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2. 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

3. Long-term efficacy and safety of subcutaneous tocilizumab in clinical trials of polyarticular or systemic juvenile idiopathic arthritis

Author

Brunner, H, Ruperto, N, Ramanan, A, Horneff, G, Minden, K, Calvo Penades, I, Alexeeva, E, Cleary, G, Stern, S, Kone-Paut, I, Maldonado Velazquez, M, Rabinovich, C, Remesal, A, Silva, C, Nikishina, I, Zucchetto, M, Brockwell, L, Gordon, O, Nagel, S, De Benedetti, F, Cuttica, R, Rama, M, Akikusa, J, Chaitow, J, Len, C, Schmeling, H, Schneider, R, Hufnagel, M, Benedetti, F, Velazquez, M, Rubio, N, Alekseeva, E, Boteanu, A, Torrent, R, Penades, I, Janow, G, Weiss, J, Lovell, D, Martin, A, Nanda, K, Wagner-Weiner, L, Zeft, A, Dare, J, Brunner H. I., Ruperto N., Ramanan A. V., Horneff G., Minden K., Calvo Penades I., Alexeeva E., Cleary G., Stern S. M., Kone-Paut I., Maldonado Velazquez M. D. R., Rabinovich C. E., Remesal A., Silva C. A., Nikishina I., Zucchetto M., Brockwell L., Gordon O., Nagel S., De Benedetti F., Cuttica R., Rama M. E., Akikusa J., Chaitow J., Len C., Schmeling H., Schneider R., Hufnagel M., Benedetti F. D., Velazquez M. D. R. M., Rubio N., Alekseeva E., Boteanu A., Torrent R. B., Penades I. C., Janow G., Weiss J., Lovell D., Martin A., Nanda K., Wagner-Weiner L., Stern S., Zeft A., Dare J., Brunner, H, Ruperto, N, Ramanan, A, Horneff, G, Minden, K, Calvo Penades, I, Alexeeva, E, Cleary, G, Stern, S, Kone-Paut, I, Maldonado Velazquez, M, Rabinovich, C, Remesal, A, Silva, C, Nikishina, I, Zucchetto, M, Brockwell, L, Gordon, O, Nagel, S, De Benedetti, F, Cuttica, R, Rama, M, Akikusa, J, Chaitow, J, Len, C, Schmeling, H, Schneider, R, Hufnagel, M, Benedetti, F, Velazquez, M, Rubio, N, Alekseeva, E, Boteanu, A, Torrent, R, Penades, I, Janow, G, Weiss, J, Lovell, D, Martin, A, Nanda, K, Wagner-Weiner, L, Zeft, A, Dare, J, Brunner H. I., Ruperto N., Ramanan A. V., Horneff G., Minden K., Calvo Penades I., Alexeeva E., Cleary G., Stern S. M., Kone-Paut I., Maldonado Velazquez M. D. R., Rabinovich C. E., Remesal A., Silva C. A., Nikishina I., Zucchetto M., Brockwell L., Gordon O., Nagel S., De Benedetti F., Cuttica R., Rama M. E., Akikusa J., Chaitow J., Len C., Schmeling H., Schneider R., Hufnagel M., Benedetti F. D., Velazquez M. D. R. M., Rubio N., Alekseeva E., Boteanu A., Torrent R. B., Penades I. C., Janow G., Weiss J., Lovell D., Martin A., Nanda K., Wagner-Weiner L., Stern S., Zeft A., and Dare J.

Published
2024

7. Creation of Induced Pluripotent Stem Cells RCMGi014-A Using Reprogramming of Urine Cells of a Patient with Fibrodysplasia Ossificans Progressiva Associated with Heterozygous Mutation in the ACVR1 Gene.

Author

Kondrateva, E. V., Grigorieva, O. V., Kurshakova, E. V., Panchuk, I. O., Pozhitnova, V. O., Voronina, E. S., Tabakov, V. Yu., Nikishina, I. P., Arsenyeva, S. V., Matkava, V. G., Lavrov, A. V., Smirnikhina, S. A., and Kutsev, S. I.

