Dallas Support Group Meeting: November 14, 2015

Today's post will cover the Dallas support group meeting I attended on November 14, 2015. If I counted correctly there were 29 attendees at this meeting, not counting the two doctors. The main guest speaker was Dr. G from Mayo Clinic in Rochester. He gave a good presentation about amyloidosis that mainly covered AL and ATTR amyloidosis and the various treatment options for those types, including currently active clinical trials. The other guest doctor was Dr. Q from MD Anderson in Houston, who I believe came at the suggestion of one of his patients who was also in attendance. MD Anderson is developing some amyloidosis expertise in diagnosing and treating mainly AL amyloidosis patients.

Of the 29 attendees, there were ten with AL amyloidosis, four with familial types, two localized, and one with wild type TTR. The remaining 12 attendees were caregivers, friends, or family members. Four or five people were attending their first meeting.

Although one of the facilitators of the support group told the group about Mom's passing near the beginning of the meeting and said some very kind words, I knew I would have to speak about it when it came my turn to talk about my experience with amyloidosis. I started fine and got through the story about how Mom's diagnosis came about, and her going on dialysis, and being on, off, and back on the kidney transplant list while dealing with various other health issues. But I struggled a little when I started to say that everything seemed to be going fine until the day she died unexpectedly due to the ruptured fistula. I had to stop for a bit, took a breath, and continued with the story. That led into my talking about what I had learned about ruptured fistulas since then, which I will discuss in a future blog post.

So I made it through my first support group meeting since Mom's passing. Maybe the next one will be a little easier for me. Or maybe not.

This post will most likely be the last one of 2015. I plan on starting 2016 with the usual year in review post, and I know there are a few articles I still need to review. I do have a few other topics planned for blog posts, so my goal is to continue posting at least monthly.

See you next year!

2015 Familial Support Group Meeting - Part 2

The previous post covered the morning of Day 1 of the 2015 Familial Support Group Meeting in Chicago. This post will cover the remainder of the meeting.

After lunch on the first day, the first three presentations were from representatives of different pharmaceutical companies (Alnylam, ISIS Pharmaceuticals, and Pfizer) that have developed drugs currently in clinical trials to treat ATTR amyloidosis. I will not go into any detail on those presentations since they deal strictly with ATTR, but some of the presentations can be viewed at this link: http://www.amyloidosissupport.org/support_groups/familial_temp.html, and summaries of all the presentations can be viewed at this link: http://files.ctctcdn.com/b2465c81401/3e266c20-d1ca-428b-8ef5-c8090c7b24b2.pdf.

The next presenter was Dr. Berk of Boston University again, who discussed some alternative treatments for amyloidosis that have been studied to varying degrees. Most of those were strictly for ATTR, but one that applies to all types of amyloidosis is doxycycline, an antibiotic developed in the 1960s that was recently discovered to have some possible benefits with regard to amyloidosis. Boston University had a recent clinical trial for doxycycline that was open to patients with all types of amyloidosis. The trial is complete but the results have not yet been published because they are still going over the data. Dr. Berk was able to tell us that 25 subjects enrolled in the trial, with five dropping out for various reasons. He did not say too much about the results, but he did indicate there were significant side effects, mainly GI issues and sun hypersensitivity.

For ATTR patients the ongoing drug development is very good news, especially given the significant progress that has been made in just the last few years. As far as drugs being developed to treat fibrinogen amyloidosis, for now it looks like our best hope will be for one of the drugs that breaks up amyloid deposits of all types.

The last presenter before the breakout sessions was Isabelle Lousada of the Amyloidosis Foundation and the Amyloidosis Research Consortium. She presented the results of a recent patient survey for ATTR patients to gather information about symptoms and quality of life issues. This survey was part of an ongoing effort to communicate with the FDA in the US about clinical trials and drug development for ATTR.

