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Brian M Feldman

Publications and source records attributed to Brian M Feldman.

At least 19 recordsLinked to original sources
PubMed2007

Validation of the oral mucositis assessment scale in pediatric cancer.

BACKGROUND: Our objective was to examine the construct validity of the Oral Mucositis Assessment Scale (OMAS) in children receiving doxorubicin chemotherapy. METHODS: Children between 6 and 18 years of age with cancer receiving doxorubicin-containing chemotherapy were included. OMAS was measured on days 7, 10, 14, and 17 after chemotherapy. Other measures of mucositis obtained concurrent with OMAS were the World Health Organization (WHO) mucositis scale and pain visual analogue scale (VAS). We also recorded analgesia administration. RESULTS: Sixteen children were studied for 45 post-chemotherapy cycles and 156 OMAS assessments were performed. OMAS was moderately correlated with WHO scores (r = 0.56; P = 0.0006) whereas correlation with the pain VAS was fair (r = 0.37; P = 0.002). OMAS also had fair correlation with the number of doses of topical analgesia (r = 0.43; P = 0.001) and with the cumulative dose of opioid analgesia (r = 0.38; P = 0.003). CONCLUSIONS: The OMAS is valid for use in mucositis clinical trials for children at least 6 years of age.

Administration, Oral↗
PubMed2006

The Quality of My Life questionnaire: the minimal clinically important difference for pediatric rheumatology patients.

OBJECTIVE: To determine parent-child agreement for the Quality of My Life (QoML) questionnaire. To establish construct validity of the QoML questionnaire. To determine the minimal clinically important difference (MCID) for the Quality of Life (QOL) and Health-Related Quality of Life (HRQOL) scales. METHODS: A total of 136 families of children with inflammatory arthritis were interviewed. The QoML questionnaire was completed for the child's current state of health, and under 2 hypothetical scenarios, where (1) there is a hypothetical small improvement, and (2) there is a hypothetical small deterioration in health. The differences between the original QOL and HRQOL scores and hypothetical improvement and deterioration scores, respectively, were calculated to give MCID scores. RESULTS: In total, 131 families completed the questionnaires. Intraclass correlation coefficients for parent proxy report and patient self-report of the QOL and HRQOL were 0.63 and 0.40, respectively. Correlations of QOL with pain and disease severity were moderately negative (r = -0.55 and -0.56, respectively, p < 0.0001). Correlations of HRQOL with pain and disease severity were strongly negative (r = -0.66 and r = -0.68, respectively, p < 0.0001). The MCID for improvement on the QOL was 7 mm, and for the HRQOL 11 mm. The MCID for deterioration in QOL was -33 mm, and for HRQOL -38 mm. CONCLUSION: The QoML questionnaire demonstrated fair parent-child agreement and good convergent construct validity. MCID scores will enable clinicians to interpret QoML questionnaire results in a clinically meaningful way.

Arthritis↗
PubMed2006

Advances in the treatment of juvenile dermatomyositis.

PURPOSE OF REVIEW: Juvenile dermatomyositis is a rare chronic inflammatory disease that primarily affects the muscles and skin. Immunosuppressive therapy has played a very important role in reducing mortality rates and morbidity. The review focuses on the spectrum of medications currently used in the treatment of juvenile dermatomyositis, highlighting new advances and unanswered questions. RECENT FINDINGS: Data regarding the treatment of juvenile dermatomyositis come almost entirely from retrospective studies with relatively small numbers of patients. Corticosteroids continue to be the accepted first-line therapy. Evidence that the addition of methotrexate at initiation of treatment allows corticosteroids to be tapered more rapidly with good outcomes exists. High-risk, refractory patients may benefit from intravenous cyclophosphamide. Results in refractory patients treated with rituximab are also encouraging. Topical immunosuppressant agents have been largely disappointing in treating rash. The effect and role of exercise in the treatment and rehabilitation of patients with juvenile dermatomyositis is an interesting new area of research. SUMMARY: Future research in the treatment of juvenile dermatomyositis should focus on improving the understanding of disease course and its predictors such that treatment protocols can be developed to provide the most benefit and least amount of medication toxicity for the individual patient.

Child↗
PubMed2006

N-of-1 trials: innovative methods to evaluate complementary and alternative medicines in pediatric cancer.

