Tag Archive for: sickle cell anemia

boy using spirometer

Hydroxyurea initiation improves spirometry results in children with sickle cell anemia

boy using spirometer

Hydroxyurea therapy may help preserve lung function over time in children with sickle cell anemia.

Children’s National researchers participated in a recent study suggesting that the use of hydroxyurea (HU) therapy in children with sickle cell anemia (SCA) may help preserve lung function over time.

Sickle cell disease is associated with various pulmonary complications, including reactive airways, acute chest syndrome (ACS), pulmonary fibrosis and pulmonary arterial hypertension, which can lead to increased morbidity and mortality. Lung function tends to decline over time in individuals with SCA, and a more irreversible restrictive pathology develops with age and increasing respiratory complications.

Hydroxyurea is a disease-modifying therapy that has been shown to lower the rates of several complications of SCD, such as ACS and painful vaso-occlusive crises. It also might help lessen the severity of airway hyperreactivity in children.

This study provides evidence that hydroxyurea therapy may have a positive impact on pulmonary function in children with SCA, which could be of interest to doctors treating this patient population. By slowing the decline in lung function, hydroxyurea therapy may help reduce the risk of pulmonary complications and improve the overall health outcomes of children with SCA.

You can read the full study, Spirometric Changes After Initiation of Hydroxyurea in Children With Sickle Cell Anemia, in the Journal of Pediatric Hematology/Oncology.

Authors on the study from Children’s National Hospital include Dinesh Pillai, M.D., Deepika Darbari, M.D., and Anastassios Koumbourlis, M.D., M.P.H.

Blood Transfusion

Hydroxycarbamide effective in sickle cell stroke prevention

Blood Transfusion

Hydroxycarbamide treatment is on par with blood transfusions for preventing stroke in patients with sickle cell anemia.

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What’s known

Strokes are common and devastating complications for patients with sickle cell anemia, often leading to severe and lifelong motor and neurocognitive problems for people with this congenital blood disorder. Results of a clinical trial published in 1998 showed that having regular blood transfusions could reduce the risk of having a first stroke by 90 percent in children with sickle cell anemia. Since then, doctors have employed this prophylactic treatment widely. However, blood transfusions can be painful, inconvenient and carry substantial risks themselves — including the potential of blood-borne infections, iron overload and immune-related reactions to blood products. Finding a way to reduce stroke risk without over-relying on blood transfusions could substantially benefit patients with sickle cell anemia.

What’s new

A team of researchers, including Naomi L.C. Luban, M.D., a Children’s National Health System hematologist and laboratory medicine specialist, tested transfusions against a drug treatment called hydroxycarbamide in a clinical trial to see if the pharmaceutical intervention could reduce strokes at least as well as transfusions. The clinical trial, known as “TCD With Transfusions Changing to Hydroxyurea (TWiTCH),” assigned 60 patients with sickle cell anemia who had abnormally high transcranial Doppler (TCD) flow velocities—a measure of blood flow in the brain that suggests elevated risk of stroke—to receive hydroxycarbamide instead of transfusions. The research team compared the outcomes for these patients with 61 other patients who received standard prophylactic transfusions. Over the 24-month study period, neither group experienced any strokes, although three transient ischemic attacks (a temporary blockage of blood flow in the brain) occurred in each group. These comparable findings suggest that hydroxycarbamide treatment, also known as hydroxyurea, is on par with transfusions for preventing strokes in patients with sickle cell anemia.

Questions for future research

Q: Does hydroxycarbamide offer a long-term way for patients with sickle cell anemia to avoid transfusions?
Q: Could hydroxycarbamide help patients with sickle cell anemia who already have suffered a stroke or who have had severe problems with blood vessels in their brains that impair blood flow?
Q: Which other treatments can help patients avoid the myriad complications that accompany sickle cell anemia?

Source: “Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anemia—TCD With Transfusions Changing to Hydroxyurea (TWiTCH): A multicentre, open-label, phase 3, non-inferiority trial.” Ware, R.E. B. R. Davis, W. H. Schultz, R.C. Brown, B. Aygun, S. Sarnaik, I. Odame, B. Fuh, A. George, W. Owen, L. Luchtman-Jones, Z.R. Rogers, L. Hilliard, C. Gauger, C. Piccone, M.T. Lee, J.L. Kwiatkowski, S. Jackson, S.T. Miller, C. Roberts, M.M. Heeney, T.A. Kalfa, S. Nelson, H. Imran, K. Nottage, O. Alvarez, M. Rhodes, A.A. Thompson, J.A. Rothman, K.J. Helton, D. Roberts, J. Coleman, M.J. Bonner, A. Kutlar, N. Patel, J. Wood, L. Piller, P. Wei, J. Luden, N.A. Mortier, S.E. Stuber, N. L. C. Luban, A.R. Cohen, S. Pressel and R.J. Adams. Published by The Lancet on Feb. 13, 2016.