Treatment Of Myasthenia Gravis Research Articles

Possible sex and racial disparities in myasthenia gravis care.

Given the importance of early diagnosis and treatment of myasthenia gravis (MG), it is critical to understand disparities in MG care. We aimed to determine if there are any differences in testing, treatment, and/or access to neurologists for patients of varying sex and race/ethnicity with MG. We used a nationally representative healthcare claims database of privately insured individuals (2001-2018) to identify incident cases of MG using a validated definition. Diagnostic testing, steroid-sparing agents, intravenous immunoglobulin (IVIG), plasma exchange (PLEX), and thymectomy were defined using drug names or CPT codes. Steroid use was defined using AHFS class codes. We also determined whether an individual had a visit to a neurologist and the time between primary care and neurologist visits. Logistic regression determined associations between sex and race/ethnicity and testing, treatments, and access to neurologists. Female patients were less likely to get a computed tomography (CT) chest (odds ratio (OR) 0.73, 95% confidence interval (CI): 0.64-0.83), receive steroids (OR: 0.85, 95% CI: 0.75-0.97), steroid-sparing agents (OR: 0.84, 95% CI: 0.72-0.97), and IVIG or PLEX (OR: 0.80, 95% CI: 0.67-0.95). Black patients were less likely to receive steroids (OR: 0.78, 95% CI: 0.63-0.96). No significant disparities were seen in access to neurologists. We found healthcare disparities in MG treatment with female and Black patients receiving less treatment than men and those of other races/ethnicities. Further research and detailed assessments accounting for individual patient factors are needed to confirm these apparent disparities.

Efgartigimod in refractory autoimmune myasthenia gravis.

Efgartigimod, a neonatal Fc-receptor inhibitor, has recently been approved as treatment for myasthenia gravis (MG). In this retrospective cohort study, we aimed to systematically assess short- and long-term effectiveness of efgartigimod in patients with refractory MG. Sixteen patients with refractory autoimmune acetylcholine receptor MG were treated with efgartigimod. Data were collected from January 2021 to March 2023 on Myasthenia Gravis Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis score (QMG), Myasthenia Gravis Composite score (MGC) and the 15-item revised version of the Myasthenia Gravis Quality of Life questionnaire (MG-QoL15r). A favorable outcome was seen in 56% of patients at the last measurement. Out of 16 patients, 50% were an MG-ADL responder after the first treatment cycle. After 4 weeks, a clinically meaningful improvement compared to baseline was seen on the MG-ADL, QMG, and MGC. There was a statistically significant improvement on the MGQoL15r from baseline to week 4. The improvement was maintained until the last measurement for the MGC and the MGQoL15r. At the last visit, all patients had discontinued 4-weekly dosages, shifting to administration frequencies of 1, 2, or 3 weeks. Drug doses could be decreased for prednisolone (n = 7), azathioprine (n = 2), and intravenous immunoglobulin (n = 9). Frequency of plasma exchange was decreased in nine patients. In patients with refractory MG, efgartigimod was effective for at least half of all patients. Patients required more frequent dosing compared to the ADAPT phase 3 trial. In 80% of the patients concurrent medication could be reduced or discontinued.

Cost-effectiveness of eculizumab and efgartigimod for the treatment of anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.

