Clinical Characteristics of <scp>Cryopyrin‐Associated</scp> Periodic Syndrome and <scp>Long‐Term Real‐World</scp> Efficacy and Tolerability of Canakinumab in Japan: Results of a Nationwide Survey-Reference-Cited by-同舟云学术

Clinical Characteristics of Cryopyrin‐Associated Periodic Syndrome and Long‐Term Real‐World Efficacy and Tolerability of Canakinumab in Japan: Results of a Nationwide Survey

Published:2024-03-07 Issue:6 Volume:76 Page:949-962
ISSN:2326-5191
Container-title:Arthritis & Rheumatology
language:en
Short-container-title:Arthritis & Rheumatology

Author:

Miyamoto Takayuki¹^ORCID,Izawa Kazushi¹^ORCID,Masui Sho¹,Yamazaki Atsue¹,Yamasaki Yuichi²^ORCID,Matsubayashi Tadashi³,Shiraki Mayuka⁴,Ohnishi Hidenori⁴,Yasumura Junko⁵,Kawabe Tomohiro⁶,Miyamae Takako⁶^ORCID,Matsubara Tomoyo⁷,Arakawa Naoya⁸,Ishige Takashi⁸,Takizawa Takumi⁸,Shimbo Asami⁹,Shimizu Masaki⁹^ORCID,Kimura Naoki⁹,Maeda Yuichi¹⁰^ORCID,Maruyama Yuta¹¹,Shigemura Tomonari¹¹,Furuta Junichi¹²,Sato Satoshi¹³^ORCID,Tanaka Hiroshi¹⁴,Izumikawa Miharu¹⁵,Yamamura Masahiro¹⁶,Hasegawa Toshio¹⁷,Kaneko Hiroshi¹⁸,Nakagishi Yasuo¹⁹,Nakano Naoko²⁰,Iida Yasunori²¹,Nakamura Tamaki²¹,Wakiguchi Hiroyuki²¹^ORCID,Hoshina Takayuki²²,Kawai Toshinao²³,Murakami Kosaku¹,Akizuki Shuji¹,Morinobu Akio¹,Ohmura Koichiro²⁴,Eguchi Katsuhide²⁵,Sonoda Motoshi²⁵,Ishimura Masataka²⁵,Furuno Kenji²⁶,Kashiwado Momoko²⁷,Mori Masaaki²⁸^ORCID,Kawahata Kimito²⁷,Hayama Koremasa²⁹^ORCID,Shimoyama Kumiko³⁰,Sasaki Natsuko²²,Ito Taisuke³⁰,Umebayashi Hiroaki³¹,Omori Tae³²,Nakamichi Seiko³³,Dohmoto Tomotsune³⁴,Hasegawa Yasuyuki³⁴,Kawashima Hisashi³⁵,Watanabe Shojiro³⁶,Taguchi Yuichiro³⁷,Nakaseko Haruna³⁸,Iwata Naomi³⁸^ORCID,Kohno Hiroki⁶,Ando Taiki¹⁷,Ito Yasuhiko³⁹,Kataoka Yuko⁴⁰,Saeki Takako⁴¹,Kaneko Utako⁴²,Murase Ayako⁴³,Hattori Seira⁴³^ORCID,Nozawa Tomo⁴³^ORCID,Nishimura Kenichi⁴³,Nakano Reiji⁴⁴,Watanabe Misa⁴⁵,Yashiro Masato⁴⁶,Nakamura Tomonori⁴⁷,Komai Toshihiko⁴⁸,Kato Kentaro¹,Honda Yoshitaka¹^ORCID,Hiejima Eitaro¹,Yonezawa Atsushi¹,Bessho Kazuhisa¹,Okada Satoshi⁵,Ohara Osamu⁴⁹,Takita Junko¹,Yasumi Takahiro¹,Nishikomori Ryuta⁵⁰^ORCID,

Affiliation:

