Evans syndrome: clinical perspectives, biological insights and treatment modalities
José Carlos Jaime-Pérez, Patrizia Elva Aguilar-Calderón, Lorena Salazar-Cavazos, David Gómez-Almaguer Department of Hematology, Internal Medicine Division, Dr José E González University Hospital, School of Medicine of the Universidad...
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Dove Medical Press
2018-10-01
|
| Series: | Journal of Blood Medicine |
| Subjects: | |
| Online Access: | https://www.dovepress.com/evans-syndrome-clinical-perspectives-biological-insights-and-treatment-peer-reviewed-article-JBM |
| _version_ | 1811302349801521152 |
|---|---|
| author | Jaime-Pérez JC Aguilar-Calderón PE Salazar-Cavazos L Gómez-Almaguer D |
| author_facet | Jaime-Pérez JC Aguilar-Calderón PE Salazar-Cavazos L Gómez-Almaguer D |
| author_sort | Jaime-Pérez JC |
| collection | DOAJ |
| description | José Carlos Jaime-Pérez, Patrizia Elva Aguilar-Calderón, Lorena Salazar-Cavazos, David Gómez-Almaguer Department of Hematology, Internal Medicine Division, Dr José E González University Hospital, School of Medicine of the Universidad Autónoma de Nuevo León, Monterrey, Nuevo León, México Abstract: Evans syndrome (ES) is a rare and chronic autoimmune disease characterized by autoimmune hemolytic anemia and immune thrombocytopenic purpura with a positive direct anti-human globulin test. It is classified as primary and secondary, with the frequency in patients with autoimmune hemolytic anemia being 37%–73%. It predominates in children, mainly due to primary immunodeficiencies or autoimmune lymphoproliferative syndrome. ES during pregnancy is associated with high fetal morbidity, including severe hemolysis and intracranial bleeding with neurological sequelae and death. The clinical presentation can include fatigue, pallor, jaundice and mucosal bleeding, with remissions and exacerbations during the person’s lifetime, and acute manifestations as catastrophic bleeding and massive hemolysis. Recent molecular theories explaining the physiopathology of ES include deficiencies of CTLA-4, LRBA, TPP2 and a decreased CD4/CD8 ratio. As in other autoimmune cytopenias, there is no established evidence-based treatment and steroids are the first-line therapy, with intravenous immunoglobulin administered as a life-saving resource in cases of severe immune thrombocytopenic purpura manifestations. Second-line treatment for refractory ES includes rituximab, mofetil mycophenolate, cyclosporine, vincristine, azathioprine, sirolimus and thrombopoietin receptor agonists. In cases unresponsive to immunosuppressive agents, hematopoietic stem cell transplantation has been successful, although it is necessary to consider its potential serious adverse effects. In conclusion, ES is a disease with a heterogeneous course that remains challenging to patients and physicians, with prospective clinical trials needed to explore potential targeted therapy to achieve an improved long-term response or even a cure. Keywords: Evans syndrome, autoimmune cytopenias, low-dose rituximab, IVIG, systemic lupus erythematosus, HSCT, sirolimus, mofetil mycophenolate, HSCT |
| first_indexed | 2024-04-13T07:26:29Z |
| format | Article |
| id | doaj.art-09e9db531331444faf606223a318d60d |
| institution | Directory Open Access Journal |
| issn | 1179-2736 |
| language | English |
| last_indexed | 2024-04-13T07:26:29Z |
| publishDate | 2018-10-01 |
| publisher | Dove Medical Press |
| record_format | Article |
| series | Journal of Blood Medicine |
