The Provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: A prospective validation study

Objective. To validate a core set of outcome measures for the evaluation of response to treatment in patients with juvenile dermatomyositis (DM). Methods. In 2001, a preliminary consensus-derived core set for evaluating response to therapy in juvenile DM was established. In the present study, the core set was validated through an evidence-based, large-scale data collection that led to the enrollment of 294 patients from 36 countries. Consecutive patients with active disease were assessed at baseline and after 6 months. The validation procedures included assessment of feasibility, responsiveness, discriminant and construct ability, concordce in the evaluation of response to therapy between physicians and parents, redundancy, internal consistency, and ability to predict a therapeutic response. Results. The following clinical measures were found to be feasible, and to have good construct validity, discriminative ability, and internal consistency; furthermore, they were not redundant, proved responsive to clinically important changes in disease activity, and were associated strongly with treatment outcome and thus were included in the final core set: 1) physician`s global assessment of disease activity, 2) muscle strength, 3) global disease activity measure, 4) parent`s global assessment of patient`s well-being, 5) functional ability, and 6) health-related quality of life. Conclusion. The members of the Paediatric Rheumatology International Trials Organisation, with the endorsement of the American College of Rheumatology and the European Leauge Against Rheumatism, propose a core set of criteria for the evaluation of response of therapy that is scientifically and clinically relevant and statistically validated. The core set will help standardize the conduct and reporting of clinical trials and assist practitioners in deciding whether a child with juvenile DM has responded adequately to therapy.

C-Peptide Levels and Insulin Independence Following Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in Newly Diagnosed Type 1 Diabetes Mellitus

Context In 2007, the effects of the autologous nonmyeloablative hematopoietic stem cell transplantation (HSCT) in 15 patients with type 1 diabetes mellitus (DM) were reported. Most patients became insulin free with normal levels of glycated hemoglobin A(1c) (HbA(1c)) during a mean 18.8-month follow-up. To investigate if this effect was due to preservation of beta-cell mass, continued monitoring was performed of C-peptide levels after stem cell transplantation in the 15 original and 8 additional patients. Objective To determine C-peptide levels after autologous nonmyeloablative HSCT in patients with newly diagnosed type 1 DM during a longer follow-up. Design, Setting, and Participants A prospective phase 1/2 study of 23 patients with type 1 DM(aged 13-31 years) diagnosed in the previous 6 weeks by clinical findings with hyperglycemia and confirmed by measurement of serum levels of anti glutamic acid decarboxylase antibodies. Enrollment was November 2003-April 2008, with follow-up until December 2008 at the Bone Marrow Transplantation Unit of the School of Medicine of Ribeirao Preto, Ribeirao Preto, Brazil. Hematopoietic stem cells were mobilized via the 2007 protocol. Main Outcome Measures C-peptide levels measured during the mixed-meal tolerance test, before, and at different times following HSCT. Secondary end points included morbidity and mortality from transplantation, temporal changes in exogenous insulin requirements, and serum levels of HbA1c. Results During a 7- to 58-month follow-up (mean, 29.8 months; median, 30 months), 20 patients without previous ketoacidosis and not receiving corticosteroids during the preparative regimen became insulin free. Twelve patients maintained this status for a mean 31 months (range, 14-52 months) and 8 patients relapsed and resumed insulin use at low dose (0.1-0.3 IU/kg). In the continuous insulin-independent group, HbA(1c) levels were less than 7.0% and mean (SE) area under the curve (AUC) of C-peptide levels increased significantly from 225.0 (75.2) ng/mL per 2 hours pretransplantation to 785.4 (90.3) ng/mL per 2 hours at 24 months posttransplantation (P<.001) and to 728.1 (144.4) ng/mL per 2 hours at 36 months (P=.001). In the transient insulin-independent group, mean (SE) AUC of C-peptide levels also increased from 148.9 (75.2) ng/mL per 2 hours pretransplantation to 546.8 (96.9) ng/mL per 2 hours at 36 months (P=.001), which was sustained at 48 months. In this group, 2 patients regained insulin independence after treatment with sitagliptin, which was associated with increase in C-peptide levels. Two patients developed bilateral nosocomial pneumonia, 3 patients developed late endocrine dysfunction, and 9 patients developed oligospermia. There was no mortality. Conclusion After a mean follow-up of 29.8 months following autologous nonmyeloablative HSCT in patients with newly diagnosed type 1 DM, C-peptide levels increased significantly and the majority of patients achieved insulin independence with good glycemic control. Trial Registration clinicaltrials.gov Identifier: NCT00315133

