Today we’ll be discussing a new set of guidelines for treating patients with sickle cell disease recently published in JAMA by the United States National Heart, Lung and Blood Institute.
Sickle cell disease affects about 100,000 people in the United States. A single amino acid substitution in the beta-globin chain of hemoglobin transforms red blood cells into a sickle or crescent shape. These abnormally shaped cells increase blood viscosity and are unable to normally interact with leukocytes, platelets, and the vascular endothelium. This can lead to both acute and chronic problems.
Here is Dr. Buchanan who helped draft the new guidelines:
“We divided them up into five major categories: health maintenance, acute complications, chronic complications, and then the two disease modifying therapies—hydroxyurea and blood transfusions. A huge section of the guidelines that is devoted to transfusions and the reason for that is simply because the importance of transfusion for these patients.”
Red blood cell transfusions prior to surgery are important because transfused red cells dilute sickled cells and mitigate potential adverse events. The guidelines recommend transfusions prior to surgery to increase hemoglobin levels to 10 g/dL.
To prevent strokes in children less than 16 years of age, the guidelines recommend examining blood flow via annual transcranial Doppler ultrasonography, and long-term transfusion therapy for the children with abnormal results.
The guidelines also strongly recommend transfusion therapy for patients with symptomatic acute chest syndrome and anemic patients with acute splenic sequestration.
For patients undergoing long-term transfusion therapies, it is strongly recommended that iron overload be assessed.
Likewise, Dr. Buchanan and the report emphasize vigilance for other transfusion complications. Here is Dr. Buchanan:
“The problem with alloimmunization in people with sickle cell disease is a substantial one. So the recommendation is quite a strong one in order to do red cell phenotyping on all sickle cell patients, identify and monitor for the development of autoantibodies.”
In addition to guidelines on transfusion support, the report outlines other recommendations such as daily oral hydroxyurea and penicillin prophylaxis in young children. The complete guidelines are online.
We’ll be back with another edition of Transfusion News on October 15th. Thanks for joining us.
References
1. DeBaun MR. The challenge of creating an evidence-based guideline for sickle cell disease. JAMA 2014;312: 1004-5.
2. Yawn BP, Buchanan GR, Afenyi-Annan AN, Ballas SK, Hassell KL, James AH, Jordan L, Lanzkron SM, Lottenberg R, Savage WJ, Tanabe PJ, Ware RE, Murad MH, Goldsmith JC, Ortiz E, Fulwood R, Horton A, John-Sowah J. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA 2014;312: 1033-48.
3. Evidence-Based Management of Sickle Cell Disease: Expert Panel Report 2014. Accessed online http://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines/. September 20, 2014.
My interests relate to cord blood stem CD34+ cell (now in 5% of MNC yields) can provide substantial numbers of gene modified cells needed for effective therapy. I thought that in the USA and elsewhere Bone marrow transplantation that Substantially increases the chances of treatment encouraging progress was being made.
Would it help patients to be informed to have hopes or would it be worrying and the time has not arrived?
I think apheretic erythroexchange has valuable advantages with respect to simple transfusion either in terms of minor alloimmunization and minor iron overload! (You can search my published paper If You wish)