Subjects
INDUCED pluripotent stem cells, FIBRODYSPLASIA ossificans progressiva, GENETIC mutation, EPIBLAST, URINALYSIS
Abstract

Fibrodysplasia ossificans progressiva (FOP) is a severe genetic disease caused by mutations in the ACVR1 gene for which there is currently no effective therapy. Therefore, obtaining iPSCs for further modeling and development of treatment methods, including gene therapy, can be very promising. The authors herein describe the generation of the induced pluripotent stem cell (iPSCs) line RCMGi014-A from urinary sediment cells of a patient with clinically expressed and genetically proven (ACVR1:c.6176G>A) FOP. These iPSCs proliferate in dense monolayer cell colonies, have a normal karyotype (46,XY), express pluripotency markers (OCT4, SOX2, TRA-1-60, SSEA-4), and show the ability to differentiate into three germ layers, which confirms their pluripotent status. [ABSTRACT FROM AUTHOR]

Published
2024
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9. Definition and Validation of the American College of Rheumatology 2021 Juvenile Arthritis Disease Activity Score Cutoffs for Disease Activity States in Juvenile Idiopathic Arthritis

Author

Trincianti, C, Van Dijkhuizen, E, Alongi, A, Mazzoni, M, Swart, J, Nikishina, I, Lahdenne, P, Rutkowska-Sak, L, Avcin, T, Quartier, P, Panaviene, V, Uziel, Y, Pruunsild, C, Vargova, V, Vilaiyuk, S, Dolezalova, P, Ringold, S, Garrone, M, Ruperto, N, Ravelli, A, Consolaro, A, Trincianti C., Van Dijkhuizen E. H. P., Alongi A., Mazzoni M., Swart J. F., Nikishina I., Lahdenne P., Rutkowska-Sak L., Avcin T., Quartier P., Panaviene V., Uziel Y., Pruunsild C., Vargova V., Vilaiyuk S., Dolezalova P., Ringold S., Garrone M., Ruperto N., Ravelli A., Consolaro A., Trincianti, C, Van Dijkhuizen, E, Alongi, A, Mazzoni, M, Swart, J, Nikishina, I, Lahdenne, P, Rutkowska-Sak, L, Avcin, T, Quartier, P, Panaviene, V, Uziel, Y, Pruunsild, C, Vargova, V, Vilaiyuk, S, Dolezalova, P, Ringold, S, Garrone, M, Ruperto, N, Ravelli, A, Consolaro, A, Trincianti C., Van Dijkhuizen E. H. P., Alongi A., Mazzoni M., Swart J. F., Nikishina I., Lahdenne P., Rutkowska-Sak L., Avcin T., Quartier P., Panaviene V., Uziel Y., Pruunsild C., Vargova V., Vilaiyuk S., Dolezalova P., Ringold S., Garrone M., Ruperto N., Ravelli A., and Consolaro A.

Abstract

Objective: To develop and validate new Juvenile Arthritis Disease Activity Score 10 (JADAS10) and clinical JADAS10 (cJADAS10) cutoffs to separate the states of inactive disease (ID), minimal disease activity (MiDA), moderate disease activity (MoDA), and high disease activity (HDA) in children with oligoarthritis and with rheumatoid factor–negative polyarthritis, based on subjective disease assessment by the treating pediatric rheumatologist. Methods: The cutoffs definition cohort was composed of 1,936 patients included in the multinational Epidemiology, Treatment and Outcome of Childhood Arthritis (EPOCA) study. Using the subjective physician rating as an external criterion, 4 methods were applied to identify the cutoffs: mapping, Youden index, 90% specificity, and maximum agreement. The validation cohort included 4,014 EPOCA patients, patients from 2 randomized trials, and 88 patients from the PharmaChild registry. Cutoff validation was conducted by assessing discriminative and predictive ability. Results: The JADAS10 cutoffs were 1.4, 4, and 13, respectively, for oligoarthritis and 2.7, 6, and 17, respectively, for polyarthritis. The cJADAS10 cutoffs were 1.1, 4, and 12, respectively, for oligoarthritis and 2.5, 5, and 16, respectively, for polyarthritis. The cutoffs discriminated strongly among different levels of pain and morning stiffness, between patients who were and those who were not prescribed a new medication, and between different levels of improvement in clinical trials. Achievement of ID and MiDA according to the new JADAS cutoffs at least twice in the first year of disease predicted better outcome at 2 years. Conclusion: The 2021 JADAS and cJADAS cutoffs revealed good metrologic properties in both definition and validation samples, and are therefore suitable for use in clinical trials and routine practice.

