Multifocal acquired demyelinating sensory and motor neuropathy (MADSAM) is a variant of chronic inflammatory demyelinating polyneuropathy (CIDP). It presents as a chronic, asymmetrical sensory-motor polyneuropathy with features of demyelination on nerve conduction studies. Management options include intravenous immunoglobulin and corticosteroid infusions. Prognosis is generally favorable but there have been reports of variable response to treatment. This condition is rare and local data on CIDP and its variants is limited, hence we report a case of a 64-year-old male presenting with 3 year history of progressive asymmetric numbness and weakness of all limbs. Sensory deficits began on the left hand and had progressed to involve the distal portions of all limbs, eventually developing weakness as well. The patient underwent multiple nerve-conduction studies, all of which showed findings congruent with MADSAM. He was given intravenous high dose methylprednisolone and was maintained on mycophenolate mofetil.

5-aminovalanoic acid (5AVA) can be used as the precursor of new plastics nylon 5 and nylon 56, and is a promising platform compound for the synthesis of polyimides. At present, the biosynthesis of 5-aminovalanoic acid generally is of low yield, complex synthesis process and high cost, which hampers large-scale industrial production. In order to achieve efficient biosynthesis of 5AVA, we developed a new pathway mediated by 2-keto-6-aminohexanoate. By combinatory expression of L-lysine α-oxidase from Scomber japonicus, α-ketoacid decarcarboxylase from Lactococcus lactis and aldehyde dehydrogenase from Escherichia coli, the synthesis of 5AVA from L-lysine in Escherichia coli was achieved. Under the initial conditions of glucose concentration of 55 g/L and lysine hydrochloride of 40 g/L, the final consumption of 158 g/L glucose and 144 g/L lysine hydrochloride, feeding batch fermentation to produce 57.52 g/L of 5AVA, and the molar yield is 0.62 mol/mol. The new 5AVA biosynthetic pathway does not require ethanol and H₂O₂, and achieved a higher production efficiency as compared to the previously reported Bio-Chem hybrid pathway mediated by 2-keto-6-aminohexanoate.
Nylons ; Lysine/metabolism* ; Hydrogen Peroxide/metabolism* ; Metabolic Engineering ; Plastics/metabolism* ; Fermentation ; Escherichia coli/metabolism* ; Aminocaproates/metabolism*

OBJECTIVES: Hepatic fibrosis is a serious pathological consequence of chronic liver disease. Mycophenolate mofetil (MMF) is a commonly used immunosuppressant after organ transplant. However, the relationship between MMF and hepatic fibrosis remains unclear. This study aims to explore the effect of MMF on hepatic fibrosis in mice and the potential mechanism. METHODS: A total of 24 mice (male, 8-week old, C57BL/6) were randomly divided into a control group, a MMF group, a carbon tetrachloride (CCl₄) group and a CCl₄+MMF group (n=6 in each group). After the mice were sacrificed, the serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels were detected. The liver tissues were taken up for Masson staining and collagen I (COL1) immunohistochemistry. The levels of transforming growth factor-β1 (TGF-β1) and α-smooth muscle actin (α-SMA) were detected by Western blotting. Finally, the levels of mRNA for TGF-β1, α-SMA, and COL1 were detected using real-time PCR. RESULTS: Compared with the CCl₄ group, the ALT and AST levels were lower (both P<0.05), the degree of liver fibrosis was alleviated, and the deposition of COL1 in the liver was significantly decreased (P<0.01) in the CCl₄+MMF group. Compared with the CCl₄ group, the protein expression levels of TGF-β1 and α-SMA were significantly decreased (both P<0.05) and the relative expression levels of TGF-β1, α-SMA and COL1 mRNA in the liver were significantly decreased (all P<0.05) in the CCl₄+MMF. CONCLUSIONS MMF could reduce CCl₄-induced hepatic fibrosis, which might be related to the inhibition of TGF-β1. This study is expected to provide a target for the treatment of hepatic fibrosis.
Male ; Animals ; Mice ; Mice, Inbred C57BL ; Mycophenolic Acid/therapeutic use* ; Carbon Tetrachloride/toxicity* ; Transforming Growth Factor beta1/genetics* ; Liver Cirrhosis/drug therapy* ; RNA, Messenger