1. Kyoto University Kyoto Japan

2. Kagoshima University Hospital Kagoshima Japan

3. Seirei Hamamatsu General Hospital Hamamatsu Japan

4. Gifu University Gifu Japan

5. Hiroshima University Hiroshima Japan

6. Tokyo Women's Medical University Hospital Tokyo Japan

7. Dokkyo Medical University Saitama Medical Center Saitama Japan

8. Gunma University Maebashi Japan

9. Tokyo Medical and Dental University Tokyo Japan

10. Osaka University Suita Japan

11. Shinshu University Matsumoto Japan

12. University of Tsukuba Tsukuba Japan

13. Saitama Children's Medical Center Saitama Japan

14. Hirosaki University Hospital Hirosaki Japan

15. Kagawa University Kagawa Japan

16. Okayama Saiseikai General Hospital Okayama Japan

17. Juntendo University Tokyo Japan

18. National Center for Global Health and Medicine Tokyo Japan

19. Hyogo Prefectural Kobe Children's Hospital Kobe Japan

20. Ehime Prefectural Central Hospital Matsuyama Japan

21. Yamaguchi University Ube Japan

22. University of Occupational and Environmental Health, Japan Kitakyushu Japan

23. National Center for Child Health and Development Tokyo Japan

24. Kobe City Medical Center General Hospital Kobe Japan

25. Kyushu University Fukuoka Japan

26. Fukuoka Children's Hospital Fukuoka Japan

27. St. Marianna University School of Medicine Kawasaki Japan

28. Tokyo Medical and Dental University, Tokyo, Japan, and St. Marianna University School of Medicine Kawasaki Japan

29. Nihon University Tokyo Japan

30. Hamamatsu University School of Medicine Hamamatsu Japan

31. Miyagi Children's Hospital Sendai Japan

32. Tokyo Metropolitan Bokutoh Hospital Tokyo Japan

33. Nagasaki University Nagasaki Japan

34. Tottori Prefectural Central Hospital Tottori Japan

35. Tokyo Medical University Tokyo Japan

36. Ehime University Matsuyama Japan

37. Department of Rheumatology Nagoya Ekisaikai Hospital Nagoya Japan

38. Aichi Children's Health and Medical Center Obu Japan

39. Nagoya City University West Medical Center Nagoya Japan

40. Matsuyama Red Cross Hospital Matsuyama Japan

41. Nagaoka Red Cross Hospital Nagaoka Japan

42. Niigata University Niigata Japan

43. Yokohama City University Yokohama Japan

44. Shizuoka Children's Hospital Shizuoka Japan

45. Toho University Tokyo Japan

46. Okayama University Hospital Okayama Japan

47. Kagoshima University Kagoshima Japan

48. The University of Tokyo Tokyo Japan

49. Kazusa DNA Research Institute Kisarazu Japan

50. Kurume University Kurume Japan

Abstract

ObjectiveWe assess the clinical characteristics of patients with cryopyrin‐associated periodic syndrome (CAPS) in Japan and evaluate the real‐world efficacy and safety of interleukin‐1 (IL‐1) inhibitors, primarily canakinumab.MethodsClinical information was collected retrospectively, and serum concentrations of canakinumab and cytokines were analyzed.ResultsA total of 101 patients were included, with 86 and 15 carrying heterozygous germline and somatic mosaic mutations, respectively. We identified 39 mutation types, and the common CAPS‐associated symptoms corresponded with those in previous reports. Six patients (5.9% of all patients) died, with four of the deaths caused by CAPS‐associated symptoms. Notably, 73.7% of patients (100%, 79.6%, and 44.4% of familial cold autoinflammatory syndrome, Muckle–Wells syndrome, and chronic infantile neurological cutaneous articular syndrome/neonatal onset multisystem inflammatory disease, respectively) achieved complete remission with canakinumab, and early therapeutic intervention was associated with better auditory outcomes. In some patients, canakinumab treatment stabilized the progression of epiphysial overgrowth and improved height gain, visual acuity, and renal function. However, 23.7% of patients did not achieve inflammatory remission with crucial deterioration of organ damage, with two dying while receiving high‐dose canakinumab treatment. Serological analysis of canakinumab and cytokine concentrations revealed that the poor response was not related to canakinumab shortage. Four inflammatory nonremitters developed inflammatory bowel disease (IBD)—unclassified during canakinumab treatment. Dual biologic therapy with canakinumab and anti–tumor necrosis factor‐α agents was effective for IBD– and CAPS‐associated symptoms not resolved by canakinumab monotherapy.ConclusionThis study provides one of the largest epidemiologic data sets for CAPS. Although early initiation of anti–IL‐1 treatment with canakinumab is beneficial for improving disease prognosis, some patients do not achieve remission despite a high serum concentration of canakinumab. Moreover, IBD may develop in CAPS after canakinumab treatment.

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Funder

Japan Agency for Medical Research and Development

Japan Society for the Promotion of Science

AMED

Publisher

Wiley

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