Soliris (eculizumab) / AstraZeneca - LARVOL DELTA

EFFICACY AND SAFETY OF IPTACOPAN IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: A REAL-WORLD STUDY OF 37 PATIENTS IN CHINA (EBMT 2026) - " Among the 37 patients, 3 had received prior eculizumab and the remainder were C5 inhibitor-naive. Iptacopan treatment achieved substantial clinical improvement in both classical PNH and PNH/BMF patients, as evidenced by transfusion-free hemoglobin rises and normalization of hemolytic markers in the majority."

Clinical • Real-world • Real-world evidence • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • 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

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

DANICOPAN ADD-ON THERAPY DEMONSTRATES POSITIVE EFFICACY AND SAFETY IN ADVANCED AGE ADULTS WITH PNH AND CLINICALLY SIGNIFICANT EXTRAVASCULAR HEMOLYSIS: PHASE 3 ALPHA TRIAL SUB-ANALYSIS (EBMT 2026) - P3 | "This sub-analysis demonstrates that danicopan add-on to ravulizumab or eculizumab has positive efficacy and safety profile in advanced age patients with PNH and csEVH. During 12 weeks of danicopan therapy, patients reported meaningful improvements in Hb and ARC, maintained transfusion avoidance, and reported low rate of AEs."

Clinical • Metastases • P3 data • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

CLINICAL BIOMARKERS FOR EARLY RECOGNITION OF SUBOPTIMAL ECULIZUMAB RESPONSE IN TRANSPLANT-ASSOCIATED THROMBOTIC MICROANGIOPATHY (EBMT 2026) - "Longitudinal biomarker profiling showed that non-responders had progressive CXCL9 elevation despite sC5b-9 control, and that adding IFNγ blockade with emapalumab improved eculizumab PK/PD, accelerated complement suppression, and enhanced survival, underscoring the pathogenic role of the complement-interferon loop (Fig3). These findings reveal that eculizumab non-response reflects complement independent persistent endothelial injury. Early monitoring of ST2, CXCL9, rUPCR, may identify patients at risk for suboptimal eculizumab response and guide precision therapies that can be integrated with complement blockade. Such strategies will be essential to improve survival and prevent irreversible vascular injury in high-risk TA-TMA."

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

THROMBOTIC MICROANGIOPATHY FOLLOWING CAR T-CELL THERAPY: A CASE SERIES FROM THE EBMT TRANSPLANT COMPLICATIONS WORKING PARTY (EBMT 2026) - "Overall, 215 EBMT CAR-T centres were contacted, and 90 responded indicating that they had no eligible patients.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

ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH DIAMOND-BLACKFAN ANAEMIA: A SINGLE-CENTER EXPERIENCE (EBMT 2026) - "All patients received long-term iron chelation (deferoxamine, deferasirox)...Before 2000, busulfan was combined with cyclophosphamide; later, the BuFluTT was used...Rituximab was included in the conditioning of the last four patients...Mild hepatic veno-occlusive disease occured in three patiens without need for therapeutic intervention (defibrotide). One patient developed severe transplant-associated thrombotic microangiopathy managed successfully with eculizumab... Allogeneic HSCT from matched related or unrelated donors is currently indicated for steroid-non-responsive or transfusion-dependent children with DBA. Our experience confirms high efficacy and acceptable safety, particularly in childern < 10 years, while the decision must be individualised and carefully weighed in older patiens.Supported by MHCZ for conceptual development 0064203"

Clinical • Acute Graft versus Host Disease • Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hepatology • Immunology • Infectious Disease • Transplantation • Transplantation Associated Thrombotic Microangiopathy • RPL5 • RPS26

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

TRANSPLANT ASSOCIATED THROMBOTIC MICROANGIOPATHY CLINICAL CHARACTERISTIC, RISK FACTORS, THERAPEUTIC MANAGEMENT AND OUTCOME: A SINGLE CENTER RETROSPECTIVE COHORT STUDY (EBMT 2026) - "At diagnosis, majority of patients were receiving tacrolimus or cyclosporine, and CNI withdrawal was implemented in all cases. A-TMA–directed therapy included supportive care alone in 25% (Defibrotide, plasma Exchange), CNI withdrawal alone in 6%, and complement-targeted therapy in 69%. Eculizumab and Ravulizumab were used in 31% and 39% of patients, respectively... Transplant-associated thrombotic microangiopathy (TA-TMA) remains a serious early complication following allogeneic HSCT, frequently associated with infection, GVHD, and viral reactivation. A higher incidence was observed in matched-related donor transplants compared with haplo-identical and matched-unrelated donors, likely reflecting the center's higher utilization of matched-related donors. Early diagnosis using Jodele criteria, prompt calcineurin inhibitor withdrawal, and complement-targeted therapy were associated with improved short-term survival."

Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Genetic Disorders • Graft versus Host Disease • Hematological Malignancies • Hypertension • Immunology • Infectious Disease • Leukemia • Sickle Cell Disease • Thrombocytopenia • Transplantation • Transplantation Associated Thrombotic Microangiopathy

COLD AGGLUTININ SYNDROME FOLLOWING ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (EBMT 2026) - "GVHD prophylaxis included Thymoglobuli"

Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases • Transplantation • CD4 • CD8 • HP

AN INTERSTING CASE OF DONOR DERIVED LEUKAEMIA POST CORD BLOOD TRANSPLANT (EBMT 2026) - " A 13-year-old boy underwent a T-cell–replete 6/8 mismatched cord blood transplant (CBT) with treosulfan-based myeloablative conditioning for high-risk post-transplant relapse of KMT2A-mutated, therapy-related AML in molecular remission...After achieving molecular remission with debulking chemotherapy, he underwent a matched unrelated donor HSCT with busulfan-based conditioning...Following venetoclax-based therapy and renewed remission, he proceeded to a second HSCT as described above.He engrafted promptly with full donor chimerism...Secondary to the GVHD process, he developed transplant-associated thrombotic microangiopathy (TA-TMA) and received eculizumab with partial response... We document the first donor-derived JMML with a PTPN11 mutation after cord blood transplant. This case underscores diagnostic challenges, the need for awareness of DDL, and the importance of standardised donor genetic screening despite ethical and logistical barriers."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Juvenile Myelomonocytic Leukemia • Leukemia • Multiple Myeloma • Transplantation • Transplantation Associated Thrombotic Microangiopathy • CD8 • KMT2A • MLLT3 • PTPN11

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

ORAL IPTACOPAN DEMONSTRATES EFFICACY AS SALVAGE THERAPY FOR HIGH-RISK TRANSPLANT-ASSOCIATED THROMBOTIC MICROANGIOPATHY: FIRST REAL-WORLD EXPERIENCE (EBMT 2026) - "Prior salvage therapies were extensive, including calcineurin inhibitor withdrawal (n=19), rituximab (n=11), and eculizumab (n=9), plasma exchange (n=7), and defibrotide (n=4). In conclusion, the oral complement inhibitor iptacopan demonstrated significant clinical efficacy as salvage therapy in high-risk TA-TMA patients, irrespective of prior eculizumab exposure."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cerebral Hemorrhage • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Meningococcal Infections • Pneumonia • Respiratory Diseases • Transplantation • Transplantation Associated Thrombotic Microangiopathy • CFB

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

DANICOPAN ADD-ON THERAPY DEMONSTRATES POSITIVE EFFICACY AND SAFETY IN ADVANCED AGE ADULTS WITH PNH AND CLINICALLY SIGNIFICANT EXTRAVASCULAR HEMOLYSIS: PHASE 3 ALPHA TRIAL SUB-ANALYSIS (EBMT 2026) - P3 | "This sub-analysis demonstrates that danicopan add-on to ravulizumab or eculizumab has positive efficacy and safety profile in advanced age patients with PNH and csEVH. During 12 weeks of danicopan therapy, patients reported meaningful improvements in Hb and ARC, maintained transfusion avoidance, and reported low rate of AEs."

Clinical • Metastases • P3 data • Complement-mediated Rare Disorders • Hematological Disorders • 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

OUTCOMES OF ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION AS THE ONLY TREATMENT AVAILABLE FOR PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA IN VENEZUELA (EBMT 2026) - "Background: Nowadays, the therapy with Eculizumab has proven to be effective in controlling the symptoms and preventing complications associated with Paroxysmal Nocturnal Hemoglobinuria (PNH)...The conditioning regimens used were: RIC with a combination of Busulfan, Fludarabine, and Cyclophosphamide for the HLA-haploidentical HCT cases, and MAC that consisted of a combination of Busulfan and Cyclophosphamide... Allogenic HCT is recognized worldwide as the only curative treatment for patients with PNH, offering an Overall Survival (OS) of 70 to 80% at 5 years of follow-up after HCT. However, there is no guide on how or when to proceed to HCT as front-line therapy.In our institution, it is feasible to carry out an allogeneic HCT as front-line therapy for PNH patients, using a RIC regimen and an identical MRD or HLA-haploidentical donor. Long-term follow-up is necessary to evaluate OS, incidence of GVHD, and other complications post HCT."

