“Sickle cell anemia is a congenital and very important disease, regardless of its incidence, with an enormous impact on the patient’s quality of life due to the consequences it can afflict even at an early age. A disabling disease, however little diagnosed. We have little certainty about the numbers of the disease because screening tests, apart from the pilot programs, have never been activated. Furthermore, there is no patient register in Italy. For this reason, the new therapy for the prevention of recurrent vaso-occlusive (VOC) crises in patients with sickle cell disease, aged 16 years and older, who presented at least two VOCs in the previous 12 months, is good news. “Thus Arcangelo Prete, president of the Italian Association of Pediatric Hematology and Oncology (Aieop) speaking this morning at the online press conference on the approval, by Aifa, of the reimbursement of Adakveo * (crizanlizumab).
“We are talking about a product – continues Prete – that we all hope can give us a great hand in solving the problems of these patients, but let us remember that the disease can manifest itself even before the age of 16”. At the press conference, entitled “Anemia sickle cell: the first targeted therapy for the prevention of recurrent vaso-occlusive crises arrives in Italy “, moderated by Ilaria Ciancaleoni Bartoli, director of the Rare Diseases Observatory, attended by Lucia De Franceschi, associate professor of internal medicine at the University of Verona, Aurelio Maggio, director of the Department of Hematology and Oncology of the “Cervello” hospital in Palermo, Raffaella Colombatti, oncohematology clinic of the Department of Women’s and Child Health – Padua hospital and coordinator of the Aieop Red Blood Cell Diseases Working Group and Luigi Boano, General manager of Novartis Oncology Italy.
“Patients suffering from sickle cell anemia live with the disease throughout their lives – underlined Colombatti -. Two recent studies, one national and one international, have explored the weight of the disease on the quality of life and have come up with very important information as regards the Italian population distributed throughout the country “.
“From the Italian study Scan (Sickle Cell Anemia Narrations) of narrative medicine – he underlines – the loneliness of patients in their path of diagnosis and therapy emerged. Just think that in 33% of cases sickle cell anemia was mistaken for another pathology, with a consequent enormous diagnostic delay and a wandering between the various structures. The study also showed that as many as 53% of patients had to be absent from work and 33% of children and students lost school days due to and if we think that in a year 80% of the days of life of children, adolescents and adults are spent with pain, not always to be managed in hospital, we can understand what impact sickle cell anemia has on the lives of patients and family members “.
Colombatti then pitted other data: “According to the international Sway (Sickle Cell World Assessment Survey) research conducted in 16 countries and which involved more than 2000 patients (including a large group of Italians), 91% of patients report at least one Vaso-occlusive crisis in the 12 months prior to the investigation, most of which managed with medical intervention, with an average variable between 5 and 6 crises per year. We understand from these scientific data, as well as from the patients’ stories, how in fact the disease weighs on their lives ”.
Recurrent vaso-occlusive crises in patients with sickle cell disease “are serious and unpredictable events”, continues Colombatti, and this “is a cause for anxiety for patients, because VOCs can occur when the child, the adolescent rather than the adult have done a whole range of programs. But it is also true that at the base of the crises there may be triggers in some seasons (autumn and winter), in certain age groups. Some infections can also cause VOCs. In children, for example, some crises are frequently triggered by the flu when they go to kindergarten and school ”.
Since 1972 Aieop “has been taking care of and assisting pediatric and adolescent patients and, in some cases, young adults with oncological and non-oncological haematological pathology – concluded Prete -. Over time, the association has taken root in the area with the intent to facilitate access to care for our patients as close as possible to their homes, to improve their quality of life and prevent them from migrating to health care “.
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