So what do you choose as a second line treatment

Was wondering if anyone had any insight or recommendations....


I have been on 90mg Pred for 5 weeks now, and count at 140 as of yesterday, so the Doctor has decided to start tapering to 50mg as from today. As this time around Pred hasnt increased my counts all that much from last week, (altho bearing in mind I started with a 2 count - 5 weeks ago and now 140 - so I ll take that anyday!!)

So now I am researching...... as I guess I am going to need second line treatment once the Steroids are stopping, as last time I stopped Steroids and my count held at 127 but then fell down to 2, so I guess it means I need 'help' to stabilise the count - and

My question being, so the doctors need to decide ( and I guess I need to make a decision), what treatment, now do they base their treatment on the following and how do they know...

Do they give you treatment to make your bone marrow make more platelets - if indeed your bone marrow is not producing enough OR do they give you treatment to make your body stop destroying platelets OR is there treatment which covers both.
Surely before they offer treatment they need to know what is actually going on with the platelets in your body.

I have been on this website looking at treatments and how do you know which option is best... I know there are side effects to ALL of them and some worse than others, so a bit worried which one to choose, (and as a patient I guess I have a say in the choice, I dont have to accept what the doctor offers?) luckily in the UK and on NHS I think most of them are available except perhaps the Nplate which I am not sure the NHS stears towards that for cost implications - could be wrong.

Or is this a trial and error for second line treatment you pick one which you think with your doctor and see what results you get and work it from there, and are you on these treatments for life? Thats the scary thought?

I know some people on here feel they can live with low counts instead of putting drugs into their system, but my only problem is I drop and I drop very quickly and it means time of work etc etc, and its not viable.

Any thoughts much appreciated.....

Fristly, it is now known that most, if not all, patients have both a platelet production and destruction problem. The antibodies that mark the platelets for destruction also damage the megakaryocytes which make new platelets. So you can attack from either direction. Usually in the UK you would try either an oral immunosuppressant like mycophenolate or the intravenous rituximab next. Both these aim at preventing destruction.

Nplate, which aims at forcing the body to make more platelets, is by far the best treatment I've had, but is no longer available on the NHS unless you've either had a splenectomy or a splenectomy is contraindicated. If a patient wants to use Nplate before splenectomy it is usual to get the indium study test to see where the platelets are being destroyed. If in the spleen, a splenectomy has more chance of working than if in the liver. If the latter and the liver is the problem then that counts as a contraindication to splenectomy and Nplate becomes available to you. There may also be other reasons that surgery is not recommended but finding a reason if you are otherwise healthy can be difficult.

Splenectomy is going out of fashion in the UK and the top doctors no longer do them. But the pros and cons of splenectomy is a whole 'nother discussion. It's a subject I feel very strongly about.

The most recent treatment guidelines are at www.bloodjournal.org/content/115/2/168.full?sso-checked=1 but be aware that they are international guidelines and the order of things is therefore slightly different than it would have been if the UK had written them on their own. ie splenectomy is still in there!

Did you also see the little video talk by Dr Provan at fhs.mcmaster.ca/medicine/hematology/ITP-2010/player.html

Thanks Ann - does the NHS offer the scan can you ask for it or do you have to be heading towards the Splenectomy route and the scan is the next option.

I have heard - but could be wrong, that there is a slight chance, that if you had a splenectomy there could be a chance later on the liver takes over and destroys platelets then you sit with the same kind of problem.

I am not keen on the Splenectomy route - well not now anyway, I think I am too new to this whole ITP situation to consider that right now.

Ill go check on the website and see if I can look at the video - thank you!

You can get the scan on the NHS. It's only done in a few places, London, Manchester, somewhere in the south west, Plymouth it may be. I was living in Canterbury when my haematologist suggested having it. I knew I would never agree to splenectomy but agreed to have it out of interest. I also got the result I wanted and it stopped the doctor from ever mentioning splenectomy again.

Unfortunately, there's more than a slight chance that a splenectomy will fail either immediately or later. That's one good reason never to have one. But the scan is useful if you want to try to get Nplate.

