Kreuth III: European consensus proposals for treatment of haemophilia with coagulation factor concentrates

Abstract

Summary 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.U. per capita. Decisions on whether to adopt a new product should not be based solely on cost. Prophylaxis for children with severe haemophilia is already recognized as the optimum therapy. Ongoing prophylaxis for individual adults should also be provided when required based on clinical decision making by the clinician in consultation with the patient. 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.

Patient‐centred value framework for haemophilia

“…Patient‐centred outcomes used to compare primary prophylaxis to on‐demand therapy have included survival, joint bleeding, other bleeding (eg intracranial haemorrhage), joint damage/arthropathy, joint function/motion, inhibitor development, complications (eg infection and need for central venous catheter) and QOL. Evidence of clinical benefit of prophylaxis compared to on‐demand treatment has led to its support by haemophilia organizations worldwide . While increased use of factor for prophylaxis raises per‐patient product costs compared to on‐demand treatment, its impact on joint function preservation also reduces disability and non‐factor healthcare costs among prophylaxis users .…”

Haemophilia care in Latin America: Assessment and perspectives

“…Access to concentrates is widely available in Argentina, Brazil, Chile, Colombia and Costa Rica. However, according to data from the 2016 WFH Annual Survey, only Argentina and Colombia reach optimal levels for treatment (4 IU FVIII per capita). In other countries (El Salvador, Mexico, Panama, Dominican Republic and Venezuela), it is considered inadequate for different reasons.…”

The current status of prophylactic replacement therapy in children and adults with haemophilia

“…FVIII or IX concentrations in plasma <0·01 iu/ml (≤1% of normal), joint bleeding episodes may occur as frequently as 20–30 times per year (Ramgren, ; Ahlberg, ) and life‐threatening bleedings such as intracranial haemorrhage (ICH) may occur (Ljung, ). According to a joint statement made by the World Health Organization and the World Federation of Hemophilia (WFH), initiating prophylactic treatment at an early age is considered to be the optimal form of therapy for a child with severe haemophilia (Berntorp et al , ; Richards et al , ; Giangrande et al , ). However, there is no universal agreement on the definition of ‘prophylactic therapy’ for haemophilia.…”