Transition of patients with metabolic bone disease from paediatric to adult healthcare services: current situation and proposals for improvement.

BACKGROUND: There are currently no models for the transition of patients with metabolic bone diseases (MBDs) from paediatric to adult care. The aim of this project was to analyse information on the experience of physicians in the transition of these patients in Spain, and to draw up consensus recommendations with the specialists involved in their treatment and follow-up. METHODS: The project was carried out by a group of experts in MBDs and included a systematic review of the literature for the identification of critical points in the transition process. This was used to develop a questionnaire with a total of 48 questions that would determine the degree of consensus on: (a) the rationale for a transition programme and the optimal time for the patient to start the transition process; (b) transition models and plans; (c) the information that should be specified in the transition plan; and (d) the documentation to be created and the training required. Recommendations and a practical algorithm were developed using the findings. The project was endorsed by eight scientific societies. RESULTS: A total of 86 physicians from 53 Spanish hospitals participated. Consensus was reached on 45 of the 48 statements. There was no agreement that the age of 12 years was an appropriate and feasible point at which to initiate the transition in patients with MBD, nor that a gradual transition model could reasonably be implemented in their own hospital. According to the participants, the main barriers for successful transition in Spain today are lack of resources and lack of coordination between paediatric and adult units. CONCLUSIONS: The TEAM Project gives an overview of the transition of paediatric MBD patients to adult care in Spain and provides practical recommendations for its implementation.

Efectividad y seguridad de los bisfosfonatos en el tratamiento de la osteoporosis infantil secundaria / Effectiveness and safety of bisphosphonates therapy in secondary osteoporosis in children

Introducción: Los estudios sobre efectividad y seguridad de los bisfosfonatos en osteoporosis infantil secundaria (OIS) son escasos. El objetivo fue analizar efectividad y seguridad de los bisfosfonatos en OIS. Pacientes y métodos: Estudio multicéntrico retrospectivo en <18 años afectos de OIS tratados con bisfosfonatos. Se recogieron variables clínicas. Se valoró densidad mineral ósea mediante el Z-score de densidad mineral ósea en columna lumbar (ZDMOcl) medido por absorciometría de rayos X de doble energía (DXA). Valoramos efectividad en función del cambio del ZDMOcl al año y a los dos años de su inicio y del descenso del número de fracturas/año. Los eventos adversos reportados fueron recogidos. Se realizó análisis descriptivo y bivariante. Resultados: Se reclutaron 32 pacientes. El ZDMOcl se incrementó al año del inicio del tratamiento ([-2,46±0,96] vs. [-1,54±1,38]; p<0,001). El número de fracturas/año disminuyó significativamente (1 [1-2] vs. 0 [0-0,61]; p<0,001). El cambio en el ZDMOcl fue mayor en los pacientes deambulantes (1,88+/- 0,72 vs. 0,55+/-0,82; p=0,07) y se correlacionó positivamente con el percentil del IMC (rho:0,564; p<0,001). El descenso del número de fracturas/año fue mayor en los pacientes con menor tasa inicial de fracturas (rho:-0,47; p=0,006) y cuanto mayor era el Z-score inicial (rho:-0,47; p=0,07). Se reportaron 10 eventos adversos leves en 7 pacientes (22%), todos con bisfosfonatos intravenosos. No se halló relación entre eventos adversos y las variables estudiadas. Conclusiones: Los bisfosfonatos son efectivos en OIS. La respuesta parece ser mejor en pacientes deambulantes, bien nutridos y en estadios precoces de la enfermedad. Resultan seguros, siendo los efectos adversos leves, aunque frecuentes. (AU)

