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Hereditary transthyretin amyloidosis (ATTRv) is a multisystemic, rare, inherited, progressive and adult-onset disease, affecting the sensorimotor nerves, heart, autonomic function and other organs. The actual scenario of pharmaceutical approaches for ATTRv amyloidosis includes five main groups: TTR stabilizers, TTR mRNA silencers, TTR fibril disruptors, inhibitor of TTR fibril seeding and gene therapy. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, able to penetrate into hepatocytes, where it selectively targets TTR mRNA, reducing TTR production. We report and discuss 9 cases of different patients with ATTRv amyloidosis successfully managed with patisiran in the real clinical practice. Literature data, as well as the above presented case reports, show that this drug is effective and safe in improving both neurological and cardiovascular symptoms of ATTRv amyloidosis, and to maintain a good QoL, independently form the stage of the disease and the involved mutation. Recent studies correlated improved functional and biochemical outcomes with a regression of amyloid burden, especially at the cardiac level. Today, patisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis and polyneuropathy and cardiovascular symptoms.

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Review Article Antineoplastic treatments can induce cardiovascular damage that may appear early or, sometimes, many years after exposure. [1] The majority of studies on CTX focus on patients treated with anthracyclines and trastuzumab. However, cardiotoxic effect has been described even for other classes of treatments such as tyrosine kinases inhibitors, antimetabolites, alkylating agents, taxanes, and radiotherapy. [1,7] The most common adverse event is a reduction in left ventricular (LV) dysfunction that may progress to overt heart failure (HF); nevertheless, clinical manifestations of CTX are broad and can include arrhythmias, ischemia, valvular heart disease, pericardial disease, arterial and pulmonary hypertension, and thrombosis [Figure 1]. Left ventricular dysfunction and heart failure LV dysfunction and HF are common and serious side effects of cancer treatment. [1] A recent report from the American Society Cardiovascular diseases are possible complications of antineoplastic treatment and may lead to premature morbidity and mortality among cancer survivors. A symptom-based follow-up is ineffective, and there are growing evidences that early detection of myocardial damage in patients treated with antineoplastic drugs is the key point to prevent the occurrence of damage and improve the prognosis of these patients. Different techniques have been proposed to monitor cardiac function in oncologic patients such as cardiac imaging (echocardiography, nuclear imaging, and cardiac magnetic resonance) and biomarkers (troponin and natriuretic peptides). The European Association of Cardiovascular Imaging/American Society of Echocardiography consensus document encourages an integrated approach to early detect cardiotoxicity.