Published
2021

10. INSAID Variant Classification and Eurofever Criteria Guide Optimal Treatment Strategy in Patients with TRAPS: Data from the Eurofever Registry

Author

Papa, R, Lane, T, Minden, K, Touitou, I, Cantarini, L, Cattalini, M, Obici, L, Jansson, A, Belot, A, Frenkel, J, Anton, J, Wolska-Kusnierz, B, Berendes, R, Remesal, A, Jelusic, M, Hoppenreijs, E, Espada, G, Nikishina, I, Maggio, M, Bovis, F, Masini, M, Youngstein, T, Rezk, T, Papadopoulou, C, Brogan, P, Hawkins, P, Woo, P, Ruperto, N, Gattorno, M, Lachmann, H, Papa R, Lane T, Minden K, Touitou I, Cantarini L, Cattalini M, Obici L, Jansson AF, Belot A, Frenkel J, Anton J, Wolska-Kusnierz B, Berendes R, Remesal A, Jelusic M, Hoppenreijs E, Espada G, Nikishina I, Maggio MC, Bovis F, Masini M, Youngstein T, Rezk T, Papadopoulou C, Brogan PA, Hawkins PN, Woo P, Ruperto N, Gattorno M, Lachmann HJ, Papa, R, Lane, T, Minden, K, Touitou, I, Cantarini, L, Cattalini, M, Obici, L, Jansson, A, Belot, A, Frenkel, J, Anton, J, Wolska-Kusnierz, B, Berendes, R, Remesal, A, Jelusic, M, Hoppenreijs, E, Espada, G, Nikishina, I, Maggio, M, Bovis, F, Masini, M, Youngstein, T, Rezk, T, Papadopoulou, C, Brogan, P, Hawkins, P, Woo, P, Ruperto, N, Gattorno, M, Lachmann, H, Papa R, Lane T, Minden K, Touitou I, Cantarini L, Cattalini M, Obici L, Jansson AF, Belot A, Frenkel J, Anton J, Wolska-Kusnierz B, Berendes R, Remesal A, Jelusic M, Hoppenreijs E, Espada G, Nikishina I, Maggio MC, Bovis F, Masini M, Youngstein T, Rezk T, Papadopoulou C, Brogan PA, Hawkins PN, Woo P, Ruperto N, Gattorno M, and Lachmann HJ

Abstract

Background: TNF receptor–associated periodic syndrome (TRAPS) is a rare autoinflammatory disease caused by dominant mutation of the TNF super family receptor 1A (TNFRSF1A) gene. Data regarding long-term treatment outcomes are lacking. Objective: To assess correlations of genotype-phenotypes in patients with TRAPS, as defined by the International Study Group for Systemic Autoinflammatory Diseases (INSAID) classification and Eurofever criteria, with treatment responses. Methods: Data from 226 patients with variants of the TNFRSF1A gene and enrolled in the Eurofever registry were classified according to the INSAID classification in groups A (pathogenic or likely pathogenic variants), B (variants of uncertain significance or not classified variants), and C (benign or likely benign variants) and screened for Eurofever criteria. Results: In group A (127 of 226 patients, 56%), all fulfilled Eurofever criteria and 20 of 127 patients (16%) developed AA amyloidosis. In group B (78 of 226 patients, 35%), 40 of 78 patients (51%) did not fulfill Eurofever criteria, displaying a lower incidence of abdominal pain (P <.02) and higher efficacy rate of on-demand nonsteroidal anti-inflammatory drugs (P <.02) and colchicine (P <.001). Group C (21 of 226 patients, 9%) presented a milder disease (P <.02) and none fulfilled Eurofever criteria. Anti-IL-1 drugs were the most frequently used in patients fulfilling Eurofever criteria, with the highest efficacy rate (>85% complete response). No patients on anti-IL-1 treatments developed AA amyloidosis, and 7 women with a history of failure to conceive had successful pregnancies. Conclusion: Anti-IL-1 drugs are the best maintenance treatment in patients with TRAPS. The diagnosis of TRAPS should be considered very carefully in patients of group B not fulfilling Eurofever criteria and group C, and colchicine may be preferable as the first maintenance treatment.

