Effectiveness and safety of tocilizumab in monotherapy in biologic-naïve and non-naïve patients with rheumatoid arthritis in a real-world setting / Eficacia y seguridad de tocilizumab en monoterapia en pacientes con artritis reumatoide naive y no naive a biológicos en un entorno del mundo real

Objective: To evaluate the effectiveness and safety of tocilizumab (TCZ) monotherapy in biologic-naïve patients with rheumatoid arthritis (RA) versus patients with previous biologic exposure in a real-world setting. Materials and methods: Non-controlled clinical-trial, 32-week prospective multicenter study including RA patients with moderate-severe disease activity starting TCZ in monotherapy who had a prior inadequate response or were intolerant to methotrexate (MTX). Effectiveness according to EULAR response evaluated at 24-week and safety at 32-weekwere assessed. Results: Of the 93 were enrolled of whom 84 (90%) were eligible for the effectiveness analysis. Biologic-naïve patients (n=46, 54.8%) were younger (51.5 versus 57.9) with shorter disease duration (6.4 versus 13.3) but presented similar comorbidities in comparison with non-naïve patients. DAS28 remission was achieved in a higher percentage in the group of patients with prior biological treatment. 89 adverse events (AE) were recorded in 50 patients, most of them non-serious AE (non-SAE) (86.3%). Conclusions: In a real world setting, TCZ exhibit similar effectiveness and safety in monotherapy in patients with RA regardless previous exposure to other biologic therapies. This study provides additional and valuable real-world findings on the use of TCZ in patients with RA.(AU)

Objetivo: Evaluar la efectividad y seguridad de la monoterapia con tocilizumab (TCZ) en pacientes con artritis reumatoide (AR) sin tratamiento biológico en comparación con pacientes con exposición previa a biológico en un entorno real.Materiales y métodos: Ensayo clínico no controlado, estudio multicéntrico prospectivo de 32 semanas que incluyó pacientes con AR con actividad de la enfermedad moderada-grave que comenzaron con TCZ en monoterapia y que tuvieron una respuesta inadecuada previa o fueron intolerantes al metotrexato. La eficacia de acuerdo con la respuesta EULAR fue evaluada a las 24 semanas y la seguridad a las 32 semanas. Resultados: De los 93 pacientes seleccionados, 84 (90%) fueron elegibles para el análisis de efectividad. Los pacientes sin tratamiento biológico previo (n=46, 54,8%) eran más jóvenes (51,5 frente a 57,9 años), con una duración más corta de la enfermedad (6,4 frente a 13,3 años), pero presentaban comorbilidades similares en comparación con los pacientes con tratamiento previo. La remisión de DAS28 se logró en un mayor porcentaje en el grupo de pacientes con tratamiento biológico previo. Se registraron 89 eventos adversos en 50 pacientes, la mayoría de ellos no graves (86,3%). Conclusiones: En un entorno del mundo real, TCZ exhibe una eficacia y seguridad similares en monoterapia en pacientes con AR, independientemente de la exposición previa a otras terapias biológicas. Este estudio proporciona hallazgos adicionales y valiosos en el contexto del mundo real sobre el uso de TCZ en pacientes con AR.(UA)

Effectiveness and safety of tocilizumab in monotherapy in biologic-naïve and non-naïve patients with rheumatoid arthritis in a real-world setting.

OBJECTIVE: To evaluate the effectiveness and safety of tocilizumab (TCZ) monotherapy in biologic-naïve patients with rheumatoid arthritis (RA) versus patients with previous biologic exposure in a real-world setting. MATERIALS AND METHODS: Non-controlled clinical-trial, 32-week prospective multicenter study including RA patients with moderate-severe disease activity starting TCZ in monotherapy who had a prior inadequate response or were intolerant to methotrexate (MTX). Effectiveness according to EULAR response evaluated at 24-week and safety at 32-weekwere assessed. RESULTS: Of the 93 were enrolled of whom 84 (90%) were eligible for the effectiveness analysis. Biologic-naïve patients (n=46, 54.8%) were younger (51.5 versus 57.9) with shorter disease duration (6.4 versus 13.3) but presented similar comorbidities in comparison with non-naïve patients. DAS28 remission was achieved in a higher percentage in the group of patients with prior biological treatment. 89 adverse events (AE) were recorded in 50 patients, most of them non-serious AE (non-SAE) (86.3%). CONCLUSIONS: In a real world setting, TCZ exhibit similar effectiveness and safety in monotherapy in patients with RA regardless previous exposure to other biologic therapies. This study provides additional and valuable real-world findings on the use of TCZ in patients with RA.

The added value of a European Reference Network on rare and complex connective tissue and musculoskeletal diseases: insights after the first 5 years of the ERN ReCONNET.

In order to address the main challenges related to the rare diseases (RDs) the European Commission launched the European Reference Networks (ERNs), virtual networks involving healthcare providers (HCPs) across Europe. The mission of the ERNs is to tackle low prevalence and RDs that require highly specialised treatment and a concentration of knowledge and resources. In fact, ERNs offer the potential to give patients and healthcare professionals across the EU access to the best expertise and timely exchange of lifesaving knowledge, trying to make the knowledge travelling more than patients. For this reason, ERNs were established as concrete European infrastructures, and this is particularly crucial in the framework of rare and complex diseases in which no country alone has the whole knowledge and capacity to treat all types of patients.It has been five years since their kick-off launch in Vilnius in 2017. The 24 ERNs have been intensively working on different transversal areas, including patient management, education, clinical practice guidelines, patients' care pathways and many other fundamental topics. The present work is therefore aimed not only at reporting a summary of the main activities and milestones reached so far, but also at celebrating the first 5 years of the ERN on Rare and Complex Connective Tissue and Musculo-skeletal Diseases (ReCONNET), in which the members of the network built together one of the 24 infrastructures that are hopefully going to change the scenario of rare diseases across the EU.

