Defitelio (defibrotide) / Medison, IRCCS San Raffaele Hospital, Jazz, SOBI - LARVOL DELTA

A CONUNDRUM IN VOD/SOS: THE CASE OF A MISSING BIOPSY (ISN-WCN 2026) - "Defibrotide remains the only available therapy.Methods Male of 34 years with recently diagnosed Acute Leukemia who received fludarabine and total body irradiation, followed by an allogeneic bone marrow transplant. Conclusion VOD/SOS remains a diagnostic challenge given the lack of pathognomonic findings. The European Society for Blood and Marrow Transplantation is the best diagnostic criteria due to its sensitivity, which allows early detection and treatment. Defibrotide is the first and only FDA-approved therapy for severe VOD/SOS."

Biopsy • Clinical • Bone Marrow Transplantation • Hematological Malignancies • Hepatology • Leukemia • Nephrology • Renal Disease

A Comparative Study of Porcine and Ovine Derived Defibrotide: Coagulation Profiles, Molecular Characteristics, and DNA Content. (PubMed, Clin Appl Thromb Hemost) - "DNA content showed no significant difference (ovine: 75.78 μg vs porcine: 79 μg/10 μg API).ConclusionOvine- and porcine-derived defibrotide demonstrated equivalent coagulation, molecular, and DNA content profiles. These findings suggest ovine mucosa is a viable alternative source for defibrotide, potentially improving accessibility, reducing cost, and addressing dietary restrictions associated with porcine-derived products."

Clinical • Journal • Preclinical • Bone Marrow Transplantation • Transplantation

BUSULFAN ASSOCIATED PULMONARY TOXICITY AND INFECTIOUS COMPLICATIONS FOLLOWING AUTOLOGOUS SCT IN A 3-YEAR-OLD CHILD WITH NEUROBLASTOMA (EBMT 2026) - "Background: High-dose busulfan/melphalan (Bu/Mel) remains a standard conditioning regimen for autologous stem cell transplantation in high-risk neuroblastoma but is associated with significant organ toxicity, particularly hepatic and pulmonary injury...A treatment with Defibrotide, Ganciclovir and Azithromycin was initiated...We started a treatment with Sildenafil, Macitentan, Epoprostenol in addition to Methylprednisolone pulse therapy and antifibrotic treatment with Nintedanib, Hydroxychloroquin and Azithromycin... This case illustrates the complex interplay between infectious complications, post-transplant inflammation and busulfan-induced pulmonary toxicity in a young neuroblastoma patient undergoing Bu/Mel conditioning and autologous SCT. Consideration of busulfan toxicities, especially when presenting with nonspecific early symptoms is important even when alternative explanations such as severe infection may appear equally plausible after autologous SCT. Early..."

Clinical • Acute Respiratory Distress Syndrome • Cardiovascular • Cystic Fibrosis • Cytomegalovirus Infection • Gastroenterology • Genetic Disorders • Hepatology • Immunology • Infectious Disease • Inflammation • Interstitial Lung Disease • Neuroblastoma • Pneumonia • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases • Septic Shock • Solid Tumor

Efficacy analysis of iptacopan in a patient with thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation: a case report. (PubMed, Front Immunol) - "The patient had an initial partial response to the C5 inhibitor eculizumab, but the disease progressed...The patient received a total of six sessions of therapeutic plasma exchange and two concurrent doses of defibrotide during the Iptacopan course...This biochemical improvement coincided with key clinical outcomes: resolution of proteinuria and the achievement of sustained red blood cell transfusion independence after day +58 and platelet transfusion independence after day +66, marking a decisive turnaround in his TA-TMA course. Treatment with the novel oral complement inhibitor Iptacopan induced significant hematological and clinical responses in this TA-TMA patient, demonstrating its potential therapeutic efficacy and warranting further clinical investigation."