Eculizumab and efgartigimod were approved to treat anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (anti-AChR Ab-positive gMG). These relatively new biological treatments provide a more rapid onset of action and improved efficacy compared with conventional immunosuppressive treatments, but at a higher cost. To assess the cost-effectiveness of eculizumab and, separately, efgartigimod, each added to conventional therapy vs conventional therapy alone, among patients with refractory anti-AChR Ab-positive gMG and those with anti-AChR Ab-positive gMG, respectively. A Markov model with 4 health states was developed, evaluating costs and utility with a 4-week cycle length and lifetime time horizon from a health care system perspective and a modified societal perspective including productivity losses from patients and caregiver burden. Model inputs were informed by key clinical trials and relevant publications identified from targeted literature reviews, and drug costs were identified from Micromedex Red Book. Costs and outcomes were discounted at 3% per year. Incremental cost-effectiveness ratios (ICERs; cost per quality-adjusted life-year [QALY] gained) were calculated for each comparison. Among the corresponding populations, lifetime costs and QALYs, respectively, for eculizumab were $5,515,000 and 11.85, and for conventional therapy, $308,000 and 10.29, resulting in an ICER of $3,338,000/QALY gained. For efgartigimod, lifetime costs and QALYs, respectively, were $6,773,000 and 13.22, and for conventional therapy, $322,000 and 9.98, yielding an ICER of $1,987,000/QALY gained. After applying indirect costs in a modified societal perspective, the ICERs were reduced to $3,310,000/QALY gained for eculizumab and $1,959,000/QALY gained for efgartigimod. Eculizumab and efgartigimod are rapidly acting and effective treatments for myasthenia gravis. However, at their current price, both therapies greatly exceeded common cost-effectiveness thresholds, likely limiting patient access to these therapies.

P.057 Achievement of minimal symptom expression in acetylcholine receptor antibody-positive participants treated with efgartigimod in ADAPT/ADAPT+

Background: A key efficacy indicator in generalized myasthenia gravis (gMG) treatment is improvement in MG-ADL score. Minimal symptom expression (MSE, MG-ADL total score of 0 or 1) is explored as a novel proposed treatment target in gMG in the phase 3 study of intravenous efgartigimod, ADAPT, and its open-label extension, ADAPT+. Methods: Post hoc analyses of acetylcholine receptor antibody positive participants in ADAPT (n=129) and ADAPT+ (n=111) were performed. Results: In ADAPT, 44.6% receiving efgartigimod achieved MSE vs 10.9% of participants given placebo. Despite less frequent assessment during ADAPT+, 40.5% of participants achieved MSE. Eighty-one percent of participants treated with efgartigimod who achieved MSE in ADAPT also achieved MSE during ADAPT+; 23% who had not achieved MSE in ADAPT did in ADAPT+. Achieving MSE was associated with substantial improvements in QMG, MGC, MG-QoL15r, and EQ-5D-5L mean scores of 11.4, 16.0, 12.4, and 0.3 points, respectively, from baseline to best score (across all visits). These drastic improvements resulted in quality of life (QoL) comparable to that of healthy populations. MSE achievement also resulted in sustained improvements in these disease-specific and QoL measures. Conclusions: Participants who achieved MSE showed substantial and consistent improvements across multiple disease measures and experienced QoL comparable to that of healthy populations.

Results Of Minimally Invasive Vats Thymectomy In Miastenia Gravis Patients Compared With More Invasive Approaches - 10-Year Experience In A Single Center.

Myasthenia gravis (MG) is an autoimmune, neurologic disease that causes a wide range of symptoms. While the transsternal, transcervical and thoracotomy approaches are accepted as effective, there is still debate regarding the VATS approach. We analyzed our center's surgical experience with thymectomy for myasthenia gravis, comparing the results of patients operated on using VATS and more invasive approaches, over a period of 10 years. A search of the department's surgical database for myasthenia gravis cases between January 2010 and January 2021, revealed a total of 40 cases. Twenty-four patients were included in the final analysis and were distributed into two groups: the VATS procedure group (group A) and the open procedure group (group B). The latter included sternotomy, thoracotomy, transcervical and hemiclamshell approaches. Only radical thymectomies were included. The established outcomes were clinical improvement defined as asymptomatic remission, reduction, or discontinuation of the medication necessary to achieve optimal symptom control. The median follow-up time was 27 months (ranging from 4 to 75 months). Videothoracoscopy radical thymectomy was performed on 12 patients. Complete remission with no medication was achieved in 1 case (8.3%), while 2 patients (16.7%) became asymptomatic with reduced medication. An improvement (reduced symptoms or decreased medication) was observed in 8 cases (66.6%). No change in clinical outcome was noted in 1 patient (8.3%). None of the patients reported worsening symptoms. Open thymectomy was performed on 12 patients. Complete remission with no medication was achieved in 1 case (8.3%), while 2 patients (16.7%) became asymptomatic with reduced medication. An improvement was noted in 6 cases (50%). No change in clinical outcome was observed in 3 patients (25%) whereas 2 of them (16.7%) experienced slightly better symptom control but with a significant increase in medication. One patient (8.3%) described the clinical results as without any significant change. None of the patients reported worsening symptoms. The videotoracoscopic approach in the treatment of myasthenia gravis is non-inferior compared to the open approach and effective in a long-term follow-up, offering all the additional benefits of less invasive surgery.

