| 1. |
- Berntorp, Erik, et al.
(författare)
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A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate P/ Humate -P: history and clinical performance.
- 2008
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Ingår i: European Journal of Haematology. Supplementum. - : Wiley. - 0902-4506 .- 0902-4441 .- 1600-0609. ; 80:s70, s. 3-35
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Forskningsöversikt (refereegranskat)abstract
- Patients with von Willebrand disease (VWD) and haemophilia A (HA) lack, to varying degrees, the von Willebrand factor (VWF) and coagulation factor VIII (FVIII) that are critical for normal haemostasis. These conditions in turn make patients prone to uncontrolled bleeding. Historically, patients with severe forms of VWD or HA were crippled before adulthood and their life expectancy was significantly reduced. Over the past decades, specific coagulation factor replacement therapies including Haemate P, have been developed to help patients achieve and maintain normal haemostasis. Haemate P is a human, plasma-derived VWF/FVIII medicinal product, which was first licensed in Germany in 1981 for the treatment of HA-associated bleeding. It has since then come to be accepted as the gold standard for both the treatment and prophylaxis of bleeding in VWD, especially in cases where desmopressin [1-deamino-8-D-arginine vasopressin (DDAVP)] has been ineffective. Haemate P was the first effectively virus-inactivated (pasteurisation: 60 degrees C for 10 h in aqueous solution) FVIII product, whereby the risk of potentially threatening infective complications of plasma-derived products was reduced. Haemate P was also shown to have a VWF multimer profile remarkably close to that of normal plasma. This bibliographic review presents previously unpublished clinical data of Haemate P, based upon internal clinical study reports of the proprietor, CSL Behring, in addition to data already presented in other publications. The data demonstrate a predictable and well-characterised pharmacokinetic profile, and a proven record of short- and long-term safety, while effectively correcting the haemostatic defects in VWD and HA. Recently available data have also shown Haemate P to be of haemostatic value in exceptional clinical circumstances including surgical interventions. By virtue of its plasma-derived combination of VWF and FVIII, in addition to its high VWF:FVIII content ratio (2.4:1), Haemate P is also associated with successful immune tolerance induction in those patients developing inhibitor antibodies. Although the theoretical risk of thromboembolic complications does exist while receiving Haemate P, as it does with any FVIII replacement therapy, the incidence of such complications has remained notably low. Given the robust data that have accumulated for the use of Haemate P, dosing recommendations are also described in this review; the recommendations are tailored to patient-specific contexts including baseline VWF and FVIII levels in plasma and the type of surgical intervention being undertaken. A wide variety of studies have also provided data on paediatric and geriatric populations, all of which have suggested that Haemate P can be safely and effectively used in a wide variety of clinical circumstances.
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| 2. |
- Khawaji, Mohammed, et al.
(författare)
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Lifelong prophylaxis in a large cohort of adult patients with severe haemophilia: a beneficial effect on orthopaedic outcome and quality of life.
- 2012
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 88:4, s. 329-335
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Tidskriftsartikel (refereegranskat)abstract
- Background: In the 1950s, Sweden initiated prophylaxis as a lifelong treatment for haemophilia. It was the first country to do so. Objective: To describe and evaluate dosing and outcome of prophylactic treatment in a large cohort of adult people with severe haemophilia who have been using prophylaxis most of their lives. Methods: Eighty-one patients born between 1932 and1992 were divided into two groups (Group A started prophylaxis at the age of ≤ 3 years; Group B at three or more years of age) and evaluated retrospectively. Outcome was evaluated using the Hemophilia Joint Health Score (HJHS) and SF-36, a measure of quality of life. Results: The median number of joint bleeds per year was 0 in both study groups; however, the annual number of joint bleeds during the final three years of observation was higher in group B than group A (p< 0.006). Twenty-five of 30 patients in group A and 27/51 patients in group B had no joint bleeds in that period. Group A had significantly better joint outcomes than group B. Patients in group A experienced better physical and social health than those in group B. Conclusions: This follow-up has provided for the first time more extensive and detailed information regarding the practice of prophylactic treatment in a large cohort of adults with severe haemophilia. The present study confirms, that early start of prophylaxis and continuing throughout the lifespan has been successful in virtually eliminating joint bleeds, preserving a close to normal joint status, and keeping patients healthy and able to live normal lives. © 2012 John Wiley & Sons A/S.
