Cablivi (caplacizumab-yhdp) / Sanofi - LARVOL DELTA

EASIX, A NEW TOOL TO PREDICT RESPONSE AND REFRACTORINESS IN IMMUNE THROMBOTIC THROMBOCYTOPENIC PURPURA? (EHA 2025) - "All received corticosteroids and plasma exchange (PE), with or without caplacizumab and/or rituximab. We propose EASIX as a possible marker for monitoring endothelial damage in iTTP. EASIX normalization by day 7, prior to ADAMTS13 recovery (day 21), may allow earlier monitoring of treatment responses. Additionally, clinical relapses triggered a significant increase in EASIX, while biochemical relapses and isolated ADAMTS13 decreases did not, suggesting that endothelial activation may not correlate with isolated ADAMTS13 declines."

IO biomarker • Acute Graft versus Host Disease • Anemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Myelodysplastic Syndrome • Oncology • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

AI-BASED RISK SCORE FOR THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP): ENHANCING DIAGNOSTIC ACCURACY AND TREATMENT OPTIMIZATION. (EHA 2025) - "ISTH guidelines also recommend to consider the treatment with caplacizumab, prior to the diagnostic confirmation, if there is a very high pretest probability of TTP (positive predictive value (PPV)>90%).Two risk scores (PLASMIC and French) are commonly used to assess pretest probability of TTP... This model provides a robust, data-driven approach for early risk stratification in TMA. By reliably excluding low-risk patients it allows detection of most TTP cases prior to ADAMTS13 testing (S=96.6%). In a subsequent step, the model accurately identifies high-risk patients with 95.7% PPV, exceeding the ISTH recommendations (>90%)."

Hematological Disorders • Thrombocytopenic Purpura

Caplacizumab treatment for acquired thrombotic thrombocytopenic purpura: single centre experience (ISTH 2025) - "They were successfully treated with rituximab or cyclophosphamide. All these patients achieved clinical remission and received further administrations of caplacizumab."

Clinical • Acute Kidney Injury • Anemia • Hematological Disorders • Nephrology • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura

Recovery of ADAMTS13 activity in immune thrombotic thrombocytopenic purpura: a retrospective study (ISTH 2025) - "On the other hand, an additive effect was observed for time to recovery of ADAMTS13 activity which increased from the cases with low levels of anti-ADAMTS13 IgG and not treated with caplacizumab to those with high levels of anti-ADAMTS13 IgG and treated with caplacizumab (Figure 1B). Table or Figure Upload"

Retrospective data • Hematological Disorders • Thrombocytopenic Purpura

An atypical presentation of acquired thrombotic thrombocytopenia (ISTH 2025) - "The patient was treated with PEX for 5 days, prednisone and caplacizumab for 33 days. Metastatic prostate cancer was diagnosed, the patient was treated with hormonal castration, achieving biochemical remission (PSA 2ng/mL). There was no recurrence of aTTP."

Acute Kidney Injury • Anemia • Genito-urinary Cancer • Oncology • Prostate Cancer • Renal Disease • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • HP

Effectiveness of adjunctive caplacizumab treatment in immune thrombotic thrombocytopenic purpura (ISTH 2025) - "Contrary, it could lead to more frequent early relapse due to premature PEX suspension and use of rituximab. Newly diagnosed iTTP patients had significantly higher inhibitor levels compared to relapse episodes (63 vs 34 U/L, p=0.045), and shorter hospitalization (10 vs 12 days, p=0.03), probably due to more CAPLA patients (30% vs 18%). Table or Figure Upload"

Hematological Disorders • Thrombocytopenic Purpura

Von Willebrand factor/ADAMTS13 fluctuations during the immune TTP follow-up (ISTH 2025) - "These trends were also noted in a woman with exacerbation and relapse, but not in two patients treated with caplacizumab (Fig.1C). Table or Figure Upload"

Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Management of immune TTP without plasma exchange: lessons learned from the Austrian experience (ISTH 2025) - "Methods Immunosuppression with steroids and rituximab was started upfront, and caplacizumab continued daily until ADAMTS13 increases >20%. Three patients had acute viral infections (cytomegalovirus, human immunodeficiency virus, hepatitis B), one had received 3 units of platelet concentrate before TTP was recognized and one had immune thrombocytopenia. Up to 3 sessions of TPE were required to normalize platelet counts."

