Two major advances in the treatment of hemophilia A guided by PK: guideline / consensus development + research data release

In the past two years, clinical research data related to PK-guided individualized preventive treatment have been published at major international conferences on hemophilia and coagulation, which further confirms the importance of PK-guided individualized preventive treatment
.

At the 15th Annual Meeting of the European Society for Hemophilia and Related Diseases (EAHAD) in 2022, the latest interim data from the AHEAD study, an ongoing non-interventionist, prospective, multicenter study (7 years of follow-up as of June 2020), compared the efficacy and factor consumption of 158 adult patients with moderate hemophilia A who received rAHF-PFM standard preventive therapy and PK-guided individualized preventive therapy, and the results showed ^{that 3}:

• Compared with patients receiving standard prophylaxis, the median annual bleeding rate (ABR) was lower (0 vs.
  2), with a higher proportion of patients with zero ABR and zero annual joint bleeding rate (AJBR) (ABR = 86% vs.
  31%; Proportion of patients with zero AJBR = 86% vs.
  59%)
  .
  

In 2021, the 63rd American Society of Hematology (ASH) published a study to analyze the application of PK-guided individualized preventive therapy in the perioperative period of hemophilia A patients, using a two-chamber model to simulate the PK curve, and collecting plasma concentration, factor consumption and blood loss data of perioperative FVIII to verify the effectiveness of
the individualized regimen.
Results show ⁴:

• A total of 46 patients were included and 50 surgeries
  were performed.
  The median FVIII consumption of PK-guided individualized therapy was reduced by 26.
  69% (511.
  7 U/kg vs.
  648.
  3 U/kg, P<0.
  001)<b11> during perioperative surgery compared with weight-based conventional prophylaxis.
  Perioperative substitution therapy in patients with hemophilia A guided by PK reduces FVIII consumption
  .
  

In 2021, the 29th International Society for Thrombosis and Haemostasis (ISTH) published a cross-sectional, retrospective burden of disease study, real-world evidence for
the CHESS II study.
A total of 155 patients aged ≥ 18 years with hemophilia A were enrolled, and studies showed ^{that 5}:

• Compared with no PK-directed prophylaxis, PK-guided individualized prophylaxis resulted in lower mean ABR (4.
  4 vs.
  1.
  6), fewer mean target joint counts (1.
  2 vs.
  0.
  6), fewer average hematologist consultations (11.
  8 vs.
  6.
  2), and lower proportion requiring hospitalization (49% vs.
  10%)
  .
  Demonstrated real-world benefits of PK-guided preventive treatment for clinical and health resource use ^outcomes5
  .