Introduction: There are few studies on effectiveness and safety of bisphosphonate therapy in secondary osteoporosis in children. The aim of this research was to analyse effectiveness and safety of bisphosphonates in secondary osteoporosis in children. Patients and methods: Multicentre retrospective study in patients younger than 18 suffering from secondary osteoporosis and who had received bisphosphonates. Clinical data were recorded. Bone mineral density was assessed in terms of bone mineral density Z-score in lumbar spine (ZBMDls) measured by dual-energy X-ray absorptiometry (DXA). Effectiveness was valued at changes in ZBMDls one and two years after the onset of bisphosphonates and at the decrease in the number of fractures a year. Adverse events reported were recorded. Descriptive and bivariant analysis were performed. Results: 32 patients were recruited. ZBMDls increased one year after the onset of treatment ([−2.46±0.96] vs. [−1.54±1.38]; p<.001). Fractures a year decreased significantly (1 [1–2] vs. 0 [0–0.61]; p<,001). ZBMDls increase was higher in patients who were able to walk (1.88±0.72 vs. 0.55±0.82; p=.07) and correlated positively with body mass index (BMI) for age percentile (rho: 0.564; p<.001). The decrease in the number of fractures a year was higher in patients with lower initial fracture rate (rho: −0.47; p=.006) and with higher initial ZBMDls (rho: −0.47; p=.07). 10 adverse events were reported in 7 patients (22%), all of them intravenous bisphosphonates related. No association was found between adverse events and studied variables. Conclusions: Bisphosphonates are effective in secondary osteoporosis in children. Response seems to be better in patients who are able to walk, well-nourished and in the early stages of the disease. Adverse events were frequent but mild. (AU)

Effectiveness and safety of bisphosphonates therapy in secondary osteoporosis in children.

INTRODUCTION: There are few studies on effectiveness and safety of bisphosphonate therapy in secondary osteoporosis in children. The aim of this research was to analyse effectiveness and safety of bisphosphonates in secondary osteoporosis in children. PATIENTS AND METHODS: Multicentre retrospective study in patients younger than 18 suffering from secondary osteoporosis and who have received bisphosphonates. Clinical data were recorded. Bone mineral density (BMD) was assessed in terms of BMD Z-score in lumbar spine (ZBMDls) measured by dual-energy X-ray absorptiometry (DXA). Effectiveness was valued at changes in ZBMDls one and two years after the onset of bisphosphonates and at the decrese in the number of fractures a year. Adverse events reported were recorded. Descriptive and bivariant analysis was performed. RESULTS: 32 patients were recruited. ZBMDls increased one year after the onset of treatment ((-2.46 ± 0.96) vs. (-1.54 ± 1.38); p < .001). Fractures a year dicreased significantly (1 (1-2) vs. 0 (0-0.61); p < .001). ZBMDls increase was higher in patients who were able to walk (1.88 ± 0.72 vs. 0.55 ± 0.82; p = .07) and correlated positively with body mass index (BMI)- for- age percentile (rho: 0.564; p < .001). The decrease in the number of fractures a year was higher in patients with lower initial fracture rate (rho: -0,47; p = .006) and with higher initial ZBMDls (rho: -0.47; p = .07). 10 adverse events were reported in 7 patients (22%), all of them intravenous bisphosphonates related. No association was found between adverse events and studied variables. CONCLUSIONS: Bisphosphonates are effective in secondary osteoporosis in children. Response seems to be better in patients who are able to walk, well-nourished and in the early stages of the disease. Adverse events were frequent but mild.

Recomendaciones para el diagnóstico y tratamiento de la baja masa ósea para la edad cronológica y la osteoporosis infantil en Atención Primaria / Recommendations for the diagnosis and treatment of low bone mass for chronological age and childhood osteoporosis in primary care

Introducción: la ausencia en nuestro medio de protocolos de manejo y de derivación de los pacientes de riesgo hace que exista una gran variabilidad en la actividad preventiva y en el manejo clínico respecto a la osteoporosis infantil en los pediatras de nuestro país. Método: recientemente, el Grupo de Trabajo de Osteogénesis Imperfecta y Osteoporosis Infantil, de la Sociedad Española de Reumatología Pediátrica (SERPE) ha publicado un documento de consenso con recomendaciones sobre el diagnóstico y tratamiento de la osteoporosis secundaria infantil. En este artículo, resumimos aquellas más relevantes en el ámbito de Atención Primaria. Un panel de expertos, compuesto por pediatras y reumatólogos, elaboró una serie de recomendaciones basadas en la evidencia tras realizar una revisión cualitativa de la literatura. El nivel de evidencia se determinó para cada sección utilizando el sistema del Centro de Medicina basada en la Evidencia de Oxford (CEBM). Se realizó una encuesta Delphi para aquellas recomendaciones con un nivel de evidencia de IV o V. Se incluyeron todas las recomendaciones que tuvieron un nivel de concordancia superior o igual al 70%. Esta encuesta se envió a todos los miembros de la Sociedad Española de Reumatología Pediátrica. Resultados: se obtuvieron 51 recomendaciones, categorizadas en ocho secciones. Las recomendaciones resultantes son: cuándo sospechar y cómo prevenir la osteoporosis infantil y la baja masa ósea según la edad cronológica; qué métodos de detección y diagnóstico utilizar; cuáles son los tratamientos actuales y cómo prevenir la osteoporosis inducida por los corticoesteroides. Conclusión: la detección precoz y un enfoque terapéutico adecuado de la baja masa mineral ósea desde Atención Primaria (AP) son fundamentales para mejorar la salud ósea de nuestra población infantil. Las recomendaciones expuestas pueden ayudar a tomar las medidas de prevención y tratamiento correctas en la población infantil de riesgo (AU)

