Abstract Number: PB0629
Meeting: ISTH 2021 Congress
Background: Severe type of haemophilia B (HB) is defined by undetectable FIX plasma level activity(<1 IU/ml) and characterized by spontaneous bleeds into joints and muscles. Without proper treatment, recurrent intraarticular bleeds inevitably lead to joint damage, resulting in function impairment and major disability.
Aims: The study goal was to perform a clinical analysis of severe HB patients including disease complications and treatment regimens, evaluation of haemophilic arthropathy and degree of of joints’ function impairment.
Methods: Study group consisted of 53 severe HB patients aged 12-74 (median 36) years. Retrospective and prospective clinical data were collected using dedicated questionaries. The Hemophilia Joint Health Score (HJHS) v.2 scale was used to evaluate the degree of joint disease. The examination included function measurement of ankles, knees and elbows, together with gait assessment.
Results: In the study group 22/53(41,5%) had life-threatening bleeding episodes including 9 (17%) intracranial haemorrhages. Spontaneous intraarticular bleeds were reported by 50/53(94,3%) and intramuscular bleeds by 40/53(75%) patients. The mean HJHS score (joints) was 25.57 ± 20.47(median 23) and gait impairment was 2 points. Due to advanced haemophilic arthropathy, 22/53(42%) patients underwent orthopaedic surgery, 11(21%) had at least one alloplasty. Prospectively collected data revealed that 37/52(71,2%) patients were prophylactic infusers, 10(19,2%) were treated “on demand” and 5(9,62%) on demand with occasional prophylactic FIX infusions. The mean annualized bleeding rate was 2.53 ± 6.54 for prophylactic users, and 15.4 ± 13.00 for “on demand” group.
Conclusions: Our study confirmed that severe haemophilia B patients are at high risk of bleeding, including life-threatening haemorrhages unless prophylactic therapy is applied. Study analysis indicated connection between the lack of optimal treatment and advanced joint desease and demonstrated that switching from “on demand” to prophylaxis was beneficial even in adult patients with preexisting severe arthropathy.
To cite this abstract in AMA style:Buczma A, Odnoczko E, Baran B, Gwozdowska A, Zawilski J, Windyga J. Clinical Assessment with the Evaluation of Joint Disease and Haemostatic Treatment in Patients with Severe Haemophilia B [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/clinical-assessment-with-the-evaluation-of-joint-disease-and-haemostatic-treatment-in-patients-with-severe-haemophilia-b/. Accessed August 15, 2022.
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