Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity
Por:
Berrueco R, González E, Deyà-Martinez A, Solsona M, García-García AP, Calzada-Hernández J, Luo Y, Vlagea A, Ruiz-Llobet A and Alsina L
Publicada:
17 oct 2023
Ahead of Print:
17 oct 2023
Categoría:
Pediatrics, perinatology and child health
Resumen:
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.
Filiaciones:
Berrueco R:
Pediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain
Instituto Nacional de Investigación Biomédica en Enfermedades Raras (CIBER ER), Instituto de Salud Carlos III, Madrid, Spain
González E:
Pediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain
Deyà-Martinez A:
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain
Clinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Clinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, Spain
Solsona M:
Pediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain
García-García AP:
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain
Clinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Clinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, Spain
Calzada-Hernández J:
Pediatric Rheumatology Unit, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain
Luo Y:
Clinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Clinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, Spain
Vlagea A:
Clinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, Spain
Immunology Department, Centre of Biomedical Diagnosis, Hospital Clínic, Barcelona, Spain
Ruiz-Llobet A:
Pediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain
Instituto Nacional de Investigación Biomédica en Enfermedades Raras (CIBER ER), Instituto de Salud Carlos III, Madrid, Spain
Alsina L:
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain
Clinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, Spain
Clinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, Spain
Department of Surgery and Surgical Specializations, Facultat de Medicina i Ciències de la Salut, Universitat de Barcelona, Barcelona, Spain
Hosp St Joan Deu, Pediat Hematol Dept, Barcelona, Spain
Inst Recerca St Joan Deu Barcelona IRSJD, Barcelona, Spain
Inst Salud Carlos III, Inst Nacl Invest Biomed Enfermedades Raras CIBER
E, Madrid, Spain
Hosp St Joan Deu, Pediat Allergy & Clin Immunol Dept, Clin Immunol &
Primary Immunodeficiencies Unit, Barcelona, Spain
Hosp Clin Barcelona, Hosp St Joan Deu, Clin Immunol Unit, Barcelona,
Spain
Univ Barcelona, Hosp St Joan Deu, Pediat Rheumatol Unit, Barcelona,
Hosp Clin Barcelona, Ctr Biomed Diag, Immunol Dept, Barcelona, Spain
Univ Barcelona, Dept Surg & Surg Specializat, Fac Med Ciencies Salut 1,
Barcelona, Spain
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