As a patient who went through the ringer trying this drug and that drug looking for an answer back in 2006, before the autoantibodies and phenotypes were known, I want to urge all physicians treating suspected autoimmune myositis to do what they can to identify the type of myositis as much as they can. A lot of time was necessarily wasted trialing treatments for "polymyositis" and "dermatomyositis", overgeneralizing results because it was not yet known that there are many types with many different disease processes. What fails for one common autoantibody's myopathy might work for another rare autoantibodies' myopathy, but that result could be buried due to statistically lumping all types of autoimmune myopathy together into just DM and PM. I think that even without clinical trials, clinical experience can be gained by coordinated record keeping of which treatments work and which fail on different autoantibodies on the cumulative patients of many specialists. In other words, I'm advocating gaining collective experience of which treatments work on which phenotype of autoimmune myopathy across the practices of many specialists in treating autoimmune myopathy. As well as accelerating the creation of clinical experience, this combined clinical experience could help guide which clinical trials are sponsored first. Please forgive me if you physicians are already doing this, I'm just a patient.
I'm retired and I don't mind revealing this part of my medical history, since it can benefit other patients. I'm a patient who has had what turned out to be anti-HMGCR necrotizing autoimmune myopathy for 16 years. On initial presentation, as well as the Bohan and Peter symptoms for PM and I had mild Gottron's papules and V-sign. Over the years I was treated with prednisone plus or or two at a time of the following prednisone sparing agents: methotrexate, azathioprine, mycophenolate, IVIG, cyclosporine C, cyclophosphamide, leflunomide, rituximab, and tacrolimus. Only the prednisone and IVIG ever caused much of an improvement, and that was just to bring my CKs from the 5,000 to 20,000 range down to between 300 and 1,000. About 12 months ago, after 15 months of this horrible disabling illness, my rheumatologist was able to get a supply of Xeljanz (tofacitinib) samples from Pfizer. For me, tofacitinib XR 11 mg/day was very successful for me in combination with IVIG Based on four months of this success, we were able to get provincial pharmacare coverage for the tofacitinib. I've been completely tapered off of prednisone for 3 months now, and we've slightly reduced my IVIG dose. My CKs are now below 230. I am feeling much better and more stable. I have had no adverse side effects at all, but it did also improve my rosacea. I want to urge the start of clinical trials of tofacitinib for treatment of anti-HMGCR myopathy.
38:40 Isn't the current thinking that each autoantibody creates its own type of autoimmune myopathy? That PM/Scl myopathy is not really an overlap autoimmune disease but rather is a separate illness that has PM and Scl as its symptoms? I'm just a patient, but I think that is what I've understood from past presentations by Dr Lisa Rider and Dr Andew Mammen. So, is it possible that what works on the disease caused by the PM/Scl autoantibody would fail a clinical trial for PM or Scl patients? That the best treatment would be missed due to overgeneralization of the disease in clinical trials, combining separate diseases into one group? Or am I confused?
“My patients are the guide for me,” you said!!! The way it should be! Thank you!
Thank you so much!
As a patient who went through the ringer trying this drug and that drug looking for an answer back in 2006, before the autoantibodies and phenotypes were known, I want to urge all physicians treating suspected autoimmune myositis to do what they can to identify the type of myositis as much as they can. A lot of time was necessarily wasted trialing treatments for "polymyositis" and "dermatomyositis", overgeneralizing results because it was not yet known that there are many types with many different disease processes.
What fails for one common autoantibody's myopathy might work for another rare autoantibodies' myopathy, but that result could be buried due to statistically lumping all types of autoimmune myopathy together into just DM and PM.
I think that even without clinical trials, clinical experience can be gained by coordinated record keeping of which treatments work and which fail on different autoantibodies on the cumulative patients of many specialists. In other words, I'm advocating gaining collective experience of which treatments work on which phenotype of autoimmune myopathy across the practices of many specialists in treating autoimmune myopathy.
As well as accelerating the creation of clinical experience, this combined clinical experience could help guide which clinical trials are sponsored first.
Please forgive me if you physicians are already doing this, I'm just a patient.
Dr Paik, on behalf of myositis patients everywhere, thank you for all that you, your team, and your colleagues do for us. We appreciate it hugely.
Thank you
How about jak inhibitor in refactory antesynthetase syndrom
I'm retired and I don't mind revealing this part of my medical history, since it can benefit other patients. I'm a patient who has had what turned out to be anti-HMGCR necrotizing autoimmune myopathy for 16 years. On initial presentation, as well as the Bohan and Peter symptoms for PM and I had mild Gottron's papules and V-sign. Over the years I was treated with prednisone plus or or two at a time of the following prednisone sparing agents: methotrexate, azathioprine, mycophenolate, IVIG, cyclosporine C, cyclophosphamide, leflunomide, rituximab, and tacrolimus. Only the prednisone and IVIG ever caused much of an improvement, and that was just to bring my CKs from the 5,000 to 20,000 range down to between 300 and 1,000.
About 12 months ago, after 15 months of this horrible disabling illness, my rheumatologist was able to get a supply of Xeljanz (tofacitinib) samples from Pfizer. For me, tofacitinib XR 11 mg/day was very successful for me in combination with IVIG
Based on four months of this success, we were able to get provincial pharmacare coverage for the tofacitinib.
I've been completely tapered off of prednisone for 3 months now, and we've slightly reduced my IVIG dose. My CKs are now below 230. I am feeling much better and more stable. I have had no adverse side effects at all, but it did also improve my rosacea.
I want to urge the start of clinical trials of tofacitinib for treatment of anti-HMGCR myopathy.
38:40 Isn't the current thinking that each autoantibody creates its own type of autoimmune myopathy? That PM/Scl myopathy is not really an overlap autoimmune disease but rather is a separate illness that has PM and Scl as its symptoms? I'm just a patient, but I think that is what I've understood from past presentations by Dr Lisa Rider and Dr Andew Mammen. So, is it possible that what works on the disease caused by the PM/Scl autoantibody would fail a clinical trial for PM or Scl patients? That the best treatment would be missed due to overgeneralization of the disease in clinical trials, combining separate diseases into one group?
Or am I confused?