Efficacy and safety of thalidomide in children with monogenic autoinflammatory diseases: a single-center, real-world-evidence study

Zhang, Caihui; Yu, Zhongxun; Gao, Sihao; Ma, Mingsheng; Gou, Lijuan; Wang, Changyan; Wang, Lin; Li, Ji; Zhong, Linqing; Zhou, Yu; Wang, Wei; Song, Hongmei

doi:10.1186/s12969-023-00881-0

Pediatric Rheumatology

Table 1 Baseline characteristics of 16 patients with monogenic autoinflammatory diseases treated with thalidomide

From: Efficacy and safety of thalidomide in children with monogenic autoinflammatory diseases: a single-center, real-world-evidence study

Characteristics (n, %)	AGS	Blau syndrome	CINCA	DADA2	FMF	HA20	PLAID	SAVI	TRAPS	Total
Num	3 (18.75)	4 (25.0)	2 (12.50)	1 (6.25)	1 (6.25)	2 (12.50)	1 (6.25)	1 (6.25)	1 (6.25)	16 (100.0)
Gender
Female	2 (25.0)	2 (25.0)	1 (12.50)	1 (12.50)	0	1 (12.50)	1 (12.50)	0	0	8 (50.0)
Male	1 (12.50)	2 (25.0)	1 (12.50)	0	1 (12.50)	1 (12.50)	0	1 (12.50)	1(12.50)	8 (50.0)
Clinical manifestations
Fever	0	1 (12.50)	1 (12.50)	1 (12.50)	0	2 (25.0)	1 (12.50)	1 (12.50)	1 (12.50)	8 (50.0)
Rash	2 (16.67)	4 (33.33)	2 (16.67)	1 (8.33)	0	1 (8.33)	1 (8.33)	1 (8.33)	0	12 (75.0)
Growth or intellectual disability	1 (16.67)	2 (33.33)	2 (33.33)	0	0	0	0	1 (16.67)	0	6 (37.50)
Gastrointestinal involvement	1 (16.67)	0	1 (16.67)	1 (16.67)	1 (16.67)	1 (16.67)	1 (16.67)	0	0	6 (37.50)
Respiratory involvement	2 (28.57)	0	2 (28.57)	0	0	1 (14.29)	1 (14.29)	1 (14.29)	0	7 (43.75)
Cardiovascular involvement	1 (14.29)	3 (42.86)	2 (28.57)	0	0	0	1 (14.29)	0	0	7(43. 75)
Joint involvement	1 (33.33)	2 (66.67)	0	0	0	0	0	0	0	3 (18.75)
Eye or ear involvement	1 (14.29)	4 (57.14)	2 (28.57)	0	0	0	0	0	0	7 (43. 75)
Hematologic involvement	2 (28.57)	1 (14.29)	2 (28.57)	1 (14.29)	0	0	1 (14.29)	0	0	7 (43. 75)
Neurologic involvement	1 (20.0)	1 (20.0)	2 (40.0)	1 (20.0)	0	0	0	0	0	5 (31.25)
Hypothyroidism	1(50.0)	0	0	0	0	1(50.0)	0	0	0	2 (12.50)
Autoantibody positivity	2 (33.33)	1 (16.67)	0	0	0	1 (16.67)	1 (16.67)	1 (16.67)	0	6 (37.50)
Elevated CRP	0	3 (27.27)	2 (18.18)	1 (9.09)	1 (9.09)	1 (9.09)	1 (9.09)	1 (9.09)	1 (9.09)	11 (68.75)
Elevated ESR	2 (22.22)	2 (22.22)	1 (11.11)	1 (11.11)	0	1 (11.11)	0	1 (11.11)	1 (11.11)	9 (56.25)
Treatment before the addition of thalidomide
Glucocorticoids	3 (27.27)	2 (18.18)	2 (18.18)	1 (9.09)	1 (9.09)	0	1 (9.09)	1 (9.09)	0	11 (68.75)
Immunosuppressive agents	3 (25.0)	3 (25.0)	1 (8.33)	1 (8.33)	1 (8.33)	1 (8.33)	0	1 (8.33)	1 (8.33)	12 (75.0)
Janus Kinase Inhibitors	3 (75.0)	0	0	0	0	0	0	1 (25.0)	0	4 (25.0)
MTX	0	3 (60.0)	1 (20.0)	1 (20.0)	0	0	0	0	0	5 (31.25)
Others	0	0	0	0	1^a (33.33)	1^b (33.33)	0	0	1^a (33.3)	3 (18.75)
Biologics	0	1 (50.0)^c	0	1 (50.0)^d	0	0	0	0	0	2 (12.50)

n (%) represents the number of positive cases in each disease category (number of positive cases in each disease category/total number of positive cases × 100%)
DADA2 adenosine deaminase 2 deficiency, PLAID PLCγ2-associated antibody deficiency and immune dysregulation, MTX Methotrexate
^aColchicine
^bHydroxychloroquine
^cEtanercept
^dTocilizumab

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ISSN: 1546-0096

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