Acupuncture and moxibustion treatment for myasthenia gravis: A systematic review and meta-analysis.

Myasthenia gravis (MG) is a common autoimmune disease that often involves the skeletal muscle of the whole body and seriously affects patients' quality of life. Acupuncture and moxibustion treatment of MG has unique advantages, the aim is to evaluate the clinical effect of acupuncture and moxibustion on MG. The literature on acupuncture and moxibustion treating MG in PubMed, CochraneLibrary, EMBASE, SCI, China Academic Journals full-text database, China Biology Medicine disc, VIP and Wanfang database were searched through computers from the establishment of the database to December 2022. A total of 11 studies were included, involving 658 patients, where 330 in the treatment group and 328 in the control group. The results of the meta-analysis showed that the treatment group performed better than the control group in improving the total clinical response rate (OR = 3.26, 95%[2.04,5.21], P < .01). Additionally, the treatment group outperformed the control group in raising the absolute clinical score (MD = -3.48, 95%CI[-5.17, -1.78], P < .01). However, there was no significant difference between the treatment group and the control group in improving the level of serum interleukin-6 receptor (MD = -1.45,95%CI[-6.85,3.95], P > .05) and OMG quantitative score (MD = -2.16,95%CI[-4.85,0.52], P > .05). The total clinical effective rate was tested for publication bias, which showed that the 2 sides of the funnel plot were asymmetrical, suggesting the possible existence of publication bias. Acupuncture and moxibustion has a good effect on MG, which is better than conventional Western medicine in improving the total clinical effective rate and absolute clinical score.

Treatment of concomitant myasthenia gravis and Lambert-Eaton myasthenic syndrome with autologous CD19-targeted CAR T cells

Myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) are autoimmune disorders affecting neuromuscular transmission. Their combined occurrence is rare, and treatment remains challenging. Two women diagnosed with concomitant MG/LEMS experienced severe, increasing disease activity despite multiple immunotherapies. Anti-CD19 chimeric antigen receptor (CAR) Tcells have shown promise for treating autoimmune diseases. This report details the safe application of anti-CD19 CAR Tcells for treating concomitant MG/LEMS. After CAR Tcell therapy, both patients experienced rapid clinical recovery and regained full mobility. Deep B cell depletion and normalization of acetylcholine receptor and voltage-gated calcium channel N-type autoantibody levels paralleled major neurological responses. Within 2months, both patients returned to everyday life, from wheelchair dependency to bicycling and mountain hiking, and remain stable at 6 and 4months post-CAR Tcell infusion, respectively. This report highlights the potential for anti-CD19 CAR Tcells to achieve profound clinical effects in the treatment of neuroimmunological diseases.

Depression and anxiety among Filipino myasthenia gravis patients seen in a public tertiary hospital in Metro Manila

Anxiety and depression complicate treatment of Myasthenia Gravis (MG) as it overlaps with somatic symptoms related to the disease itself. While both are established factors for disease severity in developed countries, similar research in underdeveloped regions is lacking. This study aims to determine the prevalence of anxiety and depression in patients with MG in a tertiary hospital in the Philippines, and to identify the sociodemographic and illness related factors that affect both mental disorders. Locally validated Hospital Anxiety Depression Scale-Pilipino (HADS-P) questionnaires were administered to 59 consenting MG patients to screen for anxiety and depression. Those who scored more than 8 for each subset of HADS-P were presumed to have anxiety, depression or both. The prevalence of anxiety and depression was 50.9% and 15.3%, respectively. Among the factors analyzed, only the female sex had a significant association with anxiety (p=0.019). The total HADS-P score (p=0.009) and HADS-P score for anxiety (p=0.008) had significant positive correlation with MGFA class. The high prevalence of anxiety and depression in MG warrants proper screening and recognition to institute proper treatment and management. This will also prevent confusion with somatic symptoms associated with MG.

