Scleroderma and related conditions
This page is provided by Thomas J. A. Lehman MD
Delivering the best care - with great care
Dr. Lehman is the
author of many textbook chapters and articles on the care of children and young
adults with scleroderma. He practices in
This information is provided as a public service. No
warranty is expressed or implied.
Being told that your child has scleroderma is a very scary
experience. The first thing you
need to know is what type of scleroderma.
Sometimes doctors use the word scleroderma when they are referring to
localized lesions of morphea (see below).
This isn’t pretty to look at, but it is rarely serious. Other times a child has linear scleroderma
with an area of tight skin on an arm or leg.
Again, this isn’t pretty, but it isn’t life threatening. Other children have progressive systemic
sclerosis or CREST syndrome. These are
the more severe forms of scleroderma that may be life-threatening. Before you do
anything else make sure you know which form of scleroderma the doctor is
talking about. It makes a big difference
in what problems you are likely to have, what medicines you should take, and in
what the outcome is likely to be. There are rare cases in which one form of scleroderma
changes to another. However, this is so
exceptionally rare that once you have been told which form of scleroderma your
child has by an experienced physician you should not worry about the others.
In
the paragraphs that follow I have given a brief summary of the different types
of scleroderma and their treatment. This
was originally written for physicians, but it’s not too technical. There is so much to say about scleroderma and
each child’s case is so different that I held back from trying to write a
long-winded discussion. Only a small
part of such a discussion would be appropriate for each viewer. Helpful organizations such as the Scleroderma
Foundation and the Juvenile Scleroderma network have links near the bottom of
the page. If you still have a lot of
questions, you can get the best answers that are relevant to your child by
talking to your physician.
Scleroderma in childhood may occur as
localized morphea, linear scleroderma, progressive systemic sclerosis or the
CREST syndrome. Linear scleroderma and morphea
are the most common of these entities. They often begin as areas of either
increased or decreased pigmentation which are reddish at first and then become progressively
hardened and 'shrunken.' Since there is no medical treatment, which will
improve the hardening without 'risk of side effects,' no intervention is
warranted unless the area of involvement overlies a joint. Treatment with
emollients is often felt to be beneficial by patients and their families.
However, if the area of linear scleroderma overlies a joint, progressive
tightening of the skin and underlying tissues may produce flexion contractures
(i.e. the inability to fully straighten the arm or leg). Physical and
occupational therapy may help to prevent this. In severe cases it can also be
treated with methotrexate, but this should only be done by a physician who is
familiar with this drug since it can have significant side effects. Surgical
intervention is generally not helpful.
Sclerodermatomyositis is an infrequent, but well-described entity. Children with sclerodermatomyositis have
characteristic changes of linear scleroderma accompanied by a
dermatomyositis-like rash, weakness, and muscle inflammation. The
dermatomyositis like component of this illness responds well to corticosteroid
therapy, but appears to be without effect on the linear scleroderma. Some
children with illness that looks like a combination of scleroderma and
dermatomyositis have mixed connective tissue disease. However, these children
do not have linear scleroderma like lesions and sclerodermatomyositis
is probably a true independent diagnosis.
Often children are not recognized to have
scleroderma until they develop Raynaud’s
phenomenon. Progressive systemic
sclerosis and CREST syndrome are potentially severe, life threatening illnesses
in children. These forms of scleroderma
can involve the heart, lungs, kidneys, and even gastrointestinal tract as well
as the skin. Because the internal organs
may be involved by the disease there can be a wide variety of symptoms. Serious damage to the internal organs can be
life threatening. No uniformly effective
therapy exists. D penicillamine is beneficial for
some patients, but requires close monitoring in view of its potential side
effects including rash, renal toxicity, leukopenia, and autoimmune phenomenon. Recent publications however have cast doubt
on its overall effectiveness and many centers no longer use this drug. In many
centers children with PSS are treated with cyclosporine and methotrexate with
beneficial effects. Newer biologic agents
are being used in children with severe disease at the Hospital for Special Surgery
and the early results are very promising.
