The national sickle cell study described in this news release from the Medical University of South Carolina could have a significant impact on the future treatment of children with sickle cell disease. We’re told in the lede that the drug hydroxyurea is as effective as blood transfusions in reducing blood flow speeds in the brains of some children with sickle cell anemia, but it doesn’t tell us on what evidence that conclusion was based. It also falls short in explaining other key details of the study, doesn’t give us any indication of the potential cost savings and doesn’t disclose any of the potential conflicts-of-interest between some of the investigators and drug companies.
Sickle cell anemia is a debilitating disorder with limited treatment options. It affects about 100,000 Americans, and disproportionately affects African Americans, according to the National Institutes of Health. Hydroxyurea is already widely used for treatment so evidence that would support its additional effectiveness for stroke prevention in children with sickle cell disease would be significant, especially if this meant replacing tedious and resource-intensive treatment with frequent blood transfusions.
The news release does not cite costs. This is unfortunate because hydroxyurea treatment would likely be less costly than standard treatment with blood transfusion.
The news release discusses benefits in only the broadest of terms, citing “positive preliminary results,” and fails to quantify those results.
The news release makes no mention of possible side effects related to the use of hydroxyurea. The NIH notes that those risks include a drop in white cell count or platelet count and, in rare cases, a worsening of anemia. According to NIH, “those side effects usually go away if a person stops taking the medication.” There were other adverse events measured in the study that should have been described as well.
The news release says the study shows that some children can be moved from transfusion to medication after at least a year, but it offers no evidence to support that assertion.
The news release should have said more about the study design (non-inferiority, so it was only designed to show that hydroxyurea was not worse than transfusions), the number/rate of stroke in each study arm (although, importantly, this was not the main outcome of the paper so talking about stroke benefit so directly is misleading), and the difference in blood velocity in each group.
The news release doesn’t promote disease mongering. It didn’t oversell the disease’s impact but it perhaps missed an opportunity to inform readers about the disease’s characteristics and rates of stroke in the sickle cell population.
The news release makes no mention of funding sources or conflicts of interest. The study itself states that it was funded by the NIH and notes that 13 of the authors, including the primary investigator, have ties to drug companies.
The news release notes that hydroxyurea is the only drug that has been approved by the Food and Drug Administration to treat sickle cell disease. We rate this Satisfactory since there are no other drug alternatives to name.
It’s understood that the drug is already being used in sickle cell treatment and is FDA approved.
The study authors say that their work is unique: “So far, no study has prospectively compared blood transfusions with hydroxycarbamide [hydroxyurea] for children with sickle cell anemia and abnormal TCD [transcranial doppler] velocities, even though this is the largest group of children with sickle cell anaemia who currently receive chronic transfusions, and no alternative treatment to transfusions is currently available.”
It concludes that the trial results “allow for a major conceptual shift in the long-term management of children with sickle cell anaemia and established cerebrovascular disease.”
The news release contains none of that context.
We didn’t observe any unjustifiable language in the release’s description of the study.