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1. Safety and effectiveness of abatacept in juvenile idiopathic arthritis: Results from the PRINTO/PRCSG registry

Author: Lovell, D, Tzaribachev, N, Henrickson, M, Simonini, G, Griffin, T, Alexeeva, E, Bohnsack, J, Zeft, A, Horneff, G, Vehe, R, Stanevica, V, Tarvin, S, Trachana, M, Del Rio, A, Huber, A, Kietz, D, Orban, I, Dare, J, Foeldvari, I, Quartier, P, Dominique, A, Simon, T, Martini, A, Brunner, H, Ruperto, N, Brunner, J, Fernandes, T, Appenzeller, S, Oliveira, S, Terreri, M, Minden, K, Hufnagel, M, Helling-Bakki, A, Herlin, T, Moreno, E, Anton, J, Del-Castillo, P, Udaondo, C, Penades, I, Brochard, K, Ramanan, A, Hashkes, P, Olivieri, A, Zulian, F, Montin, D, Peroni, D, Stanevicha, V, Cornejo, G, Wulffraat, N, Kamphuis, S, Gastanaga, M, Miraval, T, Oliveira-Ramos, F, Lazar, C, Nikishina, I, Sarychev, A, Chasnyk, V, Grebenkina, L, Suwairi, W, Koskova, E, Ally, M, Louw, I, Breedt, J, Ting, T, Taylor, J, Huggins, J, Dewitt, E, Grom, A, Schulert, G, Rodriguez-Smith, J, Morris, P, Sukumarain, S, Gitelman, M, Miller, M, Curran, M, Alperin, R, Ardalan, K, De Ranieri, D, Hiskey, M, Nolan, B, Chalom, B, Zelf, A, Spalding, S, Costanzo, D, Rennebohm, R, Waugaman, B, Brodus, E, Robinson, A, Panupattanapong, S, Rosenkranz, M, Cassidy, E, Torok, K, Kingsbury, D, Cartwright, V, Lasky, A, Brown, D, Reiff, A, Shaham, B, Marzan, K, Wagner-Weiner, L, Onel, K, Tesher, M, Edens, C, Moore, T, Syed, R, Pepmueller, P, Tuttle, P, Dalrymple, A, Barhula, S, Feller, L, Horwitz, M, Justice, M, Nocton, J, Olson, J, Williams, C, Versbsy, J, Co, D, Roth-Wojcicki, E, Correll, C, Binstadt, B, Hobday, P, Brueck, D, Gillispie-Taylor, M, Vora, S, O'Neil, K, Ballinger, S, Blakley, M, Klausmeier, T, Oliver, M, Stevens, B, Rodriguez, M, Go, E, Inman, C, Hersh, A, Stern, S, Woodward, A, Durkee, D, Boulva, S, James, K, Treemarcki, E, Goldsmith, D, Lvovich, S, Toib, D, Patel, J, Jerath, R, Sharma, N, Newhall, L, Carrasco, R, Moorthy, N, Boneparth, A, Quintero, A, Graham, T, Spence, S, Davis, A, Gotte, A, Mehta, J, Walters, H, Mian, Z, Parkinson, E, Hui-Yen, J, Steigerwald, K, Guzman, M, Gottlieb, B, Whitaker, C, Kelly, L, Succimarri, R, Hazel, E, Chedeville, G, Compillo, S, Leblance, C, Tucker, L, Cabral, D, Houghton, K, Guzman, J, Morishita, K, Stringer, E, Ramsey, S, Lang, B, Levy, D, Silverman, E, Schmeling, H, Johnson, N, Luca, N, Dhalla, M, Lovell D. J., Tzaribachev N., Henrickson M., Simonini G., Griffin T. A., Alexeeva E., Bohnsack J. F., Zeft A., Horneff G., Vehe R. K., Stanevica V., Tarvin S., Trachana M., Del Rio A. Q., Huber A. M., Kietz D., Orban I., Dare J., Foeldvari I., Quartier P., Dominique A., Simon T. A., Martini A., Brunner H. I., Ruperto N., Brunner J., Fernandes T., Appenzeller S., Oliveira S., Terreri M. T., Minden K., Hufnagel M., Helling-Bakki A., Herlin T., Moreno E., Anton J., Del-Castillo P. M. -., Udaondo C., Penades I. C., Brochard K., Ramanan A., Hashkes P. P., Olivieri A. N., Zulian F., Montin D., Peroni D., Stanevicha V., Cornejo G. V., Wulffraat N., Kamphuis S., Gastanaga M. E. P., Miraval T., Oliveira-Ramos F., Lazar C., Nikishina I., Sarychev A., Chasnyk V., Grebenkina L., Suwairi W. M. S., Koskova E., Ally M., Louw I., Breedt J., Brunner H., Ting T., Taylor J., Huggins J., Dewitt E. M., Grom A., Lovell D., Schulert G., Rodriguez-Smith J., Morris P., Sukumarain S., Gitelman M. K., Miller M., Curran M., Alperin R., Ardalan K., De Ranieri D., Hiskey M., Nolan B., Chalom B., Zelf A., Spalding S., Costanzo D., Rennebohm R., Waugaman B., Brodus E., Robinson A., Panupattanapong S., Rosenkranz M., Cassidy E., Torok K., Kingsbury D., Cartwright V., Lasky A., Brown D., Reiff A., Shaham B., Marzan K., Wagner-Weiner L., Onel K., Tesher M., Edens C., Moore T., Syed R., Pepmueller P., Tuttle P., Dalrymple A., Barhula S., Feller L., Horwitz M., Justice M., Nocton J., Olson J., Williams C., Versbsy J., Co D., Roth-Wojcicki E., Correll C., Vehe R., Binstadt B., Hobday P., Brueck D., Griffin T., Gillispie-Taylor M., Vora S., O'Neil K., Ballinger S., Blakley M., Klausmeier T., Oliver M., Stevens B., Rodriguez M., Go E., Bohnsack J., Inman C., Hersh A., Stern S., Woodward A., Durkee D., Boulva S. F., James K., Treemarcki E., Goldsmith D., Lvovich S., Toib D., Patel J., Jerath R., Sharma N., Newhall L., Carrasco R., Moorthy N., Boneparth A., Quintero A., Graham T., Spence S., Davis A., Gotte A., Mehta J., Walters H., Mian Z., Parkinson E., Hui-Yen J., Steigerwald K., Guzman M., Gottlieb B., Whitaker C., Kelly L., Succimarri R., Hazel E., Chedeville G., Compillo S., Leblance C., Tucker L., Cabral D., Houghton K., Guzman J., Morishita K., Huber A., Stringer E., Ramsey S., Lang B., Levy D., Silverman E., Schmeling H., Johnson N., Luca N., Dhalla M., Lovell, D, Tzaribachev, N, Henrickson, M, Simonini, G, Griffin, T, Alexeeva, E, Bohnsack, J, Zeft, A, Horneff, G, Vehe, R, Stanevica, V, Tarvin, S, Trachana, M, Del Rio, A, Huber, A, Kietz, D, Orban, I, Dare, J, Foeldvari, I, Quartier, P, Dominique, A, Simon, T, Martini, A, Brunner, H, Ruperto, N, Brunner, J, Fernandes, T, Appenzeller, S, Oliveira, S, Terreri, M, Minden, K, Hufnagel, M, Helling-Bakki, A, Herlin, T, Moreno, E, Anton, J, Del-Castillo, P, Udaondo, C, Penades, I, Brochard, K, Ramanan, A, Hashkes, P, Olivieri, A, Zulian, F, Montin, D, Peroni, D, Stanevicha, V, Cornejo, G, Wulffraat, N, Kamphuis, S, Gastanaga, M, Miraval, T, Oliveira-Ramos, F, Lazar, C, Nikishina, I, Sarychev, A, Chasnyk, V, Grebenkina, L, Suwairi, W, Koskova, E, Ally, M, Louw, I, Breedt, J, Ting, T, Taylor, J, Huggins, J, Dewitt, E, Grom, A, Schulert, G, Rodriguez-Smith, J, Morris, P, Sukumarain, S, Gitelman, M, Miller, M, Curran, M, Alperin, R, Ardalan, K, De Ranieri, D, Hiskey, M, Nolan, B, Chalom, B, Zelf, A, Spalding, S, Costanzo, D, Rennebohm, R, Waugaman, B, Brodus, E, Robinson, A, Panupattanapong, S, Rosenkranz, M, Cassidy, E, Torok, K, Kingsbury, D, Cartwright, V, Lasky, A, Brown, D, Reiff, A, Shaham, B, Marzan, K, Wagner-Weiner, L, Onel, K, Tesher, M, Edens, C, Moore, T, Syed, R, Pepmueller, P, Tuttle, P, Dalrymple, A, Barhula, S, Feller, L, Horwitz, M, Justice, M, Nocton, J, Olson, J, Williams, C, Versbsy, J, Co, D, Roth-Wojcicki, E, Correll, C, Binstadt, B, Hobday, P, Brueck, D, Gillispie-Taylor, M, Vora, S, O'Neil, K, Ballinger, S, Blakley, M, Klausmeier, T, Oliver, M, Stevens, B, Rodriguez, M, Go, E, Inman, C, Hersh, A, Stern, S, Woodward, A, Durkee, D, Boulva, S, James, K, Treemarcki, E, Goldsmith, D, Lvovich, S, Toib, D, Patel, J, Jerath, R, Sharma, N, Newhall, L, Carrasco, R, Moorthy, N, Boneparth, A, Quintero, A, Graham, T, Spence, S, Davis, A, Gotte, A, Mehta, J, Walters, H, Mian, Z, Parkinson, E, Hui-Yen, J, Steigerwald, K, Guzman, M, Gottlieb, B, Whitaker, C, Kelly, L, Succimarri, R, Hazel, E, Chedeville, G, Compillo, S, Leblance, C, Tucker, L, Cabral, D, Houghton, K, Guzman, J, Morishita, K, Stringer, E, Ramsey, S, Lang, B, Levy, D, Silverman, E, Schmeling, H, Johnson, N, Luca, N, Dhalla, M, Lovell D. J., Tzaribachev N., Henrickson M., Simonini G., Griffin T. A., Alexeeva E., Bohnsack J. F., Zeft A., Horneff G., Vehe R. K., Stanevica V., Tarvin S., Trachana M., Del Rio A. Q., Huber A. M., Kietz D., Orban I., Dare J., Foeldvari I., Quartier P., Dominique A., Simon T. A., Martini A., Brunner H. I., Ruperto N., Brunner J., Fernandes T., Appenzeller S., Oliveira S., Terreri M. T., Minden K., Hufnagel M., Helling-Bakki A., Herlin T., Moreno E., Anton J., Del-Castillo P. M. -., Udaondo C., Penades I. C., Brochard K., Ramanan A., Hashkes P. P., Olivieri A. N., Zulian F., Montin D., Peroni D., Stanevicha V., Cornejo G. V., Wulffraat N., Kamphuis S., Gastanaga M. E. P., Miraval T., Oliveira-Ramos F., Lazar C., Nikishina I., Sarychev A., Chasnyk V., Grebenkina L., Suwairi W. M. S., Koskova E., Ally M., Louw I., Breedt J., Brunner H., Ting T., Taylor J., Huggins J., Dewitt E. M., Grom A., Lovell D., Schulert G., Rodriguez-Smith J., Morris P., Sukumarain S., Gitelman M. K., Miller M., Curran M., Alperin R., Ardalan K., De Ranieri D., Hiskey M., Nolan B., Chalom B., Zelf A., Spalding S., Costanzo D., Rennebohm R., Waugaman B., Brodus E., Robinson A., Panupattanapong S., Rosenkranz M., Cassidy E., Torok K., Kingsbury D., Cartwright V., Lasky A., Brown D., Reiff A., Shaham B., Marzan K., Wagner-Weiner L., Onel K., Tesher M., Edens C., Moore T., Syed R., Pepmueller P., Tuttle P., Dalrymple A., Barhula S., Feller L., Horwitz M., Justice M., Nocton J., Olson J., Williams C., Versbsy J., Co D., Roth-Wojcicki E., Correll C., Vehe R., Binstadt B., Hobday P., Brueck D., Griffin T., Gillispie-Taylor M., Vora S., O'Neil K., Ballinger S., Blakley M., Klausmeier T., Oliver M., Stevens B., Rodriguez M., Go E., Bohnsack J., Inman C., Hersh A., Stern S., Woodward A., Durkee D., Boulva S. F., James K., Treemarcki E., Goldsmith D., Lvovich S., Toib D., Patel J., Jerath R., Sharma N., Newhall L., Carrasco R., Moorthy N., Boneparth A., Quintero A., Graham T., Spence S., Davis A., Gotte A., Mehta J., Walters H., Mian Z., Parkinson E., Hui-Yen J., Steigerwald K., Guzman M., Gottlieb B., Whitaker C., Kelly L., Succimarri R., Hazel E., Chedeville G., Compillo S., Leblance C., Tucker L., Cabral D., Houghton K., Guzman J., Morishita K., Huber A., Stringer E., Ramsey S., Lang B., Levy D., Silverman E., Schmeling H., Johnson N., Luca N., and Dhalla M.
Abstract: Objective: The aim of this study was to report the interim 5-year safety and effectiveness of abatacept in patients with JIA in the PRINTO/PRCSG registry. Methods: The Abatacept JIA Registry (NCT01357668) is an ongoing observational study of children with JIA receiving abatacept; enrolment started in January 2013. Clinical sites enrolled patients with JIA starting or currently receiving abatacept. Eligible patients were assessed for safety (primary end point) and effectiveness over 10 years. Effectiveness was measured by clinical 10-joint Juvenile Arthritis Disease Activity Score (cJADAS10) in patients with JIA over 5 years. As-observed analysis is presented according to the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) guidelines. Results: As of 31 March 2020, 587 patients were enrolled; 569 are included in this analysis (including 134 new users) with 1214.6 patient-years of safety data available. Over 5 years, the incidence rate (IR) per 100 patient-years of follow-up of serious adverse events was 5.52 (95% CI: 4.27, 7.01) and of events of special interest was 3.62 (95% CI: 2.63, 4.86), with 18 serious infections [IR 1.48 (95% CI: 0.88, 2.34)]. As early as month 3, 55.9% of patients achieved cJADAS10 low disease activity and inactive disease (20.3%, 72/354 and 35.6%, 126/354, respectively), sustained over 5 years. Disease activity measures improvement over 5 years across JIA categories. Conclusion: Abatacept was well tolerated in patients with JIA, with no new safety signals identified and with well-controlled disease activity, including some patients achieving inactive disease or remission.
Published: 2024

