علاج الهيموفيليا أ الشديدة والمتوسطة الشدة بدون مثبطات العامل الثامن

References

DOI: 10.1016/j.jtha.2024.05.026

• In individuals with severe and moderately severe hemophilia A without inhibitors, the ISTH Hemophilia Guideline Panel recommends prophylaxis over episodic treatment of bleeding events (strong recommendation, based on moderate-certainty evidence ⨁⨁⨁◯).
• Therefore, we defined hemophilia as severe and moderately severe when individuals enrolled in the study population had FVIII or FIX activity levels <2 IU/dL.
• In individuals with severe and moderately severe hemophilia A without inhibitors, the ISTH Hemophilia Guideline Panel suggests either prophylaxis with emicizumab or prophylaxis with FVIII concentrates (conditional recommendation, based on very low–certainty evidence ⨁◯◯◯).
• Emicizumab may offer a lower treatment burden for patients given its weekly, biweekly, or every 4-week schedule and subcutaneous administration.