Journal of Clinical Pediatrics

Diagnosis, treatment and future of children type 1 diabetes mellitus

LUO Feihong

Journal of Clinical Pediatrics. 2022, 40(5): 321-327. doi:10.12372/jcp.2022.22e0411

Abstract ( 706 )

HTML ( 73 )

PDF (1157KB) ( 602 )

Figures and Tables | References | Related Articles | Metrics

Type 1 diabetes mellitus (T1DM) is the major type of pediatric diabetes. According to the longitudinal epidemiology surveys in our national large metropolitans, the average annual incidence increasing rate is about three times higher that the average in the world, and the incidence increase in children under five years ranks the top, which indicates the younger trend of T1DM. The etiology and underlying mechanism of T1DM is complicated, with genetic susceptibility and environmental trigger factors as the main causes of T1DM. Early onset, long-term of disease course and poor glycemic control result in high incidence of chronic diabetic complications, and affect psychomotor development. Drug therapy, blood glucose monitoring, diabetes education, exercise and nutrition therapy are the fundamental measures for good glycemic control in pediatric T1DM. Artificial pancreas, stem cell islet differentiation and transplantation, and immune intervention may fundamentally improve the treatment and prognosis of T1DM in the future.

Diagnostic approach of neonatal diabetes mellitus

WANG Chunlin, LU Huifei

Journal of Clinical Pediatrics. 2022, 40(5): 328-333. doi:10.12372/jcp.2022.22e0114

Abstract ( 538 )

HTML ( 20 )

PDF (1391KB) ( 336 )

Figures and Tables | References | Related Articles | Metrics

Neonatal diabetes mellitus (NDM) is a rare monogenic disease with extensive heterogeneity in clinical phenotypes and genotypes on hidden clinical manifestations, which can easily delay diagnosis. In recent years, with the development of gene detection technology, more pathogenic genes have been gradually recognized. At present, more than 30 gene mutations are known as varied subtypes of NDM for the differences on clinical manifestations and outcomes. Genetic variation or abnormal methylation in chromosome 6q24 imprinting region is the commonest cause of transient neonatal diabetes mellitus (TNDM), and KATP gene mutations (KCNJ11, ABCC8) are the commonest cause of persistent neonatal diabetes mellitus (PNDM). About 90% of NDM children with KCNJ11 or ABCC8 mutations received oral sulfonylureas to maintain stable blood glucose levels. Early treatment can reverse part of the neurodevelopmental delay caused by KCNJ11 mutations, and improve the success rate of insulin conversion to sulfonylureas. Early accurate genetic diagnosis and typing are helpful for precise individualized treatment and prognosis determination. In this paper, genotype-phenotype, treatment and management of NDM were summarized, providing reference for pediatricians in early detection and diagnosis, precise treatment in clinical practice.

Advances in transplantation therapy in the treatment of type 1 diabetes in children and adolescents

CHEN Yao, WANG Xiumin

Journal of Clinical Pediatrics. 2022, 40(5): 334-338. doi:10.12372/jcp.2022.22e0253

Abstract ( 373 )

HTML ( 15 )

PDF (1222KB) ( 272 )

References | Related Articles | Metrics

Prevalence of type 1 diabetes in children and adolescents rises in decades of years. Medication could keep the serum level of glucose in a reasonable range rather than cure the disease. Transplantation, mainly islet transplantation, stem cell transplantation and pancreas transplantation, has attracted more attention for the possibility of curing type 1 diabetes. Rejection, islet inflammation and apoptosis hinder a successful transplantation, which may be relieved by carbon monoxide or regulatory T cell therapies. Most of studies about transplantation treatment for type 1 diabetes were on animals or adults, with few on children and adolescents. Furthermore, fecal microbiota transplantation could halt progression of human new-onset type 1 diabetes, which could be an adjuvant therapy in future days.

