The USTMA Consortium held their first annual conference for TTP patients on 22 August 2020. It was originally scheduled as an in-person conference in Chapel Hill, North Carolina, but due to COVID-19 it pivoted to a virtual conference.
The stated mission of the USTMA Consortium is to “improve outcomes in Thrombotic Thrombocytopenic Purpura (TTP) and other thrombotic microangiopathies (TMA), through observational studies, clinical trials of novel therapies and translational research in these rare diseases.”
The conference agenda covered the following items:
·
Coping with Strategies for Dealing with Chronic Disease
· The Doctors’ Golden Rules: Listen to your Patients, Learn from your Patients
So, what did I learn?
How to forecast TTP. I think my hematologist and I are now on the right track. In the past, we measured the level of the platelets to check that status of my blood. This is like looking out the window to forecast the weather. The platelet level tells if you have a problem or do not have a problem. To forecast TTP, it is better to measure the trend of the ADAMTS13 activity, one of enzymes in the blood, If the ADAMTS13 activity is trending down, then TTP could happen in the future. To monitor ADAMTS13 activity, it was recommended that ADAMTS13 activity be measured monthly for 3 months after TTP, then every 3 months for the first year, and then every 6-12 months. Currently we are measuring my platelets and my ADAMTS13 activity monthly. So far, so good.
How can we try to prevent relapse? The goal is to stay away from plasmapheresis and steroids (Prednisone). Plasmapheresis requires hospital time, nursing time, loads of plasma and either needles in your arms or a line in your jugular vein. Steroids are just bad as there are many side effects. As such if the ADAMSTS13 activity can forecast a relapse, it is recommended that the patient is prescribed Rituximab to help improve the ADAMTS13 activity. Additionally, Caplacizumab can also be prescribed as this drug will help to block platelet aggregation. Note Caplacizumab may not be approved for use in all countries, may not be known or used by your hematologist, and may not be prescribed due to its cost.
The conference discussed TTP and the brain and coping with chronic disease. Originally it was thought once recovered from TTP, the patient’s lives would return to normal. Unfortunately, studies have shown that TTP most significantly effects the brain, then the kidneys, and then the heart. As a result, many TTP patients do not fully recover. Due to the impact on the brain many patients become depressed. Discussions with recovered patients showed they are still dealing with the effects of the disease.
TTP does not impact people uniformly. About 75 percent of the time, TTP patients are woman. And is the US, about 50 percent of the patients are African American. There was no reasoning provided.
Stopping a TTP episode takes time. TTP is an auto-immune disease, so we first try to stop the auto-immune disfunction using Prednisone. This drug is quite effective, but like all other alternatives, it is slow. I have experienced this slowness. In 2017, my platelets were back to normal after 9 days and we stopped plasmapheresis, but 4 days later my platelets were 39 and I had retinal detachment. In 2019, we also stopped plasmapheresis after 9 days to allow me to go home for Christmas and Boxing Day. Unfortunately, my platelets dropped to 16 and we really had to start plasmapheresis all over again. We learned you need to taper away from plasmapheresis slowly to help prevent relapse.
All in all, the conference was successful. If you did not attend, you should watch some of the videos linked to the agenda above. I highly recommend you watch The Doctors’ Golden Rules section by Dr. James George.
Thanks, Bruce.
@BruceFightsTTP
