The Roles and Challenges of Advanced Therapies in the Management of Refractory Immune Thrombocytopenia: A Case Report and Review of the Literature

Abstract

The management of primary immune thrombocytopenia (ITP) is becoming a subject of interest as there appears to be treatment failure and resistance to modern conventional treatment, necessitating a more universal and goal-directed approach to management. Our patient is a 74-year-old male who was diagnosed with ITP six years ago and recently presented to the emergency department (ED) with complaints of melena stools and severe fatigue lasting for two days. Prior to the ED presentation, he had received multiple lines of treatment including splenectomy. On admission, the pathology after splenectomy showed a benign enlarged spleen with a focal area of intraparenchymal hemorrhage/rupture and changes compatible with ITP. He was managed with multiple platelet transfusions, IV methyl prednisone succinate, rituximab, and romiplostim. His platelet counts improved to 47,000, and he was discharged home on oral steroids with outpatient hematology follow-up. However, in a few weeks, his condition deteriorated, and he presented with an increased platelet count and further multiple complaints. Romiplostim was discontinued, and he was continued on prednisone 20 mg daily, after which he improved, and his platelet count reduced to 273,000 on 20 mg prednisone. This case calls attention to the need to review the role of combination therapy in treating refractory ITP and the prevention of complications of thrombocytosis secondary to advanced therapy. Treatment needs to be more streamlined, focused, and goal-directed. Escalation and de-escalation of treatment should be synchronized to prevent adverse complications from overtreating or undertreating.

Therapeutic Outcomes of High Dose-Dexamethasone versus Prednisolone + Azathioprine, Rituximab, Eltrombopag, and Romiplostim Strategies in Persistent, Chronic, Refractory, and Relapsed Immune Thrombocytopenia Patients

“…Moreover, we described the persistent ITP patients who had ITP that lasted between 3 and 12 months after diagnosis, While the chronic ITP patients had ITP lasting for more than 12 months [ 11 ]. Moreover, we described the refractory ITP who relapsed after splenectomy and initial response to conventional therapies (including rituximab and thrombopoietin Receptor Agonists [ 13 , 14 , 15 ]. The relapsed ITP patients who relapsed after normalization of platelets while patients were both on and off-label treatment, with the greatest response to that line recorded before relapse and subsequent requiring re-therapy [ 16 , 46 ].…”

“…The American Society of Hematology guidelines identified a group of patients as having refractory ITP if they matched two criteria: (1) they failed splenectomy and (2) they remained to have serious ITP or a bleeding risk necessitating therapy [ 1 , 12 ]. Recent studies also defined the refractory term as a lack of response to one or more conventional therapies (including rituximab and thrombopoietin Receptor Agonists [ 13 , 14 , 15 ]. Relapsed ITP was described in the patients who represented recurrent thrombocytopenia after normalization of platelets while patients were both on and off-label treatment, with the greatest response to that line recorded before relapse and subsequent requiring re-therapy [ 16 ].…”