Cablivi (caplacizumab-yhdp) / Sanofi - LARVOL DELTA

Acquired Immune Thrombotic Thrombocytopenic Purpura Following Transaxillary Transcatheter Aortic Valve Replacement: A Rare Life-Threatening Hematologic Emergency. (PubMed, Cureus) - "Treatment with plasma exchange, corticosteroids, caplacizumab, and rituximab led to rapid platelet recovery. The diagnosis of iTTP should be considered in patients presenting with unexplained thrombocytopenia and hemolysis after TAVR, even when asymptomatic. Early recognition and prompt initiation of therapy are essential to improve outcomes."

Journal • Cardiovascular • Heart Failure • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Unintended Interruption of Caplacizumab Therapy Can Compromise Outcomes in Immune-Mediated Thrombotic Thrombocytopenic Purpura: Lessons from Two Clinical Cases. (PubMed, J Clin Med) - "Treatment escalation to PEX and rituximab and reinitiation of continuous caplacizumab administration led to clinical stabilization. Even brief interruptions of caplacizumab therapy may compromise outcomes in iTTP. Continuous administration and secured availability, together with serial monitoring of ADAMTS13 activity to guide treatment duration and discontinuation, are essential to improve patient care."

Journal • Hematological Disorders • Ischemic stroke • Thrombocytopenia • Thrombocytopenic Purpura

Therapeutic Plasma Exchange in Immune-Mediated Thrombotic Thrombocytopenic Purpura: From Cornerstone to Contextualized Therapy. (PubMed, J Clin Med) - "Safe implementation of TPE-free strategies requires experienced centers, rapid ADAMTS13 testing, immediate access to caplacizumab and immunosuppression, careful patient selection, and close clinical and laboratory monitoring. Defining which patients can be treated safely without TPE is a central challenge for future trials and guideline development."

Journal • Review • Hematological Disorders • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Case report: Caplacizumab without plasma exchange for severe thrombotic thrombocytopenic purpura. (PubMed, Front Drug Deliv) - "In addition to rituximab and intravenous immune globulin, caplacizumab therapy was undertaken. This is the first report of caplacizumab use in a Jehovah's Witness patient with TTP with severe neurological compromise to the point of necessitating intubation and with this degree of anemia. With the eventual addition of cyclophosphamide therapy, we observed complete recovery of this critically ill patient without the use of plasma exchange in the treatment course."

Journal • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura • HP

TTP-Like Syndrome and Subsequent Non-Aneurysmal Subarachnoid Hemorrhage in HbSC Disease: A Case Report. (PubMed, Clin Case Rep) - "The patient received red-cell exchange, plasma exchange, caplacizumab, antibiotics, and dialysis, followed by hematologic and renal recovery. Months later, he developed a non-aneurysmal infratentorial subarachnoid hemorrhage (SAH) with obstructive hydrocephalus requiring external ventricular drainage (EVD) and posterior fossa decompression, resulting in severe persistent neurological disability. The case, therefore, highlights the severity of HbSC disease and the challenge of urgent treatment decisions under substantial diagnostic uncertainty when a TMA-like presentation overlaps with other severe SCD complications such as FES."

Journal • Acute Kidney Injury • Cardiovascular • CNS Disorders • Genetic Disorders • Hematological Disorders • Infectious Disease • Nephrology • Pneumonia • Renal Disease • Septic Shock • Sickle Cell Disease • Subarachnoid Hemorrhage • Thrombocytopenia • Thrombocytopenic Purpura • Ventriculomegaly

Postpartum Thrombotic Thrombocytopenic Purpura With Coexisting Preeclampsia With Severe Features (ASA 2026) - "Initial treatment included labetalol, nifedipine, and magnesium. She was treated with plasma exchange, high-dose corticosteroids, rituximab, and caplacizumab with platelet recovery after 7 days. This case highlights TTP as a critical diagnostic consideration in postpartum patients with apparent preeclampsia."

Gynecology • Hematological Disorders • Hypertension • Thrombocytopenia • Thrombocytopenic Purpura • HP

Real-world evolution of therapeutic strategies and outcomes in acquired thrombotic thrombocytopenic purpura: a 14-year referral center experience. (PubMed, Med Clin (Barc)) - "In iTTP, CPZ improves short-term outcomes, particularly when administered early. RTX is associated with a trend toward greater relapse-free survival. Quadruple therapy reduces the time to clinical remission and the number of plasma exchange sessions. In iTTP, the incorporation of targeted and personalized therapeutic advances into clinical practice is associated with an improvement in clinical outcomes."

