Search

Objectives: Evidence regarding the efficacy of neridronate in the treatment of complex regional pain syndrome type I (CRPS I) is increasing, however, very little data are available in paediatric age. Our aim was to analyse the safety and the efficacy of neridronate in a case series of children with CRPS I, according to the Budapest criteria, who did not respond to previous pharmacological and physical therapy., Methods: We collected data of children affected by CRPS I from three paediatric rheumatology centres who received neridronate. Efficacy was evaluated by changes in pain intensity, vasomotor changes, physical function, need for pain medications and MRI findings. Adverse effects were also documented., Results: Five children (3 females and 2 males, mean age 10.4 years, range 7-13 years) who received neridronate (4 intravenous, 1 intramuscular) were included. All patients had failed previous medical treatments (NSAIDs in 4, local steroids in 2, gabapentin, vitamin D and calcium supplementation in 1) and non-medical therapies (physiotherapy in 4, magnetotherapy in 2, laser in 1). Four out of five patients reported a significant improvement in pain (average VAS pre-treatment 9.6, post-treatment 2.6), recovery of physical function, and a reduced need for pain medications. Before treatment, all patients underwent MRI which revealed bone oedema that disappeared in the three of them after treatment. Neridronate was well-tolerated as only one patient experienced mild flu-like symptoms., Conclusions: Our data suggest that in children as in adults with CRPS I, neridronate may represent an effective and safe treatment option, particularly in those who do not respond to other pain treatments.

Objective: The main aim of this study was to define the best treatment option for multisystem inflammatory syndrome in children (MIS-C) and to analyse the role of anakinra., Methods: This is a multicentre retrospective cohort study. Patients were treated according to the attending physician's decision. The patients were divided into four groups on the basis of the first treatment at time of admittance: (i) IVIG, (ii) IVIG and methylprednisolone (≤2 mg/kg/day), (iii) IVIG with high-dose methylprednisolone (>2 mg/kg/day) and (iv) anakinra with or without IVIG and/or methylprednisolone. Primary outcomes were defined as the presence of at least one of the following features: death, the failure of initial treatment, meaning the need for additional treatment for clinical worsening and cardiac involvement at the end of follow-up., Results: Two hundred thirty-nine patients were recruited. At univariate analysis, persistent heart involvement at discharge was more frequent in those not receiving anakinra as initial treatment (3/21 vs 66/189; P = 0.047). After comparisons between the four treatment regimens, adjusting for the propensity score, we observed that early treatment with anakinra was associated with a lower probability of developing persistent heart disease at the end of follow-up (odds ratio: 0.6; 95% CI: 0.4-1.0)., Conclusion: We report that early treatment with anakinra is safe and very effective in patients with severe MIS-C. In addition, our study suggests that early treatment with anakinra is the most favourable option for patients with a higher risk of developing a severe disease outcome., (© The Author(s) 2023. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: journals.permissions@oup.com.)

Autoinflammatory diseases (AID) are a heterogeneous group of inherited conditions caused by abnormal activation of systems mediating innate immunity. Recent literature focuses on A20 Haploinsufficiency, an autoinflammatory disease with a phenotype resembling Behçet disease (BD). It is caused by loss-of-function mutations in TNFAIP3 gene that result in the activation of a pro-inflammatory pathway. In this case report we describe a one-year-old baby who came to our attention for hematochezia appeared at three months of age which was considered an expression of early-onset colitis. The following appearance of cutaneous inflammation Behçet-like and the positive family history concurred with the diagnosis of an autoinflammatory disease. Extended genetic tests in the patient allowed to identify a heterozygous variant in TNFAIP3 [NM_006290.4:c.460G > T, p.(Glu154Ter)], not previously described and not present in the GnomAD database. As a consequence the diagnosis A20 Haploinsufficiency was established and the appropriate management was started. The same TNFAIP3 variant was also found in her father who had suffered from recurrent oral aphthosis, vitiligo and thyroiditis since childhood. In conclusion, we described a young patient with a novel heterozygous mutation in TNFAIP3 who developed BD-like symptoms. We proposed that loss-of-function variants in TNFAIP3 may be associated with a very early-onset intestinal BD phenotype., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (© 2022 Zanatta, Biscaro, Bresolin, Marzaro, Sarcognato, Cataldo, Marzollo and Martelossi.)

Background: Endothelial progenitor cells (EPC) promote angiogenesis and vascular repair. Though reduced EPC levels have been shown in rheumatoid arthritis, no study has so far evaluated EPCs in children with juvenile idiopathic arthritis (JIA). We aimed to study circulating EPCs in children with JIA, their relation to disease activity, and effects of anti TNF-α treatment., Methods: Circulating EPCs were quantified by flow cytometry based on CD34, CD133 and KDR expression in peripheral blood of 22 patients with oligoarticular JIA and 29 age-matched controls. EPCs were re-assessed in children with methotrexate-resistant oligo-extended JIA before and up to 12 month after initiation of anti-TNF-alpha therapy. Plasma concentrations of inflammatory and EPC-regulating factors were measured using a multiplex array. Confocal immunofluorescence was used to demonstrate EPCs in synovial tissues., Results: Children with active JIA showed a significant reduction of relative and absolute counts of circulating progenitor cells and EPCs compared to age-matched healthy controls. CD34(+) cell levels were modestly and inversely correlated to disease activity. A strong inverse correlation was found between serum TNF-α and EPC levels. In 8 patients treated with anti TNF-α agents, the number of EPCs rose to values similar to healthy controls. CD34(+)KDR(+) EPCs were found in the synovial tissue of JIA children, but not in control., Conclusions: Children with JIA have reduced levels of the vasculoprotective and proangiogenic EPCs. While EPCs may contribute to synovial tissue remodelling, EPC pauperization may indicate an excess cardiovascular risk if projected later in life.