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| 3. |
- Miesbach, Wolfgang, et al.
(författare)
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When von Willebrand disease comes into age - A matter of change?
- 2011
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 86, s. 496-501
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Tidskriftsartikel (refereegranskat)abstract
- The population of elderly patients with von Willebrand Disease (VWD) is growing due to the improvement of medical care. The increase of co-morbidities and their treatment as well as standard preventive measures like anti-platelet or anticoagulation therapy constitute a challenge in the overall treatment of patients with coagulation disorders. Due to the lack of literature on older VWD patients, we discuss different aspects related to ageing in those patients. Plasma levels of von Willebrand factor (VWF) and factor VIII (FVIII), bleeding symptoms, treatment requirements and co-morbidities are possibly changing with age. Development of an evidence-based approach to the management of ageing VWD patients is therefore necessary.
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| 4. |
- Ar, Muhlis Cem, et al.
(författare)
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Methods for individualising factor VIII dosing in prophylaxis
- 2014
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 93, s. 16-20
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Forskningsöversikt (refereegranskat)abstract
- Haemophilia A is a sex-linked disorder characterised chiefly by recurrent, spontaneous joint and muscle bleedings resulting from deficiency of factor VIII (FVIII). Recurrent joint bleeds result in haemophilic arthropathy. Unless treated with factor replacement therapy, many patients with severe haemophilia become disabled. The first clinical evidence favouring prophylaxis originated from the studies in Sweden and the Netherlands in the 1960s. Later on, it was shown that prophylaxis could prevent arthropathy, if started early in life, or slow its progression in adults with established arthropathy. The optimal dosing of FVIII in long-term prophylaxis has still not been determined, and there is growing evidence that the dose and frequency of FVIII should be individualised. We conducted a systematic search of PubMed to identify all relevant articles on FVIII prophylaxis in severe haemophilia A. We focused on articles with detailed information about individualisation of prophylaxis. Long-term prophylaxis in haemophilia was introduced in Sweden in the late 1950s. However, standard prophylactic regimens may not be appropriate for all patients with severe haemophilia. Factors such as age, joint status, co-morbidities and differences in pharmacokinetics lead to interindividual variation in factor requirement. Dose tailoring of FVIII by clinical outcome was first described in 1994. Since then, several dose-finding studies questioned the necessity to maintain preinfusion levels of FVIII above 1%. Individualising prophylaxis by dose tailoring is now recommended. Each country should adopt policies for individualising prophylaxis in patients with severe haemophilia. This would lead to a better distribution of the available source of factor concentrates.
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| 5. |
- Berntorp, Erik, et al.
(författare)
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Comorbidities and inhibitors in adult patients with haemophilia: issues, costs and management strategies.
- 2015
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 95, s. 1-15
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Forskningsöversikt (refereegranskat)abstract
- Along with greater life expectancy in patients with haemophilia has been an increase in associated haemophilia-related (arthropathy, osteoporosis, viral infections) and age-related (cardiovascular disease, renal disease, cancer and others) comorbidities, many of which are only just emerging as the population ages. At present, experience in managing these comorbidities is limited. As the demographic shift continues, haemophilia care centres can expect to encounter more patients with greater levels of complexity. In the absence of evidence-based information to guide the management of adult patients with haemophilia, it is important that the scientific position be reviewed on a regular basis. To this end, several topics relevant to the clinical management of adult patients with haemophilia were examined in a symposium entitled Comorbidities and inhibitors in adult patients with haemophilia: issues, costs and management strategies held on 11 February 2015 in Helsinki, Finland, in conjunction with the 8th Annual Congress of the European Association for Haemophilia and Allied Disorders. This article is a summary of that event.
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| 6. |
- Berntorp, Erik
(författare)
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Introduction.
