Personalized Management and Timing of Thymectomy in Juvenile Myasthenia Gravis: Insights from Routine Clinical Scale Use in a Single-Center Retrospective Cohort—A Case Series

<b>Introduction:</b> Juvenile myasthenia gravis (JMG) is a rare autoimmune disorder with a variable clinical course and limited pediatric-specific treatment guidelines. Objective clinical scales, such as the Quantitative Myasthenia Gravis (QMG) Score and the Pediatric Myasthenia Gravis Quality of Life 15 (PM-QOL15), may support individualized management, but their role in routine practice remains underexplored. <b>Methods:</b> We retrospectively reviewed 10 seropositive JMG patients followed at a single tertiary neuromuscular clinic between 2014 and 2024. All patients underwent a systematic assessment with QMG at each visit, while PM-QOL15 was administered at the final visit. Clinical data, comorbidities, antibody status, treatment modalities, and outcomes were analyzed. Associations between treatment strategies, comorbidities, and scale scores were explored using appropriate statistical methods. <b>Results:</b> Seven patients (70%) underwent thymectomy, resulting in a reduction in mean QMG scores from 7.7 to 2.4, though residual relapses were observed. Chronic intravenous immunoglobulin (IVIG) therapy, administered to 70% of patients, did not significantly reduce relapse rates or steroid exposure and was associated with higher QMG scores in the second year, suggesting use in more severe phenotypes rather than therapeutic efficacy. Prolonged corticosteroid therapy did not improve remission time or relapse frequency and was complicated by major adverse effects in two patients. Timing of azathioprine initiation showed no significant correlation with relapse frequency. PM-QOL15 correlated strongly with mean QMG (r = 0.88, <i>p</i> < 0.001), reflecting cumulative disease burden. Patients with comorbidities required longer stabilization, although differences were not statistically significant. <b>Conclusions:</b> The routine integration of QMG and PM-QOL15 into follow-up may facilitate the earlier recognition of subclinical deterioration, provide objective measures of treatment response, and guide personalized management in JMG. Thymectomy showed benefit in selected patients, while the long-term roles of IVIG and corticosteroids remain uncertain. Larger multicenter prospective studies are warranted to confirm these findings and refine evidence-based strategies for pediatric JMG.

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Journal
Children
Published
2025-10-15
DOI
https://doi.org/10.3390/children12101389
Primary Topic
Myasthenia Gravis and Thymoma
Type
article
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article

Personalized Management and Timing of Thymectomy in Juvenile Myasthenia Gravis: Insights from Routine Clinical Scale Use in a Single-Center Retrospective Cohort—A Case Series

Nezih Onur Ermerak, Gülten Öztürk, Yasemi̇n Gökdemi̇r, Pınar Kahraman Koytak et al.
Children
Myasthenia Gravis and Thymoma
article

Personalized Management and Timing of Thymectomy in Juvenile Myasthenia Gravis: Insights from Routine Clinical Scale Use in a Single-Center Retrospective Cohort—A Case Series

Nezih Onur Ermerak, Gülten Öztürk, Yasemi̇n Gökdemi̇r, Pınar Kahraman Koytak, Bilgihan Bıkmazer, Ela Erdem Eralp, Olcay I. Unver, Burcu Karakayalı, Kayıhan Uluç, Pınar Ergenekon, Elif Acar Arslan, Gulcan Akyuz, İsmail Hakkı Akbeyaz, Sarp Özcan, Dilşad Türkdoğan
article en

Abstract

<b>Introduction:</b> Juvenile myasthenia gravis (JMG) is a rare autoimmune disorder with a variable clinical course and limited pediatric-specific treatment guidelines. Objective clinical scales, such as the Quantitative Myasthenia Gravis (QMG) Score and the Pediatric Myasthenia Gravis Quality of Life 15 (PM-QOL15), may support individualized management, but their role in routine practice remains underexplored. <b>Methods:</b> We retrospectively reviewed 10 seropositive JMG patients followed at a single tertiary neuromuscular clinic between 2014 and 2024. All patients underwent a systematic assessment with QMG at each visit, while PM-QOL15 was administered at the final visit. Clinical data, comorbidities, antibody status, treatment modalities, and outcomes were analyzed. Associations between treatment strategies, comorbidities, and scale scores were explored using appropriate statistical methods. <b>Results:</b> Seven patients (70%) underwent thymectomy, resulting in a reduction in mean QMG scores from 7.7 to 2.4, though residual relapses were observed. Chronic intravenous immunoglobulin (IVIG) therapy, administered to 70% of patients, did not significantly reduce relapse rates or steroid exposure and was associated with higher QMG scores in the second year, suggesting use in more severe phenotypes rather than therapeutic efficacy. Prolonged corticosteroid therapy did not improve remission time or relapse frequency and was complicated by major adverse effects in two patients. Timing of azathioprine initiation showed no significant correlation with relapse frequency. PM-QOL15 correlated strongly with mean QMG (r = 0.88, <i>p</i> < 0.001), reflecting cumulative disease burden. Patients with comorbidities required longer stabilization, although differences were not statistically significant. <b>Conclusions:</b> The routine integration of QMG and PM-QOL15 into follow-up may facilitate the earlier recognition of subclinical deterioration, provide objective measures of treatment response, and guide personalized management in JMG. Thymectomy showed benefit in selected patients, while the long-term roles of IVIG and corticosteroids remain uncertain. Larger multicenter prospective studies are warranted to confirm these findings and refine evidence-based strategies for pediatric JMG.

ChildrenVol. 12(10)
Marmara University (TR)
Openalex Percentile: Top 58%
Myasthenia Gravis and Thymoma
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