Sickle cell anemia, occlusion of blood vessels and pain for 39% of patients

39% of patients with sickle cell disease or sickle cell disease suffer from occlusion of blood vessels and pain. They are painful crises that affect the chest, abdomen and joints and which, sudden and frequent, are often managed in one’s home (in 24% of cases), without resorting to medical assistance. How are they addressed? Drinking and resting (74%).

This is what emerges from “Sway” (Sickle cell world assessment survey), an international survey, sponsored by Novartis, on over 2,000 patients suffering from sickle cell disease, which involved various countries including Italy.

Vessel occlusive crises (Voc) and the related acute complications, however, represent only the visible tip of the iceberg: between one pain crisis and the next the vaso-occlusion process continues to take place and produces effects that cause vascular damage, leading to progressive organ damage with reduced functionality. In the absence of adequate treatment, therefore, there can be an impact on the life expectancy of patients which, even in developed countries, is about 20 years lower than the general population.

According to the analysis, the risks of this subtle pathology are most underestimated by Italian patients. Of the 55 interviewed, in fact, as much as half declared that they do not consider it appropriate to go to the doctor or hospital in the case of VOC because they consider the available treatments not resolving and decide to stay at home waiting for the pain to pass. 72% also say they hope for a more effective therapeutic alternative than those currently available.

The strong negative impact on the quality of life of patients also emerges from the narrative survey (in progress) carried out by the Health and Health Area of ​​Istud, with the collaboration of Novartis Italia, according to which patients represent the disease as something “serious and disabling… you are young with the body of an old man… unable to live a normal life and work like everyone else with serenity ”. Furthermore, 40% of the interviewees declare that they are absent from work due to illness with an average of 47 days of work per year and, in the most serious cases, that they resort to transfusions even every 12 days.

These themes are at the center of the cycle of four meetings promoted by Inrete, with the non-conditioning contribution of Novartis, which opens today with the regional tables of Emilia-Romagna and Veneto and which will continue with Sicily and Lombardy. The appointments aim to initiate a constructive discussion on “sickle cell disease” in the presence of clinicians, technicians, local and central institutions and patient associations.

To date, the only decisive weapon to combat the disease is stem cell or bone marrow transplantation. Alternatively, patients resort to blood transfusions or, to reduce the number of seizures, hydroxyurea. However, the research does not stop and new therapeutic strategies are on the way as explained by Professor Lucia De Franceschi, Department of Medicine, University Hospital of Verona and contact person for Anemie Rare Eurobloodnet (European Network for rare diseases): “In Europe it has recently been approved a monoclonal antibody that acts on a specific target, p-selectin that mediates cell adhesion, preventing and significantly reducing vasoconstrictive crises. Its introduction into clinical practice will also allow us to evaluate its impact on the pathology in the long term as well as on the severity of clinical manifestations involving target organs such as the lung, kidney, brain, bone or liver, with a possible positive impact on the patient’s quality of life “.

Life whose expectancy in addition to being reduced implies pain, physical and cognitive deterioration with a decrease in work and school productivity in addition to a greater number of consumption of health, outpatient and hospital resources. Going to the hospital, especially for young patients, can also be a challenge. In fact, as Raffaella Colombatti, of the Pediatric Oncohematology of the University of Padua, comments, “the adolescent and young adult go to hospital less willingly because the emergency management paths of sickle cell disease are less known in the world of adults and not always the patient finds doctors who are experts on his illness, so he tries to stay at home and manage himself as long as he can. Another crucial aspect – continues Colombatti – is the age of transition: many adolescents have to learn to manage their pathology independently and the transition from the world of pediatrics to the world of adults is not easy ‘.

Also according to Fabrizio Canonici and Michel Gyslene Nkongne – respectively president and vice president of the Sickle Cell Disease Association – and Costanza Musci, councilor of the National Federation of United Onlus Associations – the role of the caregiver is not easy because “he must learn to recognize the signals that anticipate a crisis, especially in an adolescent patient, to try to minimize its impact and suffering ”. Suffering that, according to the representatives of the two associations, “must be avoided with treatments that intervene to prevent crises, because when the crisis is over you have already suffered a lot and therefore, we look forward to drugs that can minimize the effect of crises or that can prevent them “.

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