Cardiovascular • Cytomegalovirus Infection • Hematological Disorders • Hepatitis B • Hepatology • Human Immunodeficiency Virus • Immune Thrombocytopenic Purpura • Infectious Disease • Inflammation • Thrombocytopenia • Thrombocytopenic Purpura

Clinical Response and Refractoriness in Immune Thrombotic Thrombocytopenic Purpura (ISTH 2025) - P | "The anti-VWF nanobody caplacizumab is effective in rapidly terminating fatal thrombotic microangiopathyDespite frontline use of caplacizumab, prolonged times to platelet count recovery are occasionally observed in clinical practice, potentially indicating refractory iTTP...Differential analysis of time to platelet count doubling and time to platelet count normalization in patients based on the presence of concomitant diseases demonstrates a clear effect on the latter outcome parameter (Figure 2). Table or Figure Upload"

Clinical • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Thrombocytopenic Purpura • HP

Caplacizumab and immunosuppression without plasma exchange in thrombotic thrombocytopenic purpura (ISTH 2025) - No abstract available

Late-breaking abstract • Hematological Disorders • Thrombocytopenic Purpura

Therapeutic effects of a novel humanized anti-GPIb-alpha Fab fragment in a murine model of TTP (ISTH 2025) - "Current treatment for immune TTP includes daily therapeutic plasma exchange (TPE), caplacizumab (anti-VWF nanobody), and immunosuppressives (such as corticosteroids and rituximab). Treatment for hereditary TTP includes frequent plasma infusion, or factor VIII concentrate, or recombinant ADAMTS13...More importantly, CA1001 appeared to be efficacious in preventing and/or treating a histone-induced “TTP-like” syndrome in Adamts13-/- mice, demonstrated by the alleviation of thrombocytopenia, prerenal injury, and formation of microvascular thrombosis in major organ tissues in the treated group compared with those in the vehicle controls. Table or Figure Upload"

Preclinical • Cardiovascular • Hematological Disorders • Inflammation • Ischemic stroke • Myocardial Infarction • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Novel dual-action fusion proteins combining truncated ADAMTS13 and a nanobody against VWF (ISTH 2025) - "Possible treatments of TTP include recombinant ADAMTS13 or caplacizumab, which inhibits the GP1bα-VWF interaction. Both MDTCS-Syn-VWFA1 and MDTCS-RARAA-Syn-VWFA1 effectively inhibited ristocetin-activated VWF binding to platelets and prevented platelet agglutination, while ADAMTS13, MDTCS and MDTCS-RARAA had no effect. In the beads-on-a-string assay, all constructs showed dose-dependent reduction in the length and number of platelet stings on HUVECs, with MDTCS-RARAA-Syn-VWFA1 proving more effective than MDTCS-RARAA."

Hematological Disorders • Thrombocytopenic Purpura

Relapse and beyond: Navigating the long-term clinical impacts of immune thrombotic thrombocytopenic purpura. (PubMed, Transfus Apher Sci) - "While more recent advances in the use of immunotherapy (e.g. rituximab and caplacizumab) have significantly improved acute survival and short-term exacerbation/relapse prevention, long-term complications of the disease remain a concern for survivors. Providers should possess heightened awareness of long-term complications and atypical manifestations of relapse in survivors. We advocate for further research and observational cohort studies to formulate standardized guidelines for surveillance and intervention to mitigate the chronic burden of iTTP."

