Pediatric Anemia Pediatric Research and Clinical Trials

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Our research

Our clinicians are conducting innovative research on anemias and red blood cell disorders. In fact, you can say our faculty 'wrote the book' on pediatric blood disorders. Because of our long track record of innovation, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center is considered a world leader in laboratory and clinical research on blood disorders. Learn more about our research.

New developments in chronic anemia

Hemoglobin is a protein in red blood cells that helps them carry oxygen from the lungs to all parts of the body. Without enough hemoglobin, children experience anemia, which causes fatigue, pale skin, an increased heart rate and other symptoms, ranging from mild to life-threatening.

In children with sickle cell disease, hemoglobin is defective, causing red blood cells to become stiff, sticky and shaped like the letter C. In thalassemia, the body’s ability to produce hemoglobin is also compromised. Both disorders can cause severe anemia.

Boston Children’s Hospital's Stuart Orkin, MD, and Vijay Sankaran, MD, PhD, identified a way to compensate for this problem: getting red blood cells to make another type of hemoglobin that normally stops being made after birth.

Legacy of excellence

Boston Children’s has been a leader in developing new treatments for inherited anemias, such as sickle cell disease and thalassemia.

1960s:
David Nathan, MD, pediatrics and president emeritus, recognizes the important role of hypertransfusion in sickle cell disease.

1970s:
Richard Proper, MD, and Nathan demonstrated effective iron chelation by subcutaneous pump deferoxamine (Desferal®).

  • Nathan and Orkin develop the first genetic test for prenatal diagnosis of thalassemia.

1980s:

  • Orah Platt, MD, chief of the Department of Laboratory Medicine, and Nathan use hydroxyurea to induce fetal hemoglobin production in patients with sickle cell disease.

1990s:

  • Carlo Brugnara, MD, director of the Hematology Lab, and colleagues discover that the common antifungal drug, clotrimazole, prevents dehydration in red blood cells, a factor in sickle cell disease.

2000s:

  • A recent study tested the effectiveness of a new oral iron chelator, deferasirox.

  • Orkin and Sankaran identify a transcriptional regulator (BCL11A) critical for hemoglobin switching that could serve as a therapeutic target for hemoglobin disorders.

Clinical Trials

For many children with rare or hard-to-treat conditions, clinical trials provide new options.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

Boston Children's Hospital 300 Longwood Avenue, Boston, MA 02115 617-355-6000 | 800-355-7944

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