Infantile hemangiomas are benign tumors composed of blood vessel cells that grow in the first months of life. Most infantile hemangiomas are found in the skin, but sometimes they occur in other organs in the body such as the liver, spleen, intestine, airway, lungs and the central nervous system. While most are benign in their behavior, some may interfere with vital functions and leave permanent scarring. Severe hemangiomas require treatment to control the growth of this tumor. Rapid growth may lead to complications such as ulceration, bleeding, loss of vision, airway obstruction, congestive heart failure, and permanent disfigurement. We lack uniform guidelines for treatment and methods to measure response to treatment of infants with hemangiomas. The objective of this R34 proposal is to establish a planning process and develop novel interdisciplinary strategies to overcome existing barriers to the implementation of a multi-institutional clinical trial. The overall goal of the future clinical trial is to determine the safety and efficacy of combination therapy of prednisolone plus propranolol compared to propranolol alone for complicated infantile hemangiomas. The proposed R34 planning project builds upon the success of the Hemangioma Investigator Group by incorporating complementary expertise to comprehensively construct the study design and protocol, and develop tools for data management, methods of research oversight, and other essential elements of the study including a manual of operations/procedures. The proposed research will rapidly advance the field by facilitating an investigator-initiated, randomized-controlled clinical trial targeted at providing evidence-based care for the critical clinical issues related to treatment of infants with complicated hemangiomas.
Hemangiomas are benign tumors composed of blood vessel cells that grow in the first months of life, and while most are uncomplicated, some may interfere with vital functions and leave permanent scarring. Severe hemangiomas require treatment to control the growth of this tumor, but physicians lack uniform guidelines for the treatment of infants with hemangiomas. The proposed research will quickly advance the field by providing the necessary data and expertise to implement research to evaluate the efficacy of new medications in the treatment of infants with complicated hemangiomas.
| Drolet, Beth A; Frommelt, Peter C; Chamlin, Sarah L et al. (2013) Initiation and use of propranolol for infantile hemangioma: report of a consensus conference. Pediatrics 131:128-40 |
| Metry, Denise; Frieden, Ilona J; Hess, Christopher et al. (2013) Propranolol use in PHACE syndrome with cervical and intracranial arterial anomalies: collective experience in 32 infants. Pediatr Dermatol 30:71-89 |
| Kwon, Eun-Kyung Mary; Seefeldt, Marcia; Drolet, Beth A (2013) Infantile hemangiomas: an update. Am J Clin Dermatol 14:111-23 |
| Block, Robert W; Dreyer, Benard P; Cohen, Alan R et al. (2013) An agenda for children for the 113th Congress: recommendations from the pediatric academic societies. Pediatrics 131:109-19 |
| Martin, Kari; Blei, Francine; Bleib, Francine et al. (2013) Propranolol treatment of infantile hemangiomas: anticipatory guidance for parents and caretakers. Pediatr Dermatol 30:155-9 |
| Kwon, Eun-Kyung M; Joachim, Shawna; Siegel, Dawn H et al. (2013) Retrospective review of adverse effects from propranolol in infants. JAMA Dermatol 149:484-5 |
