Long‐term outcomes from prophylactic or episodic treatment of haemophilia A: A systematic review

O’Hara, Jamie; Sima, Camelia S.; Frimpter, Jeffrey; Paliargues, F.; Chu, Paula; Presch, I.

doi:10.1111/hae.13546

Cited by 20 publications

(33 citation statements)

References 36 publications

(78 reference statements)

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“…Therefore, this treatment should be continued lifelong. 1,4,9,[13][14][15][16][17][18][19][20][21][22][23][24][25][34][35][36] However, data on adult patients benefitting from prophylaxis are limited.…”

Section: Discussionmentioning

confidence: 99%

“…recommendation and strengthen comparable results of previous studies revealing the superior benefit of prophylaxis compared to on-demand treatment in adolescents and adults with HA. 9,[13][14][15][16][17][18][19][20][21][22][23][24][25][26]34 This prospective study is the longest NIS of a single FVIII concentrate reported so far in which treatment data were collected from the routine treatment of HA patients. 32 In a broad range of patients with severe as well as non-severe HA, covering all age groups up to 88 years, long-term prophylaxis with a pdFVIII concentrate reduced the ABR remarkably, in some cases down to zero.…”

Section: Ta B L E 2 (Continued)mentioning

confidence: 99%

“…7,31,38 These data, which the present report may contribute to, will help to improve HA therapy and the quality of life of patients with HA. 22 Beside non-factor treatment options such as emicizumab, 39 prophylaxis with FVIII concentrates is still widely used to provide prophylaxis and treat HA patients to attain low ABRs or even a bleed-free life.…”

Section: Ta B L E 2 (Continued)mentioning

confidence: 99%

“…These studies have revealed the benefit of (adherent) prophylaxis over on-demand treatment in adolescents and adults with HA. 9,[13][14][15][16][17][18][19][20][21][22][23][24][25][26][27][28][29] However, less information is available about the benefit of tertiary prophylaxis for reducing the annual bleeding rate (ABR) and annual joint bleeding rate. Furthermore, although follow-up documentation is limited, especially from real-life settings, opinion is shifting towards recommending lifelong prophylaxis.…”

Section: Introductionmentioning

confidence: 99%

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Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A

et al. 2020

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Introduction:Prophylaxis with factor VIII (FVIII) concentrates in children with haemophilia A (HA) is current standard of care. The benefit of prophylactic treatment for adult HA patients is not commonly accepted. Aim:To investigate the benefit of prophylaxis over on-demand treatment in adult and elderly patients with severe or non-severe HA in a real-life setting. Methods: Data from 163 patients comprising 1202 patient-years were evaluated for 7.5 (±5.3) years. The effects on the annual bleeding rate (ABR, including spontaneous and traumatic bleeds) of treatment with a plasma-derived FVIII concentrate, the patient's age and disease severity were investigated. The effect of changing the treatment from on demand to continuous prophylaxis on the patients' ABRs was further analysed. Results: Prophylaxis had the greatest effect on the ABRs of patients of any age with severe or non-severe HA. The difference in ABR of all patients treated on demand (median 31.4; interquartile range (IQR) 27.6; N = 83) compared with those treated prophylactically (median 1.3; IQR 3.6; N = 122) was statistically significant (P < .05), even for patients with non-severe HA (median 8.4; IQR 15.5; N = 11) vs median 1.5; IQR 4.2 (N = 17), P < .05). Patients, aged up to 88 years, switching from on demand to continuous prophylaxis showed the lowest median ABR (1.1; N = 51) after their regimen change.Conclusion: Any (even low-frequency) prophylaxis results in lower ABR than on-demand treatment. Patients switching to prophylaxis benefitted the most, irrespective of age or HA severity. Prophylactic treatment-even tertiary-is the regimen of choice for patients of any age, including elderly patients, with severe or non-severe HA. K E Y W O R D Sadult and elderly patients, annual bleeding rate, effectiveness, haemophilia A, plasma-derived factor VIII concentrate, prophylaxis

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Section: Discussionmentioning

confidence: 99%

Section: Ta B L E 2 (Continued)mentioning

confidence: 99%

Section: Ta B L E 2 (Continued)mentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A

et al. 2020

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“…3 Cross-sectional data on health-related quality of life and other patient-reported outcomes (PROs) in clinical trials in haemophilia are available. 4 However, there is a lack of real-world data exploring the relationships between longitudinal changes of PROs, clinical outcomes and treatment patterns/products across different countries.…”

Section: Introductionmentioning

confidence: 99%

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company

et al. 2020

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Introduction: Real-world data are lacking regarding the relationship between prospectively collected patient-reported outcomes (PROs), clinical outcomes and treatment in people with haemophilia (PWH). The Expanding Communications on Hemophilia A Outcomes (ECHO) registry was designed to address this data gap, but a range of difficulties led to early study closure. Aim: To describe the challenges faced and lessons learned from implementing a multinational haemophilia registry. Methods: The Expanding Communications on Hemophilia A Outcomes was planned as a five-year observational cohort study to collect data from 2000 patients in nine countries. Based on direct observations, feedback from patients enrolled in ECHO, challenges of the study design and input from study-sponsor representatives, the ECHO Steering Committee systematically identified the challenges faced and developed recommendations for overcoming or avoiding them in future studies. Results: The study closed after two years because few countries were activated and patient recruitment was low. This was related to multiple challenges including delayed implementation, stringent pharmacovigilance requirements, objections of investigators and patients to the burden of multiple PROs, data collection issues, lack of resources at study sites, little engagement of patients and competing clinical trials, which further limited recruitment. At study closure, 269 patients had been enrolled in four of nine participating countries. Conclusions: Researchers planning studies similar to ECHO may want to consider the barriers identified in this global registry of PWH and suggestions to mitigate these limitations, such as greater patient involvement in design and analysis, clearer assessment and understanding of local infrastructure and potential changes to the administration of the study.

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The Evolution of Hemophilia Care: Clinical and Laboratory Advances, Opportunities, and Challenges

2020

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Hemophilia A (HA) and B (HB) are X-linked bleeding disorders caused by mutations in the F8 or F9 gene that result in the absence, or reduced activity, of the corresponding clotting factor. The severity of bleeding and related complications is proportional to the amount of residual circulating functional factor. The development of a safe and effective hemophilia treatment lasted several decades and has been mainly based on clotting factor replacement. Advances in the engineering and manufacturing of clotting concentrates have led to the widespread availability of extended half-life products that reduced the number of intravenous infusions needed to achieve adequate trough levels. The recent development of new nonfactor replacement treatments and biotechnology techniques has offered therapeutic alternatives for hemophilia patients with and without inhibitors. These are characterized by an easier route of administration, low immunogenicity, and, regarding gene therapy and cell-based treatments, potential long-term protection from bleeding after a single treatment course. In this review, we analyze recent progresses in the management of hemophilia and discuss opportunities and challenges.

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Long‐term outcomes from prophylactic or episodic treatment of haemophilia A: A systematic review

Cited by 20 publications

References 36 publications

Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A

Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company

The Evolution of Hemophilia Care: Clinical and Laboratory Advances, Opportunities, and Challenges

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