I have had a wild ride last year beginning with a sudden decline in weight from 240 lbs - 165 lbs. accompanying the weight loss has also been an alarming increase in fatigue, cognitive ability, pain in my arms and legs, some times to the point where I can hardly feel my legs not to mention the sweats at least once a week and constant clammy hands.
I went to the doctor in the summer and my platelets were found to be 1.2 mil and mild spleen enlargement. I was diagnosed with ET and told to watch and wait and that I had no need for a bone marrow biopsy. I received a second opinion as a result because I believe my symptoms don’t match what I was diagnosed with. After some convincing we did the biopsy and discovered early stage fibrosis. This was three weeks ago now.
When I was diagnosed my hematologist immediately changed his tune kinda said he’s sorry and said I could be cured! He believes I am a perfect candidate for stem cell transplant. So today I go to the consultation with a stem cell specialist and they tell me that because there is a 10-8% chance of failure at their facility I’m not at high enough risk to need the SCT. He then tells me I will definitely need it with the next 5 to 10 years but let’s just do hydroxyurea until then to bring platelets down. I’m trying to plead with him that I feel weaker every month and that I’m losing my whole business and livelihood because I’m preforming at 50%. It gets harder to do physical work and not recover for three days after. That’s not sustainable when you have to work physically 5 days a week. I don’t want to wait until my disease progresses to do this. I am young I should not be thinking that I have 5 to 10 years left taking a drug and then doing a transplant at last minute.
I don’t understand why my symptoms don’t count in the decision. I would do the transplant in a heartbeat even knowing the risks. Because I don’t feel right and definitely feel it’s getting worse at a steady rate.
Has anyone felt this level of physical decline but been refused a “cure” ? I see that a number of patients and my doctor has said a number of his patients don’t have symptoms. But I do and they are severe. Any advice, maybe experience with pushing for self advocacy? Thank you so much in advance.
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Plasmapool
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Hi Plasmapool. As someone who is also young and working in a demanding job, with a diagnosis of ET, I understand the impact this can have on energy levels and concerns about remaining able to work. Can I ask where you went for this opinion?
What is the diagnosis? What's your driver mutation? (Jak, calr, MPL, triple negative)
That's interesting. I thought PMF and ET were two distinct entities. Hopefully you have found a MPN specialist who can walk you through appropriate treatment options. Wishing you all the best.
I would suggest that it is time for a third opinion, from a MPN Specialist. There are definitely other options to treat early stage MF than hydroxyurea or SCT. In fact, at your age, many MPN specialists would not recommend long-term use of hydroxyurea. Given what you describe, it is not surprising that the stem cell transplant center does not recommend SCT. I believe most would not.
Suggest a change in strategy. Instead of focusing on the treatment, focus on the treatment goals. Treating the symptoms that are a problem is ABSOLUTELY the central focus of treatment. Reducing risk of thrombosis is a short-term goal. You are also at risk for hemorrhage with PLT that high. Reducing risk of hemorrhage is also a short-term goal. As my MPN Specialist once said, "You really do not want a brain bleed." Improving the symptom of fatigue is also a critical goal. Improved cognition and reduced pain are also critical goals. The long-term goal would be to prevent progression of the MF.
Once you are clear with your treatment team on ALL of your goals, you can discuss treatment strategy. There are other options. There are the JAK inhibitors to consider along with the interferons and possibly other choices. There are very promising clinical trials underway that may even offer better options. These are options that you would need to discuss with a MPN Specialist, not a regular hematologist. You need a MPN-expert opinion to receive optimal care. Here are two links to MPN expert docs.
There is much more available when you are ready to seek the information. The MPN Voice site is an excellent resource and a great place to begin. Please do know that there are options to deal with the symptoms you are experiencing.
Wishing you all the best and success on your journey.
Wow what a thoughtful reply! Thank you for all these resources. I have another opinion next Thursday. You really shared a trove of information that I’m continuing to sift through. Thank you.
hi Plasmapool, I’m in similar position in being told last month to stay with hydroxy and steer away from stem cell transplant (SCT).
I sympathize with your frustration!! I was warned that SCT is running almost 30% failure rate, with 8-10% seeing mortality within 1-2 years.
I asked about autologous transplant with CRISPR correction, too. The problem there is that some of the bad JAK 2 mutation can’t be completely cleared (50%), so it’s not a complete cure.
I have the fatigue. And enlarged spleen. Inexplicably my weight went from 180 to 225 in last two years. I would envy your weight loss, but it does seem concerning.
If you are under 60, then waiting on the transplant doesn’t seem alarming. Be calm! It seems you have very good chance of beating this thing back completely.
Sorry it’s making work hard. I can identify with that challenge too!! Aching joints, cognitive focus diminished and fatigue!!
Hi, Plasmapool, I understand your frustration. I was diagnosed with ET at age 52 in 2008 and progressed to MF about 5 years ago. Now I’m classified as MF3 which means I’m fully fibrotic. In the last 10 or so years there has been significant research into this disease and there are several medications on the market for us now. You definitely need to find a MPN specialist to help with some relief of your symptoms.
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