Hematological treatment and prophylaxis in patients with and without inhibitors.

Abstract

This document focuses on the hematological treatment and prophylaxis that should be indicated in three groups of patients with hemophilia: Patients with inhibitors; Patients without inhibitors; and Patients receiving nonfactor replacement therapy. Inhibitors contribute to refractoriness to replacement treatment, leading to more severe bleeds. Immunotolerance and bypassing agents are essential to eradicate the inhibitors and bleeding control, respectively. Noninhibitors patients may receive standard half-life or extended half-life products, either on demand or prophylactically. Replacement factor concentrates are essential to control severe bleedings. Dose and frequency should be adjusted depending on the bleeding severity and type of factor concentrates (standard versus extended half-life products). Patients on emicizumab require bypassing agents (Novoseven is the agent of choice) in case of inhibitors and factor replacement in noninhibitor patients.