Therapeutic approaches in the treatment of juvenile dermatomyositis in patients with recent-onset disease and in those experiencing disease flare: an international multicenter PRINTO study
Rachana Hasija, Angela Pistorio, Angelo Ravelli, Erkan Demirkaya, Raju Khubchandani, Dinara Guseinova, Clara Malattia, Helena Canhao, Liora Harel, Dirk Foell, Carine Wouters, Carmen De Cunto, Christian Huemer, Yukiko Kimura, Harald Mangge, Carlo Minetti, Ellen Berit Nordal, Pierre Philippet, Rosaria Garozzo, Alberto Martini, Nicolino Ruperto, Pediatric Rheumatology International Trials Organization, Maria Apaz, Ruben Cuttica, Graciela Espada, Stella Garay, Ricardo Russo, Kevin Murray, Blanca Bica, Virginia Ferriani, Odete Hilario, Sheila Oliveira, Iloite Scheibel, Clovis Artur Silva, Flavio Sztajnbok, Dimitrina Mihaylova, Brian Feldman, Arnoldo Quezada, Ximena Norambuena, Oscar Porras, Miroslav Harjacek, Lana Tambic-Bukovac, Cecilia Coto, Pavla Dolezalova, Susan Nielsen, Pekka Lahdenne, Michel Bost, Michel Fischbach, Anne-Marie Prieur, Pierre Quartier, Danièle Sommelet, Jurgen Brunner, Guenther Dannecker, Stephan Ehl, Ivan Foeldvari, Gerd Ganser, Hans-Iko Huppertz, Hartmut Michels, Kirsten Minden, Florence Kanakoudi, Jenny Pratsidou, Olga Vougiouka, Zsolt Balogh, Riva Brik, Yosef Uziel, Roberto Barcellona, Fabrizia Corona, Elisabetta Cortis, Fernanda Falcini, Valeria Gerloni, Loredana Lepore, Francesco Zulian, Valda Stanevicha, Ruben Burgos Vargas, Raul Gutierrez, Carolina Duarte, Rebecca Ten Cate, Lisetta Van Suijlekom-Smit, Nico Wulffraat, Berit Flato, Marite Rygg, Anna Maria Romicka, Malgorzata Wierzbowska, Jose Antonio Melo-Gomes, Mirjana Jovanovic, Srdjan Pasic, Gordana Susic, Yvonne See, Jozef Rovensky, Richard Vesely, Veronika Vargova, Tadej Avcin, Immaculada Calvo, Julia Consuegra, Mari Luz Gamir, Consuelo Modesto, Juan Ros, Stefan Hagelberg, Michael Hofer, Traudel Saurenmann, Marie-Josephe Sauvain, Rym Hajri Ben Ammar, Huri Ozdogan, Ozgur Kasapcopur, Seza Ozen, Aysin Bakkaloglu, Madeleine Rooney, Helen Venning, Patricia Woo, Clarissa Pilkington, Brent Graham, Sampath Prahalad, Marilyn Punaro, Anne Reed, Phil Hashkes, Lauren Pachman, Carol Wallace, Robert Rennebohm, Gloria Higgins, Kenneth Schikler, Rachana Hasija, Angela Pistorio, Angelo Ravelli, Erkan Demirkaya, Raju Khubchandani, Dinara Guseinova, Clara Malattia, Helena Canhao, Liora Harel, Dirk Foell, Carine Wouters, Carmen De Cunto, Christian Huemer, Yukiko Kimura, Harald Mangge, Carlo Minetti, Ellen Berit Nordal, Pierre Philippet, Rosaria Garozzo, Alberto Martini, Nicolino Ruperto, Pediatric Rheumatology International Trials Organization, Maria Apaz, Ruben Cuttica, Graciela Espada, Stella Garay, Ricardo Russo, Kevin Murray, Blanca Bica, Virginia Ferriani, Odete Hilario, Sheila Oliveira, Iloite Scheibel, Clovis Artur Silva, Flavio Sztajnbok, Dimitrina Mihaylova, Brian Feldman, Arnoldo Quezada, Ximena Norambuena, Oscar Porras, Miroslav Harjacek, Lana Tambic-Bukovac, Cecilia Coto, Pavla Dolezalova, Susan Nielsen, Pekka Lahdenne, Michel Bost, Michel Fischbach, Anne-Marie Prieur, Pierre Quartier, Danièle Sommelet, Jurgen Brunner, Guenther Dannecker, Stephan Ehl, Ivan Foeldvari, Gerd Ganser, Hans-Iko Huppertz, Hartmut Michels, Kirsten Minden, Florence Kanakoudi, Jenny Pratsidou, Olga