A day of surprises

[ConniesDad]

Hi all

Yesterday I had the first face to face consultation since the pandemic. I must say I was wary of the reasons for this but went along anyway. The consultant reported that my blood tests results were unchanged and stable and the pegasys had reduced my spleen size by 0.7cm.

He said that I am quite symptomatic but my DIPSS score is still Int1 and therefore I am not eligible for ruxolitinib. He offered me the option of a drug trial but that would then rule me out for a stem cell transplant in the medium term.

He said that he feels that the time is right to think about SCT now as I am currently quite fit and healthy, all things considered, and in his opinion the Covid situation in the UK is pretty much under control now and would be less of a risk to the success of the transplant. He feels that because of the ASXL mutation my condition will only progress and the symptoms increase going forward.

I have to go back to see him in 4 weeks and come to a decision about whether to go ahead I’m finding life just about tolerable at present (although this could be partly due to lockdown fatigue) but dread the thought of my condition getting worse. I think I am leaning more towards the SCT but with a 60% 5 year survival rate it does make me worry, although my current prognosis is about 80 months from diagnosis without the SCT.

The next 4 weeks are going to require a lot of thought and research before I make my decision so any words of wisdom from this fantastic knowledgeable community will be greatly received.

Garry

[hunter5582]

That is certainly quite a dilemma to face. In the absence of evidence of progression of the MF, I am not sure that I would assume the prognostic of 80 months from date of diagnosis actually applies individually to you. Certainly the fact that you are also positive for ASXL does increase your risk of progression. The question I would ask is whether there is any evidence that the MF is progressing. If so, at what rate. Do you know whether the mutant allele burden has progressed? I believe you were considering having that checked. I would not consider making this decision without knowing that information.

You mentioned not being eligible for ruxolitinib. Did you start taking PEGylated Interferon? It is the one med that has the potential to reduce mutant allele burden and possibility slow/delay disease progression. It is unfortunate that the formulary is interfering with the chance to try ruxolitinib. As I recall it has shown good efficacy in combination with PEGylated Interferon. It seems quite strange that the NHS would authorize SCT before trying all other options for treatment. That really does not make sense.

I sure that those more knowledgeable about SCT will weigh in to discuss their knowledge and experience. No matter what you decide, please know that you have our support.

All the best to you on the next stage of your MPN journey.

[JediReject]

Hi Garry, , it's a tough one . Are you being seen by an MPN or SCT specialist at all. I'm with Hunter in that I would of thought if the feeling was you were at the Transplant stage you would of already been on Peg or Ruxolitinib as depending on your projected disease progression you might comfortably have another relatively risk free couple of years before make or break. Having said that I can tell you that it is of great benefit to be in generally good health going to Transplant and that timing aspect is part of striking the right balance in MF cases. If we were talking about Acute Myloid Leukemia it would be different.

Five reasons I went for SCT were::

1 ) the Ruxolitinib was helping only superficially and I felt my MF was scaling up

2) I was Intermediate 2 on the scale

3) I was quite high risk of transforming to AML.

4)I was still fit enough to do it

5)I had sibling donor in waiting

It's not somethingyou need to feel pressured into in my opinion. You yourself need to be convinced the time is right after exhausting all options to manage your MF, weighing up all the expert opinion and then fully committing to the go ahead.

Don't be afraid to seek a second opinion either because it's your life on the line.

Good Luck whatever path you choose.

Chris

[ConniesDad in reply to JediReject]

Hi Chris. Thanks for your input, it’s very much appreciated. My consultant haematologist is the MPN specialist at my local hospital and is also the lead for SCT. He is quite knowledgeable and is currently leading a clinical trial to combine rux and pegasys which he did discuss with me but I ruled out due to the limitations of not being able to have SCT in the medium term. He previously said that he wouldn’t recommend SCT until the risk from continuing medical management was a likely worse outcome than the SCT. He also told me that I am extremely likely to transition to AML ‘sooner rather than later’.

He also hinted that he may have recommended SCT earlier but didn’t due to the pandemic.

I think that as I’m currently quite healthy and fit despite the heavy symptom burden I will have a better chance of getting through it and also have two sisters who have agreed to be tested as potential doners.

I’ve still got some thinking to do and may well ask for a second opinion when I see him again in 4 weeks.

Thanks again for your thoughts

[JediReject in reply to ConniesDad]

No worries . . Well that's all good that you're satisfied with your Consultant, it's half the battle. It's a real plus that he or she is an MPN specialist and SCT lead , I'm not sure you will easily better those qualifications.

