Episodic versus prophylactic infusions for hemophilia A: A cost-effectiveness analysis

Smith, Peter J. S.; Teutsch, Steven M.; Shaffer, Phaedra A.; Rolka, Henry; Evatt, Bruce L.

doi:10.1016/s0022-3476(96)70076-8

Cited by 125 publications

(165 citation statements)

References 10 publications

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“…It has been shown that patients on prophylaxis still experience joint bleeds. 2,3,9,22,23 Thus, starting prophylaxis before the occurrence of the first joint bleed postpones its occurrence but does not entirely prevent joint bleeds. Animal studies have suggested that young cartilage is more susceptible to damage by hemarthroses than older cartilage.…”

Section: Discussionmentioning

confidence: 99%

The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia

Fischer

Bom

Mauser‐Bunschoten

et al. 2002

Blood

239

232

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To prevent hemophilic arthropathy, prophylactic treatment of children with severe hemophilia should be started before joint damage has occurred. However, treatment is expensive, and the burden of regular venipunctures in young children is high. With the aim of providing information on starting prophylaxis on the basis of individual patient characteristics, the effect of postponing prophylaxis on longterm arthropathy was studied in a cohort of 76 patients with severe hemophilia born between 1965 and 1985. The median age at first joint bleed was 2.2 years (range, 0.2-5.8). Prophylaxis was started at a median age of 6 years (interquartile range [IQR], 4-9), and the median annual clotting factor use on prophylaxis was 1750 IU/kg/y (31 IU/kg/wk). Hemophilic arthropathy was measured by the Pettersson score (maximum, 78 points). At a median age of 19 years, the median Pettersson score was 7 points (IQR, 0-17). After 2 decades of follow-up, the Pettersson score was 8% higher (95% confidence interval, 1%-16%) for every year prophylaxis was postponed after the first joint bleed. This effect was independent of age at Pettersson score, age at first joint bleed, and prophylactic dose used. In conclusion, most patients have their first joint bleed after the age of 2 years. Patients who start prophylaxis soon after the first joint bleed show little arthropathy in adulthood. The longer the start of prophylaxis is postponed after the first joint bleed, the higher the risk of developing arthropathy.

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Section: Discussionmentioning

confidence: 99%

The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia

Fischer

Bom

Mauser‐Bunschoten

et al. 2002

Blood

239

232

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“…(approximately £1000) calculated by Szucs et al [20] and $1380 (approximately £850) calculated by Smith et al [21]. There are several possible reasons for these differences.…”

Section: Discussionmentioning

confidence: 86%

“…Resource data were confined to clotting factor use, although such provision is believed to account for up to 93% of the total cost of care [21]. Similarly, outcomes data were only available for the number of bleeds which represent an intermediate outcome measure.…”

Section: Discussionmentioning

confidence: 99%

Assessing the effectiveness and cost-effectiveness of prophylaxis against bleeding in patients with severe haemophilia and severe von Willebrand's disease

Miners¹,

Sabin²,

Tolley³

et al. 1998

J Intern Med

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Objectives. To assess the effectiveness and cost-effectiveness of prophylaxis with clotting factor against bleeding in patients with severe haemophilia and von Willebrand's disease (vWD). Design. Treatment details that related to 179 patients with severe (Ͻ 1 u dL Ϫ1 ) haemophilia A, B and vWD were retrospectively examined for the period 1980-95. A subgroup of these patients, 25 adults and 22 children, who had previously received treatment on demand and who had switched to treating with prophylaxis, were studied in order to examine the effects of the change. The cost-effectiveness of prophylaxis was also analysed using another subgroup of 38 patients and by adjusting their treatment details by age and method of treatment. Setting. Data were obtained on patients who were solely registered at the Royal Free Hospital Haemophilia Centre (RFHHC), London, UK. Outcome measure. Bleeds. Results. The median annual number of bleeds decreased from 23.5 (range 1-107) in 1980, to 14 (range 0-45) in 1995 (P < 0.0001). Switching from treating on demand to prophylaxis reduced bleeding frequency in 41 out of 47 patients within the period of 1 year. At the base scenario, switching to prophylaxis cost an additional £547 per averted bleed; however, this figure was highly sensitive to certain variables. Conclusion. Prophylaxis can reduce bleeding frequency but requires more clotting factor than treatment on demand. More detailed proof of cost-effectiveness is likely to require the use of modelling techniques.

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“…23,24 In a study in France, the mean annual per-patient treatment cost with on-demand therapy was estimated to be USD $73,029. 25 In Germany, where guidelines advocate alternate day prophylaxis for patients with severe hemophilia A, the average annual costs vary from €40,000 to €120,000 per patient.…”

Section: Hemophilia and Factor VIII Treatment Strategiesmentioning

confidence: 99%

Using Value-of-Information Methods when the Disease Is Rare and the Treatment Is Expensive—The Example of Hemophilia A

et al. 2014

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BACKGROUND: Hemophilia A is a rare, sex-linked genetic disorder treated with intravenous administration of factor VIII (FVIII) to prevent bleeding; however, approaches vary across and within countries. Value-ofinformation (VOI) methods identify situations in which the cost-benefit evidence is sufficient to adopt one treatment strategy over another; when the evidence is insufficient, VOI methods provide the optimal sample size for additional research. OBJECTIVE: The objective of the study was to use VOI methods in a cost-benefit decision context to evaluate the current evidence in support of using (1) alternate day prophylaxis (AP), (2) tailored prophylaxis (TP) or (3) on-demand treatment (OD) with FVIII to prevent arthropathy in children with severe hemophilia A. METHODS: To apply VOI methods, several parameters such as incidence, time horizon for the decision, costs, and threshold values to avoid MRI-detected joint damage or arthropathy were defined. Two baseline threshold values of willingness to pay for avoiding arthropathy-$200,000 and $400,000-were selected for comparing the treatment strategies. RESULTS: For threshold values<$200,000, OD had a higher expected net benefit than either prophylaxis strategy, and the evidence was sufficient for its adoption. For threshold values > $400,0,00 prophylaxis strategies had higher expected net benefit; however, a new trial with 38 patients per arm was needed to compare AP and TP, yielding an expected net gain of over $17 million. In sensitivity analyses, the results were robust to assumptions regarding discount rate, trial fixed and variable costs, enrollment fraction, and the time horizon. CONCLUSIONS: In rare diseases, evidence is often scarce and insufficient for decision making. In considering the funding of new research and patient reimbursement in rare diseases, VOI methodology may provide more relevant determinations of the value and costs of additional research, compared to standard frequentist methods.KEY WORDS: expected value of sample information; hemophilia; children; prophylaxis; costs.

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Episodic versus prophylactic infusions for hemophilia A: A cost-effectiveness analysis

Cited by 125 publications

References 10 publications

The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia

The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia

Assessing the effectiveness and cost-effectiveness of prophylaxis against bleeding in patients with severe haemophilia and severe von Willebrand's disease

Using Value-of-Information Methods when the Disease Is Rare and the Treatment Is Expensive—The Example of Hemophilia A

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