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 New York City.Click here for more information about Dr. Lehman or the Hospital for Special Surgery.

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 isnít 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 isnít pretty, but it isnít 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 itís not too technical.There is so much to say about scleroderma and each childís 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 Raynaudís 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 Iíve 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, Kawasaki disease and related conditions.He practices in New York City.

Click here for more information about Dr. Lehman or the Hospital for Special Surgery.

 

My book Ėclick 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."óKathy Gaither, Juvenile Scleroderma Network, Inc.

††††† ďNavigating 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

 

††† ďDr. Tom Lehmanís experience and compassion are evident on every page of this book, and they help guide the readeróchild, parent, and healthcare professional alike Ė 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.Ē -- Jack Klippel, M.D. President and CEO of the Arthritis Foundation

 

†††† ďDr. 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!Ē-- Charles Spencer, M.D., Professor of Clinical Pediatrics, University of Chicago, La Rabida

 

 

 

Itís 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 canít 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 careĖ 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,
New York, NY 10021
212-606-1151, fax 212-606-1938, e-mail goldscout@aol.com

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