The Italian version of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ).

We report herein the results of the cross-cultural adaptation and validation into the Italian language of the parent's version of two health related quality of life instruments. The Childhood Health Assessment Questionnaire (CHAQ) is a disease specific health instrument that measures functional ability in daily living activities in children with juvenile idiopathic arthritis (JIA). The Child Health Questionnaire (CHQ) is a generic health instrument designed to capture the physical and psychosocial well-being of children independently from the underlying disease. The Italian CHAQ was already published in the literature and was therefore revalidated while the Italian CHQ was fully cross culturally adapted with 3 forward and 3 backward translations, and than validated. A total of 1,192 subjects were enrolled: 404 patients with JIA (16% systemic onset, 31% polyarticular onset, 21% extended oligoarticular subtype, and 32% persistent oligoarticular subtype) and 788 healthy children. The CHAQ clinically discriminated between healthy subjects and JIA patients, with the systemic, polyarticular and extended oligoarticular subtypes having a higher degree of disability, pain, and a lower overall well-being when compared to their healthy peers. Also the CHQ clinically discriminated between healthy subjects and JIA patients, with the systemic onset, polyarticular onset and extended oligoarticular subtypes having a lower physical and psychosocial well-being when compared to their healthy peers. In conclusion the Italian version of the CHAQ-CHQ are reliable, and valid tools for the functional, physical and psychosocial assessment of children with JIA.

Responsiveness of outcome measures in juvenile chronic arthritis. Italian Pediatric Rheumatology Study Group.

OBJECTIVE: To examine the responsiveness of the disease activity measures more commonly used in juvenile chronic arthritis (JCA) clinical trials. METHODS: Data were obtained from an open-label, non-controlled, multicentre trial designed to investigate the efficacy of methotrexate (MTX) in children with JCA. Outcome measures, including physician and parent global assessments, functional ability measures, articular variables, and laboratory indicators of systemic inflammation, were assessed at baseline and after 6 months of MTX treatment in 132 patients. Responsiveness of endpoint variables was evaluated by assessing the effect size (ES) and the standardized response median (SRM). RESULTS: Physician and parent global assessments were the more responsive instruments, showing ES and SRM above 1.0. Erythrocyte sedimentation rate, C-reactive protein, functional status measures and articular variables showed intermediate responsiveness. Morning stiffness, haemoglobin and platelet count were the least responsive instruments. CONCLUSION: The results of our analysis indicate that subjective estimations of the disease activity, either by the physician or parents, are the most responsive instruments in the assessment of the therapeutic response in children with JCA. The responsiveness of outcome measures in JCA should be further investigated in prospective controlled studies.

Performance of the preliminary definition of improvement in juvenile chronic arthritis patients treated with methotrexate. Italian Pediatric Rheumatology Study Group.

OBJECTIVE: To investigate the performance of the core set of outcome measures and the preliminary definition of improvement (PDI) in the assessment of response to methotrexate (MTX) treatment in children with juvenile chronic arthritis (JCA). METHODS: Data were obtained from an open label, non-controlled trial designed to investigate the efficacy of MTX in children with JCA. All patients had the core set of variables assessed at baseline and after six months of treatment. Variables in the core set are: (1) physician global assessment of disease activity; (2) parent or patient (if appropriate in age) global assessment of overall well being; (3) functional ability; (4) number of joints with active arthritis; (5) number of joints with limited range of motion; (6) erythrocyte sedimentation rate. The PDI specifies that to be classified as improved, a patient must show at least 30% improvement from baseline in three of any six variables in the core set, with no more than one of the remaining variables worsening by more than 30%. RESULTS: A total of 111 JCA patients were included in the study. According to the PDI, after six months of MTX treatment 73 patients (66%) were classified as improved and 38 (34%) as not improved. Among the core set variables, parent assessment detected the highest percentage of patients improved (72%) and functional assessment the lowest (37%). CONCLUSION: The PDI identifies about two thirds of patients with JCA treated with low dose MTX as improved. This proportion is similar to that expected to improve based upon a previous controlled study of low dose, oral MTX and provides preliminary evidence of the definition's validity.

Oral versus intramuscular methotrexate in juvenile chronic arthritis. Italian Pediatric Rheumatology Study Group.

OBJECTIVE: To compare the efficacy and safety of methotrexate (MTX) after oral and intramuscular administration in children with juvenile chronic arthritis (JCA). METHODS: Pediatric rheumatology centers in Italy participated in this short-term, prospective, open trial. Each investigator was allowed to choose the oral or intramuscular route of administration according to his personal preference in everyday clinical practice. Patients enrolled by each center were given MTX through the same method of administration. All patients received 10 mg/m2 of MTX each week for six months. RESULTS: A total of 257 patients with JCA (127 treated orally and 130 intramuscularly) were enrolled in the trial by 11 Italian centers. The response rate after 6 months of MTX therapy was 58% in the oral and 61% in the intramuscular cohort. The frequency of adverse side effects did not differ significantly between the two treatment groups. CONCLUSION: The results of this study suggest that MTX at the conventional dose regimen is equally effective and has a similar safety profile in children with JCA when administered orally or by intramuscular injections.

