The role of the clinical research coordinator – data manager – in oncology clinical trials

BACKGROUND: The purpose of this study was to determine the standard tasks performed by clinical research coordinators (CRCs) in oncology clinical trials. METHODS: Forty-one CRCs were anonymously surveyed, using a four-page self-administered questionnaire focused on demographics, qualifications, and professional experience. The survey questions on responsibilities consisted of an ad-hoc 32-item questionnaire where respondents had to rate the frequency of involvement in the listed activities using a 3-point scale. We defined as "standard" a task that was rated as "in all or nearly all trials" by at least half of the respondents. RESULTS: A response rate of 90% (37 out of 41) was achieved after two mailings. Less than half of the respondents had received additional training in oncology, clinical research or Good Clinical Practices (GCP). Overall, all standard tasks performed by CRCs were in the category of "monitoring activities" (those usually performed by a Clinical Research Associate "CRA") and included patient registration/randomization, recruitment follow-up, case report form completion, collaboration with the CRA, serious adverse events reporting, handling of investigator files, and preparing the site for and/or attending audits. CONCLUSIONS: CRCs play a key role in the implementation of oncology clinical trials, which goes far beyond mere data collection and/or administrative support, and directly contributes to the gathering of good quality data

Predictors of Response to Exclusive Enteral Nutrition in Newly Diagnosed Crohn´s Disease in Children: PRESENCE Study from SEGHNP

Exclusive enteral nutrition (EEN) has been shown to be more effective than corticosteroids in achieving mucosal healing in children with Crohn´s disease (CD) without the adverse effects of these drugs. The aims of this study were to determine the efficacy of EEN in terms of inducing clinical remission in children newly diagnosed with CD, to describe the predictive factors of response to EEN and the need for treatment with biological agents during the first 12 months of the disease. We conducted an observational retrospective multicentre study that included paediatric patients newly diagnosed with CD between 2014–2016 who underwent EEN. Two hundred and twenty-two patients (140 males) from 35 paediatric centres were included, with a mean age at diagnosis of 11.6 ± 2.5 years. The median EEN duration was 8 weeks (IQR 6.6–8.5), and 184 of the patients (83%) achieved clinical remission (weighted paediatric Crohn’s Disease activity index [wPCDAI] 15 mg/L and ileal involvement tended to respond better to EEN. EEN administered for 6–8 weeks is effective for inducing clinical remission. Due to the high response rate in our series, EEN should be used as the first-line therapy in luminal paediatric Crohn’s disease regardless of the location of disease and disease activityS

Aspectos clínicos y nutricionales de la intolerancia a la histamina en pacientes pediátricos con sintomatología digestiva crónica

