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

Miesbach, Wolfgang; Kittler, Sabine Friederike Karolin; Bauhofer, A.; Königs, Christoph; Becker, Thomas; Nemes, L.; Staus, Alexander; Schüttrumpf, Jörg

doi:10.1111/hae.13988

Cited by 8 publications

(11 citation statements)

References 35 publications

(148 reference statements)

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“…The initial literature search identified 236 unique articles, of which 91 were considered eligible for further screening (Figure 1). Twenty‐seven articles mentioned prophylaxis treatment for people with non‐severe HA, of which 15 were prospective 3,5,11–23 and 12 retrospective studies 9,24–34 (cohort studies, n = 10 3,11,13,24,26–28,32–34 ; observational studies, n = 11 12,15–18,22,23,25,29–31,34 ; cross‐sectional studies, n = 2, 5,9 other study types, n = 4 14,19–21 ). Most of the studies (19 out of 27) provided Level IV evidence (well‐designed case‐controlled or cohort studies).…”

Section: Resultsmentioning

confidence: 99%

“…The initial literature search identified 236 unique articles, of which 91 were considered eligible for further screening (Figure 1 ). Twenty‐seven articles mentioned prophylaxis treatment for people with non‐severe HA, of which 15 were prospective 3 , 5 , 11 , 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 , 21 , 22 , 23 and 12 retrospective studies 9 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 (cohort studies, n = 10 3 , 11 , 13 , 24 , 26 , 27 , 28 , 32 , 33 , 34 ; observational studies, n = 11 12 , 15 , 16 , 17 , 18 , 22 , 23 , 25 , 29 , 30 , 31 , 34 ...…”

Section: Resultsmentioning

confidence: 99%

“…Among eligible studies, treatment regimens were reported for 631/2769 (22.8%) and 676/2291 (29.5%) with mild or moderate HA, respectively. 9 , 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 , 21 , 22 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 50 A total of 550 of people with moderate HA were treated with factor replacement prophylaxis, although the dose, frequency and duration of the prophylaxis regimen was not always mentioned; the vast majority came from western Europe 3 , 9 , 13 , 16 , 17 , 18 , 24 , 50 and Australia. 28 One study reported short‐term (6–12 weeks) low‐dose prophylaxis in 66 children with HA and arthropathy, of whom 32 children had moderate disease severity.…”

Section: Resultsmentioning

confidence: 99%

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Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

et al. 2022

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Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life.Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making.Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life.Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion:There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype. K E Y W O R D Sarthropathy, bleeding, episodic/on-demand treatment, non-severe (mild or moderate) haemophilia A or B, prophylactic FVIII or FIX, women with haemophilia (symptomatic carriers)This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

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Section: Resultsmentioning

confidence: 99%

“…The initial literature search identified 236 unique articles, of which 91 were considered eligible for further screening (Figure 1 ). Twenty‐seven articles mentioned prophylaxis treatment for people with non‐severe HA, of which 15 were prospective 3 , 5 , 11 , 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 , 21 , 22 , 23 and 12 retrospective studies 9 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 (cohort studies, n = 10 3 , 11 , 13 , 24 , 26 , 27 , 28 , 32 , 33 , 34 ; observational studies, n = 11 12 , 15 , 16 , 17 , 18 , 22 , 23 , 25 , 29 , 30 , 31 , 34 ...…”

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

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Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

et al. 2022

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“…Prophylaxis can vary in terms of the duration of treatment: short-term prophylaxis (1–3 months) and long-term prophylaxis (continuous treatment). Of the studies included, 2 studies reported short-term prophylaxis [ 37 , 43 ], whereas 14 studies reported long-term prophylaxis [ 7 , 17 , 22 – 24 , 28 , 31 , 34 – 36 , 38 , 39 , 41 , 49 ]. Long-term prophylaxis resulted in better outcomes than short-term prophylaxis and also showed the need for continuing treatment at adolescence and adulthood.…”

