First disease-modifying therapy for knee osteoarthritis

  • Hochberg MC & al.
  • JAMA
  • 8 Oct 2019

  • International Clinical Digest
Access to the full content of this site is available only to registered healthcare professionals. Access to the full content of this site is available only to registered healthcare professionals.

Takeaway

  • Intra-articular injections of sprifermin, a recombinant human fibroblast growth factor, increases femorotibial joint cartilage thickness in knee osteoarthritis (KOA) at the highest doses, but leaves pain, stiffness, and function unchanged.

Why this matters

  • If more evidence of benefit unfolds, sprifermin could become the first disease-modifying therapy for OA.

Study design

  • 5-year, dose-finding, multicentre phase 2 trial of 549 patients aged 40-85 years with symptomatic, radiographic KOA of Kellgren-Lawrence grade 2/3.
  • Participants were randomly assigned to 1 of 5 treatment groups:
    • 100 μg sprifermin every 6 or 12 months.
    • 30 μg sprifermin every 6 or 12 months.
    • Placebo every 6 months.
  • Each treatment episode consisted of weekly intra-articular injections over 3 weeks.
  • Primary outcome: change in total femorotibial joint cartilage thickness assessed by quantitative MRI at 2 years.
  • Funding: Merck KGaA.

Key results

  • Groups 1 and 2 were the only ones to show significant improvement in femorotibial joint cartilage thickness over placebo (0.05 mm [95% CI, 0.03-0.07 mm] and 0.04 mm [95% CI, 0.02-0.06 mm], respectively).
  • None of the groups showed improvement over placebo in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) total scores, as well as WOMAC subscores for pain, stiffness, and function.

Limitations

  • Durability of results unknown.