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

The Impact of Stem Cell Transplantation on Sickle Cell Hepatopathy and Vice Versa (TCT-ASTCT-CIBMTR 2026) - "31.5% of patients received ursodiol prophylaxis, and no patients received defibrotide prophylaxis or treatment. Among 12 patients with available pre- and post-HCT HAI inflammation scores, 91.7% showed improvement or stability and of the 6 patients with elevated pre-HCT HAI fibrosis scores and post-HCT data, 66.6% demonstrated improvement or stability. Deugnier scores improved or remained stable in 55.5% of 9 patients. Our data suggest that non-myeloablative HCT can result in stable to improved liver disease with HCT outcomes comparable to patients who undergo HCT for SCD without pre-existing sickle cell hepatopathy."

Cardiovascular • Fibrosis • Genetic Disorders • Hematological Disorders • Hepatology • Immunology • Inflammation • Sickle Cell Disease • Transplantation

Characterizing an Increased Incidence of Sinusoidal Obstruction Syndrome (SOS) at a Large Academic Medical Center (TCT-ASTCT-CIBMTR 2026) - "Introduction: Sinusoidal obstruction syndrome (SOS) is a complication of hematopoietic stem cell transplant (HCT) with a mortality rate >80% in severe cases without defibrotide treatment...We characterized these cases and explored the incidence of SOS in the era of post- transplant cyclophosphamide (PTCy)...Pertinent data included: diagnostic and risk criteria, transplant regimen, comorbidity index, inotuzumab or gemtuzumab use, and death from SOS... An increased incidence of SOS is noted in our retrospective data that shows a relationship with implementation of PTCy as a GVHD platform. SOS continues to have high mortality and morbidity, including prolonged hospitalization and need for dialysis. These findings should be confirmed by a larger multicenter retrospective study."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm

Allogeneic Hematopoietic Cell Transplantation for Infantile Osteopetrosis: High Rates of Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease (SOS/VOD) and Transplant-Related Morbidity (TCT-ASTCT-CIBMTR 2026) - "Busulfan-based conditioning increases SOS/VOD risk...4/24 (17%) patients received prophylactic defibrotide...3. Successful HCT for individuals with osteopetrosis requires a measured approach to reduce transplant- related morbidity/mortality due to baseline organ dysfunction and extramedullary hematopoiesis."

Aplastic Anemia • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Liver Failure • Transplantation

Refining Allogeneic Blood and Marrow Transplantation As a Safe and Curative Option for Patients with Transfusion-Dependent Thalassemia – a Single Center Experience (TCT-ASTCT-CIBMTR 2026) - " We report our single-center experience, including the use of an augmented non-myeloablative (NMA) regimen (anti-thymocyte globulin, fludarabine, cyclophosphamide, total body irradiation 400 cGy) and post-transplant cyclophosphamide (PT-Cy)...One patient developed VOD following myeloablation, and one developed idiopathic pneumonia syndrome both recovered fully after treatment with defibrotide and etanercept, respectively... Allo-BMT is a safe, feasible, and highly effective therapy for TDT. The use of our established NMA platform, best available donor, and PT-Cy-based GVHD prophylaxis significantly abrogates the risk of post-BMT complications, mitigates GVHD, and is sufficient to provide ample donor-derived erythropoiesis resulting in long-term transfusion independence. Our experience has resulted in a paradigm shift regarding conditioning regimen intensity and donor selection for afflicted patients at our institution and highlights that all TDT patients should routinely..."

Clinical • Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Transplantation

A phase 2 trial of defibrotide for the prevention of chimeric antigen receptor T-cell-associated neurotoxicity syndrome. (PubMed, Blood Adv) - P2 | "This open-label, single-arm, phase 2 study evaluated safety and efficacy of defibrotide for prevention of CAR-T-associated neurotoxicity in patients with relapsed/refractory large B-cell lymphoma receiving axicabtagene ciloleucel. Nevertheless, results contribute valuable data for potential therapeutic insight on the management of CAR-T-associated neurotoxicity. Trial registration: ClinicalTrials.gov identifier: NCT03954106."

CAR T-Cell Therapy • Journal • P2 data • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hepatology • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Otorhinolaryngology • Pulmonary Disease • Respiratory Diseases

OUTCOME OF HEMATOPOIETIC STEM CELL TRANSPLANTATION IN INFANT LEUKEMIA: A SINGLE-CENTER RETROSPECTIVE STUDY (EBMT 2026) - "All of the 8 patients with CR2 achieved molecular remission in which 7 patients were treated with autologous CAR-T, including CD19 and CD19/CD22, and 1 patient was treated with Blinatumomab.All patients were treated with the MAC regimen, and with tacrolimus and low-dose MTX to prevent active GVHD...Major complications included veno–occlusive disease (VOD) in 9% of patients which resolved by supportive measures and defibrotide.The incidence of grade II-IV acute GVHD was 31%, and the incidence of chronic GVHD was 15% respectively... Infants with leukemia shows a low long-term survival. Hematopoietic stem cell transplantation is safe and effective in the treatment of infant leukemia. It is the most importance to make MRD negative before transplantation."

