Osteoporosis is now increasingly recognized in children due to the increased prevalence of disorders associated with bone loss. Fragility fractures represent the cardinal clinical features of ...pediatric osteoporosis and children presenting with fragility fractures deserve an accurate assessment to rule out a secondary cause. Indeed, in the pediatric population, a low bone mass is often a consequence of a chronic disease or its treatment; genetic bone disorders represent the cause of only a small fraction of cases. The position statement of the International Society for Clinical Densitometry guides physicians in interpreting densitometric data and making diagnoses of osteoporosis in children. Once a diagnosis of osteoporosis has been made, the aim is to identify children in whom bone status may deteriorate if left untreated. To date, bisphosphonates have represented the mainstay of treatment for pediatric osteoporosis. However, due to the peculiar pathophysiology of osteoporosis in this age group, a pharmacological agent with an anabolic effect on bone may provide clinicians with other therapeutic options in children. Multicenter studies are needed to optimize treatments and define optimal clinical response in treated children.
The efficacy of diet and its influence on gut microbiome composition has been largely demonstrated in inflammatory bowel disease (IBD). Little is known about its potential in the management of ...extraintestinal manifestations. We report a successful application of Crohn disease exclusion diet (CDED) in association with infliximab and methotrexate, as salvage therapy in a child affected by chronic recurrent multifocal osteomyelitis (CRMO) and Crohn disease (CD) resistant to optimized therapy. Both intestinal and bone symptoms remitted after the application of CDED. Diet may have acted on common microbic inciting agents that trigger both intestinal and bone inflammation, supporting the role of microbiota in the pathogenesis of IBD-associated extraintestinal manifestations. Our experience suggests the potential benefit of CDED in association with combined therapy in resistant patients affected by CD and extraintestinal manifestations.
Corticosteroids are the mainstay of therapy for many pediatric disorders and sometimes are life-saving. Both endogenous and synthetic derivatives diffuse across the cell membrane and, by binding to ...their cognate glucocorticoid receptor, modulate a variety of physiological functions, such as glucose metabolism, immune homeostasis, organ development, and the endocrine system. However, despite their proved and known efficacy, corticosteroids show a lot of side effects, among which growth retardation is of particular concern and specific for pediatric age. The aim of this review is to discuss the mechanism of action of corticosteroids, and how their genomic effects have both beneficial and adverse consequences. We will focus on the use of corticosteroids in different pediatric subspecialties and most common diseases, analyzing the most recent evidence.
Abstract
Immune response to biologics treatment, while widely reported, yet fails to correlate with clinical outcomes and assay to assay comparison is often not possible. Hence, we developed a new ...peptide based-detection assay to stratify pediatric patients with juvenile idiopathic arthritis (JIA) or chronic non-infectious uveitis (CNU) and monitor anti-drug antibodies (ADAbs) formed as part of an immune response to treatment with the fully human monoclonal therapeutic antibody Adalimumab. Adalimumab derived synthetic peptides were optimized for maximum immunogenicity and were tested by SP-ELISA on a development cohort of 18 JIA and CNU treated patients. The two best performing peptides able to differentiate patient groups were selected for evaluation with a larger scale ELISA testing on a total of 29 sera from pediatric patients with JIA or CNU. The results of this peptide-based assay were compared to an in-house developed SPR biosensor ADAbs assay and a commercially available bridging ELISA. The first peptide, termed HC3, was able to positively detect ADAbs in 7 out of the 29 sera, while the second peptide, called LC3, was able to detect ADAbs in 11 out of 29 sera in the evaluation group. Following statistical data evaluation, it has been found that the detection of ADAbs using the peptide-based ELISA assay positively correlates with disease progression and remission. Two synthetic peptides derived from Adalimumab may provide a beneficial tool to clinicians for monitoring patient response to such treatment and taking informed decisions for treatment alternatives.
Introduction and objectives
Immunoglobulin A vasculitis (IgAV), the most common childhood vasculitis, is associated with gastrointestinal (GI) involvement in 50–75% of cases. The aim of this study ...was to describe the characteristics of GI involvement in a cohort of hospitalized children with IgAV.
Methods
We retrospectively evaluated patients hospitalized for IgAV at Meyer Children’s University Hospital, from January 2010 to December 2020. The children’s families were interviewed by phone and asked about disease relapses.
