Understanding The Treatment Process

During the pre-apheresis visit they handed me the consent document for the transplant process. They wanted me to take it home and read it before we returned for apheresis. I read it last night. I couldn't sleep after that. All the drugs and the associated risks are mind bending. Imagine reading back-to-back all the possible side effects from all the medications and medical procedures you've ever taken/had and then consider chosing to expose your child to all of them in one concentrated period of time. It's emotionally disturbing.

The consent lays out the process and all the significant risks and side effects of the medications they typically use as well as the possibility of graft vs host disease (GVHD) and what that might mean. It's some seriously scary sh*t.

Here is the summary of the treatment plan. In general, the NIH protocol is meant to minimize the toxicity of the treatment and the reduce the chances of GVHD while providing a reasonable chance of success. The tradeoff is that the risk is increased that the donor cell do not successfully engraftment, meaning the intent of the overall process fails.

1. Apheresis
2. Preconditioning examinations
3. Pre-transplant conditioning
4. Transplant
5. Pre-engraftment prophylactic measures
6. Post-engraftment prophylactic measures
7. Long term follow-up

I've already talked about apheresis. There are risks but they are relatively minor. The exams are a full battery begins to make sure she's healthy enough to proceed.They also insert a Hickman Intervenous line so that they can get IV stuff into (and out of) her veins during the long term process without endless needle sticks. This line will stay in until the +100 day mark. This happens the week before the pre-transplant conditioning begins.

Pre-transplant conditioning takes about two weeks. First they start with a drug called Paliferman which reduces the impact of mucositis (painful sores in the mouth) caused by the conditioning process. Next she'll have 5 days of an immunosuppresant called Alemtuzumab ("Campath"). After that she'll get two days of low dose Busulfan which will kill the germ fighting cells of her immune system. They also use a anti-seizure med (Clonezepam) at this point because Busulfan can cause seizures. Next she may receive a couple "low" doses of total body irraditation (TBI). Finally they start a drug called Sirolimus ("Rapamycin") to prevent GVHD. She'll continue to take a tapering dose of Sirolimus to help prevent GVHD for up to 6 months following the transplant.

After the conditioning they do the stem cell transplant. Everyone says its anticlimactic: basically like a blood transfusion. They hang a bag of the donor's stem cells and infuse them over several hours.

For (nominally) 30 days after the transplant she'll stay in the hospital and be closely watched for infections since she'll essentially have little or no immune system. Apparently this is the worst period of discomfort for the patient with fevers, nausea, diarhea and mouth sores all possible. If infections crop up (and I'm told they will) she'll be treated with antibiotics, etc. She'll also probably have some transfusions along the way. I believe they also use some prophyllactic antibiotics and antifungals during this period.

The signs of initial engraftment typically occur in about 14-21 days. They'll be doing tests to monitor this.

After 30-45 days Elise's new immune system will hopefully have recovered enough to have some ability to fight off infections - enough that she can be released from the hospital. From that point until around transplant+100 days we'll need to stay close to the hospital so that she can be seen frequently by the medical staff. During this phase she'll be taking antibiotics and antifungals as well as the medication to prevent GVHD.

At about 100 days after the transplant most patients are ready to go home. The treatment will continue but eventually she'll be weened off most of the meds. We'll transition to a monitoring/treatment by a medical team that is closer to home. It's possible that a wide variety of things can happen after 100 days, but statistically this is apparently a significant milestone for the average patient.