“Thanks to ravulizumab, the treatment of the syndrome takes another step forward. The therapy not only radically modifies the natural history of the disease, but significantly improves the quality of life of patients and their families”. Thus Gaetano La Manna, associate professor of Nephrology at the University of Bologna, today in Milan, during an event with the press organized by Alexion, AstraZeneca Rare Disease, comments on the reimbursement of ravulizumab for the treatment of hemolytic-uremic syndrome (Seu ) that damages the blood vessel walls of the kidneys and other organs. “Today – explains the specialist – the primary objective of the treatment of the syndrome consists in the ‘switching off’ of the complement system and, in particular, of the C5 protein. To do this, until now we proceeded with the administration of eculizumab, a humanized monoclonal antibody complement inhibitor capable of blocking C5”, administered every 2 weeks. Now the interval between one administration and the next is extended to 8.
Atypical Seu “is a cornic disease with exacerbations that affects 0.5 people per million inhabitants – illustrates La Manna – Before these therapies, 50% of cases in the first clinical manifestation led to dialysis, and 75% over the years reached end-stage renal failure It occurs suddenly following a trigger event – infection, surgery, forms of systemic inflammation, childbirth, other pathologies such as hypertension – with a vague clinical picture, with a fall in platelets and an increase in other molecules typical of acute inflammation, which make diagnosis difficult.In this context there is a sudden renal failure followed by alterations in other organs with complications including neurological and cardiac because the system, out of control, affects the small vessels , causing blood clots and damage”.
“Ravulizumab changes the patient’s life path – underlines the nephrologist – Before this drug, the patient had to have plasmapheresis or plasma, only in the acute phase. There were no therapies, but an attempt was made, through plasma, to supply substances and which serve to protect themselves from inflammatory damage and which the patient does not produce due to the disease. However, the results were very poor”. You went on dialysis and even more kidney transplants were needed. “With this drug, this condition is overcome and a therapy is proposed that is capable, regardless of the trigger element – specifies the expert – of blocking the cascade of events that cause damage to the patient”.
The new monoclonal antibody “is a precision therapy – continues La Manna – It identifies a molecule with a key role in the development of the inflammatory process, the C5 component of the complement degradation pathway, an intersection of all activation pathways, very of this inflammatory process. Blocking this point means doing precision medicine, preventing the trigger event, which activates the system, from producing the damage. In fact, C5 is the molecule that is before the activation of the damage, which takes place in an attack by the system on the patient himself”.
This new therapeutic approach has a major impact on the patient and the healthcare system. Avoiding the morbid condition of dialysis 3 times a week and transplants – it was reported during the meeting – has a significant importance on the quality of life, above all and one considers that now the administration of the therapy passes from 2 to 8 weeks. Compared to the National Health Service, “just make the comparison with respect to the times for admission to dialysis which – recalls the nephrologist – has costs of 45 thousand euros / year per patient, which are considerably lengthened, if not eliminated. There are studies that have compared ravulizumab with 100% positive findings on the quality of life of patients, who in a larger population do not need dialysis or transplantation and, in the event of a transplant, maintain the organ”.
In the face of effective and long-lasting therapies, “there is a defect in the diagnosis of the pathology for a beginning that can be confused with other pathological pictures – remarks La Manna – The diagnosis is random. There are algorithms tested in hospitals, but they are often not considered due to the low possibility of identifying the key aspects of this disease.It is associated with renal insufficiency, by means that are not well codified, and one arrives at the nephrologist, who is the specialist of choice.The pathology – concludes La Manna – is very complex. It also requires medical training, which is not always aware of its existence”.
