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02 February 2017

An antibody treatment has shown promise as a new treatment for sickle cell disease, researchers reported last night.

The drug reduced the number of annual crises by 45% compared with placebo, according to the findings of the study reported in the New England Journal of Medicine.

The antibody targets the protein P-selectin, which is involved in adhesive interactions between red blood cells and other cells during vaso-occlusive crises.

Some 198 patients at 60 clinics in the USA, Jamaica and Brazil took part in the study that ran over the course of a year.

As well as showing a reduced number of crises, the study found that the median time to the first crisis increased from 1.38 months to four months.

Writing an editorial in the journal Professor Martin Steinberg, of the Boston University Medical Center, Massachusetts, USA, says doctors need to consider developing combination treatments for sickle cell disease - using hydroxyurea and other new drugs.

He points out that patient questionnaires reported "low" clinical benefit from the treatment in spite of the reduction in crises.

He says: "Treatment for sickle cell anaemia is now limited to a single drug, hydroxyurea, that helps most but not all patients.

"The trial showed that another drug, which works by a mechanism distinct from that of hydroxyurea, had beneficial effects and could be used alone or with hydroxyurea to prevent severe disease complications."

Source: Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease NEJM 2 February; doi: 10.1056/NEJMoa1611770

Link: http://www.nejm.org/doi/full/10.1056/NEJMoa1611770


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