As many of you will know Crizanlizumab (Adakveo) was recommended as a new disease modifying treatment for sickle cell anaemia  by National Institute of Clinical Excellence ( NICE) and NHS England on a Managed Access Agreement. This means that the treatment was recommended to eligible patients under an agreement to collect more data about it.

We have now been informed that preliminary findings from the ongoing worldwide clinical study called STAND (NCT038147460) indicates no statistically significant difference between Crizanlizumab 5mg/kg Crizanlizumab7.5mg/kg and placebo in making a difference to a sickle cell crisis. However, these findings are inconsistent with previous trial results from the SUSTAIN (NCT01895361) trial. As a result of this regulatory bodies such as the European Medicines Agency (EMA) will undertake a review of Crizanlizumab to evaluate the impact of these contrasting results on its currently authorised use.

We would like to reassure those who are taking Crizanlizumab , or who may be contemplating taking it, following discussions with their Health Care Professional, that it is safe. However, if you have any concerns, we recommend that you speak to your Consultant Haematologist in the first instance who will determine the best option for each patient depending on their individual situation.

We will provide more information when we have it.