I dont know much about Splenectomy but to be honest what I have read, I dont like and I just think its too risky for something thats not 100% guaranteed - just my thoughts.

I will try ask for a scan, see what they say.
I read the article - thanks so much regarding the treatments very useful, just want to be more aware so when I see the Doctors I know what they are talking about.

The usual protocol for treatments do they start you on second line and for a limited time and then see how you fair or is this treatment for 'life' how does it work?

When I was dx'd in 1974 only treatment was prednisone and then splenectomy. No other treatment existed as far as I can tell. Feel fortunate to have as many relatively healthy years as I have had.

Fiona:

Ann did a great job of explaining things, so the only thing I want to add is this. Yes, it is pretty much trial and error as far as treatments. There is no way, at this time, to know ahead of time which treatments will work and which ones will not, so you try them and see. Most people respond to Promacta or N-Plate, so one of those is never really a bad choice, especially since you've already tried Prednisone and Rituxan.

Many people do drop fast, so you are not alone there. Both Promacta and N-Plate are designed to keep counts around 50k. They are not used to normalize counts. Some people have also had remissions from those drugs, so taking them does not mean you will be on them for life.

I live the uk and my hemo put me on Revolade (promacta). I have been on it for about 3 months now (25MG daily) and so far my count has stabilised between 100 and 120 (for the last 2 months) with no side effects.

Fiona,

I am very similar to you. I didn't respond to Rituxin or prednisone. I wasn't keen on spleenectomy at the moment ( I do want to eventually have a baby, so I might do it since you can't be on many of the treatments). However, for one I am on Promacta and am doing really well on it. I also respond to Nplate, but had some bone pain on it and was tired of weekly CBC's.

Wonder if anyone can assist...

So I am on Prednisolone for the 2nd time around, due to the fact that I came off steroids the last time I was 127 then crashed in 2 weeks down to 2 - so was hospitalised. I then was given steroids again, so now tapering off them, I am on 20mg and counts are at 171 this week.

I saw the Consultant last week and I thought I would then discuss plan B - and other treatments.
I have only been on Prenisolone nothing else. He wants to taper the Prednisolone quickly and then towards the end slowly do it and see what happens. However When he dropped me from 35 mg down to 20mg I went from a count of 223 to 171 in a week.

He says depending on how quick I drop to the end and what happens will determine what treatment he decides on. But I dont want to get to the stage of possible crash again, I want to have a plan in place, so a bit nervous. The funny thing is the Haematologist I see on a weekly basis is not in agreement with the Consultant as she thinks I need to start 2nd line treatment now so that I dont have a crash again. she spoke about mycophenolate as did he - but she says its not ideal and perhaps that is why he wants to wait and see as she feels I am young and this drug can later on cause cancer.....

Any thoughts? What should I be going for and should I be waiting to see .... Just fustrating as I dont want to end up with a 2 again and spend a week in hospital trying to get to double figures again.

Fiona:

Personally, I think you are being tapered too fast. Counts tend to drop faster that way. However, going from 223 to 171 isn't something to worry about. People can sometimes go up after a taper; it can go either way and you can't base it on what happened the last time. It's not common to treat on top of what you are doing to prevent a drop. Most would just keep going until it is clear that the counts are down because if you treat to prevent drops you will never stop being on meds. Managing ITP is a balance between treating and waiting the treatments out. It is good to have a plan in place though because most treatments can take time to work, but most don't begin to treat until they hit about 30k, some less than that. You do respond fairly well to Prednisone, you just haven't attained remission.

It is very typical to have to try more than one treatment. Most people do. The choice may seem overwhelming, but the fact that there are choices is a very good thing. ITP treatments have come a long way. Mycophenolate won't get counts up fast and I agree, it's a bit toxic and isn't side effect free. I've used it for Lupus and need to consider it again soon, but I would not have tried it for ITP since there are better treatments out there. Due to your circumstances and the desire to have stable counts, Promacta or N-Plate could be good choices for you. Going to see Drew Provan is a great idea. I'm sure he will be reassuring for you.

Sandi, thanks so much you always are so re-assuring!