Vougiouka, Zsolt Balogh, Riva Brik, Yosef Uziel, Roberto Barcellona, Fabrizia Corona, Elisabetta Cortis, Fernanda Falcini, Valeria Gerloni, Loredana Lepore, Francesco Zulian, Valda Stanevicha, Ruben Burgos Vargas, Raul Gutierrez, Carolina Duarte, Rebecca Ten Cate, Lisetta Van Suijlekom-Smit, Nico Wulffraat, Berit Flato, Marite Rygg, Anna Maria Romicka, Malgorzata Wierzbowska, Jose Antonio Melo-Gomes, Mirjana Jovanovic, Srdjan Pasic, Gordana Susic, Yvonne See, Jozef Rovensky, Richard Vesely, Veronika Vargova, Tadej Avcin, Immaculada Calvo, Julia Consuegra, Mari Luz Gamir, Consuelo Modesto, Juan Ros, Stefan Hagelberg, Michael Hofer, Traudel Saurenmann, Marie-Josephe Sauvain, Rym Hajri Ben Ammar, Huri Ozdogan, Ozgur Kasapcopur, Seza Ozen, Aysin Bakkaloglu, Madeleine Rooney, Helen Venning, Patricia Woo, Clarissa Pilkington, Brent Graham, Sampath Prahalad, Marilyn Punaro, Anne Reed, Phil Hashkes, Lauren Pachman, Carol Wallace, Robert Rennebohm, Gloria Higgins, Kenneth Schikler
Abstract
Objective: To evaluate response to therapy over a 24-month period in a large prospective international cohort of patients with juvenile dermatomyositis (DM).
Methods: The study included 145 patients with recent-onset juvenile DM and 130 juvenile DM patients experiencing disease flare, all of whom were <18 years old. Disease activity parameters and therapeutic approaches in 4 geographic areas were analyzed at baseline and at 6, 12, and 24 months. Response was assessed according to the Pediatric Rheumatology International Trials Organization (PRINTO) juvenile DM response criteria, and data were reported "as observed" and in the intent-to-treat (ITT) population.
Results: Patients with recent-onset juvenile DM at baseline had higher baseline disease activity and greater improvement over 24 months when compared to juvenile DM patients experiencing disease flare at baseline. Methotrexate (MTX) or high-dose corticosteroids were administered more frequently to patients with recent-onset juvenile DM, compared to juvenile DM patients experiencing disease flare, who were more likely to receive cyclosporine. Compared to patients from Western and Eastern Europe, a higher proportion of patients from South and Central America and North America received pulse steroids, and the average steroid dosage was higher in the North American and South and Central American patients. The use of MTX was similar in all 4 regions, while cyclosporin A was more frequently used in Western Europe. In the "as observed" analysis, 57.9% of the patients with recent-onset juvenile DM and 36.4% of the patients experiencing disease flare (P<0.001) reached at least a 70% response by PRINTO criteria at 6 months; these proportions had increased at month 24 to 78.4% and 51.2%, respectively (P<0.001). Corresponding results of the ITT analysis were much lower, with only one-third of the patients able to maintain the initial assigned therapy over 24 months.
Conclusion: Patients with recent-onset juvenile DM are more likely to achieve significant clinical improvement over 24 months, when compared to patients experiencing flares of juvenile DM. Internationally, various therapeutic approaches are used to treat this disease.
Copyright © 2011 by the American College of Rheumatology.
Source: PubMed