Transplant Center: NIH

After you've decided that a stem cell transplant (aka BMT) is what you need to do, there are many different issues that you need to work through. One is the choice of a transplant center.

There are lots of high quality transplant centers around the US. However many follow treatment protocols tailored for a "typical" transplant patient, i.e. someone with life-threatening blood cancers like leukemia or
diseases which result in bone marrow failure like aplastic anemia. The outcomes for patients like Elise with a primary immune deficiency can be improved with a different treatment protocol, but there are far fewer patients to practice these protocols with and they are relatively new. So it's best to go to a transplant center that has more experience with BMT for immune disorders. There are a lot less of these simply because there are a lot less of these patients.

Given this, for Elise there were really only two choices that made sense: Children's Hospital of Los Angeles (CHLA) or National Institutes of Health (NIH) in Bethesda, MD. There are other transplant centers, but they are all effectively as remote as NIH is from us and had no particular additional benefits to consider.

A BMT takes a long time. There are 4 basic stages:

1. conditioning for transplant (12 days); 
2. transplant to transplant +45 days
3. 45 days - 100 days
4. follow-up care and recovery

During stages 1 and 2 Elise will be checked into the hospital and one of Becky or I will need to be with her at all times. During stage 3 Elise will need to be very close to the transplant center. Stage 4 can go on for months or even years but the Dr visits are typically routine appointments.

CHLA is closest to home and the support system that would provide would be nice. However we'd still need to relocate to the LA area for stages 1-3. Typically people stay at the Ronald McDonald House which is nearby.

NIH is on the other side of the country, so relocating will take on added complexity (sounds simple but I've been writing lists of things to consider, e.g. what do we do about a car?) Patients families tend to get a short term apartment in Bethesda. There are 3 pluses that swayed us to NIH. First, they are the CGD experts. Elise has been going there for several years under a study protocol (we fly back and forth), so they know her and we know them. Second, their treatment protocol includes collecting a stem cell donation from Elise in case the donor's doesn't engraft (this is an unusual risk mitigation step). And third, the transplant is part of a government-funded study so we don't need to hassle with our insurance company until the major activities have happened (we'll likely get stage 4 care at CHLA). I really don't want Elise's Dr's fighting with the insurance company over whether a medication is "standard of care" if she gets some life threatening infection.

So we've decided to go with NIH. An added benefit is that Elise says she's most comfortable there and anything to ease the apprehension is welcome.