Correlation analyses between thyroids function and diabetic ketoacidosis severity in T1DM children

JIANG Mingyu, YIN Xiaoqin, LI Pin

Journal of Clinical Pediatrics. 2022, 40(5): 339-344. doi:10.12372/jcp.2022.21e1315

Abstract ( 310 )

HTML ( 13 )

PDF (1219KB) ( 192 )

Figures and Tables | References | Related Articles | Metrics

Objective To explore the correlation between serum thyroid hormone and diabetic ketoacidosis (DKA) severity of children in type 1 diabetes mellitus (T1DM). Methods We retrospectively analyzed 167 T1DM children who were diagnosed with T1DM from January 2015 to December 2020. The clinical characteristics, laboratory indicators and thyroid function status of the four groups including non DKA, mild, moderate and severe DKA in T1DM were compared, and the related factors affecting serum free triiodothyronine (FT₃） level were analyzed. Results Among 167 patients with newly diagnosed T1DM, there were 81 males and 86 females, with a median age of 6.5 (4.0-9.9) years and a median course of disease of 14 (7-28) days. There were 104 cases in non DKA group, 20 cases in mild DKA group, 16 cases in moderate DKA group and 27 cases in severe DKA group. The FT₃, total triiodothyronine (TT₃), total thyroxine (TT₄), pH and HCO^-₃ in DKA groups were significantly lower than those in non DKA group, while the levels of anion gap and blood glucose were significantly higher than those in non DKA group, and the levels of WBC and the percentage of neutrophil in severe DKA group were significantly higher than those in other groups (P<0.05). There were 92 cases of normal thyroid function, 52 cases of non-thyroidal illness syndrome (NTIS), 10 cases of subclinical hypothyroidism, 6 cases of subclinical hyperthyroidism, 3 cases of hypothyroidism and 4 cases of hyperthyroidism. The severity of diabetes with DKA is associated with different thyroid function (P<0.01). The incidence of normal thyroid function in non DKA group was higher and the incidence of NTIS was lower. The multivariate regression analysis showed that FT₃ was negatively correlated with anion gap, blood glucose (P<0.01). Conclusions FT₃, TT₃ and TT₄ in DKA groups were lower than those in non DKA group. The incidence of NTIS in DKA group was significantly higher than that in non DKA group. Determination of thyroid hormone levels can provide a reliable basis for clinical judgment of thyroid diseases and diabetes mellitus.

Phenotype and genetic characteristics of diabetes mellitus in children

ZHANG Kaichuang, LIANG Lili, ZHANG Huiwen, WANG Ruifang, YANG Yi, SUN Yuning, HAN Lianshu, YU Yongguo, QIU Wenjuan

Journal of Clinical Pediatrics. 2022, 40(5): 345-348. doi:10.12372/jcp.2022.22e0284

Abstract ( 337 )

HTML ( 10 )

PDF (1166KB) ( 209 )

Figures and Tables | References | Related Articles | Metrics

Objective Objective To determine phenotype and genetic characteristics of monogenic diabetes in children. Methods Clinical manifestations and laboratory data at initial diagnosis of 76 children with diabetes admitted to our department from August 2020 to December 2021 were retrospectively analyzed. Whole exome sequencing was performed in 21 patients with suspected monogenic diabetes. Results A total of seven cases of monogenic diabetes were identified by whole exome sequencing (WES), all of which were maturity-onset diabetes of the young (MODY). Three cases of MODY2 were caused by GCK variants, and three cases of MODY3 were caused by HNF1A variants and one case of MODY10 caused by INS variant. Two novel variants were identified, including c.1124T>G (p.V375G) in GCK and c.110A>T (p.E37V) in INS. Five of the 7 patients (5/7) with MODY had no typical symptoms of diabetes and were admitted to the hospital due to incidentally elevated glucose level. The level of blood glucose and glycosylated hemoglobin in the MODY group (n=7) were lower than those in the T1DM group (n=62) (P<0.05), and showed no significant difference compared with those of T2DM group (n=7). The levels of insulin and C-peptide in the MODY group were higher than those in the T1DM group (P<0.05), and were significantly lower than those in the T2DM group (P<0.05). In addition, a T1DM child with retarded mental development received an additional diagnosis of 18p deletion syndrome by WES, and another T2DM child with sustained elevated liver aminotransferase received an additional diagnosis of Wilson Disease by WES. Conclusions MODY is the common type of monogenic diabetes. This study expands the mutation spectrum of MODY. When diabetes patient present with extra-pancreatic manifestations, the combination of other genetic disorders should be considered.