Context: Croton celtidifolius Baill (Euphorbiaceae) is a tree found in the Atlantic Forest in Southern Brazil, where it is commonly known as "Sangue-de-Dragão". Its red latex is used traditionally for treating ulcers, diabetes and cancer., Objective: To evaluate antitumor activities of Croton celtififolius latex in vitro and in vivo., Material and Methods: Phytochemical analyses were conducted using HPLC-DAD-MS. Cytotoxic, nuclease and pro-apoptotic properties were determined using the tetrazolium salt assay (MTT), plasmid DNA damage assay and ethidium bromide (EB)/acridine orange methods, respectively, and antitumor activity was determined in the Ehrlich ascites carcinoma (EAC) mouse model., Results: Phytochemical studies indicated a high phenol content of flavonols (45.67 ± 0.24 and 18.01 ± 0.23 mg/mL of myricetin and quercetin, respectively) and flavan-3-ols (114.12 ± 1.84 and 1527.41 ± 16.42 mg/L of epicatechin and epigallocatechin, respectively) in latex. These compounds reduced MCF-7 and EAC cell viability in the MTT assay (IC50 = 169.0 ± 1.8 and 187.0 ± 2.2 μg/mL, respectively). Latex compounds caused significant DNA fragmentation and increased the number of apoptotic cells (negative control (NC), 12%; latex, 41%) as indicated by differential staining in the EB/acridine orange assay. The in vivo latex treatment at 3.12 mg/kg/day reduced the body weight by 7.57 ± 2.04 g and increased median survival time to 17.5 days when compared to the NC group (13.0 days). In addition, the highest latex concentration inhibited tumor growth by 56%., Discussion and Conclusion: These results agree with ethno-pharmacological reports showing cytotoxicity and antitumor activity of C. celtidifolius latex. The mechanism of antitumor action may be related to direct DNA fragmentation that reduces survival and induces apoptosis.

Introduction: Obesity is a chronic disease associated with oxidative stress. Bariatric surgery for the treatment of obesity may affect biomarkers of oxidative stress., Objectives: The aim of the present study was to evaluate the effect of Roux-en-Y gastric bypass (RYGB) on blood markers of oxidative stress, such as vitamins C and E, β-carotene, reduced glutathione (GSH), catalase (CAT), ferric reducing antioxidant potential (FRAP), and thiobarbituric acid-reactive substances (TBARS)., Methods: A prospective controlled clinical trial was carried out. The participants were distributed into two groups: a control group (n=35), which was evaluated once, and a bariatric group (n=35), which was evaluated at baseline as well as 6, 12, and 24 months after surgery., Results: After surgery, the BMI decreased from 47.05±1.46 to 30.53±1.14 kg/m (P<0.001), but 25.7% of the participants regained weight after 24 months. In relation to the baseline, postsurgery reductions were found in vitamin C (31.9±4.6%, P<0.001), β-carotene (360.7±368.3%, P<0.001), vitamin E (22.8±4.1%, P<0.001), GSH (6.6±5.2%, P=0.090), CAT (12.7±5.6%, P=0.029), and FRAP (1.2±3.8%, P=0.085) 2 years after RYGB. TBARS levels decreased after 12 months (71.6±2.9%, P<0.001) in relation to the baseline but increased by 195.0±28.2% between the 12th and the 24th month (P<0.001)., Conclusion: The present findings show that oxidative stress returned 2 years after RYGB. Concentrations of vitamin C, β-carotene, GSH, CAT, and FRAP were decreased, whereas the concentration of TBARS decreased in the first year but increased in the following year, which may be partly explained by the imbalance between antioxidants and pro-oxidants.

Rib tumors are uncommon and represent 5% to 10% of all bony tumors. Regarding the benign rib lesions, costal angioma is very rare in childhood. We report a case of a rare angioma of a rib complicated by bone erosion in a young boy., (Copyright © 2012 Elsevier Inc. All rights reserved.)

Objective: To evaluate the effects of prophylactic transfusion by means of erythrocytapheresis at the beginning of the third trimester of pregnancy in women with sickle cell disease (SCD)., Methods: A cohort of 14 pregnant women with SCD who received prophylactic erythrocytapheresis transfusions at the beginning of the third trimester was retrospectively compared with a cohort of 17 pregnant women who received simple prophylactic transfusions for no indication other than SCD severity., Results: Prophylactic erythrocytapheresis transfusions were associated with a lower risk of intrauterine growth restriction (OR, 0.11; 95% confidence interval, 0.01-1.00) and oligohydramnios (OR, 0.65; 95% confidence interval, 0.45-0.92) in pregnant women with SCD., Conclusion: These results suggest that erythrocytapheresis transfusions are beneficial in women with SCD who are in the third trimester of pregnancy. Given the decrease in transfusion risks, this therapy deserves further evaluation in future trials.

Dermatomyositis (DM) in adults is frequently associated with cancer. In contrast, juvenile DM (JDM) is predominantly idiopathic and rarely reported with occult neoplasm. We describe a patient who presented with DM that was found to be a paraneoplastic manifestation of nasopharyngeal carcinoma. Although rare, paraneoplastic JDM must be suspected in the presence of unusual features such as elevated inflammatory markers or lymphadenopathy. Accurate clinical assessment including appropriate biopsies is needed before starting glucocorticoid therapy.