January 9, 2019

Untreated bleeds are common

Patient diaries show that 40% of bleeds in people with haemophilia are not treated, according to a study presented at the 60th American Society of Hematology Annual Meeting in San Diego in December 2017.

The study was part of the emicizumab development programme carried out by Genentech/Roche. Children and adults with haemophilia A used a handheld device to record real-world data about bleeds and their treatment. Participants included 103 with inhibitors and 94 without inhibitors; there were 24 children.

Adults and adolescents reported 1,596 bleeds and children reported 378 bleeds; 41% in each group were not treated. Most bleeds were reported by people without inhibitors (n=1,456) and fewer were untreated (14%).

The most common types of bleeds involved a joint (71 – 72%) or muscle (15 – 16%) with similar rates for people with or without an inhibitor; joint bleeds were less common in children (51%) but muscle bleeds were as frequent (16%).

Treated bleeds reported by adults and adolescents with inhibitors were more likely to be spontaneous (58%) than traumatic (42%) whereas the balance was more equal in patients without inhibitors (48% vs 52%) and somewhat similar in children (54% vs 46%. Untreated bleeds in adults and adolescents with inhibitors were also spontaneous in 63% of cases but more often traumatic in origin in those without inhibitors (60%). In children, the relative frequencies were again similar (46% spontaneous, 53% traumatic).

The authors suggest that clinical trials may be underestimating the true frequency of untreated bleeds in people with haemophilia A.