Analysis of genetic test results in 186 cases with short stature

WANG Lili, WU Haiying, XIE Rongrong, WANG Fengyun, CHEN Ting, CHEN Xiuli, SUN Hui, WANG Xiaoyan, ZHANG Dandan, CHEN Linqi

Journal of Clinical Pediatrics. 2022, 40(5): 349-354. doi:10.12372/jcp.2022.21e1673

Abstract ( 393 )

HTML ( 8 )

PDF (1196KB) ( 230 )

Figures and Tables | References | Related Articles | Metrics

Objective To explore the genetic etiology of short stature in children. Methods This study selected children with short stature who were treated for growth disorder from January 2017 to October 2020. The whole exome sequencing (WES) was performed to identify potential genetic etiologies, and the possible copy number variants of chromosome fragments were further improved by chromosomal microarray analysis (CMA), and the clinical phenotypic differences between gene test positive and negative groups were compared. Results A total of 186 children with short stature were included, with a median age of 7.3 (5.1-9.1) years, including 103 boys and 83 girls. A total of 69 cases of positive results were detected, with a positive rate of 37.1%. Fifty-four were tested by WES and 15 by CMA. Binary logistic regression analysis showed facial dysmorphism or skeletal abnormalities were predictors of positive gene detection results in children with short stature (all P<0.05). Conclusion Whole exome sequencing is an effective technique to detect the genetic etiology of short stature in children, and Patients with facial dysmorphism and/or skeletal abnormalities are more likely to have a known genetic etiology.

Clinical phenotypic and genetic analysis of three patients with Coffin-Siris syndrome

WU Chenchen, ZHANG Huiwen

Journal of Clinical Pediatrics. 2022, 40(5): 355-360. doi:10.12372/jcp.2022.21e1660

Abstract ( 668 )

HTML ( 9 )

PDF (1604KB) ( 416 )

Figures and Tables | References | Related Articles | Metrics

Objective To perform the phenotypic analysis and identify genetic variation locus in three patients with Coffin-Siris Syndrome (CSS). Methods Chromosome microarray analysis (CMA), trio-whole exome sequencing analysis (trio-WES), trio-whole genome sequencing analysis (trio-WGS), and Sanger sequencing were used for genetic diagnosis. The peripheral blood example of patient 3 was collected to establish an immortalized lymphocyte line, and the ARID1B protein expression was detected by Western Bolt (WB). Results All three patients visited the hospital for developmental retardation, and they all had facial dysmorphism. Patient 1 had intrauterine growth restriction, patient 2 was accompanied by recurrent upper respiratory tract infection, patient 3 had intellectual disability and abnormal hand manifestations. The CMA results of three patients were negative. The pathogenic gene loci of patient 1 and patient 2 were obtained by trio-WES analysis. Patient 1 has a de novo heterozygous splicing site mutation of c.363-3C>G in SMARCB1. There was a de novo heterozygous splicing site mutation of c.3550+1(IVS13) G>A in the ARID1B gene of patient 2. No pathogenic variations were identified in patient 3 by trio-WES. However, trio-WGS analysis revealed a de novo heterozygous exon 11 deletion in the ARID1B gene of patient 3. The three de novo mutations have not been reported previously. WB showed a significant decrease of ARID1B protein level in immortalized lymphocytes from patient 3. Conclusions The clinical manifestations of Coffin-Siris syndrome are diverse, and featured mainly as developmental retardation. The application of multiple genetic testing methods can help to confirm diagnosis of the Coffin-Siris syndrome.

Genetic and phenotypic analysis of five patients with GNAO1 gene related disorders

YANG Haibo, WEN Yongxin, ZHANG Qingping, BAO Xinhua

Journal of Clinical Pediatrics. 2022, 40(5): 361-365. doi:10.12372/jcp.2022.21e0985

Abstract ( 448 )

HTML ( 7 )

PDF (1265KB) ( 129 )

Figures and Tables | References | Related Articles | Metrics

Objective To investigate the genotypes and phenotypes features of patients with GNAO1 gene variants. Methods Genetic features, clinical manifestations and therapeutic reaction of five patients with GNAO1 gene variants were retrospectively analyzed. Results A total of 5 patients with GNAO1 gene variants were detected, including 4 patients with missense variants and 1 with splice site variation. Three variants were reported previously and two were novel variants. Two children presented with early-onset infantile epileptic encephalopathy with varying degrees of dyskinesia; three children presented with extrapyramidal symptoms (two with dystonia and one with choreoathetosis) and no seizures for the time being; profound developmental delay was observed in all five patients. Two patients with epilepsy did not response to multiple antiepileptic drugs. Two patients with dystonia underwent deep brain stimulation (DBS) therapy. The BFMDRS score decreased by 32.36% one month after surgery in one patient. However, no significant improvement was found in the other patient, and the BFMDRS score decreased by only 7.79% at 12 months postoperatively. One patient with choreoathetosis intended to accept DBS treatment later. Conclusions The majority of GNAO1 gene variants were missense mutation. The clinical manifestations of patients with GNAO1 gene variants were various, mainly presenting with developmental delay, movement disorders, and/or epilepsy. The epilepsy and the extrapyramidal symptoms responded poorly to the medical therapy, and DBS treatment could alleviate the dyskinesia in some patients.

