Cardiac allograft vasculopathy in children — treatment challenges

Cardiomyopathy in children accounts for greater than 50% of the cases of end-stage cardiac disease leading to heart transplantation in children. While early survival is excellent, late survival is limited with an average graft half-life of approximately 15 years in children. Cardiac allograft vasculopathy is a not uncommon complication of transplantation and is the leading cause of late graft loss and retransplantation in pediatric populations. Studies of the United Network of Organ Sharing database and the Pediatric Heart Transplant Study group report rates of coronary vasculopathy that increase from 5% at 2 years to 35% at 10 years. Coronary artery vasculopathy is a complex process caused by both immune mediated endothelial dysfunction and vascular changes as well as typical cardiovascular risk factors. Unfortunately, despite vigilant surveillance protocols, new onset graft dysfunction and sudden cardiac death can be the presenting symptoms of new disease. In recent years multiple medical and adjuvant therapies have been studied in relation to potential management to minimize this disease process. Further research and collaborative multi-center trials will be the most effective means of developing strategies for the prevention and treatment of coronary vasculopathy in pediatric heart transplant patients.     

Natalizumab treatment in pediatric multiple sclerosis: A case report

Pediatric multiple sclerosis (MS) with manifestations before 16 years of age occurs in 0.4–10.5% of whole MS population. The initial course of the disease is relapsing–remitting with a relapse rate generally higher than that of adults, less than 3% have a primary progressive form. Some recent reports have shown that Interferon β (IFNβ) has a strong effect in reducing the relapse rate in children with MS and is well tolerated.We report a 12-year-old girl with MS and a high relapse rate from the onset. Frequent magnetic resonance imaging (MRI) detected persisting inflammatory activity and increase of lesion burden. She continued to present acute relapses and progression of disability in spite of a treatment with IFNβ-1a at different dosages and the addition of pulse IV steroid treatment. Then, we opted for Natalizumab treatment, recently approved as a monotherapy for patients with MS who experienced inadequate response to other disease modifying therapies and never used till now in pediatric MS. Our patient showed a complete response to Natalizumab with clinical and MRI suppression of disease activity.     

Infection à VIH de l''enfant : quand débuter le traitement antirétroviral ?

Administration of highly active antiretroviral treatment (HAART) has led in the developed world to a dramatic reduction in the incidence of HIV related pediatric mortality. HAART is now the standard-of-care therapy in infected children but the occurrence of short- and long-term drug-related toxic effects and emergence of drug-resistant viral variants temper its success. In children, both CD4 cell percent and viral load have independent predictive value for disease progression, CD4 cell being the stronger predictor of AIDS and death. Concerning children aged 12 months or oder current French recommendations for immediate therapy are based on the presence of clinical symptoms (of categories B or C) or the occurrence of a severe immunodeficiency (CD4 cell percent < 15%). In infants, risk of disease progression is higher and the viral load and CD4 percent are less reliable markers. HAART should theoretically be initiated in all infants in order to prevent HIV encephalopathy and early death. However, viral failure under HAART is often encountered in children less than 12 months because of high levels of replication as well as limited data on pharmacokinetics and drug dosing. A possible alternative approach for infants without risk factor for early progression is to defer HAART under close mentoring.     

Multiple sclerosis in childhood: clinical profile in 125 patients

Multiple sclerosis (MS) has its usual onset in early adult life (average age of 30 years), but age at clinical onset varies considerably. The implications of the age of onset on the clinical presentation and course of MS are unclear. This population-based retrospective study presents data from a group of 125 patients with onset of MS before age 16 years and can thus be considered as representative of MS occurring in childhood. It demonstrates that childhood MS is more frequent in girls, that it very often has a relapsing-remitting course, that initial bouts usually involve afferent structures of the central nervous system, that recovery from these is often complete, and that the pace of the disease is slow.     

