Childhood dermatomyositis. Clinical course and long-term follow-up

The presentation, clinical course, and long-term follow-up (3-22 years) of 39 children with dermatomyositis followed from 1962-1982 is presented. The medical course of these patients was complicated by respiratory diseases (20%), gastrointestinal diseases (24%), and calcinosis (30%). Patients presenting prior to 1972 received a wide variety of treatments. Since 1972, treatment has consisted of long-term prednisone, supplemented in some patients with azathioprine. Ten patients died (eight of whom were first seen before 1972) of complications after intestinal perforation (5) or aspiration pneumonia (5). Of 29 survivors, three have persistent calcinosis and/or contractures. Improved outcome since 1972 probably relates to better clinical assessment, management of complications, and regulation of drug therapy.     

Childhood dermatomyositis and polymyositis. Treatment with methotrexate and prednisone.

The conditions of three children with dermatomyositis and one child with polymyositis were treated for nine to 31 months with combined prednisone and intravenous methotrexate (1 mg/kg/wk) when prednisone alone was ineffective in controlling the disease or when there were substantial steroid-related toxic effects. All children showed a major clinical improvement within three months despite concomitant reduction of the prednisone dose. Three children completely recovered; one patient relapsed and died. The toxic effects of methotrexate included elevated liver transaminases (3/4), nausea (2/4), abdominal pain (2/4), bone pain (2/4), mild neutropenia (1/4), and mild pruritus (1/4). Intravenous methotrexate is an effective adjunct to steroid therapy in the treatment of steroid-resistant or life-threatening dermatomyositis-polyositis or dermatomyositis-polymyositis complicated by severe steroid-related effects.     

Childhood dermatomyositis associated with intracranial tumor and liver cysts.

We present a girl with dermatomyositis, liver cysts and choroid plexus papilloma who was treated and followed for 7 years. Muscle histology revealed an inflammatory muscle disease and similar changes were detected in a brain tumor that was surgically removed at onset. Western blot analysis of the muscle revealed severely reduced calpain-3 protein. She was treated with pulse methylprednisolone treatment (800 mg i.v. for 4 days) followed by oral prednisone treatment (16 mg on alternate day) for 14 months, which improved muscle strength. Moreover, the cystic liver formations disappeared during steroid treatment. This is an unusual association of muscular disorder, steroid-responsive liver cysts, intracranial tumor and secondary calpain-3 deficiency. We speculate that this association is not coincidental, but mediated by an autoimmune attack against an antigen that is shared among the target tissues.     

Hepatosplenic cat scratch disease treated with corticosteroids.

A 3 year old girl with hepatosplenic cat scratch disease developed prolonged fever unresponsive to treatment with multiple antibiotics, including gentamicin, azithromycin, rifampin, and ciprofloxacin. Fever resolved with corticosteroid treatment.     

Unusual clinical course in a child with cystic fibrosis treated with fat emulsion.

A child diagnosed as having cystic fibrosis by customary criteria has been given regular parenteral soya oil emulsion from near birht. Sweat tests have improved, pancreatic achylia was relieved, and the child at present remains entirely well. Correction of the essential fatty acid deficiency found in cystic fibrosis may prevent some of the manifestations of the disease.     

儿童寡肌病性皮肌炎合并肺间质病变2 例报告

目的探讨托珠单抗对儿童寡肌病性皮肌炎合并肺间质病变的疗效。方法回顾分析2例寡肌病性皮肌炎合并肺间质病变患儿的临床特点及治疗与预后,并复习相关文献。结果男女各1例,女性患儿10岁11个月、男性患儿8岁5个月,起病时均有气促,但无肌肉损害的临床表现;均有典型皮疹,但肌力及肌张力正常。实验室检查以血清铁蛋白、乳酸脱氢酶、谷氨酸氨基转移酶及天冬氨酸氨基转移酶升高为主,肌酸肌酶除首次入院时稍高,复查始终在正常范围。高分辨CT示肺间质病变。临床诊断为寡肌病性皮肌炎合并肺间质病变。女性患儿经大剂量激素、环磷酰胺、环孢素、吡菲尼酮及丙种球蛋白等治疗无效死亡。男性患儿在常规激素治疗的基础上,加用托珠单抗(240 mg/次,2次),病情稳定,随访复查各指标均在正常范围内。结论儿童寡肌病性皮肌炎临床表现以及实验室检查结果不典型,死亡发生率高。联合托珠单抗治疗有效。     

Bone density in asthmatic children treated with inhaled corticosteroids.

