Thirty years of hemophilia treatment in the Netherlands, 1972-2001

Plug, Iris

doi:10.1182/blood-2004-05-2008

Cited by 105 publications

(150 citation statements)

References 26 publications

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“…Since then nationwide surveys were repeated in 1978,1985,1992 and in 2001 [21][22][23][24]. These studies aimed at assessing the medical and social consequences of haemophilia in the Netherlands.…”

Section: Settingmentioning

confidence: 99%

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Hepatitis C infection among Dutch haemophilia patients: a nationwide cross‐sectional study of prevalence and antiviral treatment

et al. 2005

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Summary. Hepatitis C is a major co‐morbidity among patients with haemophilia who received inadequately or non‐virus‐inactivated clotting factor concentrates before 1992. The objectives of this study were to investigate the prevalence of hepatitis C and the use of antiviral therapies during the last decade among patients with haemophilia in the Netherlands. We performed a cross‐sectional study and a questionnaire was sent to all 1519 patients known with haemophilia in the Netherlands between 2001 and 2002. The study population for the present study consisted of 771 patients who had received clotting factor products before 1992 of whom 638 reported their hepatitis C status. In total, 441 of the 638 (68%) patients ever had a positive test for hepatitis C virus (HCV); 344 patients (54%) had a current infection, and 97 (15%) had cleared the virus. Among 344 patients currently HCV infected, 111 (32%) had received treatment for hepatitis C, while 34% (33/97) of patients with an infection in the past had been treated for hepatitis C. In 2002 the prevalence of hepatitis C among patients with haemophilia who received clotting factor products before 1992 was 54%. The majority of patients with a current HCV infection had not been treated with antiviral therapy.

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Section: Settingmentioning

confidence: 99%

“…The mean age of patients differed according to severity of haemophilia and HCV status; patients with severe haemophilia, who were never infected, were younger [mean age 23 years, 95% confidence interval (CI) [19][20][21][22][23][24][25][26][27][28] …”

Section: Hepatitis Cmentioning

confidence: 99%

Hepatitis C infection among Dutch haemophilia patients: a nationwide cross‐sectional study of prevalence and antiviral treatment

et al. 2005

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“…This is considered the gold standard of preventive care and is implemented in many Scandinavian and Northern European countries. 21,22 Although the World Health Organization, the National Hemophilia Foundation, and the World Federation of Hemophilia recommend FVIII prophylaxis as the main approach for treating children, 20 in several countries patients are treated with an on-demand regimen, that is, FVIII infusions given only when bleeding occurs.…”

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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“…Hemarthrosis as the predominant presentation with most affected joint being knee joints were also found in Korean study 21 . However many other studies conducted in Netherlands, France and Spain showed ankle joint as mostly affected joints 22,23,24 .…”

Section: Clinical Manifestationmentioning

confidence: 99%