Hello Hal,
As promised...see below: I did a copy and paste from this ongoing research. (As I have said from my prior post regarding personal updates post-splenectomy...ITP is caused because of an issue with the complement. If you inhibit the complement, ITP will be fixed.)
The excerpt:
Up to 20% of patients with immune thrombocytopenia (ITP) have inadequate responses to current therapy and remain at risk of life-threatening bleeds. In a study presented at the 2019 American Society of Hematology annual meeting, ITP patients received an investigational complement pathway inhibitor, sutimlimab (BIVV009), biweekly for up to 21 weeks in a small trial.
In this exclusive MedPage Today video, Keith McCrae, MD, of the Cleveland Clinic, an ITP expert not involved with the study, discusses the research.
Following is a transcript of his remarks:
There are drugs that inhibit complement. The one that is approved is farther down the complement pathway. It inhibits complement 5, but this inhibits complement 1, C1s, which is actually the first component of complement that binds to an antibody once that binds to a cell, so it's working upstream. There's some human evidence that this antibody is effective in one complement-mediated disease called cold agglutinin disease or cold agglutinin-induced hemolytic anemia, but this was actually the first study in ITP, and in fact, actually, the first human study of complement inhibition in ITP, so I think that's important.
It was a small study. There were only about eight patients in this study, but about 57% -- I guess four or five of the patients -- that responded. The nice thing is they responded very quickly, within 24 hours, actually, and had significant increases in their platelet count -- I think from baseline in the 20s to over 100,000 or around maybe 80,000 to 100,000 within 24 hours, which is really quite remarkable, and within 7 days, over 200,000 in the responding patients. These responses ... I think they treated with this drug every week initially, and then maybe less than that afterwards, but the responses were durable.
They then had a washout period. Over time in the washout period when the drug was stopped, the platelet count fell again, and they re-initiated the drug and the platelet count went back up.
I think this is important for two reasons. One is it's actually the first complement-inhibition study in ITP, and secondly I think this is potentially a promising, new drug for ITP. Exactly where it will ultimately fit in the scheme of ITP treatment I don't know. There's certainly more work that needs to be done before that's decided, but I think it's an interesting abstract to be aware of.