Relationship among eccentric hip and knee torques, symptom severity and functional capacity in females with patellofemoral pain syndrome

Objective: To determine the relationships between eccentric hip and knee torques, symptom severity and functional capacity in females with patellofemoral pain syndrome (PFPS). Design: Within-subject correlational study. Setting: University biomechanics laboratory. Participants: 10 females diagnosed with PFPS. Main outcome measures: Eccentric strength of the hip abductors and lateral rotators, and knee extensors were assessed using an isokinetic dynamometer. A 10-cm visual analog scale was used to determine usual knee pain in the last week. The Anterior Knee Pain Scale (AKPS) was used to determine the functional capacity of the patients. Results: The study found that the greater the eccentric knee extensor and hip lateral rotator torques, the higher the functional capacity of the patients (p = 0.02, r = 0.72; p = 0.02, r = 0.72). It was also shown that the greater hip lateral rotator torque, the less the usual pain reported in the last week (p = 0.004, r = -0.84). Despite the lack of statistical significance (p = 0.11), it was also found a modest negative relationship between the eccentric knee extensor torque and the usual pain reported in the last week (r = -0.56) that was considered clinically meaningful (d = 1.4). Conclusions: This study showed that eccentric knee extensor and hip lateral rotator torques were associated with functional capacity and pain level in females with PFPS. Further investigations should be carried out to verify the effects of an intervention program focused on the eccentric action of these muscles with respect to the symptoms in patients with PFPS. (C) 2011 Elsevier Ltd. All rights reserved.

Long-term effects of pharyngeal flaps on the upper airways of subjects with velopharyngeal insufficiency

Objectives: To investigate the long-term effects of pharyngeal flap surgery (PFS) on nasal and nasopharyngeal dimensions of patients with velopharyngeal insufficiency (VPI) and to correlate the findings with the onset of respiratory complaints after surgery. Design/Participants: Prospective study in 58 nonsyndromic patients with repaired cleft palate and VPI, evaluated 2 days before and 5 months (POST1) and 1 year (POST2) after PFS, on average. Patients were divided into two groups: one consisting of patients with postoperative respiratory complaints (RC group) and the other without complaints (NRC group). Interventions: Superiorly based PFS. Main Outcome Measures: Respiratory complaints (self reports of mouth breathing, snoring, and other sleep obstructive events) assessed at POST1 and POST2, and minimum nasal (NCSA) and nasopharyngeal (NPA) cross-sectional areas assessed by rhinomanometry at POST2. Results: Respiratory complaints were reported by 55% and 36% of the patients evaluated at POST1 and POST2, respectively. Posterior rhinomanometry showed a significant postoperative reduction of mean NCSA in the RC and NRC groups (p < .05), to subnormal levels in some of them. The decrease was more pronounced in the RC group. No significant changes in NCSA were observed by anterior rhinomanometry. Similar results were obtained when NPA was assessed by modified anterior rhinomanometry. Conclusion: In the long-term, PFS yielded a significant reduction in upper airways dimensions beyond what should be expected and associated with persistent respiratory complaints in some cases.

Esthetic Analysis of Gingival Components of Smile and Degree of Satisfaction in Individuals With Cleft Lip and Palate

Objective: To evaluate soft tissue characteristics in individuals with cleft lip and palate and the degree of satisfaction of these individuals after rehabilitation. Setting: Hospital for Rehabilitation of Craniofacial Anomalies, Brazil. Patients: Forty-five individuals with repaired complete unilateral cleft lip and palate, aged 15 to 30 years. Interventions: One hundred thirty-five frontal facial photographs were obtained at rest and in natural and forced smile. Specialists in periodontics evaluated the soft tissue characteristics. Both patients and specialists evaluated the smiles and scored them as esthetically unpleasant, acceptable, or pleasant. Main Outcome Measures: Comparison of the cleft area with the contralateral region was performed for evaluation of soft tissue. The results of the degree of satisfaction with smile were expressed as percentages and means. The findings between patients and periodontists experienced or inexperienced with cleft care were compared. Results: Statistically significant differences were observed for alveolar process deficiency and absence of papilla in the esthetic area between groups (p < .05). Results show 84.4% of individuals considered their smile as esthetically pleasant. Specialists in periodontics of both groups scored the natural smile and forced smile as esthetically acceptable. There was a statistically significant difference in the mean of patients compared with both groups of specialists in periodontics (p < .05). Conclusions: Evaluation and knowledge of the soft tissue characteristics is extremely important for successful rehabilitation. The esthetic values and degree of patient satisfaction are essential for treatment success, since smile reconstruction should be esthetically pleasant to the patient.

