Long term tapering versus standard prednisolone treatment for first episode of childhood nephrotic syndrome: phase III randomised controlled trial and economic evaluation.

OBJECTIVE: To determine whether extending initial prednisolone treatment from eight to 16 weeks in children with idiopathic steroid sensitive nephrotic syndrome improves the pattern of disease relapse. DESIGN: Double blind, parallel group, phase III randomised placebo controlled trial, including a cost effectiveness analysis. SETTING: 125 UK National Health Service district general hospitals and tertiary paediatric nephrology centres. PARTICIPANTS: 237 children aged 1-14 years with a first episode of steroid sensitive nephrotic syndrome. INTERVENTIONS: Children were randomised to receive an extended 16 week course of prednisolone (total dose 3150 mg/m2) or a standard eight week course of prednisolone (total dose 2240 mg/m2). The drug was supplied as 5 mg tablets alongside matching placebo so that participants in both groups received the same number of tablets at any time point in the study. A minimisation algorithm ensured balanced treatment allocation by ethnicity (South Asian, white, or other) and age (5 years or less, 6 years or more). MAIN OUTCOME MEASURES: The primary outcome measure was time to first relapse over a minimum follow-up of 24 months. Secondary outcome measures were relapse rate, incidence of frequently relapsing nephrotic syndrome and steroid dependent nephrotic syndrome, use of alternative immunosuppressive treatment, rates of adverse events, behavioural change using the Achenbach child behaviour checklist, quality adjusted life years, and cost effectiveness from a healthcare perspective. Analysis was by intention to treat. RESULTS: No significant difference was found in time to first relapse (hazard ratio 0.87, 95% confidence interval 0.65 to 1.17, log rank P=0.28) or in the incidence of frequently relapsing nephrotic syndrome (extended course 60/114 (53%) v standard course 55/109 (50%), P=0.75), steroid dependent nephrotic syndrome (48/114 (42%) v 48/109 (44%), P=0.77), or requirement for alternative immunosuppressive treatment (62/114 (54%) v 61/109 (56%), P=0.81). Total prednisolone dose after completion of the trial drug was 6674 mg for the extended course versus 5475 mg for the standard course (P=0.07). There were no statistically significant differences in serious adverse event rates (extended course 19/114 (17%) v standard course 27/109 (25%), P=0.13) or adverse event rates, with the exception of behaviour, which was poorer in the standard course group. Scores on the Achenbach child behaviour checklist did not, however, differ. Extended course treatment was associated with a mean increase in generic quality of life (0.0162 additional quality adjusted life years, 95% confidence interval -0.005 to 0.037) and cost savings (difference -£1673 ($2160; €1930), 95% confidence interval -£3455 to £109). CONCLUSIONS: Clinical outcomes did not improve when the initial course of prednisolone treatment was extended from eight to 16 weeks in UK children with steroid sensitive nephrotic syndrome. However, evidence was found of a short term health economic benefit through reduced resource use and increased quality of life. TRIAL REGISTRATION: ISRCTN16645249; EudraCT 2010-022489-29.

[Inapparent charges for the assistance to nephropathic patient on dialysis].

The increasing technological effectiveness has undoubtedly produced an improvement in clinical parameters of dialysis patients, but this satisfactory therapeutic result did not follow an adequate improvement in mortality or in the perception of quality of life as per patients. Furthermore, dialysis treatment is often associated with "inapparent charges" that reduce the perception of well-being, independently of clinical changes. Thirty years ago, we carried out a national survey on inapparent charges, which represent frustrating aspects that negatively affect patients' perception of their quality of life. Thirty years later, it seemed important for us to repeat the survey to understand if Italian legislative remodeling have introduced changes in procedures and social aspects of dialysis, as preservation of quality of life is an important aspect of the replacement treatment.

The role of the clinical health psychologist in the management of chronic paediatric illness. A report of 2 cases.

Behavioural problems in chronically ill children are common and adequate provision for holistic management is necessary. It appears preferable for psychological intervention to start not when the child is referred with behavioural problems but as soon as the medical condition is diagnosed. These children, and their families, are usually in need of psychological care from the earliest stages of the disease process. Two case reports are cited in this context. The role of the clinical health psychologist is emphasised.

Psychological adaptation of siblings of chronically ill children: research and practice implications.

Studies of the psychological adjustment of physically healthy siblings to their sibling's chronic illness indicate that there is no one-to-one correspondence between the presence of a chronic illness and risk for psychological disturbance in nonafflicted children. Although the presence of a chronic illness may increase siblings' subjective distress, effects of a chronic illness on the psychological adjustment of siblings are selective and vary with age, sex, and type of illness. Chronic illness is a stressor which, in interaction with other variables, may contribute to increased risk of psychological disturbance for some siblings. Although the variables which mediate the effects of a chronic illness on siblings are as yet poorly understood, the quality of family functioning and relationships has both direct and indirect effects on siblings and deserves primary consideration in the comprehensive care of chronically ill children. Future research might profitably focus on individual differences in sibling adaptation, especially on factors which contribute to positive adjustment, the role of the family context as a mediating influence, and evaluation of preventive interventions designed to enhance sibling adaptation.