Eulalia Baselga1, Bozenna Dembowska-Baginska2, Przemysław Przewratil3, María Antonia González-Enseñat4, Dariusz Wyrzykowski5, Antonio Torrelo6, Juan-Carlos López Gutiérrez7, Magdalena Rychłowska-Pruszyńska8, Raúl de Lucas-Laguna9, Altea Esteve-Martinez10, Esther Roé11, Mohammed Zaim12, Yoann Menon12, Stéphanie Gautier12, Geneviève Lebbé13, Athmane Bouroubi12, Alain Delarue14, Jean-Jacques Voisard14. 1. Department of Dermatology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain; ebaselga@santpau.cat. 2. Department of Pediatric Oncology, Instytut Pomnik-Centrum Zdrowia Dziecka, Warsaw, Poland. 3. Department of Pediatric Oncology and Surgery, Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny, Uniwersytetu Medycznego w Łodzi, Łódź, Poland. 4. Dermatology Service, Hospital Sant Joan de Deu, Esplugues de Llobregat, Barcelona, Spain. 5. Department of Surgery and Urology for Children and Adolescents, Copernicus Podmiot Leczniczy Sp. z o.o, Gdańsk, Poland. 6. Department of Dermatology, Hospital Infantil Universitario Niño Jesús, Madrid, Spain. 7. Departments of Pediatric Surgery and. 8. Department of Surgical Oncology for Children and Youth, Instytut Matki i Dziecka, Warsaw, Poland. 9. Dermatology, Hospital Infantil La Paz, Madrid, Spain. 10. Polyclinic Dermatology, Hospital General Universitario de Valencia, Valencia, Spain. 11. Department of Dermatology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain. 12. Institut de Recherche Pierre Fabre, Toulouse, France. 13. Pierre Fabre Médicament, Boulogne-Billancourt, France; and. 14. Pierre Fabre Dermatologie, Lavaur, France.
Abstract
BACKGROUND AND OBJECTIVES: There is no consensus on optimal treatment duration for propranolol in infantile hemangioma (IH). We evaluated the efficacy and safety of oral propranolol solution administered for a minimum of 6 months up to a maximum of 12 months of age in high-risk IH. METHODS: This single-arm, open-label, phase 3 study was conducted in patients aged 35 to 150 days with high-risk IH in 10 hospitals between 2015 and 2017. The study comprised a 6-month initial treatment period (ITP) plus continuation up to 12 months of age if complete success was not achieved, a follow-up, and a retreatment period. Patients received oral propranolol twice daily (3 mg/kg per day). The primary end point was the success rate at the end of the ITP. Furthermore, the persistence of IH response and efficacy of retreatment was evaluated. RESULTS: The success rate after 6 months of treatment was 47%, increasing to 76% at the end of the ITP. Of the patients who achieved success, 68% sustained success for 3 months without treatment, and 24% required retreatment. Of the 8 patients who were retreated, 7 achieved success. Adverse events, reported by 80% of patients, were mild, which were expected in this population or known propranolol side effects. CONCLUSIONS: Oral propranolol administered beyond 6 months and up to 12 months of age meaningfully increases the success rate in high-risk IH. Success was sustained in most patients up to 3 months after stopping treatment. Retreatment was efficacious, and the safety profile satisfactory.
BACKGROUND AND OBJECTIVES: There is no consensus on optimal treatment duration for propranolol in infantile hemangioma (IH). We evaluated the efficacy and safety of oral propranolol solution administered for a minimum of 6 months up to a maximum of 12 months of age in high-risk IH. METHODS: This single-arm, open-label, phase 3 study was conducted in patients aged 35 to 150 days with high-risk IH in 10 hospitals between 2015 and 2017. The study comprised a 6-month initial treatment period (ITP) plus continuation up to 12 months of age if complete success was not achieved, a follow-up, and a retreatment period. Patients received oral propranolol twice daily (3 mg/kg per day). The primary end point was the success rate at the end of the ITP. Furthermore, the persistence of IH response and efficacy of retreatment was evaluated. RESULTS: The success rate after 6 months of treatment was 47%, increasing to 76% at the end of the ITP. Of the patients who achieved success, 68% sustained success for 3 months without treatment, and 24% required retreatment. Of the 8 patients who were retreated, 7 achieved success. Adverse events, reported by 80% of patients, were mild, which were expected in this population or known propranolol side effects. CONCLUSIONS: Oral propranolol administered beyond 6 months and up to 12 months of age meaningfully increases the success rate in high-risk IH. Success was sustained in most patients up to 3 months after stopping treatment. Retreatment was efficacious, and the safety profile satisfactory.
Authors: Virginia Albiñana; Eunate Gallardo-Vara; Juan Casado-Vela; Lucía Recio-Poveda; Luisa María Botella; Angel M Cuesta Journal: J Clin Med Date: 2022-08-04 Impact factor: 4.964