UNIGE document Scientific Article
previous document  unige:44753  next document
add to browser collection

Outcome measures in haemophilia: a systematic review

Graf, Lukas
Berntorp, Erik
Published in European Journal of Haematology. 2014, vol. 76, no. suppl., p. 2-15
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.
PMID: 24957102
Full text
Article (Published version) (116 Kb) - document accessible for UNIGE members only Limited access to UNIGE
Research group Geneva Platelet Group (13)
(ISO format)
BOEHLEN, Françoise, GRAF, Lukas, BERNTORP, Erik. Outcome measures in haemophilia: a systematic review. In: European Journal of Haematology, 2014, vol. 76, n° suppl., p. 2-15. doi: 10.1111/ejh.12369 https://archive-ouverte.unige.ch/unige:44753

340 hits

0 download


Deposited on : 2015-01-08

Export document
Format :
Citation style :