2. Defining remission in childhood-onset lupus:PReS-endorsed consensus definitions by an international task force

Author: Smith, E. M.D., Aggarwal, A., Ainsworth, J., Al-Abadi, E., Avcin, T., Bortey, L., Burnham, J., Ciurtin, C., Hedrich, C. M., Kamphuis, S., Lambert, L., Levy, D. M., Lewandowski, L., Maxwell, N., Morand, E., Özen, S., Pain, C. E., Ravelli, A., Saad Magalhaes, C., Pilkington, C., Schonenberg-Meinema, D., Scott, C., Tullus, K., Beresford, M. W., Goilav, B., Goss, N., Oni, L., Marks, S. D., Smith, E. M.D., Aggarwal, A., Ainsworth, J., Al-Abadi, E., Avcin, T., Bortey, L., Burnham, J., Ciurtin, C., Hedrich, C. M., Kamphuis, S., Lambert, L., Levy, D. M., Lewandowski, L., Maxwell, N., Morand, E., Özen, S., Pain, C. E., Ravelli, A., Saad Magalhaes, C., Pilkington, C., Schonenberg-Meinema, D., Scott, C., Tullus, K., Beresford, M. W., Goilav, B., Goss, N., Oni, L., and Marks, S. D.
Abstract: Objective: To derive childhood-onset SLE (cSLE) specific remission definitions for future treat-to-target (T2T) trials, observational studies, and clinical practice. Methods: The cSLE International T2T Task Force conducted Delphi surveys exploring paediatric perspectives on adult-onset SLE remission targets. A modified nominal group technique was used to discuss, refine, and agree on the cSLE remission target criteria.Results: The Task Force proposed two definitions of remission: ‘cSLE clinical remission on steroids (cCR)’ and ‘cSLE clinical remission off steroids (cCR-0)’. The common criteria are: (1) Clinical-SLEDAI-2 K = 0; (2) PGA score < 0.5 (0–3 scale); (4) stable antimalarials, immunosuppressive, and biologic therapy (changes due to side-effects, adherence, weight, or when building up to target dose allowed). Criterion (3) in cCR is the prednisolone dose ≤0.1 mg/kg/day (maximum 5 mg/day), whereas in cCR-0 it is zero. Conclusions: cSLE definitions of remission have been proposed, maintaining sufficient alignment with the adult-SLE definition to facilitate life-course research.
Published: 2024

3. Anakinra in Patients With Systemic Juvenile Idiopathic Arthritis: Long-term Safety From the Pharmachild Registry

Author: Giancane, G, Papa, R, Vastert, S, Bagnasco, F, Swart, J, Quartier, P, Antón, J, Kamphuis, S, Sanner, H, Glerup, M, De Benedetti, F, Tsitsami, E, Remesal, A, Moreno, E, De Inocencio, J, Myrup, C, Pallotti, C, Koné-Paut, I, Franck-Larsson, K, Malmström, H, Cederholm, S, Pistorio, A, Wulffraat, N, Ruperto, N, Giancane G, Papa R, Vastert S, Bagnasco F, Swart JF, Quartier P, Antón J, Kamphuis S, Sanner H, Glerup M, De Benedetti F, Tsitsami E, Remesal A, Moreno E, De Inocencio J, Myrup C, Pallotti C, Koné-Paut I, Franck-Larsson K, Malmström H, Cederholm S, Pistorio A, Wulffraat N, Ruperto N, Giancane, G, Papa, R, Vastert, S, Bagnasco, F, Swart, J, Quartier, P, Antón, J, Kamphuis, S, Sanner, H, Glerup, M, De Benedetti, F, Tsitsami, E, Remesal, A, Moreno, E, De Inocencio, J, Myrup, C, Pallotti, C, Koné-Paut, I, Franck-Larsson, K, Malmström, H, Cederholm, S, Pistorio, A, Wulffraat, N, Ruperto, N, Giancane G, Papa R, Vastert S, Bagnasco F, Swart JF, Quartier P, Antón J, Kamphuis S, Sanner H, Glerup M, De Benedetti F, Tsitsami E, Remesal A, Moreno E, De Inocencio J, Myrup C, Pallotti C, Koné-Paut I, Franck-Larsson K, Malmström H, Cederholm S, Pistorio A, Wulffraat N, and Ruperto N
Abstract: Objective. To evaluate the long-term safety profile of anakinra in patients with systemic juvenile idiopathic arthritis (sJIA). Methods. Data from patients with sJIA enrolled in the Pharmachild registry (ClinicalTrials.gov: NCT03932344) prior to September 30, 2018, and treated with anakinra were analyzed. The study endpoints were the occurrence of non-serious adverse events (SAEs) of at least moderate severity and SAEs, including macrophage activation syndrome (MAS), and the duration of anakinra treatment with reasons for discontinuation. All endpoints were analyzed overall by 6-month time windows, and in different treatment sets represented by those patients treated continuously with anakinra for at least 12, 18, and 24 months (set-12, -18, and -24, respectively). Results. Three hundred six patients were enrolled. Of these patients, 46%, 34%, and 28% had been treated for at least 12, 18, and 24 months, respectively. Two hundred and one AEs, mostly represented by infections, were reported for 509.3 patient-years (PY) with an overall incidence rate (IR) of 39.5 per 100 PY. Among 56 SAEs (IR 11.0/100 PY), 23.2% were infections and 19.6% MAS episodes. The IR of AEs was higher during the first 6 months of anakinra treatment, followed by decreasing IRs in the long-term treatment sets. Treatment discontinuation occurred in 76% of patients, most frequently in the first 6 months, because of inefficacy (43%), remission (31%), or AEs/intolerance (15%). No deaths or malignancies occurred during anakinra treatment. Conclusion. The results of the present study confirm the long-term safety profile of anakinra in patients with sJIA and demonstrate an overall decreasing incidence of AEs over time.
Published: 2022

4. Clinical Symptoms, Laboratory Parameters and Long-Term Follow-up in a National DADA2 Cohort.

Author: Andriessen, M.V.E., Legger, G.E., Bredius, R.G.M., Gijn, M.E. van, Hak, A.E., Muller, P.C.E.H., Kamphuis, S., Klouwer, F.C.C., Kuijpers, T.W., Leavis, H.L., Nierkens, S., Rutgers, A., Veken, L.T. van der, Well, G.T.J. van, Mulders-Manders, C.M., Montfrans, J.M. van, Andriessen, M.V.E., Legger, G.E., Bredius, R.G.M., Gijn, M.E. van, Hak, A.E., Muller, P.C.E.H., Kamphuis, S., Klouwer, F.C.C., Kuijpers, T.W., Leavis, H.L., Nierkens, S., Rutgers, A., Veken, L.T. van der, Well, G.T.J. van, Mulders-Manders, C.M., and Montfrans, J.M. van
Abstract: Contains fulltext : 296537.pdf (Publisher’s version ) (Open Access), Deficiency of adenosine deaminase-2 (DADA2) is an autosomal recessive autoinflammatory disease with an extremely variable disease presentation. This paper provides a comprehensive overview of the Dutch DADA2 cohort. We performed a retrospective cohort study in 29 ADA2-deficient patients from 23 families with a median age at inclusion of 26 years. All patients had biallelic pathogenic variants in the ADA2 gene. The most common clinical findings included cutaneous involvement (79.3%), (hepato)splenomegaly (70.8%) and recurrent infections (58.6%). Stroke was observed in 41.4% of the patients. The main laboratory abnormalities were hypogammaglobulinemia and various cytopenias. Patients presented most often with a mixed phenotype involving vasculopathy, immunodeficiency and hematologic manifestations (62.1%). In this cohort, malignancies were reported in eight patients (27.6%), of whom five presented with a hematologic malignancy and two with a basal cell carcinoma. Four patients developed hemophagocytic lymphohistiocytosis (HLH) or an HLH-like episode, of whom three passed away during or shortly after the occurrence of HLH. TNF-inhibitors (TNFi) were effective in treating vasculopathy-associated symptoms and preventing stroke, but were hardly effective in the treatment of hematologic manifestations. Three patients underwent hematopoietic cell transplantation and two of them are doing well with complete resolution of DADA2-related symptoms. The overall mortality in this cohort was 17.2%. In conclusion, this cohort describes the clinical, genetic and laboratory findings of 29 Dutch DADA2 patients. We describe the occurrence of HLH as a life-threatening disease complication and report a relatively high incidence of malignancies and mortality.
Published: 2023

5. Dysregulated endothelial cell markers in systemic lupus erythematosus:a systematic review and meta-analysis

Author: Bergkamp, S. C., Wahadat, M. J., Salah, A., Kuijpers, T. W., Smith, V., Tas, S. W., van den Berg, J. M., Kamphuis, S., Schonenberg-Meinema, D., Bergkamp, S. C., Wahadat, M. J., Salah, A., Kuijpers, T. W., Smith, V., Tas, S. W., van den Berg, J. M., Kamphuis, S., and Schonenberg-Meinema, D.
Abstract: Objectives: To perform a systematic literature review and meta-analysis on endothelial cell (EC) markers that are involved and dysregulated in systemic lupus erythematosus (SLE) in relation to disease activity, as EC dysregulation plays a major role in the development of premature atherosclerosis in SLE. Methods: Search terms were entered into Embase, MEDLINE, Web of Science, Google Scholar and Cochrane. Inclusion criteria were 1) studies published after 2000 reporting measurements of EC markers in serum and/or plasma of SLE patients (diagnosed according to ACR/SLICC criteria), 2) English language peer reviewed articles, and 3) disease activity measurement. For meta-analysis calculations, the Meta-Essentials tool by Erasmus Research Institute and of Management (ERIM) was used. Only those EC markers, which were 1) reported in at least two articles and 2) reported a correlation coefficient (i.e. Spearman’s rank or Pearson’s) between the measured levels of the EC marker and disease activity were included. For meta-analyses, a fixed effect model was used. Results: From 2133 hits, 123 eligible articles were selected. The identified SLE-related endothelial markers were involved in EC activation, EC apoptosis, disturbed angiogenesis, defective vascular tone control, immune dysregulation and coagulopathy. Meta-analyses of primarily cross-sectional studies showed significant associations between marker levels and disease activity for the following endothelial markers: Pentraxin-3, Thrombomodulin, VEGF, VCAM-1, ICAM-1, IP-10 and MCP-1. Dysregulated EC markers without associations with disease activity were: Angiopoeitin-2, vWF, P-Selectin, TWEAK and E-Selectin. Conclusions: We provide a complete literature overview for dysregulated EC markers in SLE comprising a wide range of different EC functions. SLE-induced EC marker dysregulation was seen with, but also without, association with disease activity. This study provides some clarity in the eminent complex field of EC mark
Published: 2023

6. Anakinra in Patients With Systemic Juvenile Idiopathic Arthritis: Long-term Safety From the Pharmachild Registry

Author: Giancane, G. Papa, R. Vastert, S. Bagnasco, F. Swart, J.F. Quartier, P. Antón, J. Kamphuis, S. Sanner, H. Glerup, M. De Benedetti, F. Tsitsami, E. Remesal, A. Moreno, E. De Inocencio, J. Myrup, C. Pallotti, C. Koné-Paut, I. Franck-Larsson, K. Malmström, H. Cederholm, S. Pistorio, A. Wulffraat, N. Ruperto, N. the Paediatric Rheumatology International Trials Organisation (PRINTO) and Giancane, G. Papa, R. Vastert, S. Bagnasco, F. Swart, J.F. Quartier, P. Antón, J. Kamphuis, S. Sanner, H. Glerup, M. De Benedetti, F. Tsitsami, E. Remesal, A. Moreno, E. De Inocencio, J. Myrup, C. Pallotti, C. Koné-Paut, I. Franck-Larsson, K. Malmström, H. Cederholm, S. Pistorio, A. Wulffraat, N. Ruperto, N. the Paediatric Rheumatology International Trials Organisation (PRINTO)
Abstract: Objective. To evaluate the long-term safety profile of anakinra in patients with systemic juvenile idiopathic arthritis (sJIA). Methods. Data from patients with sJIA enrolled in the Pharmachild registry (ClinicalTrials.gov: NCT03932344) prior to September 30, 2018, and treated with anakinra were analyzed. The study endpoints were the occurrence of non-serious adverse events (SAEs) of at least moderate severity and SAEs, including macrophage activation syndrome (MAS), and the duration of anakinra treatment with reasons for discontinuation. All endpoints were analyzed overall by 6-month time windows, and in different treatment sets represented by those patients treated continuously with anakinra for at least 12, 18, and 24 months (set-12, -18, and -24, respectively). Results. Three hundred six patients were enrolled. Of these patients, 46%, 34%, and 28% had been treated for at least 12, 18, and 24 months, respectively. Two hundred and one AEs, mostly represented by infections, were reported for 509.3 patient-years (PY) with an overall incidence rate (IR) of 39.5 per 100 PY. Among 56 SAEs (IR 11.0/100 PY), 23.2% were infections and 19.6% MAS episodes. The IR of AEs was higher during the first 6 months of anakinra treatment, followed by decreasing IRs in the long-term treatment sets. Treatment discontinuation occurred in 76% of patients, most frequently in the first 6 months, because of inefficacy (43%), remission (31%), or AEs/intolerance (15%). No deaths or malignancies occurred during anakinra treatment. Conclusion. The results of the present study confirm the long-term safety profile of anakinra in patients with sJIA and demonstrate an overall decreasing incidence of AEs over time. © 2022 The Journal of Rheumatology.
Published: 2022

7. International Consensus for the Dosing of Corticosteroids in Childhood-Onset Systemic Lupus Erythematosus With Proliferative Lupus Nephritis