INTRODUCCIÓN: La intolerancia a la histamina (IH) es una enfermedad poco estudiada, particularmente en pacientes pediátricos. El presente trabajo representa la serie más amplia de pacientes pediátricos con IH y el único que realiza un estudio nutricional de los pacientes. La IH se produce por una alteración en la capacidad de catabolizar la histamina ingerida con los alimentos, causada por una actividad reducida de la enzima diaminooxidasa (DAO). El consiguiente acúmulo histamínico puede causar cefalea, hipotensión o hipertensión, prurito y exantema, sibilancias y rinorrea, y diarrea, vómitos y abdominalgia. El diagnóstico se sospecha por la presencia de sintomatología típica y lo apoyará la determinación de la actividad DAO plasmática. El tratamiento consiste en realizar una dieta con alimentos bajos en histamina. La naturaleza diversa de los síntomas de la IH determina que dicha enfermedad sea frecuentemente subestimada, o los síntomas malinterpretados. Un mejor conocimiento del cuadro clínico, los síntomas, las herramientas diagnósticas, así como la respuesta al tratamiento pueden ayudar a un diagnóstico y tratamiento precoz. OBJETIVOS: Los objetivos principales de este trabajo son mejorar el conocimiento de la patología de la IH con sintomatología digestiva y en edad pediátrica, permitir un diagnóstico y tratamiento precoz y valorar la respuesta al tratamiento. Asimismo pretende analizar el impacto de dicha enfermedad y la dieta baja en histamina prescrita sobre el estado nutricional del paciente. METODOLOGÍA: Se han analizado de manera retrospectiva, prospectiva y observacional las historias clínicas de los pacientes menores de 15 años diagnosticados de IH desde septiembre de 2010 a mayo de 2014 remitidos a la consulta de gastroenterología pediátrica del Hospital Universitario Son Espases por sintomatología digestiva crónica. RESULTADOS: Se incluyeron 67 pacientes con diagnóstico de IH. La media de edad al inicio de los síntomas fue de 6 años y 5 meses. La media de edad al diagnóstico fue de 8 años y 2 meses. El intervalo medio entre el inicio de los síntomas y el diagnóstico fue de 22 meses. Este intervalo, comparando los primeros 15 casos diagnosticados con los 15 últimos casos, se redujo en 6 meses gracias a un mejor conocimiento de la enfermedad. El síntoma dominante fue el dolor abdominal inespecífico (70,1%). Los síntomas predominantes fueron el dolor abdominal (89,6%) y vómitos intermitentes (49,2%). El síntoma extradigestivo más frecuente fue la cefalea (50,7%). En 60 de los 67 casos, la actividad DAO fue inferior a 10kU/L (siendo normal a partir de 10kU/L). El valor medio fue de 7,3kU/L. El tratamiento se basó en una dieta baja en alimentos con histamina. Los 67 casos realizaron la dieta con desaparición de la sintomatología. En 64 pacientes se realizó un estudio antropométrico previo al inicio de la dieta y en 61 casos también se completó el estudio nutricional al mes de llevar la dieta baja en histamina. La media de la desviación standard o puntuación Z de peso, talla, IMC, e índices nutricionales antes de iniciar la dieta estaban en valores de normalidad según estándares de referencia. Tras un mes con dieta baja en histamina no se observaron diferencias significativas. CONCLUSIONES: La IH es un cuadro clínico subestimado. El intervalo entre el inicio de síntomas y el diagnóstico es generalmente muy prolongado, aunque podría acortarse con un mejor conocimiento de los síntomas y herramientas diagnósticas. Los síntomas predominantes son el dolor abdominal inespecífico y la cefalea. La determinación de la actividad DAO plasmática puede ayudar al diagnóstico, aunque la presencia de valores normales no necesariamente la excluye. El cuadro clínico digestivo no produce una malnutrición franca pero se puede asociar a riesgo de desnutrición. El tratamiento dietético es efectivo y no produce malnutrición.INTRODUCCIÓ: La intolerància a la histamina (IH) és una malaltia poc estudiada, particularment en pacients pediàtrics. El present treball representa la sèrie més àmplia de pacients pediàtrics amb IH i l'únic que realitza un estudi nutricional dels pacients. La IH es produeix per una alteració en la capacitat de catabolizar la histamina ingerida amb els aliments, causada per una activitat reduïda de l'enzim diaminooxidasa (DAO). El consegüent acúmul histamínic pot causar cefalea, hipotensió o hipertensió, prurito i exantema, sibilancias i rinorrea, i diarrea, vòmits i abdominalgia. El diagnòstic se sospita per la presència de simptomatologia típica i ho recolzarà la determinació de l'activitat DAO plasmàtica. El tractament consisteix a realitzar una dieta amb aliments baixos en histamina. La naturalesa diversa dels símptomes de la IH determina que aquesta malaltia sigui freqüentment subestimada, o els símptomes malinterpretats. Un millor coneixement del quadre clínic, els símptomes, les eines diagnòstiques, així com la resposta al tractament poden ajudar a un diagnòstic i tractament precoç. OBJECTIUS: Els objectius principals d'aquest treball són millorar el coneixement de la patologia de la IH amb simptomatologia digestiva i en edat pediàtrica, permetre un diagnòstic i tractament precoç i valorar la resposta al tractament. Així mateix pretén analitzar l'impacte d'aquesta malaltia i la dieta baixa en histamina prescrita sobre l'estat nutricional del pacient. METODOLOGIA: S'han analitzat de manera retrospectiva, prospectiva i observacional les històries clíniques dels pacients menors de 15 anys diagnosticats d'IH des de setembre de 2010 a maig de 2014 remesos a la consulta de gastroenterología pediàtrica de l'Hospital Universitari Són Espases per