New sickle cell drug may reduce pain

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A drug that improves blood flow in patients with sickle cell disease (SCD) appears to shorten painful episodes and increase the proportion of patients whose painful symptoms are resolved within a week, according to results of a new study in the Journal of the American Medical Association.
SCD is an inherited disease in which patients have “sickle-shaped” red blood cells. The disease can result in painful episodes in which red blood cells clump together and blood vessels become blocked.

In this study, researchers tested a new drug called purified poloxamer 188, which increases oxygen flow to tissues, thereby reducing inflammation, pain, and the duration of painful episodes in SCD patients.
To test the drug, they compared the duration of painful episodes in 255 hospitalized SCD patients who took either purified poloxamer 188 or a “dummy” pill (placebo). On average, painful episodes were 9 hours shorter in the group taking the drug compared to those taking the placebo. The results were even more pronounced in children aged 15 years or younger and in patients who were receiving another drug called hydroxyurea. Further, more patients taking the drug were able to resolve painful symptoms within a week compared to those taking the placebo.
In a related editorial, Ronald L. Nagel notes that the results are “important but modest,” and the authors suggest that further studies will be needed.

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