Journal • Real-world evidence • Hematological Disorders • Thrombocytopenic Purpura

Acquired immune-mediated thrombotic thrombocytopenic purpura with severe cholestatic liver injury and reversible acute kidney injury after ivonescimab-containing chemoimmunotherapy: a case report and literature review. (PubMed, Front Immunol) - "A 63-year-old man with stage IVB lung adenocarcinoma (cT1bN2M1c, with multiple extrathoracic rib metastases) received an ivonescimab-containing combination regimen consisting of three cycles of ivonescimab plus nab-paclitaxel and cisplatin, followed by chemotherapy alone after partial response...Treatment included corticosteroids, 10 sessions of DPMAS/TPE support with transition to FFP-based plasma exchange once iTTP was recognized, and rituximab 500 mg weekly from April 17 to May 8, 2026. Caplacizumab was not used because it was unavailable locally...The renal phenotype should be framed as severe but reversible AKI rather than dialysis-dependent renal failure. In patients receiving PD-1/VEGF-directed combination therapy, thrombocytopenia with microangiopathic hemolysis and organ injury should trigger smear review, PLASMIC scoring, urgent ADAMTS13 testing, and early mechanism-directed therapy."

Journal • Review • Acute Kidney Injury • Hematological Disorders • Hepatology • Liver Failure • Lung Adenocarcinoma • Lung Cancer • Nephrology • Non Small Cell Lung Cancer • Oncology • Renal Disease • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura

Update in treatment options for congenital and immune thrombotic thrombocytopenic purpura. (PubMed, Front Pharmacol) - "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."

Journal • Review • Cardiovascular • Gene Therapies • Hematological Disorders • Thrombocytopenic Purpura • Thrombosis

Emergent Airway Management in Thrombotic Thrombocytopenic Purpura with Seizures and Macroglossia (ASA 2026) - "Treatment with plasmapheresis, rituximab, steroids, and caplacizumab resulted in platelet and renal recovery, followed by complex tongue laceration repair. This case illustrates the challenge of difficult airway management in a post-ictal patient with macroglossia and active bleeding, and reinforces the urgency for prompt recognition of TTP, as delayed treatment is life threatening."

Acute Kidney Injury • CNS Disorders • Epilepsy • Hematological Disorders • Nephrology • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura

Caplacizumab-Enabled Treatment of Immune-Mediated Thrombotic Thrombocytopenic Purpura Without Plasma Exchange: Evidence, Patient Selection, and Practical Considerations. (PubMed, Clin Appl Thromb Hemost) - "However, a subset of patients with inadequate initial platelet responses or complex comorbidities still required rescue TPE.ConclusionTPE-free caplacizumab therapy is a promising, highly targeted strategy, though it should not replace standard therapy outside experienced centers. Safe implementation demands confirmed iTTP, clinical stability, immediate caplacizumab availability, rigorous ADAMTS13 monitoring, adequate immunosuppression, and predefined thresholds for rescue TPE."

Journal • Review • Critical care • Hematological Disorders • Immunology • Thrombocytopenic Purpura

Clinical Spectrum and Differential Diagnosis of Adult Thrombotic Microangiopathies: Real-World Experience from a Tertiary Referral Center. (PubMed, Hematol Rep) - "This study was conducted in a setting where ADAMTS13 activity testing became available only from 2015 onward and complement-targeted therapy (eculizumab) had limited accessibility throughout most of the study period, conditions that shaped both diagnostic classification and treatment outcomes. In this cohort, in the absence of ADAMTS13 testing, TTP was the most frequent subtype, while treatment non-response and renal impairment emerged as the main factors associated with mortality. These findings emphasize the need for early clinical recognition and careful subtype-based differential diagnosis, which will reduce morbidity and mortality by permitting rapid initiation of pathophysiology-based appropriate interventions, i.e., PEx, immune suppression and caplacizumab for immune TTP and anti-complement therapy for aHUS, and limiting the inappropriate use of PEx with its complications, including sepsis."

