Assessing bleeding rates, related clinical impact and factor utilization in German hemophilia B patients treated with extended half-life rIX-FP compared to prior drug therapy

Oldenburg, Johannes; Yan, Songkai; Maro, Géraldine S.; Krishnarajah, Girishanthy; Tiede, Andreas

doi:10.1080/03007995.2019.1662675

Cited by 26 publications

(35 citation statements)

References 17 publications

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“…The improvement in bleeding outcomes with EHL factors may come at a greater cost, particularly for HA. In our study, EHL FIX resulted in reduced factor utilization, a consistent finding across studies 6‐8. On the contrary, actual factor utilization increased with EHL FVIII, the difference being more marked with Adynovate compared to Eloctate.…”

Section: Discussionsupporting

confidence: 88%

The Australian experience with switching to extended half‐life factor VIII and IX concentrates: On behalf of the Australian Haemophilia Centre Directors' Organisation

Brennan

Parikh²,

McRae

et al. 2020

Haemophilia

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Introduction Extended half‐life (EHL) factor (F) VIII and FIX concentrates became available to selected haemophilia A (HA) and haemophilia B (HB) patients in Australia in March 2018. Aim To determine factor utilization and bleeding outcomes during the first 6 months of prophylaxis with EHL concentrates, and compare it to the last 6 months of prophylaxis with standard half‐life (SHL) concentrates. Methods A national, retrospective study was performed using data extracted from the Australian Bleeding Disorders Registry (ABDR). Patients with ≥3 months of EHL exposure were analysed. Results A total of 129 HA patients (86 Adynovate, 43 Eloctate) and 64 HB (Alprolix) patients were included in the study. For HA, switching to EHL FVIII resulted in decreased injection frequency (3 to 2 per week), improved ‘reduced adherence’ rates (18% to 7%), decreased median annualized bleeding rate (ABR; 2.0 to 0.0) and increased proportion of patients with zero bleeds (44% to 64%). Actual factor utilization increased by 20 IU/kg/wk on Adynovate and 4 IU/kg/wk on Eloctate. For HB, switching to EHL FIX resulted in decreased injection frequency (2 to 1 per week), improved ‘reduced adherence’ rates (35% to 11%), decreased median ABR (3.0 to 2.0) and increased proportion of patients with zero bleeds (31% to 46%). Actual factor utilization decreased by 4 IU/kg/wk. There was no clinically significant inhibitor development. Conclusion Compared to SHL, EHL FVIII resulted in improved bleeding outcomes, albeit at the expense of increased factor utilization. EHL FIX resulted in improved bleeding outcomes despite decreased factor utilization.

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Section: Discussionsupporting

confidence: 88%

The Australian experience with switching to extended half‐life factor VIII and IX concentrates: On behalf of the Australian Haemophilia Centre Directors' Organisation

Brennan

Parikh²,

McRae

et al. 2020

Haemophilia

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“…11 In Germany, in a cohort of 59 patients who switched from SHL-FIX to Idelvion ® , there was a significant reduction of bleeding rates. 12 The mean dose of Idelvion ® used was 44.2 IU/kg which is more than double of the dose used in our cohort and across the UK.…”

Section: Discussionmentioning

confidence: 71%

A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B

et al. 2020

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Introduction Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B. Aim The low doses used in real world are approximately half of the doses used in clinical trials. We aim to assess the efficacy and safety of low dose EHL‐FIX. Methods Data from a cohort of 13 patients who were switched from standard half‐life factor IX (SHL‐FIX) to Alprolix® (mean dose 31.5 IU/kg) and seven patients who switched from standard half‐life factor IX to Idelvion® (mean dose 20.2 IU/kg) were included. Results The median annualized bleeding rate was similar for SHL‐FIX (median 3, interquartile range [IQR] 1‐5) and EHL‐FIX (median 3, IQR 1‐5.25). Quality of life scores, measured using the European Quality of Life 5 Dimensions assessment were similar for SHL‐FIX (median 0.76, IQR: 0.63‐0.84) and EHL‐FIX (median 0.79, IQR: 0.58‐0.88). Conclusion This study shows that EHL‐FIX given at low doses can be effective for prevention of bleeding for persons with severe haemophilia B.

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“…In addition to data from PROLONG-9FP studies, real-world clinical data has been collated and analyzed to determine the effectiveness, safety, and clinical response of rIX-FP in routine clinical practice. Two post-marketing studies have used retrospective patient chart data to compare bleeding rates, FIX product consumption, and dosing regimens in adult/adolescent and pediatric patients with hemophilia B who switched from prior FIX prophylaxis to rIX-FP prophylaxis [30,31].…”

Section: Clinical Experiencementioning

confidence: 99%

IDELVION: A Comprehensive Review of Clinical Trial and Real-World Data

Escobar

Mancuso

Hermans

et al. 2022

JCM

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Hemophilia B is a bleeding disorder caused by a deficiency of coagulation factor IX (FIX). Treatment with FIX replacement products can increase FIX activity levels to minimize or prevent bleeding events. However, frequent dosing with standard-acting FIX products can create a high treatment burden. Long-acting products have been developed to maintain bleed protection with extended dosing intervals. Recombinant factor IX–albumin fusion protein (rIX-FP) is a long-acting product indicated for the treatment and prophylaxis of bleeding events and perioperative management in adult and pediatric patients. This review outlines data from all previously treated patients in the Prophylaxis and On-Demand Treatment using Longer Half-Life rIX-FP (PROLONG-9FP) clinical trial program and summarizes real-world data evaluating the use of rIX-FP in routine clinical practice. In the PROLONG-9FP program, rIX-FP demonstrated effective hemostasis in all patients at dose regimens of up to 21 days in patients aged ≥ 18 years and up to 14 days in patients aged < 12 years. rIX-FP has a favorable pharmacokinetic profile and an excellent safety and tolerability profile. Extended dosing intervals with rIX-FP led to high levels of adherence and reduced consumption compared with other FIX therapies. Data from real-world practice are encouraging and reflect the results of the clinical trials.

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Assessing bleeding rates, related clinical impact and factor utilization in German hemophilia B patients treated with extended half-life rIX-FP compared to prior drug therapy

Abstract: Tiede (2020) Assessing bleeding rates, related clinical impact and factor utilization in German hemophilia B patients treated with extended half-life rIX-FP compared to prior drug therapy,

Cited by 26 publications

References 17 publications

The Australian experience with switching to extended half‐life factor VIII and IX concentrates: On behalf of the Australian Haemophilia Centre Directors' Organisation

The Australian experience with switching to extended half‐life factor VIII and IX concentrates: On behalf of the Australian Haemophilia Centre Directors' Organisation

A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B

IDELVION: A Comprehensive Review of Clinical Trial and Real-World Data

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