Why is it then that the Haematologists go for say Mycophenolate over Promacta, when I am reading up on Promacta the side effects are minimum as against Mycophenolate. Some drugs havnt even been approved, so surely they should opt for drugs which are approved and have less side effects like Promacta?
I understand it will be a try and test with 2nd line treatment and hopefully I will be able to take a drug which has less side effects AND works.
Just wondered why they would choose a drug with bad side effects and long term effects over say Promacta?

Quote; "Just wondered why they would choose a drug with bad side effects and long term effects over say Promacta?"

For some doctors, it may just be what they are already familiar with.

For myself, I chose to switch from Promacta to generic Cellcept (Mycophenolate) simply because it is far less expensive.
Promacta would cost me $375 a month co-pay...of which I have to pay all $4,500 in the first two months of each year.
Generic Cellcept costs me $10 a month. Yes, it IS all about the money in my case.
$4,380.00 actual 'money-in-my-pocket' savings is nothing to sneeze at (If someone mentions obamacare I'm going to scream).

I used Promacta for 3 years, then switched to Cellcept. In the past 2 years of Cellcept, I have not noticed the side-effects as any worse than Promacta. Different, but not really worse.


.

FionaJ wrote: Just wondered why they would choose a drug with bad side effects and long term effects over say Promacta?

A lot depends on which country you live in.

I would hazard a guess that cost is a major factor in the US as well as medical insurance coverage. In UK due the the NHS cost is not a overriding factor so lot will go down on the individual Haematologist.

I live in the UK and I am currently on Promacta (Revolade) and have been for close to 6 months now and will probably be on it for a long time coming.

The very first time my platelets drop she treated me with Preds and a course of IVIG (3 years ago). Second time round it was Preds with Revolade (9 months ago).

True - some doctors go with what they have the most experience with. I'd ask her what her reasons are and I would expect a valid answer and not a run around answer. It is actually your choice. Like Jack, I have also gone with the cheaper choices at times. I couldn't see spending my kids college tuition on meds. I am too practical for that, so chose steroids many times over something that may have been easier on me. Maybe your doctors do not want the fight to get Promacta for you....who knows?

Many people tolerate Mycophenolate well. The long term side effects are usually worse than the short-term side effects. I did not do well on Mycophenolate, even at a low dose. It took away my ability to think and I was still working then. I was sluggish and had horrible brain fog. I had to quit taking it, even at a low dose. My Rheumatologist keeps pushing to get me back on it, but that and Imuran cause me to get sick often and antibiotics don't help much. I usually end up worse off than I had been before taking those meds.

Cost is the reason that NICE in England and Wales took so long to approve Nplate and Revolade. They first tried to say "no" outright for cost reasons for Nplate but at the comments stage, there were so many medics and a few patients who argued for it, NICE decided eventually to allow its use. Each weekly injection costs about £500 for my dose and if you need a higher dose it's £1,000. So no small sum. I still have about 6 grands worth of the stuff in my fridge as they kept giving me too much. It's good to know that it's there if I need it again in a hurry.

Now that NICE has declared that these drugs should o nly be used post-splenectomy, permission for its use has to be sought from the various CCGs and it is always cost that has them refuse.

I know Sandi you said the Doctors choose medication to use for their patients, which they are familiar with, question is, what makes them decide or how do I decide, to choose a medication that either makes more platelets - like NPLATE etc or go for medication which stops the body attacking platelets, which do you opt for?

It seems like my Haematologist is gearing towards immune suppresants at this stage....

Fiona:

You don't know and neither do they. Since most people tend to have both destruction and production problems, it's a matter of which you will respond to which you don't know until you've tried it. Since that is the case, people tend to go with what they can afford and which seems to suit them best as far as time and side effects. For example, I never went for IVIG because it's too temporary, too expensive and much too time consuming. You read about the drugs, read about the experiences of others and try to come up with which sounds better to you. You can then suggest it to your doctor. Being a part of the decision instead of just taking what is dished out empowers you and makes this much easier. Most people find that a doctor who works with them instead of dictating to them is a much better fit. You have to advocate for yourself. Become informed and be firm about your decisions.

