Rare Blood

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

Nine out of 10 patients with hemophilia B dosed with FLT180a were able to stop their weekly infusions of replacement factor IX.

Patients with cold agglutinin disease (CAD) had durable responses at week 53, and no new safety signals were reported.

Additional population-based studies of autoimmune hemolytic anemia and its management are needed to reduce mortality in this patient population.

New data show the types of malignancies, such as leukemias and myeloproliferative disorders, that are most common in children with Langerhans cell histiocytosis (LCH).

The patient was 52 years old and underwent a liver transplant 13 years after his hematopoietic stem cell transplant (HSCT) to treat his sickle cell disease (SCD).

Creating and validating effective risk assessment models could help facilitate appropriate prophylaxis for hemophilia, but more research is needed to confirm the prognostic value of various clinical risk factors and biomarkers.

A new report offers novel insights into a very small patient population.

Surveys of health care professionals, patients, and caregivers explored unmet needs in sickle cell disease (SCD), as well as the significant impact the disease has on everyday life.

Investigators said the benefits appeared relatively early in treatment for many patients and persisted throughout the study period.

A systematic review and meta-analysis found no clear advantage for automated red cell exchange.

Prior to the pandemic, youth with sickle cell disease were more likely to report depressive symptoms, but the opposite was true during the pandemic.

A cure for sickle cell disease could more than double the lifetime incomes of patients, the study found.

A panel of experts suggests adding caplacizumab to the historic treatment of plasma exchange and immunosuppression could lead to significant improvement in outcomes for patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP).

The findings highlight the significant financial burden of sickle cell disease.

While suggesting that the disorder be included in the differential diagnosis of thrombocytopenia following vaccination, the group emphasized that the benefits of global vaccination against the virus outweigh the risk of this rare complication.

Patients with cold agglutinin disease faced a 4.5-times greater risk of death during winter months, new data show.

Among their sample cohort of 49 patients, the researchers found that scores on the Montreal Cognitive Assessment correlated with measures of both literacy and health literacy.

Improved management of sickle cell disease has vastly improved life expectancy and reduced morbidity for affected patients, but the disease itself and its treatments carry fertility risks that many young patients and their caregivers are unaware of.

During her presentation at the Academy of Managed Care Pharmacy Nexus 2021 meeting, Aimee Tharaldson, PhD, highlighted the most anticipated specialty pharmacy drugs awaiting FDA approval, including several treatments for cancer and rare diseases.