Paraneoplastic Syndromes

[Gingergrrl]

This exchange is getting more like a PM, where we should probably continue.

I agree and happy to discuss via PM instead. I never know if this info could help someone else down the line but am thinking my situation is too obscure and maybe just applies to me?

I don't really know how to get medical professionals to move faster, other than via emergency departments, which you will want to avoid if possible. You should have a local doctor to take over your case, and follow up on the findings you have. At present the problem is not that your doctors are taking things easy, it is that you are in limbo where no one is responsible for what happens next. Some professional has to be responsible for the treatments you need.

My goal 100% is to have a local doc take over and hoping it will be the one recommended to me. Am just trying to get a sooner appt with him so I can get started. Will also have a new PCP next month but do not expect him to know about the antibodies and will still need the NM specialist. And agree with you that going to an ER or hospital could kill me with MCAS and avoid this at all cost.

Once you have that new doctor I'd say IVIG is a reasonable intervention with limited risk. It may not work, but it is unlikely to precipitate a crisis. If it fails, you still have the option of plasmaphaeresis, even before you get to B-cell depletion experiments. (I don't think your concerns about exchanging "good plasma" are relevant if the preceding treatment did not produce improvement. However, if you feel strongly about it you might go directly for plasmaphaeresis.) If a doctor who is responsible for you feels you are still sinking after trying these standard interventions, then there is a good case to be made for quasi-experimental treatment when the alternative is grim.

Am curious which treatment you view as quasi-experimental? Is that RTX, or plasmapheresis or something else?

This is what happened in the case of Rebecca2z. She has a diagnosis of EDS, but I don't think that played a major role in the decision to try Rituximab. It was just that her condition was bad and deteriorating, and her doctors were out of good traditional options. Since patients who had similar conditions had improved, and there were published reports of improvement, and no other options were promising, this was a typical justification for off-label quasi-experimental use of an existing approved medication. Her life was at risk, she was aware of the risks, and the option of doing nothing was unacceptable.

Yes, have spoken with her a lot which makes me think you might be referring to RTX? Am afraid my life will be at risk if I do nothing and my breathing continues to deteriorate at the rate it has in the last two years. Am willing to try risky treatments and all I can do now is wait to see local doc and keep trying for sooner appt. And in the meantime, am trying to learn and gather as much info as possible about all of these treatments.

Don't worry about answering here, I know this is very specific and probably not helpful for anyone else. I really appreciate all of the feedback that you have already given me.

 

[anciendaze]

I was definitely talking about B-cell depletion, and thinking about Rituximab. At this point we aren't entirely sure this qualifies as an autoimmune disease, though that is the best guess.

The effectiveness of Rituximab/Rituxan is still a research topic in a number of autoimmune diseases. Rituximab was originally approved for the treatment of lymphomas, and I know people who were treated with it for B-cell lymphomas and improved. Technically, any use to treat other conditions is an off-label use, though some are better recognized than others, but restricting medications entirely to approved uses would almost be impossible. Example: For many years Lithium salts were only approved to treat mania, but were in fact used to treat bipolar illness regardless of which phase patients were in. The question is largely one of legal and professional liability. This is one reason doctors are paid for expertise and judgment. If you limit their professional options as if they were clerks you undercut the entire point of having highly-trained medical professionals.

 

[Gingergrrl]

I was definitely talking about B-cell depletion, and thinking about Rituximab. At this point we aren't entirely sure this qualifies as an autoimmune disease, though that is the best guess.

Thanks for clarifying. I wrote something in the PM prior to seeing this. I was unclear if these Ab's are considered an autoimmune disease, or the pre-cursor or a paraneoplastic syndrome or how to label them. I know labels do not matter but they help me to understand things better.

The effectiveness of Rituximab/Rituxan is still a research topic in a number of autoimmune diseases. Rituximab was originally approved for the treatment of lymphomas, and I know people who were treated with it for B-cell lymphomas and improved.

So it was originally a form or chemotherapy but has evolved? Was discussing this in another post where I was told it is not chemotherapy which continues to confuse me b/c my doctor said it was. Am trying to figure out if it could help my situation (regardless of the chemo or non-chemo label) and struggling to grasp this part.

Technically, any use to treat other conditions is an off-label use, though some are better recognized than others, but restricting medications entirely to approved uses would almost be impossible.

I am totally okay with off-label uses of meds and want to choose whatever has the greatest chance of helping me.

 

[anciendaze]

The word chemotherapy has evolved through a number of meanings. There was a time when the only treatment which could actually prevent or cure an infectious disease was with quinine for malaria. This was not thought of as a chemical, because it was extracted from plants which made it naturally for their own protection.

Originally chemotherapy meant some pretty crude stuff used to poison pathogens like treponema pallidum which causes syphilis with synthetic stuff like mercury or arsenic compounds. This had limited success, even in known infectious diseases like TB, and fell into disfavor as a general approach to other diseases until the discovery of Protosil, the first commercial antibiotic. This set of a gold rush for new specific antimicrobial agents which has never ended.

When treatment of cancer with toxic substances (that were hoped to be more toxic to cancer cells than to healthy ones) came along, there was still living memory of the previous meaning of "chemotherapy", and this then shifted the common meaning to be cancer chemotherapy, not to be confused with "wonder drugs" like antibiotics. This seems to have had some marketing influence.

I suspect we are not at the end of this process, so that new terminology for treatment with artificial chemicals will emerge with new approaches.