Journal • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Renal Disease • Transplantation • Transplantation Associated Thrombotic Microangiopathy • IDH1 • STAG2

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION POST–CAR-T THERAPY IN CHILDREN WITH PRE-B ACUTE LYMPHOBLASTIC LEUKAEMIA: NATIONAL POLISH EXPERIENCE (EBMT 2026) - "Conditioning regimens included total body irradiation with etoposide in nine patients; treosulfan, fludarabine, and thiotepa in two patients; and fludarabine with cyclophosphamide in one patient. Post-transplant complications included one patient developing severe veno-occlusive disease, treated with defibrotide. Four children developed acute skin graft-versus-host disease (GVHD): three responded to steroid therapy, while one had steroid-resistant GVHD requiring etanercept. One child experienced graft rejection and required a second HSCT.Two patients were treated with cidofovir for adenovirus reactivation, and one subsequently developed chronic bone marrow failure... Hematopoietic stem cell transplantation remains an important therapeutic strategy in selected patients following CAR-T therapy. The primary indication for HSCT in this setting is disease relapse; however, second CAR-T infusions are increasingly considered. Allogeneic HSCT after CAR-T is also recommended as..."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • Aplastic Anemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Leukemia • Transplant Rejection • Transplantation • CD34

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION POST–CAR-T THERAPY IN CHILDREN WITH PRE-B ACUTE LYMPHOBLASTIC LEUKAEMIA: NATIONAL POLISH EXPERIENCE (EBMT 2026) - "Conditioning regimens included total body irradiation with etoposide in nine patients; treosulfan, fludarabine, and thiotepa in two patients; and fludarabine with cyclophosphamide in one patient. Post-transplant complications included one patient developing severe veno-occlusive disease, treated with defibrotide. Four children developed acute skin graft-versus-host disease (GVHD): three responded to steroid therapy, while one had steroid-resistant GVHD requiring etanercept. One child experienced graft rejection and required a second HSCT.Two patients were treated with cidofovir for adenovirus reactivation, and one subsequently developed chronic bone marrow failure... Hematopoietic stem cell transplantation remains an important therapeutic strategy in selected patients following CAR-T therapy. The primary indication for HSCT in this setting is disease relapse; however, second CAR-T infusions are increasingly considered. Allogeneic HSCT after CAR-T is also recommended as..."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • Aplastic Anemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Leukemia • Transplant Rejection • Transplantation • CD34

T CELL-DEPLETED HAPLOIDENTICAL AND MATCHED UNRELATED DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION REPRESENT SAFE AND EFFICIENT THERAPEUTIC APPROACHES FOR PEDIATRIC PATIENTS WITH BONE MARROW FAILURE SYNDROMES (EBMT 2026) - "Five patients received a conditioning regimen based on fludarabine and cyclophosphamide (FC) and three fludarabine, thiotepa, treosulfan (FTT), along with ATG-Grafalon® or thymoglobulin. One SD patient received a MUD-PBSC, conditioned with fludarabine, thiotepa and melphalan (FTM). Immunosuppression (IST) consisted of tacrolimus (or cyclosporine) and mycophenolate mofetil (MMF) in all nine patients...One case of veno-occlusive disease (VOD) in a T-haplo-HSCT patient with FA resolved completely after treatment with defibrotide... This single-center, retrospective series supports feasibility and safety of T-haplo-HSCT in rare BMF syndromes and indicates favorable outcomes in T-haplo-HSCT with fast engraftment, low GVHD, and a reduced risk of severe toxicity compared to MUD-HSCT in children."

Clinical • Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Dermatitis • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Mucositis • Neutropenia • Pediatrics • Transplantation • CD34

HAEMATOPOIETIC STEM CELL TRANSPLANT (HSCT) IN NEUROPATHIC MUCOPOLYSACCHARIDOSIS TYPE-II (MPS-II) PRESERVES NEURODEVELOPMENT AND SHOULD BE ROUTINE PRACTICE (EBMT 2026) - "He was conditioned with Busulfan and Fludarabine following the standard European Guidelines...The same donor donated PBSC and conditioning was with Fludarabine, Thiotepa and Treosulfan. Alemtuzumab serotherapy, Cyclosporin-A and mycophenolate mofetil used as GVHD prophylaxis. Defibrotide was used for VOD prophylaxis in second transplant due to experiencing mild VOD during first transplant.Multisystem evaluation including neurodevelopmental assessments were recorded over 6–7 follow-up-years... In neuropathic MPS-II, definitive therapy with HSCT in early infancy preserves neurodevelopment and significantly reduces the multi-system disease burden compared with initiating ERT in later childhood. This sibling case-comparison supports the rationale for the need of newborn screening and prompt referral for HSCT. We have separately developed a gene therapy programme to further improve outcomes of cellular therapy in MPS-II and are reporting those outcomes at this meeting."

Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Graft versus Host Disease • Hunter Syndrome • Hurler Syndrome • Immunology • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Transplantation

TREOSULFAN BASED MYELOABLATIVE CONDITIONING IN ADOLESCENT AND ADULT PATIENTS WITH HEMOGLOBINOPATHY (EBMT 2026) - "All patients received a conditioning regimen consisting of treosulfan (3 x 14 g/m²), thiotepa (2 x 5 mg/kg BW), fludarabine (4 x 40 mg/m²), and ATG-Grafalon®, with the only difference being in the timing of ATG (upfront 3 x 15 mg/kg BW in T-haplo-HSCT, 3 x 10 mg/kg BW in MSD-HSCT on days -3 to -1). Immunosuppression involved a combination of calcineurin inhibitors and mycophenolate mofetil...Three patients developed mild hepatic SOS, all responding well to defibrotide and fully recovering... Treosulfan-based conditioning proved to be safe, myeloablative, and highly immunosuppressive. It emerges as an excellent alternative to busulfan in this high-risk population, with no cases of conditioning-related graft failure, no significant mixed chimerism, and neither severe GVHD nor SOS."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Epstein-Barr Virus Infections • Gastroenterology • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Sickle Cell Disease • CD14

INCIDENCE OF SINUSOIDAL OBSTRUCTION SYNDROME (VOD/SOS) DIAGNOSED BY DOPPLER ULTRASOUND/LIVER ELASTOGRAPHY IN PATIENTS AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (EBMT 2026) - P | "Twenty-nine patients received defibrotide, with a median treatment duration of 21 days (range, 3–60)... Pre-transplant liver assessment using US-Doppler and LSM is significantly associated with subsequent SOS development, suggesting that these non-invasive approaches may serve as valuable predictive biomarkers. Prospective validation in larger multicenter studies is warranted."

Clinical • Bone Marrow Transplantation • Hepatology • Transplantation

INOTUZUMAB OZOGAMICIN AS A BRIDGE TO ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION IN RELAPSED/REFRACTORY B-ALL: A STUDY ON BEHALF OF GETH-TC (EBMT 2026) - "Defibrotide was used in all but one patient, with SOS resolution in 22 patients (67%). InO is an effective bridge to alloHCT for responsive R/R B-ALL patients. However, relapse and SOS remain major challenges, with InO exposure (>2 cycles or <50-day interval), number of prior treatment lines, and MRD status emerging as key risk factors influencing transplant outcomes."

Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hepatology • Immunology • Infectious Disease • Transplantation

EASIX OUTPERFORMS EBMT AND CIBMTR RISK MODELS IN PREDICTING VOD/SOS DESPITE DEFIBROTIDE USE IN ALLOGENEIC HSCT (EBMT 2026) - "Exposure to busulfan or total body irradiation was not significantly associated with VOD/SOS development. Despite defibrotide use, VOD/SOS remains a major complication after allo-HSCT. EASIX outperformed CIBMTR and complemented EBMT risk assessment in identifying patients at increased risk. Incorporating EASIX into the pretransplant evaluation may refine risk stratification and support targeted prophylactic strategies."

Bone Marrow Transplantation • Fibrosis • Gastroenterology • Hepatology • Immunology • Infectious Disease • Liver Cirrhosis • Otorhinolaryngology

HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE FOR MALIGNANT AND NON-MALIGNANT HEMATOLOGIC DISORDERS: A REAL-WORLD EXPERIENCE FROM QATAR (EBMT 2026) - "TBI-based conditioning with fludarabine 160 mg/m² and TBI 1200 cGy were administered to 30 patients (69.8%), whereas 13 patients (30.2%) received non-TBI conditioning...Three patients (7%) had grade 3 CRS and required tocilizumab. Clinically significant CMV infection was significantly higher in patients who did not receive letermovir prophylaxis (92%) than in those who did (11.6%). One patient developed VOD that was treated with defibrotide... Haplo-HCT with PTCy leads to excellent survival for patients who lack HLA-matched donors or access to an unrelated donor. Our results revealed universal engraftment, higher rates of disease-free survival and an improved graft-versus-host disease-free-relapse-free survival. Finally, in our cohort, both chemo and radiotherapy-based conditioning haploidentical cell transplantation resulted in low rates of relapse, non-relapse mortality and chronic GVHD."