Since you seem to want stability, the TPO's would give you the best shot at that, although it may take a while. Some people do respond to Imuran or CellCept but it takes a while and they are on those drugs for a long time or until the side effects get to them. Sometimes, due to where you live and insurance restrictions, people don't have many choices but if you do, it can be up to you.

Ann wrote: Cost is the reason that NICE in England and Wales took so long to approve Nplate and Revolade. They first tried to say "no" outright for cost reasons for Nplate but at the comments stage, there were so many medics and a few patients who argued for it, NICE decided eventually to allow its use. Each weekly injection costs about £500 for my dose and if you need a higher dose it's £1,000. So no small sum. I still have about 6 grands worth of the stuff in my fridge as they kept giving me too much. It's good to know that it's there if I need it again in a hurry.

Now that NICE has declared that these drugs should only be used post-splenectomy, permission for its use has to be sought from the various CCGs and it is always cost that has them refuse.

I am not sure where you have got your information from Ann but you can receive both Revolade and N-Plate on the NHS without having to have a splenectomy. I know as I am currently Revolade and nobody has ever gone near my spleen and my hemo as always maintained that if Revolade ever fails then N-Plate will be the next course of treatment.

Some info regarding N-Plate

NICE's initial appraisas for N-Plate were completed in Nov 2009 and was given preliminary recommendations supporting the use of romiplostim (UK name for N-Plate)

In Nov 2010 in adults with chronic ITP whose condition is refractory to standard active treatments & rescue therapies or who have severe disease & a high risk of bleeding that requires frequent courses of rescue therapies, & if the manufacturer provides romiplostim with the rebate on the list price as agreed under the patient access scheme


As for Revolade, yes a cost was a factor. In 2010 the NHS rejected the use of Revolade because of costs.

Revolade was £104,100 per QALY (quality-adjusted life-year) and Splenectomised patients £116,750 per QALY.

It was finally given the go ahead in Dec 2010 as GSK (GlaxoSmithKline) offered a undisclosed discount via the patient access scheme.

The reason N-Plate being made available had nothing to do with doctors or patients demanding it but GSK dropping its price demands.

I wonder if Promacta is part of the Glaxo/Novartis deal? And what that might do to pricing?

I also wonder if GSK will raise prices now that they've been fined nearly half a billion dollars by China for bribery.
What? A drug company bribed doctors to use their drugs? Oh no, that never happens. :S

.

Unfortunately GhostRider you are incorrect. NICE decided earlier this year to change the wording in the guidance for romiplostim as it said it could not go against the marketing authorisation. You have to do some digging on the NICE website to find things, it's a confusing site.

Eltrombopag is here www.nice.org.uk/Guidance/TA293 and it clearly states "You should be able to have eltrombopag if you have had your spleen removed, or if you have not had your spleen removed because you can’t have the operation..." Note the date, July 2013. It failed in 2010. A GSK person told me that they messed up their first application. It passed when resubmitted.

Romiplostim is here www.nice.org.uk/guidance/TA221 and has been altered recently, in May 2014, to say the same.

Romiplostim was refused in the final draft in 2010. Comments were invited, as they always are, and were received. They are still on the website for the reading. I sent in my comment although I know it would have made little difference.

I also know what was said at the meeting of NICE at the final meeting when they would usually simply agree that the draft guidelines would be made final. This time they didn't do that and reversed their decision. I was there and watched and listened with open mouth!

It was later widely acknowledged that the person who had given them their first medical opinion had little knowledge of ITP and they had received poor information. Early on in 2008/09 they seemed to be under the impression that the main problem for patients was cosmetic, embarrassing bruises and so on. They were then told by experts about people whose ITP was uncontrolled and of patients who had died from bleeding. That and the Patient Access Scheme which we never did get to know much about, and still don't, had them change their minds.

And to hear them also agree at that meeting not to force splenectomy on patients made it doubly amazing. It's such a shame that they have now changed that. It doesn't matter to me as I have a contraindication to surgery but for others I hope it isn't too long before it's changed back.

