Intensive Replacement Treatment in Haemophilia Patients With Synovitis
Status:
Not yet recruiting
Trial end date:
2022-05-01
Target enrollment:
Participant gender:
Summary
Background: Joint haemorrhage represents the most common type of bleeding episode in persons
with hemophilia (PwH). In the absence of an adequate prophylaxis with Factor VIII (for
hemophilia A) or FIX (for hemophilia B) concentrates up to 85% of patients with severe
hemophilia develop a clinically overt joint disease. Screening of early signs of arthropathy
is needed. Synovitis is widely considered as one of the parameters to be taken into account
for the diagnosis and the surveillance of joint impairment in PwH.
Aim: To assess if an intensive factor VIII replacement treatment is able at reverting
synovitis in PwH.
Methods: The present study is a randomized, open-label, cross-over study. Among patients
referred to enrolling Haemophilia Centres, consecutive patients with severe (FVIII < 1%) or
severe-moderate (FVIII < 2%) haemophilia A without inhibitors will be enrolled. The present
study will be organized in 2 phases.
- Phase 1 (US screening): All patients will undergo an ultrasound examination of elbows,
ankles and knees to define joint status and to identify presence/absence of synovitis
according to the HEAD-US system.
- Phase 2 (Intervention): Patients with US evidence of synovitis will be randomly assigned
at undergoing a PK assessment with my-PK-fit to start a prophylaxis with Adynovi®
targeting a 12% FVIII through level (PROPEL-like arm) or to continue ongoing standard
treatment (control arm). US examination of the six joints will be repeated monthly for
six months and in case of onset of symptoms that might suggest an acute bleeding
episode. After six months the two treatment arm will be switched in the frame of a
cross-over approach and all PwH will be followed for other 6 months The primary outcome
will be represented by changes in synovial status during the intensive factor VIII
replacement treatment vs standard treatment.