Study
| Phase 3, open-label, multicenter, randomized trial (AQUILA) |
| Patients with high-risk smoldering multiple myeloma without end-organ damage |
| Daratumumab subcutaneous monotherapy vs. active monitoring |
Efficacy
| PFS @ 5 yrs: 63.1% vs. 40.8% (HR: 0.49 [0.36–0.67]) |
| OS @ 5 yrs: 93.0% vs. 86.9% (HR: 0.52 [0.27–0.98]) |
| CR or better: 8.8% vs. 0% |
| VGPR or better: 29.9% vs. 1.0% |
| First-line MM treatment initiation @ 5yrs: 29.7% vs. 55.9% (HR: 0.46 [0.33–0.62]) |
Safety
| Grade ≥3 AEs: 40.4% vs. 30.1% |
| Hypertension (5.7% vs. 4.6%), infections (16.1% vs. 4.6%), pneumonia (3.6% vs. 0.5%) |
| Treatment discontinuation due to AEs: 5.7% vs. 0% |
| Deaths due to AEs: 1.0% vs. 2.0% |
N Engl J Med 2025;392:1777-88
http://doi.org/10.1056/NEJMoa2409029
Reviewed by Ulas D. Bayraktar, MD on Jun 2, 2025
