特发性炎性肌病完全临床应答相关因素的单中心真实世界研究

doi:10.19723/j.issn.1671-167X.2024.02.013

摘要/Abstract

摘要：

目的: 探究影响特发性炎性肌病(idiopathic inflammatory myopathies，IIMs)患者对常规治疗完全临床应答的相关因素。方法: 纳入2000年1月至2023年6月于北京大学人民医院就诊的IIMs患者，通过分析患者的临床特征、实验室检查、免疫学指标和治疗用药，明确影响患者对常规治疗完全临床应答的相关因素。结果: 共纳入635例IIMs患者，其中518例患者完成随访，平均随访时间36.8个月，总体完全临床应答率为50.0%(259/518)。各临床亚型中，皮肌炎(dermatomyositis，DM)、抗合成酶综合征(anti-synthetase syndrome，ASS)和免疫介导坏死性肌病(immune-mediated necrotizing myopathy，IMNM)的完全临床应答率分别为53.5%、48.9%和39.0%。未完全临床应答组与完全临床应答组相比，在临床特征方面，发热(P=0.002)和快速进展型间质性肺病(rapid progressive interstitial lung disease，RP-ILD)(P=0.014)的发生率较高；在实验室检查方面，谷草转氨酶、乳酸脱氢酶、D-二聚体、红细胞沉降率、C反应蛋白和血清铁蛋白水平较高；在治疗用药方面，激素和静脉注射免疫球蛋白(intravenous immunoglobin，IVIG)的使用比例均较高。IMNM(P=0.007)、间质性肺病(P=0.001)、谷草转氨酶高(P=0.012)、血清铁蛋白高(P=0.016)和外周血CD4⁺T细胞计数低(P=0.004)是IIMs未完全临床应答的危险因素。结论: IIMs患者的总体完全临床应答率低，IMNM亚型最低; 起病时存在间质性肺病、谷草转氨酶高、血清铁蛋白高或外周血CD4⁺T细胞计数低的患者应给予积极治疗。

关键词: 特发性炎性肌病, 临床应答, 间质性肺病, 危险因素, 自身抗体

Abstract:

Objective: To investigate the correlation factors of complete clinical response in idiopathic inflammatory myopathies (IIMs) patients receiving conventional treatment. Methods: Patients diagnosed with IIMs hospitalized in Peking University People's Hospital from January 2000 to June 2023 were included. The correlation factors of complete clinical response to conventional treatment were identified by analyzing the clinical characteristics, laboratory features, peripheral blood lymphocytes, immunological indicators, and therapeutic drugs. Results: Among the 635 patients included, 518 patients finished the follow-up, with an average time of 36.8 months. The total complete clinical response rate of IIMs was 50.0% (259/518). The complete clinical response rate of dermatomyositis (DM), anti-synthetase syndrome (ASS) and immune-mediated necrotizing myopathy (IMNM) were 53.5%, 48.9% and 39.0%, respectively. Fever (P=0.002) and rapid progressive interstitial lung disease (RP-ILD) (P=0.014) were observed much more frequently in non-complete clinical response group than in complete clinical response group. The aspartate transaminase (AST), lactate dehydrogenase (LDH), D-dimer, erythrocyte sedimentation rate (ESR), C-reaction protein (CRP) and serum ferritin were significantly higher in non-complete clinical response group as compared with complete clinical response group. As for the treatment, the percentage of glucocorticoid received and intravenous immunoglobin (IVIG) were significantly higher in non-complete clinical response group than in complete clinical response group. Risk factor analysis showed that IMNM subtype (P=0.007), interstitial lung disease (ILD) (P=0.001), eleva-ted AST (P=0.012), elevated serum ferritin (P=0.016) and decreased count of CD4⁺T cells in peripheral blood (P=0.004) might be the risk factors for IIMs non-complete clinical response. Conclusion: The total complete clinical response rate of IIMs is low, especially for IMNM subtype. More effective intervention should be administered to patients with ILD, elevated AST, elevated serum ferritin or decreased count of CD4⁺T cells at disease onset.

