HELP

Hi,
We need medical support.
A girl, 18 years old, with the diagnostic of systemic JRAm since June 2005 is under unstable medical conditions:
- all NSAIDs do not work (side effects)
- severe side effects to ciclosporine and metrotrexate
- severe side effects to prednisone (psihic concern, moonface and cushing syndrome are minore, should not count)

Currently she is on (for couple of months):
- kineret 100mk shots/daily
- Enbrel shots, twice per week
- 10mg prednisone daily

Nevertheless she is unstable: fever 102F and above, headaches, muscle pain, Reynolds syndrome, hands and feet numbing, coughing, cold/flu like symptoms. We, the parents, have the feeling we may lose her in a couple of weeks or months ...and this feeling is unchanged since June 2005. Life is very difficult without hope and guidance.

She is under medical care in the rheumatology clinic in Children Hospital Stanford. She has been seen by doctors in Seattle and Los Angeles Children Hospitals affiliated by the universities. Besides of having a given name for the syndrome, (systemic JRA) no one really understands what she really has
and are not able to keep her under a not life threatening medication or recover. She has no joints pain at all.

We need help to have the feeling she may recover sometimes .... we are horrified the disease may kill her anytime and the doctors look at her and DO NOT KNOW HOW TO STABILISE HER for not being constantly under life threatening conditions.

There is no need yet for a BMT transplant talk, because the doctors do not really guarantee anythink!  Whatever we ask the answer is almost the same: "what you (the parents) say it makes sense but we (the doctor) do not guarantee anythink!" We are concerned about her mind, lungs, kidney, heart, liver, and all the doctors say is: "your concern are justified, lets keep her watching with medication and blood tests". And this is keep going since 2005!  This is not a life and seems nobody really knows a thing! Meanwhile life is keep going down for all of us: except smart talk about immune system, virus and genetic conditions for immune disorders ...they don't know anything to really help! We need hope and action, not talk w/o results.

Please help us!
Thank you!

Systemic

[Kevin Pho, MD]

This certainly sounds like a difficult case, and you have been to one of the top treatment centers in the country.  It is unlikely that I can come up with anything different over the internet.

I can suggest some investigational therapies that can be discussed with your medical team.

This can include human recombinant anti-interleukin-6 monoclonal antibody which in small studies have been shown to help in refractory cases.

Stem-cell transplantation is another approach that you mentioned.  If medical therapy doesn't work, this can be considered.  This should be the last-resort approach due to the high risk of mortality.

Thalidomide is another approach - but needs to be used in caution in women due to the risks of birth defects.

These approaches obviously are for refractory cases.  The Enbrel and Kineret are appropriate and up-to-date treatments.   The investigational modalities mentioned above can also be considered.

Followup with your personal physician is essential.

This answer is not intended as and does not substitute for medical advice - the information presented is for patient education only. Please see your personal physician for further evaluation of your individual case.

Kevin, M.D.
kevinmd_