Session Type: ACR Late-breaking Abstract Session
Session Time: 4:30PM-6:00PM
Background/Purpose: Sprifermin, anovel recombinant human fibroblast growth factor‑18 is currentlyinvestigated as a potential disease-modifying osteoarthritis (OA) drug. Two-year primary data from a 5-year PhaseII trial (FORWARD) is presented.
Methods: Patients (pts) aged 40–85 years with symptomaticradiographic knee OA, KLG 2 or 3, and medial mJSW ≥2.5 mm in the targetknee were randomized (1:1:1:1:1) to receive 3 weekly intra-articular injectionswith double-blinded placebo (PBO) or sprifermin, administered in cycles every 6or 12 months (Fig 1). The primary endpoint was the change in total tibiofemoraljoint (TFJ) cartilage thickness from baseline (BL) to Year 2. The ITTpopulation (all randomized pts) was used for non-MRI endpoints; and the mITT(all ITT pts with BL and ≥1 post-treatment MRI up to Year 2) for MRIendpoints.
Results: The ITT population included 549 pts:median age 65 years, 69% women, 80% white, and 69% KLG2. Of these, 12.2% (sprifermin)and 19.4% (PBO) discontinued treatment within 2 years. The primary endpoint wasmet (Fig 2); there was a dose-dependent increase in total TFJ cartilagethickness, with significant differences for sprifermin 100µg q6 mo (Group 1)and 100µg q12 mo (Group 2) vs PBO (+0.03 vs -0.02 mm; p<0.001, and +0.02 vs ‑0.02mm; p<0.001, respectively). Furthermore, significant differences in changeof cartilage thickness were observed between sprifermin vs PBO in medial (Group1: +0.02 vs -0.03 mm; p=0.003) and lateral TFJ compartments (Group 1 and 2:both +0.04 vs -0.01 mm; p<0.001), and in central medial TFJ sub-regions (Group1: +0.054 vs -0.11; p<0.001). Changes in mJSW observed with X-ray weresignificantly different between sprifermin Group 1 and PBO in the lateral butnot the medial compartment. Total WOMAC scores improved by ~50% in all treatmentgroups including PBO. AEs and serious AEs were balanced between groups, and anoverall acceptable safety profile was observed.
Conclusion: To our knowledge, sprifermin is thefirst investigational agent to show prevention of cartilage loss in both thelateral and medial (including central medial) femorotibial compartments. Thesestructural benefits associated with sprifermin suggest that it may beefficacious for disease-modifying treatment of OA, and should be furtherevaluated in clinical trials.
To cite this abstract in AMA style:Hochberg MC, Guermazi A, Guehring H, Aydemir A, Wax S, Fleuranceau-Morel P, Bihlet AR, Byrjalsen I, Andersen JR, Eckstein F. Efficacy and Safety of Intra-Articular Sprifermin in Symptomatic Radiographic Knee Osteoarthritis: Results of the 2-Year Primary Analysis from a 5-Year Randomised, Placebo-Controlled, Phase II Study [abstract]. Arthritis Rheumatol. 2017; 69 (suppl 10). http://acrabstracts.org/abstract/efficacy-and-safety-of-intra-articular-sprifermin-in-symptomatic-radiographic-knee-osteoarthritis-results-of-the-2-year-primary-analysis-from-a-5-year-randomised-placebo-controlled-phase-ii-study/. Accessed November 18, 2017.
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ACR Meeting Abstracts - http://acrabstracts.org/abstract/efficacy-and-safety-of-intra-articular-sprifermin-in-symptomatic-radiographic-knee-osteoarthritis-results-of-the-2-year-primary-analysis-from-a-5-year-randomised-placebo-controlled-phase-ii-study/