International Retrospective Chart Review of Treatment Patterns in Severe Familial Mediterranean Fever, Tumor Necrosis Factor Receptor–Associated Periodic Syndrome, and Mevalonate Kinase Deficiency/Hyperimmunoglobulinemia D Syndrome

Objective: Periodic fever syndrome (PFS) conditions are characterized by recurrent attacks of fever and localized inflammation. This study examined the diagnostic pathway and treatments at tertiary centers for familial Mediterranean fever (FMF), tumor necrosis factor receptor–associated periodic syndrome (TRAPS), and mevalonate kinase deficiency (MKD)/hyperimmunoglobulinemia D syndrome (HIDS). Methods: PFS specialists at medical centers in the US, the European Union, and the eastern Mediterranean participated in a retrospective chart review, providing de‐identified data in an electronic case report form. Patients were treated between 2008 and 2012, with at least 1 year of followup; all had clinical and/or genetically proven disease and were on/eligible for biologic treatment. Results: A total of 134 patients were analyzed: FMF (n = 49), TRAPS (n = 47), and MKD/HIDS (n = 38). Fever was commonly reported as severe across all indications. Other frequently reported severe symptoms were serositis for FMF patients and elevated acute‐phase reactants and gastrointestinal upset for TRAPS and MKD/HIDS. A long delay from disease onset to diagnosis was seen within TRAPS and MKD/HIDS (5.8 and 7.1 years, respectively) compared to a 1.8‐year delay in FMF patients. An equal proportion of TRAPS patients first received anti–interleukin‐1 (anti‐IL‐1) and anti–tumor necrosis factor (anti‐TNF) biologic agents, whereas IL‐1 blockade was the main choice for FMF patients resistant to colchicine and MKD/HIDS patients. For TRAPS patients, treatment with anakinra versus anti‐TNF treatments as first biologic agent resulted in significantly higher clinical and biochemical responses (P = 0.03 and P < 0.01, respectively). No significant differences in responses were observed between biologic agents among other cohorts. Conclusion: Referral patterns and diagnostic delays highlight the need for greater awareness and improved diagnostics for PFS. This real‐world treatment assessment supports the need for further refinement of treatment practices

Concurrent Oral 1 - Therapy of rheumatic disease: OP4. Effectiveness of Rituximab in Rheumatoid Arthritis: Results from the British Society for Rheumatology Biologics Register (BSRBR)

Background: Rituximab (RTX) in combination with methotrexate (MTX) has been licensed since 2006 for the management of severe active rheumatoid arthritis (RA) in patients who have failed at least one anti-tumour necrosis factor (anti-TNF) therapy. Published clinical trials have demonstrated the efficacy of RTX in improving both clinical symptoms and patients' physical function. This study aimed to assess the effectiveness of RTX in RA patients treated in routine clinical practice by examining clinical and patient reported outcomes six months after receiving a first course of RTX. Methods: The analysis involved 550 RA patients registered with the BSRBR, who were starting RTX and were followed up for at least 6 months. Change in Disease Activity Score (DAS28) and European League Against Rheumatism (EULAR) response were used to assess the clinical response while change in Health Assessment Questionnaire (HAQ) score was used to assess the physical function of the patients 6 months after starting RTX. The change in DAS28 and HAQ was compared between seronegative and seropositive patients and anti-TNF naïve patients versus anti-TNF failures. The response was also compared between patients receiving RTX in combination with MTX, other non-biologic disease modifying anti-rheumatic drugs (nbDMARDs) or no nbDMARDs. Results: The mean (s.d.) age of the cohort was 59 (12) years and 78% of the patients were females. The patients had a mean (s.d.) of 15 (10) years of disease duration. 16% were biologic naïve while 84% were anti-TNF failures. 32% of the patients were seronegative and 68% were seropositive. The mean (95% CI) DAS28 at baseline was 6.2 (6.1, 6.3) which decreased to 4.8 (4.7, 4.9) at 6 months of follow up. 16% were EULAR good responders, 43% were moderate responders and 41% were non responders. The mean (95% CI) change in HAQ was −0.1 (−0.2, −0.1) (Table 1). The mean change in DAS28 was similar in seropositive and seronegative patients (p = 0.18) while the anti-TNF naïve patients showed a greater reduction in DAS28 scores than anti-TNF failures (p = 0.05). Patients receiving RTX in combination with MTX showed similar changes in DAS28 and HAQ compared to patients receiving RTX alone or with other nbDMARDs. Conclusions: RTX has proven to be effective in the routine clinical practice. Anti-TNF naïve patients seem to benefit more from RTX treatment than anti-TNF failures. Disclosure statement: The authors have declared no conflicts of interes

Clinical characteristics of children with Juvenile Systemic Sclerosis: follow-up of 23 patients in a single tertiary center

© 2007 Russo and Katsicas; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution Licens

Subcutaneous Abatacept in Patients With Polyarticular-Course Juvenile Idiopathic Arthritis : Results From a Phase III Open-Label Study