Author: Chalhoub, N.E. Wenderfer, S.E. Levy, D.M. Rouster-Stevens, K. Aggarwal, A. Savani, S.I. Ruth, N.M. Arkachaisri, T. Qiu, T. Merritt, A. Onel, K. Goilav, B. Khubchandani, R.P. Deng, J. Fonseca, A.R. Ardoin, S.P. Ciurtin, C. Kasapcopur, O. Jelusic, M. Huber, A.M. Ozen, S. Klein-Gitelman, M.S. Appenzeller, S. Cavalcanti, A. Fotis, L. Lim, S.C. Silva, R.M. Miramontes, J.R. Rosenwasser, N.L. Saad-Magalhaes, C. Schonenberg-Meinema, D. Scott, C. Silva, C.A. Enciso, S. Terreri, M.T. Torres-Jimenez, A.-R. Trachana, M. Al-Mayouf, S.M. Devarajan, P. Huang, B. Brunner, H.I. Abulaban, K. Aguiar, C. Ahn, S.-Y. Akoghlanian, S. Al-Abrawi, S. Aljaberi, N. Alperin, R. Angeles-Han, S. Ardalan, K. Bader-Meunier, B. Balboni, I. Barbar-Smiley, F. Baxter, S. Beary, J. Boneparth, A. Brakeman, P. Bridges, J. Burgos-Vargas, R. Cabral, D.A. Cameto, J. Carter, C. Chang, J. Chédeville, G. Chhakchhuak, C. Chiraseveenuprapund, P. Cifuentes Alvarado, M. Concannon, A. Cooper, J. Cron, R. De Carvalho, L.M. De Quattro, K. De Ranieri, D. Dizon, B. Donnelly Wrigley, C. Duong, M.D. Eberhard, A. Ede, K. Edelheit, B. Edens, C. Espada, G. Farhey, Y. Flores, F. Fritz, D. Ganguli, S. Gilbert, M. Gittar, P. Greenbaum, L. Grom, A. Gulati, G. Harry, O. Hayward, K. Henrickson, M. Hersh, A. Hiraki, L. Hiskey, M. Hoffmann, S. Hollander, M. Hom, C. Houk, L. Houk, J.B. Hsieh, E.W.Y. Hsu, J. Jensen, P. Joos, R. Jurado, R. Jusan Fiorot, F. Kallash, M. Kamphuis, S. Keltsev, V., (deceased) Khanna, S. Kim, S. Kimseng, K.J. Knight, A. Kunder, R. Lai, J. Laskin, B. Lewandowski, L. Lim, L. Linda, W.-W. Lo, M. Lovell, D. Luggen, M. Madison, J. Mansuri, A. Martin, L. Mason, S. Miller, M. Mina, R. Mohammed, A. Moncrieffe, H. Moorthy, L. Morgan, E. Mosquera, A. Muntel, E. Muscal, E. Myones, B. Nocton, J. Ogbu, E. Okamura, D. Olson, J. Orrock, J. Paim-Marques, L. Pain, C. Park, C. Patel, P. Pereira, M. Prado, R.D. Radhakrishna, S. Rheault, M. Ridgway, W. Riskalla, M. Ronis, T. Sadun, R. Sagcal-Gironella, A.C. Santos, M.C. Schikle and Chalhoub, N.E. Wenderfer, S.E. Levy, D.M. Rouster-Stevens, K. Aggarwal, A. Savani, S.I. Ruth, N.M. Arkachaisri, T. Qiu, T. Merritt, A. Onel, K. Goilav, B. Khubchandani, R.P. Deng, J. Fonseca, A.R. Ardoin, S.P. Ciurtin, C. Kasapcopur, O. Jelusic, M. Huber, A.M. Ozen, S. Klein-Gitelman, M.S. Appenzeller, S. Cavalcanti, A. Fotis, L. Lim, S.C. Silva, R.M. Miramontes, J.R. Rosenwasser, N.L. Saad-Magalhaes, C. Schonenberg-Meinema, D. Scott, C. Silva, C.A. Enciso, S. Terreri, M.T. Torres-Jimenez, A.-R. Trachana, M. Al-Mayouf, S.M. Devarajan, P. Huang, B. Brunner, H.I. Abulaban, K. Aguiar, C. Ahn, S.-Y. Akoghlanian, S. Al-Abrawi, S. Aljaberi, N. Alperin, R. Angeles-Han, S. Ardalan, K. Bader-Meunier, B. Balboni, I. Barbar-Smiley, F. Baxter, S. Beary, J. Boneparth, A. Brakeman, P. Bridges, J. Burgos-Vargas, R. Cabral, D.A. Cameto, J. Carter, C. Chang, J. Chédeville, G. Chhakchhuak, C. Chiraseveenuprapund, P. Cifuentes Alvarado, M. Concannon, A. Cooper, J. Cron, R. De Carvalho, L.M. De Quattro, K. De Ranieri, D. Dizon, B. Donnelly Wrigley, C. Duong, M.D. Eberhard, A. Ede, K. Edelheit, B. Edens, C. Espada, G. Farhey, Y. Flores, F. Fritz, D. Ganguli, S. Gilbert, M. Gittar, P. Greenbaum, L. Grom, A. Gulati, G. Harry, O. Hayward, K. Henrickson, M. Hersh, A. Hiraki, L. Hiskey, M. Hoffmann, S. Hollander, M. Hom, C. Houk, L. Houk, J.B. Hsieh, E.W.Y. Hsu, J. Jensen, P. Joos, R. Jurado, R. Jusan Fiorot, F. Kallash, M. Kamphuis, S. Keltsev, V., (deceased) Khanna, S. Kim, S. Kimseng, K.J. Knight, A. Kunder, R. Lai, J. Laskin, B. Lewandowski, L. Lim, L. Linda, W.-W. Lo, M. Lovell, D. Luggen, M. Madison, J. Mansuri, A. Martin, L. Mason, S. Miller, M. Mina, R. Mohammed, A. Moncrieffe, H. Moorthy, L. Morgan, E. Mosquera, A. Muntel, E. Muscal, E. Myones, B. Nocton, J. Ogbu, E. Okamura, D. Olson, J. Orrock, J. Paim-Marques, L. Pain, C. Park, C. Patel, P. Pereira, M. Prado, R.D. Radhakrishna, S. Rheault, M. Ridgway, W. Riskalla, M. Ronis, T. Sadun, R. Sagcal-Gironella, A.C. Santos, M.C. Schikle
Abstract: Objective: To develop a standardized steroid dosing regimen (SSR) for physicians treating childhood-onset systemic lupus erythematosus (SLE) complicated by lupus nephritis (LN), using consensus formation methodology. Methods: Parameters influencing corticosteroid (CS) dosing were identified (step 1). Data from children with proliferative LN were used to generate patient profiles (step 2). Physicians rated changes in renal and extrarenal childhood-onset SLE activity between 2 consecutive visits and proposed CS dosing (step 3). The SSR was developed using patient profile ratings (step 4), with refinements achieved in a physician focus group (step 5). A second type of patient profile describing the course of childhood-onset SLE for ≥4 months since kidney biopsy was rated to validate the SSR-recommended oral and intravenous (IV) CS dosages (step 6). Patient profile adjudication was based on majority ratings for both renal and extrarenal disease courses, and consensus level was set at 80%. Results: Degree of proteinuria, estimated glomerular filtration rate, changes in renal and extrarenal disease activity, and time since kidney biopsy influenced CS dosing (steps 1 and 2). Considering these parameters in 5,056 patient profile ratings from 103 raters, and renal and extrarenal course definitions, CS dosing rules of the SSR were developed (steps 3–5). Validation of the SSR for up to 6 months post–kidney biopsy was achieved with 1,838 patient profile ratings from 60 raters who achieved consensus for oral and IV CS dosage in accordance with the SSR (step 6). Conclusion: The SSR represents an international consensus on CS dosing for use in patients with childhood-onset SLE and proliferative LN. The SSR is anticipated to be used for clinical care and to standardize CS dosage during clinical trials. © 2021, American College of Rheumatology
Published: 2022

8. Gene signature fingerprints stratify SLE patients in groups with similar biological disease profiles: a multicentre longitudinal study

Author: Wahadat, M.J., Schonenberg-Meinema, D., Helden-Meeuwsen, C.G. van, Tilburg, S.J. van, Groot, Noortje, Schatorje, E.J.H., Hoppenreijs, E.P., Muller, P.C., Brinkman, D.M., Dvorak, D., Verkaaik, M., Berg, J.Merlijn van den, Bouchalova, K., Kamphuis, S., Versnel, M.A., Wahadat, M.J., Schonenberg-Meinema, D., Helden-Meeuwsen, C.G. van, Tilburg, S.J. van, Groot, Noortje, Schatorje, E.J.H., Hoppenreijs, E.P., Muller, P.C., Brinkman, D.M., Dvorak, D., Verkaaik, M., Berg, J.Merlijn van den, Bouchalova, K., Kamphuis, S., and Versnel, M.A.
Abstract: Item does not contain fulltext, OBJECTIVES: Clinical phenotyping and predicting treatment responses in SLE patients is challenging. Extensive blood transcriptional profiling has identified various gene modules that are promising for stratification of SLE patients. We aimed to translate existing transcriptomic data into simpler gene signatures suitable for daily clinical practice. METHODS: Real-time PCR of multiple genes from the IFN M1.2, IFN M5.12, neutrophil (NPh) and plasma cell (PLC) modules, followed by a principle component analysis, was used to identify indicator genes per gene signature. Gene signatures were measured in longitudinal samples from two childhood-onset SLE cohorts (n = 101 and n = 34, respectively), and associations with clinical features were assessed. Disease activity was measured using Safety of Estrogen in Lupus National Assessment (SELENA)-SLEDAI. Cluster analysis subdivided patients into three mutually exclusive fingerprint-groups termed (1) all-signatures-low, (2) only IFN high (M1.2 and/or M5.12) and (3) high NPh and/or PLC. RESULTS: All gene signatures were significantly associated with disease activity in cross-sectionally collected samples. The PLC-signature showed the highest association with disease activity. Interestingly, in longitudinally collected samples, the PLC-signature was associated with disease activity and showed a decrease over time. When patients were divided into fingerprints, the highest disease activity was observed in the high NPh and/or PLC group. The lowest disease activity was observed in the all-signatures-low group. The same distribution was reproduced in samples from an independent SLE cohort. CONCLUSIONS: The identified gene signatures were associated with disease activity and were indicated to be suitable tools for stratifying SLE patients into groups with similar activated immune pathways that may guide future treatment choices.
Published: 2022

9. Gene signature fingerprints stratify SLE patients in groups with similar biological disease profiles: a multicentre longitudinal study

Author: Wahadat, M.J., Schonenberg-Meinema, D., Helden-Meeuwsen, C.G. van, Tilburg, S.J. van, Groot, Noortje, Schatorje, E.J.H., Hoppenreijs, E.P., Muller, P.C., Brinkman, D.M., Dvorak, D., Verkaaik, M., Berg, J.Merlijn van den, Bouchalova, K., Kamphuis, S., Versnel, M.A., Wahadat, M.J., Schonenberg-Meinema, D., Helden-Meeuwsen, C.G. van, Tilburg, S.J. van, Groot, Noortje, Schatorje, E.J.H., Hoppenreijs, E.P., Muller, P.C., Brinkman, D.M., Dvorak, D., Verkaaik, M., Berg, J.Merlijn van den, Bouchalova, K., Kamphuis, S., and Versnel, M.A.
Abstract: Contains fulltext : 287954.pdf (Publisher’s version ) (Open Access), OBJECTIVES: Clinical phenotyping and predicting treatment responses in SLE patients is challenging. Extensive blood transcriptional profiling has identified various gene modules that are promising for stratification of SLE patients. We aimed to translate existing transcriptomic data into simpler gene signatures suitable for daily clinical practice. METHODS: Real-time PCR of multiple genes from the IFN M1.2, IFN M5.12, neutrophil (NPh) and plasma cell (PLC) modules, followed by a principle component analysis, was used to identify indicator genes per gene signature. Gene signatures were measured in longitudinal samples from two childhood-onset SLE cohorts (n = 101 and n = 34, respectively), and associations with clinical features were assessed. Disease activity was measured using Safety of Estrogen in Lupus National Assessment (SELENA)-SLEDAI. Cluster analysis subdivided patients into three mutually exclusive fingerprint-groups termed (1) all-signatures-low, (2) only IFN high (M1.2 and/or M5.12) and (3) high NPh and/or PLC. RESULTS: All gene signatures were significantly associated with disease activity in cross-sectionally collected samples. The PLC-signature showed the highest association with disease activity. Interestingly, in longitudinally collected samples, the PLC-signature was associated with disease activity and showed a decrease over time. When patients were divided into fingerprints, the highest disease activity was observed in the high NPh and/or PLC group. The lowest disease activity was observed in the all-signatures-low group. The same distribution was reproduced in samples from an independent SLE cohort. CONCLUSIONS: The identified gene signatures were associated with disease activity and were indicated to be suitable tools for stratifying SLE patients into groups with similar activated immune pathways that may guide future treatment choices.
Published: 2022

10. LONG-TERM SAFETY OF ANAKINRA IN PATIENTS WITH SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS FROM THE PHARMACHILD REGISTRY

Author: Giancane, G. Papa, R. Vastert, S. Bagnasco, F. Swart, J. F. Quartier, P. Hofer, M. Anton, J. Kamphuis, S. and Sanner, H. Glerup, M. De Benedetti, F. Tsitsami, E. and Remesal, A. Ruzafa, E. Moreno De Inocencio, J. Myrup, C. and Pallotti, C. Kone-Paut, I. Franck-Larsson, K. Malmstrom, H. and Cederholm, S. Pistorio, A. Wulffraat, N. Ruperto, N. and Giancane, G. Papa, R. Vastert, S. Bagnasco, F. Swart, J. F. Quartier, P. Hofer, M. Anton, J. Kamphuis, S. and Sanner, H. Glerup, M. De Benedetti, F. Tsitsami, E. and Remesal, A. Ruzafa, E. Moreno De Inocencio, J. Myrup, C. and Pallotti, C. Kone-Paut, I. Franck-Larsson, K. Malmstrom, H. and Cederholm, S. Pistorio, A. Wulffraat, N. Ruperto, N.
Published: 2021

11. Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: Analysis by the Pharmachild Safety Adjudication Committee

Author: Giancane, G. Swart, J.F. Castagnola, E. Groll, A.H. Horneff, G. Huppertz, H.-I. Lovell, D.J. Wolfs, T. Herlin, T. Dolezalova, P. Sanner, H. Susic, G. Sztajnbok, F. Maritsi, D. Constantin, T. Vargova, V. Sawhney, S. Rygg, M. Oliveira, S.K. Cattalini, M. Bovis, F. Bagnasco, F. Pistorio, A. Martini, A. Wulffraat, N. Ruperto, N. Cuttica, R. Garay, S.M. Brunner, J. Emminger, W. Appenzeller, S. Len, C. Saad Magalhaes, C. Telcharova-Mihaylovska, A. Harjacek, M. Jelusic, M. Estmann, A. Nielsen, S. Herrera Mora, C. Gervais, E. Koné-Paut, I. Quartier, P. Foeldvari, I. Horneff, G. Lutz, T. Minden, K. Tzaribachev, N. Trachana, M. Tsitsami, E. Vougiouka, O. Orban, I. Harel, L. Hashkes, P. Uziel, Y. Cimaz, R. Civino, A. Consolini, R. D'Angelo, G. De Benedetti, F. Filocamo, G. Fueri, E. Gallizzi, R. Maggio, M.C. Magnolia, M.G. Miniaci, A. Montin, D. Olivieri, A.N. Pastore, S. Rigante, D. Zulian, F. Rumba-Rozenfelde, I. Stanevicha, V. Panaviene, V. Rodriguez Lozano, A.L. Rubio-Perez, N. Vega Cornejo, G. Hoppenreijs, E. Kamphuis, S. Flato, B. Nordal, E.B. Abdwani, R. Miraval, T. Paz Gastanaga, M.E. Smolewska, E. Ailioaie, C. Cochino, A.-V. Laday, M. Lazar, C. Alexeeva, E. Chasnyk, V. Keltsev, V. Suwairi, W.M.S. Vijatov-Djuric, G. Vojinovic, J. Arkachaisri, T. Koskova, E. Avcin, T. Ally, M. Van Rensburg, C.J. Louw, I. Lopez, J.A. Boteanu, A.L. Calvo Penades, I. De Inocencio, J. Mesa-Del-Castillo, P. Moreno, E. Remesal, A. Hofer, M. Gok, F. Ozen, S. Ramanan, A. Pallotti, C. Villa, L. and Giancane, G. Swart, J.F. Castagnola, E. Groll, A.H. Horneff, G. Huppertz, H.-I. Lovell, D.J. Wolfs, T. Herlin, T. Dolezalova, P. Sanner, H. Susic, G. Sztajnbok, F. Maritsi, D. Constantin, T. Vargova, V. Sawhney, S. Rygg, M. Oliveira, S.K. Cattalini, M. Bovis, F. Bagnasco, F. Pistorio, A. Martini, A. Wulffraat, N. Ruperto, N. Cuttica, R. Garay, S.M. Brunner, J. Emminger, W. Appenzeller, S. Len, C. Saad Magalhaes, C. Telcharova-Mihaylovska, A. Harjacek, M. Jelusic, M. Estmann, A. Nielsen, S. Herrera Mora, C. Gervais, E. Koné-Paut, I. Quartier, P. Foeldvari, I. Horneff, G. Lutz, T. Minden, K. Tzaribachev, N. Trachana, M. Tsitsami, E. Vougiouka, O. Orban, I. Harel, L. Hashkes, P. Uziel, Y. Cimaz, R. Civino, A. Consolini, R. D'Angelo, G. De Benedetti, F. Filocamo, G. Fueri, E. Gallizzi, R. Maggio, M.C. Magnolia, M.G. Miniaci, A. Montin, D. Olivieri, A.N. Pastore, S. Rigante, D. Zulian, F. Rumba-Rozenfelde, I. Stanevicha, V. Panaviene, V. Rodriguez Lozano, A.L. Rubio-Perez, N. Vega Cornejo, G. Hoppenreijs, E. Kamphuis, S. Flato, B. Nordal, E.B. Abdwani, R. Miraval, T. Paz Gastanaga, M.E. Smolewska, E. Ailioaie, C. Cochino, A.-V. Laday, M. Lazar, C. Alexeeva, E. Chasnyk, V. Keltsev, V. Suwairi, W.M.S. Vijatov-Djuric, G. Vojinovic, J. Arkachaisri, T. Koskova, E. Avcin, T. Ally, M. Van Rensburg, C.J. Louw, I. Lopez, J.A. Boteanu, A.L. Calvo Penades, I. De Inocencio, J. Mesa-Del-Castillo, P. Moreno, E. Remesal, A. Hofer, M. Gok, F. Ozen, S. Ramanan, A. Pallotti, C. Villa, L.
Abstract: Background: To derive a list of opportunistic infections (OI) through the analysis of the juvenile idiopathic arthritis (JIA) patients in the Pharmachild registry by an independent Safety Adjudication Committee (SAC). Methods: The SAC (3 pediatric rheumatologists and 2 pediatric infectious disease specialists) elaborated and approved by consensus a provisional list of OI for use in JIA. Through a 5 step-procedure, all the severe and serious infections, classified as per MedDRA dictionary and retrieved in the Pharmachild registry, were evaluated by the SAC by answering six questions and adjudicated with the agreement of 3/5 specialists. A final evidence-based list of OI resulted by matching the adjudicated infections with the provisional list of OI. Results: A total of 772 infectious events in 572 eligible patients, of which 335 serious/severe/very severe non-OI and 437 OI (any intensity/severity), according to the provisional list, were retrieved. Six hundred eighty-two of 772 (88.3%) were adjudicated as infections, of them 603/682 (88.4%) as common and 119/682 (17.4%) as OI by the SAC. Matching these 119 opportunistic events with the provisional list, 106 were confirmed by the SAC as OI, and among them infections by herpes viruses were the most frequent (68%), followed by tuberculosis (27.4%). The remaining events were divided in the groups of non-OI and possible/patient and/or pathogen-related OI. Conclusions: We found a significant number of OI in JIA patients on immunosuppressive therapy. The proposed list of OI, created by consensus and validated in the Pharmachild cohort, could facilitate comparison among future pharmacovigilance studies. Trial registration: Clinicaltrials.gov NCT 01399281; ENCePP seal: awarded on 25 November 2011. © 2020 The Author(s).
Published: 2020

12. Long-term follow-up after CMI® Prosthesis for failed trapeziectomy with suspension arthroplasty

Author: Kamphuis, S, Lorenzini, R, Jandali, AR, Sproedt, J, Kamphuis, S, Lorenzini, R, Jandali, AR, and Sproedt, J
Published: 2020

13. Closed screw fixation versus open reduction and bone grafting in delayed union scaphoid fractures

Author: Kamphuis, S, Sproedt, J, Kamphuis, S, and Sproedt, J
Published: 2020

14. Long-term follow-up after CMI® Prosthesis for failed trapeziectomy with suspension arthroplasty

Author: Kamphuis, S, Lorenzini, R, Jandali, AR, Sproedt, J, Kamphuis, S, Lorenzini, R, Jandali, AR, and Sproedt, J
Published: 2020

15. Closed screw fixation versus open reduction and bone grafting in delayed union scaphoid fractures

Author: Kamphuis, S, Sproedt, J, Kamphuis, S, and Sproedt, J
Published: 2020

16. Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Author: Giancane, G, Swart, Jf, Castagnola, E, Groll, Ah, Horneff, G, Huppertz, Hi, Lovell, Dj, Wolfs, T, Herlin, T, Dolezalova, P, Sanner, H, Susic, G, Sztajnbok, F, Maritsi, D, Constantin, T, Vargova, V, Sawhney, S, Rygg, M, K Oliveira, S, Cattalini, M, Bovis, F, Bagnasco, F, Pistorio, A, Martini, A, Wulffraat, N, Ruperto, N, Cuttica, R, Garay, Sm, Brunner, J, Emminger, W, Appenzeller, S, Len, C, Saad Magalhaes, C, Telcharova-Mihaylovska, A, Harjacek, M, Jelusic, M, Estmann, A, Nielsen, S, Herrera Mora, C, Gervais, E, Koné-Paut, I, Quartier, P, Foeldvari, I, Lutz, T, Minden, K, Tzaribachev, N, Trachana, M, Tsitsami, E, Vougiouka, O, Orban, I, Harel, L, Hashkes, P, Uziel, Y, Cimaz, R, Civino, A, Consolini, R, D'Angelo, G, De Benedetti, F, Filocamo, G, Fueri, E, Gallizzi, R, Maggio, Mc, Magnolia, Mg, Miniaci, A, Montin, D, Olivieri, An, Pastore, S, Rigante, Donato, Zulian, F, Rumba-Rozenfelde, I, Stanevicha, V, Panaviene, V, Rodriguez Lozano, Al, Rubio-Perez, N, Vega Cornejo, G, Hoppenreijs, E, Kamphuis, S, Flato, B, Nordal, Eb, Abdwani, R, Miraval, T, Paz Gastanaga, Me, Smolewska, E, Ailioaie, C, Cochino, Av, Laday, M, Lazar, C, Alexeeva, E, Chasnyk, V, Keltsev, V, Suwairi, Wm, Vijatov-Djuric, G, Vojinovic, J, Arkachaisri, T, Koskova, E, Avcin, T, Ally, M, Van Rensburg, Cj, Louw, I, Lopez, Ja, Boteanu, Al, Calvo Penades, I, De Inocencio, J, Mesa-Del-Castillo, P, Moreno, E, Remesal, A, Hofer, M, Gok, F, Ozen, S, Ramanan, A, Pallotti, C, Villa, L &amp, Paediatric Rheumatology International Trials Organisation, (PRINTO), Rigante D (ORCID:0000-0001-7032-7779), Giancane, G, Swart, Jf, Castagnola, E, Groll, Ah, Horneff, G, Huppertz, Hi, Lovell, Dj, Wolfs, T, Herlin, T, Dolezalova, P, Sanner, H, Susic, G, Sztajnbok, F, Maritsi, D, Constantin, T, Vargova, V, Sawhney, S, Rygg, M, K Oliveira, S, Cattalini, M, Bovis, F, Bagnasco, F, Pistorio, A, Martini, A, Wulffraat, N, Ruperto, N, Cuttica, R, Garay, Sm, Brunner, J, Emminger, W, Appenzeller, S, Len, C, Saad Magalhaes, C, Telcharova-Mihaylovska, A, Harjacek, M, Jelusic, M, Estmann, A, Nielsen, S, Herrera Mora, C, Gervais, E, Koné-Paut, I, Quartier, P, Foeldvari, I, Lutz, T, Minden, K, Tzaribachev, N, Trachana, M, Tsitsami, E, Vougiouka, O, Orban, I, Harel, L, Hashkes, P, Uziel, Y, Cimaz, R, Civino, A, Consolini, R, D'Angelo, G, De Benedetti, F, Filocamo, G, Fueri, E, Gallizzi, R, Maggio, Mc, Magnolia, Mg, Miniaci, A, Montin, D, Olivieri, An, Pastore, S, Rigante, Donato, Zulian, F, Rumba-Rozenfelde, I, Stanevicha, V, Panaviene, V, Rodriguez Lozano, Al, Rubio-Perez, N, Vega Cornejo, G, Hoppenreijs, E, Kamphuis, S, Flato, B, Nordal, Eb, Abdwani, R, Miraval, T, Paz Gastanaga, Me, Smolewska, E, Ailioaie, C, Cochino, Av, Laday, M, Lazar, C, Alexeeva, E, Chasnyk, V, Keltsev, V, Suwairi, Wm, Vijatov-Djuric, G, Vojinovic, J, Arkachaisri, T, Koskova, E, Avcin, T, Ally, M, Van Rensburg, Cj, Louw, I, Lopez, Ja, Boteanu, Al, Calvo Penades, I, De Inocencio, J, Mesa-Del-Castillo, P, Moreno, E, Remesal, A, Hofer, M, Gok, F, Ozen, S, Ramanan, A, Pallotti, C, Villa, L &amp, Paediatric Rheumatology International Trials Organisation, (PRINTO), and Rigante D (ORCID:0000-0001-7032-7779)
Abstract: BACKGROUND: To derive a list of opportunistic infections (OI) through the analysis of the juvenile idiopathic arthritis (JIA) patients in the Pharmachild registry by an independent Safety Adjudication Committee (SAC). METHODS: The SAC (3 pediatric rheumatologists and 2 pediatric infectious disease specialists) elaborated and approved by consensus a provisional list of OI for use in JIA. Through a 5 step-procedure, all the severe and serious infections, classified as per MedDRA dictionary and retrieved in the Pharmachild registry, were evaluated by the SAC by answering six questions and adjudicated with the agreement of 3/5 specialists. A final evidence-based list of OI resulted by matching the adjudicated infections with the provisional list of OI. RESULTS: A total of 772 infectious events in 572 eligible patients, of which 335 serious/severe/very severe non-OI and 437 OI (any intensity/severity), according to the provisional list, were retrieved. Six hundred eighty-two of 772 (88.3%) were adjudicated as infections, of them 603/682 (88.4%) as common and 119/682 (17.4%) as OI by the SAC. Matching these 119 opportunistic events with the provisional list, 106 were confirmed by the SAC as OI, and among them infections by herpes viruses were the most frequent (68%), followed by tuberculosis (27.4%). The remaining events were divided in the groups of non-OI and possible/patient and/or pathogen-related OI. CONCLUSIONS: We found a significant number of OI in JIA patients on immunosuppressive therapy. The proposed list of OI, created by consensus and validated in the Pharmachild cohort, could facilitate comparison among future pharmacovigilance studies.
Published: 2020

17. Phenotypic variability and disparities in treatment and outcomes of childhood arthritis throughout the world: an observational cohort study

