The Community's Perspective on the Potential Impact of New Targeted Treatment for TTP

Answering TTP Foundation is excited to learn that Health Canada has approved the use of caplacizumab in combination with plasma exchange and immunosuppression for the treatment of aTTP in adults.

Canadian patients with aTTP can now have access to this targeted drug and the hope it brings for a safer and faster recovery from a TTP crisis. Answering TTP Foundation is looking forward to working with provincial and territorial partners to ensure that this targeted therapy is quickly made available at TTP treatment centers across the country and timely reimbursed by public and private plans.

Results of the HERCULES (multi centre, randomized, double-blind, placebo-controlled Phase 3 clinical study) trial indicate that "treatment with caplacizumab was associated with faster normalization of the platelet count; a lower incidence of a composite of TTP-related death, recurrence of TTP, or a thromboembolic event during the treatment period; and a lower rate of recurrence of TTP during the trial than placebo"---source: New England Journal of Medicine.


We asked patients what this new treatment means to them.

Answering TTP Foundation took an active role in the CADTH and INESS review of caplacizumab by preparing patient feedback based on surveys and interviews of our community. Two surveys (the second to address follow up questions from the committee) were conducted with a response rate of 257 and 154 respectively. More information about the review of caplacizumab and copies of the Foundation's submissions can be found on the CADTH website here.
Survey Outcome 1: This drug will saves lives
The family of one patient who had passed away stressed the value of more options ---
"One of our frustrations was that she was getting plasmapheresis but the platelets [count] just didn't move. Having one more option could have made all the difference in the world." --- Family member of deceased from 2019 survey.
"As soon as a TTP patient presents [with an episode], it is time to start treatment; there is no time for triage. Having a drug that is a "booster" could be very important. But even after the platelets seem to have stabilized, you have to continue to monitor. In our case, it dropped again over the weekend and by the time we realized he was in serious trouble, it was too late. He never got the rituximab or another drug. Our brother was robbed." --- Family member of deceased from 2019 survey.
We have seen delays have devastating consequences. "He was on plasmapheresis but the doctors were overconfident in the initial response to PP. He was responding so well; the platelets had risen so well, but it was false confidence. They recommended rituximab but it never got there. He passed away 9 or 10 days after diagnosis." --- Family member of deceased from 2019 survey.
Survey Outcome 2: This drug gets patients home faster and safer
In general, patients recognize plasmapheresis as the ubiquitous imperative treatment to deal with a TTP episode; however, it is not possible to over-emphasize the challenge of the process on the individual, both physically and emotionally, and by extension, the family. As described by one patient:
“Plasmapheresis treatments are tough. It feels as if your whole body is humming. … When I get a bag/a donor that I am more sensitive to, I might develop hives and get hot. As soon as I feel this, I would tell my nurse so she could give me another shot of Benadryl to counteract the reaction from the blood product. With every shot, I felt the rush of the Benadryl and I would try to give in and sleep. But mostly only my eyes close – the rest of my body is anxiously awake. I try not to think about the probability of a more serious reaction or side effect.
… Plasma exchange treatments save my life, but they also mean that my life is all of a sudden hijacked for extended hospital stays. This was easier when it just meant that I was off work and in hospital leaving my husband, parents, siblings and friends anxious about my recovery. This reaches a whole new level when you need to juggle kids and manage their emotions and real fears.” --- Patient respondent from 2019 survey.
More than half (56%) of the Canadian respondents required more than 20 plasma exchange treatments to achieve remission [following their most challenging TTP crisis]. It is important to appreciate how arduous and time-consuming a plasma exchange procedure can be. A single plasma exchange treatment typically takes over three hours, so this means that half of TTP patients spend 60 hours or more hooked up to a machine following a single TTP crisis.
In describing what “needing fewer plasma exchange treatments could mean”, the majority spoke about spending less time in hospital, being able to be home, being able to take care of children and family, experiencing less anxiety and need to plan (in anticipation of the next crisis), and less fear and anxiety about the procedure and the side effects. Nearly half of all Canadian respondents said they spent more than three weeks (and up to 12-plus weeks) in hospital following their most difficult TTP crisis.
In their own words "needing fewer plasma exchange treatments could mean" (2019 survey):
  • It would make me extremely happy. There are many dangers associated with plasma exchange. Also, terrible side effects.
  • A normal lifestyle. Not running to the hospital so often.
  • It's so traumatic on the body to have high dose exchange treatments, alongside side effects. The treatment takes everything out of you (literally), making recovery a long, long process and treatment pretty much taking up your days. This is mentally and physically very difficult to recover from.
  • Hopefully less of a stay in the hospital! Almost a month was a long time, especially when you have a line sutured into your jugular!
  • Needing less plasma exchange treatments would mean faster and safer recovery with less side effects from the treatment. As a frequent relapser my life during remission is also ruled in part by planning for the next TTP crisis. If the every day anxiety isn't enough, the disruption to work and kids is tremendous. My husband and I always need to consider planning for the next relapse because weeks in hospital followed by months of recovery takes emotional energy, money and time.
  • For me, plasma exchange is one of the scariest parts of TTP. While donated blood is tested and the odds of catching something are very low, the amount of plasma donations I have consumed over the years increases those odds significantly. I have always had mild allergic reaction.