ABSTRACT
Acquired thrombotic thrombocytopenic purpura (aTTP) is a thrombotic microangiopathy resulting in high mortality. Caplacizumab is approved for treatment of adults with acquired thrombotic thrombocytopenic purpura that has shown faster platelet normalization, clinical improvement, and reduced risk of recurrent/refractory disease. We report 2 cases of adolescents treated off-label with caplacizumab who were able to stop before 30 days from end of plasma exchange after platelets normalized and ADAMTS13 activity recovered to >20% to 30%. Our results show similar efficacy to other reports of patients under 18 receiving caplacizumab in first line, regardless of plasma exchange strategy, and may offer insight into early cessation criteria.
Subject(s)
Purpura, Thrombotic Thrombocytopenic , Single-Domain Antibodies , Adult , Humans , Adolescent , Purpura, Thrombotic Thrombocytopenic/therapy , von Willebrand Factor , Single-Domain Antibodies/therapeutic use , Plasma Exchange , ADAMTS13 ProteinSubject(s)
Adrenergic beta-Antagonists/therapeutic use , Eyelid Neoplasms/drug therapy , Hemangioma/drug therapy , Propranolol/therapeutic use , Adrenergic beta-Antagonists/adverse effects , Dose-Response Relationship, Drug , Female , Humans , Infant , Infant, Newborn , Male , Propranolol/adverse effects , Treatment OutcomeABSTRACT
BACKGROUND: School tobacco control policies vary widely in their strength, extensiveness, and enforcement. Currently, no standardized method exists to assess the comprehensiveness of school tobacco policies. The purpose of this study was to develop a new practical rating system for school tobacco policies, assess its reliability, and present preliminary validation data. METHODS: This study presents the systematic development of a rating system to assess the strength of school tobacco policies. Based on the empirical literature and the expertise of an advisory panel consisting of educational leaders and tobacco control advocates and practitioners, a "gold standard" school tobacco policy was developed and guided the content of the 40-point rating system. The 4 domains of the School Tobacco Policy Index were: Tobacco-free environment (14 points), Enforcement (12 points), Prevention and treatment services (6 points), and Policy organization (8 points). RESULTS: The Index was pilot-tested using 95 Missouri public school district tobacco policies and proved to be highly reliable among coders. The evaluated policies varied greatly between school districts, with the lowest total policy score of phi and the highest score of 21. School district policy scores were significantly related to a number of county-level tobacco policy characteristics, including support for a tobacco excise tax increase. CONCLUSIONS: The Index is a user-friendly, practical tool for tobacco control professionals and educators, providing them with the ability to easily evaluate their own school policies. Their evaluation efforts will be useful in strengthening existing policies and developing new comprehensive policies to protect the health of students, staff, administrators, and visitors.