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A retrospective study demonstrates the safety and efficacy of GCSF and plerixafor for CD34+ stem cell mobilization as autologous backup in sickle cell patients undergoing alternate donor HSCT, with insights relevant for gene therapy.
This article reviews the first-in-human study demonstrating durable and safe posttranscriptional silencing of BCL11A in sickle cell disease patients using a shmiR vector, resulting in stable fetal hemoglobin induction and clinical benefit o
RAAS inhibitors, used for kidney protection in sickle cell disease, may significantly exacerbate anemia by impairing erythropoiesis, warranting close hemoglobin monitoring in this vulnerable population.
A prospective multicenter study reports excellent survival, no graft failure, and very low GVHD with a thiotepa-based reduced-intensity matched sibling transplant platform for severe sickle cell disease.
A multicenter study reveals that menstrual pain and abnormal uterine bleeding are highly prevalent in women with sickle cell disease and are linked to increased hospitalizations. Low awareness of hormonal therapies highlights a critical gap
An international prospective validation confirms the PRESEV score reliably identifies adult sickle cell patients hospitalized for vaso-occlusive crisis (VOC) who are at low risk for acute chest syndrome (ACS), enabling safer outpatient mana
A multicenter randomized trial found that community health worker support produced modest but durable improvements in health-related quality of life for young adults with sickle cell disease during transition to adult care, whereas a mobile
Prompt opioid delivery in pediatric EDs for sickle cell pain significantly lowers hospitalization rates, with the greatest benefit seen when the first dose is given within 60 minutes and the second dose within 30 minutes.
New WHO guidelines offer evidence-based approaches to improve care for pregnant women with sickle cell disease, focusing on treatment and maternal outcomes.