Author: Consolaro, A, Giancane, G, Alongi, A, van Dijkhuizen, Ehp, Aggarwal, A, Al-Mayouf, Sm, Bovis, F, De Inocencio, J, Demirkaya, E, Flato, B, Foell, D, Garay, Sm, Lazăr, C, Lovell, Dj, Montobbio, C, Miettunen, P, Mihaylova, D, Nielsen, S, Orban, I, Rumba-Rozenfelde, I, Magalhães, C, Shafaie, N, Susic, G, Trachana, M, Wulffraat, N, Pistorio, A, Martini, A, Ruperto, N, Ravelli, A &amp, Abdwani R, Aghighi, Y, Aiche, Mf, Ailioaie, C, Aktay Ayaz, N, Al-Abrawi, S, Alexeeva, E, Anton, J, Apostol, A, Arguedas, O, Avcin, T, Barone, P, Berntson, L, Boteanu, Al, Boyko, Y, Burgos-Vargas, R, Calvo Penades, I, Chédeville, G, Cimaz, R, Civino, A, Consolini, R, Constantin, T, Cuttica, R, Dallos, T, Martin, N, Magni-Manzoni, S, De Cunto, C, Dolezalova, P, Ekelund, M, El Miedany, Y, Espada, G, Estmann Christensen, A, Foeldvari, I, Gallizzi, R, Ganser, G, Gerloni, V, Haas, Jp, Harel, L, Harjacek, M, Hashad, S, Herlin, T, Herrera, C, Hofer, M, Holzinger, D, Horneff, G, Huppertz, Hi, Iagăru, N, Ibanez Estrella, A, Ioseliani, M, Joos, R, Knupp Oliveira, S, Kamphuis, S, Kasapcopur, O, Katsicas, Mm, Khubchandani, R, Kondi, A, Kröger, L, La Torre, F, Laday, M, Lahdenne, P, Maggio, Mc, Magnolia, Mg, Malagon, C, Malin, M, Martino, S, Melo-Gomes, Ja, Mesa-Del-Castillo, P, Militaru, A, Minden, K, Miniaci, A, Moradinejad, Mh, Morel Ayala, Z, Nikishina, I, Norambuena, X, Nordal, Eb, Pagava, K, Panaviene, V, Pastore, S, Pieropan, S, Podda, Ra, Pruunsild, C, Putto-Laurila, A, Quartier, P, Remesal, A, Rigante, Donato, Ringold, S, Rutkowska-Sak, L, Rygg, M, Saurenmann, Rk, Sawhney, S, Scott, C, Shiari, R, Smolewska, E, Sozeri, B, Swart, Jf, Sztajnbok, F, Torcoletti, M, Tsitsami, E, Tzaribachev, N, Unsal, E, Uziel, Y, Vähäsalo, P, Varbanova, B, Vargova, V, Vesely, R, Vijatov-Djuric, G, Vilaiyuk, S, Vojinovic, J, Vougiouka, O, Weiss, P, Wouters, C, Rigante D (ORCID:0000-0001-7032-7779), Consolaro, A, Giancane, G, Alongi, A, van Dijkhuizen, Ehp, Aggarwal, A, Al-Mayouf, Sm, Bovis, F, De Inocencio, J, Demirkaya, E, Flato, B, Foell, D, Garay, Sm, Lazăr, C, Lovell, Dj, Montobbio, C, Miettunen, P, Mihaylova, D, Nielsen, S, Orban, I, Rumba-Rozenfelde, I, Magalhães, C, Shafaie, N, Susic, G, Trachana, M, Wulffraat, N, Pistorio, A, Martini, A, Ruperto, N, Ravelli, A &amp, Abdwani R, Aghighi, Y, Aiche, Mf, Ailioaie, C, Aktay Ayaz, N, Al-Abrawi, S, Alexeeva, E, Anton, J, Apostol, A, Arguedas, O, Avcin, T, Barone, P, Berntson, L, Boteanu, Al, Boyko, Y, Burgos-Vargas, R, Calvo Penades, I, Chédeville, G, Cimaz, R, Civino, A, Consolini, R, Constantin, T, Cuttica, R, Dallos, T, Martin, N, Magni-Manzoni, S, De Cunto, C, Dolezalova, P, Ekelund, M, El Miedany, Y, Espada, G, Estmann Christensen, A, Foeldvari, I, Gallizzi, R, Ganser, G, Gerloni, V, Haas, Jp, Harel, L, Harjacek, M, Hashad, S, Herlin, T, Herrera, C, Hofer, M, Holzinger, D, Horneff, G, Huppertz, Hi, Iagăru, N, Ibanez Estrella, A, Ioseliani, M, Joos, R, Knupp Oliveira, S, Kamphuis, S, Kasapcopur, O, Katsicas, Mm, Khubchandani, R, Kondi, A, Kröger, L, La Torre, F, Laday, M, Lahdenne, P, Maggio, Mc, Magnolia, Mg, Malagon, C, Malin, M, Martino, S, Melo-Gomes, Ja, Mesa-Del-Castillo, P, Militaru, A, Minden, K, Miniaci, A, Moradinejad, Mh, Morel Ayala, Z, Nikishina, I, Norambuena, X, Nordal, Eb, Pagava, K, Panaviene, V, Pastore, S, Pieropan, S, Podda, Ra, Pruunsild, C, Putto-Laurila, A, Quartier, P, Remesal, A, Rigante, Donato, Ringold, S, Rutkowska-Sak, L, Rygg, M, Saurenmann, Rk, Sawhney, S, Scott, C, Shiari, R, Smolewska, E, Sozeri, B, Swart, Jf, Sztajnbok, F, Torcoletti, M, Tsitsami, E, Tzaribachev, N, Unsal, E, Uziel, Y, Vähäsalo, P, Varbanova, B, Vargova, V, Vesely, R, Vijatov-Djuric, G, Vilaiyuk, S, Vojinovic, J, Vougiouka, O, Weiss, P, Wouters, C, and Rigante D (ORCID:0000-0001-7032-7779)
Abstract: BACKGROUND: To our knowledge, the characteristics and burden of childhood arthritis have never been studied on a worldwide basis. We aimed to investigate, with a cross-sectional study, the prevalence of disease categories, treatment methods, and disease status in patients from across different geographical areas and from countries with diverse wealth status. METHODS: In this multinational, cross-sectional, observational cohort study, we asked international paediatric rheumatologists from specialised centres to enrol children with a diagnosis of juvenile idiopathic arthritis, according to International League of Associations for Rheumatology criteria, who were seen consecutively for a period of 6 months. Each patient underwent retrospective and cross-sectional assessments, including measures of disease activity and damage and questionnaires on the wellbeing and quality of life of the children. We qualitatively compared the collected data across eight geographical areas, and we explored an association between disease activity and damage and a country's gross domestic product (GDP) with a multiple logistic regression analysis. FINDINGS: Between April 4, 2011, and Nov 21, 2016, 9081 patients were enrolled at 130 centres in 49 countries, grouped into eight geographical areas. Systemic arthritis (125 [33·0%] of 379 patients) and enthesitis-related arthritis (113 [29·8%] of 379) were more common in southeast Asia, whereas oligoarthritis was more prevalent in southern Europe (1360 [56·7%] of 2400) and rheumatoid factor-negative polyarthritis was more frequent in North America (165 [31·5%] of 523) than in the other areas. Prevalence of uveitis was highest in northern Europe (161 [19·1%] of 845 patients) and southern Europe (450 [18·8%] of 2400) and lowest in Latin America (54 [6·4%] of 849), Africa and Middle East (71 [5·9%] of 1209), and southeast Asia (19 [5·0%] of 379). Median age at disease onset was lower in southern Europe (3·5 years, IQR 1·9-7·3) than in other regions
Published: 2019

18. Dutch juvenile idiopathic arthritis patients, carers and clinicians create a research agenda together following the James Lind Alliance method: a study protocol

Author: Schoemaker, C.G., Armbrust, W., Swart, J.F., Vastert, S.J., Loosdregt, J. van, Verwoerd, A., Whiting, C., Cowan, K., Olsder, W., Versluis, E., Vliet, R., Fernhout, M.J., Bookelman, S.L., Cappon, J., Berg, J.M. ten, Schatorje, E.J., Muller, P., Kamphuis, S., Boer, J, Lelieveld, O., Net, J. van der, Jongsma, K.R., Rensen, A., Dedding, C., Wulffraat, N.M., Schoemaker, C.G., Armbrust, W., Swart, J.F., Vastert, S.J., Loosdregt, J. van, Verwoerd, A., Whiting, C., Cowan, K., Olsder, W., Versluis, E., Vliet, R., Fernhout, M.J., Bookelman, S.L., Cappon, J., Berg, J.M. ten, Schatorje, E.J., Muller, P., Kamphuis, S., Boer, J, Lelieveld, O., Net, J. van der, Jongsma, K.R., Rensen, A., Dedding, C., and Wulffraat, N.M.
Abstract: Contains fulltext : 200061.pdf (publisher's version ) (Open Access), BACKGROUND: Research on Juvenile Idiopathic Arthritis (JIA) should support patients, caregivers/parents (carers) and clinicians to make important decisions in the consulting room and eventually to improve the lives of patients with JIA. Thus far these end-users of JIA-research have rarely been involved in the prioritisation of future research. MAIN BODY: Dutch organisations of patients, carers and clinicians will collaboratively develop a research agenda for JIA, following the James Lind Alliance (JLA) methodology. In a 'Priority Setting Partnership' (PSP), they will gradually establish a top 10 list of the most important unanswered research questions for JIA. In this process the input from clinicians, patients and their carers will be equally valued. Additionally, focus groups will be organised to involve young people with JIA. The involvement of all contributors will be monitored and evaluated. In this manner, the project will contribute to the growing body of literature on how to involve young people in agenda setting in a meaningful way. CONCLUSION: A JIA research agenda established through the JLA method and thus co-created by patients, carers and clinicians will inform researchers and research funders about the most important research questions for JIA. This will lead to research that really matters.
Published: 2018

19. Dutch juvenile idiopathic arthritis patients, carers and clinicians create a research agenda together following the James Lind Alliance method: a study protocol

Author: Schoemaker, C.G., Armbrust, W., Swart, J.F., Vastert, S.J., Loosdregt, J. van, Verwoerd, A., Whiting, C., Cowan, K., Olsder, W., Versluis, E., Vliet, R., Fernhout, M.J., Bookelman, S.L., Cappon, J., Berg, J.M. ten, Schatorje, E.J., Muller, P., Kamphuis, S., Boer, J, Lelieveld, O., Net, J. van der, Jongsma, K.R., Rensen, A., Dedding, C., Wulffraat, N.M., Schoemaker, C.G., Armbrust, W., Swart, J.F., Vastert, S.J., Loosdregt, J. van, Verwoerd, A., Whiting, C., Cowan, K., Olsder, W., Versluis, E., Vliet, R., Fernhout, M.J., Bookelman, S.L., Cappon, J., Berg, J.M. ten, Schatorje, E.J., Muller, P., Kamphuis, S., Boer, J, Lelieveld, O., Net, J. van der, Jongsma, K.R., Rensen, A., Dedding, C., and Wulffraat, N.M.
Abstract: Contains fulltext : 200061.pdf (publisher's version ) (Open Access), BACKGROUND: Research on Juvenile Idiopathic Arthritis (JIA) should support patients, caregivers/parents (carers) and clinicians to make important decisions in the consulting room and eventually to improve the lives of patients with JIA. Thus far these end-users of JIA-research have rarely been involved in the prioritisation of future research. MAIN BODY: Dutch organisations of patients, carers and clinicians will collaboratively develop a research agenda for JIA, following the James Lind Alliance (JLA) methodology. In a 'Priority Setting Partnership' (PSP), they will gradually establish a top 10 list of the most important unanswered research questions for JIA. In this process the input from clinicians, patients and their carers will be equally valued. Additionally, focus groups will be organised to involve young people with JIA. The involvement of all contributors will be monitored and evaluated. In this manner, the project will contribute to the growing body of literature on how to involve young people in agenda setting in a meaningful way. CONCLUSION: A JIA research agenda established through the JLA method and thus co-created by patients, carers and clinicians will inform researchers and research funders about the most important research questions for JIA. This will lead to research that really matters.
Published: 2018

20. Dutch juvenile idiopathic arthritis patients, carers and clinicians create a research agenda together following the James Lind Alliance method: a study protocol

Author: Schoemaker, C.G., Armbrust, W., Swart, J.F., Vastert, S.J., Loosdregt, J. van, Verwoerd, A., Whiting, C., Cowan, K., Olsder, W., Versluis, E., Vliet, R., Fernhout, M.J., Bookelman, S.L., Cappon, J., Berg, J.M. ten, Schatorje, E.J., Muller, P., Kamphuis, S., Boer, J, Lelieveld, O., Net, J. van der, Jongsma, K.R., Rensen, A., Dedding, C., Wulffraat, N.M., Schoemaker, C.G., Armbrust, W., Swart, J.F., Vastert, S.J., Loosdregt, J. van, Verwoerd, A., Whiting, C., Cowan, K., Olsder, W., Versluis, E., Vliet, R., Fernhout, M.J., Bookelman, S.L., Cappon, J., Berg, J.M. ten, Schatorje, E.J., Muller, P., Kamphuis, S., Boer, J, Lelieveld, O., Net, J. van der, Jongsma, K.R., Rensen, A., Dedding, C., and Wulffraat, N.M.
Abstract: Contains fulltext : 200061.pdf (publisher's version ) (Open Access), BACKGROUND: Research on Juvenile Idiopathic Arthritis (JIA) should support patients, caregivers/parents (carers) and clinicians to make important decisions in the consulting room and eventually to improve the lives of patients with JIA. Thus far these end-users of JIA-research have rarely been involved in the prioritisation of future research. MAIN BODY: Dutch organisations of patients, carers and clinicians will collaboratively develop a research agenda for JIA, following the James Lind Alliance (JLA) methodology. In a 'Priority Setting Partnership' (PSP), they will gradually establish a top 10 list of the most important unanswered research questions for JIA. In this process the input from clinicians, patients and their carers will be equally valued. Additionally, focus groups will be organised to involve young people with JIA. The involvement of all contributors will be monitored and evaluated. In this manner, the project will contribute to the growing body of literature on how to involve young people in agenda setting in a meaningful way. CONCLUSION: A JIA research agenda established through the JLA method and thus co-created by patients, carers and clinicians will inform researchers and research funders about the most important research questions for JIA. This will lead to research that really matters.
Published: 2018

