Chris Roberson, Dr. Anne Greist, Dr. Manjusha Kumar, Sam Mbunya

Globally, approximately 515,000 infants with Sickle Cell Disease (SCD) are born every year. Approximately 80% of these cases occur in Sub‑Saharan Africa (SSA) annually, including 14,000 newborns in Kenya. In SSA, 50%–80% of children will die before the age of 5 years due to a lack of comprehensive SCD care compared to 3% in better‑resourced settings. The Academic Model Providing Access to Healthcare (AMPATH) SCD Program started in 2010 as a partnership between Moi University, Moi Teaching and Referral Hospital (MTRH), and Indiana Hemophilia and Thrombosis Center (IHTC) with a goal to improve access to comprehensive SCD care by increasing capacity through training, clinical care, research, and advocacy.

Dr. Amy Shapiro

Concizumab is an anti–tissue factor pathway inhibitor (TFPI) monoclonal antibody intended for once-daily subcutaneous prophylactic treatment for people with hemophilia A or B with and without inhibitors. Results from the phase 3 explorer7 study confirmed superiority of concizumab prophylaxis over no prophylaxis in reducing the annualized bleeding rate (ABR) in people with hemophilia A or B with inhibitors (HAwI/HBwI). Male patients aged ≥12 years were randomized 1:2 to no prophylaxis (group 1) or concizumab prophylaxis (group 2), or nonrandomly allocated to concizumab prophylaxis (groups 3 and 4). After ≥24 weeks of treatment, patients in group 1 could switch to concizumab prophylaxis. At the 56-week cutoff (defined as when all patients in groups 2-4 had completed the visit at 56 weeks or permanently discontinued treatment), bleed-related efficacy, pharmacokinetics and pharmacodynamics, and safety were assessed. Of the 133 patients enrolled (HAwI, n = 80; HBwI, n = 53), 114 received concizumab prophylaxis (groups 2-4) and 19 were randomized to no prophylaxis (group 1). After ≥24 weeks, 13 patients from group 1 switched to concizumab. Median ABR for treated spontaneous and traumatic bleeding episodes in patients receiving concizumab was 0.8 (interquartile range [IQR], 0.0-3.2) at the 56-week cutoff, consistent with the low bleeding rates (median ABR, 0.0; IQR, 0.0-3.3) at the 32-week cutoff. Concizumab and free-TFPI concentration remained stable over time. No new safety concerns were reported. Longer-term (≥1 year) efficacy and safety results of concizumab prophylaxis for HAwI/HBwI were consistent with the 32-week cutoff results in explorer7. This trial was registered at www.clinicaltrials.gov as #NCT04083781.