Treatment for life for severe haemophilia A– A cost‐utility model for prophylaxis vs. on‐demand treatment

Farrugia, Albert; Cassar, Josephine; Kimber, Mary Clare; Bansal, Megha; Fischer, Kathelijn; Auserswald, G.; O’Mahony, Brian; Tolley, Keith; Noone, Declan; Balboni, Sonia

doi:10.1111/hae.12121

Cited by 50 publications

(88 citation statements)

References 55 publications

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“…Wszystkie dostępne obecnie dane wskazują, że nawet dotknięty ciężką postacią hemofilii pacjent ma szansę na prowadzenie normalnego trybu życia, w tym na zachowanie pełnej aktywności zawodowej, bez konieczności korzystania ze świadczeń przysługujących z tytułu niepełnosprawności [40,41].…”

Section: Dyskusjaunclassified

Ocena poziomu jakości życia chorych na hemofilię

Owsianowska

2017

Pomeranian J Life Sci

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Introduction:Haemophilia is a genetic disease. Bleeding, pain, temporary or long-term lack of physical activity, and even the risk of disability could have a negative impact on the quality of life. The aim of the paper is to evaluate the quality of life of patients with haemophilia, and to provide answers to the following questions: 1. What is the quality of life of patients with haemophilia? 2. Which sphere of life is given the highest and which is given the lowest score? 3. Is there a connection between the sociodemographic data, the health situation and the quality of life? Materials and methods: In total 163 adults diagnosed with haemophilia who are mainly treated at home and living in different parts of Poland were included in the survey. All sociodemographic data were collected using an author-designed questionnaire. The quality of life was measured using the WHOQOL-100 questionnaire proposed by World Health Organization. Results: All surveyed patients were males diagnosed with haemophilia A, which is consistent with the epidemiology of the disease and the fact that haemophilia A is more frequent in Poland than haemophilia B. Most of the males (144) were diagnosed with severe haemophilia, 18 with moderate and only 6 with mild haemophilia. What is more, 126 (75%) respondents had type C hepatitis. Conclusions:In the field of 6 life spheres measured with the use of the WHOQOL-100 questionnaire the highest score -14.66 -was gained in the sphere of spirituality, religion and personal beliefs. The lowest score -12.99 -was gained in the physical sphere, mostly concerning pain and feelings of discomfort. The outcome of 12.89 was gained in the environmental sphere. After detailed analysis of the researched material there was a conclusion made: 1. A group of men with inborn haemophilia demonstrate an average quality of life, comparable to the level of life of sick people living in other European countries. 2. The researched group give the highest estimate to the spiritual sphere of life, religion and personal beliefs, while the physical and environmental spheres were given the lowest estimate. 3. The degree of seriousness of haemophilia is of core importance when it comes to the level of life. Type C hepatitis and the status of pensioner is also a serious matter.

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Section: Dyskusjaunclassified

Ocena poziomu jakości życia chorych na hemofilię

Owsianowska

2017

Pomeranian J Life Sci

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“…Recent improvements in the treatment of hemophilia patients have brought prevention of all bleeding events and their major complications potentially within reach [23,74]. The main obstacle is represented by the high cost of care.…”

Section: Expert Commentarymentioning

confidence: 99%

“…Individualized dosing requires pharmacokinetic profiling of each patient using reliable and accurate assays to measure the plasma FVIII or IX concentration, careful dose calculation in which all available information regarding the patient is incorporated, and a wide range of CFC potencies with user-friendly infusion devices available in order to arrive at the precise dose for the patient [23,29,32,35,75,76]. Consideration of the potential barriers to adherence must be addressed.…”

Section: Expert Commentarymentioning

confidence: 99%

“…Personalized care [18,19]of hemophilia patients has been shown not only to improve long-term prognoses of patients and their quality of life, but also to reduce direct costs of treatment and indirect costs to society incurred by work/school absenteeism [20][21][22][23][24][25]. And yet personalization of treatment has not been widely and fully adopted, perhaps in part because it may not be adequately incentivized for patients, physicians, and payers, and the tools for personalization may not be available to a sufficient extent.…”

Section: Introductionmentioning

confidence: 99%

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An innovative outcome-based care and procurement model of hemophilia management

Gringeri¹,

Doralt²,

Valentino

et al. 2016

Expert Review of Pharmacoeconomics & Outcomes Research

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Hemophilia is a rare bleeding disorder associated with spontaneous and post-traumatic bleeding. Each hemophilia patient requires a personalized approach to episodic or prophylactic treatment, but selfmanagement can be challenging for patients, and avoidable bleeding may occur. Patient-tailored care may provide more effective prevention of bleeding, which in turn, may decrease the likelihood of arthropathy and associated chronic pain, missed time from school or work, and progressive loss of mobility. A strategy is presented here aiming to reduce or eliminate bleeding altogether through a holistic approach based on individual patient characteristics. In an environment of budget constraints, this approach would link procurement to patient outcome, adding incentives for all stakeholders to strive for optimal care and, ultimately, a bleed-free world.ARTICLE HISTORY

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“…However, the long-term follow-up required [7,11]; and the low number of patients available render this design infeasible for the comparison of prophylactic strategies [12]. Studies modelling patient treatment and outcome are a method used to circumvent this problem [13][14][15][16]. Our group designed a micro simulation model based on data from existing cohorts [17].…”

Section: Introductionmentioning

confidence: 99%

Modelling lifelong effects of different prophylactic treatment strategies for severe haemophilia A

2016

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Background: Lifelong prophylactic replacement therapy with clotting factor concentrates is recommended for severe haemophilia. The prophylactic dose determines both clinical outcome and treatment cost. In the absence of clinical studies, computer simulation was used to explore lifelong effects and clotting factor consumption for various prophylactic dose levels, and optimize strategies for switching between prophylactic and on-demand treatment. Design and Methods: Individual patients' lifetime joint bleeds, radiological arthropathy (Pettersson score, 0-78) and consumption were simulated for each treatment strategy. Treatment effectiveness (expressed as % of patients maintaining a lifetime Pettersson score ≤14) and clotting factor consumption were modelled for lifelong prophylaxis at dose levels 1000-4500 IU kg À1 year À1, for on-demand treatment and for switching strategies. Treatment efficiency (consumption per unit of effectiveness) was used to compare strategies. Results: Compared to lifelong on-demand treatment, lifelong prophylaxis at 1000 IU kg À1 year À1 increased effectiveness from 21 to 36%, at an additional consumption of 0.9 9 10 6 IU kg À1 . For lifelong prophylaxis, each additional 1000 IU kg À1 year À1 resulted in a proportional increase in consumption by AE5 9 10 6 IU kg À1 but a less than proportional reduction in arthropathy by AE50%; consequently, increasing consumption progressively diminished treatment efficiency. Switching strategies slightly reduce effectiveness and consumption. Optimum switching criteria were similar across prophylactic dose levels. Conclusion: According to the simulation model, low-dose prophylaxis (1000 IU kg À1 year À1) improved outcome at a limited increase in consumption compared to ondemand treatment. Increasing prophylactic dose further improved health outcomes, but at decreasing efficiency. Optimal prophylactic dose should therefore be selected balancing acceptable health impact and available budget.

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Treatment for life for severe haemophilia A– A cost‐utility model for prophylaxis vs. on‐demand treatment

Cited by 50 publications

References 55 publications

Ocena poziomu jakości życia chorych na hemofilię

Ocena poziomu jakości życia chorych na hemofilię

An innovative outcome-based care and procurement model of hemophilia management

Modelling lifelong effects of different prophylactic treatment strategies for severe haemophilia A

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