Introduction: due to the lack of standardised protocols for the management and referral of at-risk patients, there is substantial variability in the prevention and clinical management of childhood osteoporosis among paediatricians in Spain.Methods: the Working Group on Osteogenesis Imperfecta and Childhood Osteoporosis of the Sociedad Española de Reumatología Pediátrica (SERPE) recently published a consensus document with recommendations on the diagnosis and management of secondary childhood osteoporosis. An expert panel comprised of paediatricians and rheumatologists carried out a qualitative literature review and developed evidence-based recommendations.For each section, the level of evidence was determined using the Oxford Centre for Evidence-based Medicine (CEBM) system. A Delphi survey was conducted for those recommendations with a level of evidence of IV or V. All recommendations for which the level of agreement was 70% or greater were included. This survey was sent to all members of the SERPE.Results: the process yielded 51 recommendations categorized into 8 sections. The resulting recommendations concern when to suspect and how to prevent childhood osteoporosis and low bone mass according to chronological age; which screening and diagnosis methods to use; the current treatments and how to prevent corticosteroid-induced osteoporosis.Conclusions: early detection and an adequate approach to the treatment of low bone mass at the primary care (PC) level are essential to improve bone health in our paediatric population. These recommendations could contribute to improving prevention and treatment measures in at-risk children. (AU)

Expert panel consensus recommendations for diagnosis and treatment of secondary osteoporosis in children.

BACKGROUND: Osteoporosis incidence in children is increasing due to the increased survival rate of patients suffering from chronic diseases and the increased use of drugs that can damage bones. Recent changes made to the definition of childhood osteoporosis, along with the lack of guidelines or national consensuses regarding its diagnosis and treatment, have resulted in a wide variability in the approaches used to treat this disease. For these reasons, the Osteogenesis Imperfecta and Childhood Osteoporosis Working Group of the Spanish Society of Pediatric Rheumatology has sounded the need for developing guidelines to standardize clinical practice with regard to this pathology. METHODS: An expert panel comprised of 6 pediatricians and 5 rheumatologists carried out a qualitative literature review and provided recommendations based on evidence, when that was available, or on their own experience. The level of evidence was determined for each section using the Oxford Centre for Evidence-based Medicine (CEBM) system. A Delphi survey was conducted for those recommendations with an evidence level of IV or V. This survey was sent to all members of the SERPE. All recommendations that had a level of agreement higher or equal to 70% were included. RESULTS: Fifty-one recommendations, categorized into eight sections, were obtained. Twenty-four of them presented an evidence level 4 or 5, and therefore a Delphi survey was conducted. This was submitted electronically and received a response rate of 40%. All recommendations submitted to the Delphi round obtained a level of agreement of 70% or higher and were therefore accepted. CONCLUSION: In summary, we present herein guidelines for the prevention, diagnosis and treatment of secondary childhood osteoporosis based on the available evidence and expert clinical experience. We believe it can serve as a useful tool that will contribute to the standardization of clinical practice for this pathology. Prophylactic measures, early diagnosis and a proper therapeutic approach are essential to improving bone health, not only in children and adolescents, but also in the adults they will become in the future.

Tuberculosis in pediatric patients treated with anti-TNFα drugs: a cohort study.

