Methyltestosterone is used mainly for replacement or substitution of diminished or absent endogenous testicular hormone.
Uses in Males
In males, methyltestosterone is used for the management of congenital or acquired primary hypogonadism such as that resulting from orchiectomy or from testicular failure caused by cryptorchidism, bilateral torsion, orchitis, or vanishing testis syndrome. Methyltestosterone also is used in males for the management of congenital or acquired hypogonadotropic hypogonadism such as that resulting from idiopathic gonadotropin or gonadotropin releasing hormone (luteinizing hormone releasing hormone) deficiency or from pituitary-hypothalamic injury caused by tumors, trauma, or radiation. If any of these conditions occur before puberty, androgen replacement therapy will be necessary during adolescence for the development of secondary sexual characteristics and prolonged therapy will be required to maintain these characteristics. Prolonged androgen therapy also is required to maintain sexual characteristics in other males who develop testosterone deficiency after puberty.
When the diagnosis is well established, methyltestosterone may be used to stimulate puberty in carefully selected males with delayed puberty. These males usually have a family history of delayed puberty that is not caused by a pathologic disorder. Brief treatment with conservative doses of an androgen may occasionally be justified in these males if they do not respond to psychologic support. Because androgens may adversely affect bone maturation in these prepubertal males, this potential risk should be fully discussed with the patient and his parents prior to initiation of androgen therapy.
(See Cautions: Pediatric Precautions.)If androgen therapy is initiated in these prepubertal males, radiographs of the hand and wrist should be obtained at 6-month intervals to determine the effect of therapy on the epiphyseal centers.
The safety and efficacy of methyltestosterone in patients with low testosterone concentrations related to aging (i.e., late-onset hypogonadism) have not been established. Although endogenous testosterone concentrations decline with aging and manifestations of hypogonadism such as decreased libido, impotence, decreased body hair growth, decreased muscle mass, increased risk of cardiovascular disease, and decreased bone mass and resultant osteoporosis may occur, it is unclear whether these symptoms are related to such decreased concentrations or to normal aging; therefore, the need to replace testosterone in aging men is unclear.
In females, methyltestosterone is used for the palliative treatment of androgen-responsive, advanced, inoperable, metastatic (skeletal) carcinoma of the breast in women who are 1-5 years postmenopausal. Primary goals of therapy in these women include ablation of the ovaries. Other methods of counteracting estrogen activity include adrenalectomy, hypophysectomy, and/or antiestrogen therapy (e.g., tamoxifen). Androgen therapy also has been used in premenopausal women with carcinoma of the breast who have benefited from oophorectomy and are considered to have a hormone-responsive tumor. The decision to use androgen therapy in women with carcinoma of the breast should be made by an oncologist with expertise in the treatment of this carcinoma.
Methyltestosterone also has been used for the prevention of postpartum breast pain and engorgement; however, the drug does not appear to prevent or suppress lactation.
In females, methyltestosterone is used in combination with estrogens for the management of moderate to severe vasomotor symptoms associated with menopause in patients who do not respond adequately to estrogens alone. While estrogen/androgen combinations were found to be effective for the management of vasomotor symptoms associated with menopause under a determination made by the FDA in 1976, formal administrative proceedings were initiated by the FDA in April 2003 to examine the effectiveness of estrogen/androgen combinations for this indication. FDA is undertaking this action because the agency does not believe there is substantial evidence available to establish the contribution of androgens to the effectiveness of estrogen/androgen combinations for the management of vasomotor symptoms in menopausal women who do not respond to estrogens alone. The FDA will allow continued marketing of combination estrogen/androgen products while the matter is under study.
Although methyltestosterone has been used in other conditions (e.g., fractures, surgery, convalescence, functional uterine bleeding), there is a lack of substantial evidence that androgens are effective in these conditions. In addition, the FDA states that there currently is no evidence to support the safety and efficacy of methyltestosterone as an aphrodisiac (i.e., to arouse or increase sexual desire or to improve sexual performance).
Misuse, Abuse, and Dependence
Because of their anabolic and androgenic effects on performance (ergogenic potential) and physique, androgens have been misused and abused by athletes, bodybuilders, weight lifters, and others, including high school- and college-aged individuals engaged in sports. Following review of data from published literature and case reports in October 2016, the FDA concluded that misuse and abuse of androgens are associated with serious adverse cardiovascular, hepatic, endocrine, and mental health effects. (
See Cautions; also .)
Serum testosterone concentrations should be evaluated in patients who may be misusing or abusing androgens (e.g., patients experiencing serious adverse cardiovascular or psychiatric effects); however, serum testosterone concentrations may be below or within the normal range in patients abusing synthetic derivatives of testosterone.