This grant application requests support for a meeting of multidisciplinary clinicians and scientists focusing on two vascular anomalies associated with high morbidity and mortality, Kaposiform hemangioendothelioma (KHE) and multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT). KHE and tufted angioma are rare vascular tumors often associated with Kasabach Merritt Phenomenon (KMP), a form of consumptive coagulopathy. MLT is another rare vascular anomaly associated with coagulopathy and life-threatening bleeding events. Since both of these disorders involve multiple organs, the disorders inherently require an interdisciplinary approach to care and research. Clinical management of infants and children with these disorders is hampered by under-recognition of these lesions, ineffective therapies, and lack of communication between multidisciplinary care providers. Investigations by our group have already contributed essential fundamental knowledge about these two disorders so that the additional expertise provided by such a workshop will quickly provide translational activities that will improve clinical practice. The proposed workshop will bring together expert clinicians and investigators in the field of vascular anomalies in an effort to exchange institutional experience and foster the collective creation of innovative treatment protocols and algorithms for these rare patient populations. The overall goal of this workshop is to provide a forum for open and transparent communication between expert clinicians, clinician scientists, and basic science researchers with experience with KHE/KMP or MLT patients. Emergent from these discussions, we expect: (1) identification of institutional inconsistencies and knowledge gaps, (2) generation of clinical care guidelines, and (3) acceleration of research directives and scientific discoveries for children with KHE with and without KMP and MLT. The collective experience of multiple physicians in this area will provide uniform and agreed upon criteria for accurate diagnosis and best practices for treatment that will be disseminated to the entire scientific community through publications in medical journals.
The proposed conference will bring together expert physicians and researchers in the field of vascular anomalies from across the country to further the scientific community's understanding in two areas: (1) kaposiform hemangioendothelioma and tufted angioma with and without Kasabach Merritt Phenomenon and (2) multifocal lymphangioendotheliomatosis with thrombocytopenia. At the conclusion of this multidisciplinary workshop, doctors will have a better understanding of how to care for infants and children with these two diseases, researchers will be able to prioritize future areas of research, and all information will be shared with scientists worldwide through the publications of results in medical journals.
| Tlougan, Brook E; Lee, Margaret T; Drolet, Beth A et al. (2013) Medical management of tumors associated with Kasabach-Merritt phenomenon: an expert survey. J Pediatr Hematol Oncol 35:618-22 |
| Drolet, Beth A; Trenor 3rd, Cameron C; Brandão, Leonardo R et al. (2013) Consensus-derived practice standards plan for complicated Kaposiform hemangioendothelioma. J Pediatr 163:285-91 |