Zilucoplan (Zilbrysq) for myasthenia gravis.

The FDA has approved the complement C5 inhibitor zilucoplan (Zilbrysq – UCB) for once-daily subcutaneous treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive. Zilucoplan is the first complement inhibitor to be approved for treatment of myasthenia gravis that can be self-administered.

Rozanolixizumab: A New Therapy in the Treatment of Myasthenia Gravis.

The aims of this article are to review the clinical aspects of rozanolixizumab, to describe clinical trial results that led to the drug's approval, and to examine the impact on patient care to aid clinical decision making. A PubMed search was conducted using the terms Rystiggo™, rozanolixizumab, rozanolixizumab therapy, and myasthenia gravis. The most recent prescribing information was also used for information relating to the drug and for identification of pertinent studies. Phase I, II, and III randomized controlled trials were all eligible for inclusion. Meeting abstracts and articles focusing on the use of rozanolixizumab or any indication other than generalized myasthenia gravis were excluded from this article. Food and Drug Administration approval of rozanolixizumab is based on the phase III MycarinG study in patients with generalized myasthenia gravis. A phase II trial explored initial clinical efficacy and safety pertaining to the dose and frequency of rozanolixizumab across 2 treatment periods in patients with moderate to severe myasthenia gravis. Rozanolixizumab is the first therapy approved to treat patients positive for both types of antibodies, anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase, in generalized myasthenia gravis. The approval of rozanolixizumab represents an advancement in therapy for generalized myasthenia gravis. The provision of individualized, targeted, and well-tolerated treatment is valuable for the patients whose myasthenia gravis is not well controlled and who are seeking a medication with a rapid onset of action to improve their symptoms and overall quality of life.

Zilucoplan: A Newly Approved Macrocyclic Peptide for Treatment of Anti-Acetylcholine Receptor Positive Myasthenia Gravis

Zilucoplan is a synthetic macrocyclic peptide approved by the Food and Drug Administration (FDA), in October 2023, for the treatment of generalized myasthenia gravis. It is considered as an orphan drug that causes the inhibition of terminal complement cascade activation with a dual mechanism of action preventing the formation of the membrane attack complex (MAC) and the destruction of the neuromuscular junction. This drug has been demonstrated to be able to treat the generalized myasthenia gravis without significant adverse effects, with good efficacy, safety, and tolerability profile. Zilucoplan is not only innovative and promising in the therapeutics of generalized myasthenia gravis, but it could also be beneficial for the treatment of other diseases as well as a model for synthesis of analogues to improve pharmacological profile.

Patient perceptions of disease burden and treatment of myasthenia gravis based on sentiment analysis of digital conversations

Myasthenia gravis (MG) is a rare, autoimmune, antibody-mediated, neuromuscular disease. This study analyzed digital conversations about MG to explore unprovoked perspectives. Advanced search, data extraction, and artificial intelligence-powered algorithms were used to harvest, mine, and structure public domain digital conversations about MG from US Internet Protocol addresses (August 2021 to August 2022). Thematic analyses examined topics, mindsets, and sentiments/key drivers via natural language processing and text analytics. Findings were described by sex/gender and treatment experience with steroids or intravenous immunoglobulin (IVIg). The 13,234 conversations were extracted from message boards (51%), social media networks (22%), topical sites (21%), and blogs (6%). Sex/gender was confirmed as female in 5703 and male in 2781 conversations, and treatment experience was with steroids in 3255 and IVIg in 2106 conversations. Topics focused on diagnosis (29%), living with MG (28%), symptoms (24%), and treatment (19%). Within 3176 conversations about symptoms, eye problems (21%), facial muscle problems (18%), and fatigue (18%) were most commonly described. Negative sentiments about MG were expressed in 59% of conversations, with only 2% considered positive. Negative conversations were dominated by themes of impact on life (29%), misdiagnosis problems (27%), treatment issues (24%), and symptom severity (20%). Impact on life was a key driver of negativity in conversations by both men (27%) and women (34%), and treatment issues was a dominant theme in conversations by steroid-treated (29%) and IVIg-treated (31%) patients. Of 1382 conversations discussing treatment barriers, 36% focused on side effects, 33% on lack of efficacy, 21% on misdiagnosis, and 10% on cost/insurance. Side effects formed the main barrier in conversations by both steroid-treated and IVIg-treated patients. Capturing the patient voice via digital conversations reveals a high degree of concern related to burden of disease, misdiagnosis, and common MG treatments among those with MG, pointing to a need for treatment options that can improve quality of life.

