Priapism in Boys and Young Men: Incidence and Prevalence

这项研究的主要目的是对患有镰状细胞病(SCD)的男孩和年轻男子的异常勃起进行全面的调查。因此，它代表着朝着确定一种有效的方法来管理异常勃起和减少阳萎的频率迈出了第一步，阳萎是SCD成年男性异常勃起的主要并发症。这项建议的目的是：1)进行纵向队列研究，以确定异常勃起的发生率与青春期生理和荷尔蒙发育阶段及早熟的关系，探索异常勃起与心理调整之间的关系；以及2)进行安慰剂对照的试点研究 将评估伪麻黄碱预防异常勃起能力的干预试验。这项建议的第一阶段涉及一项观察性研究，该研究将向所有7-29岁患有SCD的男孩和年轻男性开放。他们将每天记录阴茎异常勃起的发生情况，每6个月入院一次，对身体发育、生殖器青春期、身体成分、骨龄、促性腺激素、睾酮、胰岛素样生长因子和夜间阴茎肿胀进行密集评估。在此期间 在录取后，参与者还将完成几项心理评估。这一阶段的数据将被用来确定异常勃起和青春期、促性腺激素水平和睾酮浓度之间的联系(如果有)。这些数据还将确定异常勃起和心理调整之间是否存在关系。该提案的第二阶段将寻求将那些被发现有合格异常勃起发作的患者纳入伪麻黄碱的随机、双盲、安慰剂对照临床试验。这项初步研究的目的是确定伪麻黄碱是否能够预防异常勃起。在试验期间，每个参与者将继续保持相同的每日日记。此外，每个参与者将在试验开始和结束时被允许完成与观察阶段相同的身体、激素和心理评估。预防试验的结果衡量标准将是异常勃起的发生率、异常勃起持续时间和异常勃起发作之间的间隔时间。此外，还将对治疗臂之间的身体发育、二级性发育、身体成分和心理调整进行比较。收集的数据将确定某些关键方法的可行性，并将提供比率、标准偏差、中位数事件间隔时间的估计。这些结果在规划大规模治疗试验之前是绝对必要的，将为这一领域未来的工作提供生物学和统计学背景。

The primary goal of this study is to conduct a comprehensive investigation of priapism in boys and young men with sickle cell disease (SCD). As such, it represents the initial step toward defining an effective way to manage priapism, and to reduce the frequency of impotence that is its major complication in adult males with SCD. The aims of this proposal are: 1) to conduct a Iongitudinal cohort study in order to define the incidence of priapism in relationship to the physical and hormonal developmental stages of puberty and early maturity, to explore the relationship between priapism and psychological adjustment; and 2) to conduct a pilot, placebo-controlled intervention trial in which the ability of pseudoephedrine to prevent priapism will be evaluated. The first stage of this proposal involves an observational study that will be open to all boys and young men with SCD between the ages of 7 -29 years. They will keep daily records of the occurrence of priapism, and will be admitted every 6 months for an intensive evaluation of physical growth, genital pubertal stage, body composition, bone age, gonadotropins, testosterone, insulin-like growth factor and nocturnal penile tumescence. During these admissions, the participants will also complete several psychological assessments. Data from this phase will be used to identify the association (if any) between priapism and pubertal stage, gonadotropin levels, and testosterone concentration. The data will also determine if there is a relationship between priapism and psychological adjustment. The second stage of this proposal will seek to enroll those individuals who are found to have qualifying episodes of priapism on to a randomized, double-blinded, placebo-controlled clinical trial of pseudoephedrine. The purpose of this pilot study is to determine whether pseudoephedrine is able to prevent episodes of priapism. During this trial, each participant will continue to keep the same daily journal. In addition, each participant will be admitted at the beginning and end of the trial to complete the same physical, hormonal and psychological assessments as were performed in the observational phase. The outcome measures of the prevention trial will be the occurrence of priapism, duration of priapism and the interval between episodes of priapism. In addition, physical growth, secondary sexual development, body composition and psychological adjustment will be compared between the treatment arms. The data collected will establish the feasibility of certain key methods, and will provide estimates of rates, standard deviations, median time-to-event. These results, which are absolutely essential before a large scale treatment trial can be planned, will provide the biological and statistical background for future work in this field.