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Komansky Center Leads the Way in Benign Pediatric Hematology

NEW YORK (Mar 10, 2014)

Not all blood disorders in children are cancerous. While we often hear about acute leukemia as being the most common type of pediatric cancer, in fact most blood disorders in children are benign. Many of these are inherited and require long-term care. Treating these conditions requires special expertise and a multidisciplinary approach, often integrating cutting-edge research into the clinic.

This approach has been developed and fine-tuned over the past 30 years at NewYork-Presbyterian Phyllis and David Komansky Center for Children's Health – the preeminent center in New York, New Jersey, and Connecticut for managing the broad spectrum of benign blood disorders in children and adolescents. In two particularly strong initiatives, doctors and researchers are refining the care of young patients with platelet disorders and identifying factors that raise the risks of blood clots in hospitalized children.

Scientists also conduct pioneering basic research in the Children's Cancer & Blood Foundation laboratories related to iron metabolism, erythropoiesis (the production of red blood cells), and other advanced areas of study with the potential to improve the care of children with benign blood disorders.

Types of Pediatric Blood Disorders

There are many types of noncancerous blood disorders that may arise in children:

  • Anemia is one of the most common pediatric blood disorders and is characterized by low levels of hemoglobin – the iron-containing protein in red blood cells that transports oxygen. Nutritional deficiencies, especially a lack of adequate iron in the diet, are by far the most common cause of anemia in children as well as adolescents. Other causes may include bleeding, infections, and inherited conditions, such as sickle cell disease and thalassemia.
  • Thrombocytopenia is when a patient has low numbers of blood platelets – the blood cells the body produces to help with clotting. Without sufficient platelets, children are prone to potentially dangerous bleeding. Causes include auto-immune conditions, infections, and certain medications.
  • Neutropenia occurs when one has low levels of infection-fighting white blood cells, making a child prone to illnesses caused by bacteria.
  • Hemophilia and von Willebrand Disease are inherited bleeding disorders, caused by inadequate levels of proteins called clotting factors.
  • Thrombosis is the development of blood clots, which most often start in the legs. If a blood clot dislodges and travels to the blood vessels supplying the lungs or heart, it can be fatal. So treating them early – and preventing them from developing – is key. Children who are hospitalized for a long period of time are susceptible to developing blood clots.
  • Children may also experience bone marrow failure, in which all of the blood counts are low and patients are prone to anemia, thrombocytopenia, and neutropenia.

Pioneering the Treatment of Platelet Disorders

James Bussel, M.D., Professor of Pediatrics at Weill Cornell Medical College, leads the Platelet Disorders Center at the Komansky Center, which sees more patients with these disorders than any other hospital in the Tri-State area. Dr. Bussel has been an international leader in the field. He focuses primarily on immune thrombocytopenias and has authored seminal papers over the years which form the very basis of how these disorders are treated.

James Bussel, M.D.
James Bussel, M.D.

Most children with acute (short-term) low platelet levels can be successfully treated with more standard therapies. But those with chronic (long-term) conditions need more complex, ongoing care. These children remain at high risk of having bleeding episodes, spontaneously or triggered by even trivial trauma. When bleeding occurs, particularly in the brain, it can cause significant damage. Because of this, children with a higher risk of bleeding often need to restrict their day-to-day activities during school, sports, and travel.

The Platelet Disorders Center at the Komansky Center is world-renowned for conducting clinical trials exploring novel drugs for patients with a cause of low platelets called refractory idiopathic thrombocytopenic purpura (ITP). Dr. Bussel has pioneered the use in children of the drugs romiplostim (Nplate®) and eltrombopag (Promacta®), which boost platelet production by stimulating stem cells in the bone marrow. The scope and experience of this program make it unique worldwide.

Research is also under way in the Division of Pediatric Hematology/Oncology to understand how platelets are made. An innovative laboratory-based program led by Beau Mitchell, M.D., Clinical Assistant Professor of Pediatrics, focuses on clarifying the biology of how platelets are created in the bone marrow from stem cells. Advances in this area may be translated to the clinic to help patients in a direct translational "bench-to-bedside" manner.

Pediatric Thrombosis - a Rising Trend

Thanks to medical advances, sophisticated procedures, and complex surgeries, more children with chronic health disorders are surviving with shunts, stents, and surgical hardware inside their bodies. But while these advances are helping these children to live longer, they also place them at increased risk of forming blood clots. Children who've been treated for a disorder called short bowel syndrome and those who have had any kind of organ transplant are also at risk. Doctors have also become more aware of inherited conditions which predispose children to blood clots.

Beau Mitchell, M.D.
Beau Mitchell, M.D.

Indeed, the incidence of thrombosis among hospitalized children has risen ten- to 20-fold in recent years. This alarming rise has made it a challenge to balance the improved survival achieved through medical advances with the secondary risks of interventions, including blood clotting associated with certain treatments.

At the Komansky Center, pediatric hematology specialists have established a pediatric-focused thrombosis program. They have created a registry for any child with a blood clot, coupling clinical information on the child with data on potential risk factors from procedures, immobilization (such as being confined to a hospital bed), factors related to their environment (including lifestyle, medications, and activity), and inherited predisposition. NewYork-Presbyterian Hospital has had a formal thrombosis prevention program in place for adult patients for several years, but the program for children is new.

Nicole Kucine, M.D.
Nicole Kucine, M.D.

The goal is to use the data collected to create an algorithm – a mathematical model – to assess each child's thrombosis risk, which can be used to define the best way to prevent blood clots in that child, and in other children with similar risk factors. The Komansky Center has also established a comprehensive "anticoagulation clinic" for the short- and long-term follow-up of children who have already had a blood clot, which not only provides expert continuous care by a hematologist, but also gathers data which will guide us in the future with regard to choice of treatment type and duration.

"With more data on risk factors for blood clots in children, we will be able to pinpoint which patients need to receive preventive care, and with what drug," said Nicole Kucine, M.D., Assistant Professor of Pediatrics and lead physician for the pediatric thrombosis program. "This is a novel approach for children."

To make an appointment with a pediatric hematology specialist at NewYork-Presbyterian Phyllis and David Komansky Center for Children's Health, call (212) 746-3400. For more information, visit

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