Real-World experience with efgartigimod in patients with myasthenia gravis.

Recommendations for the treatment of myasthenia gravis (MG) have been difficult to develop because of limited evidence from large randomized controlled trials. New drugs and treatment approaches have recently been shown to be effective in phase 3 studies in seropositive generalized (g) MG. One such drug is efgartigimod, a human-Fc-fragment of IgG1, with a high affinity for the endosomal FcRn. We conducted a multicenter study to evaluate the real-world clinical and safety effects of efgartigimod in 22gMG patients. We evaluated the strategies for the timing of re-treatment with it. The participants received a total of 59 efgartigimod -treatment cycles. The median number of cycles was 2 (range 1-6). Twenty patients (86.3%) improved by at least 2 MG-ADL points after the first treatment cycle. The median MG-ADL score at baseline was 6.5 (range: 3-17) and 2.5 (range: 0-9) post-treatment (p < 0.001). A consistent improvement of at least 2 points in the MG-ADL score after each cycle occurs in 18 patients. The effect duration of the treatment was usually between 4 and 12weeks. Two major clinical patterns of treatment response were found. Treatment with efgartigimod was also associated with significant reductions of prednisone doses Overall, the treatment was safe and associated with only minor adverse events. The single fatality was apparently due tosevere respiratory failure. We found that efgartigimod is clinically effective, may be used as a steroid sparing agent and is generally safe for gMG patients. We recommend a personalized preventive treatment approach until clinical stabilization, followed by discontinuation and periodic evaluations.

Mycophenolate and methotrexate are better tolerated than azathioprine in myasthenia gravis

Azathioprine is recommended as the first-line steroid-sparing immunosuppressive agent for myasthenia gravis. Mycophenolate and methotrexate are often considered as second-line choices despite widespread consensus on their efficacy. We aimed to gather real-world data comparing the tolerability and reasons for discontinuation for these agents, by performing a national United Kingdom survey of side effects and reasons for discontinuation of immunosuppressants in myasthenia gravis. Of 235 patients, 166 had taken azathioprine, 102 mycophenolate, and 40 methotrexate. The most common side effects for each agent were liver dysfunction for azathioprine (23 %), diarrhoea for mycophenolate (14 %), and fatigue for methotrexate (18 %). Women were generally more likely to experience side effects of immunosuppressants. Azathioprine was significantly more likely to be discontinued than mycophenolate and methotrexate due to side effects. There was no significant difference in treatment cessation due to lack of efficacy. This study highlights the significant side-effect burden of treatment for myasthenia gravis. Mechanisms to reduce azathioprine toxicity should be utilised, however mycophenolate and methotrexate appear to be good treatment choices if teratogenicity is not a concern. Women are disadvantaged due to higher frequency of side effects and considerations around pregnancy and breastfeeding. Treatments with improved tolerability are needed.

Myasthenia gravis-Gender aspects and family planning

There is evidence that gender-specific differences can influence the diagnostics, treatment and long-term disease course of myasthenia gravis (MG). In women the diagnosis is often made during childbearing age. Gender-specific differences in MG and relevant aspects in routine clinical practice are presented. In addition, current studies on family planning, pregnancy and childbirth in MG are highlighted and treatment recommendations are derived. Narrative literature review. In addition to sociodemographic data, gender-specific differences encompass clinical as well as paraclinical factors, such as disease severity and antibody status. With few exceptions pregnancy is possible with good maternal and neonatal outcome. During pregnancy and peripartum, children of MG patients should be closely monitored for early detection and treatment of potential syndromes caused by diaplacental transfer of maternal antibodies. Gender-specific factors can influence the course of MG. Adequate medical counselling and multidisciplinary collaboration are essential for MG patients who wish to have children.