N-of-1 randomized controlled trials (RCTs) are randomized trials conducted within individuals and may be an attractive methodology for conducting studies of complementary and alternative medicine (CAM) in pediatric oncology. These trials may be used to determine the efficacy of an intervention in an individual, or multiple N-of-1 RCTs may be combined to estimate a population effect. There are many potential advantages to the use of N-of-1 RCTs with CAM in pediatric cancer. These advantages include the ability to determine whether CAM is effective in a specific child. In addition, the N-of-1 RCT allows parents and children to voice preferences about treatment options and allows them to directly participate in balancing adverse events and therapeutic benefits. Also, in estimation of population effects, combining multiple N-of-1 RCTs tends to require smaller sample sizes than do traditional parallel-group designs. However, there also may be several challenges to the conduct of such a trial. The use of N-of-1 RCTs may be very beneficial in evaluating CAM therapies in pediatric cancer. However, careful consideration of the advantages and disadvantages of such a design should be undertaken prior to initiating an N-of-1 RCT.

Child↗
PubMed2006

Corticosteroid treatment of refractory Kawasaki disease.

OBJECTIVE: To review the indications for corticosteroids in patients with Kawasaki disease (KD) treated by pediatric rheumatologists in Canada and to determine their efficacy on fever in patients with refractory KD. METHODS: All practicing pediatric rheumatologists in Canada identified KD patients treated with corticosteroids and completed a standard data form that included demographics, clinical and laboratory features, imaging studies, and therapeutic interventions, by chart review. RESULTS: Thirty-two patients with KD (14 female; 18 male: mean age 4.6 years) were treated with corticosteroids. Corticosteroids were used in 26 patients (81%) for persistent fever despite treatment with intravenous immunoglobulin (IVIG) (refractory KD), 5 patients (19%) for congestive heart failure, and 1 patient for persistent acute phase symptoms other than fever. The 26 patients with refractory KD are the primary subject of this report. Twenty-two patients (85%) had rapid, sustained resolution of fever after corticosteroids. There were no serious reported adverse effects. Eight patients (31%) treated with corticosteroids developed coronary artery (CA) aneurysms and 9 (35%) developed CA dilatations without aneurysms. Of those who developed CA aneurysm, 4 had aneurysms detected prior to IV methylprednisolone (MP) on echocardiograms performed on days 6-27 (mean day 13) of illness. The remaining 4 patients had CA aneurysm detected after IVMP therapy, on echocardiograms performed on days 13-49 (mean day 23) of illness, 1-25 days (mean 9 days) after IVMP. In patients with one year or more of followup, 46% had resolution of CA abnormalities. CONCLUSION: Corticosteroids are effective in the treatment of fever in most patients with IVIG-refractory KD. A multicenter prospective study is needed to determine the effect of corticosteroids on CA outcome in patients with refractory KD.

Child↗
PubMed2005

Clinical responsiveness of self-report functional assessment measures for children with juvenile idiopathic arthritis undergoing intraarticular corticosteroid injections.

OBJECTIVE: The Childhood Health Assessment Questionnaire (CHAQ), Juvenile Arthritis Functional Assessment Report (JAFAR), and Juvenile Arthritis Functional Status Index (JASI) are widely used functional measures for juvenile idiopathic arthritis (JIA) that differ in content, format, and completion time. We compared the responsiveness and child-parent agreement of the JAFAR, CHAQ, and JASI in a prospective, multicenter study. METHODS: Children and adolescents from 5 rheumatology centers were enrolled. Subjects were about to undergo therapy (intraarticular corticosteroid injections [IAS] and methotrexate or hip surgery (MTX/hip]) expected to produce a functional improvement. All subjects were studied before the intervention and at 6 weeks and 6 months posttreatment. At each study visit, the 3 measures were administered in randomized, balanced order to both parents and children. RESULTS: A total of 92 subjects (mean age 12.8 years) were enrolled in the study, 74 of which were in the IAS group. The responsiveness of all 3 measures was moderate to strong. The standardized response mean at 6 weeks for the IAS group on the JAFAR, CHAQ, and JASI was 0.41 (95% confidence interval [95% CI] 0.18, 0.64), 0.70 (95% CI 0.47, 0.93), and 0.36 (95% CI 0.13, 0.59), respectively. The CHAQ was somewhat more responsive to change at 6 weeks (IAS group: relative efficiency 0.34 [JAFAR], 0.27 [JASI]), but less responsive at 6 months (MTX/hip group: relative efficiency 5.1 [JAFAR], 3.9 [JASI]). All 3 questionnaires showed acceptable parent-child agreement, and overall, there were few differences between the 3 questionnaires. CONCLUSION: The functional outcome measures currently used for JIA are all adequately responsive for use in trials or in the clinic setting. The choice of which measure to use should therefore be based on the time available for completion, the intended clinical/research use, and the depth of content required.

