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

The impact of hematopoietic cell transplantation on sickle cell hepatopathy and vice versa (ASH 2025) - P1/2, P2 | "Ursodiol was used prophylactically in 31.5% of patients, and no patients received defibrotide prophylaxis or treatment. These findings possibly reflect the limited statistical power of this study and small subgroup sizes. 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."

Cardiovascular • CNS Disorders • Fibrosis • Genetic Disorders • Hematological Disorders • Hepatitis C • Hepatology • Immunology • Infectious Disease • Inflammation • Liver Failure • Sickle Cell Disease • Transplantation

Allogeneic hematopoietic cell transplantation for infantile osteopetrosis: High rates of sinusoidal obstruction syndrome/veno-occlusive disease and transplant-related morbidity (ASH 2025) - "Busulfan-based conditioning is widely used in myeloablative regimens in osteopetrosis alloHCT, increasing SOS/VOD risk in an already at-risk population...4/24 (17%) patients received prophylactic defibrotide...In this population, we demonstrated a high retrospective incidence of criteria fulfillment for diagnosis of VOD/SOS at 71% and 42% for Modified Seattle and Baltimore diagnostic criteria respectively, as well as a low 3-year overall survival of 36%. Our findings reinforce the need to optimize conditioning regimens as well as the approach to complications in the post-transplant setting."

Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Transplantation

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia (ASH 2025) - " CLIMB-111 is an ongoing 2-y, Phase 3 trial of exa-cel in TDT pts aged 12-35 y. The primaryendpoint is transfusion independence defined as proportion of pts maintaining a weighted averagehemoglobin (Hb) ≥9 g/dL without RBC transfusion for ≥12 consecutive months (m; TI12)...Consistent with that, there were 7 cases(7/56; 12.5%) of hepatic veno-occlusive disease; none resulted in end-organ dysfunction, all were relatedto busulfan and all resolved after defibrotide treatment... Exa-cel demonstrated durable clinical benefit for up to 6 y in adults and adolescents withTDT. After exa-cel, iron was successfully removed by IRT with no evidence of iron reaccumulation after IRTcessation. This suggests that in addition to durable TI in 98% of subjects, exa-cel potentially preventstissue iron deposition by restoring iron homeostasis via correction of underlying IE."

Clinical • Beta-Thalassemia • Genetic Disorders • Hepatology • ERFE

Inotuzumab ozogamacin in refractory or 1st or greater relapse of pediatric B-ALL as bridge to cure: A single center experience (ASH 2025) - "CD19-directedtherapies including chimeric antigen receptor T-cell therapy (CART) and blinatumomab have rescuedmultiply relapsed B-ALL patients. This single institution study shows that full-dose InO leads to MRD negative CR in relapsed/refractorypatients with B-ALL, serving as an effective bridge to subsequent therapy with CART, BMT, or CART+BMT.Over half of these patients are still alive, further demonstrating the use of InO as a bridge from diseaseto consolidative therapy. While VOD is a common concern after InO treatment, in our cohort only 2patients had defibrotide-treated VOD, both following BMT. Further adverse event evaluation is ongoing.In our small cohort the t(1; 19) translocation appears to be a negative prognostic indicator for response toInO; future studies should further investigate this association."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Hepatology • Leukemia • Pediatrics • CD22

Inotuzumab ozogamicin leads to high rates of measurable residual disease negativity for patients with B-cell acute lymphoblastic leukemia in morphologic remission (ASH 2025) - "All pts received ursodiol forhepatic sinusoidal obstruction syndrome (SOS) prophylaxis...Twenty-four pts (65%) received prior blinatumomab, 4 pts (11%) previouslyreceived CAR T, and 5 pts (14%) had previously undergone allogeneic stem cell transplant (ASCT)...In responding pts who achieved MRD-negativity (either by FCM orNGS) to InO, for pts who underwent ASCT vs. no ASCT, the 3-year OS rate was 71% vs. 56%, respectively,(p=0.6), with 3-year EFS rates of 57% vs. 44%, respectively (p=0.43).Most adverse events were low grade; SOS occurred in 3 pts (8%): 1 pt with Ph-negative ALL developedSOS 2 weeks after ASCT and recovered with defibrotide; 2 pts with Ph-positive ALL developed SOS whileon concomitant ponatinib.ConclusionInO results in high rates of MRD-negativity in pts with B-cell ALL and positive MRD, with clearance of MRDby FCM and NGS achieved in the majority of pts. Our data support the use of InO as an effective and safemethod of MRD-eradication, especially in..."

