Hamostaseologie 2025; 45(S 01): S77-S78
DOI: 10.1055/s-0044-1801660
Abstracts
Topics
T-09 Innovation and Novelties

Aspects of long-term joint health in haemophilia A: inSight Joint Health tool

Authors

  • G Goldmann

    1   Universitätsklinikum Bonn, Institut f. Exper. Hämatologie und Transfusionsmedizin, Bonn, Germany

  • N Marquardt

    1   Universitätsklinikum Bonn, Institut f. Exper. Hämatologie und Transfusionsmedizin, Bonn, Germany

  • A C Strauss

    2   Universitätsklinikum Bonn, Klinik und Poliklinik für Orthopädie und Unfallchirurgie, Bonn, Germany

  • J Oldenburg

    1   Universitätsklinikum Bonn, Institut f. Exper. Hämatologie und Transfusionsmedizin, Bonn, Germany
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Introduction: The management of hemophilia A has undergone a revolution. Improving patients`compliance with prophylaxis has become a key focus [1.] Although treatment standards are high, daily practice reveals variable outcomes: Patients are still bleeding, subclinical bleeds occur despite current prophylaxis, which still needs to be optimized [2] [3]. There has been evidence, trough that levels of 3% factor VIII (FVIII) are not adequate to fully prevent synovitis in all patients [4]. Unmet needs remain, joint and functional impairment and acute and chronic pain occur. Evidence suggests, that FVIII activity levels of up to 50% may be needed to achieve a near-zero joint bleed rate [5]. The German S2k guidelines on synovitis recommend maintaining factor trough levels of≥30% for 6 months for the treatment of chronic synovitis. One key to improve the prophylaxis and its outcome in the real world setting is an educated, committed and adherent patient [6] [7].

Method: To raise awareness for joint health and support communication between physicians and their patients, an innovative tool ​has been developed. Using innovative technology, the inSight Joint Health tool lets users explore immersive 3D models, detailed animated videos, medical scan images and informative messaging about joint health in haemophilia.​ The tool explains the pathophysiology behind subclinical and clinical bleeds and provides guidance on how long-term joint health can be preserved. It can be applied to support patient education and can help to support the shared decision making process in haemophilia treatment.

Results: The inSight Joint Health tool enables to communicate how both, clinical and subclinical bleeds, can contribute to joint damage​ and highlight that an optimized prophylactic therapy supports long-term joint health​. A step forward in the treatment of hemophilia A has recently been achieved with a new class of factor VIII concentrate, the high-sustained FVIII: Efanesoctocog alfa is a B domain-deleted single-chain FVIII connected to the D'D3 domain of von Willebrand Factor (vWF). Efanesoctocog alfa provides a 3-4 times longer half-life compared to standard and extended half-life FVIII products [8] [9] [10].

Conclusion: Achieving high FVIII activity levels with current standard and extended half-life FVIII replacement therapies is associated with a high treatment burden. Innovative treatment options may provide high sustained FVIII activity levels and improve patient outcomes [7]. Application of the tool might support haemophilia-specific acceptance and commitment to a prophylactic treatment. Physicians and patients evaluated the inSight Joint Health tool as beneficial tool,supporting shared decision making and enhancing the adherence to a prophylactic therapy regimen contributing to long-term joint health.


 

Conflict of Interest:

GG: Payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from SOBI, Takeda, Bayer, Octapharma, Novo Nordisk, Biotest and Roche; support for attending meetings and/or travel from Novo Nordisk, Biotest and Swedish Orphan Biovitrum SOBI. NM: Speaker/consultant and grant/research support from Baxalta, Bayer, Chugai, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Roche, Shire/Takeda, SOBI. JO: Research Support/P.I. from Bayer, Biotest, Chugai, CSL-Behring, Novo Nordisk, Octapharma, Pfizer, Roche, SOBI, Takeda; Consultant for Bayer, Biogen Idec, Biomarin, Biotest, Chugai, CSL-Behring, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sparks, SOBI, Takeda; Speakers Bureau for Bayer, Biogen Idec, Biomarin, Biotest, Chugai, CSL-Behring, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sparks, SOBI, Takeda; Scientific Advisory BoardBayer, Biogen Idec, Biomarin, Biotest, Chugai, CSL-Behring, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sparks, Takeda, SOBI. NM: Speaker/consultant and grant/research support from Baxalta, Bayer, Chugai, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Roche, Shire/Takeda, SOBI. ACS: Research Support/P.I. from Bayer, Chugai , Roche, SOBI, Takeda; Consultant for Bayer, Biotest, SOBI, Takeda; Speakers Bureau for Bayer, SOBI, Takeda. JO: Research Support/P.I. from Bayer, Biotest, Chugai, CSL-Behring, Novo Nordisk, Octapharma, Pfizer, Roche, SOBI, Takeda; Consultant for Bayer, Biogen Idec, Biomarin, Biotest, Chugai, CSL-Behring, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sparks, SOBI, Takeda; Speakers Bureau for Bayer, Biogen Idec, Biomarin, Biotest, Chugai, CSL-Behring, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sparks, SOBI, Takeda; Scientific Advisory BoardBayer, Biogen Idec, Biomarin, Biotest, Chugai, CSL-Behring, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sparks, Takeda, SOBI.


Publication History

Article published online:
13 February 2025

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