OBJECTIVES: To investigate the clinical effect of different immunosuppressive treatment regimens in children with ocular myasthenia gravis (OMG). METHODS: A retrospective analysis was conducted on 130 children with OMG who were treated in the Department of Neurology, Jiangxi Children's Hospital, from February 2018 to February 2023. According to the treatment regimen, they were divided into four groups: glucocorticoid (GC) group (n=29), mycophenolate mofetil (MMF) group (GC+MMF; n=33), methotrexate (MTX) group (GC+MTX; n=30), and tacrolimus (FK506) group (GC+FK506; n=38). Treatment outcomes and adverse reactions were compared among the groups. RESULTS: After 3 months of treatment, the FK506 group had significantly lower scores of Myasthenia Gravis Quantitative Scale and Myasthenia Gravis-Specific Activities of Daily Living than the other three groups (P<0.05). After 3 months of treatment, the FK506 group had a significantly lower dose of prednisone than the GC group, and after 6 and 9 months of treatment, the MMF, MTX, and FK506 groups had a significantly lower dose of prednisone than the GC group (P<0.05). After 12 months of treatment, the MMF, MTX, and FK506 groups had a significantly lower incidence rate of GC-related adverse reactions than the GC group (P<0.05). CONCLUSIONS For children with OMG, the addition of various immunosuppressants can reduce the dosage of GC and adverse reactions. Among them, FK506 shows superior efficacy compared to other immunosuppressants in the early treatment of OMG.
Humans ; Child ; Prednisone/adverse effects* ; Tacrolimus/adverse effects* ; Retrospective Studies ; Activities of Daily Living ; Immunosuppressive Agents/adverse effects* ; Myasthenia Gravis/drug therapy* ; Glucocorticoids/therapeutic use* ; Mycophenolic Acid/adverse effects*

OBJECTIVES: To investigate the difference in the therapeutic effect of mycophenolate mofetil (MMF) or cyclophosphamide (CTX) in children with Henoch-Schönlein purpura nephritis (HSPN) of different age groups. METHODS: A retrospective analysis was conducted on the clinical data of 135 children with HSPN who were treated with MMF or CTX in the Department of Nephrology, Children's Hospital Affiliated to Capital Institute of Pediatrics, from October 2018 to October 2020. According to the immunosuppressant used, they were divided into two groups: MMF group and CTX group, and according to the age, each group was further divided into two subgroups: ≤12 years and >12 years, producing four groups, i.e, the ≤12 years MMF subgroup (n=30), the >12 years MMF subgroup (n=15), the ≤12 years CTX subgroup (n=71), and the >12 years CTX subgroup (n=19). All children were followed up for at least 12 months, and the above groups were compared in terms of clinical outcomes and the incidence rate of adverse reactions. RESULTS: There was no significant difference in the complete response rate between the MMF group and the CTX group after 3, 6, and 12 months of treatment (P>0.05). There were no significant difference in the complete response rate and the incidence rate of adverse reactions between the >12 years MMF subgroup and the ≤12 years MMF subgroup at 3, 6, and 12 months of treatment (P>0.05). The >12 years CTX subgroup had a significantly lower complete response rate than the ≤12 years CTX subgroup at 6 and 12 months of treatment (P<0.05). The >12 years CTX subgroup had a significantly higher incidence rate of adverse reactions than the >12 years MMF subgroup (P<0.05). CONCLUSIONS The efficacy and adverse reactions of MMF are not associated with age, but the efficacy of CTX is affected by age, with a higher incidence rate of adverse reactions. CTX should be selected with caution for children with HSPN aged >12 years.
Child ; Humans ; Mycophenolic Acid/adverse effects* ; IgA Vasculitis/drug therapy* ; Retrospective Studies ; Cyclophosphamide/adverse effects* ; Immunosuppressive Agents/adverse effects* ; Vasculitis/drug therapy* ; Nephritis/complications*

