Bone Marrow Transplant (BMT)
For Childhood Autoimmune Diseases
JRA, Scleroderma, Lupus
Thomas J. A. Lehman MD
Chief, Division of Pediatric Rheumatology
Hospital for Special Surgery
535 E. 70 St
212-606-1151
Delivering the best
care - with great care
BMT for arthritis sounds awfully extreme, but it is
one of the new alternatives being actively considered for severe cases which
persist despite intensive and aggressive therapy. The idea is not to transplant children who
are wheel chair bound with multiple destroyed joints, but to prevent children
from ever getting that bad. Does it
make sense?? Here’s what we know. About ten percent of children with severe
systemic onset JRA (this group is only 20% of JRA cases – so now we’re talking
2% of patients) have severe disease despite NSAIDs,
high doses of steroids, methotrexate, and azathioprine or other
immunosuppressives. Many develop major
steroid induced complications with short stature, avascular necrosis of bone,
and recurrent infections. These children
suffer from severe medical complications and severe psychological
complications. In addition the
immunosuppressive drugs add to the risk of infection, and of cancer or
infertility. Suppose there was
something you could do to make it all go away?
A Dutch group transplanted one of their
severe patients when everything else had failed. That patient got dramatically better. They then transplanted three more. I attended a small meeting in
There are two types of BMT. Autologous transplant involves
harvesting the patient’s peripheral blood stem cells or bone marrow, treating
it, and then giving it back after they are treated with intensive
chemotherapy. There’s ‘no’ risk of graft
versus host disease, but the disease might come back. Allogeneic
transplants involve intensive chemotherapy and then giving the patient stem
cells from an HLA matched sibling. You
run the risk of graft versus host disease, but it is less likely the disease
will come back. :The
Dutch group did only autologous because they are ‘safer.’
What’s happened so far? Eight of 24 are much better and off steroids
after 6 – 24 months, but 12 are only somewhat better – but they’re off
steroids. However 4 children have died
of complications. Not the miracle
everyone had hoped, but these are our ‘hopeless’ cases.
At HSS we’re ‘down the block’ from Memorial
Sloan Kettering; a premier institution with extensive experience in BMT for
children. We believe we can get better
results with allogeneic transplantation and, with
Memorial’s extensive experience, we hope to have a better safety record. We have set up a protocol to provide BMT for
children with severe rheumatic diseases.
We will provide allogeneic transplants for
children who have HLA matched siblings and autologous transplants for children
who do not have a suitable donor.
This is only for those children we can’t control with conventional
therapy and there are rigorous entry criteria. We are presently screening children with
juvenile rheumatoid arthritis and scleroderma for consideration. Severe systemic lupus erythematosus and
severe dermatomyositis may also benefit from transplantation. If you have children you think might be
candidates for this therapy let us know.
Only a limited number of transplants will be done until we are sure this
is as safe as possible and effective. Each
child will be rigorously screened and we may recommend other things to try
before transplantation, or feel that there is too much damage for the risk to
be worthwhile. We are also dependent
on insurance company authorization, but we’ve been successful so far.
No one
knows whether this will prove to be the best therapy over the long term. What we know is that the current therapy
fails to be adequate for some children.
The Division of Pediatric Rheumatology at the Hospital for Special
Surgery is at the forefront of providing the best possible therapy for children
with rheumatic diseases.
Dr. Lehman is the author of many
textbook chapters and articles on the care of children and young adults with
rheumatic diseases including SLE, JRA, dermatomyositis, scleroderma,
My
book –click here to order at a discount from Amazon.com
at
Amazon.com
It’s not just
growing pains.
A guide to childhood muscle, bone, and joint pain,
rheumatic diseases and the latest treatments
Click here to see the table of
contents
It
has always been a frustration trying to answer the many questions I have
received from people over the web. I can’t
take the time and give them the detail I would like to. I have to take care of my patients. This book is a distillation of my experience
answering questions for parents and health professionals over 25 years of
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diseases, the tests, the medications, or how to be sure you are getting the
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give you the answers. If you are a general physician, a
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them. It will also answer many of your
questions about what shots to give, what precautions to take, and the other
questions families, pediatricians, and other health care providers have asked
me over the years.
Systemic
lupus erythematosus, dermatomyositis, Scleroderma, progressive systemic
sclerosis, pss, jra,
juvenile rheumatoid arthritis, childhood arthritis, growing pains, rheumatism,
children with pain, bone pain, pediatric specialists, my child hurts, chronic
disease, chronic childhood illness, the best care, Kawasaki disease, mixed
connective tissue disease, SLE, JCA, JIA, juvenile chronic arthritis, sports injuries, frequent sports injuries,
cyclophosphamide, Methotrexate, diclofenac, voltaren, Relafen, children’s health care, educational
materials, pediatric resources, public health education, health education,
school nurse materials, help for school nurses.