Depression and anxiety among Filipino myasthenia gravis patients seen in a public tertiary hospital in Metro Manila

Anxiety and depression complicate treatment of Myasthenia Gravis (MG) as it overlaps with somatic symptoms related to the disease itself. While both are established factors for disease severity in developed countries, similar research in underdeveloped regions is lacking. This study aims to determine the prevalence of anxiety and depression in patients with MG in a tertiary hospital in the Philippines, and to identify the sociodemographic and illness related factors that affect both mental disorders. Locally validated Hospital Anxiety Depression Scale-Pilipino (HADS-P) questionnaires were administered to 59 consenting MG patients to screen for anxiety and depression. Those who scored more than 8 for each subset of HADS-P were presumed to have anxiety, depression or both. The prevalence of anxiety and depression was 50.9% and 15.3%, respectively. Among the factors analyzed, only the female sex had a significant association with anxiety (p=0.019). The total HADS-P score (p=0.009) and HADS-P score for anxiety (p=0.008) had significant positive correlation with MGFA class. The high prevalence of anxiety and depression in MG warrants proper screening and recognition to institute proper treatment and management. This will also prevent confusion with somatic symptoms associated with MG.

New treatment options for generalized myasthenia gravis

Myasthenia gravis is an autoimmune disease in which autoantibodies against the postsynaptic membrane proteins of the neuromuscular junction disrupt impulse transmission thus causing pathological muscle weakness and fatigue that worsens throughout the day. Although the disease is not yet curable, most patients can achieve complete symptom control and improved quality of life with appropriate treatment. Four treatment strategies are used in clinical practice: symptomatic, immunosuppressive, immunomodulatory, and surgical treatment, which can help control the disease but are not equally effective for all patients. Symptomatic treatment with acetylcholinesterase (AChE) inhibitors is often not effective enough, so additional treatment with immunosuppressants is indicated. These are effective, but can cause systemic side effects if taken for long periods. Even polytherapy is often not sufficient enough to treat patients with myasthenia gravis. The challenges of treating this disease are encouraging to seek alternatives. Increasing attention is being paid to antibodies against acetylcholine receptors (AChRs) and other structures of the neuromuscular junction that are important in pathogenesis of myasthenia gravis. Drugs are being developed that target specific links in the immune system to reduce the risk of systemic adverse effects. Currently, only two drugs are approved for the treatment of generalized myasthenia gravis – eculizumab and efgartigimod. Both of them are safe and effective in treating generalized myasthenia gravis with prevalent anti-AChR antibodies. Currently, 10 other drugs are clinically tested for their safety and efficacy in treating patients with myasthenia gravis. In this article, we review publications that analyze biological therapy and its novelty in the treatment of myasthenia gravis. We focus more on already approved biological drugs.

Qualitative interviews to support development of a patient-reported companion measure to the Glucocorticoid Toxicity Index

IntroductionGlucocorticoids (GCs) are associated with multiple toxicities that have substantial impact on patients. We conducted qualitative interviews with patients to identify the toxicities that are most relevant from their perspective, with the goal of creating a patient-reported companion measure to the Glucocorticoid Toxicity Index (GTI), a clinician-facing instrument. MethodsThirty-one patients with recent or current GC use participated in concept elicitation interviews. Participants received GC treatment for myasthenia gravis, chronic inflammatory demyelinating polyradiculoneuropathy, vasculitis, or systemic lupus erythematosus. Transcripts were coded following a thematic analysis approach. ResultsParticipants reported more than 100 toxicities they believed to be associated with their GC medications. Common toxicities included weight gain (87%), increased appetite (84%), insomnia/sleep problems (77%), cognitive impairment/brain fog (71%), easy bruising (68%), anxiety (65%), irritability/short temper (65%), and osteoporosis (39%). These toxicities often centered on self-esteem, neuropsychiatric effects, skin toxicities, and musculoskeletal function. They can be categorized into domains such emphasizing neuropsychiatric, metabolic/endocrine, musculoskeletal, and dermatological effects, highlighting aspects of GC toxicity that patients are uniquely positioned to appreciate and report. ConclusionOur results confirm that the toxicities associated with GCs are pervasive and diverse, with substantial impact on patients' lives. These data will be used to inform the development of a patient-reported outcome measure assessing GC toxicity. This patient-reported instrument will be designed to complement the clinician-reported GTI, facilitating a more detailed understanding of the nuances of change in GC toxicity.

