Treatment Of Juvenile Myasthenia Gravis Research Articles

Treatment of juvenile myasthenia gravis with tacrolimus: A cohort study.

We investigated the proper timing, efficacy and safety of tacrolimus for juvenile myasthenia gravis (JMG). We conducted a retrospective cohort study for JMG patients treated with tacrolimus at Xiangya Hospital, Central South University, Changsha, China from 2010 to 2023. The clinical information of patients with a follow-up of more than 1 year was collected. Comparisons of clinical features between groups of patients who achieved therapeutic goal and those who did not achieve therapeutic goal as well as between groups of patients treated with tacrolimus within or after 1 year from JMG onset was carried out. Forty-three patients were enrolled, of whom 28 achieved therapeutic goal. Tacrolimus reduced glucocorticoids (GC) dosages for the 28 cases and 15 cases discontinued GC completely. Generalized myasthenia gravis (GMG) subtype had an association with a group of patients who achieved therapeutic goal (p = 0.001). Median duration from JMG onset to tacrolimus use was 10.50 months for those who achieved therapeutic goal and 36.00 months for those who did not achieve therapeutic goal (p = 0.010). The median Myasthenia Gravis Activities of Daily Living (MG-ADL) score improved significantly (p = 0.003). The initiation of tacrolimus within 1 year of JMG onset showed an association with achievement of therapeutic goal (p = 0.026). GMG subtype showed an association with a group of patients who received tacrolimus within 1 year (p = <0.001). Tacrolimus side effects were tolerable. The provision of tacrolimus within 1 year of JMG onset is effective and safe.

Clinical Characteristics and Treatment of Juvenile Myasthenia Gravis-A Single-Center Experience.

Juvenile myasthenia gravis (MG) is a rare autoimmune neuromuscular disease, often treated with anticholinesterases, corticosteroids, and immunosuppressants. However, optimal treatment durations remain unclear. This study investigated the clinical characteristics and treatment of juvenile MG, including medication duration. The administration period for all drugs, immunosuppressants, and prednisolone at doses greater than 0.35 mg/kg daily was extracted retrospectively from medical records. Nineteen participants (8 boys, 11 girls) aged 8 months to 14 years (median, 2.5 years) at onset were identified. Fourteen patients (73.7%) had ocular MG and five (26.3%) had generalized MG. Drug treatment was conducted in 18 cases; however, 7 patients did not complete the treatment. Among the patients who completed drug treatment, the duration of treatment ranged from 11 to 100 months (median, 47 months). In the six patients treated with continuous administration of prednisolone or immunosuppressants, the treatment duration ranged from 33 to 99 months (median, 56 months). No severe adverse effects requiring hospitalization were reported. The patients treated with prednisolone or immunosuppressants required at least 33 months of treatment. These results will help develop protocols for juvenile MG treatment.

Rituximab Therapy in the Treatment of Juvenile Myasthenia Gravis: The French Experience.

Corticosteroids are the first-line immunosuppressants in the management of juvenile myasthenia gravis despite their adverse effects. The place of new immunosuppressive therapies is not clearly defined by the last international consensus held in March 2019 due to the lack of clinical trials. The aim of this study is to describe the use of rituximab and its efficacy and safety in 8 main pediatric centers of the French neuromuscular reference network to propose a new place in the therapeutic strategy of juvenile myasthenia gravis. We conducted a retrospective multicenter study from January 1, 2009, to April 30, 2020, including a large cohort of children with myasthenia gravis in 8 main French pediatric reference centers of the FILNEMUS network. The type of myasthenia, different lines of immunosuppressive treatment, and clinical course of the patients were collected. To evaluate the efficacy of rituximab, we studied the clinical course of patients on immunosuppressive therapy. Outcome was defined as the clinical and therapeutic status of patients at the last visit: stable without immunosuppressants, stable with immunosuppressants, or unstable. We included 74 patients: 18 children with ocular form and 56 children with generalized form. Of the 37 patients who required immunosuppressive therapy, 27 were treated with rituximab. Patients treated with rituximab had a better outcome than patients treated with conventional immunosuppressants (p = 0.006). The use of rituximab as a first-line immunosuppressant showed a better efficacy with a discontinuation of immunosuppressants in 75% of patients (vs 25%, p = 0.04) and results in cortisone sparing (42% vs 92%, p = 0.03) compared with rituximab treatment as a second- or third-line immunosuppression. Rituximab was well tolerated; no adverse effect was observed. The use of rituximab has increased in France over the last 10 years as a first-line immunosuppressant. This study suggests good tolerability and efficacy of rituximab in juvenile myasthenia gravis. Early use appears to improve outcomes and facilitate cortisone sparing in antibody-positive generalized juvenile myasthenia. This study provides Class III evidence that for children with MG, rituximab is effective and well tolerated.

