Soliris (eculizumab) / AstraZeneca - LARVOL DELTA

OUTCOME OF SENSITIZED HEART-KIDNEY TRANSPLANT PATIENTS (WRMC 2026) - "Immunosuppression medications including ATG, eculizumab, rituximab, obinutuzumab, and IVIG were collected for all patients. Sensitized patients undergoing heart-kidney transplantation appear to have less rejection in the heart while other post-transplant outcomes are comparable. Adjustments to lower immunosuppression may be considered for these patients."

Clinical • Antibody-mediated Rejection • Cardiovascular • Congestive Heart Failure • Heart Failure • Immunology • Myocardial Infarction • Transplant Rejection • Transplantation

Treatment Pathways, Switching, and Barriers to Disease-Modifying Therapy in Hispanic Cohort of NMOSD (ACTRIMS Forum 2026) - "Disease-modifying therapy (DMT) exposures (rituximab, eculizumab, inebilizumab, ravulizumab, satralizumab, azathioprine, mycophenolate, interferons, glatiramer), reasons for discontinuation/switch, adherence/logistics, and EDSS trajectories were compiled. ResultsPatients cycled through a median of two DMTs (range: 1-4) before stabilization. In this largely Hispanic cohort, NMOSD treatment pathways were shaped by barriers as much as biology. Logistics failures, intolerance, and drug failure were the main reasons for switching therapies. Strikingly, nearly half of inebilizumab-treated patients experienced breakthrough relapses, diverging from trial outcomes and signaling a need for close monitoring and timely escalation."

CNS Disorders • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder

Bilateral Thalamic Lesion: An Extremely Rare Presentation of Neuromyelitis Optica Spectrum Disorder (ACTRIMS Forum 2026) - "Tocilizumab was initiated for relapse prevention; however, after three doses, she developed intractable headache, vomiting, and encephalopathy...Her course was complicated by pneumonia, Clostridioides difficile infection, and paroxysmal sympathetic hyperactivity, managed with clonidine and diazepam. She was subsequently transitioned to eculizumab for long-term relapse prevention... This case describes a rare pediatric presentation of NMOSD with internal capsule and sequential bilateral thalamic involvement, initially presenting with hemiplegia and mimicking both stroke and neoplasm, later complicated by intractable paroxysmal sympathetic hyperactivity. While thalamic volume loss and nuclear atrophy are more characteristic of multiple sclerosis, thalamic involvement in NMOSD is rare and typically affects nuclei associated with inflamed pathways—such as the lateral geniculate and ventral posterior nuclei—secondary to optic neuritis or transverse myelitis. Our patient,..."

Cardiovascular • CNS Disorders • Infectious Disease • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pneumonia • Rare Diseases • Respiratory Diseases

Disease burden and treatment patterns of paroxysmal nocturnal hemoglobinuria in Japan: a real-world survey. (PubMed, Int J Hematol) - "Among C5i-treated patients (n = 39), 51.3% received ravulizumab and 48.7% eculizumab, for a median (IQR) duration of 1.6 (1.0, 2.7) years. Mean (SD) EQ-5D-5L and FACIT-Fatigue scores were 0.73 (0.16) and 32.3 (7.1). Even C5i-treated patients continued to experience substantial disease burden, highlighting the need for more effective treatments to improve quality of life."

Journal • Real-world evidence • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Comparative Clinical Effectiveness Trial of Rituximab versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab to Prevent Relapses in Neuromyelitis Optica Spectrum Disorder (NMOSD) (ACTRIMS Forum 2026) - "BEST-NMOSD is the first comparative effectiveness trial comparing all approved NMOSD DMTs to rituximab. The primary endpoint combines efficacy and safety, allowing for a holistic comparison of treatments and reflecting real-world decisions. This study will deliver actionable results for patients, physicians, and regulatory authorities."

