Supplementary materials: Indirect treatment comparison of lanadelumab and a C1-esterase inhibitor in pediatric patients with hereditary angioedema

These are peer-reviewed supplementary materials for the article 'Indirect treatment comparison of lanadelumab and a C1-esterase inhibitor in pediatric patients with hereditary angioedema' published in the Journal of Comparative Effectiveness Research.Inverse probability weightingSupplementary Table 1: Eligibility criteria for the systematic literature reviewSupplementary Table 2: Baseline and prognostic factors of the SPRING and C1-INH studies as estimated per individual patient dataSupplementary Table 3: Baseline and prognostic factors considered for ITC analyses SPRING vs. Aygören Pürsün before and after IPTW Supplementary Table 4: Trial design of the SPRING and C1-INH 2019 studies used for indirect treatment comparison analysisSupplementary Figure 1: Flow chart for the identification and selection of published sources for this indirect treatment comparisonReferencesAim: To compare the efficacy and safety of lanadelumab versus other approved long-term prophylaxis (LTP) treatments in patients with pediatric hereditary angioedema (HAE) aged Materials & methods: A systematic literature review was conducted to identify studies of LTP in patients with HAE aged Results: Lanadelumab 150 mg every 2 weeks (Q2W) reduced the monthly HAE attack rate by 82.1% versus C1-INH(IV) 1000 IU twice weekly (every 3–4 days [BIW]; rate ratio [RR], 0.1792 [95% CI: 0.0296–1.0853]) and by 88.9% versus C1-INH(IV) 500 IU BIW (RR: 0.1107 [95% CI: 0.0234–0.5239]). Treatment with lanadelumab Q2W reduced the risk of total adverse events by 56.2% versus C1-INH(IV) 1000 IU BIW (RR:0.4377 [95% CI: 0.1536–1.2469]) and by 66.0% versus C1-INH(IV) 500 IU BIW (RR: 0.3401 [95% CI: 0.1234–0.9371]). Conclusion: This exploratory analysis suggested a trend toward greater efficacy and fewer adverse events with lanadelumab 150 mg Q2W compared with C1-INH(IV) BIW 1000 IU and 500 IU in pediatric patients with HAE. Future studies could potentially assess larger samples over longer perio