How we manage requests for recombinant factor VIIa (NovoSeven).
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Drug Costs↗
Biomedical subjects
Prasad Mathew
Publications and source records attributed to Prasad Mathew.
Current opinion on inhibitor treatment options.
Inhibitor development in patients with hemophilia complicates hemostatic management. Currently, high doses of factor concentrates are used to override low-titer, low-responding inhibitors during acute bleeding episodes. Bypassing agents, such as the activated prothrombin complex concentrate (aPCC), Factor Eight Inhibitor Bypassing Activity, Anti-Inhibitor Coagulant Complex, Vapor Heated (FEIBA; Baxter AG, Vienna, Austria), and activated recombinant factor VII (rFVIIa; NovoSeven, Novo Nordisk A/S, Bagsvaerd, Denmark), are commonly used to treat acute bleeding episodes in patients with high-titer, high-responding inhibitors, whereas long-term therapeutic options include inhibitor eradication using immune tolerance induction. Neither FEIBA nor rFVIIa can be monitored with a laboratory assay, making it difficult to establish optimal dosages. Comparative studies evaluating the efficacy of FEIBA and rFVIIa for bleed control have been sparse, prompting investigators to initiate crossover comparison studies to assess the efficacy and cost of aPCC and rFVIIa in the treatment of joint hemorrhages. Both the cost of therapy and the outcome of therapy will need to be considered in the development of future hemostatic agents for patients with inhibitors.
Blood Coagulation Factor Inhibitors↗
Pharmacoeconomic analysis of recombinant factor VIIa versus APCC in the treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors.
OBJECTIVE: To compare the cost-effectiveness of three treatment regimens using recombinant activated Factor VII (rFVIIa), NovoSeven, and activated prothrombin-complex concentrate (APCC), FEIBA VH, for home treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors. METHODS: A literature-based, decision-analytic model was developed to compare three treatment regimens. The regimens consisting of first-, second-, and third-line treatments were: rFVIIa-rFVIIa-rFVIIa; APCC-rFVIIa-rFVIIa; and APCC-APCC-rFVIIa. Patients not responding to first-line treatment were administered second-line treatment, and those failing second-line received third-line treatment. Using literature and expert opinion, the model structure and base-case inputs were adapted to the US from a previously published analysis. The percentage of evaluable bleeds controlled with rFVIIa and APCC were obtained from published literature. Drug costs (2005 US$) based on average wholesale price were included in the base-case model. Univariate and probabilistic sensitivity analyses (second-order Monte Carlo simulation) were conducted by varying the efficacy, re-bleeding rates, patient weight, and dosing to ascertain robustness of the model. RESULTS: In the base-case analysis, the average cost per resolved bleed using rFVIIa as first-, second-, and third-line treatment was $28 076. Using APCC as first-line and rFVIIa as second- and third-line treatment resulted in an average cost per resolved bleed of $30 883, whereas the regimen using APCC as first- and second-line, and rFVIIa as third-line treatment was the most expensive, with an average cost per resolved bleed of $32 150. Cost offsets occurred for the rFVIIa-only regimen through avoidance of second and third lines of treatment. In probabilistic sensitivity analyses, the rFVIIa-only strategy was the least expensive strategy more than 68% of the time. CONCLUSIONS: The management of minor-to-moderate bleeds extends beyond the initial line of treatment, and should include the economic impact of re-bleeding and failures over multiple lines of treatment. In the majority of cases, the rFVIIa-only regimen appears to be a less expensive treatment option in inhibitor patients with minor-to-moderate bleeds over three lines of treatment.
Blood Coagulation Factors↗
Therapeutic choices in the current millennium: hemophilia workshop highlights.
Many issues affect hemophilia care providers when managing bleeding episodes in individuals with hemophilia. A diverse group of hemophilia providers from the United States met at two workshops to discuss the issues influencing treatment choices in the current millennium and to learn about current advances in treatment of hemophilia. This paper provides a summary of the discussions at these workshops. Despite the progress made in the management of patients with hemophilia, the workshop highlighted the fact that there were still many unanswered questions.
Factor VIII↗
Recombinant activated factor VII as an adjunct to packing for liver injury with hepatic venous disruption.
Injuries to the juxtahepatic veins represent a small proportion of all liver injuries but constitute the most challenging and deadly form of hepatic trauma. Recombinant activated factor VII, established as a crucial therapy for enhancing hemostasis in hemophiliacs with inhibitors, has also been used to correct coagulopathy after traumatic injury. We report two children with hepatic venous injury requiring perihepatic packing and recombinant activated factor VII to successfully control hemorrhage.
Accidents, Traffic↗
Recombinant versus plasma-derived factor IX in the treatment of hemophilia B: proceedings of a meeting held in June 2003.
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Blood Component Transfusion↗
Conclusion.
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Blood Component Transfusion↗
Recombinant factor VIIa in management of spontaneous subcapsular liver hematoma associated with pregnancy.
