Adzynma (apadamtase alfa /cinaxadamtase alfa) / Takeda, Meiji Seika - LARVOL DELTA

Hereditary TTP and Hemolytic Anemia in Newborns. (PubMed, Pediatrics) - "Two of them receive recombinant ADAMTS13 treatment for their deficiency...We propose a diagnostic algorithm to assist clinicians in neonatal diagnosis of hTTP in hopes of mitigating the need for exchange transfusions and extensive hematologic workups later in life. Timely diagnosis would prompt targeted treatment, which can prevent long-term neurologic and renal sequelae."

Journal • Anemia • Cardiovascular • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

Recombinant ADAMTS13 in congenital thrombotic thrombocytopenic purpura: final analysis from a randomized phase 3 trial. (PubMed, Blood) - P3 | "A ~6-fold increase in ADAMTS13 activity and prolonged time with ADAMTS13 activity of ≥10% was noted in participants receiving rADAMTS13 versus PBT. Consistent with the interim analysis, results with a longer follow-up support the continued clinical benefit of rADAMTS13 prophylaxis in congenital TTP."

Journal • P3 data • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Homeostatic imbalance between von Willebrand factor and ADAMTS13 on hemorrhagic shock-induced endotheliopathy. (PubMed, Haematologica) - "In mice, recombinant ADAMTS13 decreased the adhesive activity and enhanced cleavage of plasma VWF, and improved parameters of lung dysfunction. The present study supports the conclusion that an imbalance between VWF and ADAMTS13 drives, at least in part, the endotheliopathy of trauma and the associated adverse outcomes and suggests a potential therapeutic target for severely injured patients with hemorrhagic shock."

Journal • Hematological Disorders • Pulmonary Disease • Respiratory Diseases

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

Perioperative Optimization and Multidisciplinary Management of Cesarean Delivery in a Patient with Hereditary Thrombotic Thrombocytopenic Purpura (ASA 2026) - "Robust multidisciplinary care was planned preoperatively, although final coordination and recombinant ADAMTS13 logistics required day-of refinement. She underwent uneventful/uncomplicated cesarean delivery with spinal anesthesia."

Clinical • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

A rapid ADAMTS13 activity assay using a portable readout device. (PubMed, Blood Vessel Thromb Hemost) - "Finally, inhibition of recombinant ADAMTS13 printed in a parallel line detects ADAMTS13 antibodies, allowing simultaneous quantification of ADAMTS13 activity levels and detection of ADAMTS13 antibodies. This technology enables rapid quantification of ADAMTS13 activity levels and detection of ADAMTS13 antibodies using a portable device."

Journal • Hematological Disorders • Thrombocytopenic Purpura

Recombinant ADAMTS13 Ameliorates Liver Injury and Improves Hepatic Microcirculation in a Murine Acute-on-Chronic Liver Failure Model. (PubMed, Hepatol Res) - "rADAMTS13 attenuated liver injury in a murine ACLF-like model and was associated with restoration of the ADAMTS13/VWF axis, improved hepatic microcirculation, and increased angiogenesis- and regeneration-related responses. These findings suggest that rADAMTS13 may represent a potential therapeutic approach for ACLF."

Journal • Preclinical • Fibrosis • Hepatology • Immunology • Liver Failure • CD34 • KDR

The Potential Role and Mechanism of RBM47-Mediated Genes in Atherosclerosis. (PubMed, DNA Cell Biol) - "Recombinant ADAMTS-13 was undergoing phase 3 clinical trials...However, the role of RBM47 in other genes that contain AREs and GREs, such as apoA-II, ABCA1, HMGCR, IFN-γ, IL-15, low-density lipoprotein receptor, oxidized low-density lipoprotein receptor 1, and programmed cell death 1 ligand 1, is unclear. This review focuses on the role and mechanism of RBM47-mediated genes in atherosclerosis to provide knowledge for new targets."

