Rationale for individualizing haemophilia care

Abstract

Owing to the heterogeneity in the clinical phenotype of haemophilia A and B, it is now recognized that disease severity (based on factor VIII/IX activity) may no longer be the most appropriate guide for treatment and that a 'one-size-fits-all' approach is unlikely to achieve optimal therapy. Based on the present literature and consensus views of a group of experts in the field, this article highlights key gaps in the understanding of the diverse relationships between bleeding phenotype and factors such as joint health, genetic susceptibility, laboratory parameters, quality of life and management of pain. Early prophylaxis is a potential 'gold standard' therapy and issues surrounding inhibitor development, variations in its clinical use and long-term outcomes are discussed. Comprehensive treatment should be individualized for all patients (including those with mild or moderate haemophilia and carriers). Wherever possible all patients should be given prophylaxis. However, adult patients with a milder haemophilia phenotype may be candidates for ceasing prophylaxis and switching to on-demand treatment. Regardless, all treatment (on-demand and prophylaxis) should be tailored towards both the patient's personal needs and their clinical profile. In addition, as the associations between risk factors (psychosocial, condition-related and treatment-related) and clinical features are unique to each patient, an individualized approach is required to enable patients to alter their behaviour in response to them. The practical methodologies needed to reach this goal of individualized haemophilia care, and the health economic implications of this strategy, are ongoing topics for discussion.

Defining patient value in haemophilia care

“…Can and should haemophilia be defined as a single medical condition? Medically, it is clearly defined as factor VIII or factor IX levels below 40 IU/dL, but outcomes and subsequent clinical management are much more heterogeneous: 4…”

“…Can and should haemophilia be defined as a single medical condition? Medically, it is clearly defined as factor VIII or factor IX levels below 40 IU/dL, but outcomes and subsequent clinical management are much more heterogeneous: functional outcomes and QoL are perhaps similar for individuals with severe arthropathy and people with other orthopaedic conditions, but different for mild haemophilia. Many haemophilia treatment centres worldwide provide multidisciplinary care for haemophilia, but true value‐based health care goes further: all team members, regardless of specialty, share the responsibility to improve outcomes, and are accountable for the results…”

Outcome measures for adult and pediatric hemophilia patients with inhibitors

“…First, validated and standardized outcome assessment tools are required for research, including clinical trials, and for enabling long‐term patient follow‐up . The follow‐up and clinical care of individual patients, together with resource allocation, would also benefit from standardized outcome measurement, particularly in light of growing evidence that treatment for each patient should be individualized …”

“…11,15 The follow-up and clinical care of individual patients, together with resource allocation, would also benefit from standardized outcome measurement, particularly in light of growing evidence that treatment for each patient should be individualized. 16,17 Outcome measurement is also important from an economic viewpoint. The high cost of hemophilia care, coupled with a global economic recession 18 and limited resources in even high-income countries, inevitably means that some restrictions in hemophilia care will be requested by payers over the next few years in terms of products and treatment regimens used.…”

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

“…The importance of individualizing care for hemophilia, whether it be 'on demand' (episodic treatment for acute bleeding episodes) or prophylaxis, has been much discussed in peer-reviewed literature [15][16][17]. 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].…”