Clinical • IO biomarker • Residual disease • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Hepatology • Leukemia • Renal Disease • Septic Shock • ABL1

Oral iptacopan demonstrates efficacy as salvage therapy for high-risk transplant-associated thrombotic microangiopathy: First real-world experience (ASH 2025) - "Recent advances in complement inhibition have demonstrated promising therapeutic potential, asevidenced by eculizumab (Jodele S, et al...Elevated urinary protein excretion (rUPCR ≥1 mg/mg) and elevated sC5b-9level were present in 10 and 13 patients, respectively.The treatment before iptacopan included calcineurin inhibitor withdrawal (n=18), plasma exchange (n=7),defibrotide administration (n=4), anti-CD20 therapy (n=11), and anti-C5 therapy (n=9)... The oral complement inhibitor iptacopan demonstrated significant clinical efficacy as salvage therapy inhigh-risk TA-TMA patients, particularly when administered as second-line therapy, with infections beingthe most common adverse events. These findings support further prospective evaluation of iptacopan asa targeted therapy for TA-TMA."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Infectious Disease • Meningococcal Infections • Transplantation • CFB

Impact of oral eicosapentaenoic acid on veno-occlusive disease/sinusoidal obstruction syndrome onset prevention: Single-center retrospective analysis (ASH 2025) - "Prophylactic agents such as ursodeoxycholic acid anddefibrotide have shown preventive benefits, though their efficacy remains limited.Eicosapentaenoic acid (EPA) is an omega-3 fatty acid that possesses numerous beneficial effects,including the reduction of triglyceride levels. Thirteen deaths occurred in the EPA group andten in the no-EPA group within 100 days.ConclusionsIn summary, the findings of our study suggest that oral EPA administration can safely prevent the onsetof VOD/SOS after allo-HSCT. To validate this hypothesis, a prospective multicenter clinical trial is required."

Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cerebral Hemorrhage • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Otorhinolaryngology

A case report of veno-occlusive disease (VOD) in a patient with metastatic triple-negative breast cancer receiving trastuzumab deruxtecan. (SABCS 2025) - "We present a rare case of VOD that occurred in a patient with breast cancer receiving T-DXd.Case Presentation: A 45-year-old female with triple-negative breast cancer developed a rapid metastatic recurrence following neoadjuvant chemo-immunotherapy, then rapid progression while on first-line sacituzumab govitecan...The two most implicated ADCs are gemtuzumab ozogamicin and inotuzumab ozogamicin, utilized in hematologic malignancies. Management of VOD includes defibrotide, a thrombolytic agent that stabilizes the endothelium. On review of the literature, there are reports of VOD occurring after treatment with trastuzumab emtansine, another ADC used in breast cancer... We present a rare case of VOD after receipt of T-DXd in a heavily pretreated patient with metastatic breast cancer."

Case report • Clinical • Metastases • Breast Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer

Inotuzumab ozogamicin-associated sinusoidal obstruction syndrome/veno-occlusive disease diagnosed by transjugular liver biopsy (PubMed, Rinsho Ketsueki) - "The patient died of liver failure progression that showed no improvement with defibrotide. Pathological examination at autopsy revealed enlarged endothelial cells and fibrosis of the central hepatic vein. In cases where the diagnosis of SOS/VOD is inconclusive based on clinical findings and HokUS scores, TJLB may facilitate earlier diagnosis and effective therapeutic decision-making for SOS/VOD."

Journal • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Fibrosis • Hematological Malignancies • Hepatology • Immunology • Leukemia • Liver Failure • Oncology

Prediction of Sinusoidal Obstructive Syndrome after Allogeneic Stem Cell Transplantation Using Liver Stiffness Measurement By Fibroscan (ASH 2023) - "5/7 AML pts received a median of 4 (2-5) lines of chemotherapy before HSCT; 2/4 sAML pts got azacytidine/ venetoclax and all 4 MF pts received Ruxolitinib before HSCT...In 6/11 pts with LSM ≥7.2 kPa, the conditioning was modified; Treosulfan was given instead of BU (n=3), non-DAC instead of DAC protocol (n=3), reduced intensity instead of MAC (n=4), methotrexate was omitted (n=2). One pt received Defibrotide prophylaxis... Pts with a high risk for VOD may have a high baseline LSM. In these pts, modifying the planned conditioning and GVHD prophylaxis regimens to less hepato-toxic ones may reduce the incidence of subsequent VOD. LSM elevation after HSCT should increase awareness of SOS, while pts with hyperbilirubinemia and low LSM usually suffered from other causes of liver toxicity."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Fibrosis • Gastroenterology • Genetic Disorders • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Liver Cirrhosis • Lymphoma • Myelodysplastic Syndrome • Myelofibrosis • Obesity • Oncology • Portal Hypertension • Transplantation

Still You Hesitate to Use Ino?: Inotuzumab Ozogamicin Optimized Post-Transplant Outcomes in R/R Ph-Negative B-ALL (ASH 2024) - "The standard chemotherapy (SC) group was defined to include patients who received neither InO nor blinatumomab (Blina)...All patients received SOS-specific treatment including defibrotide or recombinant human thrombomodulin...ConclusionOur results encourage the salvage chemotherapy with InO before allogeneic HCT especially in Ph-negative R/R B-ALL patients. Compared to SC, InO had a higher remission induction rate, better post-transplant prognosis, and controllable post-transplant SOS."

Post-transplantation • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Hepatology • