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Open research questions in Myasthenia Gravis and Thymoma

123 gap statements mined from Myasthenia Gravis and Thymoma papers in our 4.5M-paper local library, which holds 586 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.

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What the literature leaves open

  • There is a lack of awareness of superior herniation of the thymus among clinicians. The condition often leads to unnecessary investigations and interventions.

    Now you see it; Now you don't: Superior herniation of thymus presenting as an intermittent anterior neck mass · 2026 · DOI

  • The need for comprehensive evaluation of long-standing masses that undergo change.

    Case Report: A seventy-year-stable chest wall bronchogenic cyst with sudden enlargement: coincidence or connection with a concurrent thymic carcinoma? · 2026 · DOI

  • The follow-up duration was long-term outcomes, including predominantly short-term; thus, delayed autoimmune events, long-term prognosis and disease recurrence, remain unclear. Several limitations of the present study should be fully acknowledged when interpreting the results. Large-scale, international multicenter cohort studies are warranted for further validation.

    Real-world study of efgartigimod in AChR antibody-positive generalized myasthenia gravis: thymus status, multi-domain symptom improvement and steroid-sparing effect · 2026 · DOI

  • There is a need to identify factors associated with secondary generalization in patients with ocular-onset myasthenia gravis. There is a lack of understanding of the clinical relevance of quantitative anti-AChR antibody assessment for risk stratification in ocular-onset myasthenia gravis.

    Risk factors for disease generalization in acetylcholine receptor antibody-positive ocular myasthenia: a multicenter retrospective study · 2026 · DOI

  • To improve this, future research should focus on faster, more accessible diagnostic tools, such as highly sensitive cell-based antibody tests or portable electrophysiology devices, that can be used in community settings and smaller hospitals (Tannemaat, 2024).

    Ocular Myasthenia Gravis: Diagnosis, Management, and Risk of Disease Progression · 2026 · DOI

  • Limited evidence exists concerning the efficacy of immunosuppressive therapy in altering the natural progression of OMG. Nevertheless, owing to the small number of OMG cases, no definitive conclusions regarding treatment responsiveness can be drawn and larger-scale studies are warranted to further evaluate its efficacy.

    Eculizumab in Myasthenia Gravis: A Multicenter Retrospective Real-World Study in China · 2026 · DOI

  • Future research should focus on developing predictive biomarkers immunosuppression optimizing protocols for this high-risk population.

    Fatal Fulminant Myocarditis Following a Single Dose of Pembrolizumab in a Patient with Recurrent Thymoma: A Case Report · 2026 · DOI

  • The lack of understanding of the changes in body composition in patients with myasthenia gravis. The need for a non-invasive and informative method for evaluating the condition of patients with myasthenia gravis.

    Features of bioimpedance investigation in patients with myasthenia gravis · 2026 · DOI

  • The coexistence of stiff-person syndrome and myasthenia gravis is exceptionally rare, with fewer than 20 cases reported to date. The potential role of thymic pathology in the development of multiple autoimmune neurological disorders is not fully understood.

    Case Report: Efgartigimod: a beacon of hope for overlapping stiff-person syndrome and myasthenia gravis following thymoma · 2026 · DOI

  • Current management of MG crisis primarily involves plasma exchange or intravenous gamma-globulin, which have short-lived efficacy. The need for other therapeutic modalities for MG crisis is a recognized unmet need. There is a lack of studies evaluating the safety and efficacy of efgartigimod in adults with severe MG exacerbations.

    Prospective study of efgartigimod in severe myasthenia gravis exacerbations requiring ventilatory or enteral support · 2026 · DOI

  • Future research should focus on the development of precision therapies for MG. The identification of predictive biomarkers and the development of targeted immunotherapies are areas for future research. The study of the immune microenvironment and population heterogeneity in MG should continue.

    The immune microenvironment and population heterogeneity in myasthenia gravis: implications for precision therapeutics · 2026 · DOI

  • There is a need for a better understanding of the immune microenvironment and population heterogeneity in MG. Current therapies for MG have limitations, including variable efficacy and delayed onset.

    The immune microenvironment and population heterogeneity in myasthenia gravis: implications for precision therapeutics · 2026 · DOI

  • The relationship between peripheral B-cell depletion and clinical efficacy is not fully established. Nevertheless, the most appropriate RTX dose remains uncertain.

    Efficacy and safety of low-dose rituximab in MuSK antibody-positive myasthenia gravis: a single-center retrospective study · 2026 · DOI

  • Available evidence indicates a potential therapeutic role for telitacicept in MG, although the magnitude and durability of clinical benefits remain uncertain.

    Telitacicept in myasthenia gravis: a systematic review of clinical outcomes, steroid-sparing effects, and safety · 2026 · DOI

  • Diagnosing CMSs can be challenging due to the heterogeneous nature of the disease. Treatment options for CMSs are limited. The patient's symptoms were progressive and debilitating.