| spelling | doaj.art-09e9db531331444faf606223a318d60d2022-12-22T02:56:27ZengDove Medical PressJournal of Blood Medicine1179-27362018-10-01Volume 917118441317Evans syndrome: clinical perspectives, biological insights and treatment modalitiesJaime-Pérez JCAguilar-Calderón PESalazar-Cavazos LGómez-Almaguer DJosé Carlos Jaime-Pérez, Patrizia Elva Aguilar-Calderón, Lorena Salazar-Cavazos, David Gómez-Almaguer Department of Hematology, Internal Medicine Division, Dr José E González University Hospital, School of Medicine of the Universidad Autónoma de Nuevo León, Monterrey, Nuevo León, México Abstract: Evans syndrome (ES) is a rare and chronic autoimmune disease characterized by autoimmune hemolytic anemia and immune thrombocytopenic purpura with a positive direct anti-human globulin test. It is classified as primary and secondary, with the frequency in patients with autoimmune hemolytic anemia being 37%–73%. It predominates in children, mainly due to primary immunodeficiencies or autoimmune lymphoproliferative syndrome. ES during pregnancy is associated with high fetal morbidity, including severe hemolysis and intracranial bleeding with neurological sequelae and death. The clinical presentation can include fatigue, pallor, jaundice and mucosal bleeding, with remissions and exacerbations during the person’s lifetime, and acute manifestations as catastrophic bleeding and massive hemolysis. Recent molecular theories explaining the physiopathology of ES include deficiencies of CTLA-4, LRBA, TPP2 and a decreased CD4/CD8 ratio. As in other autoimmune cytopenias, there is no established evidence-based treatment and steroids are the first-line therapy, with intravenous immunoglobulin administered as a life-saving resource in cases of severe immune thrombocytopenic purpura manifestations. Second-line treatment for refractory ES includes rituximab, mofetil mycophenolate, cyclosporine, vincristine, azathioprine, sirolimus and thrombopoietin receptor agonists. In cases unresponsive to immunosuppressive agents, hematopoietic stem cell transplantation has been successful, although it is necessary to consider its potential serious adverse effects. In conclusion, ES is a disease with a heterogeneous course that remains challenging to patients and physicians, with prospective clinical trials needed to explore potential targeted therapy to achieve an improved long-term response or even a cure. Keywords: Evans syndrome, autoimmune cytopenias, low-dose rituximab, IVIG, systemic lupus erythematosus, HSCT, sirolimus, mofetil mycophenolate, HSCThttps://www.dovepress.com/evans-syndrome-clinical-perspectives-biological-insights-and-treatment-peer-reviewed-article-JBMFisher Evans SyndromeAutoimmune cytopeniasLow-dose RituximabIVIGSystemic Lupus ErythematosusHSCT. |
| spellingShingle | Jaime-Pérez JC Aguilar-Calderón PE Salazar-Cavazos L Gómez-Almaguer D Evans syndrome: clinical perspectives, biological insights and treatment modalities Journal of Blood Medicine Fisher Evans Syndrome Autoimmune cytopenias Low-dose Rituximab IVIG Systemic Lupus Erythematosus HSCT. |
| title | Evans syndrome: clinical perspectives, biological insights and treatment modalities |
| title_full | Evans syndrome: clinical perspectives, biological insights and treatment modalities |
| title_fullStr | Evans syndrome: clinical perspectives, biological insights and treatment modalities |
| title_full_unstemmed | Evans syndrome: clinical perspectives, biological insights and treatment modalities |
| title_short | Evans syndrome: clinical perspectives, biological insights and treatment modalities |
| title_sort | evans syndrome clinical perspectives biological insights and treatment modalities |
| topic | Fisher Evans Syndrome Autoimmune cytopenias Low-dose Rituximab IVIG Systemic Lupus Erythematosus HSCT. |
| url | https://www.dovepress.com/evans-syndrome-clinical-perspectives-biological-insights-and-treatment-peer-reviewed-article-JBM |
| work_keys_str_mv | AT jaimeperezjc evanssyndromeclinicalperspectivesbiologicalinsightsandtreatmentmodalities AT aguilarcalderonpe evanssyndromeclinicalperspectivesbiologicalinsightsandtreatmentmodalities AT salazarcavazosl evanssyndromeclinicalperspectivesbiologicalinsightsandtreatmentmodalities AT gomezalmaguerd evanssyndromeclinicalperspectivesbiologicalinsightsandtreatmentmodalities |