Therapeutic ultrasound combined with conventional physiotherapy proved superior to infrared therapy in patients with hemophilia, according to a recent study.

Alix Arnaud, Sanofi, discusses the cost comparison of therapies for major surgeries in patients with hemophilia A.

Background: Hemophilia A is an inherited bleeding disorder with an increased risk of excessive bleeding. People affected by hemophilia A with a severe bleeding phenotype are treated prophylactically with hemostatic agents. Monitoring treatment and adjusting it to the individual patient’s needs is complicated by the scarcity of laboratories equipped to perform relevant coagulation assays and the absence of point-of-care testing platforms. A novel near-patient testing platform called the EnzySystem HemA aims to address this issue by measuring Factor VIII activity and Thrombin Generation within one hour out of 100 µL of whole blood. Objective: This study assessed the #usability of the EnzySystem HemA by healthcare professionals without prior training and gathered information on its perceived #usability, effectiveness, usefulness, and acceptability. Methods: A qualitative, single-center formative #usability assessment was performed. Seven healthcare providers participated, all unaffiliated with the EnzySystem’s developer (Enzyre). Participants included one pediatric hematologist, three (pediatric) nurses, and three (pediatric) nurse practitioners. Two of the participants also held management positions. Participants performed a FVIII activity assay with a mockup of the EnzySystem HemA and were interviewed about its acceptability, #usability, effectiveness, and usefulness. Video recordings of the sessions were obtained with permission and subsequently reviewed to assess #usability; thematic analysis was performed on the interview transcripts. Results: Five participants (71%, 5/7) successfully performed the FVIII activity assay as intended. Of the remaining two participants, one applied insufficient force when inserting the blood tube, while the other was unable to transfer the blood into the EnzySystem. This latter step caused difficulties in three other participants, however they completed it correctly. Five participants (71%, 5/7) did not dispose of the EnzySystem components as intended, primarily due to compatibility issues of the disposable parts with the inlet of US standard medical waste containers. Five themes were generated from the interviews: “Functional the way it is” focuses on the acceptance of the prototype despite suggestions for various improvements; “Utility through faster results and increased access” and “Potential for patient engagement” relate to the envisioned benefits of the platform; and “Financial investment and return” and “Ensuring accuracy” reflect on potential barriers for implementation. Conclusions: Despite significant #usability issues, the need for faster and more accessible testing led participants to conclude that the current platform was acceptable for use, provided that proof of assay accuracy was demonstrated. The results of this study will form the basis for further development of the EnzySystem HemA. The effectiveness of any updates will be evaluated in future #usability studies, which will also include people with hemophilia A as participants.

Phase III trial shows once‑monthly and once‑weekly denecimig (Mim8) significantly reduces annualized bleeding rate versus standard care, with up to 95% of patients bleed‑free.

Alhemo wins FDA nod for hemophilia A and B without inhibitors, showing up to 86% fewer bleeds in a Phase III trial.

Belief BioMed and Grand Life Sciences partner to bring BBM-H803 gene therapy to 30,000 hemophilia A patients in China. Here's what it means for treatment access.

Key Points. Pathogenic deep intron variants in hemophilia A can be identified from patient iPSCs via genome editing.Our approach is potentially useful to v

npj Systems Biology and Applications - Prediction of hemophilia A severity using a small-input machine-learning framework

Early initiation of emicizumab treatment seems safe and effective for the management of acquired hemophilia A, according to a new study.

Emicizumab prophylaxis was considered potentially safe and effective in a real-world, international study of infants with hemophilia A.