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July 21st 2023
The findings show liver complications and cirrhosis as major causes of morbidity in patients with SCD, with implications for treatment and care.
Results show a high level of adherence to hydroxyurea medication is essential for lowering the incidence of negative clinical outcomes like VOCs and acute chest syndrome in children with sickle cell anemia.
Investigators address 2 questions: How can we better understand the biology of these rare disorders? How can we stimulate research and optimize the regulatory process to improve diagnosis and targeted treatment?
Findings from the study emphasize the importance of disseminating knowledge and education to reduce disease burden after examining healthcare students' knowledge and attitudes towards sickle cell trait (SCT) screening in Ghana.
According to the results, 42.8% of participants failed to agree that relevant legislature is in place for the sickle cell disease population, as it doesn't facilitate optimal access to high-quality healthcare.
"Despite advances in SCD management and high-risk pregnancy care, at the national level, outcomes in this population have not improved since the last NIS analysis of data from 1999 to 2008," investigators write.
With public health efforts and therapeutic advancements the prognosis for this rare condition has progressed, but longer life expectancy has highlighted a multitude of areas that need attention in this population.
The targeted patient population included those with sickle cell disease or beta thalassemia who were considered at high risk for complications, or otherwise ineligible for standard myeloablative hematopoietic stem cell transplant.
"There's no national guidance on how this information should be delivered back to families, which leaves it up to each state's newborn screening program to decide the best route that they see fit," Dr. Corinna Schultz explains.
Updated findings from the ongoing clinical trial investigating lovo-cel in sickle cell disease and persistent anemia allude to the possibility that a contributing factor of anemia was identified.
The observed heterogeneity between BP-CMML cases suggested distinct clinicopathological phenotypes and the investigators integrated clinical metadata with blast immunophenotype with 59 independent features.