History of pediatric hematology oncology

Pediatric Hematology Oncology as a specialty was possible because of the evolution of the science of Hematology, which developed microscopy for describing blood cell morphology and methods for quantitation of these elements. Before pediatric blood diseases could be defined, it was necessary to establish the normal blood values of infancy and childhood. The unique features of the blood of the newborn were the focus of many of the early studies. After normal values were established, specific blood disease and hematologic syndromes of children began to be described in Europe and the United States. Pediatric Hematology Oncology is a broad and complex area that encompasses perturbations of the several-formed elements of the blood and their precursors in the bone marrow, as well as the coagulation-fibrinolytic systems in the plasma, the reticuloendothelial system, and malignancies of the blood and solid tissues and organs. The interactions of the blood and nutrition have long been important areas of study. Advances in Pediatric Oncology have been particularly spectacular in the last 50 years. Using multi-modal therapy including combination chemotherapy, more than 80% of children with cancer can now be cured. During the last 50 years, Pediatric Hematology Oncology has increasingly used tools of the "new biology": immunology, biochemistry, enzymology, genetics and molecular genetics, and others. During the last century, many diseases have been recognized and defined by biochemical and genetic mechanisms, and in some instances they have been prevented or cured.     

History of WOFAPS (1963–2019)

The formation of the World Federation of Associations of Pediatric Surgery (WOFAPS) was an important unifying force in the emergence of pediatric surgery as a distinct specialty. Beginning with the formation of several national societies in the early ‘60s, an early, multinational effort was created. This was in large part fostered by the International Pediatric Association (IPA), which lent logistical support from the medical pediatric community to the pediatric surgeons. In 2001, the mission of the Federation was formalized to focus on the development and education of surgeons serving children, in all parts of the world. This was articulated in the famous Kyoto Declaration of Pediatric Surgery: “Every infant and child who suffers from an illness or disease has the right to be treated in an environment devoted to their care by a pediatric medical or surgical specialist”. This vision was unique at the time and foreshadowed the major increase in advocacy activity which has emerged in recent years. While the mission has evolved with time, the present organization continues to hold true to the guiding principles of the original founders and seeks to improve the quality of “Surgical Care for the child, no matter where they live”. Education and collaboration across borders underpins the organization’s endeavors.     

Impact du régime d''éviction sur la néophobie dans le cadre d''une allergie alimentaire chez l''enfant : étude exploratoire

OBJECTIVES: This study was designed to analyse the impact of an elimination diet in children with food allergy, and its perception by their parents on the later reticence of children to test unknown foods, food neophobia. METHODS: The degree of food neophobia of children having outgrown their allergy (mean age, 7 years 2 months) was compared to that of a sibling (9 years 5 months) using a standardized scale and a questionnaire of food friendliness. Parents were also asked to fill in a questionnaire on the disease and its burden on the family. RESULTS: Children having outgrown their allergy are more reluctant to test new foods than their non-allergic brother or sister, as shown by their scoring on the food neophobia scale and the number of unknown foods following the cure of the disease. Two factors increase the level of food neophobia, the distressing effect and the duration of the period elapsed until the diagnosis was made, as well as the distressing effect and the lack of variety in the meal preparation. CONCLUSION: Food neophobia, a normal phase between 2 and 10 years, is worsened by the elimination diet required by food allergy, especially in case of late diagnosis and when the time elapsed before diagnosis and the preparation of meals were perceived as difficult to bear.     

Les dysplasies bilatérales isolées de la hanche chez l'enfantDysplasia epiphysealis capitis femoris

Background. - Dysplasia epiphysealis capitis femoris is not well-known despite its relative frequency; its prognosis is difficult to predict. Population and methods. - Twenty children (19 boys and one girl) aged 2.5 to 7 years at diagnosis were included in the study. They had delayed capitis femoris apparition, or a fragmentation of dysplastic capitis femoris. The prognosis remained poor in 13 patients. Discussion. - Most authrs consider that prognosis of this disease is always favorable. We failed to find early prognostic criteria; radiological findings valuable for prognosis are only present after 3 to 6 years. Conclusion. - Early new imaging techniques have to be evaluated before considering impossibility to foresee the evolution.     

