Accelerated approval of Oxbryta ® (voxelotor): A case study on novel endpoint selection in sickle cell disease
Sickle cell disease (SCD) is an inherited disease characterized by hemolysis, anemia, and vaso-occlusion leading to substantial morbidity and mortality. Development of prior pharmacologic therapies exclusively utilized vaso-occlusive crisis (VOC) as a clinical efficacy endpoint; however, this focus on VOC did not capture the full extent of disease symptomatology and complications and slowed the development of new therapies. Voxelotor, a hemoglobin S polymerization inhibitor, was recently approved in the United States for the treatment of SCD in adults and adolescents 12 years of age and older through an accelerated approval pathway.
Publication date: Available online 8 October 2020Source: Hematology/Oncology and Stem Cell TherapyAuthor(s): M.M. Sulaiman, A.I. Ladu, A.M. Abba, A.A. Bukar
Condition: Sickle Cell Anemia Intervention: Drug: Voxelotor Sponsors: Elizabeth Yang; Mednax Center for Research, Education, Quality and Safety; University of California, San Francisco; Global Blood Therapeutics Recruiting
ConclusionThe prevalence of RLS among this sample of adult patients with sickle cell anemia was much higher than that previously reported for the general Turkish population. The presence of RLS was an important cause of sleep disturbance in these adult patients with SCA.
CONCLUSION: The prevalence of RLS among this sample of adult patients with sickle cell anemia was much higher than that previously reported for the general Turkish population. The presence of RLS was an important cause of sleep disturbance in these adult patients with SCA. PMID: 33029692 [PubMed - as supplied by publisher]
Conclusion: The risk of pregnancy in patients with homozygous sickle cell anemia remains high, on both the maternal and fetal sides. PMID: 33014460 [PubMed]
Abstract Total pancreatectomy with islet autotransplantation is a complex surgical approach for acute recurrent or chronic pancreatitis that frequently triggers extreme thrombocytosis (platelets ≥ 1000 × 109 /L). Thrombocytosis can be prothrombotic, so cytoreductive hydroxyurea is often initiated after this surgery; however, optimal dosing strategy and efficacy are unknown. This prospective pilot study characterized the pharmacokinetics of hydroxyurea after this procedure in children. It also compared them with previously published pediatric parameters in sickle cell anemia (SCA), the disease in which ped...
Authors: Ribeiro-Filho J, Yahouédéhou SCMA, Pitanga TN, Santana SS, Adorno EV, Barbosa CG, Ferreira JRD, Pina ETG, Dos Santos Neres JS, Leite IPR, Lyra IM, Goncalves MS Abstract INTRODUCTION: Ticagrelor is an antiplatelet agent approved for the treatment of patients with an acute coronary syndrome or a history of myocardial infarction. Considering the evidence demonstrating that ticagrelor-mediated inhibition of platelet activation and aggregation have beneficial effects in the treatment of thrombotic conditions, clinical studies have been conducted to evaluate the use of this drug for the treatment o...
CONCLUSION: Our findings confirm that thrombotic events related to EXT are rare in pediatric patients, which can inform the use of empiric anti-platelet therapy. PMID: 32979018 [PubMed - as supplied by publisher]
CONCLUSIONS: While Niprisan® appeared to be safe and effective in reducing severe painful crises over a six-month follow-up period, further trials are required to assess its role in managing people with SCD and the results of its multicentre trials are awaited. Currently, no conclusions can be made regarding the efficacy of Ciklavit® and the powdered root extract of Pfaffia paniculata in managing SCD. Based on the published results for Niprisan® and in view of the limitations in data collection and analysis of the three trials, phytomedicines may have a potential beneficial effect in reducing painful crises in ...
CONCLUSIONS: PheWAS is a viable tool for calculating risk associated with any biomarker. The current analysis provide a new approach to generating hypotheses and understanding the breadth of health disparities. Future analyses will further explore causality, risk factors, and potential confounders not accounted for here. PMID: 32958289 [PubMed - as supplied by publisher]