Complement deficiency in pediatric-onset systemic lupus erythematosus.

BACKGROUND: Pediatric-onset systemic lupus erythematosus (pSLE) accounts for about 10%-20% of all patients with SLE. Deficiencies in early complement components of the classical pathway are the strong genetic risk factor for the development of SLE. In this study, clinical and laboratory manifestations of both complement-deficient and normal complement pSLE patients were compared. MATERIALS AND METHODS: To investigate clinical and immunological manifestations of pSLE in Iran, 36 consecutive pSLE patients (onset before 18 years) who were followed up over a period of 2 years, were studied. Complement C1q and C2 levels were measured using radial immunodifusion assay and complement C3 and C4 levels were measured using nephelometry. Medical records were retrospectively evaluated from patient database of Children Medical Center Hospital. Data were assessed through descriptive analysis (confidence interval = 95%), paired t-test, and Pearson correlation test. RESULTS: Twenty-one patients (58%) had at least one component of complement deficiency. Ten patients (27%) had low C1q level, 11 patients (30.5%) had low C2, nine patients (25%) had low C3, and four patients (11%) had low C4 level. Serum level of complement in pSLE was significantly lower than the control group, except C4 (P = 0.005). The low C1q patients had an earlier age of onset of disease (P < 0.0001). The cutaneous manifestations were more frequent and much more severe in pSLE with low complement (100% vs. 73%). The frequency of renal and musculoskeletal symptoms was equal, but renal morbidity was more common in pSLE with low complement. Positivity for anti-ds-DNA was less common in pSLE with low complement (71% vs. 86%). CONCLUSION: In pSLE patients with early disease onset and more aggressive SLE manifestations and negative anti-ds-DNA test, complement deficiency should be considered.

Safety and efficacy of mitoxantrone in pediatric patients with aggressive multiple sclerosis.

OBJECTIVE: The purpose of this study was to assess the safety and efficacy of mitoxantrone (MX) in pediatric patients with aggressive multiple sclerosis (MS). METHODS: A retrospective analysis on pediatric MS patients treated with MX was performed with regards to demographic/clinical parameters and magnetic resonance imaging (MRI) findings. RESULTS: 19 definite pediatric MS cases with mean ± SD age of 15.4 ± 2.8 years underwent 20 mg MX for control of their severe/frequent relapses, high EDSS score or new and active brain MRI lesions. After a median [IQR] follow-up period of 30[12-60] months, 14 cases (73%) were relapse free; the EDSS score decreased by at least 0.5 in 16 cases (84.2%); and gadolinium-enhancing lesion volume fell by 84.2% in 16 cases. Adverse events included nausea and vomiting, fatigue, alopecia, palpitation, cardiomyopathy and mild leukopenia. All adverse events were mild and transient. CONCLUSION: Our results suggest MX is a good candidate for treatment of children with worsening RRMS and SPMS. Recommendations regarding patient selection, treatment administration, and close follow-up should be considered. Continuing research is needed to establish its efficacy and safety profile in a multinational collaboration with careful follow-up of adverse events.

Pediatric multiple sclerosis with primary progressive course--report of a retrospective cohort study in Iran.

The aims of this study were to suggest the rate of primary progressive (PP) subtype of pediatric onset multiple sclerosis (MS) in Isfahan, Iran, and describe its clinical and paraclinical features. The data of patients were retrieved from Isfahan MS Society (IMSS) database from April 2003 to August 2011. Among 3,843 MS patients of Isfahan who have been registered in IMSS, 260 patients had onset symptom when younger than the age of 18 years, of whom, 11 patients had a PP course (4.23%). The mean age at onset in pediatric primary progressive multiple sclerosis (PPMS) was 16 years (range: 13 to 17) with female preponderance (2.66:1) and disease duration of 4.73 ± 3.03 years. Ataxia was the most frequent initial symptom (7/11). Additionally, the mean Expanded Disability Status Scale and progression index was 4.31 ± 0.60 and 1.50 ± 1.21, respectively. Cerebrospinal fluid analysis showed oligoclonal immunoglobulin G bands in seven patients. Magnetic resonance imaging (MRI) demonstrated periventricular lesions in all 11 patients and spinal lesions in 9 patients. Exposure to parental smoking was recorded in seven individuals. In conclusion, PPMS is an uncommon subtype of pediatric onset MS. Cerebral lesions are more common MRI findings in pediatric PPMS patients than that in adults. The course of PPMS seems to be more progressive in the pediatric population than in adults.