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Current Challenges in the Management of Hemophilia
Kenneth A. Bauer, MD
Participating Faculty: Hemophilia: Managing the Cost of Care With a Focus on Patient Outcomes
Post Test: Hemophilia: Managing the Cost of Care With a Focus on Patient Outcomes

Current Challenges in the Management of Hemophilia

Kenneth A. Bauer, MD
One of the most serious treatment complications in patients with hemophilia is transfusion-related HCV and HIV infections.27 The high rate of HCV infection in older patients with hemophilia is a contributing factor to the increased prevalence of both renal disease and liver disease.28,29 Cirrhosis is a potential outcome for those infected with HCV, and coinfection with HIV has been shown to accelerate the development of liver failure.30

Renal Disease

Compared with the general population, patients with hemophilia are 50 times more likely to die from renal failure.31 Renal disease is partly due to the higher incidence of hypertension and HIV coinfection in patients with hemophilia compared with the general population.32,33

Cardiovascular Disease

Patients with hemophilia are also more likely to experience symptomatic cardiovascular disease and intracranial hemorrhage with life-threatening complications.34 The presence of hypertension is likely a contributing factor to the increased risk of cardiovascular disease, with higher rates of hypertension noted in patients with moderate and severe hemophilia compared with those with mild hemophilia.35


The proportion of patients with hemophilia who are overweight or obese has been estimated at 50%, which is similar to that of the general population. Overweight/ obesity in patients with hemophilia increases the likelihood of hemophilic arthropathy and decreased joint range of motion.36

Quality of Life

Over the years, advances in the treatment of hemophilia have narrowed the life expectancy gap between patients with hemophilia and the general population. Despite this, the need for frequent intravenous infusions of factor therapy, complications of treatment, and comorbidities result in an impaired QOL for patients with hemophilia. This applies particularly to those with severe disease, who require access to medical care and supplies on a regular basis. The requirement that factor therapy be refrigerated further restricts a patient’s mobility and lifestyle choices. Traveling to remote areas or regions without medical resources may be challenging. In addition, patients with hemophilia are discouraged from participating in high-risk contact sports or other activities. These restrictions can be difficult to accept for younger, active patients. Family planning concerns may arise in adulthood, and genetic counseling may be needed for the couple wishing to have children if the father has hemophilia or the mother is a known carrier.37 The financial cost associated with a lifetime of therapy and clinic visits can be substantial, and regular doctor and physical therapy appointments take up a substantial amount of both patients’ and caregivers’ time. Some patients experience chronic pain that can lead to dependence on opioids or illicit drugs.38,39 QOL is significantly improved for patients living in industrialized countries with strong family support compared with those from less developed countries and lower socioeconomic status.40

General Challenges With Hemophilia Treatment

The diagnosis and management of patients with severe hemophilia are complex. Preventive treatment (prophylaxis) is required to avoid bleeding episodes and related complications, and use of replacement therapy with coagulation factors is needed during acute bleeding episodes. Optimal care of patients with hemophilia requires a comprehensive approach that is coordinated by a multidisciplinary team of specialists and is provided at a dedicated hemophilia treatment center.6 Despite advances in the management of hemophilia, challenges remain, including the shortage of physicians trained in the management of hemostasis and thrombosis and limited access to specialized centers that provide comprehensive care.

Multidisciplinary Approach

The treatment of patients with hemophilia must be comprehensive to achieve optimal long-term results. A multidisciplinary approach is essential, and ideally includes a team of physicians, nurses, psychologists, genetic counselors, pharmacists, case/social workers, and physical therapists. Over the course of their lifetime, patients with hemophilia may need to have access to other specialists, including rheumatologists, pain specialists, infectious disease specialists, hepatologists, surgeons, nutritionists, and dentists.41

Access to Specialized Centers

Patients with hemophilia require lifelong access to specialized centers and comprehensive care, posing a challenge for many patients and providers. In one study, almost half of survey respondents who treated themselves with factor therapy at home reported concerns with the availability and affordability of their treatment. 42 Challenges also arise once a young patient enters adolescence and gains a certain degree of autonomy regarding their treatment. Programs designed to target youths with hemophilia during the transition to more autonomous management have been developed and are effective, but require the patients to have Internet access and other support.43 For patients with hemophilia, receiving dental care is also a challenge, and specialized facilities or hospitalization may be needed for procedures that would otherwise be considered routine.44

Shortage of Specialists

To further complicate the management of patients with hemophilia, fewer physicians are adequately trained to treat inherited bleeding disorders because graduates of combined hematology/oncology fellowship programs are more likely to focus on oncologic than on benign hematologic disorders.45 Patients with hemophilia require not only experienced hematologic care for their bleeding diathesis, but also care from other specialists such as dental surgeons and orthopedic surgeons who have experience with patients undergoing treatment for hemophilia. Improper or inadequate training in these areas can put the patient at unnecessary risk.

