RESUMO
The economic trend and the health care landscape are rapidly evolving across Asia. Effective real-world data (RWD) for regulatory and clinical decision-making is a crucial milestone associated with this evolution. This necessitates a critical evaluation of RWD generation within distinct nations for the use of various RWD warehouses in the generation of real-world evidence (RWE). In this article, we outline the RWD generation trends for 2 contrasting nation archetypes: "Solo Scholars"-nations with relatively self-sufficient RWD research systems-and "Global Collaborators"-countries largely reliant on international infrastructures for RWD generation. The key trends and patterns in RWD generation, country-specific insights into the predominant databases used in each country to produce RWE, and insights into the broader landscape of RWD database use across these countries are discussed. Conclusively, the data point out the heterogeneous nature of RWD generation practices across 10 different Asian nations and advocate for strategic enhancements in data harmonization. The evidence highlights the imperative for improved database integration and the establishment of standardized protocols and infrastructure for leveraging electronic medical records (EMR) in streamlining RWD acquisition. The clinical data analysis and reporting system of Hong Kong is an excellent example of a successful EMR system that showcases the capacity of integrated robust EMR platforms to consolidate and produce diverse RWE. This, in turn, can potentially reduce the necessity for reliance on numerous condition-specific local and global registries or limited and largely unavailable medical insurance or claims databases in most Asian nations. Linking health technology assessment processes with open data initiatives such as the Observational Medical Outcomes Partnership Common Data Model and the Observational Health Data Sciences and Informatics could enable the leveraging of global data resources to inform local decision-making. Advancing such initiatives is crucial for reinforcing health care frameworks in resource-limited settings and advancing toward cohesive, evidence-driven health care policy and improved patient outcomes in the region.
RESUMO
OBJECTIVE: To conduct a prospective cohort study to clarify the relationship between human leukocyte antigen (HLA) polymorphisms and the seroconversion of hepatitis B e antigen (HBeAg). STUDY DESIGN: In the prospective cohort study, 81 HBeAg-positive children with chronic hepatitis B virus (HBV) infection from 40 unrelated families were recruited and followed-up regularly for a mean period of 17.70 +/- 3.23 years. The association between HLA antigen and the age at HBeAg seroconversion was analyzed using Cox regression model with shared frailties under left truncation and right censorship. RESULTS: HLA-B61 and HLA-DQB1*0503 antigens predicted a higher HBeAg seroconversion rate (relative incidence = 6.17 and 3.22, P = .024 and .017, respectively). Within-family frailty in our sibling cohort study demonstrated a negligible or a low degree of within-family correlation with spontaneous HBeAg seroconversion in each HLA antigen. CONCLUSIONS: HLA class I antigen B61 and class II antigen DQB1*0503 are associated with earlier HBeAg seroconversion in Taiwanese children with chronic HBV infection.
Assuntos
Antígenos HLA-DQ/genética , Antígenos HLA-DR/genética , Antígenos E da Hepatite B/sangue , Hepatite B Crônica/sangue , Hepatite B Crônica/genética , Antígenos de Histocompatibilidade Classe I/sangue , Adolescente , Criança , Pré-Escolar , Estudos de Coortes , Feminino , Teste de Histocompatibilidade , Humanos , Lactente , Recém-Nascido , Masculino , Polimorfismo Genético/genética , Irmãos , TaiwanRESUMO
To elucidate the frequency of FIC1 (ATP8B1) and BSEP (ABCB11) mutations in Taiwanese children with chronic intrahepatic cholestasis with low gamma-glutamyltranspeptidase (GGT) levels, we assessed 13 unrelated patients with infantile onset chronic intrahepatic cholestasis. Liver complementary DNA sequencing was performed in 7 infants for mutation analyses of FIC1 and BSEP genes. Two distinct liver histologic features were found. Group 1 (n = 5) was characterized by bland cholestasis and group 2 (n = 8) by giant cell transformation. Group 2 patients were associated with higher transaminase levels, alpha-fetoprotein levels, and early mortality. Novel FIC1 mutations were found in all 4 patients tested in group 1, including a 74-bp deletion, a 98-bp deletion, a nonsense, and 2 missense mutations. BSEP mutations were found in 2 of the 3 patients in group 2, including 2 missense mutations and a 1-bp deletion. Phenotypic characterization is useful to differentiate FIC1- from BSEP-related disease.