21. MEFV mutations affecting pyrin amino acid 577 cause autosomal dominant autoinflammatory disease

Author: Stoffels, M., Szperl, A., Simon, A., Netea, M.G., Plantinga, T.S., Deuren, M. van, Kamphuis, S., Lachmann, H.J., Cuppen, E., Kloosterman, W.P., Frenkel, J., Diemen, C.C. van, Wijmenga, C., Gijn, M. van, Meer, J.W.M. van der, Stoffels, M., Szperl, A., Simon, A., Netea, M.G., Plantinga, T.S., Deuren, M. van, Kamphuis, S., Lachmann, H.J., Cuppen, E., Kloosterman, W.P., Frenkel, J., Diemen, C.C. van, Wijmenga, C., Gijn, M. van, and Meer, J.W.M. van der
Abstract: Item does not contain fulltext, OBJECTIVES: Autoinflammatory disorders are disorders of the innate immune system. Standard genetic testing provided no correct diagnosis in a female patient from a non-consanguineous family of British descent with a colchicine-responsive autosomal dominant periodic fever syndrome. We aimed to unravel the genetic cause of the symptoms. METHODS: Whole exome sequencing was used to screen for novel sequence variants, which were validated by direct Sanger sequencing. Ex vivo stimulation with peripheral blood mononuclear cells was performed to study the functional consequences of the mutation. mRNA and cytokine levels were measured by quantitative PCR and ELISA, respectively. RESULTS: Whole exome sequencing revealed a novel missense sequence variant, not seen in around 6800 controls, mapping to exon 8 of the MEFV gene (c.1730C>A; p.T577N), co-segregating perfectly with disease in this family. Other mutations at the same amino acid (c.1730C>G; p.T577S and c.1729A>T; p.T577S) were found in a family of Turkish descent, with autosomal dominant inheritance of familial Mediterranean fever (FMF)-like phenotype, and a Dutch patient, respectively. Moreover, a mutation (c.1729A>G; p.T577A) was detected in two Dutch siblings, who had episodes of inflammation of varying severity not resembling FMF. Peripheral blood mononuclear cells from one patient of the index family showed increased basal interleukin 1beta mRNA levels and cytokine responses after lipopolysaccharide stimulation. Responses normalised with colchicine treatment. CONCLUSIONS: Heterozygous mutations at amino acid position 577 of pyrin can induce an autosomal dominant autoinflammatory syndrome. This suggests that T577, located in front of the C-terminal B30.2/SPRY domain, is crucial for pyrin function.
Published: 2014

22. MEFV mutations affecting pyrin amino acid 577 cause autosomal dominant autoinflammatory disease

Author: Stoffels, M., Szperl, A., Simon, A., Netea, M.G., Plantinga, T.S., Deuren, M. van, Kamphuis, S., Lachmann, H.J., Cuppen, E., Kloosterman, W.P., Frenkel, J., Diemen, C.C. van, Wijmenga, C., Gijn, M. van, Meer, J.W.M. van der, Stoffels, M., Szperl, A., Simon, A., Netea, M.G., Plantinga, T.S., Deuren, M. van, Kamphuis, S., Lachmann, H.J., Cuppen, E., Kloosterman, W.P., Frenkel, J., Diemen, C.C. van, Wijmenga, C., Gijn, M. van, and Meer, J.W.M. van der
Abstract: Item does not contain fulltext, OBJECTIVES: Autoinflammatory disorders are disorders of the innate immune system. Standard genetic testing provided no correct diagnosis in a female patient from a non-consanguineous family of British descent with a colchicine-responsive autosomal dominant periodic fever syndrome. We aimed to unravel the genetic cause of the symptoms. METHODS: Whole exome sequencing was used to screen for novel sequence variants, which were validated by direct Sanger sequencing. Ex vivo stimulation with peripheral blood mononuclear cells was performed to study the functional consequences of the mutation. mRNA and cytokine levels were measured by quantitative PCR and ELISA, respectively. RESULTS: Whole exome sequencing revealed a novel missense sequence variant, not seen in around 6800 controls, mapping to exon 8 of the MEFV gene (c.1730C>A; p.T577N), co-segregating perfectly with disease in this family. Other mutations at the same amino acid (c.1730C>G; p.T577S and c.1729A>T; p.T577S) were found in a family of Turkish descent, with autosomal dominant inheritance of familial Mediterranean fever (FMF)-like phenotype, and a Dutch patient, respectively. Moreover, a mutation (c.1729A>G; p.T577A) was detected in two Dutch siblings, who had episodes of inflammation of varying severity not resembling FMF. Peripheral blood mononuclear cells from one patient of the index family showed increased basal interleukin 1beta mRNA levels and cytokine responses after lipopolysaccharide stimulation. Responses normalised with colchicine treatment. CONCLUSIONS: Heterozygous mutations at amino acid position 577 of pyrin can induce an autosomal dominant autoinflammatory syndrome. This suggests that T577, located in front of the C-terminal B30.2/SPRY domain, is crucial for pyrin function.
Published: 2014

23. MEFV mutations affecting pyrin amino acid 577 cause autosomal dominant autoinflammatory disease

Author: Stoffels, M., Szperl, A., Simon, A., Netea, M.G., Plantinga, T.S., Deuren, M. van, Kamphuis, S., Lachmann, H.J., Cuppen, E., Kloosterman, W.P., Frenkel, J., Diemen, C.C. van, Wijmenga, C., Gijn, M. van, Meer, J.W.M. van der, Stoffels, M., Szperl, A., Simon, A., Netea, M.G., Plantinga, T.S., Deuren, M. van, Kamphuis, S., Lachmann, H.J., Cuppen, E., Kloosterman, W.P., Frenkel, J., Diemen, C.C. van, Wijmenga, C., Gijn, M. van, and Meer, J.W.M. van der
Abstract: Item does not contain fulltext, OBJECTIVES: Autoinflammatory disorders are disorders of the innate immune system. Standard genetic testing provided no correct diagnosis in a female patient from a non-consanguineous family of British descent with a colchicine-responsive autosomal dominant periodic fever syndrome. We aimed to unravel the genetic cause of the symptoms. METHODS: Whole exome sequencing was used to screen for novel sequence variants, which were validated by direct Sanger sequencing. Ex vivo stimulation with peripheral blood mononuclear cells was performed to study the functional consequences of the mutation. mRNA and cytokine levels were measured by quantitative PCR and ELISA, respectively. RESULTS: Whole exome sequencing revealed a novel missense sequence variant, not seen in around 6800 controls, mapping to exon 8 of the MEFV gene (c.1730C>A; p.T577N), co-segregating perfectly with disease in this family. Other mutations at the same amino acid (c.1730C>G; p.T577S and c.1729A>T; p.T577S) were found in a family of Turkish descent, with autosomal dominant inheritance of familial Mediterranean fever (FMF)-like phenotype, and a Dutch patient, respectively. Moreover, a mutation (c.1729A>G; p.T577A) was detected in two Dutch siblings, who had episodes of inflammation of varying severity not resembling FMF. Peripheral blood mononuclear cells from one patient of the index family showed increased basal interleukin 1beta mRNA levels and cytokine responses after lipopolysaccharide stimulation. Responses normalised with colchicine treatment. CONCLUSIONS: Heterozygous mutations at amino acid position 577 of pyrin can induce an autosomal dominant autoinflammatory syndrome. This suggests that T577, located in front of the C-terminal B30.2/SPRY domain, is crucial for pyrin function.
Published: 2014

24. Treatment choices of paediatric rheumatologists for juvenile idiopathic arthritis: etanercept or adalimumab?

Author: Anink, J., Otten, M.H., Gorter, S.L., Prince, F.H., Rossum, M.A.C. van, Berg, J.M. van den, Pelt, P.A. van, Kamphuis, S., Brinkman, D.M.C., Swen, W.A., Swart, J.F., Wulffraat, N.M., Dolman, K.M., Koopman-Keemink, Y., Hoppenreijs, E.P.A.H., Armbrust, W., Cate, R. ten, Suijlekom-Smit, L.W. van, Anink, J., Otten, M.H., Gorter, S.L., Prince, F.H., Rossum, M.A.C. van, Berg, J.M. van den, Pelt, P.A. van, Kamphuis, S., Brinkman, D.M.C., Swen, W.A., Swart, J.F., Wulffraat, N.M., Dolman, K.M., Koopman-Keemink, Y., Hoppenreijs, E.P.A.H., Armbrust, W., Cate, R. ten, and Suijlekom-Smit, L.W. van
Abstract: 1 september 2013, Item does not contain fulltext, Objectives. To evaluate differences in baseline characteristics between etanercept- and adalimumab-treated JIA patients and to reveal factors that influence the choice between these TNF inhibitors, which are considered equally effective in the recent ACR recommendations for JIA treatment. Methods. Biologic-naive JIA patients with active arthritis who started treatment with adalimumab or etanercept between March 2008 and December 2011 were selected from the Dutch Arthritis and Biologicals in Children register. Baseline characteristics were compared. Focus group interviews with paediatric rheumatologists were performed to evaluate factors determining treatment choices. Results. A total of 193 patients started treatment with etanercept and 21 with adalimumab. Adalimumab-treated patients had longer disease duration prior to the start of biologics (median 5.7 vs 2.0 years) and more often a history of uveitis (71% vs 4%). Etanercept-treated patients had more disability at baseline (median Childhood Health Assessment Questionnaire score 1.1 vs 0.4) and more active arthritis (median number of active joints 6 vs 4). The presence of uveitis was the most important factor directing the choice towards adalimumab. Factors specific for the paediatric population-such as painful adalimumab injections-as well as the physician's familiarity with the drug accounted for the preference for etanercept. Conclusion. Although the two TNF inhibitors are considered equally effective, in daily practice etanercept is most often prescribed; adalimumab is mainly preferred when uveitis is present. In choosing the most suitable biologic treatment, paediatric rheumatologists take into account drug and patient factors, considering newly published data and cautiously implementing this into daily care.
Published: 2013

25. Effectiveness and safety of a second and third biological agent after failing etanercept in juvenile idiopathic arthritis: results from the Dutch National ABC Register.

Author: Otten, M.H., Prince, F.H., Anink, J., Cate, R. ten, Hoppenreijs, E.P.A.H., Armbrust, W., Koopman-Keemink, Y., Pelt, P.A. van, Kamphuis, S., Gorter, S.L., Dolman, K.M., Swart, J.F., Berg, J.M. van den, Wulffraat, N.M., Rossum, M.A.C. van, Suijlekom-Smit, L.W. van, Otten, M.H., Prince, F.H., Anink, J., Cate, R. ten, Hoppenreijs, E.P.A.H., Armbrust, W., Koopman-Keemink, Y., Pelt, P.A. van, Kamphuis, S., Gorter, S.L., Dolman, K.M., Swart, J.F., Berg, J.M. van den, Wulffraat, N.M., Rossum, M.A.C. van, and Suijlekom-Smit, L.W. van
Abstract: 1 mei 2013, Item does not contain fulltext, OBJECTIVE: To evaluate the effectiveness and safety of switching to a second or third biological agent in juvenile idiopathic arthritis (JIA) after etanercept failure. METHODS: The Arthritis and Biologicals in Children Register aims to include all Dutch JIA patients who have used biological agents. Data on the disease course were used to estimate drug survival with Kaplan-Meier and calculate adverse event (AE) rates. RESULTS: Of 307 biologically naive JIA patients who started etanercept, 80 (26%) switched to a second and 22 (7%) to a third biological agent. During 1030 patient-years of follow-up after the introduction of etanercept, 49 switches to adalimumab, 28 infliximab, 17 anakinra, four abatacept and four trial drugs were evaluated. 84% (95% CI 80% to 88%) of patients who started etanercept as a first biological agent were, after 12 months, still on the drug, compared with 47% (95% CI 35% to 60%) who started a second and 51% (95% CI 26% to 76%) who started a third biological agent. Patients who switched because of primary ineffectiveness continued the second agent less often (32%, 95% CI 12% to 53%). After etanercept failure, drug continuation of adalimumab was similar to infliximab for patients with non-systemic JIA; anakinra was superior to a second TNF-blocker for systemic JIA. AE rates within first 12 months after initiation were comparable for each course and each biological agent. CONCLUSIONS: Switching to another biological agent is common, especially for systemic JIA patients. A second (and third) agent was less effective than the first. The choice of second biological agent by the physician mainly depends on availability and JIA category.
Published: 2013

26. Tumour necrosis factor-blocking agents in persistent oligoarticular juvenile idiopathic arthritis: results from the Dutch Arthritis and Biologicals in Children Register.

