Refractory/relapse thrombocytopenia in a patient with Evans' syndrome successfully treated with zanubrutinib

Abstract

BackgroundEvans' syndrome (ES) is a rare autoimmune disorder and has high mortality rate. Due to the rarity of the disease, the treatments of ES are always emprical, including steroids, IVIG, rituximab, TPO-RAs, immunosuppressants, splenectomy and supportive therapies. However, many patients may become relapsed/refractory to a series of different interventions. Herein, we report a case that a ES patient with severe refractory/relapse thrombocytopenia was successfully treated with zanubrutinib. Case presentationA 15-year-old Chinese girl, with repeated skin petechiae and ecchymosis, was diagnosed of Evans' syndrome nally. Despite the initial treatment with dexamethasone, recombinant human thrombopoietin receptor agonist (Eltrombopag /Avatrobopa), immunoglobulin, rituximab, immunosuppressants (Cyclosporine/Azathioprine /Sirolimus) and splenectomy, the patient relapsed and nally had a dramatic and lasting recovery after being treated with zanubrutinib, a second-generation selective covalent Inhibitor of Bruton's Tyrosine Kinase. ConclusionWe concluded by reviewing the cases of refractory/relapse Evans' syndrome and the application of BTK Inhibitors for the treatment of autoimmune diseases. Zanubrutinib may be a feasible therapeutic option for patients with Evans' syndrome who do not respond well to traditional therapies. Ours is the rst published case of using covalent Inhibitor of BTK in refractory/relapse Evans' syndrome and contributes to the successful use of zanubrutinib in future clinical practice. However, the role of zanubrutinib in ES treatment requires more basic and clinical trials to test and evaluate.

Safety and Efficacy of Tyrosine Kinase Inhibitors in Immune Thrombocytopenic Purpura: A Systematic Review of Clinical Trials

“…Zanubrutinib is also a BTKI-like rilzabrutinib and orelabrutinib. A case report showed encouraging results of zanubrutinib in a 15-year-old Chinese girl with severe unresponsive ITP with Evans’ syndrome [ 32 ]. Trials are in progress in China on monotherapy, and the combination of zanubrutinib with TPO and steroids in refractory ITP patients or first-line therapy (NCT05279872, NCT05369377, NCT05369364).…”