| Scleroderma
Fact Sheet The information on this fact sheet has mostly been found on the internet. This
means that there is a bias towards treatment initiatives in America, and little
information about whether treatments are available in the UK. It also means that
the information may not be comprehensive. Patients should talk to their consultants
about treatment options available in the UK.
New treatments for Scleroderma are extremely difficult to research well. Scleroderma
affects individuals in different ways, which makes it very difficult to form homogenous
groups on which to run trials, yet small samples have no statistical power, making
it very difficult to predict the course of the disease. Also, some of the worst
effects of the disease can be life threatening, making it unethical to have a
control group that receives no treatment. 'Double blind' studies (where neither
the patient, nor the researcher, know who is being treated) are therefore very
difficult, and sometimes impossible, to run. The Analysis of results not from
a double-blind study is then open to unconscious bias and their value can be limited.
What is Scleroderma? Scleroderma (hard skin) is a little-known disease of the connective tissue,
immune system and blood vessels, which primarily affects women in the childbearing
years, though it can affect anyone of any age. It results in taut, hard, discoloured
skin and may also affect the internal organs.
Scleroderma is divided into localised forms (morphoea, linear scleroderma) and
generalised scleroderma (systemic sclerosis, SSc). SSc has a severe and a mild
form. The severe form, diffuse cutaneous SSc, affects 40% of sufferers; the mild
form, limited cutaneous SSC, 60%. In the severe form, wide areas of the skin are
affected and internal organ involvement occurs early. Scleroderma can be fatal,
with lung disease as the chief cause of death. Survivors have a greatly reduced
quality of life with breathlessness, kidney disease, heart disease, digestive
problems and reduced function in joints, muscles and hands. In the mild form,
there is limited skin involvement with the later development of gut disease (and
thus difficulty in swallowing and eating) and lung problems. Additionally there
may be calcinosis (deposits of calcium which mass under the skin and protrude),
dry eyes and mouth, ulceration and a very poor circulation.
Juvenile scleroderma differs from the adult disease in that localised forms predominate.
The juvenile disease attacks particularly the skin, muscles, joints, tendons and
bones, with internal organ involvement a rarity. Childhood morphoea may last 3-4
years then resolve spontaneously, but the linear form may lead to growth defects.
Treatments Currently there is no treatment to cure scleroderma, although there are many treatments
to deal with the symptoms. There are also a number of initiatives, some of which
are detailed below (in alphabetic order).
Antibiotic treatment
There is increasing evidence that scleroderma and other rheumatic diseases are
caused by hypersensitivity (allergic-type) reaction due to infection of mycoplasmas
or bacterial L-form variants. If this is so then it is thought that antibiotics
would be effective in addressing the cause of the illness. Treatment is tailored
to individual patients and could be life long in some instances. Results are gradual
but the following symptoms are among those reported to be improved: pain, stiffness,
restricted range of motion, dry or cracked or tight skin, skin ulcers, swallowing
difficulties and heartburn.
Trails have been done on Minocyclin - a member of the tetracycline group of antibiotics
-, which is most commonly used to treat acne. David Trentham, MD (Harvard Medical
School, Beth Israel, Deaconess Medical Centre, Boston, Massachusetts), has carried
out a small open study on 11 patients with scleroderma, sponsored by The Road
Back Foundation and the NIH. Results have to be treated with caution as the sample
was very small and the mechanism by which mynocycline might work is not understood.
However four of the patients had complete remissions. The most frequent side effects
are vomiting and diarrhoea. Minocyclin also interferes with the balance mechanism
of the inner ear with resulting dizziness, nausea and unsteadiness. It is not
prescribed to people who have problems with their kidneys.
Brown seaweed or kelp - a wound dressing made from calcium alginates, derived
from kelp, has recently been found to be useful in the treatment of sclerodermal
ulcers.
Bone Marrow Transplant / stem cell transfusion.
Currently being studied in America, the procedure is for patients whose skin is
extensively affected and whose internal organs have been mild to moderately affected
before their disease has continued for three years. Blood cells and bone marrow
are removed from the patient who is then given immunotherapy with drugs and x-ray
radiation. The process of immunotherapy destroys blood cells and bone marrow,
so the stored cells are then put back into the patient afterwards allowing the
bone marrow to grow back. In some cases bone marrow from a matched relative (brother
or sister) can be used. The process has the potential to reverse the disease.
To be eligible for the process patients must be between 18 and 60 with a diagnosis
of diffuse systemic sclerosis.
For more information about clinical trials contact:
Arthritis Clinical Research Unit
Virginia Mason Research Center R1-RC
1000 Seneca Street
Seattle, WA 98101
http://www.vmresearch.org/ar_1999_clinical.htm
Corticosteroid drugs (steroids)
They are derived from, or are synthetic variants of, the natural corticosteroid
hormones formed in the outer part (cortex) of the adrenal glands situated on top
of each kidney. Their presence in large amounts in the body helps to reduce inflammation
and suppress allergic reactions and immune system activity. (Not to be confused
with anabolic steroids which build muscle.) They relieve symptoms and may also
temporarily halt the disease.