Clinical • Post-transplantation • Real-world • Real-world evidence • Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Transplantation

DEFIBROTIDE RESCUE FOR PEDIATRIC VENO-OCCLUSIVE DISEASE/SINUSOIDAL OBSTRUCTION SYNDROME; A SINGLE-CENTER 10-YEAR EXPERIENCE (EBMT 2026) - "DF rescue offers an effective treatment for VOD. Early recognition of VOD and response to DF are major determinants of survival. The EBMT criteria offer a reliable tool for early recognition and treatment of VOD."

Clinical • Bone Marrow Transplantation • Hepatology • Otorhinolaryngology • Pediatrics

PLASMINOGEN ACTIVATOR INHIBITOR-1 (PAI-1) IS AN EARLY BIOMARKER OF SEVERE SINUSOIDAL OBSTRUCTION SYNDROME AFTER PEDIATRIC HSCT (EBMT 2026) - "Further, higher PAI-1/PLT was associated with malignant diagnosis (day +0: 175 vs. 123 ng/10⁹ platelets (p=0.001); day +30: 200 vs. 97 ng/10⁹ platelets (p=0.0073)), and with busulfan-based conditioning (day +21: 668 vs. 179 ng/10⁹ platelets (p=0.03); day +30:266 vs. 100 ng/10⁹ platelets (p= 0.0007)). PAI-1/PLT appeared to be an early, independent biomarker for the prediction of severe SOS. In patients requiring treatment with defibrotide, this difference was significant already before the start of conditioning. These results suggest that PAI-1/PLT may serve as a prognostic biomarker for severe SOS after pediatric HSCT."

Biomarker • Clinical • Bone Marrow Transplantation • Graft versus Host Disease • Hepatology • Pediatrics

REAL-WORLD EFFICACY AND SAFETY OF DEFIBROTIDE FOR SINUSOIDAL OBSTRUCTION SYNDROME - NATIONWIDE SURVEY (EBMT 2026) - "Our study showed the efficacy and safety of DF for the treatment of SOS comparable to previous studies. Both the EBMT 2016 and 2023 severity grading showed significant predictive ability for the prognosis of SOS treated with DF."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Bone Marrow Transplantation • Hepatology

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."

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

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

COMPREHENSIVE PAIN MANAGEMENT IN PAEDIATRIC ALLOGENIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT): RETROSPECTIVE AUDIT OF SPECIALIST PAIN TEAM REFERRALS AND DEVELOPMENT OF A PILOT PAIN-MANAGEMENT PATHWAY (EBMT 2026) - "All three patients were high-risk HSCT for relapsed/refractory acute leukaemia (and developed severe acute complications in the immediate post-HSCT period (grade IV GVHD, grade III VOD with GI bleeding post defibrotide and fatal grade IV VOD).Table 1: Characteristics of patients referred to pain services*Both patients received post-transplant cyclophosphamide (PTCy)**mismatched cord HSCT for high-risk paediatric AML Better, more focused management of pain, using novel pharmacological and non-pharmacological measures, can improve the HSCT experience for children, and specialist pain management services and dedicated guidance are vital. Better, more focused management of pain, using novel pharmacological and non-pharmacological measures, can improve the HSCT experience for children, and specialist pain management services and dedicated guidance are vital. Regular paracetamol can be safely used as an analgesic without masking sepsis. Use of Pregabalin is safe in the..."

Retrospective data • Bone Marrow Transplantation • CNS Disorders • Gastrointestinal Disorder • Genetic Disorders • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Mucositis • Pain • Pediatrics • Septic Shock • Sickle Cell Disease • Transplantation

INCIDENCE OF VOD AND LONGITUDINAL DYNAMICS OF EASIX AND ENDOTHELIAL BIOMARKERS AFTER ALLO-HCT WITH PTCY (EBMT 2026) - "Post-transplant cyclophosphamide (PTCy) is widely used for GVHD prophylaxis, but its effect on VOD remains unclear...All 8 (66.7%) moderate and severe cases received defibrotide, and one (8.3%) underwent TIPS placement... VOD remains a clinically significant complication after allo-HCT with PTCy, associated with relevant morbidity, organ dysfunction, and reduced survival. Complementary, findings support the central role of endothelial dysfunction in VOD pathophysiology and that biomarker monitoring may enable earlier patient risk stratification."

