| 11. |
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| 12. |
- Berntorp, Erik, et al.
(författare)
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Inhibitor treatment in haemophilas A and B: Summary statement for the 2006 International Consensus Conference
- 2006
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 12, s. 41281-41281
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Tidskriftsartikel (refereegranskat)abstract
- Participants in an international conference on the management of haemophilia patients with inhibitors developed a jointly authored summary of the findings and conclusions of the conference. Current knowledge of the genetic and immunologic mechanisms underlying inhibitor development was briefly summarized. Concerning the purported treatment-related risk factors, conference participants commented on the limitations of the available evidence and the need for more rigorous prospective research in a fully genotyped population. Other clinical considerations discussed included the unproved utility of routine surveillance, the need for assay standardization, the management of acute bleeding and approaches to joint disease prophylaxis and immune tolerance induction (ITI). A number of issues were identified as needing further investigation in larger prospective studies, ideally through international cooperation. Such studies should enrol cohorts that have been scrupulously defined in terms of mutation status and treatment exposure. Finally, conference participants urged their colleagues to participate in the currently ongoing international trials of ITI.
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| 13. |
- Berntorp, Erik
(författare)
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Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens.
- 2009
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 15, s. 1219-1227
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Tidskriftsartikel (refereegranskat)abstract
- In patients with severe haemophilia, spontaneous bleeding into joints initiates a sequence of events culminating in disabling arthropathy. Early evidence from Sweden suggested that clotting factor prophylaxis improved patient outcomes. Recent randomized, controlled trials comparing prophylaxis with on-demand treatment have definitively shown that prophylaxis reduces bleeding and improves joint outcomes in patients with severe haemophilia A. Available evidence also supports the effectiveness of prophylaxis in patients with haemophilia B. In the United States, fewer than half of all patients with severe haemophilia A or B are treated with prophylaxis, and in those receiving such treatment, adherence to prophylactic treatment regimens is low in many age groups. Barriers to prophylaxis include cost, difficulties associated with venous access and the time required for prophylactic infusions. Although concerns around adherence play an important role in the willingness of physicians to prescribe prophylaxis, individualized prophylactic regimens may help increase patient adherence. Clotting factors that are more convenient and less time-consuming to infuse also may improve adherence to prophylactic therapy. By promoting rigorous adherence to prophylactic clotting factor therapies, physicians may be able to help preserve joint function in patients with severe haemophilia.
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| 14. |
- Berntorp, Erik, et al.
(författare)
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Long-term prophylaxis in von Willebrand disease
- 2005
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Ingår i: Blood Coagulation and Fibrinolysis. - 1473-5733. ; 16:Suppl 1, s. 23-26
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Tidskriftsartikel (refereegranskat)abstract
- The majority of patients with von Willebrand disease (VWD) have a mild bleeding tendency that primarily involves mucosal bleeding. Some patients with the disorder, however, have severe episodes of mucosal or joint bleeding that can hamper daily activities and lead to significant joint impairment. Experience in the setting of severe hemophilia has shown the feasibility and benefits of prophylactic treatment to prevent bleeding and development of arthropathy. This approach also needs to be evaluated in patients with VWD who require repetitive treatment for bleeding episodes. Data from a series of 35 patients (with VWD types 3, 2A, 2B, and 1) who have received long-term prophylaxis at Malmo University Hospital and Karolinska University Hospital in Stockholm, Sweden, have demonstrated a substantial reduction of bleeding episodes since initiation of treatment. Patients who began prophylaxis at a young age (younger than 5 years) to prevent nose and mouth bleeds have had no joint bleeds and have no clinical signs of arthropathy. Treatment has been safe, with no cases of thrombosis, and no viral transmission among patients who received virus-aftenuated von Willebrand factor-containing factor VIII concentrate. These data thus suggest that long-term prophylaxis is warranted in the majority of patients with type 3 VWD and in other subtypes with severe bleeding tendencies, and that such an approach may help in the avoidance of joint disease if started early. More clinical data and controlled trials are needed in order to formulate recommendations for prophylaxis in patients with VWD.
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| 15. |
- Berntorp, Erik, et al.
(författare)
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New Approaches to Using FEIBA in the Treatment of Inhibitor Patients.
