Real-world safety and efficacy of lomitapide in homozygous familial hypercholesterolemia: interim report of special-use survey in Japan.

Aim: To evaluate the safety and efficacy of lomitapide in real-world clinical practice in Japan. Patients & methods: Interim analysis of 39 patients with homozygous familial hypercholesterolemia from an all-case surveillance study. Results: Median lomitapide dose (across 42 months) was 9.8 mg/day. 74 drug-related adverse events (AEs) were reported in 24 (61.5%) patients, including 14 (35.9%) with liver-related AEs, 19 (48.7%) with gastrointestinal disorders and 1 (2.6%) bleeding disorder. Lomitapide dose was reduced for 39.2% of drug-related AEs, withdrawn temporarily for 12.2%, and discontinued for 1 event (1.4%). Mean ± SD blood LDL-C level decreased from 225.9 ± 172.0 mg/dl (5.8 ± 4.5 mmol/l) predose to 159.4 ± 93.0 mg/dl (4.1 ± 2.4 mmol/l) at 12 months (p = 0.0245). Conclusion: This interim analysis suggests lomitapide is safe and effective in real-world clinical practice in Japan.

What is this article about? Lomitapide is a drug used to treat homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder that causes very high cholesterol levels. Because HoFH is rare, only a limited number of patients were enrolled into the clinical trials that showed it was safe and effective, before its approval. Therefore, a study is now underway to evaluate the efficacy and safety of lomitapide when it is used in daily clinical practice in Japan. We have analyzed data for 39 patients who have been enrolled in this study so far. What are the results? We found that although most patients experienced some side effects, only one patient had to discontinue lomitapide. Most side effects could be managed without having to alter lomitapide treatment, or in some cases by reducing the dose or stopping the drug temporarily. We also found that lomitapide reduced cholesterol levels. What do the results mean? The results suggest that lomitapide is generally safe and effective in patients with HoFH being treated in routine clinical practice. The study is ongoing and additional analyses will be performed when a greater number of patients have been treated.

Safety and effectiveness of adalimumab in Japanese rheumatoid arthritis patients: postmarketing surveillance report of 7740 patients.

OBJECTIVES: To confirm the safety and effectiveness of adalimumab and to evaluate the influence of the concomitant use of methotrexate (MTX). METHODS: Postmarketing surveillance of 7740 Japanese rheumatoid arthritis (RA) patients was performed. All patients who received adalimumab in the registration period were followed for 28 weeks after starting treatment for safety and 24 weeks for effectiveness. Effectiveness was measured by duration of morning stiffness, swollen and tender joint counts (28 joints), patient global assessment of disease activity, erythrocyte sedimentation rate and serum C-reactive protein. RESULTS: Comparable rates of adverse drug reactions (ADRs) were reported in this study and in the interim analysis. Age, pulmonary disease history or comorbidity, co-existing diabetes mellitus, concomitant MTX at doses of > 8 mg/week and concomitant glucocorticoids at doses of > 5 mg/day were risk factors for infections. All mean values of effectiveness measurements improved. Relatively lower disease activity at baseline, biologic-naïve, concomitant MTX use and early RA stage/low functional class were background factors contributing to the effectiveness. The combination of adalimumab with MTX improved the response to adalimumab treatment. CONCLUSION: Adalimumab, especially with concomitant use of MTX, provided significant improvement in disease activity, without any unexpected ADRs in Japanese RA patients.