Journal • Review • Alzheimer's Disease • Cardiovascular • CNS Disorders • Cognitive Disorders • Depression • Hematological Disorders • Hypertension • Mood Disorders • Psychiatry • Rare Diseases • Thrombocytopenic Purpura

Excitement builds for ISTH 2025 Congress in Washington, D.C. as late-breakthrough abstracts are announced (PRNewswire) - "The International Society on Thrombosis and Haemostasis (ISTH) prepares to welcome nearly 5,000 attendees from the global thrombosis and hemostasis community to the ISTH 2025 Congress in Washington, D.C....A key highlight of the ISTH 2025 Congress, taking place from June 21-25, 2025, is the presentation of late-breakthrough abstracts, offering new data from major clinical trials and breakthrough studies, giving attendees a first look at transformative findings."

Clinical data • Hemophilia A • Immune Thrombocytopenic Purpura • Venous Thromboembolism

Clinical Response and Refractoriness in Immune Thrombotic Thrombocytopenic Purpura (ERA 2025) - "iTTP refractoriness is extremely rare in caplacizumab-treated patients. Observation of prolonged times to platelet recovery should prompt further diagnostic workup to identify concomitant diseases and factors potentially counterfeiting uncontrolled iTTP, instead of primarily intensifying iTTP treatment."

Clinical • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Hematological Disorders • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Thrombocytopenic Purpura • HP

Thrombotic Thrombocytopenic Purpura Without Neurological Involvement: A Case Report and Review of the Diagnostic and Treatment Strategies. (PubMed, Cureus) - "Timely investigation and treatment not only saved her life but also reduced end-organ damage. This report provides an overview of the acute presentation of TTP, positive clinical findings, investigations, diagnosis, treatment, and post-treatment outcomes."

Journal • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Hematological Disorders • Nephrology • Thrombocytopenic Purpura

Critical care victory: saving a life from multi-organ failure in a life-threatening case of thrombothic thrombocytopenic purpura (TTP) (Euroanaesthesia 2025) - "Rituximab, PEX, corticosteroids may be useful in treatment. In resistant cases, caplacizumab can be life-saving(1)."

Clinical • Anemia • Cardiovascular • CNS Disorders • Critical care • Gastrointestinal Disorder • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Metabolic Disorders • Psychiatry • Schizophrenia • Thrombocytopenia • Thrombocytopenic Purpura • HP

The TMA team and TTP pathway improved outcomes in a cohort with Thrombotic thrombocytopenic purpura. (PubMed, PLoS One) - "All patients had acquired TTP. TTP-RRD at 90 days was reduced from 69% with usual care to 6% with Pathway care (95% CI 0.35 to 0.90, P = 0.0004), a relative risk reduction of 91%; TTP relapse alone at 90 days was reduced from 62% to 0% (95% CI 0.36 to 0.88, P = 0.0002) with Pathway care. The number needed to treat to prevent TTP-RRD was 1.59 at 90 days. Over the project duration usual care demonstrated a hazard ratio for TTP-RRD of 12.58 compared to Pathway care. With the intervention, the duration of TPE was increased (median 6 vs 12 sessions, P < 0.05), as was use of rituximab (31.3% vs 93.8%, 95% CI -0.36 to -0.88, P = 0.003), and caplacizumab (6.3% vs 62.5%, 95% CI -0.027 to -0.81, P = 0.001). All Pathway patients underwent biochemical surveillance, and 31% had pre-emptive rituximab to reduce possibility of clinical relapse. A structured TTP Pathway significantly reduces morbidity and aligns care with modern clinical guidelines. The TMA Team is a valuable..."

Journal • Retrospective data • Hematological Disorders • Thrombocytopenic Purpura

TARGETING VON WILLEBRAND FACTOR WITH CAPLACIZUMAB TO REDUCE THROMBO-INFLAMMATION IN ACUTE ISCHEMIC STROKE (ESOC 2025) - "Our findings suggest that vWF plays a critical role in DMT during AIS. Caplacizumab represents a promising therapeutic option for reducing DMT during AIS, through its anti-thrombotic and anti-inflammatory effect."