Biomarker • Graft versus Host Disease • Hepatology • Immunology • Myeloproliferative Neoplasm • VCAM1

INTRARENAL ARTERIAL INFUSION OF MESENCHYMAL STEM CELLS FOR THE TREATMENT OF TRANSPLANTATION-ASSOCIATED ACUTE RENAL FAILURE: A CASE REPORT (EBMT 2026) - "Despite treatment with defibrotide and plasma exchange, his condition deteriorated rapidly, progressing to acute renal failure... Currently, the patient is 2.5 years post-transplantation, continues to take antihypertensive medication orally, and has returned to school. Although his renal function has not fully normalized, it has not impacted his daily life or studies."

Case report • Clinical • Acute Kidney Injury • Bone Marrow Transplantation • Cardiovascular • CNS Disorders • Epstein-Barr Virus Infections • Hematological Disorders • Hypertension • Infectious Disease • Nephrology • Ophthalmology • Renal Disease • Transplantation • Transplantation Associated Thrombotic Microangiopathy • CASP10 • FAT4 • IRF7

THE ROLE OF DEFIBROTIDE AS PROPHYLAXIS FOR VENOCCLUSIVE DISEASE IN PEDIATRIC THALASSEMIA STEM CELL TRANSPLANTATION USING IV BUSULFAN CONDITIONING. A RETROSPECTIVE SINGLE-CENTER 10-YEAR EXPERIENCE (EBMT 2026) - "The duration of hospitalization was shortened, possibly after the switch to the TBF (Thiotepa, Busulfan, and Fludarabine) regimen.Table: Patient characteristics and outcome(a) Chi-Square Test, (b) Fisher's Exact Test, (c) Independent T TestReduced toxicity-Myeloablative chemotherapy (RT-MAC), Myeloablative chemotherapy (MAC) Despite a high incidence of around 39.5% patients having VOD in our pediatric thalassemia cohort, fortunately, none of the patients had severe VOD leading to any morbidity or mortality. Despite a high incidence of around 39.5% patients having VOD in our pediatric thalassemia cohort, fortunately, none of the patients had severe VOD leading to any morbidity or mortality. All patients were managed at the pediatric BMT unit without any significant complications. We acknowledge the limited sample size, the retrospective nature of the study, and the limited control group."

Retrospective data • Aplastic Anemia • Beta-Thalassemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Pediatrics • Transplantation

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

SYSTEMATIC LIVER STIFFNESS MEASUREMENT FOR DIAGNOSIS OF HEPATIC VENO-OCCLUSIVE DISEASE IN ADULT PATIENTS UNDERGOING ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION: A PROSPECTIVE REAL-WORLD SINGLE-CENTRE EXPERIENCE (EBMT 2026) - "All other cases received defibrotide and showed a therapeutic response, but the "very severe" patient died of multi-organ-failure associated with septic shock... In our single-centre experience, the ELASTOVOD cutoff for diagnosis (>25kPa) and exclusion (<6kPa) of VOD/SOS, together with the "stepwise algorithm" to assess LSM in the "grey zone", were effective with excellent sensitivity and specificity for both pSWE and 2D-SWE. In addition, both pSWE and 2D-SWE values confirmed the ability of discriminating between VOD/SOS and non–VOD/SOS post-HSCT severe hepatopathies. Despite the limited number of VOD/SOS cases, our work confirms LSM as bedside non-invasive diagnostic tool and represents the first validation of the ELASTOVOD study."

Clinical • Real-world • Real-world evidence • Bone Marrow Transplantation • Hepatology • Otorhinolaryngology • Septic Shock • Transplantation

BASELINE LIVER ELASTOGRAPHY PREDICTS THE RISK OF SOS/VOD IN PATIENTS UNDERGOING ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (EBMT 2026) - "Defibrotide was administered in 16 SOS/VOD cases.Patients who developed SOS/VOD had significantly higher baseline TE values compared with those who did not (10.3 ±7.4 kPa versus 6.2 ±4.3kPa; p<0.001). Baseline TE measurement may serve as a non-invasive tool to identify alloHCT recipients at increased risk of SOS/VOD. Patients with elevated TE, especially those previously exposed to hepatotoxic agents such as inotuzumab ozogamicin, may benefit from targeted preventive strategies to mitigate NRM."

Clinical • Bone Marrow Transplantation • Fibrosis • Hepatology • Infectious Disease • Transplantation