Hi, I was yesterday at the Haematology Department having my weekly blood count, as I am now on 10mg of Steroids and at a count of 173, and wanted to know what they were considering as my 2nd line treatment. I also mentioned I wanted to try the platelet production type medication instead of immune suppresants. So Nplate or eltrombopag, and I was told that according to NICE it would need to be proved that I needed them. So they normally only give those to people who have had a Splenectomy - and its failed or those that cannot have a Splenectomy or I would need to be a person who always needed emergency treatment due to low count drops. ( I thought since my count dropped from 123 to 2 in a week and I was hospitilised that should prove something - guess not)

They then said that my next course of action would be IVIG and if that doesnt work then perhaps I could 'ask' for the platelet producing medication ????
My issue with IVIG is that its time consuming and having had so much time of work for blood tests and a 7 day hospital stay its tricky for me to now think of having more time of work for either a full day or 2 days for transfusion.

I have an appointment on the 14th October with the Consultant again, so I guess I may need to further discuss with him.

In which case why am I on Revolade and still have my spleen.

Looking at what you quoted you missed out a vital part.

"Who can have eltrombopag?

You should be able to have eltrombopag if you have had your spleen removed, or if you have not had your spleen removed because you can’t have the operation, only if:

- Your chronic ITP has not improved even though you have tried other available treatments, or
- You have severe chronic ITP and a high risk of bleeding that needs frequent courses of emergency treatment."

Many people with ITP will fall under these 2 categorises. There should be no issue with any anybody who has ITP and has failed to respond to first line treatment getting Revolade on the NHS.

At no point did my Hemo even mention NICE or funding or anything like that. Other then having a CT Scan about 3 years ago where it was found my spleen was slightly enlarged nobody has ever gone near it in any way shape or form.

It was a simple case that Preds only raise my count slightly so the next step was eltrombopag. I was in and out of her office in about 10 mins.

It looks to me the problem is not with the NICE but with individual Hospitals.

FionaJ wrote: They then said that my next course of action would be IVIG and if that doesnt work then perhaps I could 'ask' for the platelet producing medication ????
My issue with IVIG is that its time consuming and having had so much time of work for blood tests and a 7 day hospital stay its tricky for me to now think of having more time of work for either a full day or 2 days for transfusion.

You're medical system is different than mine - but here's what I'd say about IVIg being the next line of treatment:
1. My former hematologist [had to change when our medical coverage changed] said she never had a patient go into remission using IVIg
2. I don't recall anyone who has ever been here saying IVIg did the trick for them

My stepmother used to say "put your elbows on the counter and ask to see the manager" - is there anyone else you can see?

I agree. Long term management with IVIG is definitely a waste of time and money. I don't understand why they put that first. It's not cheap either and when people need infusions so often it all adds up. IVIG is good for a rescue treatment or if the person doesn't respond to anything else.

I never even considered that because I didn't want to miss work either. Doctors/insurance need to realize that patients have lives too.

You have been lucky Ghost. Good for you but I can assure you that it isn't the same for everyone. Revolade has actually been used very little in the UK, you are one of the lucky few to try it.

You are reading the paragraph you quoted a bit wrongly. Both having a splenectomy or a contraindication, and the lower bit about having chronic ITP etc etc are necessary. It isn't an 'or' thing.

I'm going to an ITP conference for medics next week so will hear more about it then.

Hopefully you will get the chance to find out if it a funding issue or if it is down to the individual Hemo / Hostipal / Primary Care Trust

Good Luck and fingers crossed you can find out ☺

Dear All

Wonder if any of you have any insights or great comments for me to think on...

I am still waiting for 2nd line treatment and I have since Pred had now 2 dosages of IVIG, they seem to last for about 4 - 5 weeks and then counts are low again, so at the moment we are going down and last week count of 69. (from 209)

SO.... I went to see Dr Provan in London, just to chat and get some clarity. However I was set on asking for Nplate and my Consultant said it could be applied for. Which I thought he had done, but when I had my recent consultation on the 13th January he was then only applying for it. I know Dr Provan also mentioned Mycophenolate of Cellcept (in the USA) both good choices for me.