    First Korean Case of DOK7 Congenital Myasthenic Syndrome Responsive to Ephedrine Therapy · 2026 · DOI

  • There is a lack of reported cases of DOK7 CMS in the Korean population. The treatment response of DOK7 CMS patients to ephedrine therapy is not well understood.

    First Korean Case of DOK7 Congenital Myasthenic Syndrome Responsive to Ephedrine Therapy · 2026 · DOI

  • There is a need for real-world data on the safety and effectiveness of efgartigimod in patients with generalized myasthenia gravis by autoantibody subtype.

    Safety and effectiveness of efgartigimod in patients with generalized myasthenia gravis by autoantibody subtype: analysis of post-marketing surveillance in Japan · 2026 · DOI

  • Thymic carcinoma (TC) is a rare and aggressive malignancy with limited evidence guiding second-line treatment after prior systemic therapy.

    Real-world outcomes of second-line therapy in advanced thymic carcinoma: a multicenter Turkish Oncology Group (TOG) study · 2026 · DOI

  • However, data on infection-related outcomes in patients with MG receiving RTX are limited, particularly in the context of low-dose regimens, where systematic real-world evidence is scarce.

    Infections and hypogammaglobulinemia in myasthenia gravis patients receiving low-dose rituximab: a real-world cohort study · 2026 · DOI

  • Prospective longitudinal studies integrating thymic imaging with functional immune biomarkers are warranted to clarify the causal direction and clinical significance of HCC-associated thymic involution.

    Radiographic evidence of thymic involution in hepatocellular carcinoma: a propensity score-matched study in viral cirrhosis · 2026 · DOI

  • However, whether HCC occurrence is associated with structural thymic involution in humans, particularly among patients with liver cirrhosis, remains unclear.

    Radiographic evidence of thymic involution in hepatocellular carcinoma: a propensity score-matched study in viral cirrhosis · 2026 · DOI

  • The study is exploratory, and the number of cases is not sufficient to answer confirmatory questions. The study only serves to discover indications of possible correlations between biomarker levels and outcomes.

    Multicentric prognostic observational study on biomarker profile in immunotherapy-naïve patients with highly active generalized myasthenia gravis (PROGNO-MG): a study protocol · 2026 · DOI

  • The high cost and logistical constraints of echocardiography, PET/CT, and micro-CT acquisition limited the imaging subset size. The study required a multi-dimensional phenotyping framework to investigate the complex relationships between neuromuscular dysfunction, immune alterations, and whole-animal phenotypic changes.

    A multi-dimensional phenotyping framework reveals coordinated neuromuscular, immune, and imaging-derived phenotypes in experimental autoimmune myasthenia gravis · 2026 · DOI

  • The imaging subset size was limited due to the high cost and logistical constraints of echocardiography, PET/CT, and micro-CT acquisition. The study was not designed or powered for definitive group-level inference. The imaging-derived findings should be interpreted as exploratory and require validation in unselected cohorts.

    A multi-dimensional phenotyping framework reveals coordinated neuromuscular, immune, and imaging-derived phenotypes in experimental autoimmune myasthenia gravis · 2026 · DOI

  • Greater self-reported activity was associated with less fatigue or lower MG burden in several cross-sectional datasets, but reverse causality and residual confounding remain plausible; evidence from repeated measurements was sparse and heterogeneous.

    Physical Activity, Exercise, and Rehabilitation in Myasthenia Gravis: A Systematic Review and Narrative Synthesis · 2026 · DOI

Most-cited papers in Myasthenia Gravis and Thymoma

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Open questions raised by papers listed on this page include: (1) There is a lack of awareness of superior herniation of the thymus among clinicians (“Now you see it; Now you don't: Superior herniation of thymus presenting as an…”, 2026); (2) The follow-up duration was long-term outcomes, including predominantly short-term; thus, delayed autoimmune events, long-term prognosis and disease recurrence, remain unclear (“Real-world study of efgartigimod in AChR antibody-positive generalized…”, 2026); (3) There is a need to identify factors associated with secondary generalization in patients with ocular-onset myasthenia gravis (“Risk factors for disease generalization in acetylcholine receptor…”, 2026); (4) Owing to the small number of OMG cases, no definitive conclusions regarding treatment responsiveness can be drawn and larger-scale studies are warranted to further evaluate its efficacy (“Eculizumab in Myasthenia Gravis: A Multicenter Retrospective Real-World Study…”, 2026); (5) Future research should focus on developing predictive biomarkers immunosuppression optimizing protocols for this high-risk population (“Fatal Fulminant Myocarditis Following a Single Dose of Pembrolizumab in a…”, 2026). 123 gap statements have been mined from Myasthenia Gravis and Thymoma papers in our 4.5M-paper local library, which holds 586 papers on the topic; the gaps come from whichever of those papers state one. They are mostly the research gaps the authors state and the papers' abstracts, plus future-work, limitations and challenges passages. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one, so you can read the original claim in context.

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