Lithiase biliaire et drépanocytose homozygote en pédiatrie à Dakar (Sénégal)

BACKGROUND: Cholelithiasis is a well recognized complication of sickle-cell anemia (SCA) because of chronic hemolysis. It is usually asymptomatic but may result in acute cholecystitis or cholangitis. The aim of this study was to assess prevalence of cholelithiasis and its associated factors among Senegalese children and adolescents with SCA. PATIENTS AND METHODS: We analyzed the follow-up records and results of systematic clinical, biological and ultrasonographic examinations in 106 patients with SCA aged 11 months to 22 years (median: 10 years, 6 months), followed up in the Albert-Royer Children's Hospital (Dakar). RESULTS: Cholelithiasis was detected in 9.4% of the patients. Gender, ethnic group, geographic origin and socioeconomic conditions did not have any association with cholelithiasis frequency. The youngest patient with cholelithiasis was 7 years old, and prevalence of cholelithiasis increased with age. The mean age at the beginning of SCA follow-up was higher among patients with cholelithiasis. They were more frequently transfused and tended to present more frequent pain crises and other complications of SCA. We found no spontaneous clinical manifestations attributed to cholelithiasis. Nevertheless, provoked right upper quadrant pain was more frequently observed in patients with cholelithiasis. Fetal hemoglobin, steady-state hemoglobin, reticulocytes count, serum bilirubin, alkaline phosphatase and transaminase levels were not significantly different in these patients, compared to the others. However, those with cholelithiasis had significantly higher mean red cell volume. CONCLUSION: Prevalence of cholelithiasis appears relatively low in Senegalese patients with SCA, probably owing to the tolerance of the Senegal haplotype. Advanced age and severe chronic hemolysis are etiologic factors in evidence. We recommend systematic abdominal ultrasound at least once a year for patients older than 5 years, or when right upper quadrant pain is observed. Cholecystectomy should be performed in cases of cholelithiasis in order to prevent complications.     

Traitement de la néphropathie du lupus érythémateux disséminé chez l'enfant

The severity of renal disease in systemic lupus erythematosus is variable. Renal biopsy is important to guide the treatment. The World Health Organization classification define six different histological categories with possible transformations from one category to another. Histological signs of activity or chronicity are important with respect to prognosis and treatment. Examination of renal biopsy allows predicting the reversibility of histological lesions following therapy. Apart from histological signs of severity, other factors may influence the prognosis: arterial hypertension, initial serum creatinine, the delay between onset of renal disease and treatment, the occurrence of exacerbations of the nephropathy, and the response to therapy by the end of the first year. The prognosis of severe forms of lupus nephritis, mainly diffuse proliferative glomerulonephritis, has improved during the last 20 years. The addition of immunosuppressive agents (cyclophosphamide, azathioprine) to corticosteroids is responsible for this improvement. Methylprednisolone pulses are as effective as oral high doses of prednisone during initial treatment and have fewer side effects. Many authors advocate monthly cyclophosphamide pulses over six months, sometimes followed by quarterly pulse cyclophosphamide. However, such an approach has not been proven to be more effective than an oral course of cyclophosphamide and/or azathioprine. On follow-up, steroid therapy should be slowly tapered, and close monitoring of lupus serological parameters (anti-DNA antibodies, complement), urinary protein excretion rate, urinary sediment and renal function allow one to detect exacerbations of the disease, which may require adapted therapy. While such protocols have improved the outcome, they have potential side effects. In addition to the deleterious effect of steroids on physical appearance, often badly tolerated by adolescents, immunosuppressive treatments increase the risks of severe infectious complications and the risks of cardiovascular complications in young adults.     

