Here’s the most important thing to understand about anemia: it’s the result of some underlying disease or condition. This condition could be relatively harmless or quite serious. While it is reversible in pregnancy period or newborn child anemia, the sickle-sell anemia is a complete different situation.
Sickle cell anemia, EU ok at first therapy that prevents ‘painful crises’
Green light from the European Commission to crizanlizumab (Adakveo, Novartis), the first therapy aimed at the prevention of recurrent vaso-occlusive crises (Voc) or ‘painful crises’ in patients with sickle cell disease from 16 years of age. The drug can be given as add-on therapy to hydroxyurea / hydroxycarbamide (Hu / Hc), or as monotherapy in patients for whom Hu / Hc is inappropriate or inadequate.
Sickle cell anemia is a rare haematological disease that affects around 50,000 people in Europe. Crizanlizumab binds to P-selectin, a cell adhesion protein that plays a central role in multicellular interactions that can result in vaso-occlusion.
The European approval is based on the results of the Sustain clinical study, in which crizanlizumab – reports Novartis in a note – significantly reduced the median annual rate of Voc to 1.63, compared to 2.98 for placebo, equal to a reduction of 45%. There was also an increase of more than double the percentage of patients without VOC who completed the study, compared to placebo. Again, reductions in VOC frequency have been observed among patients regardless of sickle cell genotype and / or use of Hu / Hc)”. In the same trial, crizanlizumab was shown to reduce the median annual rate of hospitalization days by 42%.(4 days for crizanlizumab vs 6.87 days for placebo).
The therapy is now authorized in 36 countries around the world, including the US. The European approval of this therapeutic option represents very important news for patients with sickle cell anemia who after many years have at their disposal a new treatment tool capable of improving their quality of life. Only crizanlizumab, thanks to its peculiar mechanism of action, is in fact able to act on the chronic inflammatory vascular disease that underlies the numerous clinical complications present both in adolescents and adults but also in children.
Crizanlizumab, has a profile of uniqueness that makes it very interesting for clinicians, because it could help us manage even those patients who have failed or do not accept therapies considered gold standard for sickle cell anemia, such as hydroxyurea or transfusion regimens.
This milestone will allow us to achieve available to patients the first targeted drug for the prevention of recurrent vaso-occlusive crises, which upset the lives of patients from a physical, social and emotional point of view and can degenerate into acute and long-term complications.