Surgical results of video-assisted thoracoscopic thymectomy for treatment of Juvenile Myasthenia Gravis

Juvenile myasthenia gravis (JMG) is an autoimmune disease affecting the neuromuscular junction that appears before 19 years of age with varying degrees of weakness of different muscle groups. The main treatment is pharmacological, but thymectomy has also demonstrated to improve remission rates. To describe the clinical characteristics and postoperative course of pediatric patients with JMG who underwent video-assisted thoracoscopic (VATS) thymectomy. Clinical Serie: Six pa tients who underwent VATS thymectomy between March 2011 and June 2019. The age range at diag nosis was between 2 and 14 years and the average age at surgery was 7 years. All patients were under treatment with pyridostigmine bromide associated with immunosuppression with corticosteroids before surgery. The interval between diagnosis and thymectomy was 21.5 months on average. VATS was performed by left approach, and there was no perioperative morbidity or mortality. The average hospital stay was 2 days. Three patients remain with no symptoms and without corticotherapy. Two patients were on corticosteroids, but in smaller doses than previous to surgery. One patient presented a crisis requiring hospitalization and ventilatory support during follow-up. VATS thy mectomy is part of the treatment for JMG. In this series, it appears as a safe approach and its results were favorable.

Thoracoscopic thymectomy for juvenile myasthenia gravis

PurposeA randomized controlled trial of thymectomy in myasthenia gravis demonstrated improved clinical outcomes in adults, but data surrounding juvenile cases, especially those treated with minimally invasive approaches, are limited. Here, we review our experience with thoracoscopic thymectomy for juvenile myasthenia gravis (JMG) in the largest cohort to date.MethodsAll cases of thymectomy for JMG in a single tertiary referral center between 2007 and 2018 were reviewed (N = 50). Patients underwent left thoracoscopic approach with extended dissection and without use of monopolar energy. Demographics, diagnostic criteria, and clinical classification, as well as surgical data were collected. Clinical status and medications were reviewed in follow-up.ResultsThe mean age at surgery was 10.5 ± 0.8 years. Ocular disease and generalized disease each comprised half of the cohort. No patients suffered complications or increased risk of morbidity or mortality with thymectomy. At any interval of follow-up through 3.5 years, 49.8% of patients were improved compared to their pre-operative presentation, and there was a significant trend towards decreased steroid use.ConclusionThoracoscopic thymectomy is a safe treatment for juvenile myasthenia gravis in pediatric patients over a wide range of ages, body masses, and symptoms. Our experience adds evidence that pediatric patients likely benefit from thymectomy with improved clinical status and reduced medications.

The role of thymectomy in the treatment of juvenile myasthenia gravis: a systematic review.

