McCune-Albright Syndrome Medication

  • Author: Gabriel I Uwaifo, MBBS; Chief Editor: George T Griffing, MD   more...
 
Updated: Nov 16, 2011
 

Medication Summary

The goals of pharmacotherapy are to prevent complications and to reduce morbidity.

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Aromatase inhibitors

Class Summary

Testolactone typically is administered at a low dose of approximately 10 mg/kg/d and is increased gradually over a period of 3-4 weeks to an ultimate dose of 40 mg/kg/d. With adequate treatment response, serum estrone and estradiol levels are reduced. Patients who respond to treatment should continue therapy until the age of normal puberty or until bone age of 15-16 years, when epiphysial fusion plate has occurred. Among the potential adverse effects associated with medication use are transient abdominal cramping, diarrhea, and mild hepatic inflammation evidenced by elevated SGOT, SGPT, and GGT.

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Testolactone (Teslac)

 

Synthetic antineoplastic agent that is structurally distinct from the androgen steroid nucleus in possessing a 6-membered lactone ring in place of the usual 5-membered carbocyclic D-ring.

Although the precise mechanism by which testolactone produces a clinical antineoplastic effect has not been established, its principal action is reported to be the inhibition of steroid aromatase activity and the consequent reduction in estrone synthesis from adrenal androstenedione, the major source of estrogen in postmenopausal women.

Based on in vitro studies, the aromatase inhibition may be noncompetitive and irreversible. This phenomenon may account for the persistence of testolactone's effect on estrogen synthesis after drug withdrawal. Despite some similarity to testosterone, testolactone has no in vivo androgenic effect. No other hormonal effects have been reported in clinical studies in patients receiving testolactone. In one study, testolactone administered orally (1000 mg/d) was reported to increase the renal tubular reabsorption of calcium but had no effect on serum calcium concentration. The mechanism of hypocalciuric effect is unknown. No clinical effects of testolactone on adrenal function have been reported in humans; however, one study noted an increase in the urinary excretion of 17-ketosteroids in most patients treated with 150 mg/d PO.

Typically, testolactone is administered at a low dose of approximately 10 mg/kg/d and is increased gradually over a period of 3-4 wk to an ultimate dose of 40 mg/kg/d. With an adequate treatment response, serum estrone and estradiol levels are reduced. Patients who respond to treatment should continue therapy until the age of normal puberty or until bone age of 15-16 y, when epiphysial fusion plate has occurred.

Testolactone is well absorbed from the GI tract. It is metabolized to several derivatives in the liver, all of which preserve the lactone D-ring. These metabolites, as well as some unmetabolized drug, are excreted in the urine. No clear manifestation of androgenic, estrogenic or antiestrogenic, progestational or antiprogestational, and gonadotropinlike or antigonadotropic effects have been reported. Testolactone does not demonstrate anti-inflammatory, mineralocorticoidlike, or glucocorticoidlike properties. Available for PO administration as tabs, providing 50 mg testolactone per tab.

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Contributor Information and Disclosures
Author

Gabriel I Uwaifo, MBBS  Clinical and Research Attending, Assistant Professor of Medicine and Endocrinology, MedStar Clinical Research Center, MedStar Research Institute and Washington Hospital Center

Gabriel I Uwaifo, MBBS is a member of the following medical societies: American Association of Clinical Endocrinologists, American College of Physicians-American Society of Internal Medicine, American Diabetes Association, American Medical Association, American Society of Hypertension, and Endocrine Society

Disclosure: Nothing to disclose.

Coauthor(s)

Nicholas J Sarlis, MBBS, MD, PhD, FACP,  Vice President, Medical Affairs, Incyte Corporation

Nicholas J Sarlis, MBBS, MD, PhD, FACP, is a member of the following medical societies: American Association for Cancer Research, American Association for the Advancement of Science, American Association of Clinical Endocrinologists, American College of Endocrinology, American College of Physicians, American Federation for Medical Research, American Head and Neck Society, American Medical Association, American Society for Therapeutic Radiology and Oncology, American Society of Clinical Oncology, American Thyroid Association, Association for Psychological Science, Endocrine Society, European Society for Medical Oncology, New York Academy of Sciences, and Royal Society of Medicine

Disclosure: Incyte Corporation Salary Employment; Sanofi-Aventis Ownership interest Stock option/ restricted stock holder; Incyte Corporation Ownership interest Stock option/ restricted stock holder

Specialty Editor Board

Ghassem Pourmotabbed, MD†  Former Associate Professor, Department of Internal Medicine, Division of Endocrinology and Metabolism, University of Tennessee School of Medicine and Health Science Center

Ghassem Pourmotabbed, MD† is a member of the following medical societies: American Diabetes Association, American Federation for Medical Research, and Endocrine Society

Disclosure: Nothing to disclose.

Francisco Talavera, PharmD, PhD  Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Medscape Salary Employment

Arthur B Chausmer, MD, PhD, FACP, FACE, FACN, CNS  Professor of Medicine (Endocrinology, Adj), Johns Hopkins School of Medicine; Affiliate Research Professor, Bioinformatics and Computational Biology Program, School of Computational Sciences, George Mason University; Principal, C/A Informatics, LLC

Arthur B Chausmer, MD, PhD, FACP, FACE, FACN, CNS is a member of the following medical societies: American Association of Clinical Endocrinologists, American College of Endocrinology, American College of Nutrition, American College of Physicians, American College of Physicians-American Society of Internal Medicine, American Medical Informatics Association, American Society for Bone and Mineral Research, Endocrine Society, and International Society for Clinical Densitometry

Disclosure: Nothing to disclose.

Mark Cooper, MBBS, PhD, FRACP  Head, Diabetes & Metabolism Division, Baker Heart Research Institute, Professor of Medicine, Monash University

Disclosure: Nothing to disclose.

Chief Editor

George T Griffing, MD  Professor of Medicine, St Louis University School of Medicine

George T Griffing, MD is a member of the following medical societies: American Association for the Advancement of Science, American College of Medical Practice Executives, American College of Physician Executives, American College of Physicians, American Diabetes Association, American Federation for Medical Research, American Heart Association, Central Society for Clinical Research, Endocrine Society, International Society for Clinical Densitometry, and Southern Society for Clinical Investigation

Disclosure: Nothing to disclose.

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Base of the skull computed tomography scan showing extensive fibrous dysplasia. Note the asymmetrical affectation, with near-total obliteration of various neural foramina at the base of the skull. This degree of fibrous dysplasia can result in multiple cranial nerve compression neuropathies, of which blindness and deafness (from involvement of cranial nerves II and VIII) are among the most disabling.
Café au lait spot. This is a fairly large, irregular-edged ("coast-of-Maine" variety) lesion. It presents as a brownish, otherwise-asymptomatic macule/patch. The degree of pigmentation is fairly uniform.
Fibrous dysplasia of a long bone characterized by focal bony expansion, patchy areas of sclerosis, and bony cyst formation.
This plain skull radiograph shows marked macrocrania, frontal bossing, and a markedly thickened bony table in patchy areas, particularly at the base of the skull and occiput. The skull also shows the hair-on-end appearance suggestive of Paget disease or poorly controlled hemoglobinopathy (eg, beta thalassemia, sickle cell disease).
 
 
 
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