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
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,
It’s not just
A guide to childhood muscle, bone, and joint pain,
rheumatic diseases and the latest treatments
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 practice. If you want to know about the diseases, the tests, the medications, or how to be sure you are getting the best care– If you are the family member of a child with joint pains, this book will give you the answers. If you are a general physician, a pediatrician, or a nurse who cares for children with these diseases it will answer many of the questions families ask you, and you can recommend it to 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.