Survival of Dental Implants in the Cleft Area-A Retrospective Study

Objective To evaluate the survival rate of dental implants placed in the cleft area Design Retrospective study Setting Hospital for Rehabilitation of Craniofacial Anomalies, Brazil Institutional Tertiary Healthcare Center Patients 120 patients who received dental implants in the grafted cleft area in the years 1999 to 2005 Interventions Clinical data were evaluated from the records of 120 patients according to the following criteria placement grafted, cleft area, and age at surgery, age at placement of dental implants, site and dimension of implants, interval between placement of implants and the last clinical follow-up, and interval between placement and removal or indication for removal of implants Main Outcome Measures Percentage of survival rate of implants Results Mean age at placement of the bone graft was 17 6 years and 21 years at placement of implants A total of 123 cleft areas received secondary bone graft and bone graft to install implants (regraft) The mean survival rate was 34 months since placement of the implant to the last clinical follow-up and 26 months since placement of the prosthesis Seven dental implants were removed The survival rate since placement to the last clinical follow-up was 94 3% Conclusion Rehabilitation of the cleft area with dental implants is a viable and secure alternative, with good prognosis

Multidisciplinary case conference reviews: Improving outcomes for nursing home residents, carers and health professionals

Aim of study: This study sought to determine whether multidisciplinary case conference reviews improved outcomes for nursing home residents, and the effects of this team approach to resident care on carers, including the hands-on carers employed by the nursing home, and health professionals. Method: 245 residents of three Canberra nursing homes were enrolled in this non-randomised controlled trial. The intervention consisted of sessions of three case conference reviews held between 10/4/96 and 4/12/96. These sessions were attended by the General Practitioners (GPs) of the residents discussed, the GP project officer from the ACT Division of General Practice, a clinical pharmacist, senior nursing staff, other health professionals eg physiotherapist, and occasionally the resident concerned or their representative. At each review, a case presentation by the resident's GP was followed by a multidisciplinary discussion of all aspects, medical and non-medical, of the resident's care. The review concluded with a management plan for the resident. In total 75 residents were reviewed. Main outcome measures: Medication use and cost, and mortality. Results: One month after the reviews were completed comparisons between those who were reviewed and those who were not showed non-significant reductions in medication orders, medication cost, and mortality in the reviewed group. Many of the 92 recommendations in the management plans that were carried out benefited the residents (n=37) and/or carers (n=24). The responses of the GPs and the Directors of Nursing to the reviews were overwhelmingly positive. Conclusion: Recommendations arising from multidisciplinary case conferences were carried out to the benefit of patients and carers. Given the support shown by key stakeholders, multidisciplinary conferences should be used more.

The validity of self-reported energy intake as determined using the doubly labelled water technique

In the 1980s the development of the doubly labelled water (DLW) technique made it possible to determine the validity of dietary assessment methods using external, independent markers of intake in free-living populations. Since then, the accuracy of self-reported energy intake (EI) has been questioned on a number of occasions as under-reporting has been found to be prevalent in many different populations. This paper is a review of investigations using the DLW technique in conjunction with self-reported EI measures in groups including adults, children and adolescents, obese persons, athletes, military personnel and trekking explorers. In studies where a person other than the subject is responsible for recording dietary intake, such as parents of young children, EI generally corresponds to DLW determined energy expenditure. However, in instances where the subjects themselves report their intake, EI is generally under-reported when compared with energy expenditure. It was originally believed that this phenomenon of under-reporting was linked to increased adiposity and body size, however, it is now apparent that other factors, such as dietary restraint and socio-economic status, are also involved. This paper therefore aims to present a more comprehensive picture of under-reporting by tying in the findings of many DLW studies with other studies focusing particularly on the characteristics and mechanisms for under-reporting. Awareness of these characteristics and mechanisms will enable researchers to obtain more accurate self-reports of EI using all dietary recording techniques.

Effect of power Doppler and digital subtraction techniques on the comparison of myocardial contrast echocardiography with SPECT

