Pediatric Blood Disorders (Noncancer)

Dr. Hogan shares a warm moment with a patient while listening to their heart.
Dr. Marie Martinelli is part of our expert team caring for children with blood disorders.

The team at OHSU Doernbecher Children’s Hospital includes some of the nation’s top specialists in pediatric blood disorders. We work together to provide the best possible care for your child.

  • Our hematologists are blood disorder experts who only treat children.
  • Our team includes leaders of OHSU Hemostasis and Thrombosis Center, one of just 12 federally funded regional hemophilia treatment centers in the U.S.
  • We offer outreach clinics each year in Eugene and Medford.
  • Our providers are also researchers, participating in clinical trials to better understand and treat these diseases. We may be able to offer your child access to promising new treatments.
  • We run the Spots, Dots and Clots Clinic at the OHSU Center for Women’s Health. The clinic helps teens and young women with issues related to bleeding and women’s health.

Understanding pediatric blood disorders

Blood disorders can affect your child’s ability to produce blood cells or affect how their blood cells and blood chemicals work.

A blood disorder might:

  • Limit the blood’s ability to clot (to stop bleeding with platelets or clotting proteins).
  • Cause clots inside blood vessels, which can affect blood flow.
  • Affect the production of red blood cells, which help circulate oxygen through the body.
  • Affect the production of white blood cells, which fight infection.

Bleeding and clotting disorders

What is it?

Children with hemophilia have blood that doesn’t clot properly. Hemophilia can lead to internal bleeding or heavy bleeding from an injury or surgery. It is caused by a mutation in a gene. Hemophilia mainly affects boys. It can be mild, moderate or severe.

Boys with severe hemophilia are often diagnosed as newborns. They may be tested because of a family history of hemophilia or because of bleeding after circumcision. Boys with mild hemophilia may not be diagnosed until they bleed more than normal after surgery or trauma.


If left untreated, hemophilia can cause:

  • Swelling, pain or tightness in knees, elbows and ankles
  • Many bruises
  • Heavy bleeding after vaccinations or minor cuts
  • Bleeding of the mouth and gums
  • Blood in urine or stool
  • Frequent and difficult-to-stop nosebleeds


If your child is diagnosed with hemophilia, they will get preventive treatment to keep them from experiencing symptoms.

  • Your child may receive a protein called a “clotting factor” that their bodies don’t make.  The protein is given as a shot, in an IV or through a port (a small device implanted in the chest).
  • For severe hemophilia, your child will receive clotting factor therapy every few days or weeks. This is called prophylaxis.

What is it?

Thrombosis happens when a blood clot forms inside a blood vessel, preventing normal blood flow. When it happens in a large vein, most often in a leg, it’s called deep vein thrombosis.

A blood clot in a deep vein can travel to the lungs. This can cause a serious condition called a pulmonary embolism, which can lead to injury or death.

Symptoms of deep vein thrombosis:

  • Pain, aching and heaviness or warmth in a leg
  • Discoloration in the leg, or thickening of the skin
  • Swelling in a leg, ankle or foot

Symptoms of a pulmonary embolism:

  • Sudden shortness of breath
  • Irregular or rapid heart rate
  • Chest pain
  • Coughing up blood


  • Medications called anticoagulants, or blood thinners, prevent clotting. They can be given in a shot or pill.
  • Compression stockings to reduce leg swelling.
  • A filter placed in the affected vein can keep clots from traveling to the heart. It is used only when blood thinners aren’t effective or can’t be used.
  • Thrombolytic therapy uses medication to break up or dissolve clots. Tissue plasminogen activator, or tPA, is one type. It is used in children in rare situations.

What is it?

Von Willebrand disease is the most common bleeding disorder in the U.S., affecting about one in every 100 people.

It is almost always inherited genetically. This gene mutation affects your child’s ability to make a clotting protein called von Willebrand factor. Or it causes the protein to not work properly.


  • Frequent nosebleeds
  • Frequent bruising
  • Unusual bleeding during procedures such as surgery or having a tooth pulled
  • Heavy and longer-than-normal menstrual periods


  • Medication that promotes the release of von Willebrand factor. It comes as an injectable or nasal spray.
  • Clotting factor concentrates given in an IV.

What is it?

Thrombocytopenia happens when your child’s blood has an abnormally low number of platelets. Platelets are pieces of blood cells that help form clots that stop bleeding.

Many health issues can cause it, such as infections and disorders in which the immune system destroys platelets.


  • Frequent bruising
  • Small red or purple spots on the skin (petechiae)
  • Blood blisters in the mouth
  • Other signs of unusual bleeding


  • In mild cases, the condition could get better on its own. Your child may not need treatment.
  • In more serious cases, your child may need treatment. We may use medicines that change your child’s immune system.
  • In life-threatening situations, we may treat thrombocytopenia with transfusions of platelets or red blood cells.

We also diagnose and treat:

  • Other coagulation disorders
  • Qualitative platelet disorders
  • Heavy menstrual bleeding
  • Connective tissue disorders
  • Hereditary hemorrhagic telangiectasia
  • Thrombophilia
  • Factor V Leiden
  • Prothrombin gene mutation
  • Protein C deficiency
  • Protein S deficiency
  • Antithrombin deficiency
  • Stroke in children

Hemoglobin disorders

What is it?

