Abstract Number: PB0550
Meeting: ISTH 2021 Congress
Background: The development of inhibitory antibodies is a major complication of factor replacement therapy in patients with congenital hemophilia, with the risk of joint arthropathy in patients with recurrent joint bleeding. The long-term real-world effectiveness and safety of activated prothrombin complex concentrate (aPCC; Baxalta US Inc, a Takeda company, Lexington, MA, USA) have been assessed in patients with congenital hemophilia A or B with high-responding inhibitors (PwHI) in the FEIBA Global Outcome study (FEIBA GO).
Aims: To assess the effectiveness of prophylactic and on-demand aPCC therapy on joint health in PwHI and its safety.
Methods: FEIBA GO (EUPAS6691) was a post-authorization, prospective, observational, non-interventional, multicenter cohort study. Male PwHI diagnosed and prescribed treatment with aPCC (physician’s discretion) before study entry were eligible; planned observational period was 4 years. Ethics committee approval and patient consent were obtained. Data are from the safety analysis set.
Results: Fifty PwHI who were enrolled between September 3, 2014 and December 19, 2017 received aPCC prophylaxis (n=37) or on-demand treatment (n=13) at 25 sites in 11 countries (hemophilia A: n=49; hemophilia B: n=1). Overall annualized joint bleeding rate (AJBR) was lower in patients receiving prophylaxis than in those receiving on-demand therapy (Table 1). Overall, 11 (29.7%) and 4 (33.3%) PwHIs receiving prophylaxis or on-demand therapy had zero joint bleeds, respectively. Among PwHIs receiving prophylaxis or on-demand therapy, zero joint bleeds occurred in 6/18 PwHIs with 2-4 years’ follow-up and in 2/7 PwHIs with >4 years’ follow-up. A total of 177 and 31 adverse events were reported in 70.0% (28/40) and 76.9% (10/13) of PwHI receiving prophylaxis or on-demand therapy, respectively. Most frequently reported events were infections and musculoskeletal disorders (Table 2). No thrombotic microangiopathy was reported.
|By treatment regimen*||Patients completing study*†‡||By follow-up period§|
|2-4 years||>4 years|
|Median (range) age at baseline, years||15.0 (2-71)||36.0 (5-65)||17.5 (3-71)||40.0 (33-65)||–||–|
|Mean (SD)||3.8 (5.7)||6.9 (7.4)||3.6 (6.8)||5.4 (7.7)||3.0 (3.0)||6.7 (8.1)|
|Median (range)||2.3 (0-28.4)||6.3 (0-20.9)||0.5 (0-19.9)||2.7 (0-16.3)||3.0 (0-8.6)||4.8 (0-19.9)|
|*Patients could appear in >1 regimen group (prophylaxis or on-demand).
†Includes 4 patients (prophylaxis, n=2; on-demand, n=2) who had a completion visit as they had >47 months of follow-up but did not reach 48-months’ follow-up.
‡Overall follow-up for 11 patients completing study: 44.4 patient-years
§For all treatment regimens.
|n (%) of patients, n of events||Treatment regimen at time of event|
|Nasopharyngitis||6 (15.0) 8||3 (23.1) 7|
|Infections (not specified)||6 (15.0) 7||0|
|Venous device-related infection†||5 (12.5) 9||0|
|Hemarthrosis||4 (10.0) 6||1 (7.7) 1|
|Pain in extremity||2 (5.0) 2||2 (15.4) 2|
|Synovitis||4 (10.0) 5||0|
|*In ≥10% of patients receiving either regimen; patients can appear in >1 regimen group (prophylaxis or on-demand).
†Central venous catherization reported as pre-existing condition in 1 patient in the prophylaxis group (3 events) and as an adverse event in 2 patients in the prophylaxis group (2 events).
Conclusions: These real-world data further support the use of on-demand and prophylactic aPCC therapy to maintain low levels of recurrent joint bleeding in PwHI.
To cite this abstract in AMA style:Windyga J, Hermans C, Holme PA, Escuriola-Ettingshausen C, Cid AR, Khair K, Oldenburg J, Botha J, Kemenyash N, Kokot-Kierepa M, Négrier C. Real-world Joint Bleeding Rates in Patients with Congenital Hemophilia and Inhibitors: Final Data from the FEIBA Global Outcome (FEIBA GO) Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/real-world-joint-bleeding-rates-in-patients-with-congenital-hemophilia-and-inhibitors-final-data-from-the-feiba-global-outcome-feiba-go-study/. Accessed September 24, 2021.
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