Category:Endocrine therapy

Endocrine therapy is the use of medicines that act on the body's endocrine system, either by replacing a deficient hormone, by mimicking or antagonising a natural hormone, by blocking hormone synthesis at the gland, or by modulating the hormone's action at its target receptor. The category overlaps substantially with hormones (the umbrella that catalogues the medicines themselves) and with each of the specific endocrine subspecialties (diabetes medicines, thyroid hormones and antithyroid agents, sex hormone therapy, corticosteroid therapy, calcium and bone metabolism). The category exists as a single page because the underlying pharmacological strategy across these domains is the same: an endogenous signalling system has been disturbed, and a medicine is added that restores or redirects the signal.

The endocrine-therapy category includes, by clinical practice, four strategies. The first is hormone replacement, in which a missing endogenous hormone is supplied as an identical or analogous medicine: levothyroxine for hypothyroidism, insulin for type 1 diabetes and selected type 2, estradiol (with a progestin in women with an intact uterus) for menopausal symptom management, testosterone for hypogonadism, growth hormone for childhood and selected adult deficiency, glucocorticoid and mineralocorticoid replacement for adrenal insufficiency, desmopressin for central diabetes insipidus.

The second strategy is pharmacological dose of a hormone or analogue beyond the physiologic range to produce a therapeutic effect not available at replacement doses: high-dose systemic glucocorticoid for inflammation; intravenous insulin for diabetic ketoacidosis and hyperkalaemia; high-dose intravenous methylprednisolone for spinal-cord oedema and multiple-sclerosis relapse; supraphysiologic vasopressin and analogues for hepatorenal syndrome and septic shock; GLP-1 receptor agonists at higher-than-physiologic activity for diabetes control and obesity.

The third strategy is hormone antagonism, in which a synthetic medicine blocks the natural hormone's action at its receptor or earlier in the signalling pathway: the antiandrogens (bicalutamide, enzalutamide, abiraterone, cyproterone, spironolactone in this indication) in prostate cancer and in polycystic ovary syndrome; the gonadotropin-releasing hormone agonists and antagonists in prostate cancer and endometriosis; the selective estrogen-receptor modulators (tamoxifen, raloxifene) and the aromatase inhibitors (anastrozole, letrozole, exemestane) in hormone-receptor-positive breast cancer; the antithyroid agents methimazole and propylthiouracil for hyperthyroidism; the corticosteroid synthesis inhibitors (ketoconazole at supraphysiologic dose, metyrapone, osilodrostat, mitotane) for Cushing's syndrome; the progesterone-receptor antagonist mifepristone in early pregnancy termination and in Cushing's syndrome; the orexin-receptor antagonists in insomnia (acting on a hormone of arousal).

The fourth strategy is hormone-pathway modulation more broadly: incretin-pathway medicines (DPP-4 inhibitors preserving endogenous GLP-1 and GIP; GLP-1 receptor agonists adding exogenous receptor activation; SGLT2 inhibitors modulating renal glucose handling and indirectly the insulin-glucagon-incretin axis); the calcium-sensing receptor allosteric activator cinacalcet for secondary hyperparathyroidism; the soluble guanylate cyclase pathway through PDE5 inhibition.

The clinical practice of endocrine therapy has been transformed in the last twenty years by the recognition that many endocrine medicines have effects on diseases outside their original endocrine indication. The SGLT2 inhibitors reduce cardiovascular mortality in heart failure with both reduced and preserved ejection fraction; the GLP-1 receptor agonists reduce cardiovascular events in atherosclerotic disease and produce substantial weight loss in obesity (the semaglutide-tirzepatide era of metabolic medicine); the bisphosphonates reduce fracture risk in osteoporosis through their effect on bone resorption (an endocrine-pathway target through osteoclast pharmacology). The boundaries between "endocrine therapy", "cardiovascular medicine", and "metabolic medicine" are now substantially blurred.

• Antihyperglycemic agents (diabetes): the insulins, biguanides, sulfonylureas (also indexed under insulin secretagogues), DPP-4 inhibitors, SGLT2 inhibitors, thiazolidinediones, GLP-1 receptor agonists and the dual GIP/GLP-1 agonists
• Thyroid hormones and antithyroid agents: levothyroxine (T4), liothyronine (T3), thyroid extract; methimazole, propylthiouracil; radioactive iodine (radiopharmaceutical)
• Sex hormones: estrogens, progestins, androgens; the antiandrogens, the SERMs, the aromatase inhibitors, the GnRH agonists and antagonists, the progesterone-receptor antagonists
• Corticosteroids: the systemic glucocorticoids, the inhaled, topical, ophthalmic, intra-articular preparations; mineralocorticoid replacement (fludrocortisone)
• Bone and calcium: bisphosphonates (alendronate, risedronate, ibandronate, zoledronate); the RANKL antibody denosumab; teriparatide and abaloparatide (PTH analogues); calcitonin; the vitamin D analogues including calcitriol and paricalcitol; calcimimetic cinacalcet; sclerostin antibody romosozumab
• Pituitary and hypothalamic axis:
  • Growth hormone (somatropin) and growth-hormone-releasing-hormone analogues; pegvisomant (growth-hormone receptor antagonist for acromegaly)
  • GnRH agonists (leuprolide, goserelin, triptorelin, histrelin) and antagonists (degarelix, elagolix, relugolix)
  • Vasopressin and desmopressin for diabetes insipidus
  • Somatostatin analogues (octreotide, lanreotide, pasireotide) for acromegaly and carcinoid
  • Dopamine agonists (bromocriptine, cabergoline) for prolactinoma; quinagolide outside the United States
  • ACTH (cosyntropin for testing, repository corticotropin for selected immune indications)
• Carcinoid and neuroendocrine tumour:
  • Somatostatin analogues
  • Telotristat (tryptophan hydroxylase inhibitor for carcinoid diarrhoea)
• Cushing's syndrome:
  • Ketoconazole at supraphysiologic doses, metyrapone, osilodrostat, levoketoconazole (selective steroidogenesis inhibitors)
  • Mitotane (adrenolytic for adrenocortical carcinoma)
  • Mifepristone (glucocorticoid-receptor antagonist for hyperglycaemia of Cushing's)
• Metabolic / obesity:
  • GLP-1 receptor agonists (semaglutide, liraglutide) and tirzepatide for chronic weight management
  • Phentermine, phentermine-topiramate, bupropion-naltrexone, orlistat
• Diabetes insipidus and SIADH:
  • Desmopressin (DI), tolvaptan and conivaptan (SIADH and polycystic kidney disease)

The boundary of this category is "medicine acting principally on an endocrine pathway." The catecholamines (epinephrine, norepinephrine, dopamine) are functionally hormones in the strict sense but their clinical use is dominated by their cardiovascular and neurological actions rather than endocrine; they are collected under sympathomimetics and vasopressors. The antidiuretic-hormone-related medicines are listed here for diabetes insipidus and SIADH indications and under diuretics for the vaptan free-water-diuretic actions. The bone and calcium subcategory is sometimes collected as a separate "metabolic bone disease" pharmacopoeia but is included here on the basis of its endocrine targets (PTH, calcitonin, vitamin D as hormone). The medicines for diabetes and metabolic-syndrome management are full members of this category despite their broader cardiovascular effect, because the underlying pathways are endocrine.

This category page is an encyclopedia article about its subject. The actual index of medicines belonging to the category is generated automatically by the wiki engine, from category-membership declarations on the individual medicine pages, and appears at the foot of the page below the references.