Patient Insight With Collagenase Treatment for Dupuytren: A Prospective Study.

PURPOSE: There appears to be controversy regarding differing patient and physician perceptions of adverse effects (AEs) in the treatment of Dupuytren disease with collagenase clostridium histolyticum (CCH). The aim of this study was to compare the number, type, and severity of AEs perceived and reported by patients and by their physician METHODS: To assess AEs following CCH injection in a standardized way, patients were given a list of predefined complications and asked to rate their severity on a 4-point Likert scale ranging from 1 (serious) to 4 (insignificant). RESULTS: Eighty-five patients were included. Patients reported fewer AEs than their physician (mean, 1.48 vs 2.18). There was no agreement between physician- and patient-reported AEs except for skin lacerations, which showed fair agreement (κ = 0.257). CONCLUSIONS: Patients and physicians differ in their evaluation of AEs due to CCH treatment in Dupuytren disease. A fair level of agreement was observed for skin lacerations. CLINICAL RELEVANCE: Greater consensus is needed when defining AEs associated with CCH in the treatment of Dupuytren disease.

A comparison of patient-reported outcome measures for dupuytren disease: A prospective view.

Treatment success in Dupuytren´s disease has traditionally been assessed by clinical examination and physical measures, but based on patient orientation, patient-reported outcome measures (PROMs) can be used to quantify treatment impacts and health-related quality of life. The aim of this study was to compare the most widely used PROMS in Dupuytren´s disease, their psychometric properties, and their association with objectively measured hand function. We show a prospective study in which two disease-specific questionnaires (URAM scale and SDSS) and two hand-specific questionnaires (briefMHQ and PEM) were administered before and a month after the treatment with collagenase. Psychometric properties (construct validity, internal consistency, test-retest reliability, internal and external responsiveness, reliability, and minimal clinically important difference (MCID)) were calculated for all the questionnaires. Ninety-two completed both sets of questionnaires. Pre- and post-treatment scores were strongly correlated (Spearman rho >0.6) for all questionnaires. High internal consistency (Cronbach alpha >0.88) was observed for all the questionnaires. Test-retest reliability was also significant, with an ICC of >0.50 in all cases. The MCID was similar for three of the questionnaires (URAM, 11.528/45=0.256; SDSS, 5.079/20=0.254; and PEM, 21.542/77=0.215) and somewhat lower for the briefMHQ (10.617/60=0.177). No correlations were observed between treatment outcome and treated hand (r = 0.107; p = 0.31), joint (r = 0.163; p = 0.12), or finger (r = -0.151; p = 0.15). In conclusion, we did not find that any one questionnaire performed better than the other in detecting perceived changes in health status among patients with Dupuytren´s disease treated with collagenase.

Sensitivity and Specificity of the Unité Rhumatologique Des Affections De La Main (URAM) Scale for Dupuytren Contracture: A Systematic Review and Meta-Analyses.

BACKGROUND: Unité Rhumatologique des Affections de la Main (URAM) is a novel and disease-specific questionnaire for Dupuytren contracture, a fibroproliferative disease that affects hands causing progressive contracture in flexion of the fingers. OBJECTIVES: To evaluate the sensitivity and specificity of the URAM scale in Dupuytren contracture. MATERIALS AND METHODS: We performed meta-analyses of 10 articles published in PubMed, Embase, Cochrane, Google Scholar, Latin American and Caribbean Health Sciences Literature (LILACS), and in various grey literature databases that describe the use of the URAM and Tubiana scales to assess treatment outcomes in Dupuytren contracture. We built three models: a hierarchical summary receiver operating characteristic (HSROC) model to determine the optimal threshold for defining Dupuytren contracture, a difference in means model to assess the magnitude of the effect of different treatment modalities, and a meta-regression model to determine the effect on patient quality of life questionnaires such as the URAM, according to variations in Tubiana scores after treatment. RESULTS: The HSROC and bivariate models showed a sensitivity of 80.23% (95% CI: 75.66 to 84.14) and an overall specificity of 2.61% (95% CI: 1.11 to 6.05). The second model showed an overall difference in means of 1.95 (95% CI: -2.86 to -1.04) for partial fasciectomy and collagenase Clostridium histolyticum (CCH) injections, and -1.30 (95% CI: -1.77 to -0.83) for partial fasciectomy, and -2.75 (95% CI: -4.73 to -0.78) for CCH. The coefficient obtained in the meta-regression model was -1.666 (95% CI: -4.183 to 0.851). CONCLUSION: The URAM scale is highly sensitive to changes in Dupuytren contracture but has low specificity. It also showed a strong correlation with worsening of finger contracture as measured by the Tubiana scale.

