sickle cell disease

Long-Term Outcomes of Fixed Low versus Moderate Hydroxyurea Dosing in Sickle Cell Stroke Prevention: Insights from the SPRINT Cohort

By MedXY 11/02/2026

A long-term follow-up of the SPRINT trial shows no significant difference in recurrent stroke or mortality rates between initial low and moderate fixed doses of hydroxyurea in sickle cell disease, emphasizing event clustering within the first year.

Clinical Updates Hematology-Oncology

Mitapivat in Sickle Cell Disease: A Promising Oral Therapy to Improve Hemolytic Anemia and Reduce Complications

By MedXY 11/01/2026

The phase 3 RISE UP trial demonstrates that mitapivat significantly improves hemoglobin levels in sickle cell disease, offering a novel, well-tolerated oral treatment with potential to modify disease outcomes.

Hematology-Oncology Neurology news

Unveiling the Systemic Drivers of Blood-Brain Barrier Disruption in Sickle Cell Disease: Insights from Proteomic Profiling

By MedXY 09/20/2026

This study identifies systemic pathological pathways that mediate blood-brain barrier injury in sickle cell disease, linking BBB disruption to brain hypoxia and microstructural injury, highlighting new therapeutic targets to preserve neurological function.

Hematology-Oncology news

Optimizing Autologous Stem Cell Backup Mobilization with GCSF and Plerixafor in Sickle Cell Disease: Implications for Gene Therapy

By MedXY 09/18/2026

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.

Hematology-Oncology news

Sustained Genetic Silencing of BCL11A Using shmiR Vector in Sickle Cell Disease: Long-Term Safety and Efficacy

By MedXY 08/24/2026

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 over 4+ years.

Hematology-Oncology Nephrology news

Renin-Angiotensin-Aldosterone System Inhibitors Worsen Anemia in Sickle Cell Disease: Clinical and Mechanistic Insights

By MedXY 08/10/2026

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.

Hematology-Oncology news Pediatrics

Reduced-Intensity Thiotepa Plus Post-Transplant Cyclophosphamide Delivers Near-Universal Engraftment and Minimal GVHD in Matched Sibling Transplantation for Adolescents and Adults With Sickle Cell Disease

By MedXY 05/04/2026

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.

Hematology-Oncology news

Addressing the Silent Crisis: Menstrual Pain and Abnormal Bleeding as Drivers of Acute Care in Sickle Cell Disease

By MedXY 12/21/2025

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 in interdisciplinary care and reproductive health management.

Hematology-Oncology news

Validated PRESEV Score Identifies Adults with Sickle Cell VOC at Low Risk for Acute Chest Syndrome — Implications for Safer Outpatient Management

By MedXY 12/18/2025

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 management strategies in selected patients.

Hematology-Oncology news Pediatrics

Community Health Workers Halt Decline in Quality of Life During Transition to Adult Care for Young Adults With Sickle Cell Disease

By MedXY 11/17/2025

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 health intervention provided transient benefit.

Emergency Medicine Hematology-Oncology news Pediatrics

Timely Opioid Administration in Pediatric Emergency Departments Reduces Hospitalizations for Acute Sickle Cell Pain

By MedXY 10/27/2025

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.

news Rheumatology Specialties

WHO Releases Comprehensive Guidelines for Managing Sickle Cell Disease in Pregnancy

By MedXY 07/28/2025

New WHO guidelines offer evidence-based approaches to improve care for pregnant women with sickle cell disease, focusing on treatment and maternal outcomes.