- 2015
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 94, s. 1-1
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Tidskriftsartikel (refereegranskat)
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| 7. |
- Boehlen, Francoise, et al.
(författare)
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Outcome measures in haemophilia: a systematic review
- 2014
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 93, s. 2-15
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Forskningsöversikt (refereegranskat)abstract
- Haemophilia A and B are hereditary X-linked disorders due to deficiency (or absence) of coagulation factor VIII or IX, respectively. Bleeding risk is related to the severity of factor deficiency. Repeated joint bleeding can lead to a severe haemophilic arthropathy resulting in disabilities. Outcome measurements in persons with haemophilia (PWH) have been limited to laboratory evaluation (factor VIII or IX levels) and clinical outcomes (such as bleeding frequency), morbidity (for example linked with arthropathy) and mortality. Due to the new standard of care of PWH, there is a need to consider other outcome measures, such as the early detection and quantification of joint disease, health-related quality of life (QoL) and economic or cost-utility analyses. To investigate this, we performed a 10-yr systematic overview of outcome measures in haemophilia. Only clinical trials including at least 20 patients with haemophilia A or B were included. To facilitate the search strategy, eight issues of outcome measures were selected: physical scores, imaging technique scores, functional scores, QoL measurement, mortality, bleeding frequency, cost and outcome and bone mineral density. The results of these will be discussed. Clearly defined outcomes in haemophilia care are important for many reasons, to evaluate new treatments, to justify treatment strategies, to allow a good follow-up, to perform studies and to allocate resources. The use of such scoring systems is clearly recommended by experts in haemophilia care. However, most centres do not perform such scores outside clinical trials due to reasons such as lack of time and resources.
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| 8. |
- Canaro, Mariana, et al.
(författare)
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The ageing patient with haemophilia
- 2015
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 94, s. 17-22
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Forskningsöversikt (refereegranskat)abstract
- Older patients with haemophilia (PWH) face many challenges related not only to haemophilia but also to general comorbidities associated with ageing. This article discusses the clinical experience published about the high prevalence of diseases in older PWH. These conditions are managed in the general population by healthcare workers with little training in haemophilia. Haemophilic arthropathy is common in elderly PWH. Prophylaxis starting at an early age in sufficient dose regimens to prevent arthropathy did not occur in patients who are now older than around 40yr. Many PWH above this age thus have limitations in their activities of daily life. Cardiovascular diseases have become increasingly common in the growing, ageing cohort of PWH. Lifestyle issues such as sexual dysfunction may be exacerbated by the medical issues and psychological problems associated with haemophilia. Hepatitis C virus is a leading problem in PWH. Coinfection with HIV accelerates the progression to end-stage liver disease. Acute and chronic renal failure is more common in adult PWH than in general population. Other comorbidities are reviewed. The evidence is scarce, so it is imperative to report any experience regarding the diagnosis and treatment of these entities, to improve the quality of life of older PWH.
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| 9. |
- Miesbach, Wolfgang, et al.
(författare)
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Interaction between VWF and FVIII in treating VWD
- 2015
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Ingår i: European Journal of Haematology. - : Wiley. - 1600-0609 .- 0902-4441. ; 95:5, s. 449-454
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Tidskriftsartikel (refereegranskat)abstract
- In patients with von Willebrand disease (VWD), the absence of von Willebrand factor (VWF) antigen leads to the premature loss of endogenous circulating secreted factor VIII (FVIII), thereby resulting in the dual defect in haemostasis. Consequently, correcting the VWF deficiency also acts to correct the associated defect in FVIII activity because exogenous VWF forms complexes with and protects endogenous FVIII. The purpose of this study was to summarise relevant aspects of the interaction between VWF and FVIII and to analyse their effects on VWD treatment. Differences in the VWF/FVIII ratios in coagulation factor concentrates should be considered when treating VWD.
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| 10. |
- Berntorp, Erik
(författare)
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Introduction.
- 2014
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Ingår i: European Journal of Haematology. Supplementum. - : Wiley. - 0902-4506 .- 0902-4441. ; 93, s. 1-1
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Tidskriftsartikel (refereegranskat)
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