Rose McNulty

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

A recent study suggests that including a pharmacist in the hemophilia comprehensive care model may improve bleeding outcomes and medication access and adherence, and lead to cost savings.

Renal outcomes and chimeric antigen receptor (CAR) T-cell therapy efficacy were unaffected by baseline renal status in a cohort of patients with diffuse large B cell lymphoma, but acute kidney injury during treatment was associated with worse clinical outcomes.

A recent review details strategies for mitigating the risks faced by pregnant patients with hemophilia or von Willebrand disease and their children.

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

A small study suggests that comprehensive genomic profiling can help with clinical decision-making and predicting clinical outcomes in advanced biliary tract cancer, but further research is needed.

The rare condition can easily be confused with hepatocellular carcinoma, but immunohistochemical studies can help accurately diagnose intrahepatic sarcomatoid cholangiocarcinoma.

Morbidity and mortality remain high for patients with acquired hemophilia A, and advances in immunotherapy and hemostatic therapy are needed to improve outcomes.

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).

The study pooled data from 4 clinical trials to gain insight into surgical outcomes in this complex population.

Despite $2.5 million placeholder price tags, the Institute for Clinical and Economic Review draft evidence report considers gene therapy cost-effective versus comparators in both hemophilia A and B.

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.

The review highlights current strategies for myeloid cell targeting and novel agents targeting myeloid cells for cancer treatment.

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


A review published in Frontiers in Oncology explored the application of allo-SCT for myelodysplastic syndromes and myeloproliferative neoplasms.

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

The findings, which contradict prior North American studies, suggest that the incidence rate of the rare cancer continues to increase in the United States.

A significant portion of patients who receive chimeric antigen receptor (CAR) T-cell therapy experience immune effector cell–associated neurotoxicity syndrome, and this recent study suggests neurofilament light chain protein levels may hold promise as a biomarker to identify at-risk patients.

The case emphasizes the importance for clinician awareness of the potential for rare immune-mediated disorders, including acquired hemophilia A (AHA), following allogeneic hematopoietic stem cell transplantation (HSCT).

The management of myeloproliferative neoplasms (MPNs) in adolescent and young adult (AYA) patients is currently similar to that of older patients. However, comprehensive research is needed to form guidelines for this younger population.

A recent review suggests point-of-care ultrasound can help home hospital care providers assess, diagnose, and monitor a range of conditions with relatively little additional training.

As the feasibility of phase 1 clinical trials for prenatal spinal muscular atrophy (SMA) therapies is explored, patient and parent input on prenatal testing and possibly treatment is a valuable tool for guiding research discussions.

Nurse-led education is an essential aspect of care for patients with bleeding disorders, but there is currently a lack of research on specific strategies to improve patient self-management through education.

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).

The real-world study adds to previous randomized controlled trial findings suggesting that shorter courses of antibiotic therapy can be sufficient for hospitalized patients with mild or moderate community-acquired pneumonia.

A retrospective study found that adolescents and young adults (AYA) receiving tyrosine kinase inhibitors (TKI) therapy for chronic-phase chronic myeloid leukemia (CML) had similar prognoses to older patients despite also showing higher tumor burden at diagnosis.

Systemic mastocytosis and mast cell activation syndrome both negatively impact health-related quality of life (HRQOL), with mast cell activation syndrome patients reporting more significant impacts on everyday life.

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 results suggest that dipeptidyl peptidase-4 (DDP-4) inhibitors, which are widely prescribed in Germany, are not a cost-effective option despite lower drug pricing compared with empagliflozin.

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