Side effects include increased susceptibility to infection, peptic ulcers, and
the risk of diabetes/worsening of pre-existing diabetes. In children they can
retard growth. Long-term use of high doses can lead to acne, mood changes, rounding
of the face (moon-face), increased blood pressure and fluid retention, muscle
wasting, easy bruising, a fat pad on the back and osteoporosis.
Dimethyl Sulfoxide (DMSO)
In America it has been in use since the 1940's as an industrial solvent and introduced
into therapeutic practice in the 1960's as a treatment for strains, sprains, bruises
and arthritis. It is currently being studied, but not approved, for use in a number
of skin, nerve and autoimmune diseases. There have been some studies which seem
to show better survival rates among mice who were given a 3% solution of DMSO
in their drinking water.
DMSO has been in use since 1974 by the Institute of Rheumatology AMS USSR for
systemic scleroderma. Affected areas are either immersed in a DMSO solution, or
it can be applied topically, or administered subcutaneously. Research seems to
show that patients with relatively mild disease are more likely to respond favourably
than those with severe to advanced disease. DMSO seems to work on collagen leading
to greater movement ability and skin pliability with dense skin swelling lessened.
D-penicillamine
An antirheumatic drug given to slow or even halt the progression of rheumatic
arthritis has been used in scleroderma with variable effects.
Interferon-g
Interferons are a group of substances produced in human and animal cells that
have been infected with viruses or stimulated by other substances. They are thought
to promote resistance to other types of viral infection. In an open, controlled
multicenter study from Germany 32 patients suffering from diffuse or limited forms
of systemic sclerosis were given 50mg interferon-g three times a week for 1 year.
After a year patients had stabilised where they would have been expected to worsen.
Side effects were minor flu-like symptoms.
Methotrexate
This drug is capable of blocking the metabolism of cells and is therefore used
in the treatment of certain diseases characterised by abnormally rapid cell growth.
It has been used in the treatment of rheumatoid arthritis and psoriasis, although
the reason why it is helpful is not known.
Nutritional supplements
Malabsorption can be a big problem for scleroderma sufferers due to the scarring
and bacterial overgrowth of their intestines. Antibiotics are often prescribed
which help with absorption and removing bad bugs, but often liquid nutritional
supplements or intravenous nutrition is also required. Some deficiencies common
amongst scleroderma sufferers include:
· Vitamin B12 deficiency, which can lead to fatigue, memory loss and abnormal
gait.
· Vitamin D deficiency, leading to softened bones.
· Vitamin K deficiency leading to haemorrhaging.
· Vitamin E deficiency can lead to sclerodermal bowel disease.
Photopheresis
A procedure similar to dialysis in that a large-caliber IV tube is inserted into
one arm, blood is removed, treated and returned to the patient. The blood is passed
through a centrifuge, which separates white and red blood cells. The white blood
cells - the main cells responsible for autoimmune disease - are collected in a
bag, which is injected with a medicine, then exposed to ultraviolet light, then
transfused back into the patient. In Seattle a study was carried out in 1991 but
was deemed inconclusive by the FDA. Some patients in the test, however, showed
dramatic improvement with few side effects. It is not known why it should work.
For further information on photopheresis contact:
Arthritis Clinical Research Unit
Virginia Mason Research Center, R1-RC
1000 Seneca Street
Seattle WA 98101
http://www.vmresearch.org/ar_1999_clinical.htm
Psoralen-UV-A therapy (PUVA)
Psoralen is one of a class of compounds originally used in the treatment of cutaneous
diseases, initially for psoriasis, in the early 1970's by TB Fitzpatrick and JA
Parrish at Harvard. When it was activated with UVA (320-400 nm radiation) it showed
good results in controlling psoriasis. Research has been carried out on patients
with systemic sclerosis showing some improvement of symptoms and indicated that
it may be an effective treatment for patients who are undergoing skin changes.
For more information contact on this research contact:
Angelike Hofer, MD
Department of Dermatology University of Graz
Universitaetplatz3
A-8010 Graz
Austria
E-Mail: angelika.hofer@uni-graz.at
For more information on PUVA contact:
Francis P Gasparro PhD, Director
Jefferson University Photobiology Lab
Department of Dermatology and Cutaneous Biology
233 South 10th Street (Room 428)
Philadelphia
PA 19107-5541
Relaxin (brand name ConXnR)
In development, Relaxin (recombinant human relaxin H2) is a natural protein and
has been shown to inhibit excessive connective tissue build-up by decreasing collagen
production and enhancing collagen breakdown. This has led to improvements in,
among other things, the function of the lungs, the ability to extend the hands
and increased eating, gripping and reaching ability.