Clinical • CNS Disorders • Depression • Inflammation • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Ophthalmology • Optic Neuritis • Rare Diseases

MANAGEMENT OF TRANSPLANT‑ASSOCIATED THROMBOTIC MICROANGIOPATHY IN CHILDREN AFTER HSCT WITH RAVULIZUMAB: SINGLE CENTER EXPERIENCE (EBMT 2026) - "GvHD prophylaxis included ATG, rituximab, abatacept and tacrolimus or ruxolotinib in all cases...All patients received eculizumab (median no... TA-TMA is still challenging complication of HSCT. Use of Ravulizumab in refractory cases is promising strategy in pediatric patients. Future multicenter studies required for estimation of safety and efficacy of Ravulizumab in real-world practice."

Clinical • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation • Transplantation Associated Thrombotic Microangiopathy

KIDNEY TRANSPLANTATION FOLLOWING HSCT, COMPLICATED BY TA-ТМА WITH PROGRESSION TO END-STAGE RENAL DISEASE (EBMT 2026) - "Current therapy consisted of abatacept and continued eculizumab.Pathological examination of the removed kidney revealed diffuse global glomerulosclerosis, severe interstitial fibrosis, and C3d deposition in the glomeruli, consistent with advanced CKD caused by a combination of TA-TMA and viral nephritis.At the two-month follow-up, the patient remains off systemic immunosuppressive therapy with excellent kidney graft function. Kidney transplantation from a hematopoietic stem cell donor became a necessary step in the therapy of a patient with ESRD following HSCT. The presence of full donor chimerism prior to kidney transplantation allowed to minimize the volume of induction therapy before the solid organ transplant. The use of regulatory T-cells was considered as an additional option, aimed both at controlling GVHD and preventing kidney allograft rejection while promoting immune tolerance."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Kidney Disease • Cytomegalovirus Infection • Fibrosis • Genetic Disorders • Glomerulonephritis • Graft versus Host Disease • Immunology • Infectious Disease • Nephrology • Primary Immunodeficiency • Renal Disease • Respiratory Diseases • Solid Organ Transplantation • Transplant Rejection • Transplantation • Transplantation Associated Thrombotic Microangiopathy • CD34

Eculizumab Is Associated with Significantly Lower Adenovirus Clearance in Children after Hematopoietic Cell Transplantation (TCT-ASTCT-CIBMTR 2026) - " Among 25 children with adenoviremia, the first adenovirus PCR was detected a median of 62 days post-HCT, Treatment approaches included cidofovir (76%), reduction of immune suppression (32%), viral specific T cells (20%), and brincidofovir (8%)- all ecu exposed patients received ≥1 treatment and 50% of non-ecu exposed received ≥1 treatment...In bivariable models adjusting for alemtuzumab (HR 0.26, p=0.02), abatacept (HR 0.14, p=0.001), post-HCT cyclophosphamide (HR 0.28, p=0.02), grade 3-4 acute GVHD (HR 0.20, p=0.02), and absolute lymphocyte count at first adenovirus PCR (HR 0.29, p=0.02), adenovirus clearance remained significantly lower in than those exposed to ecu... In this single center retrospective study, eculizumab exposed children were 3 times less likely to clear adenovirus and 6 times more likely to die of adenovirus related mortality. While these findings need to be validated in larger cohorts, our findings merit a careful risk/benefit analysis prior to..."

Clinical • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Transplantation • Transplantation Associated Thrombotic Microangiopathy

Complement involvement in antiphospholipid syndrome. (PubMed, Immunol Lett) - "Several cases report described the use of eculizumab, an anti-C5 monoclonal antibody, to treat severe forms of APS (recurrent thrombosis, Catastrophic APS) but these studies are not sufficient and need to be more standardized. C4d measurement may be useful to assess classical and lectin pathways activation, C5a may allow evaluating the C5a/C5aR axis activity whereas, associated with sC5b9, it may also assess the terminal pathway activation but also the therapeutic efficacy of complement blocking molecules. Thus, assessment of good complement biomarkers and their kinetics needs to be done to determine personalized therapeutic options."

Journal • Review • Cardiovascular • Genetic Disorders • Hematological Disorders • Thrombosis

Paroxysmal Nocturnal Hemoglobinuria in a Young Adult Woman: A Representative Case of Recurrent Intravascular Hemolysis. (PubMed, Cureus) - "Eculizumab therapy was initiated, resulting in the resolution of hemoglobinuria and improvement in hemoglobin levels and symptoms. This case highlights the importance of considering PNH in patients with Coombs-negative hemolytic anemia and recurrent dark urine. Early recognition and timely complement inhibition are essential to reducing hemolysis, preventing thrombosis, and optimizing long-term outcomes."

