Managementul actual al trombocitopeniei imune recăzute/refractare

Abstract

Immune thrombocytopenia (ITP) is an acquired auto­im­mune bleeding disorder characterized by antibody me­dia­ted platelet destruction and impaired platelet pro­duc­tion. Adult patients often present with a chronic di­sease, displaying a stable platelet count and a low risk for bleeding. However, there is a small category of ITP patients who have instable platelet count, are at high risk for bleeding and are refractory to all forms of treat­ment. Immune thrombocytopenia is a diagnosis of ex­clu­sion; extensive diagnostic work is needed in order to classify a patient as having refractory ITP. Refractory pa­tients do not respond to steroids, i.v. immunoglobulins, throm­bo­poietin receptor agonists (TPO-RA) or rituximab, or to any other form of treatment. Combination therapy has been evaluated in small cases, with varying results. Newer treat­ments – fostamatinib, FcRn or Bruton kinase (BTK) in­hi­bitors – appear to be more effective. Exploring the bio­lo­gy of refractoriness is essential for understanding the complex pathology of this disease and for finding ways to stop its natural evolution. In summary, refractory im­mune thrombocytopenia is a complex condition that re­quires precise diagnosis and the initiation of an effective com­bi­na­tion treatment from onset.