Background: In Australia, prescribing restrictions limit access to internationally recommended second-line therapies such as rituximab and thrombopoietin agonists (TPO-A) (eltrombopag and romiplostim). Subsequent lines of therapy include an array of immunosuppressive and immune-modulating agents directed by drug availability and physician and patient preference.
Objectives:The objective of the study was to describe the use of first and subsequent lines of treatment for adult immune thrombocytopenia (ITP) in Australia and to assess their effectiveness and tolerability.Patients/Methods: A retrospective review of medical records was conducted of 322 patients treated for ITP at eight participating centers in Australia between 2013 and 2020. Data were analyzed by descriptive statistics and frequency distribution using pivot tables, and comparisons between centers were assessed using paired t tests.Results: Mean age at diagnosis of ITP was 48.8 years (standard deviation [SD], 22.6) and 58.3% were women. Primary ITP was observed in 72% and secondary ITP in 28% of the patients; 95% of patients received first-line treatment with prednisolone (76%), dexamethasone (15%), or intravenous immunoglobulin (48%) alone or in combination. Individuals with secondary ITP were less steroid dependent (72% vs. 76%) and required less treatment with a second-line agent (47% vs. 58%) in the study sample. Over half (56%) of the cohort received treatment with one or more second-line agents. The mean number of second-line agents used for each patient was 1.9 (SD, 1.2). The most used second-line therapy was rituximab, followed by etrombopag and splenectomy. These also generated the highest rates of complete response (60.3%, 72.1%, and 71.8% respectively). The most unfavorable side effect profiles were seen in long-term corticosteroids and splenectomy.
This case report describes a patient with an idiopathic acquired Factor VIII inhibitor and severe bleeding. She was treated with rituximab after failing first-line treatment with steroids and cyclophosphamide. Two months following rituximab treatment, our patient developed a succession of severe opportunistic infections requiring intensive care unit admission. Over a period of 12 weeks she required treatment for Pseudomonas aeruginosa septicaemia, herpes simplex gingivostomatitis and pharyngotonsillitis, clostridium difficile-related diarrhoea, systemic cytomegalovirus infection, pneumocystis jiroveci, and invasive pulmonary aspergillosis lung infections. After significant rehabilitation, the patient was finally discharged following a 5-month admission. This case highlights the complexity of balancing a life-threatening condition with the side effects of treatment. It also raises the issue of routine prophylaxis for immunosuppression in nonmalignant conditions, which will become a common dilemma with the expanding indications for rituximab use.
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