Neonatos en tratamiento con nutriciones parenterales individualizadas, candidatos a recibir nutriciones parenterales estandarizadas / Neonates treated with individualized parenteral nutrition who are candidates to receive standardized parenteral nutrition

OBJETIVO: cuantificar el número de pacientes neonatos en tratamiento con nutriciones parenterales individualizadas (NPI), candidatos a recibir nutriciones parenterales estandarizadas (NPE), así como el número de días. MATERIAL Y MÉTODOS: estudio prospectivo observacional y descriptivo de cohortes. Los criterios de inclusión fueron: pacientes neonatos con indicación de nutrición parenteral (NP) y prescripción individualizada. Los criterios de exclusión fueron: pacientes que no hubieran iniciado la diuresis, con necesidades nutricionales específicas, con alteraciones del equilibrio ácido-base y/o con contraindicación de la NPE. Se emplearon variables relacionadas con el paciente (sexo, peso, semanas de gestación y días de vida) y relacionadas con el tratamiento (aportes de la NPI). Fijando como criterio de conversión el volumen de NP, se calcularon los aportes teóricos con la NPE. El criterio para que un paciente fuera candidato a recibirla fue que todos los aportes teóricos estuvieran dentro de los requerimientos de referencia. RESULTADOS: se incluyeron 33 neonatos (9 mujeres) en tratamiento con NPI y con 94 prescripciones. La mediana de peso de los pacientes incluidos en el estudio fue de 2,14 (RIC: 0,9) kg, nacidos a las 35 (RIC: 3) semanas de gestación y en los que se inició NP entre los días 0 y 4. El 71 % (22/31) de los pacientes en el 54,1 % (46/85) de sus prescripciones fueron candidatos a recibir NPE administrada por vía central durante 1 a 8 días, mientras que ningún paciente fue candidato a recibirla por vía periférica. CONCLUSIONES: en nuestro centro, el 71 % de los pacientes nenonatos en tratamiento con NPI administrada por vía central son candidatos a recibir NPE, lo que fomenta la normalización del soporte nutricional en esta población

OBJETIVE: to quantify the number of neonates treated with individualized parenteral nutrition (IPN) who were candidates to receive standardized parenteral nutrition (SPN), and to calculate their treatment duration. MATERIAL AND METHODS: this was a prospective, observational, descriptive cohort study. Inclusion criteria were: neonates with indication of parenteral nutrition (PN) and individualized prescription. Exclusion criteria included: patients who had not started diuresis, with specific nutritional needs, altered acid-base balance, and/or contraindication to receive SPN. Included variables were patient-related (gender, weight, weeks of gestation, and days of life) and treatment-related regarding IPN composition. Setting the volume of PN as the conversion criterion, theoretical contributions were calculated with the SPN. The criterion for a patient to be a candidate to receive SPN was that all the theoretical contributions calculated were within the reference requirements range. RESULTS: a total of 33 neonates (9 women) received IPN with 94 prescriptions. The median weight of the patients included in the study was 2.14 (IQR, 0.9) kg, and they were born at 35 (IQR, 3) weeks of gestation. PN began between 0 and 4 days of life. In all, 71 % (22/31) of the patients in 54.1 % of their (46/85) prescriptions were candidates to receive SPN via central administration for 1 to 8 days, whereas no patient was candidate to receive SPN via peripheral administration. CONCLUSIONS: in our center, 71 % of neonates treated with central administration of IPN are candidates to receive SPN, thus promoting the normalization of nutritional support in this population

[Neonates treated with individualized parenteral nutrition who are candidates to receive standardized parenteral nutrition]. / Neonatos en tratamiento con nutriciones parenterales individualizadas, candidatos a recibir nutriciones parenterales estandarizadas.

INTRODUCTION: Objetive: to quantify the number of neonates treated with individualized parenteral nutrition (IPN) who were candidates to receive standardized parenteral nutrition (SPN), and to calculate their treatment duration. Material and methods: this was a prospective, observational, descriptive cohort study. Inclusion criteria were: neonates with indication of parenteral nutrition (PN) and individualized prescription. Exclusion criteria included: patients who had not started diuresis, with specific nutritional needs, altered acid-base balance, and/or contraindication to receive SPN. Included variables were patient-related (gender, weight, weeks of gestation, and days of life) and treatment-related regarding IPN composition. Setting the volume of PN as the conversion criterion, theoretical contributions were calculated with the SPN. The criterion for a patient to be a candidate to receive SPN was that all the theoretical contributions calculated were within the reference requirements range. Results: a total of 33 neonates (9 women) received IPN with 94 prescriptions. The median weight of the patients included in the study was 2.14 (IQR, 0.9) kg, and they were born at 35 (IQR, 3) weeks of gestation. PN began between 0 and 4 days of life. In all, 71 % (22/31) of the patients in 54.1 % of their (46/85) prescriptions were candidates to receive SPN via central administration for 1 to 8 days, whereas no patient was candidate to receive SPN via peripheral administration. Conclusions: in our center, 71 % of neonates treated with central administration of IPN are candidates to receive SPN, thus promoting the normalization of nutritional support in this population.