Sickle cell anemia is the most common inherited blood disorder. It is caused by a mutation in the hemoglobin, the protein in red blood cells that carries oxygen. All newborns in the U.S. are screened for sickle cell anemia shortly after birth.

Most blood cells are shaped like ovals. If your child has sickle cell anemia, some of their red blood cells are shaped like a "C", or like a sickle.

Their unusual shape causes sickle cells to get stuck in small blood vessels. When this happens, not enough healthy red blood cells carry oxygen through the body. Sickle cell anemia can cause pain and damage organs like the spleen. Damage to the spleen increases the risk of serious infections.

Sickle cell anemia is most common in people of African descent. Some people with Latino, southern European, Middle Eastern, Asian or Indian heritage can also inherit sickle cell anemia.


  • Signs of anemia, such as fatigue and pale skin
  • Yellowish eyes
  • Dark urine
  • Painful swelling in hands and feet
  • Pain in the abdomen, chest and lower back
  • Delayed growth


Until recently, there was no cure for sickle cell disease. Bone marrow transplants that let the body make normal red blood cells have shown promise for children younger than 16. However, transplants do have serious risks. If a transplant is an option for your child, Doernbecher’s advanced bone marrow transplant program is among the best in the nation.

In the next few years, a new kind of treatment called gene therapy may be an option for your child.

All children with sickle cell anemia need to take Penicillin daily. Other treatments that can help with your child’s symptoms include:

  • Pain relievers and drinking lots of liquid
  • Hydroxyurea, a medication that helps reduce the number of red blood cells that sickle
  • Antibiotics and vaccines to fight infection
  • Blood transfusions
  • Folic acid, a B vitamin supplement that can help red blood cells develop.

What is it?

Thalassemia is an inherited condition in which your child doesn’t produce enough hemoglobin. Hemoglobin is the protein that helps red blood cells carry oxygen through the body.

Two important parts of hemoglobin are called “alpha” and “beta.” Thalassemia can happen when your child produces low amounts of either alpha or beta hemoglobin.

The condition ranges from mild to severe. Your child could have minor, intermedia or major thalassemia.


Thalassemia causes signs of anemia (low red blood cell count) such as:

  • Shortness of breath
  • Dizziness
  • A fast heartbeat
  • Leg cramps
  • Headaches
  • Pale skin


  • Folic acid, a B vitamin supplement that can help red blood cells develop.
  • Blood transfusions, given regularly for major thalassemia.

Bone marrow failure syndromes

What is it?

Aplastic anemia occurs when your child’s bone marrow does not produce enough blood cells.


  • Tiring easily or lack of energy
  • Shortness of breath
  • Rapid heart rate
  • Pale skin
  • Frequent bruising
  • Small red or purple spots on the skin (petechiae)
  • Frequent nosebleeds or bleeding gums
  • Blood in stool
  • Frequent infections or fevers
  • Headaches


A bone marrow transplant is the only cure for severe aplastic anemia. If it’s an option for your child, Doernbecher has one of the top pediatric bone marrow transplant programs in the nation.

Immunosuppression drugs, which lower your child’s immune system response, can also help with aplastic anemia. This treatment may increase the bone marrow’s production of blood cells.

Treatment for symptoms:

  • Blood transfusions
  • Antibiotics
  • Medicine to increase white blood cell production

White blood cell disorders

What is it?

Neutropenia happens when your child doesn’t have enough neutrophils. A neutrophil is a type of white blood cell that helps fight infections from bacteria.

Neutropenia often occurs temporarily as a result of chemotherapy. It can also be caused by bone marrow infections or diseases. Rarely, it is caused by an inherited condition.


Neutropenia itself has no symptoms. Your child’s doctor may discover it during a blood test. It can put your child at risk for repeated infections.


Treatment is usually only needed in the rare, inherited type of neutropenia. In the temporary form, we will watch for and treat infections it may cause.

Other disorders we treat

  • Anemia, including iron deficiency
  • Congenital hemolytic anemia
  • Hemolytic disease of the newborn
  • Polycythemia
  • Hereditary spherocytosis and other disorders of the red cell membrane

Autoimmune disorders happen when your immune system makes antibodies against your own cells and tissues.

  • Immune thrombocytopenia (ITP) (antibodies against platelets)
  • Autoimmune hemolytic anemia (antibodies against red blood cells)
  • Evans syndrome (antibodies against both platelets and red blood cells)

Vascular disorders are caused by the abnormal growth of blood vessels or lymph channels in your body’s circulatory system.

  • Hemangioma
  • Capillary malformations
  • Cavernous malformations
  • Sturge-Weber syndrome
  • CLOVES syndrome
  • Epithelioid hemangioendothelioma
  • Gorham Stout disease
  • Kaposiform hemangioendothelioma
  • Kasabach-Merritt phenomenon
  • Klippel-Trenaunay syndrome
  • Lymphatic malformation
  • Tufted angioma
  • Blue rubber bleb nevus syndrome

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Doernbecher Children’s Hospital
700 S.W. Campus Drive
Portland, OR 97239
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Navigating sickle cell disease

A baby boy sitting in a chair smiling.

Diagnosed with sickle cell anemia as a baby, Isaiah may one day need a bone marrow transplant. His care team “uplifts our entire family,” his mom says. Read their story.