Not just complaints. This time the patients ‘promote’ the work done in centers specialized in the treatment of beta-thalassemia, a rare genetic blood disease. More than 80% positively judge the work performed by the medical-nursing staff and more than 90% appreciate the quality of the services and care provided. A ‘report card’ that emerged from the data contained in the survey ‘The patient at the Centre’ conducted on 222 people affected by thalassemia. This is the first survey ever carried out in Italy that takes a picture of the state of the art in the fight against the disease and was promoted by the Franco and Piera Cutino Foundation in collaboration with the Chair of Social Research Methodology of the Degree Course in Communication Sciences of the University of Palermo.
Seven thousand patients in Italy
Beta thalassemia is a severe congenital anemia caused by a genetic defect. “This defect – Gianluca Forni, director of the Microcitemia Department of the Galliera Hospital in Genoa – determines the lack of production of hemoglobin, the protein contained in red blood cells which is responsible for transporting oxygen in the body. The disease affects more than 7,000 people in Italy. It is possible to treat beta thalassemia with blood transfusions and therapies to remove the excess iron brought about by the same transfusions with drugs called chelators”.
Beta thalassemia, towards a new gene therapy by Letizia Gabaglio 25 January 2023
The identikit of the patient with thalassemia
The survey participants are followed up in three different reference centers of the peninsula: Catania (Policlinico), Cagliari (Microcitemico) and Ferrara (Sant’Anna). “The research also highlights – he explains Lorella Pitrolo, creator of the survey – how 70% of people with beta thalassemia, who participated in the survey, are aged between 45 and 65 years. Half are married and in 37% of cases have at least one child”. The information collected also serves to better understand how the disease has changed over time: “The data we have collected on patient profiles clearly confirms how the prognosis of the disease has changed enormously in the last 20 years – underlines Joseph Cutino, president of the Franco and Piera Cutino Foundation. The merit is to be found in therapeutic innovation and blood donations which have exponentially increased the prospects of patients who are now able to lead an almost normal life”.
Beta-thalassemia, new indications for the drug against anemia by Dario Rubino 08 March 2023
How much patients trust the therapies
Among the other survey data, there are also those relating to the effectiveness of treatments. For 73% of patients, the treatments prescribed have a positive effect on their state of health. However, 20% consider it really complex to adhere to so-called conventional therapies such as blood transfusions and the use of chelating drugs. “According to the survey – underlines Forni – patients see with increasing confidence the innovative therapies recently available also in our country, which aim to reduce the need for transfusions and limit hospital admissions as well as the use of chelating drugs. In fact, 52% of the interviewees express a propensity to change treatment”.
Beta-thalassemia, prenatal diagnosis can avoid over 400 cases a year by Tiziana Moriconi 02 May 2023
They don’t believe in healing
The survey by the Franco and Piera Cutino Foundation highlights the lack of trust that is instead reserved for therapies aimed at healing. More than 50% of patients say they have no expectations. “This trend finds an explanation in the advanced age of the people affected by the disease – he continues Aurelius May, Director of the Campus of Hematology “Cutino” of the PO “Cervello” of Palermo. There are treatments, such as gene therapies, which can definitively defeat the disease. Today they are used only in experimental protocols, dedicated, however, to younger patients. 73% of those interviewed claim that they have never participated in these clinical trials. All this does not polarize the interest of people who tend to already have positive feedback from the treatment used. In order to overcome this ‘gap’, thanks to the establishment of an International Working Group, prognostic scores on complications and mortality have been developed to identify the ideal candidates for gene therapy, even among older patients”.
The stress of transfusions in the summer
Even if they declared themselves satisfied, some critical issues persist in the daily life of the patients, starting from the need to follow the therapeutic path every day. In particular, the continuous blood transfusions, essential to counteract anemia. “Also because – underlines Cutino – especially in the summer, there is a chronic and dangerous lack of blood. A real nightmare: for this reason we invite all citizens, who are in a position to do so, to go to transfusion centers in recent months. Beta thalassemia must be less frightening than in the past also thanks to therapies which, despite some limitations, have truly revolutionized the lives of thousands of people in a positive way. We are also at the forefront in Europe on early diagnosis. However, we still need to work on many aspects in which we are lacking, such as the blood donation network. It must be strengthened as soon as possible especially to be able to face the difficult summer periods”, underlines Cutino.
A good relationship with doctors
The survey also tried to understand the quality of the doctor-patient relationship: “The impact of the disease is still very strong and to deal with it it is essential to be able to establish a virtuous dialogue with healthcare professionals”, he explains Valentino Orlando, President of United. “Over 40% of patients judge the willingness to listen on the part of both doctors and nurses to be excellent. For seven out of ten, however, the prescribed drug treatment was discussed and explained in a clear and understandable way”. The survey, carried out thanks to the non-conditioning contribution of Bristol Myers Squibb, Vertex and Agios and with the patronage of United, the ‘Giambrone, Alt Rino Vullo’ Foundation and Fasted Catania, made it possible to collect positive data which underline the excellent and qualified level of assistance that our national health system is able to guarantee.