simptomatologia digestiva crònica. RESULTATS: Es van incloure 67 pacients amb diagnòstic d'IH. La mitjana d'edat a l'inici dels símptomes va ser de 6 anys i 5 mesos. La mitjana d'edat al diagnòstic va ser de 8 anys i 2 mesos. L'interval mitjà entre l'inici dels símptomes i el diagnòstic va ser de 22 mesos. Aquest interval, comparant els primers 15 casos diagnosticats amb els 15 últims casos, es va reduir en 6 mesos gràcies a un millor coneixement de la malaltia. El símptoma dominant va ser el dolor abdominal inespecífic (70,1%). Els símptomes predominants van ser el dolor abdominal (89,6%) i vòmits intermitents (49,2%). El símptoma extradigestivo més freqüent va ser la cefalea (50,7%). En 60 dels 67 casos, l'activitat DAO va ser inferior a 10kU/L (sent normal a partir de 10kU/L). El valor mitjà va ser de 7,3kU/L. El tractament es va basar en una dieta baixa en aliments amb histamina. Els 67 casos van realitzar la dieta amb desaparició de la simptomatologia. En 64 pacients es va realitzar un estudi antropomètric previ a l'inici de la dieta i en 61 casos també es va completar l'estudi nutricional al mes de portar la dieta baixa en histamina. La mitjana de la desviació standard o puntuació Z de pes, talla, IMC, i índexs nutricionals abans d'iniciar la dieta estaven en valors de normalitat segons estàndards de referència. Després d'un mes amb dieta baixa en histamina no es van observar diferències significatives. CONCLUSIONS: La IH és un quadre clínic subestimado. L'interval entre l'inici de símptomes i el diagnòstic és generalment molt perllongat, encara que podria escurçar-se amb un millor coneixement dels símptomes i eines diagnòstiques. Els símptomes predominants són el dolor abdominal inespecífic i la cefalea. La determinació de l'activitat DAO plasmàtica pot ajudar al diagnòstic, encara que la presència de valors normals no necessàriament l'exclou. El quadre clínic digestiu no produeix una malnutrició franca però es pot associar a risc de desnutrició. El tractament dietètic és efectiu i no produeix malnutrició.INTRODUCTION: Histamine intolerance (HIT) is a poorly studied condition, particularly in paediatric patients. This work presents the largest series of paediatric HIT patients and is the only one to carry out a nutritional study of patients. HIT is caused by an impaired ability to catabolise histamine ingested with food, caused by reduced activity of the enzyme diamine oxidase (DAO). The resulting histamine accumulation can cause headaches, hypotension or hypertension, pruritus and exanthem, sibilant rhonchi, rhinorrhea, diarrhoea, vomiting and abdominal pain. The presence of typical symptoms may indicate the condition, the diagnosis of which is confirmed by determining plasma DAO activity. The treatment involves following a low-histamine diet. The diverse nature of the symptoms means HIT is often overlooked and the symptoms misinterpreted. A better understanding of the clinical profile, symptoms, diagnostic tools and treatment response can aid early diagnosis and treatment. OBJECTIVES: The main objectives of this work are to gain greater knowledge of HIT presenting gastrointestinal symptoms in paediatric patients, to allow early diagnosis and treatment and to assess the response to treatment. It also aims to analyse the impact of the disease and how the prescribed low-histamine diet affects the nutritional state of the patient. METHODS: We carried out retrospective, prospective and observational analyses of the clinical records of patients under 15 diagnosed with HIT between September 2010 and May 2014 who had been referred to the paediatric gastroenterology unit of the University Hospital Son Espases because of chronic gastrointestinal symptoms. RESULTS: 67 patients diagnosed with HIT were included. The mean age at the onset of symptoms was 6 years and 5 months. The mean age at diagnosis was 8 years and 2 months. The mean interval between the onset of symptoms and diagnosis was 22 months. This interval, comparing the first 15 cases diagnosed with the last 15 cases, was reduced by six months due to a better understanding of the disease. The dominant symptom was non-specific abdominal pain (70.1%). The predominant symptoms were abdominal pain (89.6%) and intermittent vomiting (49.2%). The most common symptom unrelated to digestion was headaches (50.7%). In 60 of the 67 cases, the DAO activity was below 10 kU/L (standard is above 10 kU/L). The average value was 7.3 kU/L. The basis of the treatment was a low-histamine diet. In the 67 cases, the diet was followed and the symptoms disappeared. In 64 patients an anthropometric study was conducted prior to beginning the diet, and in 61 cases a nutritional study was also carried out one month after beginning the low-histamine diet. The average standard deviation or Z-score for weight-to-height, BMI and nutritional indexes were normal according to standard reference values before starting the diet. After one month on the low-histamine diet no significant differences were observed. CONCLUSIONS: HIT is an underestimated clinical condition. The interval between the onset of symptoms and diagnosis is usually very prolonged, but could be shortened with a better understanding of the symptoms and diagnostic tools. The digestive clinical condition does not produce direct malnutrition but can be associated with it. The predominant symptoms are non-specific abdominal pain and headaches. The determination of plasma DAO activity can help the diagnosis, although the presence of normal values does not necessarily exclude it, creating a risk of malnutrition. Dietary treatment is effective and does not cause malnutrition