Journal • Review • Atherosclerosis • Cardiovascular • Dyslipidemia • Targeted Protein Degradation • ABCA1 • APOA2 • APOB • AXIN1 • CD36 • CD70 • CXCL8 • DKK1 • IFNG • IL10 • IL15 • PD-1 • PD-L1 • SCARB1 • SIGLEC5 • TJP1 • UBR5

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

Targeting VWF-ADAMTS13 dysregulation Prevents Thrombosis and Pulmonary Arterial Remodeling in Chronic Thromboembolic Pulmonary Hypertension (ISTH 2026) - "Aims To investigate the role of the VWF-ADAMTS13 axis in the pathogenesis of a rat model of CTEPH and to determine if recombinant ADAMTS13 reduces the disease burden and mortality...Conclusions We conclude that targeting dysregulation of the VWF-ADAMTS13 axis may represent a novel and promising therapeutic strategy to prevent persistent thrombosis and attenuate pulmonary vascular remodeling and hemodynamic burden in CTEPH. Table or Figure Upload (1) Page 2 Table or Figure Upload (2) Page 3 DOI*10.1016/j.rpth.2026.103629"

Cardiovascular • Hematological Disorders • Pulmonary Arterial Hypertension • Pulmonary Disease • Pulmonary Embolism • Respiratory Diseases • Thrombosis • TGFB1

Switching cTTP patients from plasma to recombinant ADAMTS13 (ISTH 2026) - No abstract available

Clinical

Investigating loss of B cell tolerance in Immune thrombotic thrombocytopenic purpura (ISTH 2026) - "PBMCs were incubated with recombinant ADAMTS13 protein conjugated to APC fluorophore (ADAMTS13- APC) for the identification of ADAMTS13-specific B cells...Conclusions Preliminary findings suggest that acute iTTP patients harbour B cells with enhanced BCR signalling and loss of anergic B ND cells that are ADAMTS13-specific. DOI*10.1016/j.rpth.2026.103949"

IO biomarker • Hematological Disorders • Immunology • Thrombocytopenic Purpura • CD27 • CD73 • NT5E

Functional Recovery of Triple-Alanine ADAMTS13 Mutants via C-Terminal Single-domain Antibody Fusion (ISTH 2026) - "Methods Kinetic and structural properties of rADAMTS13, MDTCSm and two fusion constructs (MDTCSm-aVWF and MDTCSm-L-aVWF, longer linker) were evaluated using FRETS-VWF73 assays and ELISAs with 12 domain- specific monoclonal antibodies (mAbs)...Activity for each construct is normalized as a percentage relative to its respective baseline without plasma. DOI*10.1016/j.rpth.2026.103959"

Hematological Disorders • Thrombocytopenic Purpura

Characterization of the anti-ADAMTS13 immune response in non-idiopathic immune-mediated thrombotic thrombocytopenic purpura. (ISTH 2026) - "Fine epitope mapping of anti-ADAMTS13 IgG was performed by ELISA using recombinant ADAMTS13 domain fragments...Table or Figure Upload (2) Mapping of anti-ADAMTS13 IgG epitopes targeting different domains of ADAMTS13, individualizing each fragment tested, in patients with idiopathic or non-idiopathic iTTP, at diagnosis. DOI*10.1016/j.rpth.2026.103951"

Hematological Disorders • Immunology • Infectious Disease • Thrombocytopenic Purpura

Monitoring ADAMTS13 recovery, conformation, and alloantibodies during plasma and recombinant therapy in cTTP (ISTH 2026) - "Background Prophylactic and on-demand treatment of patients with congenital thrombotic thrombocytopenic purpura (cTTP) is switching from fresh frozen plasma to recombinant ADAMTS13 replacement therapy...Longer follow-up will be essential to clarify the clinical relevance of pre-existing low- titer IgG alloantibodies and to optimize long-term monitoring strategies. DOI*10.1016/j.rpth.2026.103962"

Hematological Disorders • Thrombocytopenic Purpura

N-Glycans: a target for anti-ADAMTS13 antibodies in immune-mediated thrombotic thrombocytopenic purpura (ISTH 2026) - "To investigate the involvement of N-glycans in ADAMTS13 autoantibody binding, complete N-glycans were removed from recombinant ADAMTS13 using PNGase F or terminal sialic acid was removed using Neuraminidase...With previously identified immunogenic hotspots being adjacent to two of the four N-glycans present on ADAMTS13's CS domains, our findings suggest both N-glycans to help shape this predominant immunogenic region of ADAMTS13. DOI*10.1016/j.rpth.2026.103958"

