RATIONALE: Giving bortezomib together with melphalan and dexamethasone may be an effective treatment for primary amyloidosis and light chain deposition disease. PURPOSE: This phase II trial is studying how well giving bortezomib together with melphalan and dexamethasone works in treating patients with primary amyloidosis or light chain deposition disease.
OBJECTIVES: Primary * Determine the complete hematologic response rate at 12 months. Secondary * Determine the overall hematologic response rate. * Determine the organ response rate. * Determine time to treatment failure. * Determine the overall survival. OUTLINE: This is a multicenter study. Patients receive oral melphalan on days 1-4, bortezomib IV on days 1, 8, 15, and 22, and dexamethasone orally or IV on days 1, 2, 8, 9, 15, 16, 22, and 23. Treatment repeats every 4-6 weeks for up to 20 courses in the absence of disease progression or unacceptable toxicity. Blood, urine, and bone marrow aspirates are collected at baseline and periodically after treatment to permit the correlation of clinical results with measured molecular events. A single baseline peripheral blood DNA sample is collected for future association studies linking disease onset, progression, and response to administered therapy with single nucleotide polymorphisms. Blood plasma and urine samples are evaluated for proteomic markers associated with disease progression and therapeutic response. Peripheral blood RNA samples are evaluated for transcriptional response to treatment of peripheral blood lymphocytes. Bone marrow aspirates are collected to extract plasma cells by flow cytometry for gene expression profiling. Quality of life is assessed at the beginning of each course.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
35
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
Melphalan 9 mg/m2/day days 1-4
Rocky Mountain Cancer Centers/Rocky Mountain Blood & Marrow Transplant Program
Denver, Colorado, United States
Boston University Cancer Research Center
Boston, Massachusetts, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, United States
Josephine Ford Cancer Center at Henry Ford Hospital
Complete Hematologic Response
Time frame: Up to 12 months
Overall Survival
time from day of registration until day of death.
Time frame: time from day of registration until 72 months.
Time to Treatment Failure (TTF)
Time from start of treatment until date of documented disease progression, removal from protocol due to toxicity, or death from any cause.
Time frame: start of treatment until 72 months
Organ Response Rate (OrR)
Time frame: Beginning of cycles 4, 8, 12, 16 and 20, at follow up and end of study.
Overall Hematologic Response Rate (OHR)
Time frame: Beginning of cycles 4, 8, 12, 16 and 20, at follow up and end of study.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
≤28 days prior to enrollment
Day 1 of cycles 6, 12, 18 and at end of study.
≤28 days prior to enrollment
Start of each cycle
Detroit, Michigan, United States
Providence Cancer Institute at Providence Hospital - Southfield Campus
Southfield, Michigan, United States
Duke Comprehensive Cancer Center
Durham, North Carolina, United States
UPMC Cancer Centers
Pittsburgh, Pennsylvania, United States