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
The Italian Transthyretin Amyloidosis Web-Network
The study aims, by generating a large registry of patients with ATTR amyloidosis, including data at diagnosis and during follow up, to describe the natural history of ATTR amyloidosis in a real-world setting and to define and validate prognostic models, response criteria applicable at any point of the disease. The registry will also be used for data sharing and to allow the possibility of a close collaboration amongst the amyloidosis experts of the ARTC and all the physicians around the Country involved in the diagnosis and management of systemic amyloidosis. Thanks to the online registry, the diagnostic facility of the ARTC will be made available to requesting physicians.
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research. The purpose of this research is to: * Develop Clinical Guidelines for specific rare kidney diseases. These are written recommendations on how to diagnose and treat a medical condition. * Audit treatments and outcomes. An audit makes checks to see if what should be done is being done and asks if it could be done better. * Further the development of future treatments. Participants will be invited to participate on clinical trials and other studies. The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best (Home - Patients Know Best), allows patients to provide information themselves, including their own reported quality of life and outcome measures.