Retrospective data • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Ophthalmology • Septic Shock • T Acute Lymphoblastic Leukemia • Transplantation • CD22 • KMT2A

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-hostdisease (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 isdisease relapse; however, second CAR-T infusions are increasingly considered. Allogeneic HSCT after CAR-T is also recommended as..."

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

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

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

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 • Dermatology • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Mucositis • Neutropenia • Pediatrics • Transplantation • CD34

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

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

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...We started a treatment with Sildenafil, Macitentan, Epoprostenol in addition to Methylprednisolone pulse therapy and antifibrotic treatment with Nintedanib, Hydroxychloroquin and Azithromycin...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..."

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

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

PEDIATRIC ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHRONIC GRANULOMATOUS DISEASE: A CASE SERIES OF SINGLE CENTER EXPERIENCE (EBMT 2026) - "We included all patients except one patient conditioning with ATG, fludarabine and treosulfan...One patient developed VOD despite receiving defibrotide prophylaxis... HSCT is the only potentially curative treatment option in patients with CGD with significant morbidity and mortality, especially in patients who receive grafts from unrelated donors. These factors need to be considered in the decision-making process and when discussing conditioning and GVHD prophylaxis. In this retrospective study, we showed the results of our reduced toxicity myeloablative conditioning regimen is excellent overall survival, negligible GVHD, and low toxicity among in pediatric patients with CGD even in those with high-risk pre-HSCT clinical features or those with no HLA-identical family donor who transplanted match unrelated donor."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Pediatrics • Pneumonia • Primary Immunodeficiency • Respiratory Diseases • Septic Shock • Transplantation

BEYOND SUPPORTIVE CARE: ALLOGENEIC STEM CELL TRANSPLANTATION IN CHILDREN WITH FUCOSIDOSIS (EBMT 2026) - "All patients received busulfan-based conditioning regimens combined with fludarabine, ATG, and thiotepa. One patient required a second allo-HSCT because of declining donor chimerism and increasing enzyme levels suggestive of graft rejection; defibrotide was administered due to pre-existing hepatic involvement.Full donor chimerism and sustained engraftment were achieved in all patients... Allo-HSCT is a feasible and safe disease-modifying treatment option for pediatric fucosidosis, providing durable engraftment and long-term disease-free survival. Neurological stabilization or improvement may be achievable even in patients with advanced disease. These findings support early referral and timely transplantation to optimize outcomes in this rare disorder."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Disorders • Developmental Disorders • Dystonia • Epilepsy • Graft versus Host Disease • Hurler Syndrome • Immunology • Infectious Disease • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Rare Diseases • Transplant Rejection • Transplantation

THE IMPACT OF HEMATOPOIETIC CELL TRANSPLANTATION ON SICKLE CELL HEPATOPATHY AND VICE VERSA (EBMT 2026) - "Ursodiol was used prophylactically in 31.5% of patients, and no patients received defibrotide prophylaxis or treatment. Our data suggest that for this select and limited population of individuals with SCD-associated hepatopathy, non-myeloablative HCT can result in stable to improved liver disease, with HCT outcomes comparable to the population of patients who undergo HCT for SCD without pre-existing sickle cell hepatopathy."

Beta-Thalassemia • Cardiovascular • Fibrosis • Genetic Disorders • Hematological Disorders • Hepatology • Immunology • Inflammation • Liver Failure • Sickle Cell Disease • Transplantation

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

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

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

HAPLO-CORD HCT: SAVE THE SIBLING! (EBMT 2026) - "Pharmacologic immunosuppression (PTIS) with fludarabine (30mg/m2), dexamethasone (25mg/m2), and azathioprine (1mg/kg), were given. Hydroxyurea (20mg/kg) was given to reduce host erythropoiesis. Hyper transfusion, dual iron chelators Deferoxamine, Deferiprone were used to reduce severe iron overload. Myeloablative conditioning with Fludarabine (25/mg2), Thiotepa (8mg/kg), GVHD prophylaxis with ATG (4.5mg/kg), PTCY, Tacrolimus (later switched to sirolimus) MMF and ruxolitinib were used. Defibrotide prophylaxis was initiated due to a high pre-transplant VOD risk score... Patient had Grade 2 CRS responded to tocilizumab... Haplo-Cord HSCT provides a viable curative option for transfusion-dependent thalassemia when a matched donor is unavailable. This case demonstrates successful engraftment, manageable toxicity, and complete hematologic recovery with tailored prophylaxis and multidisciplinary care. By merging two graft sources, Haplo-Cord transplantation under Save the..."

Beta-Thalassemia • Bone Marrow Transplantation • Genetic Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Mucositis • CD34

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

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

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