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
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.
Venetoclax-Dexamethasone in Relapsed and/or Refractory t(11;14) Amyloidosis
The purpose of this study is assess safety, safest dose, and effectiveness of venetoclax in combination with dexamethasone in participants with t(11;14) positive relapsed (comes back) or refractory (did not get better) light chain amyloidosis.