ABSTRACT
Objective:To analyze the outcome of 15 cases with refractory systemic lupus erythematosus (SLE) treated with Belimumab, and evaluate the safety and efficacy of the therapy.Methods:A retrospective and real-world clinical research method was adopted.Fifteen children with confirmed refractory SLE and complete follow-up data were selected from the Department of Rheumatology, Beijing Children′s Hospital from April 1, 2020 to March 31, 2022.By comparing the changes of clinical symptoms, auxiliary examination results, SLE disease activity index (SLEDAI-2000) and Physician′s Global Assessment (PGA) scores as well as adverse events in different treatment periods (before treatment, 4 weeks, 8 weeks, 12 weeks, 6 months and 12 months after treatment), the safety and effectiveness of Belimumab treatment were all recorded.The counting data was expressed in percentage, the measurement data meeting the normal distribution was expressed in Mean±SD, and the two samples of measurement data were compared by t-test, P<0.05 means significant differences. Results:The ratio of male to female was 3∶2, and the onset age was (7.93±4.99) years; The basic treatment time was 4 months to 5 years and 1 month.There were 8 cases with lupus nephritis (LN), 2 cases suffering from hypocomplementemia for more than 1 year, 2 cases with central nervous system involvements, 2 cases complicated with antiphospholipid syndrome and 1 case with early-onset SLE.Of 8 LN cases, 1 case was complicated with neuropsychiatric lupus (NPLE) and distal femoral head infarction of both knees, and 3 cases were complicated with lumbar compression fractures and hip infarction.All patients were treated with regular traditional therapy to induce remission.During the maintenance period, the disease activity maintained at light to moderate levels, and it was difficult to reduce glucocorticoid.At baseline, SLEDAI-2000 score was 4-13, and PGA score was 1-2.50.Basic treatment includes glucocorticoids combined with immunosuppressants (Cyclosporine, Mycophenolate Mofetil, Leflunomide tablets) and antimalarial drugs, and Cyclophosphamide and/or Tripterygium Wilfordii were used at the same time according to the damage of target organs.The drug safety after intravenous injection of Belizumab showed that one patient in this group had respiratory tract infection symptoms 4 weeks after treatment; Another patient had a slight increase of alanine aminotransferase 8 weeks after treatment, and recovered to normal symptomatic treatment.No drug-related adverse reactions were found in the other 13 patients.After 4 weeks of treatment, the score of SLEDAI-2000 and PGA compared with the baseline level, and the difference was statistically significant (SLEDAI-2000 P=0.002; PGA P=0.006). There was no clinical recurrence.One patient with familial chilblain like lupus erythematosus showed significant improvement in rash 2 weeks after treatment, and low fever accompanied by increased rash 8 weeks after treatment; After 16 weeks of treatment, the body temperature was normal and the rash basically subsided. Conclusions:Belimumab is clinically effective in the treatment of refractory childhood SLE, with no serious adverse events reported.However, its long-term efficacy and safety need to be further studied by multi-center and long-term research with a large sample size.
ABSTRACT
Objective To evaluate the clinical efficacy and safety of endoscopic mucosal resection (EMR) assisted with magnifying chromoendoscopy in treatment of colorectal neoplasms. Methods Patients who met criteria for EMR including appropriate flat or depressed type and sessile lesions were enrolled. The association of morphology of colorectal lesions with histopathology was observed and the accuracy of estimation of invasive depth by magnifying chromoendoscopy was evaluated. Results Ninety lesions of 81 patients were reseeted by EMR (25 being sessile and 65 being flat or depressed). The histological results revealed low-grade dysplasia (LGD) in 58 lesions, high-grade dysplasia (HGD) in 20 lesions, and adenocarcinoma in 12 lesions. The average size of lesions was (1.4±0.5) cm in HGD, (1.6±0.5) cm in cancer and (1.0±0.4) cm in LGD with no significant difference (P> 0.05). It was shown that the flat and depressed lesions were more likely to be HGD or cancer as compared to sessile lesions, but with no statistical difference [41.5 % (27/65)vs. 20.0% (5/25), P= 0.084]. Moreover, the lesion with central depression was more likely to be HGD or cancer as compared to those without depressed surface [51.0% (25/49) vs. 17.1 % (7/41), P<0.01)]. The accuracy of estimating invasive depth by magnifying chromoendoscopy was 97.8% (86/90). Complete resection was confirmed histologically in 95.8% (88/90) of all lesions. Conclusions Colorectal lesions of depressed and flat types with central depression are more likely to be malignant. Estimation of invasive depth of colorectal neoplasia by magnifying chromoendoscopy in EMR treatment makes it more effective and safer.