A phase 2 trial of daratumumab monotherapy for patients with newly diagnosed cardiac stage IIIb AL amyloidosis.

Daratumumab monotherapy achieves a 75% hematologic response rate and a 65% 6-month overall survival in patients with stage IIIB AL amyloidosis.

• Why it matters: Patients with advanced cardiac AL amyloidosis have limited treatment options and poor prognosis, highlighting the need for effective therapies that improve survival and quality of life.
• What they did: A phase 2 trial enrolled 40 untreated patients with stage IIIB AL amyloidosis, administering daratumumab for up to 2 years, with some receiving additional bortezomib and dexamethasone if response was inadequate.
• The result: The treatment demonstrated rapid hematologic responses, a median overall survival of 10.4 months, and a 30% cardiac response rate at 6 months, suggesting daratumumab's feasibility and potential benefit in this high-risk population.