Journal • Real-world evidence • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Nephrology • Renal Disease • Septic Shock • Thrombocytopenia • Thrombocytopenic Purpura

Undiagnosed Congenital Thrombotic Thrombocytopenic Purpura Presenting as Peripartum Thrombotic Microangiopathy (ISTH 2026) - "Due to clinical urgency, she was treated initially for acquired TTP with plasma exchange, corticosteroids, rituximab, and caplacizumab...She subsequently started on weekly prophylactic recombinant ADAMTS13(40 IU/kg), resulting in sustained hematologic stability and clinical improvement...Conclusions Peripartum TMA should prompt early reflex ADAMTS13 assessment to identify congenital TTP, enabling timely, targeted therapy and improved maternal-fetal outcomes. DOI*10.1016/j.rpth.2026.104738"

Hematological Disorders • Thrombocytopenic Purpura

German S3 TTP guideline (2025) on the diagnosis, treatment, and follow-up of thrombotic thrombocytopenic purpura (TTP)- A summary of recommendations (ISTH 2026) - "Results For first acute episode and relapses of iTTP, the panel made a strong recommendation for adding corticosteroids to therapeutic plasma exchange (TPE), a weak recommendation for adding rituximab and a consensus-based recommendation for adding caplacizumab...For asymptomatic cTTP, the panel made weak recommendation for prophylactic plasma infusion and a neutral recommendation for prophylactic rADAMTS13 infusion...Urgent research needs were identified and presented as well. DOI*10.1016/j.rpth.2026.106207"

Hematological Disorders • Thrombocytopenic Purpura

Recurrent thrombocytopenia in immune thrombotic thrombocytopenic purpura during the caplacizumab era. (PubMed, Br J Haematol) - No abstract available

Journal • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Pharmacodynamics of Caplacizumab in Healthy Volunteers and Phase 2/3 Trial Patients with Immune-mediated Thrombotic Thrombocytopenic Purpura. (PubMed, Thromb Haemost) - P1, P2, P3 | "In a combined analysis of TITAN and HERCULES, median (IQR) change in VWF:RCo activity from baseline to day 2 was 90.2% (119.5, 61.5) in the caplacizumab group and 12.6% (20.0, 33.8) in the placebo group. This post hoc analysis demonstrated the pharmacodynamics of the rapid inhibitory effect of caplacizumab on VWF-platelet interaction (i.e., VWF:RCo suppression < 20%) that does not appear to be impacted by TPE in patients with iTTP."

Clinical • Journal • P2/3 data • PK/PD data • Hematological Disorders • Thrombocytopenic Purpura

Thrombotic Thrombocytopenic Purpura (TTP): A five-year experience at a public tertiary care center in Argentina (ISTH 2026) - "Conclusions Our findings underscore the importance of rapid ADAMTS13 testing in the management of TMA.Although plasma exchange should be initiated even without biochemical confirmation of PTT, the addition of rituximab often depends on this result and improves the survival of these patients. On the other hand, biochemical relapse may precede clinical relapse, so monitoring these patients is key to establishing early treatment, especially in our environment, where drugs such as caplacizumab are not yet available. The observed prevalence and mortality rates highlight the need for specialized centers capable of performing both activity and inhibitor assays to ensure timely intervention and long-term monitoring. Table or Figure Upload (1) Page 2 Table or Figure Upload (2) DOI*10.1016/j.rpth.2026.103972"

Cardiovascular • Hematological Disorders • Thrombocytopenic Purpura • Thrombosis

Post Kidney Transplant Presentation of Acquired Thrombotic Thrombocytopenic Purpura (aTTP): A Case of Pre-existing ADAMTS13 Inhibitors (ISTH 2026) - "Aims To report a rare case of early-onset post-transplant aTTP in a patient with no prior hematological history, highlighting the diagnostic process and the effectiveness of a multimodal therapeutic strategy including Caplacizumab...Standard immunosuppression was initiated with steroids, mycophenolate mofetil, tacrolimus, and anti-thymocyte globulin (ATG)...Treatment commenced with daily plasma exchange (PEX) and Rituximab (RTX, 375 mg/sqm), alongside adjusted baseline immunosuppression to mitigate infectious risk...The retrospective finding of pre-existing inhibitors suggests that the surgical stress or induction therapy may have triggered the clinical manifestation of a previously silent autoimmune state. Page 2 DOI*10.1016/j.rpth.2026.106226"