Journal • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis • HP

A Decade of Precision: PK/PD-Guided Eculizumab Therapy for TA-TMA Management (TCT-ASTCT-CIBMTR 2026) - "Achieving survival gains exceeding four-fold improvement with a median of only eight doses per patient exemplifies how collaborative, data-driven dosing can "do more with less." This model highlights pharmacists as essential partners in precision biologic therapy, integrating science, stewardship, and patient-centered care in complex transplant populations. Evaluate eculizumab utilization and dosing efficiency in high-risk TA-TMA patients treated with a PK/PD guided protocol Demonstrate the impact of eculizumab precision dosing, adherence and stewardship Describe the role of clinical pharmacists and the medical teams in guiding initiation, escalation and discontinuation of eculizumab"

PK/PD data • Bone Marrow Transplantation • Transplantation • Transplantation Associated Thrombotic Microangiopathy

Characterization and Outcomes of Switching Tacrolimus to Sirolimus in Patients with Transplant-Associated Thrombotic Microangiopathy (TCT-ASTCT-CIBMTR 2026) - "GVHD prophylaxis included post-transplant cyclophosphamide (PTCy)/Tac/Mycophenolate mofetil (MMF) (43%), Tac/methotrexate (33%), and Tac/MMF (24%)...Nine patients (22%) received at least one dose of eculizumab (range 1 – 18 doses)... In patients with TA-TMA, transitioning from Tac to Siro may represent a cost-effective strategy for at least partial TA-TMA resolution without a significant increase in severe GVHD. Interpretation of these outcomes are limited by incomplete assessment of TA-TMA biomarkers across timepoints. These finding warrant study in larger cohorts with comparison against Tac continuation."

Clinical • Acute Graft versus Host Disease • Cardiovascular • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hypertension • Immunology • Infectious Disease • Septic Shock • Thrombocytopenia • Transplantation • Transplantation Associated Thrombotic Microangiopathy

A Case Report of Transplantation Associated Thrombotic Microangiopathy (TA-TMA) in a Patient with Double Heterozygous MCP/CD46 and Cfh-H3 Risk Haplotypes (TCT-ASTCT-CIBMTR 2026) - "The patient received fludarabine/melphalan conditioning and post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil for GVHD prophylaxis...The patient was initiated on eculizumab at a dose of 900 mg weekly for four weeks...3 . Identify key clinical takeaways from this case to improve recognition, genetic evaluation, and individualized management of TA-TMA in future transplant recipients."

Case report • Clinical • Acute Kidney Injury • Antibody-mediated Rejection • Atypical Hemolytic Uremic Syndrome • Bone Marrow Transplantation • CNS Disorders • Complement-mediated Rare Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Multiple Myeloma • Nephrology • Thrombocytopenia • Transplantation • Transplantation Associated Thrombotic Microangiopathy • CD46

Treatment of Severe, Eculizumab Refractory TA-TMA in a Pediatric Patient (TCT-ASTCT-CIBMTR 2026) - "He tolerated his first autologous SCT (conditioned with thiotepa/cyclophosphamide) well without any toxicities. He initially tolerated the chemotherapy regimen (melphalan, etoposide, carboplatin) for his second autologous SCT...His eculizumab was spaced to weekly on D+132 as he transitioned to receiving dinutuximab therapy, abdominal radiation was delayed...We aim to raise awareness of this need and report successful treatment with multiple lines of therapy that included dexamethasone, emapalumab, TPE, and rituximab...Evaluate different therapeutic strategies for refractory TA-TMA 3. Understand treatment options of refractory TA-TMA that allows for continued clinical treatment of underlying neuroblastoma"

Clinical • Hematological Disorders • Infectious Disease • Neuroblastoma • Pediatrics • Renal Disease • Solid Tumor • Thrombocytopenia • Transplantation Associated Thrombotic Microangiopathy • CXCL9

Multiomics Reveal Interferon-Induced Cell Senescence As a Novel Mechanism of Eculizumab Resistance in TA-TMA (TCT-ASTCT-CIBMTR 2026) - "Discuss novel mechanisms of eculizumab resistance. Discuss potential therapies to prevent eculizumab resistance."

