Efficacy of emicizumab therapy in two adult patients with type 3 von Willebrand disease.

Type 3 von Willebrand disease (T3VWD) is a rare inherited bleeding disorder caused by the absence of von Willebrand factor (VWF). The traditional treatment for T3VWD has been VWF concentrates, but their effectiveness may be limited due to the development of alloantibodies. Emicizumab, a bispecific mAb, has shown promise in treating hemophilia A and is being studied as prophylaxis for T3VWD. In this case series, two patients with T3VWD received emicizumab prophylaxis and experienced a significant reduction in bleeding episodes and improved quality of life with fewer healthcare encounters. Although breakthrough bleeding was rare, one patient experienced a terminal intracranial bleed. Despite limited clinical experience with emicizumab in T3VWD, these cases suggest that emicizumab may be a valuable prophylactic option for patients with T3VWD. Further research is needed to determine the long-term efficacy and safety profile of emicizumab and optimal therapy for breakthrough bleeds in this patient population.

Inaccuracy of Initial Clinical Mobility Assessment in Venous Thromboembolism Risk Stratification.

BACKGROUND: Venous thromboembolism risk increases in hospitals due to reduced patient mobility. However, initial mobility evaluations for thromboembolism risk are often subjective and lack standardization, potentially leading to inaccurate risk assessments and insufficient prevention. METHODS: A retrospective study at a quaternary academic hospital analyzed patients using the Padua risk tool, which includes a mobility question, and the Johns Hopkins-Highest Level of Mobility (JH-HLM) scores to objectively measure mobility. Reduced mobility was defined as JH-HLM scores ≤3 over ≥3 consecutive days. The study evaluated the association between reduced mobility and hospital-acquired venous thromboembolism using multivariable logistic regression, comparing admitting health care professional assessments with JH-HLM scores. Symptomatic, hospital-acquired thromboembolisms were diagnosed radiographically by treating providers. RESULTS: Of 1715 patients, 33 (1.9%) developed venous thromboembolism. Reduced mobility, as determined by the JH-HLM scores, showed a significant association with thromboembolic events (adjusted OR: 2.53, 95%CI:1.23-5.22, P = .012). In contrast, the initial Padua assessment of expected reduced mobility at admission did not. The JH-HLM identified 19.1% of patients as having reduced mobility versus 6.5% by admitting health care professionals, suggesting 37 high-risk patients were misclassified as low risk and were not prescribed thrombosis prophylaxis; 4 patients developed thromboembolic events. JH-HLM detected reduced mobility in 36% of thromboembolic cases, compared to 9% by admitting health care professionals. CONCLUSION: Initial mobility evaluations by admitting health care professionals during venous thromboembolism risk assessment may not reflect patient mobility over their hospital stay. This highlights the need for objective measures like JH-HLM in risk assessments to improve accuracy and potentially reduce thromboembolism incidents.

Development of a Medication-Use Evaluation Template for Andexanet Alfa in the Reversal of Anticoagulation With Factor Xa Inhibitors.

Medication-use evaluations are meant to ensure that medication-use processes are consistent with prevailing standards of care, assure optimal use of therapy, and reduce the risk of medication-related problems. Reversal agents for direct oral anticoagulants are a worthy focus for medication-use evaluations for reasons of efficacy, safety, and cost. A multidisciplinary team of experts developed 2 medication-use evaluation templates illustrating the application of professional society guidelines to the appropriate use of andexanet alfa.