Cost of Therapy

Perhaps one of the most significant challenges facing patients, as well as their physicians, is the cost of therapy. Factor replacement is an expensive and necessary treatment. The use or choice of therapy depends on the severity of disease and presence of inhibitors.21 In one study of 1164 patients with hemophilia A or B with and without inhibitors, mean healthcare expenditures were evaluated. Among patients with hemophilia A, the mean healthcare expenditures were nearly 5 times higher in the inhibitor group than in the noninhibitor group.46


The use of prophylaxis is currently recommended as first-line treatment for children with severe hemophilia, and the use of prophylaxis is increasing for adult patients.47 Prophylactic treatment can be primary, secondary, or tertiary. Primary prophylaxis commences before the second joint bleed and before any established joint disease. Secondary prophylaxis occurs after the second joint bleed (at which point a bleeding pattern has likely been established) and ideally before any joint damage. Tertiary prophylaxis takes place when joint disease is already present and established (often in adulthood).41

Several studies have demonstrated the benefits of early prophylaxis—reducing the risk of spontaneous bleeding and subsequent complications of hemarthrosis.48-50 Other demonstrated benefits of early prophylaxis include prevention of intracranial hemorrhage and other serious bleeds, prevention of pain, improvements in QOL, and reduction in long-term disability.49

Despite the clinical advantages of prophylaxis, barriers to implementation exist among physicians and patients. Physicians may be uncertain about the optimal prophylactic regimen,49 particularly since the recent FDA approval of several therapies for this indication. For patients, barriers to implementation of prophylaxis include the need for venous access with associated potential for complications (surgical placement of indwelling lines, infections, catheter-related deep vein thrombosis), high cost, and potential need for home nursing or clinic visits to administer infusions until someone at home learns to perform infusions.

A recent survey published in 2012 by Ragni and colleagues regarding current prophylactic practices at 62 US hemophilia treatment centers showed substantial variability regarding the timing of initiating prophylaxis and the specific prophylaxis regimen used. Only 25% of the hemophilia treatment centers surveyed started prophylaxis after the first bleeding episode (of any type), and just 16% began after the second hemorrhage.51 It is clear that managed care health professionals who treat patients with hemophilia would benefit from education regarding optimal prophylaxis regimens that are based on recent information and research.

The pediatric population receiving prophylactic regimens initially incurs higher costs than the population treated on an on-demand basis. The benefit of prophylaxis, however, may outweigh the cost of complications that reduce QOL later in adulthood. Pediatric patients with severe hemophilia A who receive prophylaxis experience less joint destruction and fewer bleeding episodes than those who receive on-demand therapy.52,53 This ability to prevent hemophilic arthropathy, which is one of the most significant factors reducing the QOL in older patients, is one of the greatest advantages to a prophylactic regimen. Benefits can even be seen in adult patients who switch to a secondary prophylactic treatment course after prior on-demand therapy. One year of secondary prophylaxis increases QOL, reduces hemarthroses, and improves musculoskeletal joint assessments.54 Although many teenagers and young adults opt to discontinue prophylaxis in favor of less frequent, on-demand therapy, they experience more bleeding episodes and a decreased QOL. In a study of 38 participants comparing those maintained on a prophylactic regimen with 2 groups of patients receiving on-demand therapy, the prophylactic group experienced significantly fewer bleeding episodes and an improved QOL.55 Another long-term benefit of primary prophylaxis is fewer hospitalizations and surgical procedures on joints, which lowers medical care costs despite the increased expense of factor products.

Current Therapies

Current therapy for hemophilia A and B is replacement of the deficient coagulation factor using either plasma-derived or recombinant factor replacement products. Standard of care is to begin prophylactic therapy in childhood to avoid the long-term complication of joint destruction. However, less than half of adults continue prophylactic therapy with the remainder infusing factor concentrates on demand.56 A disadvantage of prophylactic therapy is the frequency of venipuncture required—roughly 2 to 3 times a week and potentially more often if breakthrough bleeding occurs. The relatively short half-lives of the factor replacements— approximately 10 to 14 hours for factor VIII and 15 to 24 hours for factor IX—creates the frequency requirement.57 This drawback may be why adult patients opt for less frequent, on-demand treatment regimens. Younger patients also often require means of venous access other than peripheral veins, including central lines and arteriovenous fistulas.58

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