Transplantation Associated Thrombotic Microangiopathy

Outcomes of Allogeneic HSCT in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in Resource Constrained Settings. (TCT-ASTCT-CIBMTR 2026) - "The findings of this study confirm that most patients with PNH can be cured with hematopoietic stem cell transplantation in our settings as Complement inhibitors like Eculizumab is not available and unaffordable for our patients. 1- i will gain experience in presenting original research to professional audience, it will enhance my ability to communicate scientific data effectively. 2-i will get a chance to engage with experts in my field to recieve constructive feed back on my work, helping refine my research approach."

Clinical • Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Complement-mediated Rare Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis • CD55 • CD59

Determinants of Eculizumab Response in High-Risk Transplant-Associated Thrombotic Microangiopathy (TCT-ASTCT-CIBMTR 2026) - "Explore therapeutic strategies beyond complement blockade. Learners will examine how integrating IFN γ blockade with emapalumab can enhance eculizumab PK/PD, accelerate complement suppression, and improve survival, highlighting the complement –interferon loop as a novel therapeutic target ."

Bone Marrow Transplantation • Renal Disease • Transplantation • Transplantation Associated Thrombotic Microangiopathy • CXCL9 • IFNG

Refractory AMR: Cutting to the Core of Management (ISHLT 2026) - "The central questions are:· What is your treatment of choice now? proteasome inhibitors, daratumumab, tocilizumab, eculizumab, extracorporeal photopheresis (ECP), or splenectomy?· What challenges have you encountered with real-world use (toxicity, access, relapse)?· In hindsight, would you have omitted rituximab in the prior treatment course, and why?At the end of the talk there will be a vote for best treatment from the audience"

Transplantation

THROMBOTIC MICROANGIOPATHY FOLLOWING CAR T-CELL THERAPY: A CASE SERIES FROM THE EBMT TRANSPLANT COMPLICATIONS WORKING PARTY (EBMT 2026) - "Among lymphoma patients, CAR T-cell products included axi-cel (n=3), brexu-cel (n=1) and tisa-cel (n=1)...Three were given treatment for their TMA (corticosteroids, antihypertensive therapy, or ravulizumab)...Treatment included corticosteroids, plasma exchange, tocilizumab, and long-term eculizumab... This case-series suggests that TMA after CAR T-cell therapy, although rare, is clinically significant and frequently requires management. All patients exhibited preceding CRS and most ICANS, hinting that inflammatory triggers may play a relevant role. Continued vigilance and dedicated prospective data collection are needed to characterise incidence and treatment strategies of TMA after CAR-T-cell therapy."

CAR T-Cell Therapy • Clinical • B Cell Lymphoma • Cardiovascular • Follicular Lymphoma • Hematological Malignancies • Hypertension • Infectious Disease • Large B Cell Lymphoma • Lymphoma • Mantle Cell Lymphoma • Non-Hodgkin’s Lymphoma • Renal Disease • Thrombocytopenia • Transplantation

Practical Considerations for Infection Prevention With the Clinical Use of Complement Inhibitors. (PubMed, Clin J Am Soc Nephrol) - "Currently, five complement inhibitors are approved by the United States Food and Drug Administration as effective for the treatment of kidney diseases: eculizumab, ravulizumab, avacopan, iptacopan, and pegcetacoplan. This review summarizes the infectious risks associated with complement inhibitors and highlights key clinical considerations for their safe and effective use in the treatment of kidney diseases. It is intended to serve as an accessible resource for providers utilizing these agents in clinical practice."

