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- Beeton, K, et al.
(author)
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Recent developments in clinimetric instruments
- 2006
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In: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 12:Suppl. 3, s. 102-107
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Journal article (peer-reviewed)abstract
- Assessment of impairment and function is essential in order to monitor joint status and evaluate therapeutic interventions in patients with haemophilia. The improvements in the treatment of haemophilia have required the development of more sensitive tools to detect the more minor dysfunctions that may now be apparent. This paper outlines some of the recent developments in this field. The Haemophilia Joint Health Score (HJHS) provides a systematic and robust measure of joint impairment. The MRI Scoring System has been designed to provide a comprehensive scoring system combining both progressive and additive scales. The Functional Independence Score for Haemophilia (FISH) has been developed to assess performance of functional activities and can be used in conjunction with the Haemophilia Activities List (HAL) which provides a self report measure of function. It is recommended that both measures are evaluated as these tools measure different constructs. Further refinement and testing of the psychometric properties of all of these tools is in progress. More widespread use of these tools will enable the sharing of data across the world so promoting best practice and ultimately enhancing patient care.
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- Berntorp, Erik, et al.
(author)
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Long-term prophylaxis in von Willebrand disease
- 2005
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In: Blood Coagulation and Fibrinolysis. - 1473-5733. ; 16:Suppl 1, s. 23-26
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Journal article (peer-reviewed)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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| 6. |
- Castaman, G., et al.
(author)
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Hemorrhagic symptoms and bleeding risk in obligatory carriers of type 3 von Willebrand disease: an international, multicenter study
- 2006
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In: Journal of Thrombosis and Haemostasis. - : Elsevier BV. - 1538-7933 .- 1538-7836. ; 4:10, s. 2164-2169
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Journal article (peer-reviewed)abstract
- Objectives: We undertook an international, multicenter study to describe the clinical picture and to estimate the bleeding risk in a group of obligatory carriers of type 3 von Willebrand disease (VWD). Patients and methods: Obligatory carriers (OC) of type 3 VWD were identified by the presence of offspring with type 3 VWD or by being an offspring of a type 3 patient. Normal controls were age- and sex-matched with the obligatory carriers. A physician-administered standardized questionnaire was used to evaluate hemorrhagic symptoms at presentation. A score system ranging from 0 (no symptom) to 3 (hospitalization, replacement therapy, blood transfusion) was used to quantitate bleeding manifestations. Odds ratios were computed for each symptom. Results: Ten centers participated to the study, enrolling a total of 35 type 3 VWD families, with 70 OC. A total of 215 normal controls and 42 OC for type 1 VWD were also included. About 40% of type 3 OC had at least one bleeding symptom compared to 23% of normal controls and 81.8% of type 1 OC (P < 0.0001 by chi-squared test), showing that type 3 OC clearly represent a distinct population from type 1 OC. The clinical situations associated with an increase of bleeding risk in type 3 OC were epistaxis [odds ratio 3.6; 90% confidence intervals (CI) 1.84-21.5], cutaneous bleeding (odds ratio 5.5; 90% CI 2.5-14.1) and postsurgical bleeding (odds ratio 16.3; 90% CI 4.5-59). The severity of bleeding score correlated with the degree of factor (F) VIII reduction in plasma. Conclusions: OC for type 3 VWD represent a distinctive population from type 1 OC. These patients, however, present with more frequent bleeding symptoms in comparison to normal controls, especially in case of significantly low FVIII. Desmopressin and/or tranexamic acid might be useful to prevent or treat bleeding in these cases.
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