Published
2021

21. Efficacy and Safety of Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis With and Without Fever at Baseline: Results From an Open-Label, Active-Treatment Extension Study

Author

Brunner, H, Quartier, P, Alexeeva, E, Constantin, T, Koné-Paut, I, Marzan, K, Schneider, R, Wulffraat, N, Chasnyk, V, Tirosh, I, Kallinich, T, Kuemmerle-Deschner, J, Wouters, C, Lauwerys, B, Nikishina, I, Trachana, M, Vougiouka, O, Martini, A, Lovell, D, Levy, J, Vritzali, E, Ruperto, N, Brunner HI, Quartier P, Alexeeva E, Constantin T, Koné-Paut I, Marzan K, Schneider R, Wulffraat NM, Chasnyk V, Tirosh I, Kallinich T, Kuemmerle-Deschner J, Wouters C, Lauwerys B, Nikishina I, Trachana M, Vougiouka O, Martini A, Lovell DJ, Levy J, Vritzali E, Ruperto N, Brunner, H, Quartier, P, Alexeeva, E, Constantin, T, Koné-Paut, I, Marzan, K, Schneider, R, Wulffraat, N, Chasnyk, V, Tirosh, I, Kallinich, T, Kuemmerle-Deschner, J, Wouters, C, Lauwerys, B, Nikishina, I, Trachana, M, Vougiouka, O, Martini, A, Lovell, D, Levy, J, Vritzali, E, Ruperto, N, Brunner HI, Quartier P, Alexeeva E, Constantin T, Koné-Paut I, Marzan K, Schneider R, Wulffraat NM, Chasnyk V, Tirosh I, Kallinich T, Kuemmerle-Deschner J, Wouters C, Lauwerys B, Nikishina I, Trachana M, Vougiouka O, Martini A, Lovell DJ, Levy J, Vritzali E, and Ruperto N

Abstract

Objective: To evaluate the long-term efficacy and safety of canakinumab and explore prediction of response in patients with systemic juvenile idiopathic arthritis (JIA) with or without fever at treatment initiation. Methods: At enrollment, patients with active systemic JIA (ages 2 to <20 years) started open-label canakinumab (4 mg/kg every 4 weeks subcutaneously). Efficacy measures included the adapted American College of Rheumatology (ACR) Pediatric 50/70/90 criteria, the Juvenile Arthritis Disease Activity Score (JADAS), and clinically inactive disease and clinical remission on medication, evaluated by either the JADAS or ACR criteria. Results: Of the 123 patients (70 with fever and 52 without fever [fever status was not reported for 1 patient]), 84 (68.3%) completed the study (median duration 1.8 years). Comparable efficacy (adapted ACR Pediatric 50/70/90/100) was observed by day 15 in both subgroups (60.0%/48.6%/37.1%/24.3% in those with fever and 67.3%/48.1%/34.6%/19.2% in those without fever), and further increased thereafter. By month 6, clinical remission according to the JADAS or the ACR criteria was achieved in 17 (24.3%) and 26 (37.1%), respectively, of patients with fever and 9 (17.3%) and 12 (23.1%), respectively, of patients without fever. Median time to onset of clinical remission according to the JADAS or ACR criteria was 57 and 30 days, respectively, in those with fever, and 58 and 142 days, respectively, in those without fever. An adapted ACR Pediatric 50 response by day 15 was the strongest predictor of achieving clinical remission according to the JADAS (odds ratio [OR] 13 [95% confidence interval (95% CI) 4, 42]; P < 0.0001) or glucocorticoid discontinuation (OR 19 [95% CI 3, 114]; P = 0.002). Of the 71 of 123 patients (57.7%) who received glucocorticoids at study entry, 27 (38.0%) discontinued glucocorticoids and 21 (29.6%) reached a dose of <0.2 mg/kg/day, with no difference between those with and those without fever; 13 patients (10.

