nerds

Aplastic Anemia Q&A

While I have a few extra days here at the hospital, I thought I’d tell you exactly what’s going on with my disease and treatment. I still have many people who aren’t totally clear (as well as myself), but I’ve spent a good bit of time reviewing the literature like a good little nerd. So here goes.

What is Aplastic Anemia?

Your bone marrow produces all the blood cells in your body. This is happening constantly. All these cells start out as “stem” cells which then evolve into specific types of cells. The 3 major types are Red Blood Cells, White Blood Cells, and Platelets.

With Aplastic Anemia, the bone marrow stops producing these cells, and is replaced by fat in the bone. Because the marrow stops working, they call the marrow ‘plastic’.  They refer to this as “bone marrow failure.”

aplastic anemia cells

Aplastic Anemia is characterized by a significant reduction in those 3 types of blood cells: reds, whites, and platelets. This condition is known as “pancytopenia”. That’s what the doctors saw first on my blood work.

Aplastic Anemia is characterized by its severity. I have “Severe” aplastic anemia because my counts are so low. I believe when I came here I was actually “very severe.” The biggest risks from aplastic anemia are infection and bleeding. People can have “mild” or “moderate” anemia and continue onward, but no one survives without treatment in severe aplastic anemia.

While anyone can get Aplastic Anemia at any age, it’s more common in teenage males.

What’s with the blood counts?

Blood counts are critical to measuring my response and progress.  Steady (yet slow) increases in these numbers are good signs of response.

snoop as count

The red blood cells carry oxygen in the blood. The oxygen is attached to something called ‘hemoglobin’. When the hemoglobin is low, I get fatigued and out of breath. The low normal red blood count is about 4.5.  Today, mine is 2.7. Not too bad. My hemoglobin is 8.2, and the low normal is about 14. Again, not bad, but they tend to transfuse red cells when hemoglobin hits 8. I’ve been exercising regularly and hope that it’s been keeping my hemoglobin up. These cells live the longest in the blood, up to 120 days.

The white blood cells help fight infection.  There are several kinds of white blood cells. The most important cells for me are the “neutrophils” since they fight bacterial infection. Normally, they should be around 1700. But 500 is a good number to be at to reduce the infection risk. My biggest problem is that I’m only at 40. That makes me “neutropenic” and leaves me very susceptible to infection.  There’s nothing to do to increase the neutrophils unfortunately. We just have to wait. These cells unfortunately live in the blood only about 24 hours.

The platelets help stop bleeding.  They are normally about 150. When they are low (<10), I start to notice things like petichia (little red dots on my skin) or more easy bruising, or even sores in the mouth.  When that happens, I need to get platelet transfusions. Right now, I’m running at 32. These cells stay in the blood around 6 days.

petichia
My petichia before coming for treatment was pretty bad.

What causes Aplastic Anemia?

Most of the time, the cause is unknown. In fact, they really only find a cause in about 25% of cases. These causes can be genetic, environmental, viral, or even nutritional! But all those causes have been ruled out in my case. Therefore, I am among the 75% of ‘unknown etiology.’ This makes it much more difficult to fix.

They believe in most of these cases that it’s an ‘autoimmune’ disease. In this case, my own immune system attacks my bone marrow for some reason, thinking it’s a foreign body. During this attack, my specialized white blood cells in my bone marrow, called, “T-cells” are thought to destroy the bone marrow.  It’s similar to what you may have heard about rheumatoid arthritis or lupus, except that my immune system attacked my bone marrow rather than my joints or skin.

In my case, it happened relatively quickly. I had normal counts a year previous, and really didn’t have symptoms until about November, but wasn’t diagnosed until January.

What’s the treatment for Aplastic Anemia?

The treatment for severe Aplastic Anemia really depends on several factors, including age. Younger patients usually go straight to a bone marrow transplant. Older patients like myself start with “immunosupression therapy.”  Even if I needed a bone marrow transplant, the immunosupression therapy is considered a ‘conditioning regimen’ for the transplant.

