Treatment adherence in hemophilia

Thornburg, Courtney D.; Duncan, Natalie

doi:10.2147/ppa.s139851

Cited by 138 publications

(185 citation statements)

References 73 publications

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“…Patients with severe haemophilia who do not receive prophylaxis usually have 30‐35 joint bleedings a year, and they are at high risk of developing severe arthropathy. With increasing severity and number of affected joints, BMD significantly decreases . In our study, thirty five patients (70%) were on low‐dose prophylactic factor VIII therapy with a dose of 15 U/kg twice weekly.…”

Section: Discussionmentioning

confidence: 99%

Effect of low‐dose factor VIII prophylaxis therapy on bone mineral density and 25(OH) vitamin D level in children with severe haemophilia A

et al. 2019

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Background Decreased bone mineral density (BMD) is a significant morbidity in haemophilia. Vitamin D is important for the bone health of people with haemophilia. Regular factor VIII prophylaxis can prevent bleeding and arthropathy. Aim To determine the 25(OH) vitamin D level in severe haemophilia A patients and correlate it to their Hemophilia Joint Health Score (HJHS) and dual‐energy X‐ray absorptiometry (DEXA). We also compared the 25(OH) vitamin D and DEXA in haemophilia A and healthy children and in haemophilia A children on prophylaxis versus on‐demand therapy. Methods Fifty severe haemophilia A patients were compared to 50 age‐matched healthy boys. Patients were recruited from the Pediatric Hematology Clinic, Ain Shams University from May 2017 to April 2018. Full medical history was taken with emphasis on frequency of bleeding episodes, duration and amplitude of pain assessed by the pain score. Weight, height, body mass index and HJHS were assessed. 25(OH) vit‐D3, calcium, phosphorus and alkaline phosphatase were measured. BMD was assessed using Lunar DEXA, paediatric software. Results People with haemophilia had significantly lower 25(OH) vit‐D3 (P < .001) and DEXA z‐score (P < .001) than controls. Seventy per cent of patients were on factor VIII prophylaxis twice weekly (15U/kg/dose). Significant difference was found regarding DEXA z‐score (P = .012), 25(OH) vit‐D3 (P = .033) and HJHS (P = .022) among patients on prophylaxis and on‐demand therapy. Conclusion Severe haemophilia A patients showed significantly lower 25(OH) vit‐D3 and DEXA than controls. Hence, vitamin D deficiency should be tested in all people with haemophilia for early diagnosis and treatment. Low‐dose prophylaxis in severe haemophilia preserves BMD and increases vitamin D. Further studies are required to evaluate the effect of different prophylaxis protocols on BMD and haemophilic arthropathy.

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Section: Discussionmentioning

confidence: 99%

Effect of low‐dose factor VIII prophylaxis therapy on bone mineral density and 25(OH) vitamin D level in children with severe haemophilia A

et al. 2019

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“…9 Our results, based on the perspectives of patients, suggest that visualization techniques could be a helpful patient-centred care strategy. 17,18 In another study, 19 Several decision aids have been developed for haemophilia. A previous qualitative study demonstrated that a better understanding determines the ability to practice prophylaxis.…”

Section: Discussionmentioning

confidence: 99%

“…Indeed, a good relationship between care providers and PWH has been found to be associated with treatment adherence in haemophilia 17,18. This resulted in increased patient quality of life (QoL) and enhanced illness perception and stabilization of joint health after 1 year.…”

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confidence: 99%

Patient‐centred care in haemophilia: Patient perspectives on visualization and participation in decision‐making

et al. 2019

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Introduction and Aim The British Columbia Adult Haemophilia Team recently adopted a patient‐centred care approach. The team presented visual information on an individual's pharmacokinetic profile and bleed history and encouraged patients to participate in treatment decisions. This qualitative study explored how this approach changed patients’ understanding of haemophilia and how it facilitated them to make treatment decisions. Methods We interviewed 18 males with mild, moderate or severe haemophilia, using a convenience sample from the adult haemophilia clinic at St. Paul's hospital in Vancouver, Canada. Interviews were recorded and transcribed verbatim and analyzed using descriptive content analysis. Results Most participants reported that reviewing visual information with the Clinic Team helped them in their communication with their care providers during their annual review clinic appointment. Despite this improved communication, for some the most important feature of their treatment was that they had switched from on‐demand treatment to prophylactic treatment in recent years and were able to prevent bleeds. Almost half of the participants reported that the visual information presented increased their understanding of haemophilia and the pharmacokinetics of coagulation factor. Three patients improved their treatment adherence or had changed their prophylaxis schedules based on this. Most participants felt that they were involved in decision‐making about their treatment schedule, which they appreciated. On the other hand, two participants thought the Clinic Team should make these decisions. Conclusion Participants perceived the patient‐centred prophylaxis approach helpful because it enhanced communication with the Clinic Team, increased their understanding of haemophilia and pharmacokinetics of coagulation factor and facilitated treatment decisions.

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“…Among the barriers to achieving optimal outcomes is adherence to the FVIII dosing regimen which mandates frequent, intravenous injections of concentrates of FVIII and achieving circulating FVIII levels sufficient to prevent bleeding including with physical activity …”

Section: Introductionmentioning

confidence: 99%

Emicizumab: Review of the literature and critical appraisal

Rodríguez-Merchán¹,

Valentino

2018

Haemophilia

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Introduction Emicizumab‐kywh (ACE910) is a recombinant, humanized, asymmetric bispecific antibody that functions to bring activated FIX (FIXa) and zymogen FX into an appropriate steric conformation to medicate the activation of FX to FXa thereby mimicking the cofactor function of FVIIIa. Aim The objective of this manuscript was to review the development and potential role for emicizumab in the treatment of patients with haemophilia A with and without inhibitors. Methods A Cochrane Library and PubMed (MEDLINE) search focusing on emicizumab in haemophilia was conducted. Results In total, 37 citations were retrieved and serve as the database for the literature reviewed herein. Once‐weekly subcutaneous injection of emicizumab at three dose levels has been shown to be effective as prophylaxis to prevent bleeding in a majority haemophilia A patients with inhibitors to FVIII. Likewise, prevention of bleeding was also observed in more than two thirds of patients without inhibitors to FVIII. One antidrug antibody to emicizumab has been reported in over 600 treated patients, two have developed thromboembolic events and three thrombotic microangiopathy. These thrombotic complications have occurred in conjunction with FVIII‐bypassing agents, and none have been observed following recommendations from the manufacturer regarding concomitant use of bypassing agents. The median annual treated bleeding rates were decreased in patients with as well as those without an inhibitor to FVIII. Conclusion The principal advantage of emicizumab is subcutaneous administration and effectiveness irrespective of the presence of inhibitors. Emicizumab could conceivably represent a new epoch in the treatment of people with haemophilia A.

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Treatment adherence in hemophilia

Cited by 138 publications

References 73 publications

Effect of low‐dose factor VIII prophylaxis therapy on bone mineral density and 25(OH) vitamin D level in children with severe haemophilia A

Effect of low‐dose factor VIII prophylaxis therapy on bone mineral density and 25(OH) vitamin D level in children with severe haemophilia A

Patient‐centred care in haemophilia: Patient perspectives on visualization and participation in decision‐making

Emicizumab: Review of the literature and critical appraisal

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