Treatment Overview

Living with Acromegaly means navigating physical changes that often manifest slowly over years. The condition results from the pituitary gland producing too much Growth Hormone (GH), typically due to a non-cancerous tumor. Patients often experience enlarged hands and feet, changes in facial features, and uncomfortable symptoms like joint pain, excessive sweating, and fatigue. The emotional impact of these visible and internal changes can be significant, making daily life challenging.

Treatment is critically important to normalize hormone levels and mitigate severe long-term health risks, including heart disease, high blood pressure, and diabetes. The goal is to shut down the excessive production of Growth Hormone and its downstream messenger, Insulin-like Growth Factor-1 (IGF-1). Because Acromegaly is caused by an underlying tumor, treatment requires a specialized, multi-step approach tailored to the tumor size and how the patient responds to therapy (National Institute of Diabetes and Digestive and Kidney Diseases, 2022).

Overview of treatment options for Acromegaly

The overall treatment strategy for Acromegaly prioritizes the rapid control of hormone levels. While surgical removal of the pituitary tumor (transsphenoidal surgery) is generally considered the first-line treatment for cure, medications play an essential and often lifelong role.

Medications are typically used when surgery fails to normalize GH and IGF-1 levels, when the tumor cannot be completely removed, or sometimes as a primary treatment to shrink a large tumor before surgery. The pharmacological approach involves using drugs that either stop the pituitary tumor from releasing GH or block the hormone’s effects on the body. This dual approach ensures both the source of the problem and its downstream effects are managed effectively.

Medications used for Acromegaly

Three main classes of medications are used to treat Acromegaly:

  1. Somatostatin Receptor Ligands (SRLs): These are often the first-line medical therapy used after surgery. Examples include octreotide and lanreotide. They are usually given as long-acting injections once or twice a month. These medications aim to suppress the tumor’s ability to produce Growth Hormone.
  2. Dopamine Agonists: These are the least potent class and are primarily used for small tumors or mild elevations in GH. Cabergoline and bromocriptine are common examples, usually taken orally.
  3. Growth Hormone Receptor Antagonists: Pegvisomant is the key drug in this class. It does not stop the pituitary from making GH, but instead blocks the action of GH at the cellular level. This is highly effective at normalizing IGF-1 levels when other treatments fail.

Studies show that SRLs effectively control hormone levels in a majority of patients, leading to shrinkage of the tumor in many cases and noticeable symptom relief, such as decreased sweating and headaches (Mayo Clinic, 2023).

How these medications work

The major drug classes work by interrupting the hormonal signaling process at different points.

Somatostatin Receptor Ligands (SRLs) mimic somatostatin, a naturally occurring brain hormone that inhibits GH secretion. When injected, these medications bind to receptors on the pituitary tumor cells, effectively telling the tumor to stop releasing Growth Hormone. This lowers the GH levels circulating in the blood.

Growth Hormone Receptor Antagonists (like pegvisomant) work further down the chain. They physically block the GH receptors found primarily on liver cells. Because GH cannot bind to these receptors, the liver cannot produce IGF-1. This normalization of IGF-1 is the key to stopping the unwanted growth and metabolic effects of Acromegaly.

Dopamine Agonists work by attaching to dopamine receptors on the tumor, which, paradoxically, can inhibit GH release in some pituitary tumors.

Side effects and safety considerations

Acromegaly medication is specialized and requires close monitoring. Somatostatin Receptor Ligands (SRLs) commonly cause GI issues (diarrhea, nausea, abdominal pain, and potential gallstones). Growth Hormone Receptor Antagonists necessitate monitoring liver enzymes for elevation.

Dopamine Agonists can cause headaches, dizziness, and mild nausea. Patients on long-term medical therapy must have regular blood tests (GH, IGF-1, liver enzymes) to ensure safety, effectiveness, and allow for timely hormone adjustments.

Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care.

References

  1. Mayo Clinic. https://www.mayoclinic.org
  2. National Institute of Diabetes and Digestive and Kidney Diseases. https://www.niddk.nih.gov
  3. National Organization for Rare Disorders. https://rarediseases.org
  4. National Institutes of Health. https://www.nih.gov

Medications for Acromegaly

These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Acromegaly.