This study aimed to evaluate the efficacy and safety of mycophenolate mofetil (MMF) or tacrolimus (TAC) compared with azathioprine (AZA) as maintenance therapy for active lupus nephritis (ALN). Patients with ALN who responded to 24 weeks of induction treatment were enrolled. Patients who received MMF or TAC as induction therapy continued MMF or TAC treatment during the maintenance period, whereas those who received intravenous cyclophosphamide were subjected to AZA treatment. The primary endpoint was the incidence of renal relapse. Secondary endpoints included extrarenal flares and composite endpoints (deaths, end-stage renal disease, or doubling of serum creatinine levels). A total of 123 ALN patients (47 in the MMF group, 37 in the TAC group, and 39 in the AZA group) were enrolled. The median follow-up time was 60 months. Ten MMF-treated patients, ten TAC-treated patients, and eight AZA-treated patients experienced renal relapses (P = 0.844). The cumulative renal relapse rates in the MMF group (P = 0.934) and TAC group (P = 0.673) were similar to the renal relapse rate in the AZA group. No significant difference in the incidence of severe adverse event was observed among the groups. Long-term maintenance therapies with MMF or TAC might have similarly low rates of renal relapse and similar safety profiles compared with AZA.
Humans ; Mycophenolic Acid/adverse effects* ; Azathioprine/adverse effects* ; Tacrolimus/therapeutic use* ; Lupus Nephritis/complications* ; Immunosuppressive Agents ; Treatment Outcome ; Recurrence

OBJECTIVE: To systematically evaluate the hemostatic efficacy of tranexamic acid and ε-aminocaproic acid in total hip arthroplasty (THA) and total knee arthroplasty (TKA). METHODS: Randomized controlled trials (RCT) and retrospective case-control studies about tranexamic acid and ε-aminocaproic acid for the comparison of THA or TKA were searched electronically in PubMed, EMbase, Cochrane Library, China National Knowledge Infrastructure (CNKI), Wanfang, VIP from the time of building databases to July 2020. Two investigators carried out literature screening and data extraction according to the inclusion and exclusion criteria respectively. The methodological quality of the included randomized controlled studies was evaluated through the Cochrane Handbook, and the methodological quality of the included retrospective case-control studies was evaluated through the NOS scale. Blood loss, the incidence of thrombosis complications, per capita input of hemoglobin were Meta-analyzed by Review Manager 5.3 software. RESULTS: A total of 6 articles were included, including 4 RCTs and 2 retrospective case-control studies. A total of 3 174 patients, including 1 353 in the tranexamic acid group and 1 821 in the ε-aminocaproic acid group. Meta-analysis results showed that there were no difference statistical significance in blood loss [MD=-88.60, 95%CI(-260.30, 83.10), P=0.31], blood transfusion rate [OR=1.48, 95%CI(0.96, 2.27), P=0.08], thrombotic complications [OR=0.80, 95%CI(0.07, 8.83), P=0.85], per capita hemoglobin input [MD=0.04, 95%CI(-0.02, 0.10), P=0.18] between tranexamic acid group and ε-aminocaproic acid group during THA. While in TKA, the blood loss of the tranexamic acid group was less than that of the ε-aminocaproic acid group [MD=-147.13, 95%CI(-216.52, -77.74), P<0.0001], the difference was statistically significant. The blood transfusion rate [OR=1.30, 95%CI(0.74, 2.28), P=0.37], thrombotic complications [OR=0.95, 95%CI(0.38, 2.36), P=0.92], per capita hemoglobin input [MD=-0.00, 95%CI(-0.05, 0.06), P=0.48], tourniquet time [MD=1.54, 95%CI(-2.07, 5.14), P=0.40] were similar between two groups, the difference was not statistically significant. CONCLUSION In THA, tranexamic acid and ε-aminocaproic acid have similar hemostatic effects, while in TKA, tranexamic acid can effectively reduce the patient's blood loss and has a better hemostatic effect. Tranexamic acid is recommended as one of the first choice hemostatic drugs for TKA.
Aminocaproic Acid/therapeutic use* ; Antifibrinolytic Agents/therapeutic use* ; Arthroplasty, Replacement, Hip/methods* ; Arthroplasty, Replacement, Knee/methods* ; Blood Loss, Surgical/prevention & control* ; Hemoglobins ; Hemostatics ; Humans ; Tranexamic Acid/therapeutic use*