Volumetric trabecular bone mineral density of the lumbar spine (vTBMD) and distal radius (rTBMD) were measured in 20 prepubertal white asthmatic children treated with moderate to high doses of inhaled corticosteroids. The median standard deviation score for vTBMD (0.20, -0.56 to 2.09) and rTBMD (-0.04, -0.82 to 1.39) were within the normal range.     

Severe anorexia nervosa treated with total parenteral nutrition. Clinical course and influence on clinical chemical analyses

Severe anorexia nervosa in which psychiatric treatment has failed is a life-threatening condition since the degree of emaciation can be profound. Nine young women with a weight loss of 25-50% of their habitual weights were given total parenteral nutrition (TPN) over 3-8 weeks through a tunneled central venous silicone catheter. They initially received a mean of 55 kcal/kg body weight/24 hours increasing to 65-70 kcal/kg body weight during the first week. The initial bradycardia and hypotension were normalized and body weight increased with a mean of 2.5 kg/week. All patients showed a general somato-psychic improvement after two weeks of TPN. The electrolyte disturbances seen at the beginning were normalized during the first weeks of treatment and the previously reported life-threatening electrolyte disturbances and dehydration in connection with TPN treatment were not seen. The only adverse effect registered was a rise in liver enzymes in two patients who were given greater than 80 kcal/kg and day. With a reduction of the caloric supply these changes normalized, and TPN could be continued. After 3-8 weeks the oral intake had normalized and TPN was discontinued. This study confirms that TPN can be used successfully and with minimal risk in severe anorexia nervosa until the patients have normalized their oral intake.     

Monitoring of side effects in patients treated with corticosteroids of cytotoxic drugs (author's transl)]

Side effects have to be monitored carefully in all patients receiving drugs profoundly affecting their metabolism. Corticosteroids produce the well-known Cushing's syndrome. Children rarely develop gastric ulcers or disturbances of haemostasis. If steroids are administered for more than one year, the eyes have to be examined for signs of cataract. Rarely, the pseudotumor cerebri syndrome (increased intracranial pressure) may be observed. A monitoring schedule adapted from adult medicine for paediatric purposes is proposed in the text. Nowadays, mostly combinations of cytotoxic drugs are used. Often, doses are as high as the patient can tolerate them. Effects of these drugs are especially marked upon rapidly proliferating tissues such as the haematopoietic system and gastrointestinal mucosa. Immunosuppression may have very serious consequences. Infections caused by organisms usually considered as little pathogenic (e.g., pneumocystis carinii) may become deleterious; some infections may remain concealed or produce fever as the only symptom. Particular side effects of certain cytotoxic drugs are described in the text. Many therapeutic regimens are now standardized, as are monitoring schedules. Frequency and nature of the necessary controls depend upon the disease treated and the kind of therapy applied, as well as on its intensity. Both hospital and family doctors are indispensable for this surveillance; they must remain in close contact with each other.     

Exhaled nitric oxide fractions are well correlated with clinical control in recurrent infantile wheeze treated with inhaled corticosteroids

