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

and

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  to 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.