Luanda

Researchers are evaluating the safety and effects of starting daily low dose aspirin (100 mg) in pregnant women with Sickle Cell Disease. The study compares beginning aspirin use in the first trimester (6-13 weeks) versus the second trimester (14-27 weeks) of pregnancy. This prospective, randomized controlled study includes 450 female participants in multiple maternity hospitals in Luanda, Angola, aiming to understand how timing of aspirin use impacts pregnancy outcomes in this group. Participants will be randomly assigned in equal groups to start daily low dose aspirin either in the first or second trimester. The aspirin treatment continues daily until week 36 of pregnancy or until delivery, whichever happens first. The study is designed to closely monitor and compare the effects of aspirin use started at these two different pregnancy stages. Each participant will be enrolled throughout their pregnancy, including a screening visit, the randomized treatment period, and a follow-up period lasting six weeks after delivery. Researchers will assess key outcomes such as maternal mortality, live birth events, and late abortion events over a two-year period. The study includes regular consultations and monitoring to track safety and health outcomes for both mother and baby.

Sickle cell anemia (SCA) is a common and serious blood disorder affecting over 300,000 newborns annually, especially in sub-Saharan Africa where many children die before age 5 due to limited diagnosis and care. This trial evaluates the use of hydroxyurea, a once-daily oral medication that is the standard treatment for children with SCA in wealthier countries. The study aims to bring a precision medicine approach to Africa, focusing on safe and effective hydroxyurea dosing with minimal laboratory monitoring. The trial involves 400 children aged 6 months to 12 years with SCA in Luanda, Angola. It tests individualized hydroxyurea dosing guided by pharmacokinetics using a battery-powered machine and an automated algorithm. The goal is to find safe and effective dosing strategies that require less frequent lab tests, making hydroxyurea more accessible in resource-limited settings. Hydroxyurea treatment will be closely monitored for safety and efficacy over approximately 24 months. Participants will be assessed for sickle cell-related adverse events and other health issues throughout the study. Researchers will track clinical outcomes, laboratory safety, and dosing effectiveness. The study includes careful monitoring of blood counts and drug levels, with the aim of establishing evidence-based guidelines for hydroxyurea use in sub-Saharan Africa, ultimately supporting wider access and safer treatment for children with SCA.

Researchers are investigating how genetic modifiers influence hemoglobinopathies, including sickle cell disease and beta-thalassemia. These diseases vary widely in severity, and while some genetic factors have been identified, more are believed to exist that impact disease outcomes. This large-scale, multi-ethnic genome-wide association study (GWAS) aims to discover new genetic modifiers, validate known ones, pool existing genetic data, standardize disease descriptions, and develop risk scores to better classify patients. The study will perform GWAS using SNP chips on blood samples collected during routine clinical visits or existing biobank DNA samples. Participants with various hemoglobinopathy genotypes will be included without restrictions on gender or ethnicity. The research will analyze genetic factors related to survival, complications like stroke, renal impairment, pain syndromes, and responses to treatments such as hydroxyurea and iron chelation. Data collected will contribute to a comprehensive research resource combining genomic, phenotypic, and functional information. Participants will provide consent and contribute blood samples if DNA is not already available. Researchers will gather worldwide demographic and clinical data from multiple centers. The primary outcome measured over five years is the identification of genetic modifiers influencing disease traits and treatment responses. This extensive monitoring and data collection aim to improve understanding and risk stratification of hemoglobinopathies globally.