From the voices of people with haemophilia A and their caregivers: Challenges with current treatment, their impact on quality of life and desired improvements in future therapies

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Abstract

Introduction

Haemophilia A is a chronic disease requiring frequent intravenous infusions of recombinant factor VIII. Previous studies have shown that challenges associated with current treatments may have significant impacts on quality of life (QoL) that are as important as the health outcomes conferred by the therapy. Emerging therapeutic innovations offer the potential to mitigate treatment‐related challenges, and it is therefore important to develop a better understanding of patient and caregiver experiences with existing haemophilia A treatments in order to characterize the full value of new treatments.

Aim

To gather firsthand perspectives from people with haemophilia A (PWHA) and caregivers on the challenges with current treatment, their impact on QoL and desired improvements in future therapies.

Methods

Qualitative insights were gathered from 20 non‐inhibitor PWHA or caregivers of PWHA across Canada through one‐on‐one interviews; insights were further explored through focus group sessions to uncover overarching themes and prioritize issues with current treatments.

Results

PWHA and caregivers identified several challenges, including administration of intravenous infusions, coordination of treatment schedules and ensuring adequate medication and supplies. Participants described how these challenges impact psychosocial well‐being, physical health, personal/social life and work. Alternate modes of administration and longer‐lasting treatment effects were identified as desired improvements over current treatments.

Conclusion

This study emphasizes the impact that existing haemophilia A treatments have on psychological well‐being, employment opportunities and adherence to treatment regimens. These considerations may help to inform decision‐making for policymakers and health systems around the true value of new therapies entering the haemophilia market.

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Most cited references 19

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A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study).

A Gringeri, , S Mackensen … (2011)

Prevention of arthropathy is a major goal of hemophilia treatment. While studies in adults have demonstrated an impact of prophylaxis on the incidence of joint bleeds and patients' well-being in terms of improved quality of life (QoL), it is unclear whether or not prophylaxis influences the outcome and perception of well- of children with hemophilia. This randomized controlled study compared the efficacy of prophylaxis with episodic therapy in preventing hemarthroses and image-proven joint damage in children with severe hemophilia A (factor VIII <1%) over a 10-year time period. Forty-five children with severe hemophilia A, aged 1-7 years (median 4), with negative clinical-radiologic joint score at entry and at least one bleed during the previous 6 months, were consecutively randomized to prophylaxis with recombinant factor VIII (25 IU kg(-1) 3 × week) or episodic therapy with ≥25 IU kg(-1) every 12-24 h until complete clinical bleeding resolution. Safety, feasibility, direct costs and QoL were also evaluated. Twenty-one children were assigned to prophylaxis, 19 to episodic treatment. Children on prophylaxis had fewer hemarthroses than children on episodic therapy: 0.20 vs. 0.52 events per patient per month (P < 0.02). Plain-film radiology showed signs of arthropathy in six patients on prophylaxis (29%) vs. 14 on episodic treatment (74%) (P < 0.05). Prophylaxis was more effective when started early (≤36 months), with patients having fewer joint bleeds (0.12 joint bleeds per patient per month) and no radiologic signs of arthropathy. This randomized trial confirms the efficacy of prophylaxis in preventing bleeds and arthropathy in children with hemophilia, particularly when it is initiated early in life. © 2011 International Society on Thrombosis and Haemostasis.

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Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A.

S BJÖRKMAN, Emilie S. Fritsch, … (2009)

The role of prophylactic factor VIII (FVIII) to decrease hemophilic bleeding and arthropathy is well established. The rationale for this strategy is to convert patients with severe hemophilia A to a moderate clinical phenotype by reducing time spent with a FVIII level <1 IU dL(-1). Studies to date, however, have not demonstrated a strong link between FVIII level and the bleeding rate. To assess the effect of FVIII level on break-through bleeding in patients with severe hemophilia A on prophylaxis. This study analysed data from 44 patients aged 1-6 and 99 patients aged 10-65 years with severe hemophilia A (FVIII <1 IU dL(-1)) who were treated with prophylactic FVIII as part of clinical studies assessing pharmacokinetics, safety and efficacy of a recombinant FVIII (Advate). Each patient had pharmacokinetic measurements and FVIII infusions recorded, and these were used to calculate time spent with a FVIII below 1, 2 and 5 IU dL(-1). The data demonstrate that increasing time with a FVIII below 1 IU dL(-1) is associated with increased total bleeds and hemarthroses. Lack of adherence to the intended frequency of FVIII infusion was the most important determinant of low FVIII and increased bleeding. In children aged 1-6 years, the rate of bleeding was also influenced by FVIII half-life and clearance. These data have important implications for the management of patients with severe hemophilia.

