Juvenile Scleroderma Update
Written by Dr. Thomas J.A. Lehman
Hospital for Special Surgery, New York, NY
For the first time the 2004 annual meeting of
the American College of Rheumatology featured a full 90 minute
clinical symposium on childhood scleroderma. Organized by the
members of the Juvenile Scleroderma Working group this symposium
features sessions on progressive systemic sclerosis, localized
forms of scleroderma and new advances in describing the disease.
One of the key obstacles to improving the
care of children with scleroderma has been a failure by doctors
to clearly define which group of patients they are talking
about. We all know every patient is different from other
patients, but it’s also true that ‘not all scleroderma is the
same.’ Children with localized scleroderma may have strikingly
different degrees of involvement. Although it’s less obvious,
children with PSS can have strikingly different involvement as
well. When discussing problems or therapies doctors need to be
sure they are talking about the same groups of children.
(Imagine cooking pies with round green fruit with a firm white
flesh, stems, and a seeded core. If we all had apples there
would be no problem. But what is someone had pears? They fit the
description, but the results don’t make sense. We need to make
sure our descriptions are specific enough that we don’t make
The 90 minute session at the ACR meeting
concentrated on three areas. Thomas J. A. Lehman MD from New
York gave a 30 minute presentation on the early recognition of
children with PSS and the importance of promptly initiating
therapy to prevent long term damage. There still isn’t complete
agreement on the best therapy for these children. Methotrexate,
cyclosporine, cyclophosphamide and newer agents which block
endothelial cell signaling were all discussed.
Dr. Francesco Zulian from the University of
Padua in Italy followed with an excellent presentation on the
varied forms of localized scleroderma in childhood. This is a
very confusing area. At the summer 2004 meeting in Padua 20
experts from around the world met to discuss proper
characterization of these children so we could be sure we were
all talking about the same thing. By the end of the first
afternoon it was clear we were going to have a difficult time.
Everyone recognized the typical forms of focal scleroderma (a
tight band on the leg or arm, hard round spots on the body, or
tight hard skin on the scalp), but everyone had examples of
children who clearly had more than one type of involvement. Does
that mean it’s all the same thing? We don’t know yet. Much more
needs to be done.
The final half hour presentation was made by
Dr. Nicolo Ruperto from the Gaslini hospital in Genoa Italy. Dr.
Ruperto (in abstentia since his wife had just delivered a baby –
he sent a dvd) reported on the discussions of criteria for the
diagnosis of PSS which were held in Padua. Working with the
group of 20 experts and 150 case histories of children with
scleroderma and similar diseases Dr. Ruperto was able to define
a set of criteria which could be used to define children with
PSS with increased certainty. These criteria will be very
important in allowing us to make sure everyone in a study has
the same disease. However, they aren’t writing in stone. There
were several children where even the 20 world experts couldn’t
quite agree on the proper diagnosis.
What do these things mean to children with
scleroderma and their families? They mean that a significant
group of pediatric investigators has now been organized to bring
rapid progress to the treatment and care of children with
scleroderma. So far the Juvenile Scleroderma working group has
only had two meetings, but we look forward to moving forward as
quickly as possible to find better drugs and an improved outcome
for children with scleroderma. Want to help? It all costs money.
If you have ideas for helping to support the next big meeting
let us know. So far the meetings have been in Italy because the
Italian parents of children with scleroderma were willing to do
the hard work. They’ve done two. Hopefully we can find the
resources in the US to support the next one.
keep in mind, this webpage is for your information only.
Please check with your child's physician for any treatments.
For more information on Juvenile Scleroderma, contact:
Juvenile Scleroderma Network, Inc.
1204 W. 13th Street, San Pedro, CA 90731
Tel: (310)519-9511 (Pacific Time)
24 Hour Support Line: 1-866-338-5892 (toll-free)
Speak to another JSD parent for emotional and logistical support
provided by home-based JSD volunteers. For medical advice, please
contact your child's physician.
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