Role of mTOR inhibition in the treatment of patients with renal angiomyolipomas

Abstract

Purpose To describe the role of mammalian target of rapamycin (mTOR) inhibition in the treatment of tuberous sclerosis complex (TSC) patients with renal angiomyolipoma in relation to available clinical data and clinical practice guidance for the nurse practitioner (NP). Data sources A review of the scientific literature, key clinical congresses, and key clinical trials. Conclusions TSC‐associated renal angiomyolipomas have a propensity to grow over time and predispose patients to serious and life‐threatening consequences. Surgery or invasive interventional therapies may not be the most optimal treatments because of the multiple, bilateral growth pattern of TSC‐associated renal angiomyolipomas. Targeted therapies, such as mTOR inhibitors, which have demonstrated efficacy in maintaining and reducing renal angiomyolipoma size, are of great benefit to patients. Implications for practice Treatment with everolimus, an oral mTOR inhibitor, offers patients a noninvasive pharmacotherapeutic treatment option. The NP, as a key member of the healthcare team overseeing TSC patients, must be knowledgeable about the safety and efficacy of mTOR inhibitors as their use in the patient population increases.

Renal Angiomyolipoma: The Good, the Bad, and the Ugly

“…Everolimus, another mTOR inhibitor and rapamycine derivative, has been studied the most. Currently, the Food and Drug Administration and European Medicines Agency approve this agent for treating AML in the setting of TSC [323536]. The position of mTOR inhibitors in the management of sporadic AML still remains to be determined [2].…”

“…Another concern for treating physicians is how long the treatment with those drugs should continue, as the effect of mTOR inhibition is reversible. To continue the treatment for AML, consideration should be given to long-term complications, cost, and safety, compared with other treatment options [3236]. …”

Transplant immunosuppressive drugs in urology

“…AML are benign tumors comprised of blood vessels, smooth muscle, and adipose tissue which can occur sporadically (70-80% of AML) or as part of the TSC. TSC is a rare disorder characterized by growths of benign tissue (hamartomas) of which AML is the most common (29). Though benign, AML have the potential to develop aneurysms and subsequent hemorrhage, particularly when they reach over 4 cm (30).…”

İyi Huylu Böbrek Tümörlerinde Medikal Tedavi

“…Coombs tarafından 2013 yılında yayınlanan derlemede mTOR inhibitörlerinin (sirolimus ve everolimus) renal AML tedavisindeki rolü incelenmiştir. Bu derlemede TSK ilişkili AML'de lezyonların bilateral ve çok sayıda olması nedeni ile cerrahi ve invaziv girişimsel işlemlerin uygun olmadığı ve mTOR inhibitörlerinin AML boyutunu azaltarak hastalara büyük fayda sağladığı sonucuna varılmıştır (29).…”