Hydroxyurea and transfusion therapy for the treatment of sickle cell disease | oneSCDvoice
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guides & guidelines

Hydroxyurea and transfusion therapy for the treatment of sickle cell disease

key information

source: American Society of Hematology

year: 2014

authors: Susan E. Creary, John J. Strouse

summary/abstract:

Sickle cell anemia (SCA) refers to the clinically similar disorders HbSS or HbSβ0-thalassemia. Sickle cell disease (SCD) refers to all disease genotypes, including SCA and compound heterozygous disorders, such as HbSC, HbSβ+-thalassemia, and other less common variants. The carrier state for hemoglobin S (HbAS or sickle cell trait) is not a form of SCD. Hydroxyurea was approved by the FDA in 1998 for the treatment of clinically severe SCA in adults. Its primary mechanism of action is induction of fetal hemoglobin. Treatment benefits include reduced frequency of acute sickle cell pain and acute chest syndrome, reduced need for blood transfusions and hospitalizations, and possibly improved survival.

Transfusion may be used to treat acute complications of SCD and to prevent chronic complications. Transfusion may also be used in the perioperative period in patients with SCD to prevent vasoocclusive crises, stroke, or acute chest syndrome after surgery. Transfusions can be lifesaving but carry a risk of severe adverse effects including death. Many hazards, such as risk of alloimmunization, are amplified in SCD. Many best practices to minimize adverse effects remain under investigation. RBC units should include matching for C, E, and K antigens (Moderate recommendation, low-quality evidence).

More extensive matching may be done when RBC alloantibodies are present. Consult the blood bank for evaluation of a possible DHTR if acute anemia, pain, or jaundice occur within 3 weeks after a blood transfusion (Strong recommendation, moderate-quality evidence). In patients with SCA who are not chronically transfused and who are therefore at risk for hyperviscosity due to high percentages of circulating HbS-containing erythrocytes, avoid transfusing to a target Hb >10 g/dL (Moderate recommendation, low-quality evidence).

In patients who receive chronic transfusion therapy, perform serial assessment of iron overload by methods such as liver biopsy or MRI techniques (Strong recommendation, moderate-quality evidence). Administer iron chelation therapy, in consultation with a hematologist, to patients with SCD and with documented transfusion-acquired iron overload (Moderate recommendation, moderate-quality evidence).

organisation: The Ohio State University School of Medicine; John Hopkins University School of Medicine

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