A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This is a 48-month observational, prospective, multicentre study. The overall aim of the
 study is to evaluate the long-term effectiveness of Elocta treatment on joint health in
 patients treated prophylactically with Elocta in a real-world setting.
Haemophilia A is a rare genetic disorder estimated to occur in one out of 10,000 live births,
 characterized by a deficiency in coagulation factor VIII causing impaired haemostasis and
 prolonged bleeding episodes. Moderate haemophilia, defined as < 5%, and severe haemophilia,
 defined as < 1% of normal factor VIII activity result in frequent and spontaneous bleeds into
 muscles and joints, commonly the elbows, knees, and ankles. Bleeding into joints can cause
 acute pain and swelling and can result in reduced joint range of motion, long-term cartilage
 damage and debilitating haemophilic arthropathy. Early use of prophylaxis with factor VIII
 replacement is recommended following diagnosis of haemophilia A to maintain joint health and
 prevent joint destruction. However, despite the use of prophylaxis many patients still
 experience joint bleeds which may lead to joint deterioration over time. The risk of joint
 bleeds increases with the amount of time spent below certain FVIII trough levels, e.g. 1, 3
 or 5 IU/dL. Thus, there is probably a relation between the intensity of the prophylactic
 treatment regimen and joint health. Elocta is an extended half-life rFVIII product (EHL
 rFVIII), with a slower clearance as compared to conventional FVIII products. Treatment with
 Elocta will therefore provide the treater with a greater flexibility for individualizing
 prophylaxis as compared to conventional FVIII. Higher trough levels can be reached with
 Elocta without increasing factor usage or injection frequency. The treater can instead choose
 to reduce the injection frequency or the factor consumption without lowering trough levels.

 The purpose of this study is to evaluate the effectiveness of Elocta on joint health over a
 long observation period (48 months). The study will explore the influence on long term joint
 health of different Elocta prophylaxis regimens leading to different trough levels.
Inclusion Criteria:

 -  Provided signed and dated informed consent by the patient, or the patient's legally
 authorized representative(s) for patients under the legal age, before any
 study-related activities are undertaken. Assent should be obtained from paediatric
 patients according to local regulations

 -  Have a diagnosis of haemophilia A

 -  At enrolment on prophylactic treatment with Elocta, independent of participation in
 the study

 Exclusion Criteria:

 -  Enrolment in another concurrent clinical interventional study, or intake of an
 Investigational Medicinal Product (IMP), within three months prior to inclusion in
 this study

 -  Presence of factor VIII antibodies (inhibitors) (≥0.60 Bethesda Unit [BU]/mL) at the
 latest available inhibitor test