Adolescent↗
PubMed2005

Long-term outcomes in juvenile dermatomyositis: how did we get here and where are we going?

Juvenile dermatomyositis (JDM) affects two to four children per million. Prior to treatment with corticosteroids, JDM had a high mortality rate (> 30%) and left 50% of those who survived with serious permanent impairments. After the introduction of corticosteroids, mortality rapidly dropped to less than 10%, and is currently reported to be less than 2% to 3%. Because most children now survive this illness, there is greater interest in long term outcomes. However, review of the literature shows while much is known about mortality, relatively little is known about long term outcomes such as physical function, quality of life, pain, educational and vocational achievement, patient satisfaction, and ongoing disease activity. Furthermore, the literature that has been published has not typically used the same outcomes making comparisons across studies difficult. Current efforts to identify key outcomes and validate measures for those outcomes will allow researchers in the future to provide this much needed information.

Adolescent↗
PubMed2005

Seven items were identified for inclusion when reporting a Bayesian analysis of a clinical study.

OBJECTIVE: (1) To generate a list of items that experts consider most important when reporting a Bayesian analysis of a clinical study, (2) to report on the extent to which we found these items in the literature, and (3) to identify factors related to the number of items in a report. STUDY DESIGN AND SETTING: Based on opinions from 23 international experts, we determined the items considered most important when publishing a Bayesian analysis. We then performed a literature search to identify articles in which a Bayesian analysis was performed and determined the extent to which we found these items in each report. Finally, we examined the relationship between the number of items in a report and journal- and article-specific attributes. RESULTS: Our final set of seven items described the prior distribution (specification, justification, and sensitivity analysis), analysis (statistical model and analytic technique), and presentation of results (central tendency and variance). There was >99% probability that more items were reported in studies with a noncontrolled study design and in journals with a methodological focus, lower impact factor, and absence of a word count limit. CONCLUSION: We developed a set of seven items that experts believe to be most important when reporting a Bayesian analysis.

Bayes Theorem↗
PubMed2005

Inflammatory myopathies in children.

Juvenile idiopathic inflammatory myopathies are rare conditions that are probably autoimmune in nature. Juvenile dermatomyositis (JDM) is the most common inflammatory myopathy. This article describes a recent patient who presented with typical JDM and uses her case to discuss aspects of the childhood inflammatory myopathies.

Adolescent↗
PubMed2005

Treating children with arthritis: towards an evidence-based culture.

We live in a culture of evidence-based medicine. Many areas of medicine have embraced this culture. However, for unusual diseases, like the childhood arthritides, there is little evidence. To provide this evidence a culture change must occur in pediatric rheumatology. The most convincing reason to make this change comes from the field of childhood oncology. Through successive clinical trials, collaborative oncology study groups have discovered cures for many childhood cancers. The most convincing studies are randomized trials; however, these are difficult to do. Collaborative trial groups and innovative designs are needed for an acceptable culture change in childhood arthritis. Recently a number of collaborations have been developed to help further the study of pediatric rheumatology. The best known are the Pediatric Rheumatology Collaborative Study Group in North America, and the Paediatric Rheumatology International Trials Organization in Europe, South America, and Asia. A new North American collaborative study group has formed--the Childhood Arthritis and Rheumatology Research Alliance (CARRA)--to undertake investigator-initiated clinical trials. These groups might potentially lead the way to a new evidence-based culture for childhood arthritis.

Adolescent↗
PubMed2005

Eye findings in patients with juvenile dermatomyositis.

OBJECTIVE: Reports of eye involvement in juvenile dermatomyositis (JDM), including significant retinopathy with visual loss, have led some to recommend routine formal ophthalmologic assessments for all patients with JDM at diagnosis. Our objective was to document the frequency and spectrum of eye involvement in patients followed in a single clinic caring for children with JDM. METHODS: A chart review was conducted of formal ophthalmologic consultation notes for patients with JDM followed at the Hospital for Sick Children between 1981 and 2002. RESULTS: Ophthalmologic assessments were found for 82 of 108 patients with JDM. The mean age at diagnosis of JDM was 7.0 years and 68.3% were female. Forty-five patients (55.6%) had abnormal eye examinations. Lid manifestations, found in 37 patients (45.7%), were the most common abnormality. Fourteen patients (17.1%) had corticosteroid-induced cataracts. Two patients had retinal abnormalities; one had a small retinal hemorrhage, the other an incidental chorioretinal scar. Neither had impairment of vision. No patient had uveitis. CONCLUSION: Eyelid and lens abnormalities are common in patients with JDM, while retinopathy is rare. As lid lesions and cataracts are easily detected by non-ophthalmologists, and retinal lesions are rare, we feel that JDM patients without visual symptoms do not require routine formal ophthalmologic assessment for disease manifestations.

