Bortezomib and Dexamethasone Followed by ASCT Compared With ASCT Alone in Treating Patients With AL Amyloidosis

Arm 1: The BD regimen included bortezomib 1.3 mg/m2 i.v. and dexamethasone 40 mg p.o. on days 1, 4, 8 and 11 of the 21 day cycle. This process was repeated for 2 cycles. After two cycles of BD therapy, the collection of peripheral blood stem cells (PBSC) should be completed within 4 weeks. Recommended dose of drug is as follows: granulocyte colony-stimulating factor (G-CSF) 5-10ug/kg on days 1-5 will be given, then peripheral blood stem cells will be collected on days 5-6 for 2×10^6 CD34+ cells /kg. Patients will receive ASCT therapy in 8 weeks after collection of PBSC (Recorded as day 0), while melphalan (day -2) with a dose of 140 or 200 mg/m2 (choosing a dose according to the degree of risk for patients). Melphalan will be administered by central venous catheter.

Arm 2: the patients who assigned to arm 2 will receive ASCT alone as an initial treatment. The process of ASCT is as same as arm 1.

Inclusion criteria

• Patients with newly diagnosed AL amyloidosis
• Abnormal M protein or free light chain detected in serum and/or urine
• ECOG score 0-2 points
• No absolute neutrophil count of ANC less than or equal to 1000 within 14 days before enrollment
• No platelet count of less than or equal to 50K within 14 days before enrollment
• Serum bilirubin must lower than 2.0 mg/dl within 14 days before enrollment
• Serum creatinine must lower than 2.0 mg/dl within 14 days before enrollment
• Must have LVEF at least 45% by ECHO within 14 days of enrollment
• Pulmonary Function Tests must show DLCO at least 50%

Exclusion criteria

• Subjects have received or are currently receiving systematic treatment with steroids (not including an emergent short-term use of steroids before randomization up to 4 days, maximum dose of 40mg/d)
• Pregnant and breastfeeding women, delivery term women or unwilling to take birth control measures during the study
• Subjects suffering from multiple myeloma
• Grade 2 or more than grade 2 peripheral neuropathy or neuropathic pain according to National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 3
• Known or suspected hypersensitivity to dexamethasone, bortezomib, mannitol, boron, or heparin (if use catheters)
• Subjects suffering from uncontrolled or severe cardiovascular disease, including myocardial infarction, class III-IV heart failure defined by New York Heart Association (NYHA), uncontrolled angina, clinical significant pericardial disease or cardiac amyloidosis (Other contraindications are not suitable for transplant patients) within 6 months before enrollment
• Subjects suffering from serious physical disease and mental illnesses which may interfere the study
• Subjects receiving other pilot study or treatment within 4 weeks before enrollment