Author: Anink, J., Otten, M.H., Prince, F.H., Hoppenreijs, E.P.A.H., Wulffraat, N.M., Swart, J.F., Cate, R. ten, Rossum, M.A.C. van, Berg, J.M. van den, Dolman, K.M., Koopman-Keemink, Y., Armbrust, W., Kamphuis, S., Pelt, P.A. van, Gorter, S.L., Suijlekom-Smit, L.W. van, Anink, J., Otten, M.H., Prince, F.H., Hoppenreijs, E.P.A.H., Wulffraat, N.M., Swart, J.F., Cate, R. ten, Rossum, M.A.C. van, Berg, J.M. van den, Dolman, K.M., Koopman-Keemink, Y., Armbrust, W., Kamphuis, S., Pelt, P.A. van, Gorter, S.L., and Suijlekom-Smit, L.W. van
Abstract: 1 april 2013, Item does not contain fulltext, Objective. Because TNF inhibitors are not approved for persistent oligoarticular JIA (oJIA), although they are used off-label, we evaluated their effectiveness in patients in this category. Methods. Persistent oJIA patients were selected from the Dutch Arthritis and Biologicals in Children (ABC) register, an ongoing multicentre prospective study that aims to include all Dutch children with JIA using biologic agents. Response was assessed by the JIA core-set disease activity variables and modified Wallace criteria for inactive disease. Results. Until February 2011, 16 persistent oJIA patients (68.8% females) had been included in the register. Median age of onset was 8.4 years [interquartile range (IQR) 2.1-13.5 years]; history of uveitis in 18.8%; ANA-positive 56.3%. All had previously used MTX, and 81.3% had used IA CSs. Median follow-up after the introduction of biologic treatment was 13.7 months (IQR 8.3-16.7 months). Fourteen patients started etanercept and two patients who had active arthritis as well as uveitis started adalimumab. Although patients with persistent oJIA had few affected joints [median of two active joints at the start of biologic (IQR 1-3)], the patient/parent assessments of pain [median visual analogue score (VAS) 51 (IQR 1-64)] and well-being [median VAS 44 (IQR 6-66)] were high. Additionally, their physician evaluated the disease activity as moderately high [median VAS 36 (IQR 4-65)]. After 3 months this decreased to 0 (IQR 0-30) and 63% achieved inactive disease. After 15 months the disease was inactive in 9/10 observed patients. TNF inhibitors were tolerated well. Conclusion. TNF blocking agents seem an effective and justifiable option in persistent oJIA when treatment with IA CS injections and MTX has failed.
Published: 2013

27. Treatment choices of paediatric rheumatologists for juvenile idiopathic arthritis: etanercept or adalimumab?

Author: Anink, J., Otten, M.H., Gorter, S.L., Prince, F.H., Rossum, M.A.C. van, Berg, J.M. van den, Pelt, P.A. van, Kamphuis, S., Brinkman, D.M.C., Swen, W.A., Swart, J.F., Wulffraat, N.M., Dolman, K.M., Koopman-Keemink, Y., Hoppenreijs, E.P.A.H., Armbrust, W., Cate, R. ten, Suijlekom-Smit, L.W. van, Anink, J., Otten, M.H., Gorter, S.L., Prince, F.H., Rossum, M.A.C. van, Berg, J.M. van den, Pelt, P.A. van, Kamphuis, S., Brinkman, D.M.C., Swen, W.A., Swart, J.F., Wulffraat, N.M., Dolman, K.M., Koopman-Keemink, Y., Hoppenreijs, E.P.A.H., Armbrust, W., Cate, R. ten, and Suijlekom-Smit, L.W. van
Abstract: 01 september 2013, Item does not contain fulltext, Objectives. To evaluate differences in baseline characteristics between etanercept- and adalimumab-treated JIA patients and to reveal factors that influence the choice between these TNF inhibitors, which are considered equally effective in the recent ACR recommendations for JIA treatment. Methods. Biologic-naive JIA patients with active arthritis who started treatment with adalimumab or etanercept between March 2008 and December 2011 were selected from the Dutch Arthritis and Biologicals in Children register. Baseline characteristics were compared. Focus group interviews with paediatric rheumatologists were performed to evaluate factors determining treatment choices. Results. A total of 193 patients started treatment with etanercept and 21 with adalimumab. Adalimumab-treated patients had longer disease duration prior to the start of biologics (median 5.7 vs 2.0 years) and more often a history of uveitis (71% vs 4%). Etanercept-treated patients had more disability at baseline (median Childhood Health Assessment Questionnaire score 1.1 vs 0.4) and more active arthritis (median number of active joints 6 vs 4). The presence of uveitis was the most important factor directing the choice towards adalimumab. Factors specific for the paediatric population-such as painful adalimumab injections-as well as the physician's familiarity with the drug accounted for the preference for etanercept. Conclusion. Although the two TNF inhibitors are considered equally effective, in daily practice etanercept is most often prescribed; adalimumab is mainly preferred when uveitis is present. In choosing the most suitable biologic treatment, paediatric rheumatologists take into account drug and patient factors, considering newly published data and cautiously implementing this into daily care.
Published: 2013

28. Effectiveness and safety of a second and third biological agent after failing etanercept in juvenile idiopathic arthritis: results from the Dutch National ABC Register.

Author: Otten, M.H., Prince, F.H., Anink, J., Cate, R. ten, Hoppenreijs, E.P.A.H., Armbrust, W., Koopman-Keemink, Y., Pelt, P.A. van, Kamphuis, S., Gorter, S.L., Dolman, K.M., Swart, J.F., Berg, J.M. van den, Wulffraat, N.M., Rossum, M.A.C. van, Suijlekom-Smit, L.W. van, Otten, M.H., Prince, F.H., Anink, J., Cate, R. ten, Hoppenreijs, E.P.A.H., Armbrust, W., Koopman-Keemink, Y., Pelt, P.A. van, Kamphuis, S., Gorter, S.L., Dolman, K.M., Swart, J.F., Berg, J.M. van den, Wulffraat, N.M., Rossum, M.A.C. van, and Suijlekom-Smit, L.W. van
Abstract: 01 mei 2013, Item does not contain fulltext, OBJECTIVE: To evaluate the effectiveness and safety of switching to a second or third biological agent in juvenile idiopathic arthritis (JIA) after etanercept failure. METHODS: The Arthritis and Biologicals in Children Register aims to include all Dutch JIA patients who have used biological agents. Data on the disease course were used to estimate drug survival with Kaplan-Meier and calculate adverse event (AE) rates. RESULTS: Of 307 biologically naive JIA patients who started etanercept, 80 (26%) switched to a second and 22 (7%) to a third biological agent. During 1030 patient-years of follow-up after the introduction of etanercept, 49 switches to adalimumab, 28 infliximab, 17 anakinra, four abatacept and four trial drugs were evaluated. 84% (95% CI 80% to 88%) of patients who started etanercept as a first biological agent were, after 12 months, still on the drug, compared with 47% (95% CI 35% to 60%) who started a second and 51% (95% CI 26% to 76%) who started a third biological agent. Patients who switched because of primary ineffectiveness continued the second agent less often (32%, 95% CI 12% to 53%). After etanercept failure, drug continuation of adalimumab was similar to infliximab for patients with non-systemic JIA; anakinra was superior to a second TNF-blocker for systemic JIA. AE rates within first 12 months after initiation were comparable for each course and each biological agent. CONCLUSIONS: Switching to another biological agent is common, especially for systemic JIA patients. A second (and third) agent was less effective than the first. The choice of second biological agent by the physician mainly depends on availability and JIA category.
Published: 2013

29. Tumour necrosis factor-blocking agents in persistent oligoarticular juvenile idiopathic arthritis: results from the Dutch Arthritis and Biologicals in Children Register.

Author: Anink, J., Otten, M.H., Prince, F.H., Hoppenreijs, E.P.A.H., Wulffraat, N.M., Swart, J.F., Cate, R. ten, Rossum, M.A.C. van, Berg, J.M. van den, Dolman, K.M., Koopman-Keemink, Y., Armbrust, W., Kamphuis, S., Pelt, P.A. van, Gorter, S.L., Suijlekom-Smit, L.W. van, Anink, J., Otten, M.H., Prince, F.H., Hoppenreijs, E.P.A.H., Wulffraat, N.M., Swart, J.F., Cate, R. ten, Rossum, M.A.C. van, Berg, J.M. van den, Dolman, K.M., Koopman-Keemink, Y., Armbrust, W., Kamphuis, S., Pelt, P.A. van, Gorter, S.L., and Suijlekom-Smit, L.W. van
Abstract: 01 april 2013, Item does not contain fulltext, Objective. Because TNF inhibitors are not approved for persistent oligoarticular JIA (oJIA), although they are used off-label, we evaluated their effectiveness in patients in this category. Methods. Persistent oJIA patients were selected from the Dutch Arthritis and Biologicals in Children (ABC) register, an ongoing multicentre prospective study that aims to include all Dutch children with JIA using biologic agents. Response was assessed by the JIA core-set disease activity variables and modified Wallace criteria for inactive disease. Results. Until February 2011, 16 persistent oJIA patients (68.8% females) had been included in the register. Median age of onset was 8.4 years [interquartile range (IQR) 2.1-13.5 years]; history of uveitis in 18.8%; ANA-positive 56.3%. All had previously used MTX, and 81.3% had used IA CSs. Median follow-up after the introduction of biologic treatment was 13.7 months (IQR 8.3-16.7 months). Fourteen patients started etanercept and two patients who had active arthritis as well as uveitis started adalimumab. Although patients with persistent oJIA had few affected joints [median of two active joints at the start of biologic (IQR 1-3)], the patient/parent assessments of pain [median visual analogue score (VAS) 51 (IQR 1-64)] and well-being [median VAS 44 (IQR 6-66)] were high. Additionally, their physician evaluated the disease activity as moderately high [median VAS 36 (IQR 4-65)]. After 3 months this decreased to 0 (IQR 0-30) and 63% achieved inactive disease. After 15 months the disease was inactive in 9/10 observed patients. TNF inhibitors were tolerated well. Conclusion. TNF blocking agents seem an effective and justifiable option in persistent oJIA when treatment with IA CS injections and MTX has failed.
Published: 2013

30. Tumour necrosis factor-blocking agents in persistent oligoarticular juvenile idiopathic arthritis: results from the Dutch Arthritis and Biologicals in Children Register.

Author: Anink, J., Otten, M.H., Prince, F.H., Hoppenreijs, E.P.A.H., Wulffraat, N.M., Swart, J.F., Cate, R. ten, Rossum, M.A.C. van, Berg, J.M. van den, Dolman, K.M., Koopman-Keemink, Y., Armbrust, W., Kamphuis, S., Pelt, P.A. van, Gorter, S.L., Suijlekom-Smit, L.W. van, Anink, J., Otten, M.H., Prince, F.H., Hoppenreijs, E.P.A.H., Wulffraat, N.M., Swart, J.F., Cate, R. ten, Rossum, M.A.C. van, Berg, J.M. van den, Dolman, K.M., Koopman-Keemink, Y., Armbrust, W., Kamphuis, S., Pelt, P.A. van, Gorter, S.L., and Suijlekom-Smit, L.W. van
Abstract: 01 april 2013, Item does not contain fulltext, Objective. Because TNF inhibitors are not approved for persistent oligoarticular JIA (oJIA), although they are used off-label, we evaluated their effectiveness in patients in this category. Methods. Persistent oJIA patients were selected from the Dutch Arthritis and Biologicals in Children (ABC) register, an ongoing multicentre prospective study that aims to include all Dutch children with JIA using biologic agents. Response was assessed by the JIA core-set disease activity variables and modified Wallace criteria for inactive disease. Results. Until February 2011, 16 persistent oJIA patients (68.8% females) had been included in the register. Median age of onset was 8.4 years [interquartile range (IQR) 2.1-13.5 years]; history of uveitis in 18.8%; ANA-positive 56.3%. All had previously used MTX, and 81.3% had used IA CSs. Median follow-up after the introduction of biologic treatment was 13.7 months (IQR 8.3-16.7 months). Fourteen patients started etanercept and two patients who had active arthritis as well as uveitis started adalimumab. Although patients with persistent oJIA had few affected joints [median of two active joints at the start of biologic (IQR 1-3)], the patient/parent assessments of pain [median visual analogue score (VAS) 51 (IQR 1-64)] and well-being [median VAS 44 (IQR 6-66)] were high. Additionally, their physician evaluated the disease activity as moderately high [median VAS 36 (IQR 4-65)]. After 3 months this decreased to 0 (IQR 0-30) and 63% achieved inactive disease. After 15 months the disease was inactive in 9/10 observed patients. TNF inhibitors were tolerated well. Conclusion. TNF blocking agents seem an effective and justifiable option in persistent oJIA when treatment with IA CS injections and MTX has failed.
Published: 2013

31. Treatment choices of paediatric rheumatologists for juvenile idiopathic arthritis: etanercept or adalimumab?

Author: Anink, J., Otten, M.H., Gorter, S.L., Prince, F.H., Rossum, M.A.C. van, Berg, J.M. van den, Pelt, P.A. van, Kamphuis, S., Brinkman, D.M.C., Swen, W.A., Swart, J.F., Wulffraat, N.M., Dolman, K.M., Koopman-Keemink, Y., Hoppenreijs, E.P.A.H., Armbrust, W., Cate, R. ten, Suijlekom-Smit, L.W. van, Anink, J., Otten, M.H., Gorter, S.L., Prince, F.H., Rossum, M.A.C. van, Berg, J.M. van den, Pelt, P.A. van, Kamphuis, S., Brinkman, D.M.C., Swen, W.A., Swart, J.F., Wulffraat, N.M., Dolman, K.M., Koopman-Keemink, Y., Hoppenreijs, E.P.A.H., Armbrust, W., Cate, R. ten, and Suijlekom-Smit, L.W. van
Abstract: 01 september 2013, Item does not contain fulltext, Objectives. To evaluate differences in baseline characteristics between etanercept- and adalimumab-treated JIA patients and to reveal factors that influence the choice between these TNF inhibitors, which are considered equally effective in the recent ACR recommendations for JIA treatment. Methods. Biologic-naive JIA patients with active arthritis who started treatment with adalimumab or etanercept between March 2008 and December 2011 were selected from the Dutch Arthritis and Biologicals in Children register. Baseline characteristics were compared. Focus group interviews with paediatric rheumatologists were performed to evaluate factors determining treatment choices. Results. A total of 193 patients started treatment with etanercept and 21 with adalimumab. Adalimumab-treated patients had longer disease duration prior to the start of biologics (median 5.7 vs 2.0 years) and more often a history of uveitis (71% vs 4%). Etanercept-treated patients had more disability at baseline (median Childhood Health Assessment Questionnaire score 1.1 vs 0.4) and more active arthritis (median number of active joints 6 vs 4). The presence of uveitis was the most important factor directing the choice towards adalimumab. Factors specific for the paediatric population-such as painful adalimumab injections-as well as the physician's familiarity with the drug accounted for the preference for etanercept. Conclusion. Although the two TNF inhibitors are considered equally effective, in daily practice etanercept is most often prescribed; adalimumab is mainly preferred when uveitis is present. In choosing the most suitable biologic treatment, paediatric rheumatologists take into account drug and patient factors, considering newly published data and cautiously implementing this into daily care.
Published: 2013

32. Effectiveness and safety of a second and third biological agent after failing etanercept in juvenile idiopathic arthritis: results from the Dutch National ABC Register.

