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Emicizumab may yield clinically relevant improvements in joint health in patients with hemophilia, according to results presented at the 61st American Society of Hematology (ASH) Annual Meeting in Orlando, Florida.
Previous results from the phase 3 Haven 3 trial (ClinicalTrials.gov Identifier: NCT02847637) indicate that emicizumab has a favorable safety profile and has led to reductions in joint bleeds. In this analysis, researchers examined the effect of emicizumab prophylaxis on bone and joint health.
Joint health was assessed using the hemophilia joint health score (HJHS) as a measure, where lower scores indicate better joint health; 107 patients with hemophilia A were evaluated at baseline and at week 49 of prophylaxis. In addition, multiple biomarkers of bone health were evaluated in 117 patients with hemophilia A at baseline and after 3, 6, 12, and 18 months of prophylaxis. Of these patients, 78 were also included in the HJHS assessment.
At baseline, patients who had previously received factor VIII prophylaxis and who had no target joints reported lower HJHS scores. In patients who had 1 or more target joints, total HJHS decreased a median of 2.25 points from baseline to week 49.
Levels of most bone and joint biomarkers at baseline were similar to published levels in healthy individuals, though there was significant variability. Baseline biomarker levels were similar between patients receiving factor VIII prophylaxis and patients receiving on-demand treatment, as well as between patients with or without target joints. No significant differences were observed during the study period. However, a potential association between cartilage oligomeric matrix protein and baseline HJHS was seen (Pearson correlation coefficient, 0.46; P =.0001). “Based on further investigations, this correlation may be substantially driven by age,” noted Anna Kiialainen, PhD, of F Hoffmann-La Roche Ltd in Switzerland, in her presentation.
“Clinically relevant improvements in HJHS were observed for HAVEN 3 participants with target joints after as little as 48 weeks of emicizumab,” Dr Kiialainen concluded. “Additional data are needed to better understand the long-term effect of emicizumab prophylaxis on bone and joint health.”
Disclosures: Some authors have declared affiliations with the pharmaceutical industry, including employment. Please refer to the original study for a full list of disclosures.
1. Kiialainen A, Miggli M, Kempton CL, et al. Bone and joint health markers in persons with hemophilia A (PwHA) treated with emicizumab in HAVEN 3. Oral presentation at: 61st ASH Annual Meeting and Exposition; December 7-10, 2019; Orlando, FL. Abstract 626.