Azziz R, Sanchez L A, Knochenhauer E S, Moran C, Lazenby J, Stephens K C, Taylor K, Boots L R
Departments of Obstetrics and Gynecology, University of Alabama at Birmingham, Birmingham, Alabama 35233, USA.
J Clin Endocrinol Metab. 2004 Feb;89(2):453-62. doi: 10.1210/jc.2003-031122.
The objective of the present study was to estimate the prevalence of the different pathological conditions causing clinically evident androgen excess and to document the degree of long-term success of suppressive and/or antiandrogen hormonal therapy in a large consecutive population of patients. All patients presenting for evaluation of symptoms potentially related to androgen excess between October 1987 and June 2002 were evaluated, and the data were maintained prospectively in a computerized database. For the assessment of therapeutic response, a retrospective review of the medical chart was performed, after the exclusion of those patients seeking fertility therapy only, or with inadequate follow-up or poor compliance. A total of 1281 consecutive patients were seen during the study period. Excluded from analysis were 408 patients in whom we were unable to evaluate hormonal status, determine ovulatory status, or find any evidence of androgen excess. In the remaining population of 873 patients, the unbiased prevalence of androgen-secreting neoplasms was 0.2%, 21-hydroxylase-deficient classic adrenal hyperplasia (CAH) was 0.6%, 21-hydroxylase-deficient nonclassic adrenal hyperplasia (NCAH) was 1.6%, hyperandrogenic insulin-resistant acanthosis nigricans (HAIRAN) syndrome was 3.1%, idiopathic hirsutism was 4.7%, and polycystic ovary syndrome (PCOS) was 82.0%. Fifty-nine (6.75%) patients had elevated androgen levels and hirsutism but normal ovulation. A total of 257 patients were included in the assessment of the response to hormonal therapy. The mean duration of follow-up was 33.5 months (range, 6-155). Hirsutism improved in 86%, menstrual dysfunction in 80%, acne in 81%, and hair loss in 33% of patients. The major side effects noted were irregular vaginal bleeding (16.1%), nausea (13.0%), and headaches (12.6%); only 36.6% of patients never complained of side effects. In this large study of consecutive patients presenting with clinically evident androgen excess, specific identifiable disorders (NCAH, CAH, HAIRAN syndrome, and androgen-secreting neoplasms) were observed in approximately 7% of subjects, whereas functional androgen excess, principally PCOS, was observed in the remainder. Hirsutism, menstrual dysfunction, or acne, but not alopecia, improved in the majority of patients treated with a combination suppressive therapy; although more than 60% experienced side effects.
本研究的目的是评估导致临床明显雄激素过多的不同病理状况的患病率，并记录在一大组连续患者中抑制性和/或抗雄激素激素治疗的长期成功程度。对1987年10月至2002年6月期间因可能与雄激素过多相关的症状前来评估的所有患者进行了评估，并将数据前瞻性地保存在计算机数据库中。为了评估治疗反应，在排除仅寻求生育治疗、随访不足或依从性差的患者后，对病历进行了回顾性审查。在研究期间共诊治了1281例连续患者。分析中排除了408例我们无法评估激素状态、确定排卵状态或未发现任何雄激素过多证据的患者。在其余873例患者中，分泌雄激素肿瘤的无偏患病率为0.2%，21-羟化酶缺乏型经典肾上腺皮质增生（CAH）为0.6%，21-羟化酶缺乏型非经典肾上腺皮质增生（NCAH）为1.6%，高雄激素性胰岛素抵抗黑棘皮病（HAIRAN）综合征为3.1%，特发性多毛症为4.7%，多囊卵巢综合征（PCOS）为82.0%。59例（6.75%）患者雄激素水平升高且有多毛症，但排卵正常。共有257例患者纳入激素治疗反应评估。平均随访时间为33.5个月（范围6 - 155个月）。86%的患者多毛症改善，80%的患者月经功能障碍改善，81%的患者痤疮改善，33%的患者脱发改善。观察到的主要副作用为不规则阴道出血（16.1%）、恶心（13.0%）和头痛（12.6%）；只有36.6%的患者从未抱怨有副作用。在这项对有临床明显雄激素过多的连续患者的大型研究中，约7%的受试者存在特定的可识别疾病（NCAH、CAH、HAIRAN综合征和分泌雄激素肿瘤），而其余患者存在功能性雄激素过多，主要是PCOS。大多数接受联合抑制治疗的患者多毛症、月经功能障碍或痤疮有所改善，但脱发未改善；尽管超过60%的患者有副作用。
