Recently I read an interesting article about a new med that’s been shown to speed the production of blood platelets by as much as 40% which could potentially improve the lives of so many across this great country of ours.
The drug in question, called caplacizumab, was studied in patients with a blood disease better known as thrombotic thrombocytopenic purpura (TTP) and I think the idiopathic version, ITTP. But it made me wonder if this drug might also work to treat people with aplastic anemia?
It just so happens that a dear friend of mine has a brother who was diagnosed with aplastic anemia a few years ago. He suspected he developed it as a result of taking methotrexate prescribed for his RA. He was told there was nothing he could do but receive blood transfusions, that as we know, will only work but so long before your body rejects the blood because of iron buildup. He wasn’t a candidate for a bone marrow transplant because of his age. But instead of giving up, he contacted John’s Hopkins and NIH for consultations to join a clinical trial for a drug called eltrombopag.
And here’s the HUGELY inspirational kicker…
After joining the trial and taking eltrombopag, he didn’t meet the endpoint. Doctors told him that the drug wasn’t working for him. He fought back, wrote letters, consulted with his doctors, and after several more months, somehow convinced doctors to do another arm of the study. Months passed and he got his wish! He joined another trial for the drug and THIS time he responded, albeit slightly, but damnitall, he DID respond to the drug. In other words, he was an “outlier” – he didn’t fit the norm of responses in the clinical trial because his body needed longer on the drug before it could work for him. Today, he feels like his life is almost normal again and he’s feeling soooo much better. He hasn’t had a transfusion in more than 8 months!
What have you got to say about that?! Have you been rejected from a clinical trial for aplastic anemia? Sound off! I want to hear from you!
