During the event ‘Trombocitopenia immune primaria: 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 with primary immune thrombocytopenia in Italian contexts’ was presented, conducted by CliCon Srl. The round table also offered the opportunity to illustrate the results of the international studio ‘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 requires 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.
“On the basis of the analysis conducted in the Italian context – explained Luca Degli Esposti, president of CliCon Health, Economics and Outcomes Research – the prevalence of primary immune thrombocytopenia was equal to 262 cases / million patients. About two thirds of patients are results in treatment with corticosteroid drugs and, among these, most had an exposure of more than 3 months of therapy. This experience confirmed the possibility of refining the estimates on epidemiology and methods of treatment of patients with rare diseases using the data health and, consequently, to be able to have a further element of knowledge for the management of the disease and the evaluation of drug therapies “.
The observational analysis 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 far above 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 courses of treatment 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 hematological imaging, Policlinico Gemelli, “there is a general agreement to limit the use of frontline steroids to no longer than 2-6 weeks, considering other lines of therapy in case of non-response or in case of persistence of thrombocytopenia after cortisone discontinuation. Continuing long-term cortisone treatment involves a significant risk of hypertension, diabetes, hyperlipidemia, gastropathy, osteoporosis, cataracts, and greater ease of infection. There are also other effects with a notable impact on quality of life: mood alterations, nervousness, insomnia, weight gain with typical features such as a ‘full moon’ face, acne, headache, increased sweating. In any case, the use of steroids in the short 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 have a positive impact on 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. 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 clinician’s point of view, by contrast, 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 of Aipit (Italian association of immune thrombocytopenic 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 level of platelets will drop enough to cause bleeding, the possibility of having to resort to further treatments or hospitalization. Due to its unpredictability and chronic fatigue, a strongly impacting symptom as also shown by the iWISh study, the Itp involves many limitations. example in sporting activity 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 constant fear for one’s own health and how frustrating it is not to be able to organize and live freely because of something that is beyond our control. “
A new therapeutic approach therefore it is possible, indeed desirable, to improve the quality of life of patients suffering from this rare blood disorder.