Found 7 Approved Drugs for Acromegaly

Octreotide Acetate

Brand Names
Sandostatin, Octreotide, Bynfezia

Octreotide Acetate

Brand Names
Sandostatin, Octreotide, Bynfezia
Form: Injection, Kit
Method of administration: Subcutaneous, Intravenous, Intramuscular
FDA approval date: October 21, 1988
Classification: Somatostatin Analog
BYNFEZIA PEN is a s o matostatin analogue indicated: Acromegaly : To reduce blood levels of growth hormone (GH) and insulin growth factor 1 (IGF-1; somatomedin C) in acromegaly patients who have had inadequate response to or cannot be treated with surgical resection, pituitary irradiation, and bromocriptine mesylate at maximally tolerated doses. (1.1) Carcinoid Tumors : For the symptomatic treatment of patients with m eta static c a rcinoid t u mo rs where it suppresses or inhibits the severe diarrhea and flushing episodes associated with the disease. (1.2) Vasoactive Intestinal Peptide Tumors (VIPomas) : For the treatment of profuse watery diarrhea associated with VIP-secreting tumors. (1.3) Limitations of Use Improvement in clinical signs and symptoms, or reduction in tumor size or rate of growth, were not shown in clinical trials performed with octreotide injection; these trials were not optimally designed to detect such effects.

Mycapssa

Generic Name
Octreotide

Mycapssa

Generic Name
Octreotide
Form: Capsule
Method of administration: Oral
FDA approval date: July 06, 2020
Classification: Somatostatin Analog
MYCAPSSA is indicated for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide. MYCAPSSA is a somatostatin analog indicated for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide (‎ 1 ).

Somavert

Generic Name
Pegvisomant

Somavert

Generic Name
Pegvisomant
Form: Kit
FDA approval date: October 01, 2016
SOMAVERT is indicated for the treatment of acromegaly in patients who have had an inadequate response to surgery or radiation therapy, or for whom these therapies are not appropriate. The goal of treatment is to normalize serum insulin-like growth factor-I (IGF-I) levels. SOMAVERT is a growth hormone receptor antagonist indicated for the treatment of acromegaly in patients who have had an inadequate response to surgery or radiation therapy, or for whom these therapies are not appropriate. The goal of treatment is to normalize serum insulin-like growth factor-I (IGF-I) levels. ( 1 )

Cycloset

Generic Name
Bromocriptine

Cycloset

Generic Name
Bromocriptine
Form: Tablet, Capsule
Method of administration: Oral
FDA approval date: January 13, 1998
Classification: Ergot Derivative
Hyperprolactinemia-Associated Dysfunctions Bromocriptine mesylate is indicated for the treatment of dysfunctions associated with hyperprolactinemia including amenorrhea with or without galactorrhea, infertility or hypogonadism. Bromocriptine mesylate treatment is indicated in patients with prolactin-secreting adenomas, which may be the basic underlying endocrinopathy contributing to the above clinical presentations. Reduction in tumor size has been demonstrated in both male and female patients with macroadenomas. In cases where adenectomy is elected, a course of bromocriptine mesylate therapy may be used to reduce the tumor mass prior to surgery. Acromegaly Bromocriptine mesylate therapy is indicated in the treatment of acromegaly. Bromocriptine mesylate therapy, alone or as adjunctive therapy with pituitary irradiation or surgery, reduces serum growth hormone by 50% or more in approximately ½ of patients treated, although not usually to normal levels. Since the effects of external pituitary radiation may not become maximal for several years, adjunctive therapy with bromocriptine mesylate offers potential benefit before the effects of irradiation are manifested. Parkinson’s Disease Bromocriptine mesylate tablets are indicated in the treatment of the signs and symptoms of idiopathic or postencephalitic Parkinson’s disease. As adjunctive treatment to levodopa (alone or with a peripheral decarboxylase inhibitor), bromocriptine mesylate therapy may provide additional therapeutic benefits in those patients who are currently maintained on optimal dosages of levodopa, those who are beginning to deteriorate (develop tolerance) to levodopa therapy, and those who are experiencing “end of dose failure” on levodopa therapy. Bromocriptine mesylate therapy may permit a reduction of the maintenance dose of levodopa and, thus may ameliorate the occurrence and/or severity of adverse reactions associated with long-term levodopa therapy such as abnormal involuntary movements (e.g., dyskinesias) and the marked swings in motor function (“on-off” phenomenon). Continued efficacy of bromocriptine mesylate therapy during treatment of more than 2 years has not been established. Data are insufficient to evaluate potential benefit from treating newly diagnosed Parkinson’s disease with bromocriptine mesylate. Studies have shown, however, significantly more adverse reactions (notably nausea, hallucinations, confusion and hypotension) in bromocriptine mesylate-treated patients than in levodopa/carbidopa-treated patients. Patients unresponsive to levodopa are poor candidates for bromocriptine mesylate therapy.

Signifor

Generic Name
Pasireotide

Signifor

Generic Name
Pasireotide
Form: Injection, Kit
Method of administration: Subcutaneous
FDA approval date: December 14, 2012
Classification: Somatostatin Analog
SIGNIFOR LAR is a somatostatin analog indicated for the treatment of: Patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option.
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