The role of thymectomy in the treatment of juvenile myasthenia gravis (JMG) is poorly defined. The objective of this systematic review was to evaluate the effect of thymectomy on survival, disease severity, and peri-operative complications for patients with JMG. A search of MEDLINE, EMBASE, and the Cochrane Library (1/1/2000-3/1/2016) identified all English language, human studies of thymectomy for JMG. The population was patients with JMG age≤18years who underwent thymectomy (comparator group was unexposed to thymectomy). Outcomes included survival, disease severity, and post-operative complications. Data extraction was performed by independent reviewers. Sixteen retrospective studies included 1131 participants with JMG and 488 (43%) underwent thymectomy. Post-operative improvement in JMG severity occurred for 77% (n=376/488). Comparisons of thymectomy to non-operative management were mixed. Post-operative complications were poorly recorded. Power to compare surgical approaches was limited. Outcomes specific to antibodies, surgical pathology findings, severity of JMG, and timing of thymectomy were sparse. Existing data regarding thymectomy for JMG are limited and entirely retrospective. The majority of patients who underwent thymectomy had improvement in disease severity and post-operative complications were rare. Prospective, multicenter study of thymectomy for JMG is warranted.

Results of surgical treatment for juvenile myasthenia gravis

IntroductionRadical or extended thymectomy is an effective treatment for myasthenia gravis in the adult population. There are few reports to demonstrate the effectiveness of this treatment in patients with juvenile myasthenia gravis. ObjectiveThe main objective of this study was to show that extended transsternal thymectomy is a valid option for treating this disease in paediatric patients. ResultsTwenty-three patients with juvenile myasthenia gravis underwent this surgical treatment in the period between April 2003 and April 2014; mean age was 12.13 years and the sample was predominantly female. The main indication for surgery, in 22 patients, was the generalised form of the disease (Osserman stage II) together with no response to 6 months of medical treatment. The histological diagnosis was thymic hyperplasia in 22 patients and thymoma in one patient. There were no deaths and no major complications in the postoperative period. After a mean follow-up period of 58.87 months, 22 patients are taking no medication or need less medication to manage myasthenic symptoms. ConclusionsExtended (radical) transsternal thymectomy is a safe and effective surgical treatment for juvenile myasthenia gravis.

Resultados del tratamiento quirúrgico en la miastenia gravis juvenil

IntroductionRadical or extended thymectomy is an effective treatment for myasthenia gravis in the adult population. There are few reports to demonstrate the effectiveness of this treatment in patients with juvenile myasthenia gravis. ObjectiveThe main objective of this study was to show that extended transsternal thymectomy is a valid option for treating this disease in paediatric patients. ResultsTwenty-three patients with juvenile myasthenia gravis underwent this surgical treatment in the period between April 2003 and April 2014; mean age was 12.13 years and the sample was predominantly female. The main indication for surgery, in 22 patients, was the generalised form of the disease (Osserman stage II) together with no response to 6 months of medical treatment. The histological diagnosis was thymic hyperplasia in 22 patients and thymoma in one patient. There were no deaths and no major complications in the postoperative period. After a mean follow-up period of 58.87 months, 22 patients are taking no medication or need less medication to manage myasthenic symptoms. ConclusionsExtended (radical) transsternal thymectomy is a safe and effective surgical treatment for juvenile myasthenia gravis.

Efficacy and safety of thoracoscopic thymectomy in the treatment of juvenile myasthenia gravis

Thymectomy is a well-established treatment for generalized myasthenia gravis in adults, but predictors of long-term efficacy and the optimum timing for intervention in juvenile myasthenia remain controversial. To review the preoperative presentation, surgical experience, and long-term neuromuscular follow-up in patients undergoing thoracoscopic thymectomy in a single institution. A retrospective chart review of all patients undergoing thoracoscopic thymectomy for myasthenia gravis at a tertiary referral center between 2000 and 2010 and compared to an historical cohort of trans-sternal thymectomies performed between 1970 and 1995. Age at diagnosis, presurgical medications and hospitalizations, preoperative chest imaging, presence of acetylcholinesterase antibodies, Osserman Stage, time to operative intervention, length of follow-up, DeFillipi remission scale, as well as operative and post-operative data (length of surgery, blood loss, need for chest tube, length of intubation, length of hospital stay, pathology, and complications) were recorded. Fifteen patients undergoing thoracoscopic thymectomy were identified with a mean age of 11.3years at time of diagnosis and average treatment duration of 12.5months prior to operative intervention. Of these patients, most presented with Osserman Stage IIB (8) or III (5) disease. Two patients presented with Osserman Stage IIa disease. There were no reported complications, no conversions to an open approach, and an average length of stay of 2.6days. Average length of follow-up was 37.5months, available on 13 of 15 patients. Nine of 13 (69%) were improved (DeFillippi Class 2 or 3) at 1-month follow-up, however, the pattern of remission waxed and waned, with only 50% reporting improvement at 1year, 86% at 2years and 75% at 3years. Only one patient was totally off medication. No patients required postoperative hospitalization for respiratory crisis. Thoracoscopic thymectomy offers a safe approach to thymic resection in children with JMG with little associated morbidity and a short hospital stay, but should not be considered curative. Rather it appears to make generalized JMG more amenable to long-term medical management.