Author: Otten, M.H., Prince, F.H., Anink, J., Cate, R. ten, Hoppenreijs, E.P.A.H., Armbrust, W., Koopman-Keemink, Y., Pelt, P.A. van, Kamphuis, S., Gorter, S.L., Dolman, K.M., Swart, J.F., Berg, J.M. van den, Wulffraat, N.M., Rossum, M.A.C. van, Suijlekom-Smit, L.W. van, Otten, M.H., Prince, F.H., Anink, J., Cate, R. ten, Hoppenreijs, E.P.A.H., Armbrust, W., Koopman-Keemink, Y., Pelt, P.A. van, Kamphuis, S., Gorter, S.L., Dolman, K.M., Swart, J.F., Berg, J.M. van den, Wulffraat, N.M., Rossum, M.A.C. van, and Suijlekom-Smit, L.W. van
Abstract: 01 mei 2013, Item does not contain fulltext, OBJECTIVE: To evaluate the effectiveness and safety of switching to a second or third biological agent in juvenile idiopathic arthritis (JIA) after etanercept failure. METHODS: The Arthritis and Biologicals in Children Register aims to include all Dutch JIA patients who have used biological agents. Data on the disease course were used to estimate drug survival with Kaplan-Meier and calculate adverse event (AE) rates. RESULTS: Of 307 biologically naive JIA patients who started etanercept, 80 (26%) switched to a second and 22 (7%) to a third biological agent. During 1030 patient-years of follow-up after the introduction of etanercept, 49 switches to adalimumab, 28 infliximab, 17 anakinra, four abatacept and four trial drugs were evaluated. 84% (95% CI 80% to 88%) of patients who started etanercept as a first biological agent were, after 12 months, still on the drug, compared with 47% (95% CI 35% to 60%) who started a second and 51% (95% CI 26% to 76%) who started a third biological agent. Patients who switched because of primary ineffectiveness continued the second agent less often (32%, 95% CI 12% to 53%). After etanercept failure, drug continuation of adalimumab was similar to infliximab for patients with non-systemic JIA; anakinra was superior to a second TNF-blocker for systemic JIA. AE rates within first 12 months after initiation were comparable for each course and each biological agent. CONCLUSIONS: Switching to another biological agent is common, especially for systemic JIA patients. A second (and third) agent was less effective than the first. The choice of second biological agent by the physician mainly depends on availability and JIA category.
Published: 2013

33. High prevalence of methotrexate intolerance in juvenile idiopathic arthritis: development and validation of a methotrexate intolerance severity score

Author: Bulatovic, M., Heijstek, M.W., Verkaaik, M., Dijkhuizen, E.H. van, Armbrust, W., Hoppenreijs, E.P.A.H., Kamphuis, S., Kuis, W., Egberts, T.C., Sinnema, G., Rademaker, C.M.A., Wulffraat, N.M., Bulatovic, M., Heijstek, M.W., Verkaaik, M., Dijkhuizen, E.H. van, Armbrust, W., Hoppenreijs, E.P.A.H., Kamphuis, S., Kuis, W., Egberts, T.C., Sinnema, G., Rademaker, C.M.A., and Wulffraat, N.M.
Abstract: Item does not contain fulltext, OBJECTIVE: To design and validate a new questionnaire for identifying patients with methotrexate (MTX) intolerance, and to determine the prevalence of MTX intolerance in patients with juvenile idiopathic arthritis (JIA) using this questionnaire. METHODS: The MTX Intolerance Severity Score (MISS) questionnaire was constructed, consisting of 5 domains: stomach ache, nausea, vomiting, sore mouth, and behavioral symptoms. The domains each consisted of 3 questions pertaining to the presence of a symptom upon, prior to (anticipatory), and when thinking of (associative) MTX intake. The MISS questionnaire was validated in 86 patients by determining its discriminative power between patients with and those without MTX intolerance, identified as such by a gold standard (physician's opinion). Using the MISS questionnaire, the prevalence of MTX intolerance was determined in 297 JIA patients. RESULTS: The MISS questionnaire discriminated well between MTX-intolerant and MTX-tolerant patients. A cutoff score of 6 yielded the best sensitivity (88%) and specificity (80%). MTX intolerance was found in 150 (50.5%) of 297 patients. Of 220 patients receiving oral MTX, 98 (44.5%) experienced MTX intolerance, whereas 67.5% of 77 patients receiving parenteral MTX experienced intolerance to the drug (P = 0.001). CONCLUSION: Our findings indicate that the MISS questionnaire is a highly sensitive and specific tool for the diagnosis of MTX intolerance, and that there is a high prevalence of MTX intolerance among JIA patients. The prevalence of intolerance in patients receiving parenteral MTX exceeds that in patients receiving oral MTX. The frequent occurrence of anticipatory and associative symptoms suggests that classic conditioning plays an important role in MTX intolerance.
Published: 2011

34. High prevalence of methotrexate intolerance in juvenile idiopathic arthritis: development and validation of a methotrexate intolerance severity score

Author: Bulatovic, M., Heijstek, M.W., Verkaaik, M., Dijkhuizen, E.H. van, Armbrust, W., Hoppenreijs, E.P.A.H., Kamphuis, S., Kuis, W., Egberts, T.C., Sinnema, G., Rademaker, C.M.A., Wulffraat, N.M., Bulatovic, M., Heijstek, M.W., Verkaaik, M., Dijkhuizen, E.H. van, Armbrust, W., Hoppenreijs, E.P.A.H., Kamphuis, S., Kuis, W., Egberts, T.C., Sinnema, G., Rademaker, C.M.A., and Wulffraat, N.M.
Abstract: Item does not contain fulltext, OBJECTIVE: To design and validate a new questionnaire for identifying patients with methotrexate (MTX) intolerance, and to determine the prevalence of MTX intolerance in patients with juvenile idiopathic arthritis (JIA) using this questionnaire. METHODS: The MTX Intolerance Severity Score (MISS) questionnaire was constructed, consisting of 5 domains: stomach ache, nausea, vomiting, sore mouth, and behavioral symptoms. The domains each consisted of 3 questions pertaining to the presence of a symptom upon, prior to (anticipatory), and when thinking of (associative) MTX intake. The MISS questionnaire was validated in 86 patients by determining its discriminative power between patients with and those without MTX intolerance, identified as such by a gold standard (physician's opinion). Using the MISS questionnaire, the prevalence of MTX intolerance was determined in 297 JIA patients. RESULTS: The MISS questionnaire discriminated well between MTX-intolerant and MTX-tolerant patients. A cutoff score of 6 yielded the best sensitivity (88%) and specificity (80%). MTX intolerance was found in 150 (50.5%) of 297 patients. Of 220 patients receiving oral MTX, 98 (44.5%) experienced MTX intolerance, whereas 67.5% of 77 patients receiving parenteral MTX experienced intolerance to the drug (P = 0.001). CONCLUSION: Our findings indicate that the MISS questionnaire is a highly sensitive and specific tool for the diagnosis of MTX intolerance, and that there is a high prevalence of MTX intolerance among JIA patients. The prevalence of intolerance in patients receiving parenteral MTX exceeds that in patients receiving oral MTX. The frequent occurrence of anticipatory and associative symptoms suggests that classic conditioning plays an important role in MTX intolerance.
Published: 2011

35. High prevalence of methotrexate intolerance in juvenile idiopathic arthritis: development and validation of a methotrexate intolerance severity score

Author: Bulatovic, M., Heijstek, M.W., Verkaaik, M., Dijkhuizen, E.H. van, Armbrust, W., Hoppenreijs, E.P.A.H., Kamphuis, S., Kuis, W., Egberts, T.C., Sinnema, G., Rademaker, C.M.A., Wulffraat, N.M., Bulatovic, M., Heijstek, M.W., Verkaaik, M., Dijkhuizen, E.H. van, Armbrust, W., Hoppenreijs, E.P.A.H., Kamphuis, S., Kuis, W., Egberts, T.C., Sinnema, G., Rademaker, C.M.A., and Wulffraat, N.M.
Abstract: Item does not contain fulltext, OBJECTIVE: To design and validate a new questionnaire for identifying patients with methotrexate (MTX) intolerance, and to determine the prevalence of MTX intolerance in patients with juvenile idiopathic arthritis (JIA) using this questionnaire. METHODS: The MTX Intolerance Severity Score (MISS) questionnaire was constructed, consisting of 5 domains: stomach ache, nausea, vomiting, sore mouth, and behavioral symptoms. The domains each consisted of 3 questions pertaining to the presence of a symptom upon, prior to (anticipatory), and when thinking of (associative) MTX intake. The MISS questionnaire was validated in 86 patients by determining its discriminative power between patients with and those without MTX intolerance, identified as such by a gold standard (physician's opinion). Using the MISS questionnaire, the prevalence of MTX intolerance was determined in 297 JIA patients. RESULTS: The MISS questionnaire discriminated well between MTX-intolerant and MTX-tolerant patients. A cutoff score of 6 yielded the best sensitivity (88%) and specificity (80%). MTX intolerance was found in 150 (50.5%) of 297 patients. Of 220 patients receiving oral MTX, 98 (44.5%) experienced MTX intolerance, whereas 67.5% of 77 patients receiving parenteral MTX experienced intolerance to the drug (P = 0.001). CONCLUSION: Our findings indicate that the MISS questionnaire is a highly sensitive and specific tool for the diagnosis of MTX intolerance, and that there is a high prevalence of MTX intolerance among JIA patients. The prevalence of intolerance in patients receiving parenteral MTX exceeds that in patients receiving oral MTX. The frequent occurrence of anticipatory and associative symptoms suggests that classic conditioning plays an important role in MTX intolerance.
Published: 2011

36. Tuberculosis in a nine-year-old girl treated with infliximab for systemic juvenile idiopathic arthritis.

Author: Armbrust, W., Kamphuis, S., Wolfs, T.W., Fiselier, T.J.W., Nikkels, P.G.J., Kuis, W., Wulffraat, N.M., Armbrust, W., Kamphuis, S., Wolfs, T.W., Fiselier, T.J.W., Nikkels, P.G.J., Kuis, W., and Wulffraat, N.M.
Abstract: Contains fulltext : 59183.pdf (publisher's version ) (Closed access)
Published: 2004

37. Tuberculosis in a nine-year-old girl treated with infliximab for systemic juvenile idiopathic arthritis.

Author: Armbrust, W., Kamphuis, S., Wolfs, T.W., Fiselier, T.J.W., Nikkels, P.G.J., Kuis, W., Wulffraat, N.M., Armbrust, W., Kamphuis, S., Wolfs, T.W., Fiselier, T.J.W., Nikkels, P.G.J., Kuis, W., and Wulffraat, N.M.
Abstract: Contains fulltext : 59183.pdf (publisher's version ) (Closed access)
Published: 2004

38. Tuberculosis in a nine-year-old girl treated with infliximab for systemic juvenile idiopathic arthritis.

Author: Armbrust, W., Kamphuis, S., Wolfs, T.W., Fiselier, T.J.W., Nikkels, P.G.J., Kuis, W., Wulffraat, N.M., Armbrust, W., Kamphuis, S., Wolfs, T.W., Fiselier, T.J.W., Nikkels, P.G.J., Kuis, W., and Wulffraat, N.M.
Abstract: Contains fulltext : 59183.pdf (publisher's version ) (Closed access)
Published: 2004

39. Cerebrospinal fluid levels of amino acids in infants and young children with chronic renal failure

Author: Gerrits, G.P.J.M., Kamphuis, S., Monnens, L.A.H., Trijbels, J.M.F., Schröder, C.H., Koster, A.M., Gabreëls, F.J.M., Gerrits, G.P.J.M., Kamphuis, S., Monnens, L.A.H., Trijbels, J.M.F., Schröder, C.H., Koster, A.M., and Gabreëls, F.J.M.
Abstract: Item does not contain fulltext
Published: 1998

40. Cerebrospinal fluid levels of amino acids in infants and young children with chronic renal failure

Author: Gerrits, G.P.J.M., Kamphuis, S., Monnens, L.A.H., Trijbels, J.M.F., Schröder, C.H., Koster, A.M., Gabreëls, F.J.M., Gerrits, G.P.J.M., Kamphuis, S., Monnens, L.A.H., Trijbels, J.M.F., Schröder, C.H., Koster, A.M., and Gabreëls, F.J.M.
Abstract: Item does not contain fulltext
Published: 1998

41. Cerebrospinal fluid levels of amino acids in infants and young children with chronic renal failure

Author: Gerrits, G.P.J.M., Kamphuis, S., Monnens, L.A.H., Trijbels, J.M.F., Schröder, C.H., Koster, A.M., Gabreëls, F.J.M., Gerrits, G.P.J.M., Kamphuis, S., Monnens, L.A.H., Trijbels, J.M.F., Schröder, C.H., Koster, A.M., and Gabreëls, F.J.M.
Abstract: Item does not contain fulltext
Published: 1998

42. Developmentally regulated expression of two novel platelet-derived growth factor alpha-receptor transcript in human teratocarcinoma cells

Author: Mosselman, S., Claesson-Welsh, L., Kamphuis, S., Zoelen, E.J.J. van, Mosselman, S., Claesson-Welsh, L., Kamphuis, S., and Zoelen, E.J.J. van
Abstract: Contains fulltext : 250022.pdf (Publisher’s version ) (Open Access)
Published: 1994

43. Developmentally regulated expression of two novel platelet-derived growth factor alpha-receptor transcript in human teratocarcinoma cells

Author: Mosselman, S., Claesson-Welsh, L., Kamphuis, S., Zoelen, E.J.J. van, Mosselman, S., Claesson-Welsh, L., Kamphuis, S., and Zoelen, E.J.J. van
Abstract: Contains fulltext : 250022.pdf (Publisher’s version ) (Open Access)
Published: 1994

44. Developmentally regulated expression of two novel platelet-derived growth factor alpha-receptor transcript in human teratocarcinoma cells

Author: Mosselman, S., Claesson-Welsh, L., Kamphuis, S., Zoelen, E.J.J. van, Mosselman, S., Claesson-Welsh, L., Kamphuis, S., and Zoelen, E.J.J. van
Abstract: Contains fulltext : 250022.pdf (Publisher’s version ) (Open Access)
Published: 1994

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