Results from a post hoc analysis of the Phase II SUSTAIN study of crizanlizumab, a humanized anti-P-selectin monoclonal antibody being investigated for the treatment of sickle cell disease (SCD), have been published in the American Journal of Hematology. The analysis showed that more patients treated with crizanlizumab did not experience a vaso-occlusive crisis (VOC) vs those treated with placebo (35.8% vs 16.9%), specifically patients with a history of 2-10 VOCs in the previous year.
VOCs are a painful complication of SCD and the main reason why patients seek medical care in hospitals. VOCs, which are triggered by multi-cell adhesion, are associated with increased morbidity and mortality, and can result in stroke, as well as organ damage or failure. Currently, treatment options for VOCs are limited.
‘The unpredictable, intense painful crises that patients with sickle cell disease experience are the hallmark of the disease and the primary cause of hospitalizations in this patient population,’ said Abdullah Kutlar, MD, Director, Sickle Cell Center at the Medical College of Georgia, Augusta University, Augusta, Georgia, and primary author of the SUSTAIN analysis. ‘I am encouraged that results from this post hoc analysis of SUSTAIN study data found that crizanlizumab could substantially delay or prevent these crises, which also may mean less organ damage in the long run.’
‘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,’ said Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development. ‘This is another example of what we mean when we say we are reimagining medicine.’
About the SUSTAIN trial
The Phase II SUSTAIN trial was a multicenter, multinational, randomized, placebo-controlled, double-blind,12-month study to assess safety and efficacy of the anti-P-selectin antibody crizanlizumab with or without concomitant use of hydroxyurea therapy in sickle cell disease patients with sickle cell-related pain crises. Primary results were published in The New England Journal of Medicine and showed that crizanlizumab reduced the median annual rate of sickle cell pain crises (SCPCs) by 45.3% compared to placebo (1.63 vs 2.98, p=0.010) in patients with or without hydroxyurea therapy.
Adverse events that occurred in 10% or more of the patients in either active-treatment group (2.5 mg/kg; 5 mg/kg) and at a frequency that was at least twice as high as that in the placebo group were arthralgia, diarrhea, pruritus, vomiting, and chest pain. There were no apparent increases in infections with crizanlizumab treatment.
Source: https://www.novartis.com/news/media-releases/novartis-analysis-shows-crizanlizumab-seg101-increased-number-patients-free-sickle-cell-pain-crises-vs-placebo-during-sustain-study