Collagenous gastritis in the pediatric age

Collagenous gastritis (CG) is an uncommon condition known in the pediatric age. It is characterized by the presence of subepithelial collagen bands (> 10 μm) associated with lymphoplasmacytic infiltration of the stomach's lamina propria. Symptoms manifested by patients with CG may be common with many other disorders. It typically manifests with epigastralgia, vomiting, and iron deficiency during pre-adolescence. This condition's pathophysiology remains unclear. In contrast to adults, where association with collagenous colitis and other autoimmune conditions is more common, pediatric involvement is usually confined to the stomach. Drugs of choice include proton pump inhibitors and corticoids. A case is reported of a 12-year-old girl with abdominal pain and ferritin deficiency who was diagnosed with CG based on gastric biopsy and experienced a favorable outcome

Histamine intolerance as a cause of chronic digestive complaints in pediatric patients

Introduction: histamine intolerance (HI) is a poorly described disease in gastroenterology that may present with predominant digestive complaints. The goals of this study include a report of two cases diagnosed in a pediatric gastroenterology clinic. Material and methods: observational, retrospective study of patients diagnosed with HI from September 2010 to December 2011 at the pediatric gastroenterology clinic of a tertiary hospital. They were deemed to have a diagnosis of HI in the presence of 2 or more characteristic digestive complaints, decreased diamino oxidase (DAO) levels and/or response to a low histamine diet with negative IgE-mediated food allergy tests. Results: sixteen patients were diagnosed. Males predominated versus females (11/5). Mean age at symptom onset was 4 years (6 months vs. 13 years and 6 months) and mean age at diagnosis was 6 years and 6 months (17 months vs. 13 years and 11 months), with an interval of 2 years and 1 month between symptom onset and diagnosis (5 months vs. 4 years). Predominant symptoms included diffuse abdominal pain (16/16), intermittent diarrhea (10/16), headache (5/16), intermittent vomiting (4/16), and skin rash (2/16). The diagnosis was established by measuring plasma diamino oxidase levels, which were below 10 kU/L (normal > 10 kU/L) in 14 cases, and symptom clearance on initiating a low histamine diet. In two patients DAO levels were above 10 kU/L but responded to diet. Treatment was based on a diet low in histamine-contaning food, and antihistamines H1 y H2 had to be added for two cases. Conclusions: histamine intolerance is a little known disease with a potentially relevant incidence. Predominant complaints include diffuse abdominal pain, diarrhea, headache, and chronic intermittent vomiting. Its diagnosis is based on clinical suspicion, plasma DAO measurement, and response to a low histamine diet. Management with the latter provides immediate improvement