Rare Blood

Guidance for Evaluating for Bleeding Disorders in Suspected Child Abuse

September 26th 2022, 11:15pm

Article

The recommendations aim to make this type of evaluation more consistent in cases when children have suspicious bruising or bleeding patterns.

Children With Sickle Cell Anemia Using Medicaid Less Likely to Get Stroke Screening, Pain Treatment

September 20th 2022, 7:42pm

Article

In 2019, less than half of children aged 2 to 16 years with sickle cell anemia received the recommended screening for stroke, a common complication of the disease.

Review Highlights Advances in SCD Treatment, Potential Targets for Novel Agents

September 17th 2022, 11:05pm

Article

The review discusses hydroxyurea and 3 additional drugs approved by the FDA—L-glutamine, crizanlizumab, and voxelotor—as well as agents currently being investigated to treat sickle cell disease (SCD).

Sutimlimab Is Effective, Well Tolerated in Refractory Chronic Immune Thrombocytopenia

September 17th 2022, 3:30pm

Article

Sutimlimab demonstrated rapid efficacy in the study cohort, and platelet level improvements were sustained for the duration of treatment in nearly half of the participants.

Bendamustine/Rituximab vs Sutimlimab Monotherapy for CAD Explored in New Review

September 13th 2022, 10:36pm

Article

A review published in Transfusion Medicine Reviews discussed the 2 therapeutic approaches and their pros and cons for treating cold agglutinin disease (CAD).


The Impact of Orphan Drug Legislation on the Beta Thalassemia Treatment Landscape

September 9th 2022, 1:58pm

Article

Orphan drug designation incentives have helped boost enthusiasm for researching and developing drugs for beta thalassemia, a new review concludes.

Hemoglobin Improvement Correlates With Better HRQOL in Paroxysmal Nocturnal Hemoglobinuria

September 1st 2022, 1:27pm

Article

Post hoc analyses of the phase 3 PEGASUS trial found that clinical and hematological improvements were associated with better patient-reported fatigue and physical function outcomes in paroxysmal nocturnal hemoglobinuria, for an overall bettering of health-related quality of life (HRQOL).

Patients With Sickle Cell Disease Less Likely to Receive Specialized Care

August 21st 2022, 5:08pm

Article

A recent analysis of Medicaid claims data found that individuals with sickle cell disease are seeing hematologists at a lower rate than patients with other chronic genetic diseases.

FDA Approves First Gene Therapy for Beta-thalassemia

August 17th 2022, 6:46pm

Article

The FDA Wednesday approved betibeglogene autotemcel (Zynteglo), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Impact of Cold Agglutinin Disease on Daily Life Seen in New Survey

August 15th 2022, 4:48pm

Article

Symptoms associated with cold agglutinin disease fluctuated throughout the course of the disease, and many patients were not satisfied with management measures.