Section: Resultsmentioning

confidence: 99%

Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review

Sun

Zhou

2021

Orphanet J Rare Dis

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Background Despite the advantages of prophylactic treatment for hemophilia, patients tend to discontinue or not adhere to it because of several challenges such as long-term use, high cost, young patients transitioning to adolescents, and switch to self-infusion or self-care. The goal of this systematic literature review is to emphasize adherence to and efficiency of prophylactic treatment in adults. Methods A literature review was conducted in PubMed, Embase, and Cochrane databases until April 2021 according to PRISMA guidelines, and the protocol was registered with PROSPERO (CRD42020220085). Studies evaluating the efficacy of prophylaxis in enhancing the quality of life were included. Results A total of 31 articles involving 2379 patients with hemophilia were included in this systematic review. Of these, 26 studies were observational, questionnaire-based studies, and 5 were randomized controlled trials. The majority of studies reported lower annualized bleeding rates in patients receiving prophylaxis compared with those receiving on-demand treatment or those who discontinued prophylaxis. Standard-dose prophylaxis was reported to be effective in most of the studies. In developing countries like China, data suggest that low doses were administered because of limited available resources. However, standard dose or individualized prophylaxis should be provided to prevent joint damage in the long term. Compared with adults, greater adherence to treatment was observed in patients aged < 16 years. Conclusion This systematic review emphasizes the importance of adherence to prophylaxis among young adults transitioning from childhood. In countries like China, low-dose prophylaxis can help in preventing joint bleeds in the short term, but in the long term, standard-dose therapy has shown high adherence among young adults and better joint health, in turn improving the quality of life.

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“…In addition, factor prophylaxis helped people with hemophilia to move freely, with little fear of trauma, and favoured a better quality of life [4,5]. Different replacement therapy regimens are currently available, and patients with hemophilia A and B may receive either prophylaxis or on-demand treatment by using different types of clotting factor concentrates [6,7]. The opportunity of an efficient prophylaxis reduces the risk of bleedings and should also reduce patients' and caregivers' fear of the noxious consequences of physical activity.…”

Section: Introductionmentioning

confidence: 99%

Promoting Sports Practice in Persons with Hemophilia: A Survey of Clinicians’ Perspective

Lassandro

Accettura

Giordano

2021

IJERPH

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Historically, people with hemophilia have been warned to avoid physical activities as a possible cause of bleeding; however, currently, sport is considered necessary, especially in the developmental age, for providing a good quality of life. A survey was proposed to a group of hematologists and sports physicians working in Puglia, Italy, to explore their approach to physical activities for their patients with hemophilia and to obtain suggestions about possible interventions to promote the access of patients to sports. The survey was answered by 6 hematologists and 15 sports physicians. In total, 71% (about six patients/year/physician) of patients with hemophilia seen by sports physicians asked for counseling about sports, and 67% (about five patients/year/physician) actually practiced sports. On the other hand, only 31% (about 16 patients/year/hematologist) of patients asked hematologists questions on sports, and only 16% (about seven patients/year/hematologist) of patients with hemophilia and that were followed-up by hematologists practiced sports. The sports most often recommended to patients with hemophilia by physicians included swimming, athletics, tennis, running and gymnastics. According to hematologists, physical activity was very efficient in improving the quality of life of patients; stability of joints; their psychological, social and musculoskeletal wellbeing; and in reducing the risk of bleedings. On the other hand, physical activity was considered less important in all these areas by sport physicians. In conclusion, answers to this survey suggested that sports could be promoted among hemophilic patients by increasing the sports physicians’ knowledge about hemophilia and their special role in this area. In addition, interviewed clinicians were of the opinion that increased awareness of specific guidelines and clinical practice protocols among both hematologists and sports physicians could be beneficial. Finally, answers suggested that access to fitness certification should be facilitated.

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

Cited by 8 publications

References 35 publications

Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review

Promoting Sports Practice in Persons with Hemophilia: A Survey of Clinicians’ Perspective

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