Adolescent↗
PubMed2004

Revised versions of the Childhood Health Assessment Questionnaire (CHAQ) are more sensitive and suffer less from a ceiling effect.

OBJECTIVE: To determine the relative discriminant validity of 3 new versions of the Childhood Health Assessment Questionnaire (CHAQ) and to determine the relative concordance between children and their parents. METHODS: The parents of 48 children with musculoskeletal disability and 101 nondisabled controls were given the CHAQ and the 3 revised versions in random order. Children older than 5 years also completed the questionnaires. RESULTS: All 3 new versions of the CHAQ were more sensitive at differentiating patients from controls (relative efficiency 1.35-1.65); the versions with 8 added items were even more sensitive (relative efficiency 1.79-2.32). The new versions of the CHAQ suffered less from a ceiling effect and were more normally distributed. Concordance between children and parents was moderate to high for all versions. CONCLUSION: Researchers and clinicians should consider using a revision of the CHAQ. By being more sensitive at differentiating patients, the revised versions of the CHAQ will allow fewer subjects to be studied and will be more able to detect differences between patients in the clinic.

Activities of Daily Living↗
PubMed2004

Inpatient versus outpatient management of low-risk pediatric febrile neutropenia: measuring parents' and healthcare professionals' preferences.

PURPOSE: Our primary objective was to describe and compare parents' and healthcare professionals' strength of preference scores for outpatient oral antibiotic relative to inpatient parenteral antibiotic treatment for low-risk febrile neutropenic children. Our secondary objective was to identify predictors of strength of preference for oral outpatient treatment. METHODS: Respondents were parents of children receiving cancer chemotherapy, and pediatric oncology healthcare professionals. First, the inpatient and outpatient options were described, and the respondent indicated their initially preferred option. The respondent next ranked how important seven factors (including "fear/anxiety" and "comfort") were in making their initial choice. The threshold technique was then used to elicit the respondent's strength of preference score for oral outpatient, relative to parenteral inpatient management. RESULTS: There were 75 parent and 42 healthcare-professional respondents. There was no significant difference (P =.08) in the proportions of parents (40 of 75; 53%) and healthcare professionals (30 of 42; 71%) who initially would choose outpatient management. For parents, stronger preference for oral outpatient therapy was associated with higher anticipated quality of life for the parent and child at home relative to hospital, lower importance rank for "fear/anxiety," and higher importance rank for "comfort." Conversely, for professionals, only lower importance rank for "fear/anxiety" was associated with higher strength of preference scores for outpatient oral antibiotic management. CONCLUSION: Only 53% of parents would choose outpatient oral antibiotic management for low-risk febrile neutropenia. Predictors of strength of preference scores for outpatient oral antibiotic relative to inpatient parenteral antibiotic treatment differed between parent and professional respondents.

Administration, Oral↗
PubMed2004

Normal scores for nine maneuvers of the Childhood Myositis Assessment Scale.

OBJECTIVE: To document and evaluate the scores that normal, healthy children achieve when performing 9 maneuvers of the Childhood Myositis Assessment Scale (CMAS). METHODS: A total of 303 healthy children, 4-9 years of age, were scored as they performed 9 CMAS maneuvers. The data were then evaluated to determine whether normal scores for some maneuvers are age and sex dependent. RESULTS: All children were able to achieve maximum possible scores for the supine to prone, supine to sit, floor sit, floor rise, and chair rise maneuvers. All but 2 4-year-olds achieved a maximum possible score for the arm raise/duration maneuver. Performance of the head lift and sit-up maneuvers varied significantly, depending primarily on age. Children in all age groups had less difficulty performing the leg lift than the head lift or sit-up. CONCLUSION: The normative data generated by this study are of value for interpreting the serial CMAS scores of children with idiopathic inflammatory myopathies.

Child↗
PubMed2004

Validation and clinical significance of the Childhood Myositis Assessment Scale for assessment of muscle function in the juvenile idiopathic inflammatory myopathies.

