Thrombocytopenia and Other Platelet Disorders
Improving Platelet Function in Children
Platelets are blood cells that stop bleeding by clumping together and plugging up injuries to blood vessels, such as from a cut. If a child experiences excessive bleeding with no known cause and which continues longer than usual, something may be wrong with the body's platelets. There can be too few platelets (thrombocytopenia) or too many platelets (thrombocytosis), or the platelets may not be working well (thrombocytopathy).
NewYork-Presbyterian Komansky Children's Hospital and NewYork-Presbyterian Morgan Stanley Children's Hospital have established pediatric platelet disorders programs that offer onsite screening, diagnostic and genetic testing, treatment, counseling, education, and support for children with platelet disorders and their families. Our goal is to provide care that enables your child to live a healthy, active, and safe life.
Types of platelet disorders
Thrombocytopenia (low platelet count) develops when the body does not make enough platelets or when there is a disorder that causes platelets to be destroyed by the body's immune system. There are many causes of low platelet counts. The most common platelet disorder in children and adolescents is immune thrombocytopenia (ITP, also called idiopathic thrombocytopenia), a condition in which the immune system attacks the platelets and platelet-producing cells ("megakaryocytes"). Thrombocytopenia can also be inherited, caused by infections/drugs, or result from antibodies being passed from a mother to her newborn.
Thrombocytosis (high platelet count). A high platelet count in children is typically a reaction to an underlying infection, inflammation, or iron-deficiency anemia, but rarely indicates something more serious. Our hematologists can guide you on what is causing your child’s high platelet count and what additional tests are required.
Thrombocytopathy (abnormal platelet function). Abnormal platelet function can be inherited and may be a part of a rare genetic disorder. It can also be an acquired problem and related to another underlying condition. In this group of disorders, platelets do not work properly. Additional specialized testing is required if the platelet number is either normal or low-normal, and the platelets do not appear to be working properly. Some cases of rare inherited platelet disorders may have both abnormal platelet counts and function and be misdiagnosed as ITP. Some of these disorders may carry additional risks such as cancer; an accurate diagnosis and counseling are required.
Symptoms of platelet disorders
Bleeding caused by a platelet disorder may appear as a purplish stain on the skin referred to by its size:
- Petechiae (small red or purple dots on the skin)
- Purpura (medium-sized spots on the skin)
- Ecchymoses (larger areas of discoloration or bruising).
Bleeding may also happen in other parts of the body, such as the mucous membranes and gums, from the nose (nosebleeds), and the digestive tract (appearing as blood in the stool).
Diagnosing platelet disorders
The first step in your child's care is making an accurate diagnosis. We will perform blood tests and take a thorough medical history to determine the cause of the excess bleeding and analyze platelet counts and function. If the disorder is inherited, we may conduct genetic testing to determine the cause and offer genetic testing and counseling to interested family members.
Our Approach to Care
Our care teams include pediatric hematologists with years of experience treating platelet disorders, as well as nurses and nurse practitioners, physical therapists, social workers, genetic counselors, and special laboratory personnel who perform platelet function tests. When your child approaches young adulthood, we provide transitional care to encourage them to become independent advocates of their health.
How we treat platelet disorders
We will tailor your child's care to his or her personal needs.
Medical treatment. In mild cases of ITP, no treatment may be needed other than careful observation by your child's doctor. However, if the platelet count gets very low or bleeding is significant, your child may receive medication. Conventional therapies include corticosteroids, immunoglobulin or anti-D immunoglobulin, and drugs that make the immune system less likely to attack. More recently, our doctors set the standard for the use of romiplostim and eltrombopag in children. These are newer drugs that boost platelet production.
Surgery. If medical treatments do not work well enough to control your child's ITP, we may recommend surgical removal of the spleen (splenectomy). However, this form of treatment is reserved for children with very serious cases and those that do not respond well to other therapies.
Why Choose Us
Established over four decades ago, the Platelet Disorders Program at NewYork-Presbyterian Komansky Children’s Hospital was one of the first programs dedicated to children with platelet disorders such as ITP, as well as inherited disorders of platelet production or function. Today children from all over our region come to NewYork-Presbyterian for care. Research conducted over the years has contributed greatly to the development of newer treatment approaches for these disorders. Children are now able to be treated almost entirely on an outpatient basis. We also offer clinical trials of innovative therapies. Call us today to make an appointment.