OBJECTIVE: To study the efficacy and safety of mycophenolate mofetil (MMF) versus cyclophosphamide (CTX) in the treatment of children with Henoch-Schönlein purpura nephritis (HSPN) and nephrotic-range proteinuria. METHODS: A prospective clinical trial was conducted in 68 pediatric patients who were admitted to the Department of Nephrology, Children's Hospital Affiliated to Capital Institute of Pediatrics and who were diagnosed with HSPN and nephrotic-range proteinuria from August 2016 to November 2019. The patients were randomly divided into two groups:MMF treatment ( RESULTS: At months 3, 6, and 12 of treatment, there was no significant difference in the complete remission rate and the response rate between the MMF treament and CTX treatment groups ( CONCLUSIONS MMF and CTX have similar efficacy and safety in the treatment of HSPN children with nephrotic-range proteinuria.
Child ; Cyclophosphamide/adverse effects* ; Humans ; Immunosuppressive Agents/adverse effects* ; Mycophenolic Acid/adverse effects* ; Nephritis/drug therapy* ; Prospective Studies ; Proteinuria/etiology* ; Purpura, Schoenlein-Henoch/drug therapy* ; Retrospective Studies

OBJECTIVE: To analyze the clinical outcomes of engraftment, graft-versus-host disease (GVHD) and survival in the patients with AML1-ETO positive acute myeloid leukemia (AML) treated with unrelated umbilical cord blood transplantation (UCBT). METHODS: Forty-Five patients with high-risk refractory AML1-ETO positive AML were treated with a single UCBT in a single center from July 2010 to April 2018. All the patients underwent a myeloablative preconditioning regimen，and cyclosporine A (CSA) combined with mycophenolate mofetil (MMF) was used to prevent GVHD. RESULTS: The median value of total nucleated cells (TNC) in cord blood was 5.21 (1.96-12.68)×10/kg recipient body weight, and that of CD34+ cells was 5.61 (0.56-15.4)×10/kg recipient weight. The implantation rate of neutrophil at 42 d and that of platelet at 120 d were 95.6% and 86.7%, respectively. The median time of absolute neutrophil count (ANC)＞0.5×10/L and platelet 20×10/L were 16 (12-18) d and 37 (17-140) d after transplantation, respectively. The cumulative incidence of Ⅰ -Ⅳ grade acute GVHD (aGVHD) at 100 d after transplantation was 48.9% (95% CI 33.5%-62.6%), Ⅱ-Ⅳ grade aGVHD occurred in 12 cases (33.3%) (95% CI 20%-47.2%) , and Ⅲ-Ⅳ grade a GVHD in 8 cases (20%) (95% CI 9.8% -32.8%). In 5 cases of 40 patients survived over 100 days, the chronic GVHD (cGVHD) occurred after transplantation, among which 4 were localized, and 1 was extensive. 3 patients relapsed, and the 2-year cumulative relapse rate was 9.5% (95% CI 2.4%-22.8%). The median follow-up time was 23.5 (0.9-89.67) months, 10 patients died, 2-year disease-free survival rate (DFS) was 72.7%, and overall survival rate (OS) was 75.5%. Multivariate analysis showed that Ⅲ-Ⅳ. acute GVHD (aGVHD) affected overall survival. CONCLUSION UCBT is an effective rescue treatment for patients with high-risk refractory AML1-ETO positive AML.
Cord Blood Stem Cell Transplantation ; Core Binding Factor Alpha 2 Subunit ; Graft vs Host Disease ; Humans ; Leukemia, Myeloid, Acute ; Mycophenolic Acid ; Oncogene Proteins, Fusion ; Peripheral Blood Stem Cell Transplantation ; RUNX1 Translocation Partner 1 Protein ; Transplantation Conditioning

Adolescent ; Busulfan ; therapeutic use ; Child ; Child, Preschool ; Cyclophosphamide ; therapeutic use ; Cyclosporine ; therapeutic use ; Female ; Hematopoietic Stem Cell Transplantation ; methods ; Humans ; Infant ; Leukemia ; drug therapy ; mortality ; therapy ; Leukemia, Myeloid, Acute ; drug therapy ; mortality ; therapy ; Male ; Mycophenolic Acid ; therapeutic use ; Precursor Cell Lymphoblastic Leukemia-Lymphoma ; drug therapy ; mortality ; therapy ; Retrospective Studies ; Treatment Outcome