Published
2020

22. Phenotypic and genotypic characteristics of cryopyrin-associated periodic syndrome: a series of 136 patients from the Eurofever Registry

Author

Levy, R, Gérard, L, Kuemmerle-Deschner, J, Lachmann, H J, Koné-Paut, I, Cantarini, L, Woo, P, Naselli, A, Bader-Meunier, B, Insalaco, A, Al-Mayouf, S M, Ozen, S, Hofer, M, Frenkel, J, Modesto, C, Nikishina, I, Schwarz, T, Martino, S, Meini, A, Quartier, P, Martini, A, Ruperto, N, Neven, B, and Gattorno, M

Published
2015
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23. Etanercept treatment for extended oligoarticular juvenile idiopathic arthritis, enthesitis-related arthritis, or psoriatic arthritis: 6-year efficacy and safety data from an open-label trial

Author

Foeldvari, I, Constantin, T, Vojinovic, J, Horneff, G, Chasnyk, V, Dehoorne, J, Panaviene, V, Susic, G, Stanevicha, V, Kobusinska, K, Zuber, Z, Dobrzyniecka, B, Nikishina, I, Bader-Meunier, B, Breda, L, Dolezalova, P, Job-Deslandre, C, Rumba-Rozenfelde, I, Wulffraat, N, Pedersen, R, Bukowski, J, Vlahos, B, Martini, A, Ruperto, N, Foeldvari I., Constantin T., Vojinovic J., Horneff G., Chasnyk V., Dehoorne J., Panaviene V., Susic G., Stanevicha V., Kobusinska K., Zuber Z., Dobrzyniecka B., Nikishina I., Bader-Meunier B., Breda L., Dolezalova P., Job-Deslandre C., Rumba-Rozenfelde I., Wulffraat N., Pedersen R. D., Bukowski J. F., Vlahos B., Martini A., Ruperto N., Foeldvari, I, Constantin, T, Vojinovic, J, Horneff, G, Chasnyk, V, Dehoorne, J, Panaviene, V, Susic, G, Stanevicha, V, Kobusinska, K, Zuber, Z, Dobrzyniecka, B, Nikishina, I, Bader-Meunier, B, Breda, L, Dolezalova, P, Job-Deslandre, C, Rumba-Rozenfelde, I, Wulffraat, N, Pedersen, R, Bukowski, J, Vlahos, B, Martini, A, Ruperto, N, Foeldvari I., Constantin T., Vojinovic J., Horneff G., Chasnyk V., Dehoorne J., Panaviene V., Susic G., Stanevicha V., Kobusinska K., Zuber Z., Dobrzyniecka B., Nikishina I., Bader-Meunier B., Breda L., Dolezalova P., Job-Deslandre C., Rumba-Rozenfelde I., Wulffraat N., Pedersen R. D., Bukowski J. F., Vlahos B., Martini A., and Ruperto N.

Abstract

Background: To describe the 6-year safety and efficacy of etanercept (ETN) in children with extended oligoarticular juvenile idiopathic arthritis (eoJIA), enthesitis-related arthritis (ERA), and psoriatic arthritis (PsA) Methods: Patients who completed the 2-year, open-label, phase III CLinical Study In Pediatric Patients of Etanercept for Treatment of ERA, PsA, and Extended Oligoarthritis (CLIPPER) were allowed to enroll in its 8-year long-term extension (CLIPPER2). Children received ETN at a once-weekly dose of 0.8 mg/kg, up to a maximum dose of 50 mg/week. Efficacy assessments included the JIA core set of outcomes, the JIA American College of Rheumatology response criteria (JIA-ACR), and the Juvenile Arthritis Disease Activity Score (JADAS). Efficacy data are reported as responder analyses using a hybrid method for missing data imputation and as observed cases. Safety assessments included treatment-emergent adverse events (TEAEs). Results: Out of 127 patients originally enrolled in CLIPPER, 109 (86%) entered CLIPPER2. After 6 years of trial participation (2 years in CLIPPER and 4 years in CLIPPER2), 41 (32%) patients were still taking ETN, 13 (11%) entered the treatment withdrawal phase after achieving low/inactive disease (of whom 7 had to restart ETN), 36 (28%) discontinued treatment for other reasons but are still being observed, and 37 (29%) discontinued treatment permanently. According to the hybrid imputation analysis, proportions of patients achieving JIA ACR90, JIA ACR100, and JADAS inactive disease after the initial 2 years of treatment were 58%, 48%, and 32%, respectively. After the additional 4 years, those proportions in patients who remained in the trial were 46%, 35%, and 24%. Most frequently reported TEAEs [n (%), events per 100 patient-years] were headache [28 (22%), 5.3], arthralgia [24 (19%), 4.6], and pyrexia [20 (16%), 3.8]. Number and frequency of TEAEs, excluding infections and injection site reactions, decreased over the 6-year period from