Clinical and therapeutic management of rheumatoid arthritis with biological disease-modifying antirheumatic drugs: RADAR study.

To describe the clinical and therapeutic management of rheumatoid arthritis (RA) patients with biological disease-modifying antirheumatic drugs (bDMARDs), alone or in combination with conventional synthetic DMARDs (csDMARDs), as well as analysing changes over time in bDMARD use. An observational, retrospective, multicentre study was conducted in the rheumatology departments of 10 public Spanish hospitals. Patients with RA treated with bDMARDs at baseline who had medical records available in the data collection period 2013-2016 were included. All visits to rheumatology departments recording any type of bDMARD modification (dose, etc.) were collected. Clinical characteristics, concomitant treatment, resource use, work productivity and quality of life (QoL) were recorded. 128 patients were included: 81 received first-line bDMARD treatment, 28 second-line bDMARD treatment and 19 received third or later lines. Mean study follow-up was 4.1 years. Assessment of DAS28 was available in 54.6% of visits. At baseline, 48.7% of patients had moderate-high disease activity. At final observation, 69.5% of patients continued with the first bDMARD. Tumour necrosis factor blockers were administered to 85.2% of patients in first line, 45.7% in second line and 18.1% in third or later lines. At final observation, 80.2% of patients still felt pain/discomfort. As expected, those with higher disease activity had higher loss of work productivity and lower QoL, as assessed by DAS28, than patients with lower disease activity. Drugs represented 82.6% of the total cost. In this Spanish cohort of 128 patients, most patients remained on the first prescribed bDMARD, despite remaining signs and symptoms.

Documento de Recomendaciones de la Sociedad Española de Reumatología para el manejo clínico del paciente con artritis reumatoide que no puede utilizar metotrexato / Recommendations by the Spanish Society of Rheumatology for the management of patients diagnosed with rheumatoid arthritis who cannot be treated with methotrexate

El objetivo es establecer recomendaciones para el manejo del paciente con artritis reumatoide (AR) que no puede utilizar metotrexato (MTX) por contraindicación, toxicidad o falta de adherencia farmacológica, y establecer las estrategias terapéuticas más eficaces y seguras. Se realizó un análisis cualitativo de la evidencia científica disponible hasta junio de 2015. Se utilizó un Delphi con un panel de 17reumatólogos para consolidar la opinión de expertos en aquellas recomendaciones con ausencia o baja calidad científica. Se elaboraron 18recomendaciones, y 14 de ellas abordan aspectos de seguridad. Se han actualizado las recomendaciones sobre la contraindicación del MTX y su toxicidad, y se recomienda como una opción terapéutica preferente la utilización de monoterapia biológica en pacientes con contraindicación, intolerancia o circunstancias que desaconsejan el uso de MTX. Existe evidencia científica de buena calidad que contraindica y extrema la utilización de MTX en pacientes con AR con determinados perfiles clínicos (AU)

To establish a set of recommendations for the management of patients diagnosed with rheumatoid arthritis (RA) who cannot be treated with methotrexate (MTX) due to contraindications, drug toxicity or lack of adherence, and to establish therapeutic strategies more effective and safer in these RA patients. A qualitative analysis of the scientific evidence available to June 2015. The 2-round Delphi technique of consensus was used to collect and establish expert opinion based on the participants’ clinical experience when only low quality evidence was available. A total of eighteen recommendations were developed for the management of this patient profile. Fourteen of these recommendations were related to drug safety aspects. Recommendations on contraindication and toxicity of MTX have been updated. The experts recommend the use of biological monotherapy, a preferred treatment option, in patients whose profiles reveal a contraindication, intolerance or circumstances that prevent us against the use of MTX. There is some high-quality scientific evidence that supports contraindication and establishes certain conditions of MTX use in RA patients with specific clinical profiles (AU)

Recommendations by the Spanish Society of Rheumatology for the management of patients diagnosed with rheumatoid arthritis who cannot be treated with methotrexate. / Documento de Recomendaciones de la Sociedad Española de Reumatología para el manejo clínico del paciente con artritis reumatoide que no puede utilizar metotrexato.

To establish a set of recommendations for the management of patients diagnosed with rheumatoid arthritis (RA) who cannot be treated with methotrexate (MTX) due to contraindications, drug toxicity or lack of adherence, and to establish therapeutic strategies more effective and safer in these RA patients. A qualitative analysis of the scientific evidence available to June 2015. The 2-round Delphi technique of consensus was used to collect and establish expert opinion based on the participants' clinical experience when only low quality evidence was available. A total of eighteen recommendations were developed for the management of this patient profile. Fourteen of these recommendations were related to drug safety aspects. Recommendations on contraindication and toxicity of MTX have been updated. The experts recommend the use of biological monotherapy, a preferred treatment option, in patients whose profiles reveal a contraindication, intolerance or circumstances that prevent us against the use of MTX. There is some high-quality scientific evidence that supports contraindication and establishes certain conditions of MTX use in RA patients with specific clinical profiles.