Haemophilia : the official journal of the World Federation of Hemophilia
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Letter Randomized Controlled Trial
Efficacy of celecoxib in the treatment of joint pain caused by advanced haemophilic arthropathy in adult patients with haemophilia A.
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Rare bleeding disorders (RBDs) are inherited deficiencies of coagulation factors such as fibrinogen, factor (F) II, FV, FVII, combined FV+FVIII, FX, FXI and FXIII. These disorders usually have a low prevalence in the general population and constitute approximately 3-5% of all coagulation disorders. ⋯ The clinical picture of RBDs is highly variable and can vary markedly from mild to severe, making both diagnosis and optimal treatment quite challenging. This review focuses on: (i) the efforts to establish a bleeding assessment tool adequate to RBDs, (ii) the optimal management of patients affected with FXI deficiency and (iii) the correlation between clinical severity and laboratory diagnosis when determining the minimum coagulant activity required to prevent bleeding in each RBD.
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This report summarizes recommendations relating to haemophilia therapy arising from discussions among experts from 36 European countries during the Kreuth III meeting in April 2013. To optimize the organization of haemophilia care nationally, it is recommended that a formal body be established in each country to include the relevant clinicians, national haemophilia patient organization, health ministry, paying authority and (if appropriate) regulatory authorities. The minimum factor VIII consumption level in a country should be 3 I. ⋯ Children with inhibitors who have failed, or who are not suitable for, immune tolerance therapy should be offered prophylaxis with bypassing agents. Single factor concentrates should be used as therapy wherever possible in patients with rare bleeding disorders. Orphan drug designation for a factor concentrate should not be used to hinder the development, licencing and marketing of other products for the same condition which have demonstrably different protein modification or enhancement.
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Joint pain related to haemophilia affects large numbers of people and has a significant impact on their quality of life. This article reviews evidence about behavioural and psychological aspects of joint pain in haemophilia, and considers that evidence in the context of research on other chronic pain conditions. The aim is to inform initiatives to improve pain self-management among people with haemophilia (PWH). ⋯ Increased pain acceptance predicts better mental quality of life, so acceptance-based approaches to self-management could potentially be adapted for PWH. Pain self-management interventions could include elements designed to: improve assessment of pain; increase understanding of the difference between acute and chronic pain; improve adherence to clotting factor treatment; improve knowledge and understanding about the benefits and costs of using pain medications; improve judgements about what is excessive use of pain medication; increase motivation to self-manage pain; reduce negative emotional thinking about pain; and increase pain acceptance. The influence of behavioural and psychological factors related to pain are similar in haemophilia and other chronic pain conditions, so there should be scope for self-management approaches and interventions developed for other chronic pain conditions to be adapted for haemophilia, provided that careful account is taken of the need to respond promptly to acute bleeding pain by administering clotting factor.
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Randomized Controlled Trial Multicenter Study Clinical Trial
Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors.
Factor replacement therapy for the treatment of moderate to severe haemophilia A and B can be complicated by the production of inhibitory alloantibodies to factor VIII (FVIII) or factor IX. Treatment with the nanofiltered anti-inhibitor coagulant complex, Factor Eight Inhibitor Bypassing Activity (FEIBA NF), is a key therapeutic option for controlling acute haemorrhages in patients with high-titre inhibitors or low-titre inhibitors refractory to replacement therapy. Given the high risk for morbidity and mortality in haemophilia patients with inhibitors to FVIII or FIX, we conducted this Phase 3 prospective study to evaluate whether prophylaxis with FEIBA NF is a safe and effective treatment option. ⋯ Total utilization of FEIBA NF for the treatment of bleeding episodes was significantly higher during on-demand therapy than prophylaxis (P = 0.0067). There were no differences in the rates of related adverse events between arms. This study demonstrates that FEIBA prophylaxis significantly reduces all types of bleeding compared with on-demand treatment, and the safety of prophylaxis is comparable to that of on-demand treatment.