BACKGROUND: Spontaneous subcapsular liver hemorrhage is a rare but life-threatening complication of pregnancy. Optimal management of an expanding hematoma or ruptured capsule has not been established. CASES: We report 3 patients with preeclampsia and hemolysis, elevated liver enzymes, and low platelets (HELLP) syndrome with spontaneous subcapsular liver hematomas. The first 2 patients with ruptured liver hematomas experienced life-threatening hemorrhage. The third patient experienced uncontrollable vaginal bleeding, liver hemorrhage, and was in imminent danger of capsule rupture. Despite aggressive surgical intervention and traditional blood component therapy, adequate hemostasis could not be achieved in any of these patients. Recombinant factor VIIa was used to achieve hemostasis in all three patients. CONCLUSION: Recombinant factor VIIa is an effective adjunct in the treatment of preeclamptic patients with expanding or ruptured subcapsular liver hematoma.
Adult↗
Thrombophilia and pregnancy complications.
The implications of currently available data on the association of gestational vascular complications with thrombophilia are presented in this consensus report. Screening is recommended for women with the following previous complications: fetal loss including three or more first trimester loss, two or more second trimester loss, or any stillbirth; early, severe or recurrent preeclampsia and severe intrauterine growth restriction. Maternal antithrombotic therapy is currently evaluated in women with thrombophilia and previous complications.
Abortion, Habitual↗
The use of rFVIIa in non-haemophilia bleeding conditions in paediatrics. A systematic review.
From its original envisioned use in patients with hemophilia and inhibitors, recombinant factor VIIa has been increasingly used in a variety of non-hemophilia bleeding/hemorrhagic situations with great efficacy. Most of the reported work has been in adult patients. This paper sets out to review its use in the pediatric non-hemophilia patients and the varied conditions it has been tried and used. Most of the published literature has shown that this agent is efficacious, safe and can be used as an adjunctive measure in the achievement of hemostasis. However, most of the published work is mainly anecdotal, case reports or small series. Randomized trials in children are eagerly awaited.
Blood Coagulation Disorders↗
Novel applications of recombinant factor VIIa for the management of pediatric coagulopathic diseases.
Recombinant factor VIIa (rFVIIa) was envisioned for the treatment of bleeding in hemophilia patients with inhibitors. In response to tissue factor expressed upon vessel wall injury, rFVIIa amplifies the thrombin burst primarily on membrane surfaces, including activated platelets. Because it is functional at a key point in the clotting cascade, rFVIIa shows promise as a therapeutic option for various bleeding situations. The prothrombin time (PT) is often used as a surrogate to monitor rFVIIa therapy. Using laboratory and clinical measures of outcome, the use of rVIIa as a therapeutic option in treating several different pediatric coagulopathic conditions is described.
Adolescent↗
Pain and distress from bone marrow aspirations and lumbar punctures.
OBJECTIVE: To compare the efficacy of 3 different pharmacologic regimens to relieve pain and distress in children with cancer undergoing bone marrow aspirations (BMAs) and lumbar punctures (LPs). DESIGN: Retrospective cohort study with crossovers for some patients. PATIENTS AND METHODS: The pain and distress ratings of patients undergoing BMAs (n = 73) and LPs (n = 105) were examined in a comparison of 3 different interventions: (1) a topical eutectic mixture of lidocaine and prilocaine (EMLA cream), (2) oral midazolam and EMLA cream, or (3) propofol/fentanyl general anesthesia. The choice of the intervention depended on patient/parent request. A validated faces pain scale was completed by the child or parent following each BMA or LP. The faces pain scale includes ratings of the severity of pain (from 0 = none to 5 = severe) and ratings of how frightened (from 0 = not scared to 5 = scared) the child was prior to each procedure. Comparisons of the pain and distress ratings were made among all patients for their first procedure and also within individual patients who had received >1 of the 3 interventions. Independent comparisons between the first treatments received by each patient were analyzed using Kruskal-Wallis tests. Comparisons of different crossover treatments received by individual patients were analyzed using Wilcoxon tests. RESULTS: For all first procedures, mean +/- SD pain and distress ratings during LPs were significantly lower when propofol/fentanyl was used (n = 43; 0.4 +/- 1.0 and 1.4 +/- 1.7) versus either EMLA (n = 29; 2.4 +/- 1.7 and 2.9 +/- 1.9) or midazolam/EMLA (n = 33; 2.4 +/- 1.8 and 2.7 +/- 1.8), respectively. Pain and distress ratings during BMAs were also significantly lower with propofol/fentanyl (n = 29; 0.5 +/- 1.0 and 1.2 +/- 1.7) versus EMLA (n = 21; 3.5 +/- 1.6 and 3.3 +/- 1.8) or midazolam/EMLA (n = 23; 3.3 +/- 1.5 and 3.0 +/- 1.9), respectively. When data were analyzed within each patient, these differences were also present. CONCLUSIONS: Children receiving propofol/fentanyl general anesthesia experienced significantly less procedure-related pain and distress than did those receiving either EMLA or oral midazolam/EMLA.
Adolescent↗
Cellular neurothekeoma of the chest wall.
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Child↗