Renée Maas

171 Phospholamban R14del Cardiomyopathy: a systematic summary of the pathophysiological mechanisms 7 response. These common pathways and biomarkers could have a clinical potential and prognostic associations merit further investigation to develop optimal management and therapeutic strategies for PLN0R14del patients. Moreover, given the potential role of inflammation in disease progression, it would be important to explore therapeutic interventions that target the immune system in PLN-R14del-associated cardiomyopathy. This could involve testing anti-inflammatory drugs or other immune-modulating therapies in animal models or clinical trials. Current treatments for PLNR14del patients The current optimal medical treatment in patients carrying the PLNR14del mutation follows the guidelines for heart failure and arrhythmias for the prevention of sudden cardiac death (SCD).202,203 The pharmaceutical therapeutic agents that are used include β-blockers and angiotensin-converting enzyme (ACE) inhibitors.204 Due to a lack of specificity, these medical treatments only slow down the progression of the disease at best and possibly alleviate the symptoms to some degree. In most cases of disease progression, additional interventions have to be implemented,205,206 such as implantable cardioverter-defibrillators (ICD), a left ventricular assist device (LVAD)207 or a heart transplant for patients with end-stage PLN-R14del disease. In the Netherlands, about 25% of the heart transplants performed yearly are PLN-R14del related.208 However, heart transplants are limited by donor shortage, surgical complications, and long-term effects of immunosuppressive therapy.209 Therefore, a better understanding of the molecular mechanisms could result in a tailored PLN-R14del-specific therapy. Since the discovery of PLN-R14del cardiomyopathy, many studies focusing on this disease have been performed. Here, we summarize the potential pharmacological agents available which could be more promising in treating PLNR14del compared to the current strategies (Table 2).

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