Objective-To compare the accuracy and feasibility of harmonic power Doppler and digitally subtracted colour coded grey scale imaging for the assessment of perfusion defect severity by single photon emission computed tomography (SPECT) in an unselected group of patients. Design-Cohort study. Setting-Regional cardiothoracic unit. Patients-49 patients (mean (SD) age 61 (11) years; 27 women, 22 men) with known or suspected coronary artery disease were studied with simultaneous myocardial contrast echo (MCE) and SPECT after standard dipyridamole stress. Main outcome measures-Regional myocardial perfusion by SPECT, performed with Tc-99m tetrafosmin, scored qualitatively and also quantitated as per cent maximum activity. Results-Normal perfusion was identified by SPECT in 225 of 270 segments (83%). Contrast echo images were interpretable in 92% of patients. The proportion of normal MCE by grey scale, subtracted, and power Doppler techniques were respectively 76%, 74%, and 88% (p < 0.05) at > 80% of maximum counts, compared with 65%, 69%, and 61% at < 60% of maximum counts. For each technique, specificity was lowest in the lateral wail, although power Doppler was the least affected. Grey scale and subtraction techniques were least accurate in the septal wall, but power Doppler showed particular problems in the apex. On a per patient analysis, the sensitivity was 67%, 75%, and 83% for detection of coronary artery disease using grey scale, colour coded, and power Doppler, respectively, with a significant difference between power Doppler and grey scale only (p < 0.05). Specificity was also the highest for power Doppler, at 55%, but not significantly different from subtracted colour coded images. Conclusions-Myocardial contrast echo using harmonic power Doppler has greater accuracy than with grey scale imaging and digital subtraction. However, power Doppler appears to be less sensitive for mild perfusion defects.

Randomised controlled trial of telemedicine for new neurological outpatient referrals

Objective-To test the hypothesis that telemedicine for new patient referrals to neurological outpatients is as efficient and acceptable as conventional face to face consultation. Methods-A randomised controlled trial between two groups: face to face (FF) and telemedicine (TM). This study was carried out between a neurological centre and outlying clinics at two distant hospitals linked by identical medium cost commercial interactive video conferencing equipment with ISDN lines transmitting information at 384 kbits/s. The same two neurologists carried out both arms of the study. Of the 168 patients who were suitable for the study, 86 were randomised into the telemedicine group and 82 into the face to face group. Outcome measures were (I) consultation process: (a) number of investigations; (b) number of drugs prescribed; (c) number of patient reviews and (2) patient satisfaction: (a) confidence in consultation; (b) technical aspects of consultation; (c) aspects surrounding confidentiality. Diagnostic categories were also measured to check equivalence between the groups: these were structural neurological, structural non-neurological, nonstructural, and uncertain. Results-Diagnostic categories were similar (p>0.5) between the two groups. Patients in the telemedicine group had significantly more investigations (p=0.001). There was no difference in the number of drugs prescribed (p>0.5). Patients were generally satisfied with both types of consultation process except for concerns about confidentiality and embarrassment in the telemedicine group (p=0.017 and p=0.005 respectively). Conclusion-Telemedicine for new neurological outpatients is possible and feasible but generates more investigations and is less well accepted than face to face examination.

Single institution outcomes of treatment of severe aplastic anaemia

Background: In severe aplastic anaemia, the treatment of choice for young patients with a human leucocyte antigen-matched sibling is now established as allogeneic bone marrow transplantation (BMT). In older patients and in those without a matched sibling donor, immunosuppressive therapy is the usual first option. 'Alternative' marrow donors are emerging as an option for those without a matched sibling donor. Aims: To review 10 years of local experience in treating severe aplastic anaemia with BMT and immunosuppressive therapy with emphasis on long-term outcomes. Methods: A retrospective analysis was performed of all patients with severe aplastic anaemia presenting to the Royal Brisbane and Royal Children's Hos- pitals between 1989 and 1999. Data were abstracted regarding patient demographics, pretreatment characteristics and outcome measures, including response rates, overall survival and long-term complications. Results: Twenty-seven consecutive patients were identified, 12 treated with immunosuppression alone and 15 with BMT. In these two groups, transfusion independence was attained in 25% and 100%, respectively, with overall survival being 36% and 100%, respectively. Those treated with immunosuppression were significantly older (median 41.5 versus 22 years, P = 0.008). Long-term survivors of either treatment had extremely low morbidity. Three patients carried pregnancies to term post-transplant. Three patients received alternative donor BMT with correspondingly excellent survival. Conclusions: Patients treated with allogeneic BMT for severe aplastic anaemia enjoyed extremely good long-term survival and minimal morbidity. Patients treated with immunosuppressive therapy had a poorer outcome reflecting their older age and different usage of therapies over the past decade. Optimal treatment strategies for severe aplastic anaemia remain to be determined.

