Smilowska Katarzyna, Carvalho Vanessa, Szejko Natalia, Costa João, Moro Elena, Antonini Angelo
Department of Neurology, Regional Hospital, Sosnowiec, Poland.
Department of Neurology, Unidade Local de Saúde de Santa Maria, Lisbon, Portugal.
Eur J Neurol. 2025 Jan;32(1):e70024. doi: 10.1111/ene.70024.
Parkinson's disease (PD) is a neurodegenerative disorder affecting both sexes, but differences exist between male and female in clinical manifestations, functional impact of symptoms and hormonal influences. Therefore, representativeness of females in PD trials indirectly determines the external validity of the clinical research in this field.
To estimate the representativeness of female in infusion therapy trials for advanced PD.
PubMed and EMBASE databases were searched (1980 to September 2023), along with congress abstracts, to identify controlled clinical trials and large non-controlled studies on infusion therapies in PD enrolling >100 patients. Random-effect meta-analysis was conducted to estimate mean pooled prevalence of females included in the studies. Subgroup analyses were conducted accordingly to study design and intervention.
We included 15 studies (six studies on levodopa-carbidopa intestinal gel, six on subcutaneous levodopa, two on subcutaneous apomorphine, and one on levodopa-carbidopa-entacapone intestinal gel). Sex was not a randomisation stratification factor in any of these studies. Only one study explored differences in the outcome estimated according to sex. Overall, the proportion of female included was 38% (95% CI:33%-43%; I = 74%), without differences between studies assessing different type of interventions (p = 0.72) or between study design (p = 0.35). In two studies, females represented the majority of included patients.
Female with advanced PD are underrepresented in infusion therapy trials. Most trials have overlooked sex-based biological differences that can impact clinical and functional outcomes, raising concerns about the generalizability of these findings to real-world contexts.
帕金森病（PD）是一种影响两性的神经退行性疾病，但男女在临床表现、症状的功能影响和激素影响方面存在差异。因此，PD试验中女性的代表性间接决定了该领域临床研究的外部有效性。
评估晚期PD输注治疗试验中女性的代表性。
检索了PubMed和EMBASE数据库（1980年至2023年9月）以及会议摘要，以确定纳入100名以上患者的PD输注治疗的对照临床试验和大型非对照研究。进行随机效应荟萃分析，以估计研究中纳入女性的平均合并患病率。根据研究设计和干预措施进行亚组分析。
我们纳入了15项研究（6项关于左旋多巴-卡比多巴肠凝胶，6项关于皮下注射左旋多巴，2项关于皮下注射阿扑吗啡，1项关于左旋多巴-卡比多巴-恩他卡朋肠凝胶）。在这些研究中，没有一项将性别作为随机分层因素。只有一项研究探讨了根据性别估计的结果差异。总体而言，纳入的女性比例为38%（95%CI：33%-43%；I=74%），评估不同类型干预措施的研究之间（p=0.72）或研究设计之间（p=0.35）没有差异。在两项研究中，女性占纳入患者的大多数。
晚期PD女性在输注治疗试验中的代表性不足。大多数试验忽视了可能影响临床和功能结果的基于性别的生物学差异，这引发了对这些研究结果在现实世界中可推广性的担忧。