Ghdifan S, Verin E, Couderc L, Lubrano M, Michelet I, Marguet C. Exhaled nitric oxide fractions are well correlated with clinical control in recurrent infantile wheeze treated with inhaled corticosteroids.
Pediatr Allergy Immunol 2010: 21: 1015–1020.
© 2010 John Wiley & Sons A/S Fractional exhaled nitric oxide (FeNO) is a non‐invasive marker of bronchial inflammation in asthma. However, the interest of FeNO measurement remained limited in infantile wheeze. The aim of this prospective study was to evaluate the feasibility and reproducibility of FeNO off‐line measurement in very young children with recurrent wheeze and to assess whether clinical control of infantile wheeze correlates with FeNO levels. Two exhalation samples were collected in mylar balloon during quite tidal breathing. FeNO measurements were performed off‐line by a NO analyzer. The participating patients were aged ≤36 months, wheezes had started before the age of 24 months, and they were receiving maintenance treatment with inhaled corticosteroids for at least 3 months duration. The studied population comprised of 40 uncontrolled infants with persistent wheezy respiratory symptoms, median age 14.5 months, and 40 with optimal controlled infantile wheeze, median age 14 months. The reproducibility was excellent (r = 0.95; p < 0.0001). There was a significant difference in FeNO levels between the groups of persistent wheeze and well‐controlled infants: 19.8 (2.5–99.3) ppb vs. 7.7 (0.6–29.5) ppb, p < 0.0001. At a FeNO level >15 ppb, the predictive values for uncontrolled disease were as follows: positive predictive value = 65%, negative predictive value = 90%. FeN0 levels were not increased by atopy or passive tobacco. Off‐line assessment of FeNO is feasible, reproducible, and well accepted in wheezy very young children. Optimal clinical control of infantile wheeze appeared to be associated with the control of bronchial inflammation when evaluated by FeNO measurements.     

Impaired growth in children with asthma during treatment with conventional doses of inhaled corticosteroids

We describe 6 (4F, 2M) prepubertal children with moderate asthma diagnosed at a mean age of 2.8 years. All patients were treated with inhaled corticosteroids in a dose of between 300 and 800 mcg of beclomethasone diproprionate (becotide) daily, given either as an aerosol or rotahaler. Mean height velocity SDS decreased from −0.8(range +0.5 to −2.0)to −3.2(range −1.3 to −4.8) when the dose was increased. Alternatively, when the dose was reduced or stopped, mean height velocity SDS increased from −3.2 (range −2.0 to −4.8) to +0.8 (range −1.2 to +2.7). Careful assessment of height velocity is indicated in all children receiving treatment with inhaled corticosteroids.     

Childhood sporadic pheochromocytoma: clinical profile and outcome in 19 patients

Sporadic pheochromocytoma is a rare tumor of childhood and accounts for less than 1% of cases of hypertension. We describe the presentation and outcome of 19 adolescents with sporadic pheochromocytoma seen over past 10 years at a tertiary care center in north India. The mean age (+/- SD) at presentation was 15.1 +/- 2.4 years with range from 9-18 years. The male to female ratio was 12:7. The lag time between onset of symptoms to diagnosis ranged from 1 month to 5 years with mean (+/- SD) of 1.09 +/- 1.02 years. The majority of children presented with hypertension and paroxysms. Paroxysms, characterized by the triad of headache, palpitations and sweating, was present in 13 (68%) of these patients. Twelve (63%) patients had postural fall in blood pressure, ten (53%) had abdominal pain, four (21%) had visual blurring, and three (16%) each had palpable abdominal mass and significant weight loss at presentation. Nausea and vomiting are common symptoms in children with pheochromocytoma and were present in six (32%) and three (16%) patients, respectively. Café-au-lait macule was present in only two (11%) patients. Urinary vanilyl mandelic acid (VMA) was found to be significantly high in ten (53%) patients, and urinary epinephrine and norepinephrine in eight (42%). Six (32%) patients had both VMA and urinary epinephrine and norepinephrine within normal limits and five (26%) had significant elevation of both. The tumor was localized by ultrasonography in 17 (89%) patients and by computed tomography in 18 (95%), and in one patient it was localized by 131I-MIBG scan. Sixteen (84%) patients had adrenal pheochromocytoma (including four with bilateral masses), while the remaining three (16%) had abdominal extra-adrenal pheochromocytoma originating from sympathetic ganglions. The mean (+/- SD) diameter of the tumor was 4.4 +/- 1.7 cm, ranging from 2.2-7.5 cm. Pre-operatively, hypertension was managed by phenoxybenzamine in six (32%), sustained release prazosin in 12 (63%), beta-blockers in 14 (74%), calcium channel blockers in 12 (63%), and angiotensin converting enzyme inhibitors and diuretics in only two (11%) patients. Eighteen (85%) patients underwent exploratory laparotomy for removal of the tumor. On follow-up, 13 (72%) patients became normotensive, while six (32%) patients continued to have hypertension. In conclusion, childhood pheochromocytoma is characterized by atypical symptomatology; ultrasonography is a useful modality in localizing the lesions in the majority of patients; surgery is rewarding in most patients.     

