Following my previous post I will carry on telling you about the treatments that Corey has received.
The 'main' treatment for MPS1 / Hurler Syndrome is a Bone Marrow Transplant (BMT). Also known as a Cord Blood Transplant as donated umbilical cord blood can be used or the medical term - Hematopoietic Stem Cell Transplantation (HSCT).
It is best for Hurler kids to be 'transplanted' before they are 2 years old because even though the damage already done by the storage of GAGs is irreversible, the sooner the better for it to work best slowing down further damage.
The problem with this is getting children diagnosed before they are 2 because the disease is so rare (1 in 100,000) lots of doctors don't know enough about it. This is where I count ourselves 'lucky' to be able to have a transplant before Corey was 1 and why it is so important to raise awareness of MPS.
Corey's transplant was from a very special Spanish mum who donated her son's cord blood at birth. We will never know who she is but she was the first person to save Corey's life.
The BMT process wipes out the body's immune system. The actual transplant is just like a blood infusion. The new blood stem cells rebuild the immune system by doing their thing and hopefully they 'engraft' without complication and hey presto his body now produces the missing enzyme.
This treatment is extremely risky as something like a cold virus could potentially kill you when you have no immune system and then there's graft versus host disease, veno-occlusive disease and many other complications which have taken the lives of children during this process. There is also a risk that it won't work and the body will reject the new cells - sometimes another transplant can be done and sometimes you're left on enzyme therapy for life.
We were extremely lucky in that Corey 'sailed' through transplant, it wasn't until later that he developed complications.
No comments:
Post a Comment