Long-term treatment with deferiprone enhances left ventricular ejection function when compared to deferoxamine in patients with thalassemia major

Transfusion and iron chelation treatment have significantly reduced morbidity and improved survival of patients with thalassemia major. However, cardiac disease continues to be the most common cause of death. We report the left-ventricular ejection fraction, determined by echocardiography, in one hundred sixtyeight patients with thalassemia major followed for at least 5 years who received continuous monotherapy with deferoxamine (N = 108) or deferiprone (N = 60). The statistical analysis, using the generalized estimating equations model, indicated that the group treated with deferiprone had a significantly better left-ventricular ejection fraction than did those treated with deferoxamine (coefficient 0.97; 95% CI 0.37; 1.6, p = 0.002). The heart may be particularly sensitive to iron-induced mitochondrial damage because of the large number of mitochondria and its low level of antioxidants. Deferiprone, because of its lower molecular weight, might cross into heart mitochondria more efficiently, improving their activity and, thereby, myocardial cell function. Our findings indicate that the long-term administration of deferiprone significantly enhances left-ventricular function over time in comparison with deferoxamine treatment. However, because of limitations related to the design of this study, these findings should be confirmed in a prospective, randomized clinical trial

Serial echocardiographic left ventricular ejection fraction measurements: a tool for detecting thalassemia major patients at risk of cardiac death

Cardiac damage remains a major cause of mortality among patients with thalassemia major. The detection of a lower cardiac magnetic resonance T2* (CMR-T2*) signal has been suggested as a powerful predictor of the subsequent development of heart failure. However, the lack of worldwide availability of CMR-T2* facilities prevents its widespread use for follow-up evaluations of cardiac function in thalassemia major patients, warranting the need to assess the utility of other possible procedures.In this setting,the determination of left ventricular ejection fraction (LVEF)offers an accurate and reproducible method for heart function evaluation. These findings suggest a reduction in LVEF≥7%, over time, determined by 2-D echocardiography, may be considered a strong predictive tool for the detection of thalassemia major patients with increased risk of cardiac death. The reduction of LVEF≥7% had higher (84.76%) predictive value. Finally, Kaplan–Meier survival curves of thalassemia major patients with LVEF≥7% showed a statistically significant decreased probability of survival for heart disease (p=0.0022). However, because of limitations related to the study design, such findings should be confirmed in a large long-term prospective clinical trial

Italian patients with hemoglobinopathies exhibit a 5-fold increase in age-standardized lethality due to SARS-CoV-2 infection.

Since the beginning of the COVID-19 pandemic, concerns have been expressed worldwide for patients with hemoglobinopathies and their vulnerability to SARS-CoV-2 infection. Data from Lebanon confirmed a role of underlying comorbidities on COVID-19 severity, but no deaths among a cohort of thalassemia patients.1 Patients with sickle cell disease (SCD) displayed a broad range of severity after SARS-CoV-2 infection, spanning from a favorable outcome unless pre-existing comorbidities (UK cohort)2 to high case mortality in US.3 History of pain, heart, lung, and renal comorbidities was identified as risk factors of worse COVID-19 outcomes by the US SECURE-SCD Registry.4 While Italy experienced a death rate in the general population among the highest in the world, preliminary data from the first wave of the pandemic showed a lower than expected number of infected thalassemia patients (updated up to April 10, 2020), likely due to earlier and more vigilant self-isolation compared to the general population.

La rappresentazione del sé corporeo in condizioni di patologie croniche diversamente trattate

Il contributo presenta uno studio sulla rappresentazione del sé corporeo in bambini con patologie croniche diversamente trattate, al fine di esplorare possibili risorse funzionali alla gestione della condizione di rischio costituita dalla patologia; nello specifico, gli indicatori delle risorse vanno individuati, su un piano qualitativo, nell’adeguatezza dell’immagine del corpo, nell’integrità dell’immagine di sé, nell’integrazione di mappe cognitive e, su un piano quantitativo, nel quoziente di maturità cognitiva. Lo studio ha previsto il coinvolgimento di un gruppo di 52 bambini (età media 10 anni) di cui un 50% con patologie croniche che prevedono trattamenti invasivi, caratterizzati da "pratiche" sul corpo costanti che alterano la serenità del quotidiano (es. trasfusioni, cateteri venosi, iniezioni di insulina, microinfusioni), l’altro 50% con cardiopatie sottoposti a trattamenti solo farmacologici. L’indagine ha previsto l’uso del Disegno della Figura Umana. I risultati, pur mostrando, in generale, livelli medi di adeguatezza e di integrità corporea, sottolineano differenze statisticamente significative in relazione alla tipologia di trattamento, laddove i bambini sottoposti a trattamenti invasivi presentano livelli più elevati sia di adeguatezza che di integrità corporea. Relativamente al quoziente di maturità, invece, non si evidenziano differenze tra i due gruppi, sottolineando la presenza di alcune risorse cognitive funzionali all’adattamento alla patologia