OBJECTIVES

Patients with atypical hemolytic uremic syndrome (aHUS) may require ongoing treatment of varying duration. The C5 complement inhibitor eculizumab provides transformative benefits in aHUS; however, knowledge gaps remain regarding treatment patterns/preferences. To address this, we sought to characterize real-world eculizumab use, disease triggers, dialysis and plasma exchange/infusion (PE/PI) in patients with aHUS using US claims data.

METHODS

Eculizumab-treated patients with aHUS were identified from the Optum Clinformatics^® Data Mart database from 1-Jan-2007 to 31-Mar-2019, during which eculizumab was the only available therapy. Because no ICD-9 code exists for aHUS, inclusion and exclusion criteria reflected those used in eculizumab clinical trials. The index date represents date of first eculizumab claim. aHUS triggers (conditions for which ≥1 claim was made 0–3 months before diagnosis) were identified and post-index claims for dialysis, PE/PI, and eculizumab dosage data were analyzed.

RESULTS

Overall, 86 patients (65% female, mean age 48 years) were identified, with 55 (64%) and 44 (51%) patients still enrolled at 12 and 18 months post-index, respectively. Continuously treated patients had a mean of ~2 eculizumab claims/month after the first month of treatment. Patients had claims for the following potential aHUS triggers: infections (20%), chemotherapy (67%), lupus (9%), and bone marrow transplant (5%). At 12 months post-index, 51% of patients (28/55) were receiving ongoing eculizumab treatment, of which 14% and 61% reported potential triggers of infection and chemotherapy, respectively. Median number of dialysis claims were 5.0, 3.0, 2.0, and 1.5 at 1, 6, 12, and 18 months post-index, respectively. Over the 18-month post-index period, median eculizumab dosage was 1200 mg, and two patients reported PE/PI claims.

CONCLUSIONS

These data illustrate the heterogeneity of treatment patterns, disease triggers, and use of supportive therapies in eculizumab-treated patients with aHUS, providing additional evidence that eculizumab decreases the need for dialysis.

Sponsor: Alexion Pharmaceuticals, Inc.