Dose-dependent effects of deflazacort and prednisone on growth and skeletal maturation.

Deflazacort (DFZ), a new glucocorticoid which has recently become available, is expected to have less negative effects on growth and skeletal maturation than conventional steroids, in children treated long term. To verify this hypothesis, a multicentre trial was organized to evaluate the effects of DFZ vs prednisone (PDN) on statural growth and skeletal maturation in a group of prepubertal children requiring glucocorticoid therapy for at least 6 months/year. The results of an analysis of 55 children (aged 3-12 years, 24 with connective tissue disease and 31 with kidney glomerular disorders) treated randomly with either DFZ (31 patients) or PDN (24 patients) and followed for a mean period of about 22 months (16 months under steroid therapy) are presented. The observation period was split up into the following phases according to dose and administration regimen: daily, high-dose therapy; alternate-day, high-dose therapy; low-dose therapy; suspension of treatment. The height, statural age, skeletal age and body weight velocities (i.e. the increase/year) were considered. In spite of large intra-individual and inter-individual variability, the results suggest that DFZ has a lower negative impact on indicators of growth. During high-dose daily administration, the height velocity tended to be lower in the PDN group and the impairment of skeletal maturity was significantly less for DFZ than for PDN. During an alternate-day regimen, height velocity was slightly higher in the PDN group and skeletal age velocity was higher in the DFZ group. It seems that steroid effects on statural growth and bone maturation occur in parallel.(ABSTRACT TRUNCATED AT 250 WORDS)

[Clinical assessment of a group of children with juvenile rheumatoid arthritis receiving long-term treatment with oral gold salt]. / Valutazione clinica su un gruppo di bambini affetti da artrite reumatoide giovanile in terapia con sali d'oro orali a lungo termine.

The authors assess the efficacy of gold salt treatment for juvenile rheumatoid arthritis. The study was carried out on 16 children suffering from mono-pauciarticular, polyarticular and systemic arthritis. Treatment consisted of the administration of auranofin alone in a group of 8 children and auranofin associated to corticosteroids in a second group of 8 children. A marked improvement in clinical conditions was observed with slight transitory side effects at follow-up after 12 and 24 months.

Statural growth and skeletal maturation in rheumatic prepubertal children treated with a third generation glucocorticoid (deflazacort) versus prednisone. An interim study.

Deflazacort, a new glucocorticoid (DFZ) which has recently become available, is known to have lower adverse effects on the skeletal metabolism and is expected to inhibit growth to a lesser extent in long-term treated children than earlier cortisone analogues. With the aim of verifying this hypothesis a multicenter study was planned to compare the effects of deflazacort and prednisone on linear growth and skeletal maturation in a group of prepubertal children requiring chronic steroid therapy. The data presented in this interim analysis refer to 24 children (11 females and 13 males ranging in age from 2.4 to 11.8 yrs) with rheumatoid arthritis (18), systemic lupus erythematosus (4) or dermatomyositis, selected from the total sample of 65 subjects included in the trial. They were randomly allocated to DFZ or PDN treatment and received the minimum effective dose of either steroid for at least 6 months per year. Longitudinal height measurements were obtained with standard instruments and techniques, and the bone age was assessed according to Greulich and Pyle. The following indicators of growth retardation were considered: bone age delay (difference between bone age and chronological age), statural age delay (statural age, with respect to the 50th percentile minus the chronological age) and statural age loss (statural age with respect to the individual height percentile at the first observation minus the chronological age).(ABSTRACT TRUNCATED AT 250 WORDS)

[Therapeutic strategy in the acute onset phase of infantile rheumatoid arthritis. Comparison of the main anti-inflammatory agents]. / Strategie terapeutiche nella fase acuta d'esordio dell'artrite reumatoide infantile. Confronto tra i principali presidi antiflogistici.

The effectiveness of some anti-inflammatory drugs has been compared in a series of 35 patients suffering from infant rheumatoid arthritis at onset and at the third month of disease. The results show that the clinical response to the various therapeutic protocols differs with the protocol and the stage of the disease in consideration, that disease monitoring in the early stage benefits from the serological examinations and that there is a low incidence of significant side-effects.

[Accidental inhalation of a foreign body. Presentation of 2 cases with atypical symptoms]. / Inalazione accidentale di corpo estraneo. Presentazione di due casi a sintomatologia atipica

Problems related to the inhalation of foreign bodies by children are discussed and two cases with unusual symptom pictures are presented. It is shown that some instances of acute respiratory or cardiorespiratory insufficiency of unknown origin in infancy may in fact be attributable to a foreign body in the respiratory system.