Oral Octreotide as a Treatment for Acromegaly

According to Healio, an open-label study of oral octreotide has reported that the treatment is able to maintain its response in acromegaly patients without additional safety signals. This data is very exciting, as it means that oral octreotide can control insulin-like growth factor I (IGF-I) levels for an extended period. Therefore, this therapy can be added to medical professionals’ arsenal of treatment options for acromegaly.

About the Study

The open-label study was a continuation of the CHIASMA OPTIMAL trial, a double-blind and placebo-controlled study of oral octreotide as a treatment for acromegaly. To learn more about the therapy, researchers enrolled 20 patients from the placebo group and 20 from the oral octreotide group and administered 60 mg doses of the therapy every day. Based on the specific patient, this number may have been lowered to 40 mg or raised to 80 mg. By the end of the trial, three patients were taking the low dose, 10 the original dose, and 27 the highest dose.

In terms of endpoints, researchers were looking for the number of patients who went on to complete all 48 weeks of follow-up and the proportion of participants who finished the trial as responders. At the end of the study results were:

  • 90% of the original oral octreotide group completed the open-label extension
    • 70% of the placebo group finished the trial
  • 92.6% maintained a response throughout the 48 weeks
  • Most common adverse events dealt with the gastrointestinal system
    • Nearly all were mild to moderate in severity
  • No new safety concerns


The FDA has approved patients taking 20mg or 40mg doses of oral octreotide twice a day, but this study saw participants started at 60 mg. In response to this information, researchers are examining the optimal starting dose.

Looking at the data from the trial, researchers noticed that adverse events happened less frequently when patients are started on a higher dose of oral octreotide. In the original trial, 96.4% of the octreotide group saw adverse events, as did 57.9% of the placebo group. All of these patients were started on 40 mg. In the follow-up, all patients started with 60 mg, which led to adverse events in 53.6% in the first group and 31.6% in the second.

More research needs to be done into dosage, but medical professionals are very excited about what they’ve learned so far in regards to a higher starting dosage.

About Acromegaly

Noncancerous tumors called adenomas sit on the pituitary gland in affected individuals with acromegaly, causing it to release an excess of growth hormone (GH). This excess leads to abnormal and significant growth. Symptoms typically appear slowly, with abnormal growth beginning in the appendages and making them disproportionate to the rest of the body. Further symptoms include:

  • Protruding jaw and brow bone
  • Thick, oily skin
  • Excessive sweating
  • Enlarged organs
  • Enlarged nasal bone
  • Enlarged facial features

Surgery is typically the first treatment option, as it is the quickest and most effective option. It may not work for all patients, so other options include dopamine agonists, somatostatin analogs (SSAs), and GH receptor antagonists (GHRAs). In the end, your doctor should individualize your treatment plan to your specific symptoms and needs.

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