Long-term outcome in patients with juvenile dermatomyositis: A case series

BackgroundFollow-up of juvenile dermatomyositis (JDM) patients has demonstrated the impact of the disease on several organs in the long term.ObjectiveAs there is little information on the long-term outcome of JDM, we aimed to assess long-term outcomes in a series of JDM patients.MethodsAfter selection of JDM patients, a consultation with a dermatologist and a rheumatologist was held for each patient. Cutaneous, muscle, and disease damage was assessed using different validated scores including the abbreviated Cutaneous Assessment Tool (aCAT), 8-muscle Manual Muscle Testing (MMT8), Childhood Myositis Assessment Scale (CMAS), Myositis Damage Index (MDI), Childhood Health Assessment Questionnaire (CHAQ), and Health Assessment Questionnaire (HAQ). Long-term disease outcomes were recorded including growth and pubertal development, educational and vocational achievement, and development of comorbidities.ResultsSeven patients were included in the study. After a mean follow-up of 14.9 ± 8.8 years, the mean aCAT score was 0.57 ± 1.4 and only one patient had a positive aCAT activity score. The mean aCAT damage score was 1.4 ± 1.3 and five (71%) patients had a score of ≥ 1. Five (71.4%) patients had normal muscle strength with an MMT8 score of > 72, and none had severe muscle weakness (MMT8 ≤ 32, and CMAS < 35). The mean total extent of damage according to the MDI was ≥ 1 in five (71%) patients and mainly involved the skin. Two (29%) patients had mild disability according to the CHAQ/HAQ disability index. In terms of quality of life, no patient had a score of < 40 (1 SD below the mean for healthy controls).ConclusionsBased on validated cutaneous and musculoskeletal scores, our study demonstrated the good functional outcomes of JDM at long-term follow-up.     

Plasma xanthine levels in low birthweight infants treated or not treated with theophylline.

The use of theophylline in the management of apnoea in the newborn was studied in 33 preterm infants. Infants received a dose of 3 mg/kg, 13 of them every six hours, the remaining 20 every eight hours. All the infants had significantly fewer apnoeic episodes. In a pharmacokinetic study, the half life of theophylline was 30.3 +/- 7.2 hours and the clearance rate was 23.9 +/- 5.06 ml/kg per hour (means and SD). The plasma theophylline level remained constant at between 13 and 15 mg/l from the 5th day of treatment but, at the same time, the plasma levels of caffeine rose to a mean level of 4.4 mg/l. Caffeine was detectable in plasma at birth, and in preterm infants not receiving theophylline; plasma levels of caffeine tended to be similar to the levels in their mothers' milk. These observations have led to clear conclusions on the optimum timing and dosage of theophylline, and on the need to monitor plasma levels of both theophylline and caffeine in newborn infants treated with theophylline.     

A multicenter prospective randomized trial of corticosteroids in primary therapy for Kawasaki disease: clinical course and coronary artery outcome

OBJECTIVE: To investigate the role of corticosteroids in the initial treatment of Kawasaki disease (KD). STUDY DESIGN: Between September 2000 and March 2005, we randomly assigned 178 KD patients from 12 hospitals to either an intravenous immunoglobulin (IVIG) group (n = 88; 1 g/kg for 2 consecutive days) or an IVIG plus corticosteroid (IVIG+PSL) group (n = 90). The primary endpoint was coronary artery abnormality (CAA) before a 1-month echocardiographic assessment. Secondary endpoints included duration of fever, time to normalization of serum C-reactive protein (CRP), and initial treatment failure requiring additional therapy. Analyses were based on intention to treat. RESULTS: Baseline characteristics of groups were similar. Fewer IVIG+PSL patients than IVIG patients had a CAA before 1 month (2.2% vs 11.4%; P = .017). The duration of fever was shorter (P < .001) and CRP decreased more rapidly in the IVIG+PSL group than in the IVIG group (P = .001). Moreover, initial treatment failure was less frequent (5.6% vs 18.2%; P = .010) in the IVIG+PSL group. All patients assigned to the IVIG+PSL group completed treatment without major side effects. CONCLUSIONS: A combination of corticosteroids and IVIG improved clinical course and coronary artery outcome without causing untoward effects in children with acute KD.