Screening for neuroblastoma: a 9–year birth cohort–based study in Niigata, Japan

Between April 1985 and March 1994 (9 years), 229 346 babies were born in Niigata prefecture, and 201 888 (88.0%) underwent mass screening (MS) for neuroblastoma at 6 months of age. To date, 29 infants have been screen–detected as having neuroblastoma (1 :7908). All screen–detected patients survived after removal of the primary tumor. In the same birth cohort, 17 additional children were clinically diagnosed as having neuroblastoma. The cumulative incidence rate of neuroblastoma at 5 years of age was 10.5 per 100 000 live births in the 5–year birth cohort before MS was introduced, and 18.6 per 100000 in the first 5–year birth cohort after MS was introduced. These values were not statistically different. The birth cohort incidence rate increased significantly to 22.2 per 100000 ( p < 5% compared with before MS) after the method of MS was changed to high–power liquid chromatography (HPLC), even though this latest birth cohort has not been followed for 5 years. The population–based mortality rate from neuroblastoma was 5.9 per 100 000 in the 5–year birth cohort before MS, and 4.5 per 100000 after MS in the first 5–year birth cohort, using the vanillymandelic acid (VMA) spot test. These values were not statistically significant. In contrast, no death was observed in the next 4–year birth cohort after MS using HPLC; however, this birth chohort has not yet been followed for 5 years. Mass screening, neuroblastoma     

Gas chromatographic-mass spectrometric metabolic profiling of patients with fatal infantile mitochondrial myopathy with de Toni-Fanconi-Debré syndrome

The metabolic profiles of three patients with fatal infantile mitochondrial myopathy with de Toni-Fanconi-Debré syndrome were studied by simultaneous analysis, after urease treatment of urinary organic acids, carbohydrates, polyols and amino acids using gas chromatography/mass spectrometry (GC/MS). All three patients persistently showed lactic aciduria, phosphaturia, glucosuria and generalized amino aciduria. This abnormal urinary metabolic profile was observed before the onset of any clinical symptoms, indicating that chemical diagnosis may be done presymptomatically. In one patient, the concentration of lactate increased in parallel with the severity of the clinical condition, whereas the urinary levels of 3-hydroxybutyrate, amino acids and glucose fluctuated and showed only a general tendency to increase with the clinical course. The above results suggest that simultaneous GC/MS analyses, without fractionation, of urinary metabolites facilitate not only the early chemical diagnosis either before or after the first onset, but also follow-up studies, providing an important index for the evaluation of the severity and clinical course in patients with this disorder.     

严重脓毒症和脓毒性休克早期目标导向治疗

严重脓毒症是PICU患儿死亡的主要原因之一.尽管抗生素和综合治疗手段不断改进,但其病死率依然居高不下.目前,研究证实早期目标导向治疗能显著降低严重脓毒症和脓毒性休克患者的病死率.本文介绍了目前国内外专家公认的早期目标导向治疗方案,旨在指导儿科医师尽早规范化地治疗严重脓毒症患儿,改善预后,提高生存率.     

Le DRESS syndrome, une réaction d''hypersensibilité aux médicaments, qui reste mal connue des pédiatres

The DRESS syndrome (Rash with Eosinophilia and Systemic Symptoms) is a drug hypersensitivity reaction poorly known by paediatricians. It occurs within 1 to 8 weeks of treatment. Clinical features associate in variable patterns, fever, rash, lymphadenopathies, arthritis and potentially life-threatening damage (hepatitis, nephritis, pneumonitis), hyperleucocytosis and eosinophilia. This condition must be early recognized in order to immediately stop suspect drugs. A 6.5 year old girl had a febrile rash, hyperleucocytosis, lymph nodes and cytolitic hepatitis probably due to phenobarbital. Diagnosis of DRESS syndrome was performed only 13 days after the beginning of the eruption. Evolution was favorable but characterized by the recurrence of the febrile eruption with pleuritis. DRESS syndrome is a well described disease that occurs during treatment with a number drugs, particularly anti-epileptic drugs. Steroid therapy and immunoglobulins are proposed for treatment but have not been evaluated.     

