ABSTRACT
Thrombotic thrombocytopenic purpura (TTP) is a low prevalence disease characterized by severe deficiency of the enzyme ADAMTS13, leading to the development of thrombotic microangiopathy (TMA) and often resulting in severe organ disfunction. TTP is an extremely serious condition and, therefore, timely and appropriate treatment is critical to prevent life-threatening complications.Over the past 25 years, significant advances in the understanding of the pathophysiology of immune TTP have led to the development of readily available techniques for measuring ADAMTS13 levels, as well as new drugs that are particularly effective in the acute phase and in preventing relapses. These developments have improved the course of the disease.Given the complexity of the disease and its various clinical and laboratory manifestations, early diagnosis and treatment can be challenging.To address this challenge, a group of experienced professionals from the Catalan TTP group have developed this consensus statement to standardize terminology, diagnosis, treatment and follow up for immune TTP, based on currently available scientific evidence in the field. This guidance document aims to provide healthcare professionals with a comprehensive tool to make more accurate and timely diagnosis of TTP and improve patient outcomes.
Subject(s)
Purpura, Thrombocytopenic, Idiopathic , Purpura, Thrombotic Thrombocytopenic , Humans , Purpura, Thrombotic Thrombocytopenic/diagnosis , Purpura, Thrombotic Thrombocytopenic/therapy , Purpura, Thrombotic Thrombocytopenic/etiology , ADAMTS13 Protein , Consensus , von Willebrand Factor , RecurrenceABSTRACT
Eltrombopag is effective and safe in immune thrombocytopenia (ITP). Some patients may sustain their platelet response when treatment is withdrawn but the frequency of this phenomenon is unknown. We retrospectively evaluated 260 adult primary ITP patients (165 women and 95 men; median age, 62 years) treated with eltrombopag after a median time from diagnosis of 24 months. Among the 201 patients who achieved a complete remission (platelet count >100 × 10(9) /l), eltrombopag was discontinued in 80 patients. Reasons for eltrombopag discontinuation were: persistent response despite a reduction in dose over time (n = 33), platelet count >400 × 10(9) /l (n = 29), patient's request (n = 5), elevated aspartate aminotransferase (n = 3), diarrhea (n = 3), thrombosis (n = 3), and other reasons (n = 4). Of the 49 evaluable patients, 26 patients showed sustained response after discontinuing eltrombopag without additional ITP therapy, with a median follow-up of 9 (range, 6-25) months. These patients were characterized by a median time since ITP diagnosis of 46.5 months, with 4/26 having ITP < 1 year. Eleven patients were male and their median age was 59 years. They received a median of 4 previous treatment lines and 42% were splenectomized. No predictive factors of sustained response after eltrombopag withdrawal were identified. Platelet response following eltrombopag cessation may be sustained in an important percentage of adult primary ITP patients who achieved CR with eltrombopag. However, reliable markers for predicting which patients will have this response are needed.
Subject(s)
Benzoates/administration & dosage , Erythropoiesis/drug effects , Hematinics/administration & dosage , Hydrazines/administration & dosage , Purpura, Thrombocytopenic, Idiopathic/drug therapy , Pyrazoles/administration & dosage , Adult , Aged , Blood Platelets/drug effects , Blood Platelets/pathology , Chronic Disease , Drug Administration Schedule , Drug Monitoring , Female , Follow-Up Studies , Humans , Male , Middle Aged , Platelet Count , Purpura, Thrombocytopenic, Idiopathic/pathology , Purpura, Thrombocytopenic, Idiopathic/surgery , Receptors, Thrombopoietin/agonists , Receptors, Thrombopoietin/genetics , Receptors, Thrombopoietin/metabolism , Recurrence , Remission Induction , Retrospective Studies , Splenectomy , Treatment OutcomeABSTRACT
Drug-induced immune thrombocytopenia may occur secondary to several chemotherapeutic agents or new targeted monoclonal antibodies, but thrombocytopenia induced by trastuzumab is a very rare occurrence. We report a case of severe thrombocytopenia related to the administration of trastuzumab six months after the first exposure.