However I wouldn't wait to get your sisters tested as you likely know it's a 1in 4 hit so your transplant team might have to go the register. I knew my sister was a match 8 years before I needed her donation.

That AML transitioning aspect was quite a major for me because i was informed if it happens I would need immediate high dose chemo to put me in remission from it then the SCT which though reduced intensity was more chemo prior to stem cell infusion. Taken together would reduce my chances of a successful outcome.

Worth checking out and bearing in mind.

Chris

[ConniesDad in reply to JediReject]

Thank you 🙏

[hunter5582 in reply to ConniesDad]

Seeking a second opinion from another MPN Specialist is a good idea. No matter how much I like and trust a doctor, I always get a second opinion before deciding about any surgery. Particularly one as significant as a SCT. There should be no issue at all regarding getting the second opinion. It is just good judgment.

[Ticotopia]

I hope that your sisters are the perfect match! That probably also will guide you decision process. I have all my fingers and toes crossed for you.

[ConniesDad in reply to Ticotopia]

Thank you 🙏

[MFBMT2011]

Hi Garry. I commented quite a bit on your first post last year and my views still stand. Having been written off by my initial haematologist I took guidance from an MPN specialist who said SCT was best for my future, it was just case of when. I chose not to wait as MF can move slowly or cliff edge, even going to AML which increases the bumps on the road. To me it was a no-brainer and I was passed to the SCT team. Even then your general health has to be confirmed ( a cousin of mine had ALL and was declined SCT after medical tests) and you need a donor. I didn't match my sisters but did have multiple matches so consider myself lucky as not everyone gets a match. If you don't then this can cause SCT to take a different route or make drugs an imperative. Rux' did not exist (was in stage 3 trials) when I was diagnosed and I wasn't given positive info' on other available drugs although now many seem to do well on them. Bottom line is that they do not cure, only SCT has the potential to do that, but brings with a risk.

Good luck with making your decision, it looks like a when not an if. Just don't leave it too late as that can increase the risk.

Chris (Princess Leia version)

[katiewalsh]

Hi. I agree with Hunter about considering a second opinion depending on who is advising you. I don’t know where you are but a doctor can be the specialist for MPNs & in charge of SCT and have limited experience and then there are others in the UK & US who treat a high volume of MF patients, are on top of all the current research & therapies & have great familiarity with SCT. I’d encourage you to assess just how familiar your doctor is with MF & SCT. It’s great that you’re in such good health so that SCT is an option should you go that way. Keep us posted. Katie

[ConniesDad in reply to katiewalsh]

Hi Katie. Thanks for your reply. I’m in Sheffield UK and the hospital I attend is the regional specialist hospital covering 1/2 million population in the city plus will cover the other regional towns in South Yorkshire, a total catchment area of approximately 1.5 - 2 million. I know my consultant is quite well regarded in the MPN community here in the UK and is involved in the MPN voice events, etc. He is also involved in leading research and national drug trials. Additionally he is a lead locally in SCT for leukaemia as well as MPNs. All this does lead me to have great confidence in him but I am still considering asking for a second opinion, possibly from Prof. Somerville at Christie’s in Manchester. Does anyone have any opinion on him?Garry

[katiewalsh in reply to ConniesDad]

Hi. Your current doc certainly sounds excellent. I’m sorry but I only know of a doctor at Guys in London. If you don’t get feedback on the doc you named you might try starting a new post where more will see your question. Best of luck. Katie

[Cja1956]

You do have a tough decision to make and I feel for you. I switched to an mpn specialist in 2019, after 11 years with the same hematologist. After a BMB, extensive bloodwork, and a review of my previous records, she diagnosed me with post Et Mf intermediate 1 and said I would need an SCT in about 2-5 years. She immediately made me an appointment with a SCT specialist. They sent my sister a test kit. She was not a match and they put me on the donor list. I don’t know how old you are, but I’m 65 now and I feel the older you get, the more likely you will develop other health issues. I would probably see if you can get your sisters tested as as soon as possible. I also agree with the others about a second opinion even though your doctor seems extremely qualified. Also, the place she sent me for my consultation only performs SCTs up to age 70, but other places go as high as 75. (I’m in the US). So that’s another factor. If and when she does say it’s time, I’m still not sure if I have the mental and physical stamina to go through with it. There are many variables to consider and only you can make that decision. I wish you all the best. Cindy

[ConniesDad in reply to Cja1956]

Hi Cindy. Thanks for your reply. I will have another appointment in 3-4 weeks and I will then be placed on the list for the SCT process to get underway, including the hospital assessment of my sisters, if indeed I decide to proceed at this point.

I will also ask for a second opinion at this point

Thanks

Garry