Published
2019

24. Clinical characteristics and genetic analyses of 187 patients with undefined autoinflammatory diseases

Author

Ter Haar, N, Eijkelboom, C, Cantarini, L, Papa, R, Brogan, P, Kone-Paut, I, Modesto, C, Hofer, M, Iagaru, N, Fingerhutová, S, Insalaco, A, Licciardi, F, Uziel, Y, Jelusic, M, Nikishina, I, Nielsen, S, Papadopoulou-Alataki, E, Olivieri, A, Cimaz, R, Susic, G, Stanevica, V, van Gijn, M, Vitale, A, Ruperto, N, Frenkel, J, Gattorno, M, Ter Haar NM, Eijkelboom C, Cantarini L, Papa R, Brogan PA, Kone-Paut I, Modesto C, Hofer M, Iagaru N, Fingerhutová S, Insalaco A, Licciardi F, Uziel Y, Jelusic M, Nikishina I, Nielsen S, Papadopoulou-Alataki E, Olivieri AN, Cimaz R, Susic G, Stanevica V, van Gijn M, Vitale A, Ruperto N, Frenkel J, Gattorno M, Ter Haar, N, Eijkelboom, C, Cantarini, L, Papa, R, Brogan, P, Kone-Paut, I, Modesto, C, Hofer, M, Iagaru, N, Fingerhutová, S, Insalaco, A, Licciardi, F, Uziel, Y, Jelusic, M, Nikishina, I, Nielsen, S, Papadopoulou-Alataki, E, Olivieri, A, Cimaz, R, Susic, G, Stanevica, V, van Gijn, M, Vitale, A, Ruperto, N, Frenkel, J, Gattorno, M, Ter Haar NM, Eijkelboom C, Cantarini L, Papa R, Brogan PA, Kone-Paut I, Modesto C, Hofer M, Iagaru N, Fingerhutová S, Insalaco A, Licciardi F, Uziel Y, Jelusic M, Nikishina I, Nielsen S, Papadopoulou-Alataki E, Olivieri AN, Cimaz R, Susic G, Stanevica V, van Gijn M, Vitale A, Ruperto N, Frenkel J, and Gattorno M

Abstract

Objectives To describe the clinical characteristics, treatment response and genetic findings in a large cohort of patients with undefined systemic autoinflammatory diseases (SAIDs). Methods Clinical and genetic data from patients with undefined SAIDs were extracted from the Eurofever registry, an international web-based registry that retrospectively collects clinical information on patients with autoinflammatory diseases. Results This study included 187 patients. Seven patients had a chronic disease course, 180 patients had a recurrent disease course. The median age at disease onset was 4.3 years. Patients had a median of 12 episodes per year, with a median duration of 4 days. Most commonly reported symptoms were arthralgia (n=113), myalgia (n=86), abdominal pain (n=89), fatigue (n=111), malaise (n=104) and mucocutaneous manifestations (n=128). In 24 patients, relatives were affected as well. In 15 patients, genetic variants were found in autoinflammatory genes. Patients with genetic variants more often had affected relatives compared with patients without genetic variants (p=0.005). Most patients responded well to non-steroidal anti-inflammatory drugs (NSAIDs), corticosteroids, colchicine and anakinra. Complete remission was rarely achieved with NSAIDs alone. Notable patterns were found in patients with distinctive symptoms. Patients with pericarditis (n=11) were older at disease onset (33.8 years) and had fewer episodes per year (3.0/year) compared with other patients. Patients with an intellectual impairment (n=8) were younger at disease onset (2.2 years) and often had relatives affected (28.6%). Conclusion This study describes the clinical characteristics of a large cohort of patients with undefined SAIDs. Among these, patients with pericarditis and intellectual impairment appear to comprise distinct subsets.

