Rare Blood

A panel of specialists proposed 26 essential elements for comprehensive pediatric sickle cell disease care, laying groundwork for standardized guidelines and the establishment of accredited care centers in the future.

Over the course of voxelotor treatment, hemoglobin levels and hemolysis markers improved, with results sustained over the treatment period.

The analysis emphasizes the association between vaso-occlusive crisis frequency and health care costs, which are inflated largely due to inpatient visits.

A recently published report describes the diagnosis and treatment of a case of iron-deficiency anemia–associated thrombocytopenia secondary to recurrent nosebleeds.

Population-based data is a crucial aspect to evaluating current care quality and improving outcomes for patients with sickle cell disease.

A review published in the journal Immunotherapy summarizes the applications of sutimlimab and emphasizes the importance of individualizing treatment for patients with cold agglutinin disease.

Findings of a recent study suggest that complications of sickle cell disease (SCD) significantly contribute to morbidity and are more prevalent among older patients.

An analysis of patient-reported outcomes from the pivotal CARDINAL trial of sutimlimab in cold agglutinin disease found that quality of life (QOL) improved and persisted throughout treatment.

Currently, there are no clear guidelines for managing extramedullary hematopoiesis in transfusion-dependent beta thalassemia.

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

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.

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

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


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

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).

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.

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.

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

The case of warm autoimmune hemolytic anemia (AIHA) highlights the potential for life-threatening acute hemolysis if patients do not receive timely treatment.

With adherence estimated at less than 50% in children, adolescents, and young adults, a recent study stresses the importance of treatment adherence for patients with sickle cell disease receiving hydroxyurea and the benefit for those who do.

Current standard modalities for detecting and quantifying monoclonal immunoglobin in patients with cold agglutinin disease lack adequate sensitivity. Heavy chain/light chain assay may be more effective.

This retrospective study and prospective follow-up provide insight into predictors of thrombotic episodes in patients with autoimmune hemolytic anemia (AIHA) and indicators that anticoagulant prophylaxis may be beneficial to certain patients.

The Institute for Clinical and Economic Review (ICER) released the report, which supports the long-term value of betibeglogene autotemcel (beti-cel) for the treatment of beta thalassemia.

A patient who failed to respond to rituximab and steroids later saw dramatic improvement with sutimlimab