Rare Disease Report

Crizanlizumab Reduces Pain in Patients with Sickle Cell Disease

NOVEMBER 05, 2018
Rachel Lutz
Treatment with crizanlizumab reduced pain in patients with sickle cell disease who were experiencing vaso-occlusive crisis (VOC), according to a release from drug maker Novartis.

VOCs are the most common and painful complications for people with sickle cell disease as well as the main reason patients go to the hospital for treatment. VOCs are also associated with increased morbidity and mortality and can result in stroke or organ damage or failure. There are few available treatment options for VOC.

In a new study, Abdullah Kutlar, MD and investigators from the Medical College of Georgia analyzed 132 patients for 52 weeks in a phase 2 trial to determine the annual rate of VOC seen in patients treated with crizanlizumab compared with a placebo. Each of the patients enrolled in the study had experienced at least 2 VOCs in the 12 months prior to study initiation. A total of 83 patients experienced between 2 and 4 VOC events and 49 patients experienced between 5 and 10 VOCs. Most of the patients had homozygous hemoglobin S (HbSS), the most common sickle cell disease subtype. These patients were split evenly among the study cohorts. The remaining participants were classified as “non-HbSS” patients.

A total of 67 patients received crizanlizumab 5 mg/kg and 65 received placebo.

According to the study investigators, crizanlizumab can prevent VOCs in patients with 2-4 events in the year leading up to the study initiation. The drug was found to prevent these VOC events for 17 of 42 patients in the treatment arm.

Crizanlizumab also prevented VOCs in patients with between 5 and 10 events in the year leading up to the study intervention. It worked for 7 of 25 patients in the treatment group and only 1 patient out of 24 in the placebo cohort saw improvement.

For the patients with HbSS, crizanlizumab treatment was found to be effective in 15 of the 47 patients in the treatment group compared with 8 of the 47 placebo patients.

Some of the patients in the study used concomitant hydroxyurea therapy to treat their sickle cell disease, but crizanlizumab was still able to prevent VOC events in 14 out of 42 patients in the intervention cohort, and 7 out of 40 patients in the placebo group.   

“The most surprising observation to me was the number of patients that did not experience a VOC event during the study,” primary study author Dr. Kutlar told Rare Disease Report® in an interview. “This was true both for the patients who had 2 to 4 VOCs in the previous year (40.5% vs 24.4% for placebo) and those who had 5 to 10 VOCs (28% vs 4.2% for placebo), indicating the efficacy of this drug.”

The group is continuing to hold discussions with health authorities on their progress.

“If approved by the US Food and Drug Administration, crizanlizumab will be used as a very effective intervention for preventing VOCs,” Dr. Kutlar added.

The most common adverse events—occurring at least twice as often in the active group than the placebo—were arthralgia, diarrhea, pruritis, vomiting, and chest pain. The investigators reported no apparent increases in infections when using crizanlizumab.

“The insights gained from this analysis and others from the SUSTAIN study, strengthen our belief that crizanlizumab may become an important new therapeutic option for sickle cell patients who continue to need step changes in medical innovation,” Samit Hirawat, MD, the Head of Novartis Oncology Global Drug Development said in a statement.

The study, “Effect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: A SUSTAIN study analysis,” was published in The American Journal of Hemotology.

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