Ataxia telangiectasia-like disorder 1 caused by mutation of MRE11 gene: a case report

JIA Qianfang, CUI Qingyang, ZHOU Fujun

Journal of Clinical Pediatrics. 2022, 40(5): 366-369. doi:10.12372/jcp.2022.21e0599

Abstract ( 329 )

HTML ( 13 )

PDF (1747KB) ( 231 )

Figures and Tables | References | Related Articles | Metrics

To improve the understanding of clinical phenotype and genotype of ataxia telangiectasia-like disorder 1 (ATLD1). Clinical data of a child with ATLD1 were retrospectively analyzed and related literatures were reviewed. An 11-year-old female patient was admitted to the hospital for regression in motor and intelligence development for more than two years. Clinical features include positive findings in hand rotation test, Romberg's sign, and finger-nose test, weakly positive findings in heel-knee-tibia test and left ankle clonus, other findings are grade IV muscle strength in all four limbs, poor coordination, and body swaying from side to side during walking. Two variants in MRE11 gene, c.728G>C and c.68G>C, were found by whole genome sequencing, which have not been reported. Diagnosis of ATLD was made, and rehabilitation with electronic biofeedback, low-frequency pulses, joint release training, traction, occupational therapy, hand function training, and exercise classes were ineffective. There are many causes of motor developmental delay and intellegence delay in children. Timely genetic testing helps early confirmed diagnosis. Guided prenatal counseling and diagnosis helps to avoid over-treatment and birth defects.

Comparison of common complications of transcatheter and surgical closure of perimembranous ventricular septal defects

ZHAO Xiaopei, ZHANG Yongwei, XIAO Tingting

Journal of Clinical Pediatrics. 2022, 40(5): 370-375. doi:10.12372/jcp.2022.21e1088

Abstract ( 291 )

HTML ( 5 )

PDF (1188KB) ( 215 )

Figures and Tables | References | Related Articles | Metrics

Objective To analyze the common complications of the transcatheter and the surgical approach to correct perimembranous ventricular septal defect (pmVSD) in children, and to provide a basis for clinically individualized optimal treatment plan. Methods The clinical data of hospitalized children diagnosed with pmVSD between June 2014 and April 2020 were retrospectively analyzed. Based on the final surgical approach, 284 patients were divided into interventional and repair groups. Common postoperative complications and follow-up data between these two groups were analyzed. Results Of the 499 children finally enrolled. 284 cases were in the interventional group, 130 males and 154 females, with a median age of 42.0 (33.0-56.0) months, and 215 cases were in the repair group, 100 males and 115 females, with a median age of 40.0 (30.0-60.0) months. There was no statistically significant difference in the incidence of residual shunt, aortic valve, mitral valve, and tricuspid regurgitation within 1 week after surgery between the two groups (P>0.05). The incidence of new arrhythmias and complete and incomplete right bundle branch block within 1 week postoperatively was higher in the repair group than in the intervention group, and the difference was statistically significant (P<0.05). At the end of follow-up, residual shunt, valve regurgitation and conduction block were significantly improved in both groups, with no statistically significant differences between the two groups (P>0.05). Conclusion Interventional occlusion and surgical repair are equally effective in treating perimembranous ventricular septal defects in children, and interventional treatment is less invasive and has fewer complications.

