2026-07-09 | [Transient acquired thrombotic thrombocytopenic purpura developed during bortezomib, lenalidomide, and dexamethasone therapy for multiple myeloma].

A 59-year-old woman was diagnosed with symptomatic Bence Jones protein, lambda (λ) type, associated with multiple myeloma (BJP-λ MM). Considering her renal dysfunction, she initially received bortezomib and dexamethasone. After renal function improved, she received one cycle of bortezomib, lenalidomide, and dexamethasone (BLD) therapy and achieved stringent complete response. She presented with general fatigue and jaundice before the second cycle of BLD. Laboratory testing revealed: total bilirubin, 4.8 mg/dl; indirect bilirubin, 4.1 mg/dl; lactate dehydrogenase, 978 U/l; hemoglobin, 7.4 g/dl; haptoglobin, <10 mg/dl; platelet count, 23,000/µl; and creatinine, 0.96 mg/dl. A hemogram revealed 1% schistocytes. ADAMTS13 activity was undetectable and the ADAMTS13 inhibitor level was 1.8 Bethesda units. A diagnosis of acquired thrombotic thrombocytopenic purpura (aTTP) was made, and treatment was initiated with prednisolone and rituximab. Plasma exchange was not performed because thrombocytopenia did not progress. The first dose of rituximab restored platelet count, and ADAMTS13 inhibitor became undetectable. The clinical course in this case was consistent with drug-induced aTTP.

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2026-07-09 | Update in treatment options for congenital and immune thrombotic thrombocytopenic purpura.

Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy resulting either from congenital deficiency (cTTP) or acquired (immune) deficiency (iTTP) of A Disintegrin and Metalloprotease with ThromboSpondin-type 1 motif, member 13 (ADAMTS13). Deficiency of ADAMTS13 leads to disseminated platelet thrombosis and organ dysfunction. High mortality of cTTP is prevented by plasma infusion to replace the deficient protease, or more recently by infusion of recombinant ADAMTS13. Standard treatment of iTTP includes steroids, plasma exchange, and rituximab, with or without caplacizumab. Although standard treatment of iTTP improves mortality, refractory cases persist, indicating the need for additional treatment options. This review summarizes the status of novel treatment options for cTTP and iTTP, including additional recombinant ADAMTS13 products, ADAMTS13 gene therapies, plasma cell-directed therapies (bortezomib, daratumumab) as well as novel inhibitors of von Willebrand factor activity.

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2026-07-07 | Postmarketing Safety Concerns With Caplacizumab: A Real-World Pharmacovigilance Study Based on the FDA Adverse Event Reporting System Database.

To analyze data from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) database to characterize the adverse effects of caplacizumab, including associated adverse events (AEs) and safety signals in real-world clinical practice. and obtain evidence to support the rational use of this drug in the treatment of thrombotic thrombocytopenic purpura (TTP). Postmarketing AE reports associated with caplacizumab were retrieved from the FAERS database. Safety signals were identified using four disproportionality analysis methods: the reporting odds ratio, proportional reporting ratio, Bayesian Confidence Propagation Neural Network (BCPNN), and multi-item gamma Poisson shrinker. The Weibull distribution was used to analyze the onset time of AEs, and subgroup analyses were performed according to sex, age group, and reporter type. In total, 1144 reports involving caplacizumab were analyzed. Most AEs were nonserious. Among the 401 reports with documented onset times, 162 (40.4%) described AEs occurring within 7 days of administration. Common bleeding-related events (e.g., epistaxis, gingival bleeding, gastrointestinal hemorrhage, heavy menstrual bleeding, and vaginal hemorrhage) were consistent with product labeling. Decreases in platelet count and in disintegrin and metalloproteinase with thrombospondin type 1 motif 13 (ADAMTS13) activity were interpreted as manifestations of the underlying disease (acquired TTP) rather than drug-induced AEs. Newly identified safety signals included contusion, injection site pain, injection site bruising, injection site erythema, injection site haemorrhage, pruritus and rash. These findings underscore the importance of pharmacovigilance and postmarketing surveillance for caplacizumab. Clinicians should closely monitor bleeding events and injection site reactions during treatment. To ensure the safe use of caplacizumab, the mechanisms underlying these potential signals should be elucidated, and targeted monitoring strategies should be developed.

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