Published
2019

25. INSAID Variant Classification and Eurofever Criteria Guide Optimal Treatment Strategy in Patients with TRAPS: Data from the Eurofever Registry

Author

Papa R, Lane T, Minden K, Touitou I, Cantarini L, Cattalini M, Obici L, Jansson AF, Belot A, Frenkel J, Anton-Lopez J, Wolska-Kusnierz B, Berendes R, Remesal A, Jelusic M, Hoppenreijs E, Espada G, Nikishina I, Maggio MC, Bovis F, Masini M, Youngstein T, Rezk T, Papadopoulou C, Brogan PA, Hawkins PN, Woo P, Ruperto N, Gattorno M, Lachmann HJ, and Pediatric Rheumatology International Trials Organization (PRINTO), the EUROTRAPS

Subjects
Colchicine, Autoinflammatory diseases, AA amyloidosis, TRAPS, Anakinra
Abstract

BACKGROUND: TNF receptor-associated periodic syndrome (TRAPS) is a rare autoinflammatory disease caused by dominant mutation of the TNF super family receptor 1A (TNFRSF1A) gene. Data regarding long-term treatment outcomes are lacking. OBJECTIVE: To assess correlations of genotype-phenotypes in patients with TRAPS, as defined by the International Study Group for Systemic Autoinflammatory Diseases (INSAID) classification and Eurofever criteria, with treatment responses. METHODS: Data from 226 patients with variants of the TNFRSF1A gene and enrolled in the Eurofever registry were classified according to the INSAID classification in groups A (pathogenic or likely pathogenic variants), B (variants of uncertain significance or not classified variants), and C (benign or likely benign variants) and screened for Eurofever criteria. RESULTS: In group A (127 of 226 patients, 56%), all fulfilled Eurofever criteria and 20 of 127 patients (16%) developed AA amyloidosis. In group B (78 of 226 patients, 35%), 40 of 78 patients (51%) did not fulfill Eurofever criteria, displaying a lower incidence of abdominal pain (P < .02) and higher efficacy rate of on-demand nonsteroidal anti-inflammatory drugs (P 85% complete response). No patients on anti-IL-1 treatments developed AA amyloidosis, and 7 women with a history of failure to conceive had successful pregnancies. CONCLUSION: Anti-IL-1 drugs are the best maintenance treatment in patients with TRAPS. The diagnosis of TRAPS should be considered very carefully in patients of group B not fulfilling Eurofever criteria and group C, and colchicine may be preferable as the first maintenance treatment.

Published
2021

26. INSAID Variant Classification and Eurofever Criteria Guide Optimal Treatment Strategy in Patients with TRAPS: Data from the Eurofever Registry

Author

Papa, R., Lane, T., Minden, K., Touitou, I., Cantarini, L., Cattalini, M., Obici, L., Jansson, A.F., Belot, A., Frenkel, J., Anton, J., Wolska-Kusnierz, B., Berendes, R., Remesal, A., Jelusic, M., Hoppenreijs, E.P., Espada, G., Nikishina, I., Maggio, M.C., Bovis, F., Masini, M., Youngstein, T., Rezk, T., Papadopoulou, C., Brogan, P.A., Hawkins, P.N., Woo, P., Ruperto, N., Gattorno, M., Lachmann, H.J., Papa, R., Lane, T., Minden, K., Touitou, I., Cantarini, L., Cattalini, M., Obici, L., Jansson, A.F., Belot, A., Frenkel, J., Anton, J., Wolska-Kusnierz, B., Berendes, R., Remesal, A., Jelusic, M., Hoppenreijs, E.P., Espada, G., Nikishina, I., Maggio, M.C., Bovis, F., Masini, M., Youngstein, T., Rezk, T., Papadopoulou, C., Brogan, P.A., Hawkins, P.N., Woo, P., Ruperto, N., Gattorno, M., and Lachmann, H.J.

