Myelofibrosis: What factors lead to treatment?

Hi Betsy, I think one of the more frequently repeated frustrations I see regarding blood cancers relates to ‘Why is there a delay in treatment?”. For some conditions, where disease progression is slow, active surveillance (watch and wait) is a better option than jumping in with medications. Studies have shown treating some of our blood conditions early, exposes patient unnecessarily to drugs and side effects and it doesn’t change the outcome of over delaying the intervention. With more aggressive cancers, then action is taken immediately of course.

When necessary, medications are given to help minimize the discomfort from MF symptoms…such as what you’re experiencing with the enlarged spleen. That’s why your doctor is suggesting Ojjaara to help reduce your spleen size.

I just happened to find a very recently updated article this morning on Healthline.com . It jumped out at me and seemed timely for this new MF conversation you started. ☺️
Healthline.com:
How is myelofibrosis treated and managed?
https://www.healthline.com/health/how-is-myelofibrosis-treated-and-managed
You brought up a really good point in the last paragraph about people more concerned about having the SCT, which could potentially cure their MF vs continuing to take medication while the MF progresses and perhaps eventually may no longer respond to drugs. However, there are many lines of treatments available for MF patients which would be considered before a BMT/SCT. A bone marrow transplant is generally the last resort because it can come with its own set of side effects and risks.

Another article from Healthline you might like:
https://www.healthline.com/health/managing-multiple-myeloma/when-treatment-stops-working
I can’t remember, Betsy, are you considered in a higher risk group so that a transplant is pretty much a given?

Hi @davi0937 ,
I am very sorry your spleen is bothering you and maybe something going on with your heart. Honestly, I have not had that happen to me still yet after 1.5 years of being diagnosed with PMF. I do not have any symptoms still, but consistently high platelets and slightly high white cells on lab work. I have normal red cells and hematocrit. The day of my bone marrow biopsy in 2/2025, my lab work had mysteriously lowered (by 400 platelets) with me taking nothing, slightly low red cells, and higher white cells. That all went away on labs done after which are very similar to labs done before my bone marrow biopsy so super strange! I believe you posted earlier that you have CALR mutation and are the same age as I, 66, with upcoming birthdays soon just as I do, but you have some other mutation that puts at more risk for progression and maybe that’s why you want the bone marrow transplant as soon as possible? The bone marrow specialist I saw said he does not recommend transplant ahead of need for one as it has its own risks also. I hope you can find out when it is the right time for you to have one and it goes well. Maybe you can get a few different opinions?

@1pearl Yes - you are right the MDs a re recommending wait. I have MPL (10% of PMF have this). CALR is less risky. I just don’t like wearing my body down with disease waiting. I want to go into transplant strong. I did get a second opinion at Univ of MN a few weeks ago. I am seeing a transplant team 9/1. I haven’t told my Mayo team. It’s interesting that when I have visits I have to ask questions instead of them helping me to understand. I sent my Mayo MD a question about using risk generators and what my risk looks like and he didn’t directly answer me. I feel like if I had had my transplant last year at 65 (before I had a heart murmur) it would have been less difficult. I need to look at the information Lori sent before I conclude anything. Wishing you well! Betsy

@loribmt
Thank you Lori for your response. I’ll take a look at the links. I’m PMF - MPL with genomic SRSF2. This puts me into intermediate risk primarily to transition to AML. My blasts are pretty low right now.

The fact is I feel pretty good! Just leaving on the ferry from camping on Madeline Island. I want to completely rely on my team like you did but it seems like I’m not getting clear information. Maybe I’m not asking too right questions? I’ll keep you posted!

Thank you again for your mentorship!

Hi @davi0937 ,
I am sorry about my mistake of thinking you have CALR. I totally understand why you would just like to get the bone marrow transplant done now if doctors are saying you are increased risk for problems because of mutations you have. It is very frustrating when doctors who are supposed to be helping us just do not really seem to be helping us at all! I am frustrated with the ones I have seen too!

@1pearl - I think there is a middle way for me. I most likely am doing too much thinking and researching. I need to do a better job of organizing my questions and get comfortable with the incredible expertise. @1pearl i continue to be grateful for you being a part of this journey!

@davi0937 Hi Betsy, Even if we ask the right questions the answers may not provide all the answers. Hah, how’s that for sounding like a politician. In actuality, our blood conditions are so complex, filled with nuances that aren’t always predictable. Our doctors have research protocols, statistical information, education and experience to rely on. But they also have to be as flexible as we are with adapting the right treatment to our needs. None of us are ‘one size fits all’ when it comes to blood cancers. Don’t let that stop you from asking questions.

Not to get off topic, but ah…Madeline Island. Many a fond memory of classes I attended there over the years. I’m glad you had a lovely time being surrounded by amazing scenery and Lake Superior…nature bathing at its finest! Hugs.

@loribmt and others

It’s interesting that a risk calculator is used and has things like pulmonary and cardiac measurements along with other ‘age’ related conditions. If those things determine your risk then why ‘watch and wait’? The symptoms that we are asked about are all external like itching, night sweats. I’m not seeing the correlation.

I was at an event at the UM last night for healthcare executives and providers. One of the conversations I had was about the new pancreatic cancer drug extending life from 6 months to 13 months. The provider I chatted with discussed a theory a colleague brought forward- that time in clinics and treatments should be considered as well. This is an interesting concept. What we all look for is time. What does that time look like? Anyway not really a blood cancer issue specifically but interesting in the face of impact. I’ll keep you updated on my questions related to Stem Cell Transplant versus watching. Betz

I was diagnosed with Myelofibrosis (“MF”) about 5 years ago, maybe longer ago. Lost like 50 pounds and couldn’t take my head off the pillow. Not sure if I was dying. My wife says yes. Then, about 1 1/2 years later, a PetScan ordered in response to me going to the ER with abdominal discomfort revealed suspicion of Erdheim Chester Disease (“ECD”) - a very, very rare blood cancer. 11 days in the hospital for multiple tests confirmed it: ECD. But, there’s a pill for that called Cotellic made by Genentech. The pill immediately attacked my symptoms that I thought were all coming from the MF. I gained 30 pounds back, and I’m back to life. Still feel anemic, although my hemoglobin is in the 12s. Still fighting the fight. I’m 72.