Indirect Comparison of Lanadelumab and Intravenous C1-INH Using Data from the HELP and CHANGE Studies: Bayesian and Frequentist Analyses
| العنوان: | Indirect Comparison of Lanadelumab and Intravenous C1-INH Using Data from the HELP and CHANGE Studies: Bayesian and Frequentist Analyses |
|---|---|
| المؤلفون: | Giovanna Devercelli, Mia Malmenäs, Joan Mendivil, Gagan Jain, Matthias Hunger, Katrin Haeussler |
| المصدر: | Drugs in R&D Drugs in R&D, Vol 21, Iss 1, Pp 113-121 (2021) |
| سنة النشر: | 2021 |
| مصطلحات موضوعية: | medicine.medical_specialty, Injections, Subcutaneous, Bayesian probability, Attack rate, Lanadelumab, Antibodies, Monoclonal, Humanized, 030226 pharmacology & pharmacy, Drug Administration Schedule, 03 medical and health sciences, 0302 clinical medicine, Pharmacotherapy, Frequentist inference, Internal medicine, medicine, Humans, Poisson Distribution, Original Research Article, Randomized Controlled Trials as Topic, 030203 arthritis & rheumatology, Pharmacology, Cross-Over Studies, Proportional hazards model, business.industry, lcsh:RM1-950, Angioedemas, Hereditary, Bayes Theorem, medicine.disease, lcsh:Therapeutics. Pharmacology, Treatment Outcome, Clinical Trials, Phase III as Topic, Hereditary angioedema, Administration, Intravenous, Kallikreins, business, Complement C1 Inhibitor Protein, Monte Carlo Method, Rare disease |
| الوصف: | Background Hereditary angioedema (HAE) with C1-esterase inhibitor (C1-INH) deficiency is a rare disease associated with painful, potentially fatal swelling episodes affecting subcutaneous or submucosal tissues. HAE attacks recur with unpredictable severity and frequency throughout patients’ lives; long-term prophylaxis is essential for some patients. In the absence of head-to-head studies, indirect treatment comparison (ITC) of long-term prophylactic agents is a valid approach to evaluate comparative efficacy. Methods We conducted an ITC using data from the placebo-controlled HELP study (assessing patients receiving lanadelumab 300 mg every 2 or 4 weeks) and the 12-week, parallel arm, crossover CHANGE study (assessing intravenous C1-INH). Outcomes of interest were attack rate ratio (ARR) and time to attack after day 0 (TTA0) and after day 70 (TTA70). Two ITC methodologies were used: a Bayesian approach using study results to update non-informative prior distributions to posterior distributions on relative treatment effects, and a frequentist approach using patient-level data from HELP and CHANGE to generate Poisson regressions (for ARR) and Cox models (for TTA0 and TT70). Results Both Bayesian and frequentist analyses suggested that lanadelumab reduced HAE attack rate by 46–73% versus intravenous C1-INH. Relative to intravenous C1-INH, risk of first attack after day 0 was comparable between intravenous C1-INH and both lanadelumab doses; risk of first attack after day 70 was reduced by 81–83% with lanadelumab 300 mg every 2 weeks, compared with C1-INH. Conclusions Findings from these two ITC methodologies support the favorable efficacy of lanadelumab in reducing the HAE attack rate and extending attack-free intervals in patients with HAE. Supplementary Information The online version contains supplementary material available at 10.1007/s40268-021-00337-4. |
| تدمد: | 1179-6901 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4cbb9dd6eea0a700e2f7005f2ebdb4bcTest https://pubmed.ncbi.nlm.nih.gov/33646565Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....4cbb9dd6eea0a700e2f7005f2ebdb4bc |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 11796901 |
|---|