Paradigm Shift for Hereditary Hemophilia: Towards Precision Medicine

The risks that come with living a “normal” life for a former hemophilia patient is likely to shift the paradigm in the treatment of hemophilia, according to an article published in Blood Reviews.

Patients with hemophilia A (HA) or B (HB) experience spontaneous bleeding episodes which threatened both their lives and limbs. These bleeding occurrences are currently treated with regular infusions of deficient coagulation factor (FVIII or FIX). Researchers are now examining new treatment modalities, which include prophylaxis with subcutaneous long-acting non-factor products that may enhance in vivo thrombin efficacy. Some of treatments are currently under investigation, but in other cases are already being successfully administered to patients with hemophilia.

The authors noted that both hemophilia patients with and without inhibitors already take advantage of non-factor products. For patients who also need bypassing agents, or FVIII concentrates for breakthrough bleeds, thromboembolic events may sometimes occur.

To improve treatment outcomes and provide comprehensive care, physicians require a thorough knowledge of individual needs to best tailor a treatment plan for hemophilia patients. This strategy includes:

  1. Identifying which hemophilia patients are less likely to benefit from a given intervention
  2. Defining the optimal dosing and scheduling of bypassing agents (or FVIII) to employ in combination with non-factor products
  3. Establishing tests to monitor in vivo thrombin generation
  4. Improving communication and deliver results to individuals

“As individual outcomes will be improved and the risk of adverse events minimized, non-factor products will come into wider use within the hemophilia community, and patients will hopefully have no more risks of breakthrough bleeds,” the authors wrote. “The risks of a normal life for a ‘former hemophilia patient’ is likely to change the treatment landscape and the structure of hemophilia Centers.”

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