Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Malec, Lynn; Matino, Davide

doi:10.1111/hae.14866

Cited by 8 publications

(2 citation statements)

References 61 publications

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“…This is true also for all the upcoming new generation of therapeutic molecules, including the ultra‐long FVIII molecule efanesoctocog alfa and improved FVIII mimetics that should overcome some of the limitations of current similar treatment options greatly raising the bar of protection into the non‐hemophilic range 44 …”

Section: New Therapeutic Goals and Related Outcomesmentioning

confidence: 97%

Benefits and risks of non‐factor therapies: Redefining haemophilia treatment goals in the era of new technologies

Mancuso,

Croteau,

Klamroth

2024

Haemophilia

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IntroductionOver the last decades progress in haemophilia treatment has been remarkable and prophylaxis with clotting factor concentrates in haemophilia A and B has been established as the standard of care in individuals with haemophilia and a severe bleeding phenotype. Besides clotting factor products with prolonged half‐life non‐factor therapies were developed which enable prophylaxis via subcutaneous administration. Factor VIIIa mimetics like emicizumab facilitate the coagulation pathway and are used in routine clinical practice for indivdiduals with haemophilia A. Rebalancing therapeutic agents like fitusiran, concizumab, marstacimab and serpin PC block the anticoagulant pathway and clinical trials using these products in individuals with haemophilia A and B are ongoing.Aim and MethodsA narrative review to asess the benefits and risks of non‐factor therapies taking in to account re‐defined haemophilia treatment goals.ResultsProphylaxis for prevention of bleeds using non‐factor products by subcutaneous administration is effective and results in reductions of bleeding episodes in individuals with haemophilia A or B with and without inhibitors. The treatment with emicizumab showed tolerable safety both in clinical trials and long‐term real‐world observations with few thrombotic events. In some clinical trials with rebalancing therapies (fitusiran and concizumab) thrombotic events occurred. Monitoring of the haemostatic function of novel therapies especially with concomitant haemostatic treatment is not yet established.ConclusionWith the advent of novel therapeutic agents including factor concentrates with ultra‐long half‐life and improved FVIIIa mimetics aimed at raising the bar of protection into the non‐hemophilic range redefinition of haemophilia treatment goals is eagerly needed.

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Section: New Therapeutic Goals and Related Outcomesmentioning

confidence: 97%

Benefits and risks of non‐factor therapies: Redefining haemophilia treatment goals in the era of new technologies

Mancuso,

Croteau,

Klamroth

2024

Haemophilia

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“…Data have emerged to suggest that a factor trough level of 1 IU/dL is insufficient to prevent bleeds in individuals with severe hemophilia, particularly those with higher levels of activity. 45,46 Thus, by extrapolation, many individuals with moderate hemophilia may also benefit from regular prophylaxis. 4 There is also emerging evidence that prophylactic therapy in patients with severe hemophilia may result in better musculoskeletal outcomes compared with some patients with moderate disease receiving episodic treatment.…”

Section: On-demand Treatment and Home Therapymentioning

confidence: 99%

Nonsevere Hemophilia: The Need for a Renewed Focus and Improved Outcomes

Dolan,

Fijnvandraat,

Lenting

et al. 2024

Semin Thromb Hemost

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People with nonsevere hemophilia (PWNSH) are phenotypically more diverse than those with severe hemophilia. Perceptions relating to a “nonsevere” phenotype have contributed to fewer research initiatives, fewer guidelines on optimal management, and a lack of standards for surveillance and clinical assessment for affected individuals. In many cases, episodes of abnormal bleeding could, if investigated, have led to earlier diagnosis. Furthermore, the major recent developments in therapy for hemophilia have largely focused on severe disease and, as a group, PWNSH have not been included in many key clinical trials. Benefiting people with severe disease, innovative replacement therapies have generally targeted factor levels that are above those present in a large proportion of PWNSH. Therapeutic advances can lead to improvement in phenotype for people with severe hemophilia over that currently experienced by many PWNSH. As a result, we are approaching a point where PWNSH may, in many countries, have a higher risk of bleeding and restriction in lifestyle than those with severe disease but with more limited therapeutic options. Given the multiple major advances in treatment for people with hemophilia, it is timely to review the aspects of nonsevere disease, to ensure equity in care and management for all individuals with this condition.

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The good, the bad and the ugly of pain in haemophilia: Recent evidence on the epidemiology, molecular mechanisms and knowledge gaps preventing optimal treatment

Benemei,

Mattia,

Di Minno

2024

Haemophilia

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IntroductionHaemophilia is an inherited, X‐linked blood clotting disorder caused by the deficiency of coagulation factors VIII (FVIII, haemophilia A) or IX (FIX, haemophilia B). Spontaneous bleeds are common in severe forms of haemophilia and can also occur in moderate and mild haemophilia. Severe or repeated bleeding at a joint can evolve into chronic haemophilic arthropathy, with functional damage of the joint, disability, and intense chronic articular pain. Nonetheless, acute and chronic pain may emerge due to secondary conditions related to bleedings.AimThis narrative review aims to critically discuss the most recent evidence about pain in haemophilia to give healthcare professionals a clear picture of current knowledge hence favouring the optimisation of clinical management of pain.MethodsExtensive literature search with the terms ‘hemophilia’ AND ‘pain’, focusing on the time window 2021–2023.ResultsAcute and chronic pain is a critical aspect of haemophilia at all ages. It should be considered a multifaceted phenomenon, with a positive role as an early emergency signal of a clinical event (haemarthrosis), and numerous detrimental aspects linked to its burden that heavily affects the health‐related quality of life, with psychological and social consequences.ConclusionDespite its prevalence and frequency in people with haemophilia, pain is often underestimated by healthcare professionals, leading to insufficient and inadequate treatment, also due to uncertainty linked to the presence of the coagulation disorder or arthritic flares.

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Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Cited by 8 publications

References 61 publications

Benefits and risks of non‐factor therapies: Redefining haemophilia treatment goals in the era of new technologies

Benefits and risks of non‐factor therapies: Redefining haemophilia treatment goals in the era of new technologies

Nonsevere Hemophilia: The Need for a Renewed Focus and Improved Outcomes

The good, the bad and the ugly of pain in haemophilia: Recent evidence on the epidemiology, molecular mechanisms and knowledge gaps preventing optimal treatment

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