HSCT...what does that really mean?

The Procedure

It's called hematopoietic stem-cell transplantation (HSCT). The words "stem-cell" and "transplant" are a bit misleading. This is not the type of regenerative treatment that we all read about in the news nor is Rachelle getting cells from a donor.

The positive results from this treatment are produced by using chemotherapy to very strongly suppress Rachelle's immune system. The objective is to kill the components of Rachelle's immune system that have the "memory" to target her myelin. By erasing this "immune memory" the hope is to essentially stop the auto-immune disease process.

Once the immune system is suppressed with chemo, Rachelle will get a transfusion of her own stem-cells which help facilitate the healing of her immune system. These stem-cells were collected 2 weeks ago and are being stored until she is ready for the infusion. Hence, the name "hematopoietic stem-cell transplantation" which means "getting a transplant of your own stem-cells".

 

Is this the same treatment used for cancer?

That is an excellent question! The answer is yes and no.

Rachelle's treatment is based on the procedure used to treat various for forms of cancer. These procedures are called bone marrow transplants or stem-cell transplants. Developed 40 years ago, 50,000 cancer patients per year undergo these procedures.

For cancer, the goal is to fully kill the entire immune system including the bone marrow where immune cells are produced. To accomplish this, radiation is often used in conjunction with a more powerful chemotherapy cocktail. These protocols are called "myeloablative" and patients often require a bone marrow transplant from a donor to survive.

Unlike cancer, the protocol used to treat CIDP is less toxic. For CIDP, the target is only the "immune memory" and trials have shown that this can be accomplished with a less toxic protocol. Therefore, Rachelle's treatment is considered "non-myeloablative" (also referred to as transplant-lite) which is less risky than most cancer protocols.

The Process

Simply stated, Rachelle is getting chemo. She will experience many of the side-effects you know about including losing her hair (as you have already seen).

Transplant (3 Weeks): Rachelle will be admitted to Northwestern hospital and will be inpatient for the entire duration of this phase.

The transplant starts with "conditioning" which is the process of suppressing (killing) her immune system with chemo. For 5 days Rachelle will get infusions of cyclophosphamide and rATG.

After a day of rest, Rachelle's harvested stem-cells are infused back into her. This is considered the "transplant".

For the next few weeks, Rachelle will remain in isolation in the hospital as we wait for her immune system to start to recover ("engraftment"). This is the phase when she is most at risk of infection since she essentially has no immune system.

 

Recovery

Once Rachelle's blood levels start to show signs of recovery she will come home. For the first several months she will need to be very careful as her immune system will still be compromised. She will avoid sick people, crowds, be careful of what she eats, etc.

Eventually, her new immune system will strengthen and the risk of infection subsides. However, it will take as long as a year to fully recover from the effects of the treatment. During this time she will most likely be tired, experience some pain and a few other side effects.

Schedule

All of the testing and treatments are done in Chicago at Northwestern University Hospital. Rachelle's Doctor is Dr Richard Burt and his department is called DIAD.

June 11 - (approximately) June 27: Transplant

Can I send something?

That is so nice of you to ask!

It's best to send things to our house as they are very strict about what's permitted in the hospital isolation ward. For example, you can not send flowers, balloons, fresh fruit or other perishable food items as they can all pose a risk of carrying bacteria.

Feel free to email/text us for our home address. 

Things to know

CHEMO

Worst part is the Lasix (3X per day) and fluids (24 hours). The purpose is to flush-out the chemo. We're talking 15 bathroom trips per Lasix dose.   

First dose is at 6AM and the last is 10PM. No chance of sleep for two hours after.

The Blood Cell Count is the transplant scorecard. It provides insight into Rachelle's status and progress. 

Each morning, around 5AM, blood is drawn to run labs. It's a full work-up but the primary focus is to count four types of blood cells.

1) White Blood Cells (WBCs): Many different cell types which together comprise the immune system.

Normal Range: 3.5 - 10.5

Data Significance: Provides a general measurement of Rachelle's immune strength. However, this is not a critical metric for monitoring the engraftment process nor guiding infusion decisions.     

2) Neutrophils: A type of WBC that are the frontline defenders against bacteria and fungi.

Normal Range: 1.5 - 8.0

Data Significance: This is the most important metric. Once the neutrophil count falls below .5, Rachelle is considered "neutropenic". She is most susceptible to infection and feels the worst.

Rachelle will start to feel better as her immune system comes back online. The transfusion of her stem cells on Day 0 helps facilitate this process. 

"Engraftment" is accomplished when the Neutrophils climb back above .5.   

3) Platelets: Aid in clotting to prevent excessive bleeding.

Normal Range: 140 - 390

Data Significance: A transfusion of platelets is administered when the count falls below 20. 

4) Red Blood Cells (Hemoglobin): Carry oxygen from the lungs to all the cells in the body.  

Normal Range: 11.6 - 17.5

Data Significance: A transfusion of whole blood is administered when the count falls below 8.


Expected Blood Count Drop and Recovery

Putting it all together, the Cytoxin (chemo) and rATG (anti T-lymphocyte) at first cause a slight rise in blood cell counts but then drive a steep decline.

Counts bottom out around Day +2. Rachelle will be pretty sick at this point. Once her blood cell counts start to increase she will feel better. 

The infusion of her stem cells on Day 0 help facilitate recovery and shorten the amount of time she is Neutropenic. 

Engraftment is accomplished when the Neutrophil count >.5 and occurs around Day +10. This is when Rachelle goes home.

The below graph depicts the process. 

 

Chart format from George Goss.

The Count

 Note: The naming of days using negative numbers prior to transplant and positive numbers post transplant is how the medical team tracks the process.

There are a lot of different infusions - hydration, diuretics, pre-meds, steroids and chemo. Between all the bathroom visits from the fluids and changing the bags, it's going to be hard to get much rest. 

The PICC line is a thin catheter that runs from her upper arm to her heart. It's used in the hospital to administer IVs, fluids, meds, etc., and can remain in place for weeks compared to a standard IV that lasts 3 days.

The line is about the diameter of spaghetti compared to the neck vas catheter which was more like a slurpy straw.