Registered trials on novel therapies for myasthenia gravis: a cross-sectional study on ClinicalTrials.gov

Novel biologics in MG therapy research is on the rise. This research aimed to investigate the characteristics of registered trials on novel therapies for myasthenia gravis on ClinicalTrials.gov. This cross-sectional study used a descriptive approach to assess the features of the included trials on ClinicalTrials.gov. We found 62 registered trials from 2007 to 2023 on ClinicalTrials.gov. The results showed a yearly rise in the number of registered trials (r = 0.76, p < 0.001). Following 2017, more industry-sponsored trials were conducted (91.5% [43] vs. 60% [9], p = 0.009), fewer results were released (10.6% [5] vs. 60% [9], p = 0.001), and more trials entered phase 3 (67.4% [31] vs. 20% [2], p = 0.001). The most researched novel medications were neonatal Fc receptor inhibitors (51.2% [21]), complement inhibitors (39.0% [16]), and B cell depletors (14.6% [6]). According to the website’s data, the neonatal Fc receptor inhibitors and complement inhibitors were effective in treating myasthenia gravis patients in three trials (NCT03315130, NCT03669588, and NCT00727194). This study provides valuable insights into the profile of registered trials on novel therapies for myasthenia gravis. More clinical studies are needed in the future to prove the value of its application.

Comparative effectiveness and safety of intravenous methylprednisolone and tacrolimus monotherapy in ocular myasthenia gravis with unsatisfactory prednisone responses: a retrospective study

BackgroundOral prednisone has been recognized as the first-line therapy for the treatment of ocular myasthenia gravis (OMG). However, its long-term use is complicated by numerous adverse effects and is ineffective for some OMG patients in reaching remission. This study aimed to evaluate the effectiveness and safety of intravenous methylprednisolone (IVMP) and tacrolimus monotherapy for OMG patients with unsatisfactory responses to conventional prednisone therapy.MethodsWe retrospectively reviewed 57 OMG patients who had not achieved satisfactory improvement after prednisone therapy and thereby received IVMP or tacrolimus monotherapy for at least 6 months. Ocular symptoms were evaluated by the ocular-quantitative MG (QMG) score at each time point. A ≥ 2-point fall in ocular QMG score was defined as the cut-off point to indicate clinical improvement. Logistic regression analysis was performed to identify factors associated with the efficacy of IVMP at discharge. Adverse events were recorded.ResultsBoth IVMP and tacrolimus monotherapy demonstrated significant clinical efficacy, with no statistical differences observed at the study endpoint. The proportions of patients who reached the cut-off point for efficacy evaluation were higher in the IVMP group than in the tacrolimus group (1, 3, and 6 months: 51.7% (15/29) vs 12.0% (3/25), p = 0.002; 69.0% (20/29) vs 40.0% (10/25), p = 0.033; 69.0% (20/29) vs 46.4% (13/28), p = 0.085, respectively). Multivariate logistics analysis showed that high ocular QMG scores at baseline indicated favourable responses to IVMP treatment (OR = 1.781; 95% CI 1.066–2.975; p = 0.028). All the adverse events were transient and tolerable.ConclusionOur findings suggest that both IVMP and tacrolimus monotherapy hold promise as viable treatment options for OMG patients with unsatisfactory responses to oral prednisone. The study supports the safety and effectiveness of both therapies, with IVMP exhibiting faster improvement and favourable efficacy in patients with high ocular QMG scores.