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Abstract Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare disease and has occasionally been described after vaccination, especially against viral agents. We present a case of a patient who presents with the classic pentad of TTP a few days after receiving the first dose of the mRNA Pfizer COVID‐19 vaccine. To our knowledge, this is the second report of a de novo TTP following mRNA Pfizer COVID‐19 vaccination.

Acquired thrombotic thrombocytopenic purpura (aTTP) is a life-threatening microangiopathy usually characterized by microangiopathic hemolytic anemic, thrombocytopenia, and end-organ ischemia associated with disseminated microvascular platelet-rich thrombi and severe deficiency (activity

BACKGROUND: In Saudi Arabia (KSA) and the United Arab Emirates (UAE), only limited epidemiological data and treatment guidance exist for acquired thrombotic thrombocytopenic purpura (aTTP), a rare, life-threatening blood disorder. AIMS: Expert insights from KSA and UAE were used to obtain local epidemiological data, to characterize current disease management and unmet needs, and to formulate recommendations for the improvement of the diagnosis and treatment of aTTP. Costs and socioeconomic burden were a secondary focus. METHODS: Hematologists from KSA and UAE with clinical experience in the diagnosis and management of aTTP individually answered questions on the burden and management of aTTP via an online survey. Based on these insights, a draft consensus was discussed and refined jointly by the hematologists in a live session for each country. Payers provided information on the economic burden and cost of aTTP management. RESULTS: The experts estimate the incidence of aTTP to 5–6 (KSA) and 1–2 (UAE) per 1,000,000 person-years and anticipate it increasing. Most of the presenting patients are aTTP-naive. Recurrent disease is rare. Diagnosis of aTTP should involve ADAMTS13 activity testing. Plasma exchange and immunosuppression are the current standard of care. Key unmet needs include a lack of awareness of aTTP, access to rapid testing and novel treatments to improve outcomes. CONCLUSION: The expert consensus to address the unmet needs and improve aTTP outcomes include increasing aTTP awareness and access to ADAMTS13 testing; the development of national guidelines; and, additionally, strategies to improve patients' long-term quality of life.

Abstract Background Idiopathic membranous nephropathy (MN) is one of the major glomerulonephritis that cause nephrotic syndrome. The phospholipase A2 receptor (PLA2R) has recently been identified as an endogenous antigen of idiopathic MN. Thrombotic thrombocytopenic purpura (TTP) is a disorder characterized by schistocytes, hemolytic anemia, thrombocytopenia, and organ dysfunction which occurs as a result of thrombi. Patients with acquired TTP have autoantibodies against a disintegrin and metalloprotease with thrombospondin type 1 motif 13 (ADAMTS13). These autoantibodies act as an inhibitor and cause ADAMTS13 deficiency. Idiopathic MN and acquired TTP are usually considered as independent autoimmune diseases. We experienced a patient who developed TTP during the conservative treatment of idiopathic MN, with the coexistence of ADAMTS13 inhibitor and anti-PLA2R antibody. Case presentation A 73-year-old man presented with thrombocytopenia, hemolytic anemia, disturbance of consciousness, and acute kidney injury after 4-year course of biopsy-proven idiopathic MN. ADAMTS13 activity was undetectable and the ADAMTS13 inhibitor was identified. Additionally, he was positive for anti-PLA2R antibody. The patient did not have any diseases that could cause secondary thrombotic microangiopathy, and he was diagnosed with acquired TTP. Steroid therapy and plasma exchange were initiated and the acquired TTP resolved. MN achieved remission 3 months after the anti-PLA2R antibody disappeared. Conclusions This is the first reported case of acquired TTP developed during conservative treatment of idiopathic MN, with both ADAMTS13 inhibitor and anti-PLA2R antibody positive at the onset of the TTP. The present case suggests that idiopathic MN might be associated with the development of some cases of acquired TTP.

Abstract Background Thrombotic thrombocytopenic purpura (TTP) is associated with more deleterious outcomes in patients with systemic lupus erythematosus (SLE). However, ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) levels and ADAMTS13 inhibitor were not routinely assayed in most previous studies. The objective of this study is to compare the characteristics and outcomes of immune-mediated TTP (iTTP) in patients with and without SLE. Methods The medical data of 28 patients with iTTP from Peking Union Medical College Hospital were analysed. ADAMTS13 activity and ADAMTS13 inhibitor were measured in all patients. Results All 28 patients had ADAMTS13 inhibitor and severe ADAMTS13 deficiency. iTTP was considered SLE-related (SLE-TTP) in 10 patients and primary (primary iTTP) in 18 patients. Renal involvement on presentation was more severe in patients with primary iTTP as determined by higher serum creatinine (162.7 ± 110.6 vs 73.3 ± 13.4 μmol/L, p

Tugrul Elverdi, Ahmet Emre Eskazan Division of Hematology, Department of Internal Medicine, Cerrahpasa Faculty of Medicine, Istanbul University-Cerrahpasa, Istanbul, Turkey Abstract: Thrombotic thrombocytopenic purpura (TTP) is a rare disease with a mortality rate of over 90% if left untreated. Therapeutic plasma exchange (PEX) is the mainstay of treatment of acquired TTP (aTTP), and with the introduction of PEX, the mortality rate declined dramatically below 20%. Although PEX together with corticosteroids are the backbone of the upfront management of patients with aTTP with successful outcomes, patients may remain refractory and/or relapse following an initial response to this treatment. There are some therapeutic options, which can be used among these patients, helping in improving outcomes of aTTP. Caplacizumab (formerly ALX-0081 or ALX-0681) is a humanized single-variable domain immunoglobulin that recognizes the human von Willebrand factor (vWF) A1 domain and inhibits the vWF–platelet glycoprotein 1b-alpha (GP1b-α) interaction. The drug was first developed for the prevention of thrombosis in high-risk patients with acute coronary syndrome undergoing percutaneous coronary intervention; however, drug development for this indication has been discontinued. Recently, caplacizumab received its first approval following Phase II TITAN and Phase III HERCULES trials in the European Union (EU) for the treatment of acute episode of aTTP in adult patients, in addition to PEX and immunosuppression. This review focuses on the use of caplacizumab as an emerging treatment option in patients with aTTP. Keywords: acquired thrombotic thrombocytopenic purpura, ADAMTS13, aTTP, caplacizumab, ultra-large von Willebrand factor multimers