Clinical • Bone Marrow Transplantation • Complement-mediated Rare Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Transplantation • CD34

EFFICACY AND SAFETY OF IPTACOPAN IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: A REAL-WORLD STUDY OF 37 PATIENTS IN CHINA (EBMT 2026) - " Among the 37 patients, 3 had received prior eculizumab and the remainder were C5 inhibitor-naive. Iptacopan treatment achieved substantial clinical improvement in both classical PNH and PNH/BMF patients, as evidenced by transfusion-free hemoglobin rises and normalization of hemolytic markers in the majority."

Clinical • Real-world • Real-world evidence • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

CASE REPORT: THE USE OF PEGCETACOPLAN PERI-HAEMATOPOIETIC STEM CELL TRANSPLANT FOR PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA / APLASTIC ANAEMIA (EBMT 2026) - "She did not have an adult fully matched sibling donor so underwent immunosuppression with horse anti-thymocyte globulin (hATG), cyclosporin and eltrombopag...The conditioning regimen consisted of rATG, fludarabine, cyclophosphamide and one fraction of 2.00 Gy total body irradiation as per the protocol by DeZern et al (Blood 2023)... Use of eculizumab during the peri-transplant period has been described. We are not aware of any previously described cases of pegcetacoplan as a bridging therapy to allograft for patients with PNH and AA. The case demonstrates that this novel complement inhibitor can be used during the transplant conditioning and continued after stem cell infusion with successful engraftment and clearance of the PNH clone."

Case report • Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Complement-mediated Rare Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Pneumonia • Septic Shock • Thrombosis • Transplantation

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

TRANSPLANT-ASSOCIATED THROMBOTIC MICROANGIOPATHY DRIVES MORTALITY IN PEDIATRIC HSCT: INSIGHTS FROM A SINGLE-CENTER COHORT (EBMT 2026) - "Nine patients were diagnosed during the clinical course and treated with eculizumab... TA-TMA significantly contributes to TRM in pediatric HSCT recipients. Its heterogeneous and sometimes fulminant presentations highlight the need for heightened clinical awareness. In response, we implemented a structured screening protocol for TA-TMA."

Clinical • Acute Respiratory Distress Syndrome • Aplastic Anemia • Bone Marrow Transplantation • CNS Disorders • Epilepsy • Graft versus Host Disease • Hematological Disorders • Immunology • Pediatrics • Respiratory Diseases • Transplantation • Transplantation Associated Thrombotic Microangiopathy

ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH DIAMOND-BLACKFAN ANAEMIA: A SINGLE-CENTER EXPERIENCE (EBMT 2026) - "All patients received long-term iron chelation (deferoxamine, deferasirox)...Before 2000, busulfan was combined with cyclophosphamide; later, the BuFluTT was used...Rituximab was included in the conditioning of the last four patients...Mild hepatic veno-occlusive disease occured in three patiens without need for therapeutic intervention (defibrotide). One patient developed severe transplant-associated thrombotic microangiopathy managed successfully with eculizumab... Allogeneic HSCT from matched related or unrelated donors is currently indicated for steroid-non-responsive or transfusion-dependent children with DBA. Our experience confirms high efficacy and acceptable safety, particularly in childern < 10 years, while the decision must be individualised and carefully weighed in older patiens.Supported by MHCZ for conceptual development 0064203"

Clinical • Acute Graft versus Host Disease • Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hepatology • Immunology • Infectious Disease • Transplantation • Transplantation Associated Thrombotic Microangiopathy • RPL5 • RPS26

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

THROMBOTIC MICROANGIOPATHY FOLLOWING CAR T-CELL THERAPY: A CASE SERIES FROM THE EBMT TRANSPLANT COMPLICATIONS WORKING PARTY (EBMT 2026) - "Overall, 215 EBMT CAR-T centres were contacted, and 90 responded indicating that they had no eligible patients.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

CLINICAL BIOMARKERS FOR EARLY RECOGNITION OF SUBOPTIMAL ECULIZUMAB RESPONSE IN TRANSPLANT-ASSOCIATED THROMBOTIC MICROANGIOPATHY (EBMT 2026) - "Longitudinal biomarker profiling showed that non-responders had progressive CXCL9 elevation despite sC5b-9 control, and that adding IFNγ blockade with emapalumab improved eculizumab PK/PD, accelerated complement suppression, and enhanced survival, underscoring the pathogenic role of the complement-interferon loop (Fig3). These findings reveal that eculizumab non-response reflects complement independent persistent endothelial injury. Early monitoring of ST2, CXCL9, rUPCR, may identify patients at risk for suboptimal eculizumab response and guide precision therapies that can be integrated with complement blockade. Such strategies will be essential to improve survival and prevent irreversible vascular injury in high-risk TA-TMA."

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