Zhou Jiachen, Kuba Anna, Nilius Sigrid, Pilipczuk Olga, Tarancón Thaïs, Tennigkeit Frank
UCB, Cambridge, MA, USA.
UCB, Warsaw, Poland.
Neurol Ther. 2025 Jun;14(3):1093-1103. doi: 10.1007/s40120-025-00750-w. Epub 2025 May 3.
Few published population-based studies report the incidence or prevalence of juvenile myasthenia gravis (JMG) due to the rarity of the disorder. Despite recent progress in new targeted treatments and ongoing developments, there remains a critical need for novel and effective therapies specifically for JMG. Most treatments used for JMG are only approved for adult patients with MG. Thus, a thorough evaluation and understanding of the basic epidemiology of JMG is needed.
We conducted a population-based retrospective study to estimate the annual incidence and prevalence of JMG in the US from 2010 to 2020 by analyzing the Merative™ MarketScan Commercial Claims and Encounters Database (CCAE) and Multi-State Medicaid Database (MDCD).
The incidence of JMG in 2020 was 5.9 [95% confidence interval (CI) 3.3-9.7] per million person-years in CCAE and 8.7 (95% CI 6.0-12.3) per million person-years in MDCD, with considerable variation across the study period. The prevalence of diagnosed JMG remained fairly consistent, with 25.3 (95% CI 19.9-32.2) per million population in CCAE and 37.6 (95% CI 31.9-44.4) per million population in MDCD in 2020. Both databases consistently showed higher incidence and prevalence among girls compared with boys. No clear pattern was observed in incidence by age of onset over the study period, whereas prevalence generally increased with age. Both incidence and prevalence by age of onset were higher in MDCD than CCAE. Furthermore, higher incidence and prevalence were observed among Black population compared with White population. Overall, no obvious increasing or decreasing trend was observed during the study period.
The incidence and prevalence of JMG in the US, previously understudied, may be higher than earlier research suggested, possibly due to limited research into the epidemiology of JMG. This finding implies that the actual burden of JMG could be greater than previously estimated.
由于青少年型重症肌无力（JMG）较为罕见，很少有基于人群的已发表研究报告其发病率或患病率。尽管在新的靶向治疗方面取得了进展且仍在不断发展，但对于专门针对JMG的新型有效疗法仍有迫切需求。大多数用于JMG的治疗方法仅被批准用于成年重症肌无力患者。因此，需要对JMG的基本流行病学进行全面评估和了解。
我们通过分析Merative™ MarketScan商业理赔与就诊数据库（CCAE）和多州医疗补助数据库（MDCD），开展了一项基于人群的回顾性研究，以估算2010年至2020年美国JMG的年发病率和患病率。
2020年，CCAE中JMG的发病率为每百万人口年5.9 [95%置信区间（CI）3.3 - 9.7]，MDCD中为每百万人口年8.7（95% CI 6.0 - 12.3），在整个研究期间存在相当大的差异。确诊JMG的患病率保持相对稳定，2020年CCAE中为每百万人口25.3（95% CI 19.9 - 32.2），MDCD中为每百万人口37.6（95% CI 31.9 - 44.4）。两个数据库均一致显示，女孩的发病率和患病率高于男孩。在研究期间，未观察到发病年龄与发病率之间的明显模式，而患病率通常随年龄增加。MDCD中按发病年龄划分的发病率和患病率均高于CCAE。此外，与白人相比，黑人的发病率和患病率更高。总体而言，在研究期间未观察到明显的上升或下降趋势。
美国JMG的发病率和患病率此前研究较少，可能高于早期研究所表明的水平，这可能是由于对JMG流行病学的研究有限。这一发现意味着JMG的实际负担可能比之前估计的更大。