All of these regimens have significant possible side effects and none is
uniformly effective. Children with severe scleroderma should be under the care
of an experienced specialist and may require all of these drugs over time or even
a combination of agents. Nifedipine is another drug,
which has been effective in providing at least transient relief from the
Raynaud's phenomenon and may have a beneficial effect on pulmonary involvement
in children with scleroderma.
Catastrophic kidney involvement and high blood
pressure is infrequent in childhood scleroderma, but should be treated with captopril or a related angiotensin
converting enzyme inhibitor if it occurs. Unfortunately, despite the fact that
renal involvement is infrequent, if significant heart and or lung involvement
is present the long-term (ten to 15 year) prognosis for children with
progressive systemic sclerosis remains poor. Over time many of these children
develop increasing difficulty with oxygen exchange in the lungs due to
pulmonary fibrosis. This places increasing stress on the heart and results in
an increasing risk of pneumonia or right-sided heart failure. Although
digitalization may seem warranted, it has been associated with a significant
frequency of arrhythmias and should be avoided if possible. The best hope for
these children is to be aggressively treated once their scleroderma has been
recognized in the hope of preventing or slowing the progress of internal organ
involvement. Children with progressive systemic sclerosis or CREST syndrome
should be carefully followed so that any evidence of increasing internal organ
involvement can be dealt with in the best possible fashion.
We don't have all the answers for children with
scleroderma. The most important thing is
that your child is cared for by a physician with experience who will pursue and
beneficial outcome. You can get much more information
than I’ve been able to put here from my book (see below). Research to find the
best treatment continues. In the interim families should make sure their
children receive care from experienced physicians who understand scleroderma
and can give them the best possible care.
If
you want a more detailed discussion of scleroderma in childhood click here for
information regarding a book I have written.
Further help can be
obtained from the Juvenile Scleroderma Network
and the Scleroderma Foundation
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,
Click
here for more information about Dr. Lehman or the
Hospital for Special Surgery.
My book
–click here to order at a discount from Amazon.com!!
at
Amazon.com
"A must have for any family with a child who has a rheumatic
disease! I personally found this book to very informative. Dr. Lehman, as
always, gives families a clear understanding of their choice of treatments and
what they can expect from their child's rheumatic disease."—Kathy
Gaither, Juvenile Scleroderma Network, Inc.
“Navigating
the health care system as an adult can be daunting, but doing so with a child
who has a rheumatic disease can be a particularly anxiety-provoking experience
for the entire family. Education is the key to understanding and to reducing
the stress and fear that goes along with caring for an ill child. Dr. Lehman's
book provides a thorough explanation of disease process, therapeutics,
diagnostics and coping strategies all in a very friendly easy to read manner.
The Scleroderma Foundation is excited about this guide and believes that it
would be a great addition to the family library and to providing the insight
necessary to becoming an informed, effective partner in their child's
care."--Donna Kohli,
President & CEO, Scleroderma Foundation
“Dr.
Tom Lehman’s
experience and compassion are evident on every page of this book, and they help
guide the reader—child,
parent, and healthcare professional alike – through the world of childhood arthritis. This book is an absolute gem written with a
single goal in mind: improve the lives
of kids with arthritis.” -- Jack Klippel, M.D. President and
CEO of the Arthritis Foundation
“Dr. Lehman has given
parents and families of children with arthritis the first book that speaks to
the parent and child as equals. His book
explains the illnesses, the medications, the lab tests, and the disease course
in simple, understandable lay language and givens them valuable insight into
how a pediatric rheumatologist thinks.
Bravo!”-- Charles Spencer, M.D., Professor of
Clinical Pediatrics, University of Chicago, La Rabida
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
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.
Click here if you are
interested in making an appointment to see Dr. Lehman
For books about living with childhood rheumatic disease click here.
I HOPE THIS
INFORMATION IS USEFUL TO YOU.
This site provided by Thomas J. A. Lehman MD
Chief, Division of Pediatric
Rheumatology
The Hospital for Special Surgery
535 E 70 St,
212-606-1151, fax 212-606-1938,
e-mail goldscout@aol.com
