Severe haemophilia A damages joints in young boys through repeated haemarthroses which are treated as they occur with injections of recombinant factor VIII. Fortunately, after the disasters of hepatitis and HIV-contaminated cryoprecipitate in the 1980s, this provides a completely safe treatment. This trial assesses whether in boys under 30 months of age, prophylactic factor VIII given by injection on alternate days might provide better joint protection, and it does.
Age-related macular degeneration is mostly a disease of elderly people who have smoked; we know that complement 3 is important in its pathogenesis but we don’t know much about the genetics involved. Here a genetic study identified a complement 3 polymorphism in a southern English cohort with MD; this was then cross-checked with a cohort from another country – Scotland. And the association holds. Aye.
There have been enough trials of neuroprotective agents for stroke over the last decade to fill a large book. They all begin with promising results in animal models, yield possible benefits in small pilot studies, and then flop when tried in larger randomised trials. Here the trials used an agent called NXY-059 and were known as the SAINT I and SAINT II trials respectively. The drug didn’t work, and these SAINTs will join all the others who from their labours rest.
In fact we don’t really have a good intervention for acute ischaemic stroke, judging from a short clinical review here. Intravenous thrombolysis using rt-PA (alteplase) provides very modest benefits if administered in the first 3 hours, and it’s possible that clot retrieval and/or intra-arterial thrombolysis may produce better results but possibly at the expense of more haemorrhage.