Immunosupression therapy starts by killing off my immune system that’s attacking my bone marrow. As you many of you have already read, they start with an infusion of horse-serum (ATG) for 4 days in the hospital. That kills all the white cells. Then, they provide a drug called “cyclosporine” which suppresses the action of the new T-cells I’m forming. The cyclosporine lasts at least 6 months. Cyclosporine is commonly used to prevent rejection of organ transplants.

Unfortunately, high blood pressure and subsequent seizures are side effects of the cyclosporine. They are having trouble managing my blood pressure, so I have to wait a little while on the cyclosporine. That’s what’s keeping me here.

As you know, I’m participating in a clinical trial here at the NIH. Dr. Neal Young, my physician here at NIH, actually ‘invented’ and researched the ATG-Cyclosporine protocol. As part of my clinical trial, they are using a drug called eltrombopag for 3 months in addition to the cyclosporine. I started that a few days ago.

Eltrombopag is approved by the FDA to increase platelet levels. Dr. Young and his colleague Dr. Townsley were using it for treatment after unsuccessful initial courses of ATG-cyclosporine. They began to see 40% of the patients increase their blood cell levels. So, they wanted to do a clinical trial on its effectiveness as a ‘first-line’ treatment.

Other medications are an important part of treatment to prevent infections and ‘serum-sickness’.  The most important part of my treatment moving forward is to take the medications correctly (a major task within itself!) and to prevent and monitor infections.

pills

What’s the prognosis?

Untreated, severe aplastic anemia is fatal. But, there have been overall survival rates reported as high as 95% after 3 years!

Younger patients do better than older patients. There’s generally a 60-70% response rate with my treatment. Because I’m over 40, my chances of success are a little less than a younger person.  I basically have a 50/50 shot that my current treatment will be successful.  That number might be increased with the addition of the clinical trial drug they are giving me now.

Even if I do respond to this treatment, it may not be fixed. Based on the literature, 1/3 of patients ‘relapse’, and 10-15% evolve into a more serious condition, including leukemia.

If I don’t respond to this treatment by 3 months, the options are to provide another round of immunosupression therapy similar to this one, or if I have a perfect sibling match for bone marrow (which would be only a 30% chance), then I would be a candidate for a bone marrow transplant.

There’s not much else to do but be careful, calm, and pray.

keep-calm-and-fight-your-disease

 

 

 

 

11 thoughts on “Aplastic Anemia Q&A”

  1. Thanks for the update and your continuing display of courage under extreme duress. There must be about 5000 folks in your prayer chain. I have seen anything like it except when Carter and Hunter were born. This just in!!! Protestant prayers are the prayer of choice.

  2. Request: do what your doctors tell you, visualize sitting at the Brew Shack surrounded by all of those who love you, watch some Arrested Development, try catching up with me in Candy Crush, set aside your inner nerd and any further statistics, atleast for the time being. Trust that your body / your doctors/ medications / and the prayers from family, friends and even strangers are going to get you on the plane back to Green Willow, where you literally have an army ready and waiting to be with you through the remainder of your recovery. Don’t forget, not for one second #ugotdis!

  3. Phil I am praying I am a 100% match if you need it!!!
    Even though you broke multiple bones in my body as a kid, I still only want you to get better. Sending love and prayers your way tonight and every day…Marla

  4. Wow!…that is a lot to digest. I have a good feeling about all if this and ammpraying for a complete recovery…sending good vibrations to you. :)

  5. Hey Phil — Bulldog & Linda are flying just off your wing……no doubt’s……the A-A picked a fight with the wrrong guy! Baton Rogue better get rteady because Phil will be back sooner than later! — Tom & linda

  6. Phil,
    I can’t tell you how much we are pulling for you. Thanks for letting all of us know more about aplastic anemia, it has been many years for me as far as studying this disease and I did not remember much about it. I know you are one very, very strong guy and have a strong will and attitude, all great resources to go with your medical treatment. If you like, I can come prepare some fish tacos for you and maybe Andre will join me, how about that when you finish getting better? Our very, very best.

    Tom

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