Dose and outcome of care in haemophilia – how do we define cost‐effectiveness?

Fischer, Kathelijn; Berg, H. M. Van Den; Thomas, Robert A.; Kumar, Sathish; Poonnoose, Pradeep Mathew; Viswabandya, Auro; Kavitha, M.; Bhattacharji, Sara; Srivastava, Alok

doi:10.1111/j.1365-2516.2004.01047.x

Cited by 19 publications

(18 citation statements)

References 17 publications

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“…In Mexico, the factor usage ranges between 530 and 817 IU/kg/year which are still relatively low [7]. Most data regarding the prevention of arthropathy have been from developed countries, where factor replacement is generally above 1,500 IU/kg/year [20].…”

Section: Discussionmentioning

confidence: 99%

Functional independence score in hemophilia: A cross‐sectional study assessment of Mexican children

Tlacuilo‐Parra¹,

Villela-Rodriguez²,

Garibaldi-Covarrubias³

et al. 2009

Pediatric Blood & Cancer

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BackgroundThe Functional Independence Score in Hemophilia (FISH) is a performance‐based assessment tool used to measure the patients functional ability so far only used in patients with severe hemophilia. Its aim is to determine if FISH is useful in patients with mild and moderate disease.ProcedureIn a cross‐sectional study 90 children 60 hemophilic and 30 sex‐, race‐, and age‐matched healthy males were assessed. Patients between 5 and 16 years of age were selected each patient was evaluated in seven activities under three categories: self‐care (grooming and eating, bathing, and dressing), transfers (chair and squat), and locomotion (walking and step climbing). Each activity was graded from 1 to 4 according to the amount of assistance required to perform the activity with total scores ranging from 7 to 28.ResultsAs a whole, the mean age of the patients was 10.0 ± 3.4 years with a mean FISH of 25.8 ± 3.6 (range 15–28). There were no differences in the FISH between healthy males and patients with mild hemophilia; however, the score was significantly higher in patients with mild hemophilia (28 ± 0) than patients with moderate (26.2 ± 2.5; P = 0.004) or severe hemophilia (24.0 ± 4.7; P = 0.0006). The most affected activities were squatting, walking, and step climbing.ConclusionsA significant decrease in functional ability was demonstrated according to the severity of hemophilia, especially for those activities involving weight‐bearing demands like locomotion and step climbing. Of seven activities evaluated, changes were observed in the group with moderate and severe hemophilia, but no changes detected in patients with mild disease. Pediatr Blood Cancer 2010;54:394–397. © 2009 Wiley‐Liss, Inc.

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Section: Discussionmentioning

confidence: 99%

Functional independence score in hemophilia: A cross‐sectional study assessment of Mexican children

Tlacuilo‐Parra¹,

Villela-Rodriguez²,

Garibaldi-Covarrubias³

et al. 2009

Pediatric Blood & Cancer

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“…starting after the age of 2 years or after 2 or more joint bleeds) (Chambost & Ljung, 2005). The total annual consumption of factor on prophylaxis regimens varies considerably, and there is evidence that the lower doses used in the Netherlands have been as effective at protecting the joints as the higher doses used in Sweden (Fischer et al , 2004). The clinical variability of ‘severe’ haemophilia is an important factor; some patients with severe haemophilia have few joint bleeds; they have their first bleed at an older age (van Dijk et al , 2005b), and this subgroup of phenotypically milder disease may successfully stop prophylaxis in early adulthood (van Dijk et al , 2005c).…”

Section: Treatment Of Haemophiliamentioning

confidence: 99%

Optimal haemophilia care versus the reality

Bolton‐Maggs

2006

Br J Haematol

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SummaryHaemophilia A and B are inherited bleeding disorders whose diagnosis and management is generally well established and best provided by specialists in a comprehensive care setting. Patients may be put at unnecessary risk if appropriate expertise is not sought for the management of accidents and surgery. The delivery of a high quality comprehensive service to patients with bleeding disorders depends upon defined standards and a network of haemophilia centres in the UK with similar models in other countries. In developing countries, despite a shortage or absence of treatment products, development of local expertise results in an improved outlook and reduction in mortality. Optimal care for severe haemophilia includes accurate diagnosis, early and adequate factor replacement for bleeding episodes and the provision of prophylaxis from an early age to prevent joint bleeding and the consequent arthropathy. Haemophilia treatment is expensive resulting in considerable inequity in provision of care across the world. Despite decades of experience, optimal treatment levels are not robustly defined. Transfusion-transmitted infections continue to have a significant impact on patient management. The development of inhibitory antibodies seriously complicates the management both in morbidity and cost. While gene therapy has not yet produced the hoped-for cure, new technologies will produce improved products.

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“…However, treatment is more effective if prophylaxis is started early in life. The decrease in bleeding episodes is also related to the duration of secondary prophylaxis [22,23,24]. The duration of the prophylactic treatment in the present study varied from occasional prophylactic infusion to prophylaxis during the entire course of the study (0.1–5.2 months), thus the observation time was short.…”

Section: Discussionmentioning

confidence: 99%