Results
In the study period, 118 children had GI involvement, corresponding to 75% of children hospitalized for IgAV. Their median age was 7 years (interquartile range 6–9). The most frequent GI manifestations were abdominal pain (96%), bleeding (71%, mostly occult), vomiting (58%), and diarrhea (17%). GI complications, observed in 18%, were intussusception (14%), appendicitis (3%), gallbladder hydrops (2%), and ileal perforation (1%). Abdomen ultrasound, performed in all cases, was abnormal in 68%. Abdomen X-ray, performed in 19 patients, showed pathologic findings in 84% of them. Selected children with severe manifestations also underwent abdomen computed tomography (2/118; 2%) and GI endoscopy (5/118; 4%). Steroids were used in 80 (67.8%) cases. The use of second- and third-line therapies was necessary in three cases. Relapses, investigated in 80 cases (68%), occurred in 21% of them.
Key Points
•
The present retrospective study describes a cohort of 118 patients hospitalized for gastrointestinal (GI) involvement in immunoglobulin A vasculitis (IgAV) at a tertiary care children’s hospital from January 2010 to December 2020.
•
The most frequent GI manifestations were abdominal pain (96%), vomiting (58%), and diarrhea (17%). GI bleeding was reported in 71% of children, and it was occult in most cases.
•
GI complications, occurring in 18% of cases, were intussusception (14%), appendicitis (3%), gallbladder hydrops (2%), and ileal perforation (1%).
•
Steroid treatment has been successfully used in severe GI manifestations; however, our data do not support its association with low relapse risk.
Conclusions
The present study describes a large pediatric cohort of GI involvement in IgAV. Steroid treatment should be used when GI manifestations are severe. The association of steroid use with relapse risk is not currently established.
Deficiency of adenosine deaminase 2 (DADA2) is a rare monogenic autoinflammatory disease, whose clinical phenotype was expanded since the first cases, originally described as mimicker of ...polyarteritis nodosa, with immunodeficiency and early-onset stroke.
A systematic review according to PRISMA approach, including all articles published before the 31st of August 2021 in Pubmed and EMBASE database was performed.
The search identified 90 publications describing 378 unique patients (55.8% male). To date 95unique mutations have been reported. The mean age at disease onset was 92.15 months (range 0-720 months), 32 (8.5%) showed an onset of the first signs/symptoms after 18 years old and 96 (25.4%) after 10 years old. The most frequent clinical characteristics described were cutaneous (67.9%), haematological manifestations (56.3%), recurrent fever (51.3%), neurological as stroke and polyneuropathy (51%), immunological abnormalities (42.3%), arthralgia/arthritis (35.4%), splenomegaly (30.6%), abdominal involvement (29.8%), hepatomegaly (23.5%), recurrent infections (18.5%), myalgia (17.9%), kidney involvement (17.7%) etc. Patients with skin manifestations were older than the others (101.1 months SD ± 116.5, vs. 75.3 SD ± 88.2, p 0.041), while those with a haematological involvement (64.1 months SD ± 75.6 vs. 133.1 SD ± 133.1, p < 0.001) and immunological involvement (73.03 months SD ± 96.9 vs. 103.2 SD ± 112.9, p 0.05) are younger than the others. We observed different correlations among the different clinical manifestations. The use of anti-TNFα and hematopoietic cell stems transplantation (HCST) has improved the current history of the disease.
Due to this highly variable phenotype and age of presentation, patients with DADA2 may present to several type of specialists. Given the important morbidity and mortality, early diagnosis and treatment are mandatory.
The objective of the study is to evaluate the efficacy of corticosteroids in Sydenham chorea. This is a retrospective observational study. Clinical information of children with Sydenham chorea were ...collected. Outcome of Sydenham chorea was evaluated in consideration of presence or absence of corticosteroid therapy. Thirty patients were enrolled. A total of 15 were treated with prednisone, 15 received symptomatic drugs or no treatment. Patients who were treated with prednisone showed faster improvement (4 vs 16 days;
p
= 0.002) and shorter median time of remission (30 vs 135 days;
p
< 0.001).
Conclusion
: Our study showed that corticosteroid therapy is an effective treatment of Sydenham chorea.
What is Known:
• Steroid treatment in Sydenham chorea is widely used but it is not standardized.
• Few manuscript report a beneficial use of steroids in Sydenham chorea if compared with no treatment.
What is New:
• Steroid treatment seems to be effective in both clinical remission and clinical improvement of symptoms among patients with Sydenham chorea.
• Steroid treatment seems to be superior to conventional treatment.
