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$Unique_ID{BRK04158}
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$Title{Purpura, Thrombotic Thrombocytopenic}
$Subject{Purpura, Thrombotic Thrombocytopenic TTP Moschowitz Disease
Hemolytic-Uremic Syndrome (HUS) Thrombotic Microangiopathy ('TTP-HUS' Complex)
Idiopathic Thrombocytopenic Purpura (ITP) Schoenlein-Henoch Purpura
Thrombocytopenia}
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Copyright (C) 1989, 1991 National Organization for Rare Disorders, Inc.

653:
Purpura, Thrombotic Thrombocytopenic

** IMPORTANT **
It is possible that the main title of the article (Thrombotic
Thrombocytopenic Purpura) is not the name you expected.  Please check the
SYNONYM listing to find the alternate names and disorder subdivisions covered
by this article.

Synonyms

     TTP
     Moschowitz Disease

Information on the following diseases can be found in the Related
Disorders section of this report:

     Hemolytic-Uremic Syndrome (HUS)
     Thrombotic Microangiopathy ('TTP-HUS' Complex)
     Idiopathic Thrombocytopenic Purpura (ITP)
     Schoenlein-Henoch Purpura
     Thrombocytopenia

General Discussion

** REMINDER **
The information contained in the Rare Disease Database is provided for
educational purposes only.  It should not be used for diagnostic or treatment
purposes.  If you wish to obtain more information about this disorder, please
contact your personal physician and/or the agencies listed in the "Resources"
section of this report.

Thrombotic Thrombocytopenia Purpura (TTP) is a rare, serious blood
disease.  Major symptoms may include a severe decrease in the number of blood
platelets (thrombocytopenia), abnormal destruction of red blood cells
(hemolytic anemia), and disturbances in the nervous system.  Kidney
dysfunction and fever are also common.

Symptoms

In addition to thrombocytopenia and hemolytic anemia, blood platelets may
clot in the blood vessels of many organs which may block the normal flow of
blood.  Disturbances in the nervous system may include headaches, mental
changes, slight or partial paralysis (paresis), seizures, or coma.

Fever, blood plasma proteins in the urine (proteinuria), and a very small
number of red blood cells in the urine (hematuria) may also occur.  Patches
of purplish discoloration (purpura) resulting from abnormal bleeding into the
mucous membranes (the thin, moist layer lining the body's cavities) and into
the skin is another feature of TTP.  Abnormally heavy bleeding (hemorrhage),
weakness, fatigue, lack of color (pallor), and abdominal pain with nausea and
vomiting may also be present.  In half of TTP patients, an increased level of
creatinine is found in their blood serum.

Acute renal failure occurs in only about 10% of TTP patients.  Urine flow
is very low.  Within days, swelling of the feet, shortness of breath,
headache, and fever may occur.  Retention of water and salt in the blood may
lead to high blood pressure, changes in brain metabolism, and congestion in
the heart and lungs.  Acute renal failure may lead to a buildup of potassium
in the blood (hyperkalemia) which may cause irregular heartbeat.

Abnormalities in the retina (the light-sensitive layer of the eye) have
been found in female TTP patients after taking oral contraceptives.
Clearness of vision is usually not affected.

There may be possible serious complications during pregnancy in TTP
patients.  In general, TTP often occurs suddenly with great severity and may
recur or persist.

Causes

The exact cause of TTP is not known.  It may be due to an infectious agent or
to an autoimmune reaction.  Autoimmune disorders are caused when the body's
natural defenses (antibodies) against invading organisms suddenly begin to
attack healthy tissue.

Some people may have a hereditary predisposition to TTP since family
members have become affected years apart.

TTP may be influenced by hormones.  In some cases of TTP, relapses
coincide with the use of oral contraceptives and with menstrual cycles
(cyclic TTP).

TTP can occur as a consequence of AIDS, the AIDS-related complex, or the
human immunodeficiency virus (HIV) infection.

Affected Population

One in a million people are affected with TTP each year.  Two-thirds of TTP
patients are women.  It usually affects people between 20 to 50 years old.

TTP is occasionally associated with pregnancy and collagen-vascular
diseases (a group of diseases affecting connective tissue).

TTP appears to occur more frequently than usual in intravenous drug
addicts and homosexual men who have human immunodeficiency virus (HIV)
infection.

Related Disorders

Symptoms of the following disorders can be similar to those of Thrombotic
Thrombocytopenia Purpura (TTP).  Comparisons may be useful for a differential
diagnosis:

Hemolytic-Uremic Syndrome (HUS) is characterized by acute renal failure,
a severe decrease in the number of blood platelets (thrombocytopenia), and
abnormal destruction of red blood cells (hemolytic anemia).  HUS tends to
occur in children less than 4 years old and usually after an infectious
disease.  In adults, it may affect young women as a complication of pregnancy
or in the postpartum period.  HUS may have a common origin and development
as TTP and treatment is similar.  (For more information on this disorder,
choose "HUS" as your search term in the Rare Disease Database.

Thrombotic Microangiopathy is the occurrence of TTP and HUS together.  It
is also called the 'TTP-HUS' complex.  It has been suggested that TTP and HUS
are actually variants of the same disease.

