A multicenter, prospective study of bortezomib and dexamethasone as induction treatment followed by high dose melphalan (HDM) and autologous stem cell transplantation (SCT) in patients with de novo amyloid light chain (AL) amyloidosis.
Completed
- Conditions
- de novo amyloid (AL) amyloidosis
- Registration Number
- NL-OMON23469
- Lead Sponsor
- Stichting Hemato-Oncologie voor Volwassenen Nederland(HOVON), HOVON Data Center
- Brief Summary
PMID: 30923094
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 50
Inclusion Criteria
1. Biopsy proven, systemic, untreated AL amyloidosis requiring systemic chemotherapy;
2. Age 18 -70 years inclusive at the time of signing the informed consent form;
Exclusion Criteria
1. Multiple Myeloma stage II and III (Durie and Salmon);
2. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form;
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Hematological CR rate 6 months after auto-SCT. Patients are considered a success if they received HDM and auto-SCT and are in CHR at 6 months after auto-SCT; all other patients are considered a failure.
- Secondary Outcome Measures
Name Time Method 1. Overall survival measured from the time of registration. Patients still alive or lost to follow up are censored at the day they were last known to be alive;<br /><br>2. Progression Free Survival, (hematological), i.e. time from registration until hematological progression, relapse or death, whichever occurs first;<br /><br>3. Hematological response rate rate after induction therapy;<br /><br>4. Response rate, hematological and organ;<br /><br>5. Time to response, hematological and organ;<br /><br>6. Duration of response, hematological and organ;<br /><br>7. Time to next AL amyloidosis therapy;<br /><br>8. Safety (type, frequency, and severity of adverse events (AE) and relationship of AE to study drug;<br /><br>9. Exploratory assessment of multiparameter flow cytometry quantification of bone marrow plasma cells and change in amyloid deposition in abdominal fat aspiration samples;<br /><br>10. Evaluation of prognostic factors for survival included in the hematological and organ response criteria.