Abstract

Item does not contain fulltext, BACKGROUND: TNF receptor-associated periodic syndrome (TRAPS) is a rare autoinflammatory disease caused by dominant mutation of the TNF super family receptor 1A (TNFRSF1A) gene. Data regarding long-term treatment outcomes are lacking. OBJECTIVE: To assess correlations of genotype-phenotypes in patients with TRAPS, as defined by the International Study Group for Systemic Autoinflammatory Diseases (INSAID) classification and Eurofever criteria, with treatment responses. METHODS: Data from 226 patients with variants of the TNFRSF1A gene and enrolled in the Eurofever registry were classified according to the INSAID classification in groups A (pathogenic or likely pathogenic variants), B (variants of uncertain significance or not classified variants), and C (benign or likely benign variants) and screened for Eurofever criteria. RESULTS: In group A (127 of 226 patients, 56%), all fulfilled Eurofever criteria and 20 of 127 patients (16%) developed AA amyloidosis. In group B (78 of 226 patients, 35%), 40 of 78 patients (51%) did not fulfill Eurofever criteria, displaying a lower incidence of abdominal pain (P < .02) and higher efficacy rate of on-demand nonsteroidal anti-inflammatory drugs (P < .02) and colchicine (P < .001). Group C (21 of 226 patients, 9%) presented a milder disease (P < .02) and none fulfilled Eurofever criteria. Anti-IL-1 drugs were the most frequently used in patients fulfilling Eurofever criteria, with the highest efficacy rate (>85% complete response). No patients on anti-IL-1 treatments developed AA amyloidosis, and 7 women with a history of failure to conceive had successful pregnancies. CONCLUSION: Anti-IL-1 drugs are the best maintenance treatment in patients with TRAPS. The diagnosis of TRAPS should be considered very carefully in patients of group B not fulfilling Eurofever criteria and group C, and colchicine may be preferable as the first maintenance treatment.

Published
2021

34. OP0027 AS-RELATED TCR BETA CLONOTYPES ARE PRESENT IN DIFFERENT INFLAMED TISSUES OF PATIENTS WITH SPONDYLOARTHROPATHIES

Author

Komech, E., primary, Barinova, A., additional, Shmidt, E., additional, Korotaeva, T., additional, Koltakova, A., additional, Nikitina, N., additional, Belousova, E., additional, Shapina, M., additional, Rodionovskaya, S., additional, Nikishina, I., additional, Vahlyarskaya, S., additional, Atarshchikov, D., additional, Klescheva, E., additional, Chudakov, D., additional, Lukyanov, S., additional, and Zvyagin, I., additional

Published
2021
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45. FRI0457 LONG-TERM OUTCOMES AND TREATMENT EFFICACY IN PATIENTS WITH TNF RECEPTOR-ASSOCIATED AUTOINFLAMMATORY SYNDROME (TRAPS) FROM THE EUROFEVER INTERNATIONAL REGISTRY

Author

Papa, R., primary, Lane, T., additional, Bovis, F., additional, Minden, K., additional, Touitou, I., additional, Cantarini, L., additional, Cattalini, M., additional, Obici, L., additional, Jansson, A., additional, Belot, A., additional, Woska-Kuśnierz, B., additional, Berendes, R., additional, Remesal, A., additional, Jelusic, M., additional, Espada, G., additional, Nikishina, I., additional, Hoppenreijs, E., additional, Maggio, M. C., additional, Youngstein, T., additional, Rezk, T., additional, Papadopoulou, C., additional, Brogan, P., additional, Hawkins, P. N., additional, Woo, P., additional, Ruperto, N., additional, Gattorno, M., additional, and Lachmann, H. J., additional

Published
2020
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48. PRINTO/PRES international website for families of children with rheumatic diseases: www.pediatric-rheumatology.printo.it

Author

Ruperto, N, Garcia-Munitis, P, Villa, L, Pesce, M, Aggarwal, A, Fasth, A, Avcin, T, Bae, S-C, Balogh, Z, Li, C, De Inocencio, J, Dibra, M, Dolezalova, P, Miedany, Y El, Flato, B, Harjacek, M, Huppertz, H-I, Kanakoudi-Tsakalidou, F, Wulffraat, N, Lahdenne, P, Melo-Gomes, J A, Mihaylova, D, Nielsen, S, Nikishina, I, Ozdogan, H, Pagava, K, Panaviene, V, Prieur, A-M, Romicka, A-M, Rumba, I, Shafaie, N, Susic, G, Takei, S, Uziel, Y, Vesely, R, Woo, P, and Martini, A

Published
2005

50. Spondyloarthritis: modern terminology and definitions

Author

Erdes, Sh F, primary, Rebrov, A P, additional, Dubinina, T V, additional, Badokin, V V, additional, Bochkova, A G, additional, Bugrova, O V, additional, Gaydukova, I Z, additional, Godzenko, A A, additional, Dubikov, A A, additional, Ivanova, O N, additional, Korotaeva, T V, additional, Lapshina, S A, additional, Nesmeyanova, O B, additional, Nikishina, I P, additional, Otteva, E N, additional, Raskina, T A, additional, Rumyantseva, O A, additional, Sitalo, A V, additional, and Smirnov, A V, additional

Published
2019
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