Plain language summary of the MPOWERED trial comparing the effectiveness, safety, and patient experiences' of monthly injections and oral medication used to treat acromegaly

What is this summary about? Oral octreotide is the first oral medication (taken by mouth, twice a day) in the class of drugs called somatostatin receptor ligands that is available in the USA for maintaining control of acromegaly. It was approved by the US Food and Drug Administration (FDA) for people who have had their disease respond to, while also tolerating, longacting monthly injectable versions of the somatostatin receptor ligands octreotide or lanreotide (referred to as †injectables'), that can also be referred to as somatostatin analogs. Although people with acromegaly often are able to control hormone levels with injectables, some could still experience inadequate symptom control or injection site reactions (such as pain, redness or bumps in and around the area of injection) related to this type of medicine. This summary reports the results from a phase 3 study, called MPOWERED comparing the injectables to oral octreotide. The full title of this study is called The Maintenance of Acromegaly Patient with Octreotide Capsules Compared With injections - Evaluation of Response Durability). This summary also includes insights and perspectives from an acromegaly patient advocate and from a nurse practitioner who closely follows people with acromegaly. What was the aim of the study? The main aim of the study was to look at how well oral octreotide worked and how safe it was, compared with injectables, in people with acromegaly. All study patients had control of growth hormone secretion on injectables prior to being switched to oral octreotide. Researchers also asked participants in the study questions about the impact of the treatment on their health and quality of life (known as patient-reported outcome measures). What were the results? The main finding of this study was that oral octreotide is as effective as injectables in maintaining normal growth hormone markers in the blood in patients who responded to both therapies. The side effects overall were similar between groups. In general, participants in the study treated with oral octreotide had mild to moderate side effects that typically went away as treatment was continued. The most common side effects in the oral octreotide group were flatulence (gas) and nausea. The most common side effects in the injectables group were flatulence and diarrhea.