- 2006
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Ingår i: Seminars in Thrombosis and Hemostasis. - : Georg Thieme Verlag KG. - 1098-9064 .- 0094-6176. ; 32:Suppl 2, s. 22-27
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Tidskriftsartikel (refereegranskat)abstract
- Managing hemophilia becomes particularly difficult in patients with inhibitory antibodies, especially in those requiring surgery or with refractory bleeding events. Equally challenging are those patients who develop autoantibodies against factor VIII (FVIII) in the absence of a prior history of FVIII deficiency (acquired hemophilia). Physicians seeking both short- and long-term treatment strategies for bleeding events must often rely on FVIII-bypassing agents such as activated prothrombin complex concentrate (e.g., factor eight bypassing activity [FEIBA VH, Baxter BioScience, Westlake Village, CA]) or recombinant factor VIIa (rFVIIa [NovoSeven, NovoNordisk, Bagsvaerd, Denmark]). Surgical procedures in patients with inhibitors present a considerable challenge, from both a risk-benefit and a cost-benefit aspect. Hemostasis is difficult to achieve in these patients and new treatment options are being explored. Similarly challenging are refractory bleeds, the management of which is likely to benefit from a systematic treatment approach.
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| 16. |
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| 17. |
- Berntorp, Erik
(författare)
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Pharmacoeconomics of factor dosing in the haemophilia population.
- 2006
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 12 Suppl 4, s. 70-73
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Tidskriftsartikel (refereegranskat)abstract
- Treatment of haemophilia is extremely costly due to the short biological half-life of infused factor and pricing issues. This paper examines the impact of different dosing schedules on factor consumption, via a review of literature on dosing regimens used for prophylaxis with a focus on pharmacokinetics (PK). Pharmacokinetics were found to have an important role for pharmacoeconomics in factor dosing both for assessment of the treatment and for developing new treatment protocols but the clinical response to treatment must always guide the dosing schedule. In order to better understand pharmacoeconomics during prophylaxis, controlled prospective studies are needed but much can also be learned from studies of existing cohorts that have been treated for decades.
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| 18. |
- Berntorp, Erik
(författare)
-
Prophylaxis and treatment of bleeding complications in von Willebrand disease type 3.
- 2006
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Ingår i: Seminars in Thrombosis and Hemostasis. - : Georg Thieme Verlag KG. - 1098-9064 .- 0094-6176. ; 32:6, s. 621-625
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Tidskriftsartikel (refereegranskat)abstract
- Treatment of type 3 von Willebrand disease (vWD) relies on infusion with plasma-derived factor concentrates containing von Willebrand factor (vWF). Patients with types 1 and 2 vWD who do not respond satisfactorily after receiving desmopressin need treatment with concentrates. The rationale for long-term prophylaxis in vWD is obvious: prophylaxis has been successfully used in hemophilia, and joint hemorrhages with development of hemophilic arthropathy can occur, especially in type 3 vWD. In Sweden, prophylaxis for vWD began during the 1960s, and we now have experience from a cohort of 37 patients treated for a median of 11 years (range, 2 to 45 years). The majority of subjects (n = 28) have type 3 vWD. The mean dose used for treatment is 24 units factor VIII/kg body weight given one to three times weekly. Indications for prophylaxis have included joint bleeds, bleeds from nose and mouth, menorrhagia and gastrointestinal bleeds. The annual number of bleeds has decreased dramatically following onset of prophylaxis. We conclude that long-term prophylactic treatment of vWD is warranted in the majority of cases with type 3 and in some cases, depending on the clinical phenotype, for patients with other subtypes. Additional studies are ongoing in an international effort, the von Willebrand Disease Prophylaxis Network.
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| 19. |
- Berntorp, Erik
(författare)
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Prophylaxis in von Willebrand disease.
- 2008
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 14 Suppl 5, s. 47-53
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Tidskriftsartikel (refereegranskat)abstract
- This paper reviews current issues regarding short-term (i.e. for surgery and invasive procedures) and long-term (i.e. regular infusions to prevent bleeding on a permanent or temporary basis) prophylaxis treatment using replacement concentrates for patients with von Willebrand disease (VWD) who do not respond satisfactorily to desmopressin. The standard treatment of these patients is with factor concentrates containing von Willebrand factor (VWF) and factor VIII (FVIII). When dosing these concentrates, the broad variations in content and quality of VWF as well as the FVIII content in the products should be considered. Peri-operative management strategies will depend on the VWD subtype, baseline VWF and FVIII levels, and size of procedure. FVIII level and VWF ristocetin cofactor activity may both be used to determine concentrate potency and to monitor treatment. Long-term prophylaxis, which has become a state-of-the-art approach in haemophilia, is not very common in VWD. However, more recent data suggest that a substantial number of VWD patients could benefit from prophylactic treatment with VWF-containing concentrates. For example, 35 Swedish VWD patients who required prophylaxis (mainly because of nose/mouth bleeds and joint bleeds) showed a substantial overall reduction in bleeding episodes and there were no signs of arthropathy in children who began prophylaxis before the age of 5 years since initiation of treatment with Haemate P/Humate-P. Studies of prophylaxis in VWD are urgently needed to develop evidence-based guidelines for this approach; the VWD International Prophylaxis study, for example, has commenced by the VWD Prophylaxis Network.
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| 20. |
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