Blood Disorders in Children

  • Dr. Ambrusko, MD with patient.

The Roswell Park Oishei Children’s Cancer and Blood Disorder program was created for patients with all types of pediatric cancers and blood disorders. Unlike pediatric cancer, which can sometimes be treated aggressively and quickly, blood disorders are oftentimes ongoing. Our program offers the comprehensive support that your child and family needs every stage of the patient’s blood disorder journey.

Blood disorders we treat include:

Sickle Cell Disease in Children

  • Sickle cell disease (SCD) describes a group of inherited red blood cell disorders. Children born in the United States with SCD are detected at birth, during a newborn screening program.
  • Diagnosis is confirmed by a simple blood test.
  • Some children will have complications within their first year of life.
  • Some children have complications later in childhood.

Treating Children with Sickle Cell Disease

Treating SCD is complex and takes the specialized care of a pediatric hematologist.

Medicines and Vaccines

With SCD, the spleen doesn’t work the right way and it is easier for children with SCD to get severe infections. Because of this, children are prescribed daily penicillin to lower the chance of infection caused by the pneumococcus bacteria.

Most children with SCD should take hydroxyurea, a daily medicine that raises the amount of hemoglobin F that is shown to reduce or prevent certain SCD complications.

Children with SCD should receive all recommended childhood vaccines including:

  • Pneumococcus
  • Meningococcus
  • Influenza

Screenings and Tests

Children with SCD need frequent blood tests to set up a “baseline” for complications such as anemia.

Children from ages 2 to 16 should have a Transcranial Doppler (TCD) ultrasound screening yearly to learn if they are at a higher risk for stroke. If the test comes back abnormal, regular blood transfusions can lower the chance of having a stroke.

Transfusions

Some children will benefit from a red blood cell transfusion regimen to treat and prevent certain SCD complications. Transfused red blood cells have normal hemoglobin and can help lower the chance for blocked blood vessels and improve oxygen delivery to the tissues and organs.

We also offer erythrocytapheresis, a special kind of chronic transfusion therapy where patients undergo a filtering process of the blood to replace sickle red blood cells with normal hemoglobin.

The Cure for Children with Sickle Cell Disease

At this time, hematopoietic stem cell transplantation (HSCT) is the only cure for SCD. Here is how it works:

  • Donor stem cells are taken from the bone marrow or blood of someone who does not have SCD, but is otherwise a genetic match with the child.
  • Once the stem cells are taken from the donor, the child with SCD is treated with drugs that destroy or reduce his or her own bone marrow stem cells.
  • At that point, the donor stem cells are injected into the child and make a home in the bone marrow, where they begin to replace the recipient’s cells.
  • The new stem cells will make red blood cells that contain normal hemoglobin and do not sickle anymore.

Thrombosis in Children

Your child can be treated for thrombosis in our new family-centered facility. Our doctors are experts in treating all illnesses in the group of thrombotic disorders and thrombophilias.Thrombosis is when a blood clot forms inside a blood vessel, called a thrombus, and blocks the flow of blood through the circulatory system.

The main causes of thrombosis include:

  • Conditions that make the blood coagulate easily
  • Injury to nearby tissues
  • Conditions that prevent blood from flowing normally

If a thrombus grows large enough to reduce blood flow, it can cause tissues to not get needed oxygen.

Treating Children with Thrombosis

Our doctors will carefully look at your child’s case and decide on the best treatment plan.

Your child may receive these treatments:

  • Anticoagulants – medicines that help thin the blood
  • Thrombolytic agents – medicines that help break up clots
  • Replacement factors – natural substances that control clotting

Patients’ treatment needs vary based on what causes the clots. Some children need treatment on a short-term basis, while others may need treatment for life.

Hemophilia in Children

Hemophilia is a genetic disorder in which a child cannot stop bleeding. Even though this is a lifelong condition, if it is well-managed your child can have a healthy, active life.

Signs and Symptoms of Hemophilia

Signs and symptoms vary, depending on each child’s level of clotting factors.

For example, if a child’s clotting factor is slightly lower, he or she may bleed mainly after surgery or trauma. If it is severe, the child may have sudden bleeding, which includes:

  • Nosebleeds
  • Many large or deep bruises
  • Blood in urine or stool
  • Pain, swelling or tightness in joints
  • Unexplained irritability in infants

Hemophilia may appear in baby boys as lengthy bleeding after circumcision. For boys who aren’t circumcised and for girls, hemophilia may appear as easy bruising when the child becomes mobile. Most often, the first episode of bleeding happens by the time a child is 2 years old.

Whether your child has been diagnosed with hemophilia or you suspect your child may have the disorder, we can help you find answers.

We have a team of doctors who specialize in blood disorders and an entire support staff to provide your child with the best care. Inpatient care is provided at Oishei Children’s and outpatient care is offered at the WNY Hemophilia Center.

The Vascular Anomalies Program for Children

We provide a monthly clinic to see and treat children who have vascular anomalies (a defect in blood vessels that can affect capillaries, arteries, veins, lymphatics or a combination of these), or tumors. Our team is there for you and includes:

  • A pediatric surgeon
  • A pediatric dermatologist
  • A pediatric interventional radiologist
  • A pediatric hematologist-oncologist

General Hematology Care for Children

Anemia

Anemia is caused when the body doesn’t have enough red blood cells or hemoglobin (the part of red blood cells that binds to oxygen and gives blood its red color) to carry oxygen to tissues and organs. There are many types of anemia, and they are very different in their causes and treatments.

Symptoms of anemia:

  • Anemia in children is often found during routine well-child visits
  • Some cases are found when children present with common symptoms such as:
    • Pallor
    • Listlessness
    • Fatigue

The first thing we do is learn if the anemia is caused by one of three main groups:

  • Blood loss
  • Lower red blood cell production (often from low iron)
  • Increased red blood cell destruction

We may also review other parts of the complete blood count, and study how the red blood cells look to help learn the type of anemia.

From there, more lab tests will be done, based on the suspected cause of anemia. In some cases, we may need to examine the bone marrow.

Idiopathic Thrombocytopenic Purpura

Idiopathic thrombocytopenic purpura (ITP) is a platelet disorder that can lead to easy bleeding or extreme bruising.

The disorder is also called immune thrombocytopenia and affects children and adults. Often, children get ITP after a viral infection and usually recover fully without treatment. If your child doesn’t have signs of bleeding and their platelet count isn't too low, he or she may not need any treatment.

In rare cases, the number of platelets may be so low that dangerous internal bleeding occurs. If your child needs treatment, our specialized doctors are experts at caring for children with ITP.

Von Willebrand Disease

Von Willebrand disease (VWD) is a bleeding disorder where a person either has low levels of a protein called Von Willebrand factor or the protein doesn’t work well.

If blood doesn’t clot well, heavy bleeding that is hard to stop can take place. The bleeding can damage a child’s internal organs.

Typically, when a blood vessel is injured a person begins to bleed. Small fragments called platelets stick together to stop the bleeding. Von Willebrand factor acts like glue to help the platelets stick together.

Von Willebrand factor also carries clotting factor VIII (8), another protein that helps make blood clot. Factor VIII is the protein that's missing or doesn't work well in people who have hemophilia bleeding disorder.

However, VWD is milder and more common than hemophilia. This disease is the most common genetic bleeding disorder and affects both boys and girls.

Our doctors have the knowledge and expertise to treat your child if he or she has this condition.

Rare Blood Disorders in Children

We know that learning your child has a rare blood disorder can be devastating. You can call on our team of hematology specialists for their expertise in treating patients with unusual diseases and for the resources they provide in complex cases.