OBJECTIVE: To examine the measurement characteristics of the Childhood Myositis Assessment Scale (CMAS) in children with juvenile idiopathic inflammatory myopathy (juvenile IIM), and to obtain preliminary data on the clinical significance of CMAS scores. METHODS: One hundred eight children with juvenile IIM were evaluated on 2 occasions, 7-9 months apart, using various measures of physical function, strength, and disease activity. Interrater reliability, construct validity, and responsiveness of the CMAS were examined. The minimum clinically important difference (MID) and CMAS scores corresponding to various degrees of physical disability were estimated. RESULTS: The intraclass correlation coefficient for 26 patients assessed by 2 examiners was 0.89, indicating very good interrater reliability. The CMAS score correlated highly with the Childhood Health Assessment Questionnaire (C-HAQ) score and with findings on manual muscle testing (MMT) (r(s) = -0.73 and 0.73, respectively) and moderately with physician-assessed global disease activity and skin activity, parent-assessed global disease severity, and muscle magnetic resonance imaging (r(s) = -0.44 to -0.61), thereby demonstrating good construct validity. The standardized response mean was 0.81 (95% confidence interval 0.53, 1.09) in patients with at least 0.8 cm improvement on a 10-cm visual analog scale for physician-assessed global disease activity, indicating strong responsiveness. In bivariate regression models predicting physician-assessed global disease activity, MMT remained significant in models containing the CMAS (P = 0.03) while the C-HAQ did not (P = 0.4). Estimates of the MID ranged from 1.5 to 3.0 points on a 0-52-point scale. CMAS scores corresponding to no, mild, mild-to-moderate, and moderate physical disability, respectively, were 48, 45, 39, and 30. CONCLUSION: The CMAS exhibits good reliability, construct validity, and responsiveness, and is therefore a valid instrument for the assessment of physical function, muscle strength, and endurance in children with juvenile IIM. Preliminary data on MID and corresponding levels of disability should aid in the clinical interpretation of CMAS scores when assessing patients with juvenile IIM.

Adolescent↗
PubMed2004

Effect of intracranial bleeds on the health and quality of life of boys with hemophilia.

OBJECTIVES: To compare the health, physical function, and quality of life (QoL) of boys with hemophilia with and without a history of intracranial hemorrhage (ICH). STUDY DESIGN: Of 172 patients with hemophilia A or B, 18 (10%) had at least one episode of ICH. For outcome assessments, 16 of 18 (89%) boys with ICH and 32 controls, matched (1:2) for age and severity of hemophilia, were available. The outcome measures were neurologic function, physical function, and QoL. RESULTS: The median age of the boys at the first ICH was 5.9 months (range, 1 day to 2.7 years). Boys with ICH had a higher incidence of inhibitors and lower mean household income. Neurologic examination was abnormal in seven of 16 (44%) boys with ICH and nine of 32 (28%) controls (P=.3). The mean physical function in boys with ICH was lower (82%+/-25%) compared with controls (93.5%+/-12%, P=.045). The QoL was decreased in boys with ICH compared with controls (6.8+/-3.2 vs 8.5+/-1.4, P=.02), whereas health-related QoL was not significantly different between groups. CONCLUSION: The poorer long-term outcomes of boys with hemophilia appropriately treated for ICH, especially in the domain of QoL, suggest that new strategies to prevent ICH and to manage ICH effectively in this population are needed.

Adolescent↗
PubMed2004

Clinical and cost implications of target joints in Canadian boys with severe hemophilia A.

OBJECTIVE: To compare target joint-associated costs incurred by boys with severe hemophilia A 1 year before and 1 year after development of a target joint (pre-TJ, post-TJ). STUDY DESIGN: Resource utilization data were extracted retrospectively from medical and hemophilia clinic charts and patient diaries for 16 boys attending the Hospital for Sick Children (HSC)'s comprehensive care hemophilia program. Resources examined included drugs, medical care, hospitalization, laboratory tests, therapies, and transfusions received. All costs were figured using standard price lists and were discounted using an annual rate of 3%. RESULTS: Fifteen of the 16 boys developed at least one target joint, defined as three bleeds into any single joint within a consecutive 3-month period, at an average age of 54 months (range, 15-94 months), with ankles being most often affected, followed by elbows and knees (46% vs 28% and 23%, respectively). The total cost of treating a boy with on-demand Factor VIII (FVIII) increased by 119% after development of a target joint, from $20,091 (in 2002 Canadian dollars [$CDN]) in the year before to $43,890 in the year after target joint development. Factor VIII use accounted for 87% of the total cost in the year before target joint development and 93% in the year after. CONCLUSIONS: This study identified substantial increased costs of care associated with target joint development. This finding provides further support for more aggressive treatment aimed at reducing target joints-either more aggressive treatment of joint bleeds or institution of primary prophylactic therapy at an early age.

Adolescent↗