This is part three of my talk.
My name is Lucio Luzzatto,
I'm a hematologist at
the Muhimbili University of Health and Applied Sciences in Dar-es-Salaam, Tanzania.
The notion that by blocking complement,
you could avoid hemolysis PNH is not a new concept.
But of course we didn't have the technology until
the technology of monoclonal antibodies was introduced.
So, can we take advantage of this to treat these patients?
So this for a hematologist,
are the very many aspects of the clinical burden of PNH.
Not only there is anemia,
but there is risk of thrombosis,
there is the risk of
annal depletion through into intravascular hemolysis causing abdominal pain.
It can also cause dysphagia, pulmonary hypertension,
erectile dysfunction, and multiple problems in pregnancy.
So, many of these are complicated in an indirect way,
but they all stem from the basic process which I have illustrated.
Until recently, the treatment of PNH
was based on support of the patient including blood transfusion,
possible radical treatment through
bone marrow transplantation, and sometimes immunosuppression.
I cannot go now into how that works.
Also, we needed anticoagulant prophylaxis to prevent
thrombosis and various procedure in pregnancy.