INTRODUCCIÓN: Objetivo: cuantificar el número de pacientes neonatos en tratamiento con nutriciones parenterales individualizadas (NPI), candidatos a recibir nutriciones parenterales estandarizadas (NPE), así como el número de días. Material y métodos: estudio prospectivo observacional y descriptivo de cohortes. Los criterios de inclusión fueron: pacientes neonatos con indicación de nutrición parenteral (NP) y prescripción individualizada. Los criterios de exclusión fueron: pacientes que no hubieran iniciado la diuresis, con necesidades nutricionales específicas, con alteraciones del equilibrio ácido-base y/o con contraindicación de la NPE. Se emplearon variables relacionadas con el paciente (sexo, peso, semanas de gestación y días de vida) y relacionadas con el tratamiento (aportes de la NPI). Fijando como criterio de conversión el volumen de NP, se calcularon los aportes teóricos con la NPE. El criterio para que un paciente fuera candidato a recibirla fue que todos los aportes teóricos estuvieran dentro de los requerimientos de referencia. Resultados: se incluyeron 33 neonatos (9 mujeres) en tratamiento con NPI y con 94 prescripciones. La mediana de peso de los pacientes incluidos en el estudio fue de 2,14 (RIC: 0,9) kg, nacidos a las 35 (RIC: 3) semanas de gestación y en los que se inició NP entre los días 0 y 4. El 71 % (22/31) de los pacientes en el 54,1 % (46/85) de sus prescripciones fueron candidatos a recibir NPE administrada por vía central durante 1 a 8 días, mientras que ningún paciente fue candidato a recibirla por vía periférica. Conclusiones: en nuestro centro, el 71 % de los pacientes nenonatos en tratamiento con NPI administrada por vía central son candidatos a recibir NPE, lo que fomenta la normalización del soporte nutricional en esta población.

"Validation of a spanish version of the 'Unité Rhumatologique Des Affections De La Main' (URAM) scale".

BACKGROUND: In health care, quality-of-life surveys and questionnaires related to care are becoming increasingly important as a measure of its quality. There is currently no Spanish version of the Unité Rhumatologique des Affections de la Main (URAM) scale, which makes it suitable for hand pathology. The purposes of this study are to develop a Spanish version of the URAM and perform a transcultural adaptation of it, analyzing the result for reliability, validity, and sensitivity to changes. METHODS: The questionnaire was evaluated for patients with Dupuytren's disease and Carpal Tunnel Syndrome. The cohort study subjects were interviewed at three points in time (baseline, three days after intervention, and one month after), administering the QuickDASH, URAM, and SF-12 (CF12 = physical component, CM12 = mental component) questionnaires at baseline and after intervention; and only the URAM at 3 days. Content validity was evaluated using Cronbach's α. The distribution of the factorial loads of the items and the pattern of the answers were checked. Responsiveness was evaluated by the size of the effect and the reliable rate of change. Convergent and divergent validity was performed using Spearman's r between the different questionnaires. RESULTS: The study was conducted with 106 patients. The mean baseline scores were: URAM = 14.8, QuickDASH = 41.6, CF12 = 39.3 and CM12 = 49.4. Ceiling or floor effects were not observed in the Spanish URAM. The Cronbach α = 0.853 explains 49.6% of the variance. The study had a high reproducibility (intraclass correlation coefficient (ICC) = 0.939). Size effect, measured as differences in scores, was moderate for URAM (-0.69) and QuickDASH (-0.51); and low for CF12 and CM12. The correlation of URAM with QuickDASH was high (r = 0.716), and moderate with DD and CTS. CONCLUSION: The Spanish version of the URAM is a valid and reliable tool for use in assessing hand pathology.

Adverse Effects of Collagenase in the Treatment of Dupuytren Disease: A Systematic Review.

BACKGROUND: Collagenase clostridium histolyticum (CCH) has proven to be both safe and effective in the treatment of Dupuytren disease (DD). The medium-term outcomes are similar to those achieved with surgery, and most adverse effects are self-limiting and considered to be mild or moderate. OBJECTIVE: Our objective was to conduct a systematic review of the adverse effects of CCH in DD since the release of the drug to evaluate the incidence, severity, classification, and definitions of these effects. METHODS: We analyzed the literature in terms of modifications to the original treatment protocol and grouped adverse effects according to their pathophysiological origin. RESULTS: We included 28 clinical studies and five case reports or case series analyzing 4456 patients with a mean age of 63.6 years. Mean follow-up was 7.07 months (range 3-24); the mean number of patients per study was 148 (range 5-1082). The studies did not classify the adverse effects they reported into groups. The most common effects were peripheral edema (54.4%), bruising (42.9%), and upper limb pain (28.3%). Significant biases were observed for use of terminology, demarcation of sites of involvement, severity criteria, and assessment methods. CONCLUSION: A simpler and clearer consensus-based classification system would enable better evaluation and comparison of the adverse effects of CCH in the treatment of DD. Consideration of inflammatory phenomena as part of the drug's mechanism of action would significantly reduce overall rates of adverse effects.