Idiopathic Thrombocytopenic Purpura (ITP) is a blood disease with no
specific known cause.  It is characterized by thrombocytopenia, abnormal
bleeding into the skin and mucous membranes, and anemia.  ITP occurs most
frequently in children and young adults, and more frequently in females than
males.  A viral infection may precede ITP.  (For more information on this
disorder, choose "ITP" as your search term in the Rare Disease Database).

Schoenlein-Henoch Purpura is one of a group of disorders characterized by
purplish or brownish-red discolorations of the skin caused by abnormal
bleeding into the skin and mucous membranes.  This blood disorder may affect
the skin, joints, gastrointestinal system, kidneys, and in a few cases the
central nervous system.  Major symptoms may include a rash, fever, weakness,
pain in the joints or abdomen, vomiting, blood in the stool, and anemia.

Central nervous system symptoms may include headaches, perceptual
changes, and seizures.  The exact cause of Schoenlein-Henoch Purpura is
unknown, but it may be related to an extreme allergic response to foods,
drugs, or insect bites.  (For more information on this disorder, choose
Schoenlein-Henoch" as your search term in the Rare Disease Database.)

Thrombocytopenia is a general term referring to a group of blood
disorders including TTP, HUS, and ITP.  It is characterized by a severe
decrease in the number of blood platelets and excessive bleeding into the
skin or mucous membranes.  Anemia may occur producing weakness, fatigue, and
signs of congestive heart failure.  (For more information on this disorder,
choose "Thrombocytopenia" as your search term in the Rare Disease Database).

Therapies:  Standard

Rapid diagnosis and immediate treatment is very important in TTP.  The
current drug treatment of TTP is prednisone which may relieve inflammation
and suppress the immune system.  Additionally, fresh frozen plasma (the fluid
part of the blood) is infused into the patient's vein.  Plasma infusion
should be used with extreme caution.

Genetic counseling may be of benefit for patients and their families when
TTP has affected other family members.  Other treatment is symptomatic and
supportive.

Therapies:  Investigational

Plasmapheresis and plasma exchange may be of benefit in some cases of TTP.
This procedure is a method for removing unwanted substances (toxins,
metabolic substances and plasma parts) from the blood.  Blood is removed from
the patient and blood cells are separated from plasma.  The patient's plasma
is then replaced with other human plasma and the blood is retransfused into
the patient.  Plasma infusion is also being studied as a treatment for TPP
patients.  The infusion process uses substances missing from the TPP
patient's blood and replaces them through infusion of new plasma into the
patient.  No blood is removed from the patient during this process.  These
therapies are still under investigation to analyze side effects and
effectiveness.  More research is needed before plasmapheresis and plasma
infusion can be recommended for use in all but the most severe cases of TTP.

Whole blood exchange may also be of benefit in some cases of TTP and is
still under investigation.  It involves the simultaneous withdrawal of the
patient's blood and the replacement of a donor's blood.

Other drugs such as vincristine (an immunosuppressant), dipyridamole, and
aspirin may be considered when TTP is not responsive to other treatment.
Also, drugs that destroy blood platelet clots (antiplatelet) may be of
benefit to TTP patients.  However, the side effects and the effectiveness of
these drugs are uncertain.

The orphan drug, Defibrotide, is being investigated for the treatment of
TTP.  Interested doctors may contact:

     Crinos International
     Via Belvedere 1
     22079 Villa Guardia (Como)
     Italy

The effectiveness of removing the spleen (splenectomy) of TTP patients is
also being investigated.

This disease entry is based upon medical information available through
September 1991.  Since NORD's resources are limited, it is not possible to
keep every entry in the Rare Disease Database completely current and
accurate.  Please check with the agencies listed in the Resources section for
the most current information about this disorder.

Resources

For more information on Thrombotic Thrombocytopenic Purpura, please contact:

     National Organization for Rare Disorders (NORD)
     P.O. Box 8923
     New Fairfield, CT  06812-1783
     [phone]

     NIH/National Heart, Lung and Blood Institute (NHBLI)
     9000 Rockville Pike
     Bethesda, MD  20892
     [phone]

For Genetic Information and genetic counseling referrals:

     March of Dimes Birth Defects Foundation
     1275 Mamaroneck Avenue
     White Plains, NY  10605
     [phone]

     Alliance of Genetic Support Groups
     35 Wisconsin Circle, Suite 440
     Chevy Chase, MD  20815
     (800) 336-GENE
     [phone]

References

INTERNAL MEDICINE, 2nd Ed.:  Jay H. Stein, ed.-in-chief; Little, Brown and
Co., 1987.  Pp. 860, 1006-1007.

THROMBOTIC THROMBOCYTOPENIC PURPURA.  A REVIEW:  S.J. Sierakow and E.J.
Kucharz; Cor Vasa (1988:  issue 30(1)).  Pp. 60-72.

HORMONAL DEPENDENT THROMBOTIC THROBOCYTOPENIC PURPURA (TTP).  R.S.
Holdrinet, et al.;  Scand J Haematol (March, 1983:  issue 30(3)).  Pp. 250-
256.

THROMBOTIC THROMBOCYTOPENIC PURPURA IN PATIENTS WITH THE ACQUIRED
IMMUNODEFICIENCY SYNDROME (AIDS)-RELATED COMPLEX.  A REPORT OF TWO CASES:
J.M. Nair, et al.; Ann Intern Med (August 1, 1988:  issue 109(3)).  Pp. 209-
212.