Clinical application of a pediatric nutrition screening-assessment tool in gastroenterology ward

WANG Zhixin, LU Lina, WANG Jinling, YAN Weihui, CAI Wei, WANG Ying

Journal of Clinical Pediatrics. 2022, 40(5): 376-381. doi:10.12372/jcp.2022.21e1763

Abstract ( 395 )

HTML ( 11 )

PDF (1244KB) ( 551 )

Figures and Tables | References | Related Articles | Metrics

Objectives To recognize the nutritional status of hospitalized children by the pediatric nutrition screening-assessment tool, to analyze the differences in clinical outcomes and biochemical indicators under different nutritional status, and to provide evidence for reasonable nutritional intervention. Methods A pediatric nutrition screening-assessment tool, named as Pediatric Nutrition Screening APP, developed by Working group of Pediatrics, Chinese Society of Parental and Enteral Nutrition was used to nutritional screening and assessment of children admitted to the Division of Gastroenterology and Nutrition Xinhua Hospital from March 2018 to October 2020, and to evaluate children at risk of malnutrition and analyze the difference in clinical outcomes and biochemical indicators under different nutritional status. Results A total of 624 children were enrolled, including 368 males and 256 females. There were 158 children in the 0-1 year old group, 95 children in the 1-3 years old group, and 371 children in elder than 3 years old group. Nutrition screening showed that the incidence of malnutrition risk was 56.25%, and the risk of malnutrition was 89.24%, the highest in the 0-1 year old group. Grouped by disease, children with short bowel syndrome have the highest risk of malnutrition at 90.05%, followed by acute pancreatitis at 88.89%, and chronic diarrhea at75.00%. Compared with the non-malnutrition risk group, the children in the malnutrition risk group had significantly longer duration of hospitalization, more hospitalization expenses, higher infection rates, and significantly reduced total protein, albumin, hemoglobin, and 25-(OH)-Vitamin D levels (all P<0.05). Nutrition assessment indicated that the incidence of moderate malnutrition and severe malnutrition were 35.89% and 16.37%, respectively. The incidence of moderate malnutrition and severe malnutrition in the 0-1 year old group were the highest, 34.61% and 36.54%, respectively (both P<0.05). In the normal group, moderate malnutrition group, and severe malnutrition group, the duration of hospitalization, hospitalization expenses, and infection rate stepped up, and the levels of total protein, albumin levels stepped down (all P<0.05). Conclusions The risk of malnutrition and the incidence of moderate and severe malnutrition were higher in children in gastroenterology ward. Children under small age had higher risk of malnutrition and incidence of moderate to severe malnutrition. Under malnutrition status, children had longer hospitalization, increased hospitalization expenses, and higher infection rate, and lower levels of total protein, albumin, and 25-hydroxyvitamin D.

Structure analysis of the gut microbiota in asthmatic children with different control levels

ZOU Yutong, HUANG Linsheng, ZHONG Hui, YANG Rong, GU Li

Journal of Clinical Pediatrics. 2022, 40(5): 382-387. doi:10.12372/jcp.2022.21e1496

Abstract ( 269 )

HTML ( 7 )

PDF (1385KB) ( 105 )

Figures and Tables | References | Related Articles | Metrics

Objectives To observe the difference of gut microbiota in children with asthma at different control levels. Methods Thirty children aged 10 to 12 with asthma were selected from January 2020 to December 2020 and divided into well-controlled group (16 patients) and non-control / partial control group (14 patients) according to the grading standard of asthma control level. Twelve healthy children were selected as the control group. Fresh fecal samples were collected from three group for the detection of 16S rRNA of gut microbiota. Results A total of 30 children with asthma were included. There were 16 cases in the well-controlled group, 10 males and 6 females, aged (12.19±1.11) years; 14 cases in the uncontrolled/partially controlled group, 8 males and 6 females, aged (12.92±1.59) years; and 12 cases in the control group, 6 males and 6 females, aged (13.17±1.03) years. The differences in gender and age among the three groups were not statistically significant (P>0.05). The duration of treatment was (1.22±0.18) years in the well-controlled group and (1.24±0.15) years in the uncontrolled/partially controlled group, with no statistically significant differences (P>0.05). The difference in the absolute value of FEF₅₀ among the three groups was statistically significant (P<0.05). The differences in Alpha diversity index (4 indices) among the three groups were not statistically significant (P>0.05). The relative abundance of Veillonella species in the uncontrolled/partially controlled group was 0.06 (0.01-0.25)%, 0.53 (0.19-0.92)% in the well-controlled group and 0.17 (0.09-0.82)% in the control group, with statistically significant differences among the three groups (P<0.05). A two-by-two comparison revealed that the abundance of Veillonella was significantly lower in the uncontrolled/partially controlled group compared to the well-controlled group as well as the control group, and the difference was statistically significant (P<0.05). ROC curve analysis showed that the area under the curve for the prediction of Veillonella abundance on the level of asthma control was 0.82 (95% CI: 0.66 to 0.98). Conclusions The abundance of Veillonella in the intestinal flora of poorly controlled asthmatic children was significantly reduced, which may be a guideline for the assessment of asthma control level as well as adjuvant therapy.

