Promacta -- Yea or Nay?

[David M]

All,

Here's a question re: Promacta...

I'm 60 years old, in pretty good health -- other than having moderate aplastic anemia. My situation is (so far) we are monitoring my blood counts every few months, and over the years (since roughly 2000) they have been pretty stable -- low counts, just not dangerously low.

My HGB tends to run in the 9.0 - 10.0 range... I believe 14.0 (and above) is the "normal" for a male my age. Although I have the stamina to go to work every day, to do yard work (if I take lots of breaks), and do some moderate exercise (again, with lots of breaks), my endurance is annoyingly short. I find myself huffing and puffing and having to sit down after a short time of exertion. I'm not a total slug at this point -- I still get up and about and do things that require energy -- I just tire out too quickly... and I sometimes talk myself out of taking on some tasks that I would have done if I felt I had the energy (if that makes sense).

Overall, I'm not on any medications, not needing transfusions, have no abnormal bruising or bleeding, and am not having overly frequent infections (just the normal amount anyone would have). Platelets tend to run in the 40-80 range; neutrophils in the 1.1-1.5 range; HGB in the 9.0-10.0 range.

My question is -- should I request my hematologist to prescribe me Promacta? Would the reward be worth the risk? If it boosted my red counts to the point my HGB went up to ~14, would it really make that much difference? How do I make this decision?

Any wisdom you folks can share would be welcome.

Thanks in advance!

[Sally C]

Hi David,
I can't advise you but I will give you my husband's experience with Promacta.
After over 125 blood/platelet transfusions, he was the first MDS patient at NIH to be in the Promacta clinical trial. They had great success with AA patients. He started Promacta in the spring of 2011 and stopped in August, 2012 when his platelets hit 100,000. Here we are in 2024 and his reds are usually normal or near normal, his whites always normal after having neutrophils hitting 0.0, and his platelets hover around 80,000 - 90,000. He has not had a transfusion since. He had no side effects while he was taking it. It truly performed a Miracle for us.
Please keep us posted if you decide to try it. All the best,
Sally

[Neil Cuadra]

David,

The primary use of Promacta (a brand name for eltrombopag) is to boost platelets, and it's benefited many patients. But if anemia (low HGB) is your primary concern, then among available drug treatments I'd expect an erythropoiesis-stimulating agent (ESA) to be a more likely recommendation from your hematologist.

ESAs include Aranesp (a brand name for darbepoetin alfa), Epogen (a brand name for epoetin alfa), and Procrit (a brand name for epoetin alfa).

In my layperson's opinion, wait and watch is still a reasonable approach to take, even though slightly low HGB doesn't give you the stamina you've previously had. It may not be worth risking the side effects of an ESA or other drug to try to increase your red blood cell count, when it's currently "low but livable."

Many people live with low counts by making small adjustments in their lifestyle: a little less strenuous activity, taking care of your diet, exercise, and sleep, avoiding activities likely to lead to bumps and bruises, etc. If you stick with watch-and-wait, and your counts drop into a range of more concern, you and your hematologist may then lean more toward starting a drug treatment.