Hematological Disorders • Thrombocytopenic Purpura

A rapid ADAMTS13 activity assay using a portable readout device. (ISTH 2026) - "To demonstrate that the ADAMTS13 lum can also simultaneously detect ADAMTS13 inhibitors within the same sample, parallel microfluidic lines were printed with rADAMTS13 and tested with samples without or with an antibody directed against ADAMTS13...Page 2 Table or Figure Upload (2) Fig.2: Comparison of ADAMTS13 lum to commercial ADAMTS13 assays. DOI*10.1016/j.rpth.2026.103948"

Hematological Disorders • Thrombocytopenic Purpura

Accelerated Clearance of Recombinant ADAMTS13 by Binding Alloantibodies in Congenital TTP: A Case-Based Registry Analysis (ISTH 2026) - "Regular monitoring, including platelet counts, is essential, and frequent rADAMTS13 supplementation may function as an immune tolerance induction strategy. Table or Figure Upload (1) The treatment and laboratory findings in the caes Page 2 Table or Figure Upload (2) Detection of rADAMTS13 binding allo-antibodies DOI*10.1016/j.rpth.2026.103955"

Clinical • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Switch from plasma to recombinant ADAMTS13 in congenital thrombotic thrombocytopenic purpura: the French experience. (ISTH 2026) - "Conclusions The French experience supports rhADAMTS13 as the new therapeutic standard for cTTP and highlights the need for multidisciplinary follow-up to optimize its long-term use and monitoring. DOI*10.1016/j.rpth.2026.103950"

Cardiovascular • Hematological Disorders • Obstetrics • Thrombocytopenic Purpura

Targeting pathogenic VWF/ADAMTS13 dysregulation attenuates CTEPH progression. (PubMed, bioRxiv) - "Collectively, these findings identify dysregulation of the VWF-ADAMTS13 axis as a key driver of pulmonary thrombosis and vascular remodeling in CTEPH and support therapeutic targeting of this pathway as a potential disease-modifying strategy. VWF-ADAMTS13 dysregulation promotes persistent pulmonary thrombosis and platelet-driven arterial remodeling within pulmonary arterioles.Recombinant ADAMTS13 treatment or VWF ablation abrogates pulmonary arterial thrombosis and halts vascular remodeling in CTEPH."

Journal • Cardiovascular • Hematological Disorders • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Pulmonary Embolism • Respiratory Diseases • Thrombosis • TGFB1

SUCCESSFUL TREATMENT OF A PATIENT WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP) WITH RECOMBINANT ADAMTS13 - A CASE REPORT (EHA 2026) - "Summary/Conclusion This is the first Croatian experience with recombinant ADAMTS13, which is very effective and safe for patiens with cTTP. It restores normal hemostasis, prevents TTP relapses, with higher efficacy compared to standard therapy."

Case report • Clinical • Chronic Kidney Disease • CNS Disorders • Cognitive Disorders • Hematological Disorders • Immunology • Infectious Disease • Pneumonia • Pruritus • Renal Disease • Respiratory Diseases • Respiratory Syncytial Virus Infections • Thrombocytopenia • Thrombocytopenic Purpura • Urticaria

EVOLVING MANAGEMENT OF CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA WITH RECOMBINANT ADAMTS13 (EHA 2026) - "Summary/Conclusion Our case highlights the efficacy of recombinant ADAMTS13 as prophylactic therapy. Future studies should focus on optimizing dosing strategies, particularly during high-risk states such as pregnancy, to better define maternal and fetal safety profiles, as well as to compare long-term disease control with FFP-based prophylaxis."

Cardiovascular • Hematological Disorders • Infectious Disease • Thrombocytopenic Purpura • Thrombosis

Acute kidney injury in a patient with congenital thrombotic thrombocytopenic purpura (ERA 2026) - "Patient was referred to hematology to assess qualification for recombinant ADAMTS13 treatment...AKI in patients with congenital TTP can be reversible, without significant long-term impact on kidney function. Why this case is exeptional The case reveals an extraordinary course of AKI in a patient with thrombocytopenia."

Clinical • Acute Kidney Injury • Atypical Hemolytic Uremic Syndrome • Hematological Disorders • Infectious Disease • Nephrology • Renal Disease • Thrombocytopenic Purpura • CRP • HP

A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma (clinicaltrials.gov) - P=N/A | N=50 | Recruiting | Sponsor: Takeda | Not yet recruiting ➔ Recruiting

Enrollment open • Real-world evidence • Hematological Disorders • Thrombocytopenic Purpura