Clinical Outcome of Juvenile Myasthenia Gravis After Extended Transsternal Thymectomy in a Chinese Cohort

The role of surgical treatment for juvenile myasthenia gravis (MG) remains unclear. Here, we performed a retrospective study to evaluate the predictors of clinical outcome of juvenile MG treated with extended transsternal thymectomy. A total of 141 consecutive juvenile MG patients underwent extended transsternal thymectomy at an academic hospital over a 20-year period were reviewed. Thymectomy was performed in patients resistant to pyridostigmine therapy, with generalized symptoms or ocular MG with partial response to pyridostigmine for more than 2 years. Variables potentially affecting responses to extended transsternal thymectomy were evaluated using Kaplan-Meier analysis and Cox regression modeling. Complete stable remission (CSR) is defined as asymptomatic without medication for more than 12 months. There were 96 patients with ocular MG and 45 generalized MG, the median age at disease onset was 6 years and that at operation was 12 years. Among 135 patients with complete postoperative follow-up, 34 (25.2%) achieved CSR, 28 (20.7%) experienced pharmacologic remission, 61 (45.2%) improved, 5 (3.7%) remained stable, and 7 (5.2%) deteriorated. The results indicated the disease-onset age greater than 6 years and age at operation greater than 12 years were both positively associated with CSR responses. Postoperative steroid treatments in ocular MG and preoperative disease duration in generalized MG (>12 months) were negatively associated with CSR responses. Extended transsternal thymectomy for Chinese juvenile MG patients has an efficacy comparable with reports from other ethnicities. Juvenile patients with disease-onset age greater than 6 years, age at operation greater than 12 years, and shorter disease duration of generalized MG are associated with favorable clinical outcomes.

Thymectomy for inducing remission in juvenile myasthenia gravis

The aim of this study was to determine the efficacy of thymectomy to induce remission in juvenile myasthenia gravis. A retrospective review of all patients undergoing a thymectomy for the treatment of juvenile myasthenia gravis was performed at a single tertiary referral centre between 1997 and 2009 (N = 8). All cases were moderate to severe cases (Osserman stage ≥ 2a). All operations were open. Median follow-up was 18 months (range 1-77). Postoperative progress was assessed using the De Filippi classification of remission. Only two minor complications were reported. Mean operative time was 120 min (range 80-290 min). Mean postoperative stay was 17 days (range 3-52 days). Remission was observed in 5 of 8 patients (62%) at last known follow up. Spontaneous remission rates for myasthenia gravis are quoted to be between 20 and 29% while remission rates following thymectomy are 29-68% at 3 years. Although not all of our patients have had 3 years of follow up-the remission rate of 62% demonstrated by this study is encouraging. If reproducible over a larger series this provides evidence of the efficacy of thymectomy for treating juvenile myasthenia gravis in selected patients.

