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.
Keywords: Treatment guidelines; pharmacotherapy; renal; safety; treatment.
©2013 The Author(s) ©2013 American Association of Nurse Practitioners.