An international group of 15 hematology specialists from across the globe has agreed on a number of recommendations for the management of patients with hemophilia B, according to a study published in Therapeutic Advances in Hematology.
The consensus statement was very broad in scope, covering 5 main areas: choice of factor product, therapeutic agent monitoring considerations, dose optimization, inhibitor management, and gene therapy. Thirty-three recommendations were evaluated in the first round of online voting, with a consensus reached on 29 of them during the second round of live voting.
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Here are some highlights from the consensus statements that were agreed upon:
- Physicians should consider prophylaxis with factor IX (FIX) as a therapeutic choice for all patients with severe hemophilia B. To monitor FIX therapy, laboratories should consider using proficiency testing for the particular product under investigation and use validated assays.
- Doses and frequency of prophylactic therapy should be adapted to the clinical phenotype and lifestyle considerations of the patient.
- Clinicians should conduct a review of product-specific characteristics, patient phenotype, and joint status to determine whether pharmacokinetic analysis is useful in guiding individualized prophylaxis dosing.
- Physicians should conduct inhibitor screening routinely in all patients with severe hemophilia B, and that scrutiny should intensify if a patient develops an allergic reaction to FIX or demonstrates inadequate response to FIX replacement therapy.
- The first-line treatment for FIX inhibitor eradication should be immune tolerance induction protocols combined with FIX and immunosuppressive agents.
The consensus statement says current studies on gene therapy show promise for its use as a future treatment option in patients with severe hemophilia B. Should gene therapy be introduced as a form of treatment, long-term safety and efficacy follow-ups may be needed, it says.
The authors concluded, “It is hoped that the recommendations for clinical management provided here will complement existing guidelines and support the optimal management of [people with hemophilia B].”
Hart DP, Matino D, Astermark J, et al. International consensus recommendations on the management of people with haemophilia B. Ther Adv Hematol. 2022;13:20406207221085202. doi:10.1177/20406207221085202