Journal • ANCA Vasculitis • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Infectious Disease • Influenza • Meningococcal Infections • Nephrology • Pneumococcal Infections • Pneumonia • Renal Disease • Respiratory Diseases • Vasculitis

Consensus Recommendations for the Diagnosis and Treatment of Neuromyelitis Optica Spectrum Disorders (NMOSD): The MENACTRIMS Guidelines. (PubMed, CNS Drugs) - "For acute treatment: initiate high-dose intravenous methylprednisolone promptly and use plasma exchange early for severe or steroid-refractory attacks. For long-term immunotherapy, monoclonal antibodies (rituximab, inebilizumab, eculizumab, ravulizumab, satralizumab, or tocilizumab) are recommended according to availability and patient factors; conventional immunosuppressants remain alternatives when biologics are inaccessible. Guidance is provided for pediatric patients and for pregnancy and breastfeeding, including planning after ≥ 12 months of disease stability and early postpartum treatment resumption. These MENACTRIMS guidelines aim to improve NMOSD outcomes across the region by promoting accurate diagnosis and timely, effective therapy."

Journal • Review • CNS Disorders • Immunology • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Pediatrics • Rare Diseases • Solid Tumor

Changes in Blood Cells and Complements During Relapse Prevention Therapies for Aquaporin-4 Antibody-Positive Neuromyelitis Optica Spectrum Disorder. (PubMed, Int J Mol Sci) - "They were divided into the following treatment groups: glucocorticoids and/or immunosuppressants (GC/IS, n = 22), inebilizumab/rituximab (anti-CD19/20, n = 13), satralizumab (anti-IL-6R, n = 22), and eculizumab/ravulizumab (anti-C5, n = 13). It also showed that anti-C5 therapy strongly suppressed total complement activity but did not affect the C3 and C4 levels or blood counts. These findings may have implications for the mode of action of the drugs and the risk of adverse drug reactions, including infections."

Journal • CNS Disorders • Infectious Disease • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

MANAGEMENT AND OUTCOMES OF PEDIATRIC TA-TMA AFTER ALLOGENEIC HCT: A UK MULTICENTER RETROSPECTIVE STUDY (EBMT 2026) - "Twenty-eight patients (66%) required admission to the pediatric intensive care unit for a median of 11 days (IQR, 28), primarily for respiratory support (n=22), inotropic therapy (n=9), and renal replacement therapy (n=10).Regarding treatment, 6 patients (14%) received defibrotide alone; 18 (43%) received complement inhibitors (predominantly eculizumab, with one case each of ravulizumab and nomacopan); 16 (38%) received defibrotide followed by a complement inhibitor (including one concomitant administration); and 3 (5%) received complement inhibitors followed by defibrotide... TA-TMA is a highly aggressive complication following HCT. Both defibrotide and complement inhibitors demonstrated similar ORR; however, complement inhibitors achieved more CR across sC5b-9 strata. Complement inhibitors should be considered first-line therapy, even when sC5b-9 levels are not elevated."

Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Infectious Disease • Pediatrics • Transplantation Associated Thrombotic Microangiopathy

SAFETY OF RAVULIZUMAB USE IN PREGNANCY: INSIGHTS FROM A GLOBAL PHARMACOVIGILANCE ANALYSIS (EBMT 2026) - P | "This analysis provides real-world insights on the use of ravulizumab in pregnancy and suggests no unexpected safety signals, similar to analyses on the use of eculizumab during pregnancy. These findings may inform clinical decision-making. As additional data are needed, a global observational study (NCT06312644) evaluating the safety of ravulizumab during pregnancy is currently ongoing."

Adverse events • Clinical • Aplastic Anemia • Atypical Hemolytic Uremic Syndrome • Cardiovascular • CNS Disorders • Complement-mediated Rare Disorders • Diabetes • Gestational Diabetes • Hepatology • Hypertension • Immunology • Metabolic Disorders • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

REAL-WORLD ANALYSIS OF RAVULIZUMAB SAFETY AND EFFECTIVENESS IN ADVANCED AGE PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: INSIGHTS FROM THE INTERNATIONAL PNH REGISTRY (EBMT 2026) - P | "Ravulizumab demonstrated effective control of PNH in C5i-naive and eculizumab-experienced patients in the real-world setting, regardless of age at initiation. LDH control and transfusion outcomes improved in both age groups, with no significant differences. Incidence of MAVEs and TEs during treatment was low."

Clinical • Metastases • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Complement-mediated Rare Disorders • Genito-urinary Cancer • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Meningococcal Infections • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Cell Carcinoma • Septic Shock • Solid Tumor