OBJETIVO: Investigar la farmacocinética , la eficacia y la seguridad del tratamiento con abatacept subcutáneo (SC) durante 24 meses en pacientes con artritis idiopática juvenil (AIJ) de curso poliarticular . MÉTODOS: En este estudio de fase III, abierto, internacional, multicéntrico, de un solo brazo , los pacientes con AIJ poliarticular (cohorte 1, edades de 6 a 17 años y cohorte 2, edades de 2 a 5 años) en quienes el tratamiento con ≥1 modificadores de la enfermedad el fármaco antirreumático no tuvo éxito recibió abatacept subcutáneo escalonado por peso semanalmente 10 a <25 kg (50 mg), 25 a <50 kg (87,5 mg), ≥ 50 kg (125 mg). Los pacientes que habían reunido el Colegio AIJ-Americano de Reumatología criterios de mejoría del 30% (AIJ logrado una respuesta ACR-30) en el mes 4 se les dio la opción de continuar SC abatacept al mes 24. El criterio de valoración principal fue la abatacept estacionarioEstado de la concentración mínima sérica (Cminss) en la cohorte 1 en el mes 4. Otras medidas de resultado incluyeron JIA-ACR 30, 50, 70, 90, 100 y el estado de enfermedad inactiva , la mediana de la puntuación de actividad de la enfermedad de la artritis juvenil en 71 articulaciones utilizando la C- nivel de proteína reactiva (JADAS-71-CRP) a lo largo del tiempo , seguridad e inmunogenicidad. RESULTADOS: La mediana abatacept Cminss en mes 4 (punto final primario) y en el mes 24 estaba por encima del objetivo terapéutico de exposición (10 g / ml) en ambas cohortes. El porcentaje de pacientes que habían logrado respuestas JIA-ACR 30, 50, 70, 90 o 100 o tenían respuestas inactivas a la enfermedad en el mes 4 ( población por intención de tratar) fue 83,2%, 72,8%, 52,6%, 28,3%, 14,5% y 30,1%, respectivamente, en la cohorte 1 (n = 173) y 89,1%, 84,8%, 73,9%, 58,7%, 41,3% y 50,0% , respectivamente, en la cohorte 2 (n = 46); las respuestas se mantuvieron hasta el mes 24. La mediana (rango intercuartílico) JADAS-71-CRP mejoró desde el inicio hasta el mes 4 cohorte 1, de 21,0 (13,5, 30,3) a 4,6 (2,1, 9,4); cohorte 2, de 18,1 (14,0, 23,1) a 2,1 (0,3, 4,4). Las mejoras se mantuvieron a mes 24, en el que el tiempo 27 de 173 pacientes (cohorte 1) y 11 de 22 pacientes (cohorte 2) habían alcanzado una remisión Jadas-71-CRP. No se informaron eventos adversos inesperados; 4 de 172 pacientes (2,3%) en la cohorte 1 y 4 de 46 (8,7%) en la cohorte 2 desarrollaron anti abatacept anticuerpos, sin efectos clínicos. CONCLUSIÓN: -Peso estratificado SC abatacept produjo diana terapéuticos exposiciones en todos los grupos de edad y peso, fue bien tolerado, y la mejora de los síntomas AIJ poliarticular de más de 24 meses.Objective: To investigate the pharmacokinetics, effectiveness, and safety of subcutaneous (SC) abatacept treatment over 24 months in patients with polyarticular-course juvenile idiopathic arthritis (JIA). Methods: In this phase III, open-label, international, multicenter, single-arm study, patients with polyarticular JIA (cohort 1, ages 6–17 years and cohort 2, ages 2–5 years) in whom treatment with ≥1 disease-modifying antirheumatic drug was unsuccessful received weight-tiered SC abatacept weekly: 10 to <25 kg (50 mg), 25 to <50 kg (87.5 mg), ≥50 kg (125 mg). Patients who had met the JIA–American College of Rheumatology 30% improvement criteria (achieved a JIA-ACR 30 response) at month 4 were given the option to continue SC abatacept to month 24. The primary end point was the abatacept steady-state serum trough concentration (Cminss) in cohort 1 at month 4. Other outcome measures included JIA-ACR 30, 50, 70, 90, 100, and inactive disease status, the median Juvenile Arthritis Disease Activity Score in 71 joints using the C-reactive protein level (JADAS-71–CRP) over time, safety, and immunogenicity. Results: The median abatacept Cminss at month 4 (primary end point) and at month 24 was above the target therapeutic exposure (10 μg/ml) in both cohorts. The percentage of patients who had achieved JIA-ACR 30, 50, 70, 90, or 100 responses or had inactive disease responses at month 4 (intent-to-treat population) was 83.2%, 72.8%, 52.6%, 28.3%, 14.5%, and 30.1%, respectively, in cohort 1 (n = 173) and 89.1%, 84.8%, 73.9%, 58.7%, 41.3%, and 50.0%, respectively, in cohort 2 (n = 46); the responses were maintained to month 24. The median (interquartile range) JADAS-71–CRP improved from baseline to month 4: cohort 1, from 21.0 (13.5, 30.3) to 4.6 (2.1, 9.4); cohort 2, from 18.1 (14.0, 23.1) to 2.1 (0.3, 4.4). Improvements were sustained to month 24, at which time 27 of 173 patients (cohort 1) and 11 of 22 patients (cohort 2) had achieved JADAS-71–CRP remission. No unexpected adverse events were reported; 4 of 172 patients (2.3%) in cohort 1 and 4 of 46 (8.7%) in cohort 2 developed anti-abatacept antibodies, with no clinical effects. Conclusion: Weight-stratified SC abatacept yielded target therapeutic exposures across age and weight groups, was well tolerated, and improved polyarticular JIA symptoms over 24 months