Tsuyoshi Kaneko1, Satoru Sasaki2, Naoko Baba3, Katsuyoshi Koh4, Kiyoshi Matsui5, Hiroyuki Ohjimi6, Nobukazu Hayashi7, Atsuko Nakano8, Kentaro Ohki9, Yoshihiro Kuwano10, Akira Morimoto11, Zenshiro Tamaki12, Mariko Kakazu13, Kazuo Kishi14, Tomoki Oyama15, Atsushi Sato16, Rumiko Kato17, Takeshi Higuchi17. 1. Department of Surgical Specialties, National Center for Child Health and Development, Tokyo, Japan. 2. Center for Vascular Anomalies, KKR Tonan Hospital, Hokkaido, Japan. 3. Department of Dermatology, Kanagawa Children's Medical Center, Kanagawa, Japan. 4. Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan. 5. Department of General Medicine, Kanagawa Children's Medical Center, Kanagawa, Japan. 6. Department of Plastic and Reconstructive Surgery, School of Medicine, Fukuoka University, Fukuoka, Japan. 7. Department of Dermatology, Toranomon Hospital, Tokyo, Japan. 8. Division of Otorhinolaryngology, Chiba Children's Hospital, Chiba, Japan. 9. Department of Hematology and Oncology, Gunma Children's Medical Center, Gunma, Japan. 10. Department of Dermatology, Showa General Hospital, Tokyo, Japan. 11. Department of Pediatrics, Jichi Medical University School of Medicine, Tochigi, Japan. 12. Department of Dermatology, University of Tokyo Graduate School of Medicine, Tokyo, Japan. 13. Department of Hematology and Oncology, Okinawa Prefectural Nanbu Medical Center and Children's Medical Center, Okinawa, Japan. 14. Department of Plastic and Reconstructive Surgery, Keio University School of Medicine, Tokyo, Japan. 15. Department of Plastic Surgery, Hyogo Prefectural Kobe Children's Hospital, Hyogo, Japan. 16. Department of Hematology and Oncology, Miyagi Children's Hospital, Miyagi, Japan. 17. Maruho, Kyoto, Japan.
Abstract
BACKGROUND: There have been few reports on the efficacy and safety of oral propranolol at 3 mg/kg/day for infantile hemangioma (IH) in Japanese patients. METHODS: A multicenter, open-label phase III study was conducted to evaluate the efficacy and safety of oral propranolol solution in Japanese infants aged 35-150 days with proliferating IH. Thirty-two patients were enrolled in the study, received propranolol solution for 24 weeks at 3 mg/kg/day, and completed the study. RESULTS: The success rate (complete or nearly complete resolution) at week 24 (primary endpoint) was 78% (95%CI: 60-91%). The improvement rate since the previous visit was 100% (32/32) after week 5. Overall, the IH surface area, maximum diameter, and color intensity all decreased over time. Consistency in assessment between the centralized and the investigator on-site assessments was observed in 26 patients. Of the 32 patients, 11 needed further treatment other than the study drug. The incidence of adverse events (AE) and drug-related AE was 97% and 31%, respectively. AE that occurred in ≥two patients were either typical of propranolol use (such as blood pressure decrease) or common events in infants. AE that resulted in dose reduction were observed in two patients, but no serious AE or AE that led to study drug discontinuation were observed. CONCLUSION: Oral propranolol solution at 3 mg/kg/day is effective and safe in Japanese IH patients.
BACKGROUND: There have been few reports on the efficacy and safety of oral propranolol at 3 mg/kg/day for infantile hemangioma (IH) in Japanese patients. METHODS: A multicenter, open-label phase III study was conducted to evaluate the efficacy and safety of oral propranolol solution in Japanese infants aged 35-150 days with proliferating IH. Thirty-two patients were enrolled in the study, received propranolol solution for 24 weeks at 3 mg/kg/day, and completed the study. RESULTS: The success rate (complete or nearly complete resolution) at week 24 (primary endpoint) was 78% (95%CI: 60-91%). The improvement rate since the previous visit was 100% (32/32) after week 5. Overall, the IH surface area, maximum diameter, and color intensity all decreased over time. Consistency in assessment between the centralized and the investigator on-site assessments was observed in 26 patients. Of the 32 patients, 11 needed further treatment other than the study drug. The incidence of adverse events (AE) and drug-related AE was 97% and 31%, respectively. AE that occurred in ≥two patients were either typical of propranolol use (such as blood pressure decrease) or common events in infants. AE that resulted in dose reduction were observed in two patients, but no serious AE or AE that led to study drug discontinuation were observed. CONCLUSION: Oral propranolol solution at 3 mg/kg/day is effective and safe in Japanese IH patients.