The remainder of the afternoon of the first day was used for six simultaneous breakout sessions taking place in smaller meeting rooms at the hotel. Attendees could visit whatever breakout sessions they wanted to, and move around as desired. The six topics were:

• Neuropathy
• Cardiac
• Non-ATTR Variants
• Genetic Issues
• Clinical Trials
• Caregiving and Coping

It should be no surprise that Cathy T and I attended the session on non-ATTR variants. This session was facilitated by doctors Stern and Picken, who gave presentations in the morning, and by Dr. Martha Skinner, Professor Emerita of Boston University. It was Dr. Skinner who sat down with me and Mom in June of 2010 and went over the results of Mom's Boston evaluation, called us the following month to tell us Mom had fibrinogen amyloidosis, and then called me in February of 2011 to tell me I had tested positive for the mutation. So I certainly have a special fondness for her, and in fact the only time I really teared up this entire weekend was when Dr. Gertz introduced Dr. Skinner before the breakout sessions and said some very kind words about her contributions to amyloidosis research and what she has meant to the amyloidosis community. (Or maybe my eyes got a little watery at that moment due to allergies. It's hard to know for sure.)

I am sure our breakout session was one of the smaller ones, with approximately 20 people attending in addition to the three doctors. There was not a lot of discussion about fibrinogen amyloidosis, but we did hear something interesting about liver-only transplants for AFib. You may recall from previous article reviews that there is some disagreement in the medical community about whether or not a liver-only transplant is ever an appropriate treatment for fibrinogen amyloidosis. Specifically, the National Amyloidosis Centre (NAC) in London has been reluctant to recommend liver-only transplants, instead opting for combined liver and kidney transplants for patients who are good candidates for that. Dr. Stern stated she had spoken about this to one of the doctors at the NAC, who said he would recommend a liver-only transplant in healthy patients under the age of 50. That age cutoff seems a little too low in my opinion, especially considering patients with ATTR get liver transplants into their 70s, but at least the NAC is willing to consider it and hopefully over time relax their criteria after some successes.

That wraps up Day 1 of the meeting. Day 2 was the Question and Answer session with the doctors, where attendees can submit their questions to be answered by the panel of doctors. I actually submitted the same question I submitted at the 2013 meeting, hoping to get a little better answer than I got in 2013. Here is my question:

An asymptomatic carrier of a genetic mutation develops symptoms and has a biopsy which is Congo Red positive for amyloid. Does the amyloid from this biopsy need to be typed by more advanced analysis such as mass spec?

I do think this question was answered (and asked) better this time than it was in 2013. Dr. Dispenzieri said Mayo would also do mass spectrometry on it to confirm the type, and they do mass spec on all amyloid biopsies anyway. Dr. Berk said further analysis of the biopsy would not be necessary if it fit within the clinical picture of what would be expected in the early stages of the disease. He said he would want to rule out any other cause of the amyloid, such as a plasma cell dyscrasia, so he would probably do a bone marrow biopsy to rule out AL amyloid. So that is good information to know in case I ever start developing symptoms and have a kidney biopsy that is Congo Red positive for amyloid.

The summary of the whole Q & A session is available in the PDF file at this link: http://www.amyloidosissupport.org/support_groups/fam_qanda.pdf. You can see that the questions cover a very wide range of topics, usually generating some interesting discussions. There was one question about the CPHPC trials being done in the UK, and Dr. Dispenzieri spoke about that. She said the results are encouraging and it is definitely safe, but a lot more work needs to be done. I do plan on reviewing the recently published article about the Phase 1 trials for CPHPC+antibody early in 2016, since some of the participants had fibrinogen amyloidosis.

That's about it for the 2015 meeting. My trip home was uneventful, unlike the trip home from the 2011 meeting when my luggage was destroyed, or the 2013 meeting when Mom started throwing up after we picked up our bags and then made a trip to the ER the following day.

The next post will cover the November support group meeting in Dallas.

=====Monthly Blog Status Update===== 

As of November 30, 2015:

Total posts: 160 (2 in November)

Total pageviews: 31,800 (~1400 in November)

Email subscribers: 14 (increased by 2)

Total number of countries that have viewed the blog: 106

No new countries visited the blog in November.

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Edit 12-31-15: Corrected country count.
Edit 3-24-16: Corrected typo in the penultimate paragraph.