Stem cell transplantation for patients with Evans syndrome

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.

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“…In older patients with significant comorbidities who lack a human leukocyte antigen-identical donor, autologous HSCT could be performed. 144 …”

Section: Third-line Treatmentsmentioning

confidence: 99%

Evans syndrome: clinical perspectives, biological insights and treatment modalities

Jaime‐Pérez¹,

Aguilar-Calderón²,

Salazar‐Cavazos³

et al. 2018

JBM

113

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“…3 Concomitant neutropenia is observed in up to 50% of the patients. 4 ES is usually a chronic disease and frequently refractory to conventional treatment with immunosuppression, i.v. Igs, splenectomy, cytostatic drugs and monoclonal Abs against CD20.…”

mentioning

confidence: 99%

Successful allogeneic cord blood transplantation in a patient with Evans syndrome leads to correction of hereditary angioedema type I as secondary effect

Klausegger

Wiednig

Urban

et al. 2012

Bone Marrow Transplant

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“…Immunosuppressive drugs (cyclosporine A, mycophenolate mofetil, azathioprine), cytostatic agents (cyclophosphamide, vincristine), monoclonal antibodies (rituximab, alemtuzumab), and splenectomy are considered as second‐line treatment . Autologous and allogeneic hematopoietic stem cell transplantation (HSCT) have also been used as a treatment modality . Despite various treatment options, AC become frequently chronic or relapsing with a long‐term mortality of as high as 50% mainly due to hemorrhagic and infectious complications .…”

Section: Introductionmentioning

confidence: 99%

Treatment‐refractory multi‐lineage autoimmune cytopenia after unrelated cord blood transplantation: Remission after combined bortezomib and vincristine treatment

Waespe

Zeilhofer

Güngör

2014

Pediatric Blood & Cancer

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Autoimmune cytopenias (AC) after allogeneic hematopoietic stem cell transplantation (HSCT) are associated with a dismal prognosis. We describe a 1-year-old female with multi-lineage AC occurring on day +43 after HSCT. Multi-agent treatment with high-dose prednisolone, intravenous immunoglobulins, cyclosporine A, mycophenolate mofetil, sirolimus, and rituximab was unsuccessful. Combined treatment with bortezomib and vincristine in addition to ongoing immunosuppressive therapy was started on day +414 with transfusion-independence after day +444. Immunosuppressants were tapered until day +638. On day +1,121 the patient remained in remission. Bortezomib with vincristine may be a promising treatment modality for refractory AC after HSCT that requires further study.

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Stem cell transplantation for patients with Evans syndrome

Cited by 3 publications

References 47 publications

Evans syndrome: clinical perspectives, biological insights and treatment modalities

Evans syndrome: clinical perspectives, biological insights and treatment modalities

Successful allogeneic cord blood transplantation in a patient with Evans syndrome leads to correction of hereditary angioedema type I as secondary effect

Treatment‐refractory multi‐lineage autoimmune cytopenia after unrelated cord blood transplantation: Remission after combined bortezomib and vincristine treatment

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