Key words: Idiopathic inflammatory myopathies, Clinical response, Interstitial lung disease, Risk factors, Autoantibodies

中图分类号:

R593.26

赖展鸿,李嘉辰,贠泽霖,张永刚,张昊,邢晓燕,邵苗,金月波,王乃迪,李依敏,李玉慧,栗占国. 特发性炎性肌病完全临床应答相关因素的单中心真实世界研究[J]. 北京大学学报（医学版）, 2024, 56(2): 284-292.

Zhanhong LAI,Jiachen LI,Zelin YUN,Yonggang ZHANG,Hao ZHANG,Xiaoyan XING,Miao SHAO,Yuebo JIN,Naidi WANG,Yimin LI,Yuhui LI,Zhanguo LI. A unicenter real-world study of the correlation factors for complete clinical response in idiopathic inflammatory myopathies[J]. Journal of Peking University (Health Sciences), 2024, 56(2): 284-292.

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Variables	CCR (n=259)	NCCR (n=259)	P value
Female	195 (75.3)	190 (73.4)	0.615
Age at onset/years	49.3±15.0	50.2±13.6	0.460
DM	146 (56.4)	127 (49.0)	0.095
ASS	60 (23.2)	63 (24.3)	0.757
IMNM	46 (17.8)	72 (27.8)	0.006
Fever	75 (29.0)	109 (42.1)	0.002
Dyspnea	103 (39.8)	91 (35.1)	0.276
Gottron’s sign/papules	158 (61.0)	166 (64.1)	0.468
Mechanic’s hands	72 (27.8)	82 (31.7)	0.336
Heliotrope rash	99 (38.2)	110 (42.5)	0.325
ILD	159 (61.4)	189 (73.0)	0.005
RP-ILD	84 (32.4)	111 (42.9)	0.014
C-ILD	72 (27.8)	67 (25.9)	0.620

Variables	CCR (n=259)	NCCR (n=259)	P value
WBC/(×10⁹/L)	7.53±3.37	7.81±3.66	0.363
Lymphocyte/(×10⁹/L)	1.47±0.84	1.36±0.84	0.146
PLT/(×10⁹/L)	228.0±84.5	223.0±83.1	0.483
ALT/(U/L)	33.0 (20.3, 56.0)	45.5 (20.0, 103.0)	0.509
AST/(U/L)	34.0 (24.2, 69.0)	63.5 (31.0, 132.0)	0.008
LDH/(U/L)	289 (232, 519)	429 (290, 631)	0.001
CK/(U/L)	77.0 (34.5, 251.0)	76.0 (26.8, 775.0)	0.627
D-dimer/(μg/L)	322 (195, 487)	425 (282, 714)	0.002
ESR/(mm/L)	17.5 (8.9, 41.8)	36.5 (12.8, 58.3)	0.003
CRP/(mg/L)	3.43 (0.63, 20.40)	8.03 (2.00, 28.50)	0.002
Ferritin/(μg/L)^a	229 (83, 1 116)	566 (272, 1 588)	0.020
IgG/(g/L)	14.50±6.43	13.90±5.37	0.214
C3/(g/L)	1.00±0.27	1.01±0.27	0.760
C4/(g/L)	0.25±0.12	0.24±0.10	0.575

Variables	CCR (n=259)	NCCR (n=259)	P value
Glucocorticoid, n (%)	206 (79.5)	226 (87.3)	0.018
Prednisone ≤ 15 mg/d^a	63 (24.4)	49 (19.0)	0.135
15 mg/d＜prednisone＜30 mg/d^a	9 (3.5)	9 (3.5)	＞0.999
Prednisone ≥ 30 mg/d^a	119 (46.1)	125 (48.4)	0.597
Pulse IVMP	67 (25.9)	75 (29.0)	0.431
Immunosuppressants, n (%)	166 (64.1)	156 (60.2)	0.365
CTX	96 (37.1)	94 (36.3)	0.855
CSA	54 (20.8)	51 (19.7)	0.743
TAC	10 (3.9)	17 (6.6)	0.166
MMF	20 (7.7)	11 (4.2)	0.095
JAKi, n (%)	2 (0.8)	8 (3.1)	0.055
IVIG, n (%)	25 (9.7)	42 (16.2)	0.026

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