Long-term follow-up after thymectomy for myasthenia gravis: thoracoscopic vs open

Purpose The aim of this study was to determine if patients are in remission or weaning off medication after thymectomy for myasthenia gravis (MG) and to examine the thoracoscopic versus open approaches. Methods A retrospective review of all patients who underwent thymectomy for MG at a tertiary referral center between 1992 and 2004 (N = 14). Six patients (42.9%) underwent thoracoscopic resection. Eight patients underwent open resection; 5 (35.7%) had median sternotomy and 3 (21.4%) by transcervical approaches. Follow-up was obtained in 12 (85.7%) of 14 patients by both chart review and telephone. The mean follow-up was 43.0 months (range, 4-111 months). Statistical significance was determined by Student's t test or Fisher's Exact Test. Results The thoracoscopic group had a mean operating time of 138.8 minutes compared with 139.8 minutes in the open group ( P = .9). The thoracoscopic group had a mean estimated blood loss of 7.5 mL compared with 52.5 mL in the open group ( P = .02). The mean length of stay for the thoracoscopic group was 1.5 days (range, 1-2 days) and was 10.6 days (range, 3-41 days) in the open group ( P = .13). Three (60%) of 5 patients were entirely off medication in the thoracoscopic group at the time of follow-up compared with 3 (50%) of 6 patients in the open group ( P = 1.0). In the thoracoscopic group, 5 (83.3%) of 6 were in class 1 to 3 of the DeFilippi classification (complete remission or improved with decreased medication requirements). One patient had no change in symptoms (class 4). In the open group, 5 (83.3%) of 6 were classified as DeFilippi 1 to 3 at the time of follow-up, and one patient had worsening symptoms (class 5). Conclusions Both thoracoscopic and open approaches to thymectomy in patients with MG are effective, with more than 80% of patients in both groups in remission or with improvement at the time of follow-up. The thoracoscopic group has the added benefits of decreased estimated blood loss, decreased length of hospital stay, and improved cosmesis. We advocate the thoracoscopic approach for thymectomy in the treatment of juvenile MG.

Thoracoscopic thymectomy in juvenile myasthenia gravis

Background: Although transsternal thymectomy is an effective method in the treatment of juvenile myasthenia gravis (JMG) it is traumatic in pediatric patients. Thoracoscopic thymectomy offers an effective and less traumatic approach with respect to cosmesis and postoperative recovery. Methods: A retrospective analysis of 6 consecutive patients treated with thoracoscopic thymectomy was performed. Perioperative parameters and cost analysis were compared with those of 6 consecutive open procedures performed before the study. Results: Thoracoscopic thymectomy can be performed in patients as young as 1.6 years. There was no conversion to open procedure and no perioperative morbidity and mortality. The length of operating time and the surgical cost of thoracoscopic procedure were not significantly different from those of open procedure. The length of hospitalization, however, was significantly shorter with thoracoscopic procedure, and hence the overall cost was significantly reduced (P <.05). An intermediate follow-up shows that outcome after thoracoscopic procedure is equally as effective as open procedure. Conclusions: Thoracoscopic thymectomy offers an equally effective but cosmetically more acceptable approach than sternotomy. It has a quicker recovery period and appears to be a less costly alternative to transsternal thymectomy. J Pediatr Surg 35:768-770. Copyright © 2000 by W.B. Saunders Company.

Immunosuppressive treatment for juvenile myasthenia gravis.

The result of immunosuppressive treatment in 20 cases of generalized myasthenia gravis with onset before age 16 were analysed. The patients age at the beginning of immunosuppressive treatment ranged from 10 to 22 years. Thirteen patients suffered from a severe form of myasthenia gravis with respiratory attacks, the remaining 7 demonstrated a moderately severe form with bulbar symptoms dominating the clinical picture. Indication for immunosuppressive treatment was lack of or only slight improvement after thymectomy. Prednisone was used in 17 cases, high dosage of methylprednisolone in 8 (in one case twice), azathioprine in 11 and cyclophosphamide in 10. Best results were obtained with cyclophosphamide since significant improvement was recorded in 14/20 of patients. Corticosteroids, i.e. prednisone and high-dose methylprednisolone proved to be of limited value.

143. Surgical treatment for juvenile myasthenia gravis

143. Surgical treatment for juvenile myasthenia gravis