Cardiovascular • Ischemic stroke

Resistant thrombotic thrombocytopenic purpura successfully treated with caplacizumab preceding acute myeloid leukemia. (PubMed, Pol Arch Intern Med) - No abstract available

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Thrombocytopenic Purpura

Crystals and Crisis: A Rare Case of Cocaine-induced Thrombotic Thrombocytopenic Purpura (ATS 2025) - "Case: A 47-year-old male with history of polysubstance use disorder (Cocaine, alcohol ) on methadone presented to the emergency room after being found agitated and combative on the roadside...Blood cultures grew MSSA and E. coli, with positive toxicology for fentanyl and cocaine...He was timmediately started on plasmapheresis, rituximab, and high-dose methylprednisolone, leading to clinical improvement and discharge...Plasmapheresis combined with high-dose steroids remains the cornerstone of treatment, with caplacizumab shown to reduce ICU stay from 10 to 3 days. Empirical initiation of plasmapheresis can substantially improve outcomes, with the PLASMIC score serving as a valuable tool in guiding therapeutic decisions."

Clinical • Anemia • Gastroenterology • Hematological Disorders • Renal Disease • Thrombocytopenic Purpura • HP

Management of STEMI and Cardiogenic Shock Secondary to Thrombotic Thrombocytopenic Purpura (ATS 2025) - "Shock Team was activated, and the patient was started on milrinone and transferred to a tertiary center for consideration of VA ECMO. Due to invasiveness and risk VA ECMO was deferred and the patient completed 5 courses of plasmapheresis and started on Caplacizumab...Cases report that thrombosis led to arrhythmia and cardiac arrest or coronary occlusion led to cardiogenic shock and mechanical failure prior to arrest. It is unclear if prior myocardial injury, comorbidities or timing of initial treatment alter the morbidity or mortality of these patients but early recognition of disease and transfer to facilities capable of mechanical assistance may play a pivotal role."

Anemia • Cardiovascular • Cognitive Disorders • Congestive Heart Failure • Fibrosis • Gastrointestinal Disorder • Heart Failure • Hematological Disorders • Hypotension • Immunology • Myocardial Infarction • Nephrology • Renal Disease • Thrombocytopenic Purpura • Thrombosis • HP

Immune Thrombotic Thrombocytopenic Purpura: A Review. (PubMed, JAMA) - "Addition of caplacizumab shortens time to normalization of platelet count and reduces recurrences while receiving the drug but increases bleeding risk. Monitoring ADAMTS13 activity in survivors and initiation of rituximab for those with low ADAMTS13 activity reduces the risk of clinical relapse."

Journal • Cardiovascular • CNS Disorders • Epilepsy • Hematological Disorders • Myocardial Infarction • Pain • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

A Case Series of Three Pediatric Patients With Refractory TTP and the Novel use of Daratumumab (ASPHO 2025) - "He therefore completed a second course of steroids and mycophenolate mofetil (MMF) was added...Case 2: A 16-year-old female developed acquired TTP, which was initially treated with high dose steroids, plasma exchange, rituximab, MMF, and caplacizumab...He received multiple therapies for HLH, including anakinra, high dose steroids, and etoposide... These three cases highlight the challenges of managing refractory TTP in children and the subsequent immune system activation that can occur, as shown by the associated HLH diagnosis in Case 3. Therefore, treating refractory TTP may warrant the use of adjunct novel immunosuppressive agents, such as daratumumab. Nonetheless, given the rarity and complexity of TTP, further research is needed to better understand the efficacy and safety of daratumumab and other therapies in pediatric populations."

Clinical • IO biomarker • Cardiovascular • CNS Disorders • Epilepsy • Glomerulonephritis • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Hypertension • Immunology • Pediatrics • Rare Diseases • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura

Early caplacizumab and obinutuzumab enable successful treatment of relapsing thrombotic thrombocytopenic purpura without therapeutic plasma exchange: a case report. (PubMed, Front Immunol) - "We firstly report a 55-year-old female with a history of relapsing TTP who was managed exclusively with caplacizumab, steroids and the second generation fully humanized anti-CD20 monoclonal antibody obinutuzumab during a relapse without the need of TPE throughout the whole disease course. This case illustrates the safety and effectiveness of a TPE-free TTP management using prompt initiation of caplacizumab and obinutuzumab."

Journal • Hematological Disorders • Thrombocytopenic Purpura