Clinical research on cellular immune reconstitution after allogeneic hematopoietic stem cell transplantation in children

SONG Na, SUN Ming, QI Shanshan, WANG Zhuo, YANG Li, LU Wenjie, WU Min, XIA Wei, CHEN Yan, XIONG Hao

Journal of Clinical Pediatrics. 2022, 40(5): 388-394. doi:10.12372/jcp.2022.21e1384

Abstract ( 284 )

HTML ( 5 )

PDF (1279KB) ( 169 )

Figures and Tables | References | Related Articles | Metrics

Objective To investigate the clinical characteristics of immune reconstitution after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children. Methods Children with complete clinical data who received allo-HSCT in the department of hematology and oncology from July 2019 to October 2020 were selected as the study subjects. The absolute count of peripheral blood lymphocyte subsets (CD3⁺ T cells, CD4⁺ T cells, CD8⁺ T cells, CD16⁺ CD56⁺ NK cells, CD19⁺ B cells, CD4⁺ CD25⁺ Treg cells) was dynamically monitored on day 0, 14, 21, 28, 60 and 100 after transplantation. The children were divided into non-aGVHD group and aGVHD group according to the presence or absence of acute graft-versus-host disease (aGVHD), and the differences of lymphocyte subsets between the two groups were compared. Results A total of 75 children received allo-HSCT, and 42 of them with complete clinical data after 100 days of transplantation were included in this study. There were 20 boys and 22 girls with a median age of 5.4 (2.8~9.3) years. There were statistically significant differences in the levels of CD3⁺ T cells, CD4⁺ T cells, CD8⁺ T cells, CD16⁺ CD56⁺ NK cells, CD19⁺B cells, CD4⁺CD25⁺ Treg cells and CD4⁺/CD8⁺ ratio at different time points before and after transplantation (P<0.05). CD16⁺CD56⁺ NK cells recovered the earliest and reached the level of pre-transplantation at 21 days post-transplantation. CD3⁺ T cells almost reached the pre-transplantation level at 60 days after transplantation, while CD8⁺ T cells exceeded the pre-transplantation level at 60 days after transplantation. The aGVHD occurred in 16 of 42 children, and the median time of aGVHD occurrence was 20.0 (6.0-36.0) days. At 28d and 60d after transplantation, the absolute count of CD16⁺ CD56⁺ NK cells in aGVHD group was lower than that in non-aGVHD group, the difference was statistically significant (P<0.05). Conclusions CD16⁺ CD56⁺ NK cells were the earliest lymphocyte subsets reconstructed after allo-HSCT in children. NK cell reconstitution is closely related to the occurrence of aGVHD after allo-PBSCT, which may be used as an effective predictor of prognosis in patients with aGVHD.

Drug treatment progress on juvenile dermatomyositis

SUN Yanru, LIU Li

Journal of Clinical Pediatrics. 2022, 40(5): 395-400. doi:10.12372/jcp.2022.21e1485

Abstract ( 550 )

HTML ( 15 )

PDF (1240KB) ( 220 )

Figures and Tables | References | Related Articles | Metrics

Juvenile dermatomyositis (JDM) is an autoimmune inflammatory myopathy in childhood that can affect the whole body, mainly the skin and proximal muscles. It can also affect vital organs (such as: lungs, joints, and gastrointestinal tract etc), often accompanied by the presence of specific antibodies. At present, the treatment of JDM is very difficult. The first-line drugs are glucocorticoids and methotrexate. For patients with poor therapeutic effects, some second-line or third-line drugs may play a supportive role. In refractory patients, intravenous injection of immunoglobulin or cyclophosphamide may be effective, while biologics and small molecule targeted drugs are gradually used in clinical practice. In addition to glucocorticoids, the choice of drugs often requires a combination of clinical symptoms, serum specific antibodies, pathological results and response to initial treatment, and continuous adjustments during the treatment process. The purpose of this review is to provide an overview the drugs currently used in the treatment of JDM, observation of therapeutic effects and strategies for drug selection, and to provide ideas for clinical treatment.

Table of Content