Alcoholic liver disease: A clinical series in an Australian private practice

Background and Aim: The published literature on alcoholic liver disease (ALD) in Australia lacks a large clinical series out of private practice as distinct from hospital-based hepatology referral units. This series describes the presentation and clinical features of ALD in a consecutive series out of metropolitan private practice in Australia. Methods: A retrospective descriptive study by case-note review found 297 cases of ALD at a Brisbane practice over 20 years. The main outcome measures were: clinical features and stage at presentation, reasons for referral, and the predictive value of aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio. Results: Most patients (57.9%) had no symptoms of liver disease and 29 patients (9.8%) had neither symptoms nor signs. Cirrhosis was found in 41% of patients and hepatitis-fibrosis was found in 26% of patients. The male to female (M: F) ratio was 4.7:1. The AST/ALT ratio was not reliably predictive of ALD stage. The average reported daily alcohol intake was 131 g. Females drank less on average and presented a more vigorous clinical picture. Conclusions: This series presents the spectrum of ALD in a metropolitan Australian private practice. Many patients are asymptomatic on presentation. All heavy drinkers should be targeted for early investigation without waiting for volunteered symptoms or abnormal physical signs. The male to female ratio in ALD is higher than hitherto reported. The AST/ALT ratio is not generally applicable in the staging of ALD. The differences from hospital series data suggest the demography and epidemiology of ALD in Australia are incomplete, and further study is warranted. (C) 2001 Blackwell Science Asia Pty Ltd.

Towards a definition of "difference" in osteoarthritis

To assess existing information regarding detectable differences in osteoarthritis (OA), a systematic literature search was conducted up to December 1999. Thirty-three articles were considered methodologically relevant to the definition and categorization of detectable differences in OA. It was determined that the musculoskeletal literature contains a wealth of information that relates to observed changes, much of which is derived from the clinical trials literature, but there have been relatively few methodological studies that have systematically evaluated the nature, categorization, and relevance of the change. Furthermore, most of those that have been published take the perspective of an individual or groups of experts other than that of the patient. This summary of the current literature reveals that the diverse sources of information go part way towards developing an understanding of detectable differences and their importance in the area of OA research and clinical practice. Stakeholders' interests as well as factors that modulate perceptions of importance need to be taken under consideration. In particular, the patient's perspective of the importance of change at an individual level requires further evaluation. This area of clinical research is relatively underdeveloped, but there is considerable opportunity for progress.

Swallowing outcomes following laryngectomy and pharyngolaryngectomy

Objectives: To determine the incidence of dysphagia (defined as the inability to manage a diet of normal consistencies) at hospital discharge and beyond 1 year post-surgery and examine the impact of persistent dysphagia on levels of disability, handicap, and well-being in patients. Design: Retrospective review and patient contact. Setting: Adult acute care tertiary hospital. Patients: The study group, consecutively sampled from January 1993 to December 1997, comprised 55 patients who underwent total laryngectomy and 37 patients who underwent pharyngolaryngectomy with free jejunal reconstruction. Follow-up with 36 of 55 laryngectomy and 14 of 37 pharyngolaryngectomy patients was conducted 1 to 6 years postsurgery. Main Outcome Measures: Number of days until the resumption of oral intake; swallowing complications prior to and following discharge; types of diets managed at discharge and follow-up; and ratings of disability, handicap, and distress levels related to swallowing. Results: Fifty four (98%) of the laryngectomy and 37 (100%) of the pharyngolaryngectomy patients experienced dysphagia at discharge. By approximately 3 years postsurgery, 21 (58%) of the laryngectomy and 7 (50%) of the pharyngolaryngectomy patients managed a normal diet. Pharyngolaryngectomy patients experienced increased duration of nasogastric feeding, time to resume oral intake, and incidence of early complications affecting swallowing. Patients experiencing long-term dysphagia identified significantly increased levels of disability, handicap, and distress. Patients without dysphagia also experienced slight levels of handicap and distress resulting from taste changes and increased durations required to complete meals of normal consistency. Conclusions: The true incidence of patients experiencing a compromise in swallowing following surgery has been underestimated. The significant impact of impaired swallowing on a patient's level of perceived disability, handicap, and distress highlights the importance of providing optimal management of this negative consequence of surgery to maximize the patient's quality of life.

Physical outcome measurements via the Internet: reliability at two Internet speeds

We have developed a software application to enable interactive rehabilitation via the Internet. The reliability of the telemedicine application was examined by comparing it with face-to-face assessment. The physical outcome measures assessed were knee range of motion, quadriceps muscle strength, limb girth and an assessment of gait. One therapist performed both in-person and Internet-based measurements of all outcome measures on 20 normal subjects. There was good agreement between the two techniques (the 95% limits of agreement included zero for all the variables studied). Internet assessments were conducted at two bandwidths: ISDN at 128 kbit/s and the telephone network at 17 kbit/s. Bandwidth had no significant influence on any of the measures. This study suggests that Internet-based physiotherapy interventions delivered to the home are suitable for further development.