Dados do Trabalho

Título

Human Plasma-Derived C1 Inhibitor (C1-INH) for Short-Term Prophylaxis in Hereditary Angioedema with normal C1-INH

Resumo

RATIONALE: Hereditary Angioedema (HAE) is a disease with autosomal dominant inheritance, associated with C1 inhibitor (C1-INH) deficiency and with normal C1-INH. The accumulation of bradykinin is responsible for mucosal and submucosal edema, mainly affecting the subcutaneous tissue, gastrointestinal tract, and upper airways, pathomechanism well-defined for HAE-C1-INH, however uncertain for HAE-nC1INH. The most frequently reported triggers are stress, trauma, infection, and dental, surgical or endoscopy procedures. Human plasma-derived C1-INH (pdC1-INH) is indicated for short-term prophylaxis (STP) in HAE-C1INH, however not defined for HAE-nC1-INH. This study aims to evaluate the STP in this population. METHOD: This is a multicenter, observational, retrospective study, in patients over 12 years old, with a confirmed diagnosis of HAE who underwent high-risk procedures after receiving pdC1-INH as STP. Data collection was carried out using a questionnaire with personal information, diagnosis, and medication in use. The patients were divided into three groups:: G1 (HAE-C1-INH), G2 (HAE-FXII), and G3 (HAE-UNK). RESULTS: Seventy-three infusions were evaluated in 44 patients (70F:3M, median age: 39 years (13- 67y): G1) HAE-C1-INH (37 infusions), G2) HAE-FXII (22 infusions) and G3) HAE-UNK (14 infusions). The indications for STP were: dental procedure (G1=12; G2=3; G3=2); diagnostic procedure (G1=8; G2=5; G3=5); elective surgery (G1=9; G2=5; G3=6); cesarean section (G1=5; G2=7; G3=1); normal birth (G1=2; G2=2; G3=0); others (G=1). Edema after the procedure occurred in 4/37 (10,8%) for G1, 2/22 (9%) for G2, and 3/14 (21,4%) for G3. DISCUSSION: The study demonstrated the effectiveness of pdC1-INH for STP, however, less successful for HAE-UNK in comparison to to HAE-FXII and HAE-C1INH. The occurrence of edema after the procedures was not dose-dependent. This is the first study of STP in patients with HAE-FXII and HAE-UNK.