Primary immune thrombocytopenia, prolonged use of corticosteroids compromises quality of life

During the event ‘Primary immune thrombocytopenia: changing the approach to change life’, promoted by Inrete with the non-conditioning contribution of Novartis, the study ‘Analysis of the epidemiology and drug use of patients suffering from Primary immune thrombocytopenia in Italian contexts ‘, conducted by CliCon Srl. The round table also offered the opportunity to illustrate the results of the international study’ iWISh ‘which photographs the negative impact on patients’ quality of life, also linked to prolonged therapy with corticosteroids. Primary immune thrombocytopenia (Itp) is a rare form of autoimmune blood clotting disease characterized by platelet deficiency. A little known pathology, but with negative effects on the quality of life of the people who suffer from it and which needs an optimization of the therapeutic management currently characterized by the prolongation of therapy with corticosteroids beyond the 3 months provided for by the guidelines. Guidelines that establish a precise therapeutic path capable of promoting an improvement in the quality of life of patients and, at the same time, helping to make healthcare resources more efficient. “Based on the analysis conducted in the Italian context – explained Luca Degli Esposti, President CliCon Health, Economics and Outcomes Research – the prevalence of primary immune thrombocytopenia was 262 cases / million serviceable. About two thirds of patients were treated with corticosteroid drugs and, among these, most had a exposure of more than 3 months of therapy. This experience has confirmed the possibility of refining the estimates on epidemiology and methods of treatment of patients with rare diseases using the health data available and, consequently, to have an additional knowledge element for management. of the pathology and the evaluation of pharmacological therapies “. The observational analysis and is based on a national sample of approximately 9 million patients, of which 2,869 affected by Ito. Of the latter, 65.6% – being treated with corticosteroids – had an average age of 49.9 years. Patients treated with corticosteroids with a duration of at least one cycle longer than 3 months are 46.1%, while approximately 67.7% of the cycles observed showed exposure much higher than this threshold. As evidenced by the study, in fact, the average duration of corticosteroid therapy cycles is 6.8 months in treated patients. Finally, the share of treatment cycles with corticosteroids over the year reaches 25.5%. As noted by Valerio de Stefano, director of Uoc, Hematology Service and Day Hospital, Department for radiotherapy, oncology and hematology, Policlinico Gemelli, “c ‘it is a general consensus to limit the use of first-line steroids to a period not exceeding 2-6 weeks, considering other lines of therapy in case of non-response or in case of persistence of thrombocytopenia after cortisone discontinuation. Long-term cortisone treatment involves a significant risk of hypertension, diabetes, hyperlipidemia, gastropathy, osteoporosis, cataracts, and greater ease of infections. There are also other effects with a significant impact on quality of life: mood alterations, nervousness, insomnia, weight gain with typical features such as a ‘full moon’ face, acne, headache, increased sweating. However, the term should not be demonized, as it is fundamental in the first approach to ITP, but these considerations must be kept very well in mind in clinical practice “. Intervening on the therapeutic path would also positively affect the quality of life of people affected by Itp. In fact, as stated by 90% of the 1,507 patients interviewed in the iWISh survey, the possibility of using alternative treatments, based for example on the intake of drugs orally, would also positively affect the quality of life. This is a very important aspect, but unfortunately not sufficiently taken into consideration. The research, in fact, has shown a misalignment between the perception of patients and that of doctors, especially with regard to one of the most evident and heavy effects of the disease: ‘fatigue’. 50% of patients consider it one of the most burdensome elements of the disease, while 73% reported difficulty concentrating due to this symptom. From the point of view of clinicians, on the contrary, only 38% of their patients felt fatigued and, of these, they felt that 46% experienced a high level of fatigue. “In chronic ITP – commented Barbara Lovrencic, president Aipit (Italian Association of Thrombocytopenic Immune Purpura) – what most impacts the quality of life is the instability of the platelet level, because it involves constant anxiety about whether and when the platelet level will drop so much as to cause bleeding, the possibility of having to further treatments or hospitalization. Due to its unpredictability and chronic fatigue, a strongly impacting symptom as also shown by the iWISh study, ITP involves many limitations such as in sports activities and in the possibility of traveling (both for tourism and for work), but also in work and study. With Covid-19 we have all experienced firsthand what it means to live with the constant fear for one’s health and how frustrating it is not being able to organize and live freely because of something that is beyond our control. “A new therapeutic approach is therefore possible, indeed desirable, to improve the quality of life of patients suffering from this rare blood disorder.