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Barriers to compliance with prophylaxis therapy in haemophilia.

Philip J M Johnson, S Geraghty, Beth M Hacker (2001)

Prophylaxis, or the practice of routine replacement infusions of clotting factor concentrate in persons with severe haemophilia, is a demanding medical regimen. Prophylactic infusions require direct venepuncture or sterile entry into a central venous access device on a regular basis. A telephone survey was conducted to elicit information regarding the barriers to compliance with prophylaxis. The Mountain States Regional Haemophilia and Thrombosis Center has recommended prophylaxis to 52 male patients with haemophilia A or B. The haemophilia nurse attempted to contact all of these patients or their parents, and contact was made with 38 (73.1%) of them. Respondents were asked about the following issues: their decision to initiate prophylaxis; their self-rated compliance; the challenges, barriers, and facilitators of prophylaxis; and their perceived value of the therapy. Four patients (10.5%) elected not to begin prophylaxis. Of the 34 persons who began prophylaxis, 20 respondents (58.8%) rated their compliance as excellent. Nearly one-third of the families with excellent compliance (giving 75-100% of prescribed infusions) stated that the time-consuming nature of prophylaxis was the most significant challenge of the regimen. In addition, 58.3% of the families that gave less than the prescribed number of infusions reported that the time commitment was the primary reason for missing infusions. Knowledge of the benefits of prophylaxis was the primary facilitator of compliance for 44.1% of families. Ninety-seven percent of respondents rated prophylaxis as very valuable. These data show that despite the known benefits of prophylaxis, it is a demanding medical regimen, and compliance is imperfect. In addition, this study underscores the importance of providing continuing support and education for patients and families who are implementing prophylaxis.

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Author and article information

Contributors

Jayson M. Stoffman: jstoffman@cancercare.mb.ca

Journal

Journal ID (nlm-ta): Haemophilia

Journal ID (iso-abbrev): Haemophilia

Journal ID (doi): 10.1111/(ISSN)1365-2516

Journal ID (publisher-id): HAE

Title: Haemophilia

Publisher: John Wiley and Sons Inc. (Hoboken )

ISSN (Print): 1351-8216

ISSN (Electronic): 1365-2516

Publication date (Electronic): 23 April 2019

Publication date (Print): May 2019

Volume: 25

Issue: 3 ( doiID: 10.1111/hae.2019.25.issue-3 )

Pages: 433-440

Affiliations

[ ¹ ] Shift Health Toronto Ontario Canada

[ ² ] Canadian Hemophilia Society Montreal Quebec Canada

[ ³ ] Health Sciences North Children's Treatment Centre Sudbury Ontario Canada

[ ⁴ ] London Health Sciences Centre London Ontario Canada

[ ⁵ ] Children's Hospital London Health Sciences Centre London Ontario Canada

[ ⁶ ] Pediatrics and Child Health University of Manitoba Winnipeg Manitoba Canada

Author notes

[*] [* ] Correspondence

Jayson M. Stoffman, University of Manitoba, Pediatrics and Child Health, Winnipeg, Manitoba, Canada.

Email: jstoffman@ 123456cancercare.mb.ca

Article

Publisher ID: HAE13754

DOI: 10.1111/hae.13754

PMC ID: 6850753

PubMed ID: 31016823

SO-VID: eb6a3340-4652-4743-a970-efa5255a17b5

License:

This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.

History

Date received : 17 August 2018

Date revision received : 14 March 2019

Date accepted : 15 March 2019

Page count

Figures: 0, Tables: 3, Pages: 8, Words: 5084

Funding

Funded by: Hoffman‐La Roche Limited (Canada)

Custom metadata

source-schema-version-number 2.0

cover-date May 2019

details-of-publishers-convertor Converter:WILEY_ML3GV2_TO_JATSPMC version:5.7.1 mode:remove_FC converted:12.11.2019

ScienceOpen disciplines: Hematology

Keywords: adherence,employment,haemophilia,psychological well‐being,quality of life,treatment

Data availability:

ScienceOpen disciplines: Hematology

Keywords: adherence, employment, haemophilia, psychological well‐being, quality of life, treatment

From the voices of people with haemophilia A and their caregivers: Challenges with current treatment, their impact on quality of life and desired improvements in future therapies

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Abstract

Introduction

Aim

Methods

Results

Conclusion

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Clinical Social Work and Health Intervention

Most cited references 19

A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study).

Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A.

Barriers to compliance with prophylaxis therapy in haemophilia.

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Contributors

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