Clinical • Hematological Disorders • Immunology • Nephrology • Thrombocytopenic Purpura • Transplantation • HP

Korean expert consensus on the management of thrombotic thrombocytopenic purpura. (PubMed, Korean J Intern Med) - "Although effective treatments, such as therapeutic plasma exchange, immunosuppressive therapy with rituximab, and caplacizumab, have significantly improved survival, important challenges remain. This consensus statement provides updated recommendations for diagnostic approaches, initial and adjunctive therapies, management of refractory disease, ADAMTS13 monitoring, and long-term follow-up. By integrating international evidence with local circumstances, this document aims to provide Korean clinicians with practical, upto-date guidance to enhance the routine care of patients with TTP."

Clinical guideline • Journal • Review • Cardiovascular • Hematological Disorders • Thrombocytopenic Purpura • Thrombosis

Severe Immune-Mediated Thrombotic Thrombocytopenic Purpura With High-Titer ADAMTS13 Inhibitors: A Case Report. (PubMed, Cureus) - "The patient received therapeutic plasma exchange, corticosteroids, caplacizumab, and rituximab, resulting in rapid hematologic remission. An extensive diagnostic workup excluded alternative thrombotic microangiopathies. This case highlights the importance of early clinical recognition, systematic exclusion of alternative thrombotic microangiopathies, and prompt initiation of multimodal therapy in patients with suspected iTTP. It also illustrates the potential value of serial monitoring of ADAMTS13 activity and inhibitor titers as complementary biomarkers for assessing immunologic response and guiding therapeutic management."

Journal • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Management of Immune TTP without Therapeutic Plasma Exchange in Severe Episodes: Subgroup Analysis of the ATMAR and REACT-2020 Cohorts (ISTH 2026) - "Caplacizumab was used frontline in 98.6%, rituximab in 91.8%, and corticosteroids in all patients. P values were calculated using the log-rank test. Page 3 DOI*10.1016/j.rpth.2026.103794"

Hematological Disorders • Thrombocytopenic Purpura

Plasma Exchange in Patients with Thrombotic Thrombocytopenic Purpura: A Single-Center Retrospective Case Series (ISTH 2026) - "Rituximab was administered in 42.8% of patients, mainly for relapsed or refractory disease...Limited access to caplacizumab may contribute to longer treatment duration and increased complications. Expanded access to targeted therapies is essential to improve outcomes and reduce reliance on intensive plasma exchange. DOI*10.1016/j.rpth.2026.103969"

Retrospective data • Hematological Disorders • Septic Shock • Thrombocytopenic Purpura

Immune-Mediated Thrombotic Thrombocytopenic Purpura in a Patient With Systemic Lupus Erythematosus and Recent Epstein-Barr Virus Infection: A Diagnostic Challenge. (PubMed, Cureus) - "Progressive microangiopathic hemolytic anemia with worsening schistocytosis and a high PLASMIC score prompted urgent initiation of plasma exchange before ADAMTS13 confirmation. The patient was successfully treated with plasma exchange, corticosteroids, rituximab, and caplacizumab."

Journal • Autoimmune Hemolytic Anemia • Epstein-Barr Virus Infections • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus • Thrombocytopenia • Thrombocytopenic Purpura

Thrombotic Thrombocytopenic Purpura: Clinical Presentation, Treatment Strategies, and Outcomes in a Five-Year Single-Center Case Series (ISTH 2026) - "Upon diagnostic suspicion, all patients initiated corticosteroids, therapeutic plasma Exchange and, once diagnosis was confirmed, rituximab. Early integration of caplacizumab into the treatment regimen significantly reduces mortality and shortens the duration of hospitalization. DOI*10.1016/j.rpth.2026.105361"

Clinical • Gynecology • Hematological Disorders • Thrombocytopenic Purpura

Postmarketing Safety Concerns With Caplacizumab: A Real-World Pharmacovigilance Study Based on the FDA Adverse Event Reporting System Database. (PubMed, Pharmazie) - "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."

Adverse events • Journal • P4 data • Real-world evidence • Dermatology • Hematological Disorders • Pain • Pruritus • Thrombocytopenic Purpura