Superoxide Dismutase (SOD) and Liposomal Superoxide Dismutase (LIPSOD)
Superoxide dismutase is an enzyme that in its different forms is associated with
copper, zinc and manganese. Its function in the body is to keep the membranes
fluid and the tissues and muscles supple. Without SOD the body would harden up
and wither.
SOD is available in health stores, however, unfortunately it seems that this product
has no effect, as taken orally, it is destroyed in the gut and so is unable to
affect the tissues it is supposed to benefit. In order to do some good SOD must
be injected or taken in liposomal forms (encapsulated in a liposome 'delivery
vehicle'). It is thought to be beneficial for 'hardening' diseases and disorders.
Much of the pioneering work on the use of SOD and LIPSOD has been done by A. M
Michelson of the Institut de Biologie Physico-Chimique in Paris, and research
is being carried out in Japan and America. In one woman with severe scleroderma
who received two injections of LIPSOD weekly, a significant regression of the
disease was observed.
Thalidomide
Thalidomide has been used to clear severe skin reaction associated with leprosy
treatment, and more recently in chronic inflammatory skin and mucous membrane
disorders. Theoretically it has the potential to have a positive effect on scleroderma.
A study is planned to use thalidomide in treating scleroderma by The Rockefeller
University Hospital, New York City.
UVA - 1 therapy
UVA-1 radiation penetrates the skin and has an effect on epidermal structures
and midermal and deep dermal components, especially blood vessels. It is being
investigated as a safer alternative to PUVA for treating long-term conditions.
Much of the early work in this realm has been done in Europe where efficient sources
of UVA-1 radiation are more widely available. Initial results are promising but
more detailed tests need to be carried out. Pilot studies have shown that it can
soften and reduce the diameter of sclerotic legions and in some instances clear
plaques completely.
For further information contact:
Jefferson University Photobiology Lab
Department of Dermatology and Cutaneous Biology
233 South 10th St (Room 428)
Philadelphia
PA 19107-5541
Tel: 215 503 3327
E-mail: forondoc@AOL.com
Other
Muscle aches and pains can be helped by therapies such as physiotherapy, chiropractic,
acupuncture, massage, hydrotherapy and relaxation. Heartburn can be treated with
antacids. Other medications used to alleviate symptoms of the disease are aspirin,
nonsteroidal anti-inflammatory drugs, and blood pressure medication.
References
- The CaF Directory (Contact a Family) of Specific Conditions and Rare Syndromes
in Children with their Family Support Networks.
- The Efficacy of Long-Term Application of Dimethyl Sulfoxide in a complex therapy
of Patients with Systemic Scleroderma, U.V. Murav'ev, A.P. Aliab'eva, I.A Sigidin,
and N.G. Guseva.
- Hunzelmann N, Anders S, Fierlbeck G et al. Systemic Scleroderma. Multicenter
trial of 1 year of treatment with recombinant interferon gamma. Arch Dermatol
1997; 133:609-613
Resources Organisations
The Scleroderma Society
3 Caple Rd
London
NW10 8AB
Tel: 020 8961 4912
http://www.sclerodermasociety.co.uk
The Raynaud's & Scleroderma Association
112 Crewe Road
Alsager
Stoke-on-Trent
ST7 2JA
Tel: 01270 872776
http://www.raynauds.demon.co.uk/
Offers help to families of newly diagnosed children and adults, support to families
of sufferers, information about the condition and names of appropriate medical
consultants, a quarterly newsletter and information pack.
UK Scleroderma Group Central Register of UK Systemic Sclerosis Patients
Centre for Rheumatology
Royal Free and University College Medical School
Rowland Hill Street
London NW3 2PF
Tel: 0171 830 2360
This register aims to increase the numbers of UK patients available for research.
The Scleroderma Foundation
12 Kent Way Suite
101 Byfield,
MA 01922
Tel: (001) 978 463 5843
http://www.scleroderma.org/
Websites
- http://www.worldchat.com/public/scleroderma/greatlinks.htm
- from this site you can access other relevant websites.
Further Reading
These are some of the references that have been passed to us; the list is not
exhaustive. We have not necessarily read the books, and cannot say how easy it
will be to get them.
- Scammell Henry The New Arthritis Breakthrough, ISBN: 0871318431
- Scammell Henry Scleroderma: The Proven Therapy That Can Save Your Life. ISBN:
0871318423
- Mayes Maureen Scleroderma Book: A Guide for Patients and Families. ISBN: 0195115074
-
Melvin Jeanne Scleroderma: Caring for Your Hands & Face. ISBN: 1569000069
- Clements Philip Systemic Sclerosis. ISBN: 0683017403
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