Stratégies de prévention de l’obésité chez l’enfantPrevention strategies of childhood obesity

Considering the high prevalence and the increasing trends, obesity is now considered as a public health problem in numerous countries. The main aim of the National Program of Nutrition and Health is to stop the increasing prevalence of childhood obesity. In this frame work, a group of experts has established a new presentation of the corpulence curves, adapted for clinical practice, to define normal weight and obesity. Weight status is now currently assessed on the basis of weight and height measurements, after computing the Quetelet index or body mass index (BMI) corresponding to weight (m) divided by square of height (weight/height2). As body proportion varies during growth, age must be taken into account. Various curves were published. In 1982, based on the French sample of the international growth study, BMI curves were published. They were revised in 1991. The third and 97th centiles define the normal weight range. Overweight is defined by BMI values greater than the 97th centile. In the year 2000, a new international definition was established. Two centiles were constructed to define overweight and obesity. The new BMI charts adapted for clinical practice, proposed by the French National program of nutrition and health, include the French reference curves plus the centile defining obesity in the international definition. Thus, in the new French charts, the area above the 97th centile is split in two levels (degree 1 obesity and degree 2 obesity). Drawing the BMI curve for each child, like drawing weight and height curves, is a simple act which can be done routinely. The age at adiposity rebound (an indicator predicting the risk of adult obesity) can be read from the curve. It allows to identify an early phase of obesity development, even at the time when overweight is not yet clinically visible. When obesity appears clearly, the identification is easy. The use of BMI curves is particularly useful in two situations: (1) in very young overweight children, the curves allow to identify children who have a real risk of developing obesity. (2) By the age of 6 years, when due to normal physiological variations, clinical assessment can be misleading. The BMI curves allow to identify children at risk. When a child is identified as having a real risk of obesity, simple preventive measures, adapted for each subject, could avoid a development toward massive obesity, which may become difficult to reduce if managed too late.     

Données actuelles sur la clinique de l’autisme

Autism is a neurodevelopmental disorder diagnosed on the basis of three behaviorally altered domains: social deficits, impaired language and communication, and stereotyped and repetitive behaviors. The early recognition of the disorder, as early as 2 years, is an important challenge, because early treatments are more efficient in helping children to develop their adaptation skills, allowing their better integration in the society, with less suffering and a lower level of handicap. Therefore, we describe the symptoms that may lead first degree practioners to suspect autistic disorders as early as possible, and how they can help the children and their parents to be directed to the appropriate services for diagnostic and treatment.     

Attachement désorganisé et sémiologie autistique

This paper firstly presents a literature review of the relationships between autistic symptomatology and disorganized attachment. Both have close semiological features but are distinct dimensions, arising within different context and may therefore be associated and be mistaken one for another. Recognition of their difference has important implications for both prognosis and treatment as for working alliance with parents. The discussion is highlighted by the case of Harry, who presented with a picture of autistic-like symptomatology but linked in fact with a disorganized attachment. His favourable evolution and the precisely acknowledged changes with this child during a four years treatment in a day care facility give weight to this hypothesis. An important therapeutic work can be done when early relation troubles exist between a mother and her child. The maternal perinatal depression screening and the child attachment troubles’ prevention are necessary, as it is often the case with migrant and impoverished population as recent meta-analyses show the impact of accumulated psychosocial risk factors on the increase of attachment disorganization.     

