What the trial was testing
The trial enrolled 40 patients with al amyloidosis. The study was sponsored by Columbia University and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
57% had a partial response or better in relapsed AL amyloidosis.
Journal of Clinical Oncology · 2020 · NCT01222260
These findings — that achieved partial response or better in relapsed AL amyloidosis on bendamustine + dex — were published in the Journal of Clinical Oncology and represent the headline result of the study.
Researchers tracked outcomes across 40 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with al amyloidosis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Bendamustine (Treanda) is FDA-approved for chronic lymphocytic leukemia and lymphoma but is used off-label for AL amyloidosis. The first-line FDA-approved option for AL amyloidosis now includes daratumumab combinations. Ask a hematologist with amyloidosis expertise about treatment.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open al amyloidosis trials
Ohio State University Multiple Myeloma and Amyloidosis Data Registry and Sample Resource
The investigators are researching patients with diseases of their plasma cells in order to improve their quality and length of life. The investigators have created a database of patient information, blood samples, and bone marrow tissue in order to achieve the following three goals: * Surveillance: The investigators want to track what treatments patients get or don't get, how effective they are, how they feel, what complications they suffer, how long they stay in remission, and how long they live. * Contact: Because myeloma and amyloidosis are rare, less than 700 patients are diagnosed in the state of Ohio each year, patients often feel they don't have accurate information. The investigators want to provide them access to our clinical team (both phone and email consultations, even office visits for patients that can come to Columbus) as well as information regarding informational events pertaining to your disease and local support groups. * Research: Because nearly all myeloma and amyloid patients relapse and treatment is eventually unsuccessful, our focus is to develop more effective treatments that not only prolong life, but cure the disease. Periodically the investigators will inform them about clinical trials studying new drugs or treatment paradigms.
The Norwegian Immunotherapy in Multiple Myeloma Study
The goal of this observational study is to study the effectiveness and complications of novel immunotherapies used in the treatment of multiple myeloma in routine care in Norway. The aim is to close knowledge gaps, generate evidence for future clinical trials and contribute to future consensus on how to monitor for adverse events, and what mitigation strategies should be implemented, so that we can increase patient survival and quality-of-life.