Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity

被引：3

作者：

Berrueco, Ruben ^{[1
,2
,3
]}

Gonzalez-Forster, Elisa ^{[1
,2
]}

Deya-Martinez, Angela ^{[2
,4
,5
]}

Solsona, Maria ^{[1
,2
]}

Garcia-Garcia, Ana ^{[2
,4
,5
]}

Calzada-Hernandez, Joan ^{[6
]}

Yiyi, Luo ^{[4
,5
]}

Vlagea, Alexandru ^{[5
,7
]}

Ruiz-Llobet, Anna ^{[1
,2
,3
]}

Alsina, Laia ^{[2
,4
,5
,8
]}

机构：

[1] Hosp St Joan Deu, Pediat Hematol Dept, Barcelona, Spain

[2] Inst Recerca St Joan Deu Barcelona IRSJD, Barcelona, Spain

[3] Inst Salud Carlos III, Inst Nacl Invest Biomed Enfermedades Raras CIBER E, Madrid, Spain

[4] Hosp St Joan Deu, Pediat Allergy & Clin Immunol Dept, Clin Immunol & Primary Immunodeficiencies Unit, Barcelona, Spain

[5] Hosp Clin Barcelona, Hosp St Joan Deu, Clin Immunol Unit, Barcelona, Spain

[6] Univ Barcelona, Hosp St Joan Deu, Pediat Rheumatol Unit, Barcelona, Spain

[7] Hosp Clin Barcelona, Ctr Biomed Diag, Immunol Dept, Barcelona, Spain

[8] Univ Barcelona, Dept Surg & Surg Specializat, Fac Med Ciencies Salut 1, Barcelona, Spain

来源：

FRONTIERS IN PEDIATRICS | 2023年 / 11卷

关键词：

pediatrics; autoimmune hemolytic anemia; autoimmune thrombocytopenia; Evans syndrome; autoimmune diseases; mycophenolate mofetil; PRIMARY IMMUNODEFICIENCIES; THROMBOCYTOPENIC PURPURA;

D O I：

10.3389/fped.2023.1174671

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Second-line treatments of autoimmune cytopenias (AC) are not well-defined in children. Mycophenolate mofetil (MMF) is an immunosuppressant agent that has been demonstrated to be safe and effective in this setting. A retrospective observational study was conducted in 18 children with prolonged AC who received MMF, in order to describe clinical and biological markers of response. The overall response rate of MMF at 20-30 mg/kg per day was 73.3%. All patients with Evans syndrome (n = 9) achieved complete response. Among the patients with monolineage AC (n = 9), those with an underlying inborn errors of immunity (IEI), tended to respond better to MMF. No biological markers related to treatment response were found. Rather, lymphocyte subpopulations proved useful for patient selection as a marker suggestive of IEI along with immunoglobulin-level determination.

引用

页数：7

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