So I was dead set against an immune supressant, and wanted Nplate. There is a chance I wont get Nplate. But when I started reading and doing research I now wonder if Nplate is the drug I want.
I know they keep platelets around 50 and close close monitoring. But I know someone here had a stroke on Nplate and I do worry about that, who is to say once you start the injections your platelets count will not fly up into 3 figures and not just 50. So I worry about that now.

I also worry about injecting myself and also knowing that I will always for a long time have to go weekly.
I know that we can live on a count of 50, but I almost want to get a medication that like IVIG or Pred pushes my counts up higher ( I know its a mental thing and I need to get around it)

So then there is Mycophenolate which Dr Provan also suggested and could work for me, BUT I know there is a stimga or there is documentation and warnings of Cancer - secondary cancer or skin cancer.

So thats worrying for me a lot, as I dont have much luck when it comes to health at the moment.
Monitoring when on Mycophenolate not sure if its done as closely as Nplate.

The big question at the moment is this.... I can start Mycophenolate as soon as about 2 weeks time as its available and just needs the go ahead from my NHS Consultant (which my Hameotologist said he would probably prefer to do that than push for Nplate)

She also suggested I email to Provan and ask him these questions regarding Cancer etc, as she has had many patients recently who have responded very quickly and well on Mycophenolate but she cannot answer the cancer question as she has not had enough patients and for a lengthy time.

So that is my dilemma start something now and finally hopefully carry on with my life or wait and hold out for Nplate - but then I dont know how long that will be... they keep saying a few months a few months.....


OR am I missing another treatment that is good?

I guess so lucky we have options as in the earlier days not many options out there...

Fiona:

First of all, it's great that you get about a month from IVIG, so you can do that until you make a decision on another drug. IVIG is buying you time here....you can use that time and not feel pressured into making a decision.

N-Plate may cause your count to be three digits sometimes, and that's okay. Going over 50k does not mean instant stroke. It is a concern, but not one that you have to stress about and lose sleep over. Most of the people here seem to do quite well with it. As for the injection part, I've never had to self-inject N-Plate but I did have to self-inject Methotrexate once a week for almost a year. It's not as hard as it seems and even though the office nurse showed me how to do it, I forgot when I went home. I watched a Youtube video a couple of times and got the hang of it quickly. The needles are tiny and barely hurt.

As for Mycophenolate, I'm not a huge fan of that for ITP but it is an option. I've tried it for Lupus with no success and I felt that the side effects outweighed the benefits. All immunosuppressants carry an increased risk of cancer, even Prednisone, and that risk is increased with many years of taking immunosuppressants. This treatment also may not give you normal counts; some only manage to get a small bump in counts. So there's that.

All treatments have risks and it's good that you are taking that seriously and trying to find one that will be the safest long-term. The problem is that you can't see the future so you won't have the benefit of knowing in advance what will be the best option. All you can do is choose one that might seem the best for you now. It's okay to live life without having normal counts. If I had it to do all over again I would not have treated to obtain normal counts, but thinking was different back then. I would have done just enough to keep safe counts. I know you have a problem with that. I've read stories of people having strokes after IVIG because counts went too high too fast so high counts are not always the best thing either. It's very difficult to manage ITP and keep the count exactly where you want it to be.

Take your time and think things through. You'll figure it out and one of these days, you'll feel more at peace with it.

Regarding the availability of Revolade Eltrombopag on the NHS in the UK. I was diagnosed with ITP last August, I have been healthy all my life and was admitted to hospital out the blue with a platelet count of 2. I was on Prednisolone for about 10 weeks and my platelet count briefly got to just over 20 before settling down around 10. Splenectomy was mentioned as a possible treatment but the Doctor recommended I choose between self inject Romiplostim or Eltrombopag daily tablet as a second line treatment. I choose Eltrombopag and my count jumped to 140 but for the last 2 months has settled around 80 to 90.
So it is early days for me with Eltrombopag but for now it is keeping my platelet count at a safe level, I have no side effects and feel completely normal.