Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity
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...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-10-01
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| Series: | Frontiers in Pediatrics |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fped.2023.1174671/full |
| _version_ | 1797658930864717824 |
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| author | Rubén Berrueco Rubén Berrueco Rubén Berrueco Elisa González-Forster Elisa González-Forster Angela Deya-Martinez Angela Deya-Martinez Angela Deya-Martinez María Solsona María Solsona Ana García-García Ana García-García Ana García-García Joan Calzada-Hernández Luo Yiyi Luo Yiyi Alexandru Vlagea Alexandru Vlagea Anna Ruiz-Llobet Anna Ruiz-Llobet Anna Ruiz-Llobet Laia Alsina Laia Alsina Laia Alsina Laia Alsina |
| author_facet | Rubén Berrueco Rubén Berrueco Rubén Berrueco Elisa González-Forster Elisa González-Forster Angela Deya-Martinez Angela Deya-Martinez Angela Deya-Martinez María Solsona María Solsona Ana García-García Ana García-García Ana García-García Joan Calzada-Hernández Luo Yiyi Luo Yiyi Alexandru Vlagea Alexandru Vlagea Anna Ruiz-Llobet Anna Ruiz-Llobet Anna Ruiz-Llobet Laia Alsina Laia Alsina Laia Alsina Laia Alsina |
| author_sort | Rubén Berrueco |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-11T18:06:32Z |
| format | Article |
| id | doaj.art-c1cf8f55a80f457ea9aee86ea7dab839 |
| institution | Directory Open Access Journal |
| issn | 2296-2360 |
| language | English |
| last_indexed | 2024-03-11T18:06:32Z |
| publishDate | 2023-10-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pediatrics |
| spelling | doaj.art-c1cf8f55a80f457ea9aee86ea7dab8392023-10-17T06:28:09ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602023-10-011110.3389/fped.2023.11746711174671Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunityRubén Berrueco0Rubén Berrueco1Rubén Berrueco2Elisa González-Forster3Elisa González-Forster4Angela Deya-Martinez5Angela Deya-Martinez6Angela Deya-Martinez7María Solsona8María Solsona9Ana García-García10Ana García-García11Ana García-García12Joan Calzada-Hernández13Luo Yiyi14Luo Yiyi15Alexandru Vlagea16Alexandru Vlagea17Anna Ruiz-Llobet18Anna Ruiz-Llobet19Anna Ruiz-Llobet20Laia Alsina21Laia Alsina22Laia Alsina23Laia Alsina24Pediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, SpainInstitut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, SpainInstituto Nacional de Investigación Biomédica en Enfermedades Raras (CIBER ER), Instituto de Salud Carlos III, Madrid, SpainPediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, SpainInstitut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, SpainInstitut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, SpainClinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, SpainClinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, SpainPediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, SpainInstitut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, SpainInstitut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, SpainClinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, SpainClinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, SpainPediatric Rheumatology Unit, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, SpainClinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, SpainClinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, SpainClinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, SpainImmunology Department, Centre of Biomedical Diagnosis, Hospital Clínic, Barcelona, SpainPediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, SpainInstitut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, SpainInstituto Nacional de Investigación Biomédica en Enfermedades Raras (CIBER ER), Instituto de Salud Carlos III, Madrid, SpainInstitut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, SpainClinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, Hospital Sant Joan de Déu, Barcelona, SpainClinical Immunology Unit, Hospital Sant Joan de Déu-Hospital Clínic Barcelona, Barcelona, SpainDepartment of Surgery and Surgical Specializations, Facultat de Medicina I Ciències de la Salut, Universitat de Barcelona, Barcelona, SpainSecond-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.https://www.frontiersin.org/articles/10.3389/fped.2023.1174671/fullpediatricsautoimmune hemolytic anemiaautoimmune thrombocytopeniaEvans syndromeautoimmune diseasesmycophenolate mofetil |
| spellingShingle | Rubén Berrueco Rubén Berrueco Rubén Berrueco Elisa González-Forster Elisa González-Forster Angela Deya-Martinez Angela Deya-Martinez Angela Deya-Martinez María Solsona María Solsona Ana García-García Ana García-García Ana García-García Joan Calzada-Hernández Luo Yiyi Luo Yiyi Alexandru Vlagea Alexandru Vlagea Anna Ruiz-Llobet Anna Ruiz-Llobet Anna Ruiz-Llobet Laia Alsina Laia Alsina Laia Alsina Laia Alsina Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity Frontiers in Pediatrics pediatrics autoimmune hemolytic anemia autoimmune thrombocytopenia Evans syndrome autoimmune diseases mycophenolate mofetil |
| title | Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity |
| title_full | Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity |
| title_fullStr | Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity |
| title_full_unstemmed | Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity |
| title_short | Mycophenolate mofetil for autoimmune cytopenias in children: high rates of response in inborn errors of immunity |
| title_sort | mycophenolate mofetil for autoimmune cytopenias in children high rates of response in inborn errors of immunity |
| topic | pediatrics autoimmune hemolytic anemia autoimmune thrombocytopenia Evans syndrome autoimmune diseases mycophenolate mofetil |
| url | https://www.frontiersin.org/articles/10.3389/fped.2023.1174671/full |
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