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Objective: To delineate urine biomarkers that forecast response to therapy of lupus nephritis (LN)., Methods: Starting from the time of kidney biopsy, patients with childhood-onset systemic lupus erythematosus who were diagnosed with LN were studied serially. Levels of 15 biomarkers were measured in random spot urine samples, including adiponectin, α-1-acid glycoprotein (AGP), ceruloplasmin, hemopexin, hepcidin, kidney injury molecule 1, monocyte chemotactic protein-1, lipocalin-like prostaglandin D synthase (LPGDS), transforming growth factor-β (TGF-β), transferrin, and vitamin D binding protein (VDBP)., Results: Among 87 patients (mean age 15.6 yrs) with LN, there were 37 treatment responders and 50 nonresponders based on the American College of Rheumatology criteria. At the time of kidney biopsy, levels of TGF-β (p < 0.0001) and ceruloplasmin (p = 0.006) were significantly lower among responders than nonresponders; less pronounced differences were present for AGP, hepcidin, LPGDS, transferrin, and VDBP (all p < 0.05). By Month 3, responders experienced marked decreases of adiponectin, AGP, transferrin, and VDBP (all p < 0.01) and mean levels of these biomarkers were all outstanding (area under the receiver-operating characteristic curve ≥ 0.9) for discriminating responders from nonresponders. Patient demographics and extrarenal disease did not influence differences in biomarker levels between response groups., Conclusion: Low urine levels of TGF-β and ceruloplasmin at baseline and marked reduction of AGP, LPGDS, transferrin, or VDBP and combinations of other select biomarkers by Month 3 are outstanding predictors for achieving remission of LN. If confirmed, these results can be used to help personalize LN therapy.

Objective: To (1) estimate the health-related quality of life (HRQOL) of children with childhood-onset systemic lupus erythematosus (cSLE) and compare it to that of normative cohorts; (2) assess the relationship of HRQOL with cSLE disease activity and damage; and (3) determine the effects of changes of disease activity on HRQOL., Methods: Patients with cSLE (n = 98) followed every 3 months completed HRQOL measures, the Pediatric Quality of Life Inventory Generic Core scale (PedsQL-GC), the Rheumatology Module (PedsQL-RM), and the Child Health Questionnaire (CHQ). The British Isles Lupus Activity Group Index (BILAG) was used to measure organ-system-specific disease activity. Physicians rated the course of cSLE between visits., Results: At baseline, mean (standard deviation, SD) score [parent report] of the PedsQL-GC and the PedsQL-RM was 75 (17) and 79 (14), respectively; the mean (SD) of the CHQ physical summary score (CHQ-PHS) was 49 (7) and that of the CHQ psychological summary score was 42 (12). Higher BILAG scores, especially in the general, musculoskeletal, neurological, and vascular, but not the mucocutaneous, renal, cardiovascular, or hematological BILAG domains, were associated with a significantly lower HRQOL. Patients with damage had lower HRQOL than those without damage. All HRQOL measures included were at most modestly responsive to clinically important changes with cSLE., Conclusion: HRQOL with cSLE is significantly lower than that reported in healthy populations. Organ-specific involvement with cSLE has a differential effect on HRQOL. Higher disease activity and damage are associated with significantly lower HRQOL as measured by the PedsQL-RM and the CHQ-PHS, and worsening of cSLE leads to a further decline.

Objective: The Childhood Health Assessment Questionnaire (CHAQ) is a commonly used measure of disability and physical function for children with juvenile rheumatoid arthritis (JRA), whose scores range between 0 (no disability) and 3 (very severe disability), with a smallest potential difference in the CHAQ score of individuals at 0.125. We estimated minimal clinically important differences (MCID) of the CHAQ for worsening and improvement that were actually experienced by children with JRA using patient, parent, and clinical perspectives., Methods: Changes in CHAQ scores were calculated for parent (n = 92) and patient ratings (children age > or = 8 yrs only; n = 67) between subsequent clinic visits. Changes in patient well being and disease activity and the occurrence of flare or important improvement between visits served as external standards for the MCID. MCID were defined as the median changes of the CHAQ scores of individual patients who had a minimal important improvement or worsening between visits., Results: The median change in CHAQ scores of patients who rated themselves or were rated by others as unchanged was often 0. Depending on the external standard used, the MCID for improvement of the CHAQ was -0.188 at most, while the MCID for worsening was at most +0.125., Conclusion: The MCID of the CHAQ for both improvement and worsening are often at or close to the level of the smallest potential difference, suggesting that the CHAQ is relatively insensitive to important short term changes in children with JRA. This may warrant a change in the calculation of the global CHAQ score, or the development of more sensitive functional measures.

Objective: To determine the frequency and severity of adverse reactions associated with high dose intermittent intravenous corticosteroids (IVCS) in children with rheumatic disease., Methods: Prospective documentation of adverse reactions associated with IVCS given to 213 pediatric rheumatology patients over a 4 year period., Results: Forty-six of the 213 children (22%) reported an adverse reaction. The 46 patients received 2622 doses of IVCS. Twenty-one patients (10% of all patients studied) had behavioral changes, including altered mood (14), hyperactivity (4), psychosis (2), disorientation (1), and sleep disturbances (3). Nonbehavioral adverse reactions included headache (5.2%), abdominal complaints (4.7%), pruritus (4.2%), vomiting (3.8%), hives (2.3%), hypertension (2.3%), bone pain (1.5%), dizziness (1.5%), fatigue (1%), lethargy (1%), hypotension (1%), tachycardia (1%), hyperglycemia (1%), fracture (1%), tremor (0.5%), anaphylaxis (0.5%), ulcer (0.5%), and "gray appearance" (0.5%). Using chi-squared analysis, there were no statistical differences in ethnicity (p = 0.54) or diagnosis (p = 0.46) between patient groups, with or without adverse reactions. There was a significant statistical association between history of drug induced cutaneous reaction and adverse reactions to IVCS (p < 0.01)., Conclusion: IVCS are associated with a spectrum of adverse reactions in children with rheumatic disease, of which volatile behavior is the most frequent. Children with a history of drug induced cutaneous reaction are more likely to have an adverse reaction to IVCS.

The use of steroids combined with cytotoxic drugs has increased in the last decade. The concomitant increase of opportunistic infections has contributed significantly to morbidity and mortality of patients treated with immunosuppressive agents. We describe a child with dermatomyositis who developed disseminated Nocardia brasiliensis infection while receiving steroids and methotrexate. Infectious etiology was established by gram stain. The patient was treated successfully. Disseminated Nocardia brasiliensis infection is rare with a high reported mortality. Diagnosis may be delayed secondary to insidious onset, similarity of clinical manifestations to other pathogens and slow growth in routine culture media. Nocardia should be considered early in the evaluation of infection in patients treated with immunosuppressive agents.