BACKGROUND: Adult patients receiving anti-TNFα drugs are at increased risk of tuberculosis (TB), but studies in pediatric populations are limited, and the best strategy for latent tuberculosis infection (LTBI) screening in this population remains controversial. We describe the prevalence of LTBI prior to anti-TNFα therapy and the long-term follow-up after biological treatment initiation in a cohort of children and adolescents. METHODS: Cohort observational study in children and adolescents receiving anti-TNFα agents in a tertiary-care pediatric hospital. LTBI was ruled out prior to the implementation of anti-TNFα drugs by tuberculin skin test (TST), and, from March 2012 on, QuantiFERON Gold-In Tube test (QTF-G). During anti-TNFα treatment, patients were evaluated every 6 months for TB with history and physical examination. TST/QTF-G were not repeated unless signs or symptoms consistent with TB arose or there was proven TB contact. RESULTS: The final cohort consisted of 221 patients (56.1% female; 261 treatments), of whom 51.7%/30.0%/17.3% were treated with etanercept/adalimumab/infliximab, respectively, for a variety of rheumatic diseases (75.6%), inflammatory bowel disease (20.8%), and inflammatory eye diseases (3.6%). The median (IQR) age at diagnosis of the primary condition was 6.8 years (2.7-11.0) and the duration of the disease before implementing the anti-TNFα agent was 1.8 years (0.6-4.2). LTBI was diagnosed in 3 adolescent girls (prevalence rate: 1.4%; 95% CI: 0.4-4.2) affected with juvenile idiopathic arthritis: TST tested positive in only 1, while QTF-G was positive in all cases (including 2 patients already on etanercept). They all received antiTB chemoprophylaxis and were later (re)treated with etanercept for 24-29 months, without incidences. No incident cases of TB disease were observed during the follow-up period under anti-TNFα treatment of 641 patients-year, with a median (IQR) time per patient of 2.3 years (1.4-4.3). CONCLUSIONS: In our study, the prevalence of LTBI (1.4%) was similar to that reported in population screening studies in Spain; no incident cases of TB disease were observed. In low-burden TB settings, initial screening for TB in children prior to anti-TNFα treatment should include both TST and an IGRA test, but systematic repetition of LTBI immunodiagnostic tests seems unnecessary in the absence of symptoms or known TB contact.

Artritis idiopàtica juvenil. Revisió i nous conceptes / Artritis idiopática juvenil. Revisión y nuevos conceptos / Juvenile idiopathic arthritis. Review and new concepts

L'artritis idiopàtica juvenil és la malaltia reumàtica més freqüent en la infància. Representa un grup heterogeni que inclou diferents formes de presentació d'artritis de causa desconeguda en pacients menors de 16 anys. L'etiopatogènia no és gaire coneguda, però es postula que té lloc com a conseqüència d'un estímul desconegut sobre un individu genèticament predisposat. L'aparició de nous tractaments els darrers anys ha significat un gran avenç en el tracta-ment, ja que s'han aconseguit grans millores en el pronòs-tic i la qualitat de vida. És necessari que tots els pediatres coneguin les característiques més importants de la malaltia i dels tractaments utilitzats, per tal que puguin participar de manera coordinada amb l'equip de reumatologia pe-diàtrica en l'atenció d'aquests pacients

La artritis idiopática juvenil es la enfermedad reumática más frecuente en la infancia. Representa un grupo heterogéneo que incluye diferentes formas de presentación de artritis de causa desconocida en pacientes menores de 16 años. La etiopatogenia no es muy conocida, pero se postula se desarrolla consecuencia de un estímulo desconocido sobre un individuo genéticamente predispuesto. La aparición de nuevos tratamientos en los últimos años ha significado un gran avance en el tratamiento, ya que se han logrado grandes mejoras en el pronóstico y la calidad de vida. Es necesario que todos los pediatras conozcan las características más importantes de la enfermedad y los tratamientos utilizados, para que puedan participar de manera coordinada con el equipo de reumatología pediátrica en la atención de estos pacientes (AU)

Juvenile idiopathic arthritis is the most common rheumatic disease in childhood. It represents a diverse group that includes different forms of presentation of arthritis of unknown cause in patients under 16 years. The pathogenesis is not very well known, but it is postulated that it develops as a result of unknown stimuli on a genetically predisposed individual. The emergence of new treatments in recent years has meant a breakthrough in treatment, since they have made great improvements in the prognosis and quality of life. It is necessary that all pediatricians know the most important characteristics of the disease, so they can participate in a coordinated manner with the paediatric rheumatology team, in the care of these patients (AU)