Allergologie pédiatrique. Quoi de neuf ? Une revue de la littérature internationale d''octobre 2002 à septembre 2003

Most epidemiological studies published in 2002-2003 confirm, in a large number of children, the results of previous studies. Most important results show that the risk of severe and persistent atopy and/or asthma is significantly higher in children with numerous risk factors than in children with a limited number of risk factors. Moreover, risks of severity and persistence are increased in children with early onset allergic disease, and with severe symptoms at the time of diagnosis. Effects of early exposure to furred pets are related to the degree of exposure at home, but are also modulated by the degree of exposure in the general population. In contrast with previous results, a large pediatric study shows that, at the age of 5 years, the prevalence of atopic diseases is inversely correlated with the number of vaccine infections. The efficacy of sublingual-swallow hyposensitization is long-lasting (up to 4-5 years after the discontinuation of treatment) in children with asthma due to house dust mite allergy. In contrast, individualized homeopathy, as an adjunct in the treatment of childhood asthma, is not superior to placebo in improving the quality of life of children with mild to moderate asthma. Supplementation of infant formulas with viable but not heat-inactivated probiotic bacteria is beneficial in the management of atopic dermatitis and cow's milk allergy. Finally the prevalence of peanut allergy has significantly increased between 1989 and 1994-1996 in European children, and at present, in France, the management in schools of children with food allergy is clearly inadequate.     

Treatment of non-metastatic,non-seminomatous malignant germ-cell tumours in childhood: Experience of the “Société Française D'oncologie Pédiatrique” MGCT 1985–1989 study

The members of the French Society of Pediatric Oncology treated, between January 1985 and June 1989, 67 cases of non-metastatic, non-seminomatous malignant germ cell tumours (nSGCT) in sites other than the brain. They used a clinical pre- and post-surgical TNM-type classification in order to standardize the treatment regardless of tumour site. The intensity of the treatment was decreased in comparison with the previous regimen (elimination of adriamycin, reduction in the length of treatment). The actuarial 2-year disease-free survival rate is 80%; results are excellent for patients with clinical stage I and II tumours and permit cure with moderate chemotherapy, avoiding undesirable late effects. On the other hand it is inadequate for patients with stage III suggesting that the initial chemotherapy should be intensified for these latter patients in future protocols. © 1993 Wiley-Liss, Inc.     

Pediatric radiologists: who we are and what we do. Results of a membership survey of the Society for Pediatric Radiology--1999

BACKGROUND: There is a need for reliable monitoring of workforce trends in the field of pediatric radiology by the Society for Pediatric Radiology. In addition, the Society should periodically assess itself as to its mission and relevance to its members via membership surveys. OBJECTIVE: The Membership Committee of the Society for Pediatric Radiology, 1999, conducted a 54-question survey to determine the makeup of its members, job profiles, satisfaction with services of the Society for Pediatric Radiology, and its official journal, Pediatric Radiology. MATERIALS AND METHODS: Seven hundred fifty surveys were given to active members of the Society for Pediatric Radiology in the United States and Canada. There were 275 surveys returned for an overall response of 37%. RESULTS: Mean age of members is 48 years with 99% of respondents working. Membership is 34% female and 66% male. Women members of the Society are younger (45 vs 49 years, P = 0.0012) and work less hours (47.8 vs 51.0, P = 0.0135) than men. Fifty-seven percent of respondents practice in a freestanding children's hospital, 29% in a "children's hospital within a hospital," and 14% are in community hospitals or an office-based practice. Eighty-two percent of the responding pediatric radiologist's time is spent in performing examination on children, with only 18% spent on adult work or administration. Forty-eight percent work at more than one office. Sixty-one percent worked evenings or weekends, excluding night call. Two hundred twenty-two of 275 respondents had received a Certificate of Added Qualification. The meeting "for CME credit" was considered the most important benefit of Society membership. Most respondents read select articles in Pediatric Radiology. There was sentiment to decrease esoteric case reports in favor of review articles. Only 19% of respondents submitted their articles to Pediatric Radiology initially. CONCLUSION: Pediatric radiologists are a diverse membership with the common goal of advocating for healthcare and imaging in children. Survey information given to the leadership of the Society will help the organization remain responsive to its members.