Summary - Conversation with Duke

Mandy and I spoke with Dr. Kurtzberg at Duke today. I didn't know at the time, but one of my former co-workers told me that Dr. Kurtzberg did a bone marrow transplant for her brother who had brain cancer. He was 10 at the time and now he's a scientist! :) She gave us a lot to think about, but also reassured us that Univ of Minnesota is one of only two right places to be. Here are the critical insights from our chat:

1. Treatment at a place with lots of Hurler experience is critical - Seems there are a lot of little disease-specific nuances that require special attention. For example, two vertebra are not quite right in Hurler kids, requiring special head positioning during treatment to prevent respiratory complications.
2. Good news: Hurler kids tolerate the chemo treatment relatively well - According to Dr.Kurtzberg, most don't even get all that sick/ nauseous during the 9 days of chemotherapy. They do lose their appetites, but IV feeding takes care of that.
   
3. Cord blood sounds better than bone marrow - Because it comes from newborns, there are a lot of non-blood cells in the cord blood that may help with other development issues. For example, there are neurons that can help with neurological development, etc. Bone arrow doesn't have these benefits. She also said that cord blood yields better outcomes. Some marrow kids end up with more orthopedic problems, and she sees a lot of "normal" cord blood kids. Finally, it only takes 10 days to find a match, so that won't hold up treatment
   
4. Younger is better - Not just because you head off a lot of the degenerative activity, but also because you can infuse them with more cells (not sure how this works...)
5. Kids have a much harder time in the month following transplant - that first month is critical, and we'll be praying and praying - you should to! The first two weeks, her blood count will be very low
6. Lots of tests will need to be done to make sue Gracie has no viruses before she starts chemo. Apparently, that is what causes most of the complications.
   
7. Graft versus host disease (GVHD) occurs in about 20% of cord blood transplants - it's serious in about 5%. They have a lot of treatments for this.
   
8. The big new question that came up is around the combination of enzyme replacement therapy and transplant. She is against it, although Gracie is scheduled to start it next week. I didn't get the details of why, so I hope that Mandy can chime in with a post explaining the issues. Needless to say, we have a new round of questions for the docs at Univ of MN.
9. Another question we want to answer is the difference between successful transplant (meaning the graft "takes") and the level of enzyme function. Most studies focus on the former as the success metric. I would say that from a quality of life perspective, the latter is probably more important.
   

Sarah, Jimmy, and Gracie had a great day at Univ of Minnesota, and they have been really impressed with the care team thus far. Given that we are here, and that they like it, I think the plan is to move forward with treatment in Minnesota. Duke sounds pretty good too, but we had a couple of reservations based on the call.

Other great news, our cousin Tracy knows a pediatric neurologist (Dr. PeePee, she calls him) in Minneapolis who will give us the good, bad, and ugly of all of Gracie's physicians. Thanks Tracy!

Another interesting note: Sarah found an online group of families who've been through this before. I hope she will summarize her experience with that, because it sounds like a terrific resource. They seem to also really support the Minnesota option.

We're all cautiously excited. Gracie will get a central line across the next couple of days, and pending our grilling them, she will start ERT next week. Much more to come!