More on Reduced Intensity Conditioning, Cord Blood, and HLH

A lot more information today. Some days it feels like we’re drowning in medical data and decisions. Michelle and I have been working through the options and doing our best to understand each path using our own research and what we learn from parents who have gone through this, which we then speak to our doctors about and clarify until we feel able to make a decision.

With regard to RIC, we’ve been going back and forth about using that or ablative/high-dose conditioning. One route is the more conservative route that has more successful cases behind it, but greater future danger for Zoe’s health and development. The other is much more modern approach with less patient data to point to, but can result in a very clean, relatively safe cure for Zoe’s condition.

We have pushed Dr. P and our nurse coordinator pretty hard for answers to every possible angle of this in the past few days. We’ve presented other cases of RIC/HLH/Cord Blood patients with very similar protocols but which were not successes and asked them to help us understand what will be different for Zoe. We’ve pressed them for examples of successes with this combination of factors. We’ve asked for numbers on Duke’s track record with this combination as well.

At this point we feel fully satisfied that, for Zoe’s case, for her current condition and the team of people treating her, RIC is the way we should proceed. It’s a tough decision, but this is the direction that feels right.

In recent years there have been a number of advances that will assist Zoe. The team at Duke was the first to do an unrelated cord blood transplant, and they do more than any other institution. That helps our confidence level as well.

Their process includes several additional medications to the standard protocol that help raise the chances of a successful graft, one of the concerns when using RIC with cord blood and likely a factor in the failure to graft in some patients in the past. They know now to continue GVHD medications longer than was once felt necessary, with past cases at various institutions proving clearly that stopping too early can cause the graft to fail.

What we’re seeing is that HLH is a relatively young disease and that, while the treatment phase is well established with the HLH-2004 protocol, the cure phase is still being perfected. This leaves a lot of fear and doubt, and the old saying that if you ask 5 doctors you’ll get 5 answers doesn’t help very much when it starts to ring true.

In the end we have to look at where Zoe is right now, and where medicine is right now, and do our best to make the right decision. Hopefully we have.

A Talk with the Duke PBMT unit

I was able to catch up with a contact person from the Duke Pediatric Bone Marrow Transplant unit for an initial discussion, some vitals, and to answer a few questions. As I’ve mentioned the BMT is the part of this entire process (since her treatment started at least) which scares us the most, so many of my questions went to learning more about the unit’s experience and outcomes.

First, some links with more information for those interested:

The National Marrow Donor Program


Duke’s Pediatric Bone Marrow Transplant Unit

Our contact with the PBMT unit, Ms. C, was very helpful and went really far in discussing some of my issues, further than I expected to be quite honest. It was very reassuring despite not getting to some of the answers I was most after, due simply to her not having access to some of the outcome data.

We talked about the blood and marrow donor issue, and what was considered the best donor type. Maya is, as Zoe’s sibling, the clear favorite as a donor. Her chance is 25% to be a match to Zoe’s marrow. Next after that is what’s called Unrelated Cord Blood. Cord Blood comes from the umbilical cord just after birth, and is more often than not discarded. It contains blood extremely rich in Stem Cells, which are used for a number of purposes in both disease and, in our case, treatment — the cells work in place of matched marrow donor material. We were not to my knowledge offered a chance to save our blood with Zoe or Maya, but apparently it’s common ┬áto be offered a for-pay service to bank the blood for circumstances exactly like this we now find ourselves in. I doubt we would have availed ourselves of such a service, but looking back I can at least understand it’s use now.

The cord blood that is saved after childbirth (not for a fee, simply by hospital practice in some facilities) is then placed in a cord blood bank after whatever process they use to determine it’s safe to use, and when people need it as Zoe now does, the doctors treating whoever needs it apply to reserve matching blood for the procedure. In our case, after Maya’s blood is tested if she is not a match, Duke PBMT will immediately reserve matching blood for her procedure from one of the banks. I’m told that unlike the past, finding matching cord blood for this procedure is not an issue for certain demographics — minorities tend to have a lower chance of finding a match, but I don’t know the specifics of why.

Ms. C and I talked statistics.

  • Duke has done a grand total of 14 BMT procedures for patients suffering from HLH based on the information available to her. This is a rare disease indeed.
  • Of those, the oldest patient was 3 years and the youngest was 4months; consider Zoe will likely be around 5-6 months when she has hers if we have to proceed
  • The majority of patients were under 1 year at the time of the BMT
  • Statistically younger patients have better outcomes; they have recently seen studies to indicate that this is due to higher donor material concentration — smaller, younger bodies receive a more concentrated donation during the transplant; Ms. C mentioned that in some cases they give a double dose of sorts to ensure there is sufficient material
  • The first patient was treated in 1993, and since then there have been around 1 or 2 per year with some HLH-free years at Duke
  • 10 out of 14 patients had unrelated cord blood
  • 2 out of 14 had sibling donors
  • 1 out of 14 had a related partial match donor, and the last 1 of 14 had an unrelated partial match donor; my guess is that both of those procedures took place before cord blood was more standard

All of the patients we discussed had similar prep regimens, however Ms. C stated unequivocally that the level of care in general has risen substantially since 1993 when far less was known about the disease. Zoe will benefit from the progress doctors have made in recent years.

Our next steps with Duke will entail a phone call with Dr. M of the PBMT unit to dig a little deeper into our questions. I suggested we wait until after they had received all of our blood for testing, so that we’re not asking questions that they can’t provide answers to. After that, if it becomes clear that Zoe will need the BMT, we will look at traveling to Durham to meet with the doctors and coordinator to work out the fine points.