Abstract

TREATMENT OF SECONDARY IMMUNE THROMBOCYTOPENIA UNRESPONSIVE TO STEROIDS IN ADULT-ONSET STILL’S DISEASE: A CASE REPORT SUCCESFULLY TREATED WITH A COMBINATION THERAPY OF RITUXIMAB AND ELTROMBOPAG

Author(s): Darlo Brono1, 2, Elena Rossi3, Annamaria Paglionico1, Valentina Varriano1, Valerio De Stefano3 and Eliza Gremese1,4*

Rationale: Adult-onset Still’s disease (AOSD) is a rare systemic autoinflammatory disease with potentially severe life-threatening complications. Among them, coagulation disorders such as disseminated intravascular coagulopathy and thrombotic microangiopathy may occur. Nevertheless, alterations in platelet count may have several other causes. We present the first case of a patient with AOSD complicated by immune thrombocytopenic purpura (ITP) and the individualized therapeutic approach.

Patient concerns: An 18-year-old female suffering from AOSD on immunosuppressive therapy with methotrexate and anakinra was admitted to our ward due to severe thrombocytopenia, unresponsive to glucocorticoids.

Diagnosis: A bone marrow aspirate showed normal megakariocytes, with no schistocytes, nor hemophagocytosis, thus suggesting increased peripheral destruction as the possible underlying pathological mechanism. The detection of anti-platelet antibodies against Gp Ib/IX epitopes confirmed the ITP hypothesis. In addition, Helicobacter pylori (HP) infection was identified in the gastric biopsy.

Interventions and outcomes: Due to the resistance of thrombocytopenia to high-dose steroids and intravenous immunoglobulins (IVIg), second-line therapy with rituximab (RTX) was started, combined with eltrombopag for persistent recurrent bleeding. HP infection treatment was prescribed and urea breath test (performed after one month) confirmed eradication of HP.

Four months after discharge, platelet counts returned steadily to the normal range; prednisone was tapered to 5 mg/day and eltrombopag continued. Oral cyclosporine was chosen as maintenance immunosuppressive treatment and another course of RTX was administered after six months.

Conclusion: Thrombocytopenia in AOSD can represent a diagnostic and clinical challenge due to many potential differential diagnoses. This is the first reported case of ITP in a patient with AOSD, in which combination therapy with RTX and a thrombopoietin receptor agonist (eltrombopag) was used, with the resolution of thrombocytopenia. Concomitant HP infection was recognized and treated. Abbreviations: AOSD: Adult-onset Still's Disease, ITP: Immune thrombocytopenic purpura, HP: Helicobacter pylori, IVIg: Intravenous immunoglobulins, RTX: Rituximab, TPO-RAs: Thrombopoietin receptor agonists