Italy was the first Western country to be hit by the SARS-CoV-2 epidemic. There is now mounting evidence that a minority of children infected with SARS-CoV2 may experience a severe multisystem ...inflammatory syndrome, called Multisystem inflammatory Syndrome associated with Coronavirus Disease 2019 (MIS-C). To date no universally agreed approach is available for this disease. MAIN BODY: as Italy is now facing a second hity of COVID-19 cases, we fear a recrudescence of MIS-C cases. We have, therefore, decided to prepare a report that will help clinicians to face this novel and challenging disease. We propose a diagnostic algorithm, to help case definition and guide work-up, and a therapeutic approach. MIS-C should be promptly recognized, based on the presence of systemic inflammation and specific organ involvement. Early treatment is crucial, and it will be based on the combined use of corticosteroids, high-dose immunoglobulins and anti-cytokine treatments, depending on the severity of the disease. Ancillary treatments (such as. aspirin and thrombo-profilaxis) will be also discussed.
we propose a document that will help physicians to diagnose and treat MIS-C patients. Given the level of evidence available and the methodology used, this document should not be interpreted as a guideline; the final decision about the optimal management should still be taken by the caring physician, on an individual basis.
To describe clinical and epidemiological characteristics of a Kawasaki syndrome cohort. In a monocentric, retrospective, observational study, between February 1982 and August 2018, we enrolled 361 ...children, aged 1 month to 24.4 years. Coronary artery lesions were detected in 20.2% of patients: 16% had coronary ectasia, and 4.15% had coronary aneurisms. A significant difference regarding age at disease onset (
p
= 0.025), fever duration (
p
< 0.0001), CRP (
p
= 0.001) and day of first IVIG administration (
p
< 0.0001) was detected among group. A significant correlation between coronary artery lesions and disease onset < 6 months (
p
= 0.009), second IVIG dose (
p
< 0.001) and male gender (
p
= 0.038) has been detected. Median long-term follow-up was 10.2 years (1–36 years). At the last available follow-up, patients without coronary involvement and coronary ectasia had normal cardiological tests, conversely, in patients with aneurisms, 8/13 showed persistent aneurisms at echocardiography, one ECG repolarization alterations, and one ST depression at the peak of effort during ergometric test.
Conclusion
: Children with lower age, longer fever, higher level of CRP and retard in IVIG administration are at higher risk to develop coronary artery lesions. Our long-term follow-up analysis confirms, over 36 years of observation, the benign course of Kawasaki syndrome even in coronary artery lesion patients, if timely treated.
What is already known about this topic?
•
Stopping cardiologic assessment in no risk patients results economically advantageous, timesaving and able to reduce emotional discomfort in children and their families.
•
Age at disease onset, fever duration, CRP level, and day of first IVIG administration are possible risk factors for coronary artery lesions
What is New?
•
During 36 years of observation in real life, our study shows the benign course of Kawasaki syndrome without coronary artery lesions after 6–8 weeks from the disease onset.
•
Age < 6 months at disease onset is strongly related with coronary artery lesion development.
In this multicenter retrospective study we aimed to evaluate the outcome of cardiac involvement in children affected by multisystem inflammatory syndrome (MIS-C), assessed through cardiac magnetic ...resonance (CMR). Children referring to three Italian tertiary pediatric centers between February 2020 and November 2021 with a diagnosis of MIS-C, who underwent CMR during a follow-up visit, were enrolled. Demographic, clinical, laboratory, treatment, and outcome data were collected. Twenty MIS-C patients (aged 9–17, median 12 years) were included in the study. Heart involvement at onset was testified by hypotension/shock (55%), laboratory evidence of myocardial involvement (100%), reduced LV ejection fraction (EF) on echocardiography (83%), and/or need for inotrope agents (40%); they all presented good clinical, laboratory, and echocardiographic response to treatment. CMR was performed after a median interval of 3 months from discharge. Pericardial effusion and myocardial edema were found in 5% of patients. Mild residual left ventricular (LV) dysfunction was found in 20% of patients, all showing normal echocardiographic LVEF at discharge. Minimal myocardial scars were found in 25% by late gadolinium enhancement (LGE). One patient was evaluated at two consecutive time points, showing partial resolution of a myocardial scar after 7 months from its first finding.
Conclusion
: Despite the severity of heart involvement in the acute MIS-C phase, the mid-term cardiac outcome is good. Direct cardiac tissue viral invasion may be involved in MIS-C pathogenesis.
What is Known:
• Heart involvement is common in MIS-C, but conflicting findings have been shown regarding cardiac outcome when assessed through cardiac MRI.
What is New:
• Midterm cardiac MRI shows mild abnormalities in patients recovered from MIS-C with any grade of severity of cardiac involvement at presentation.
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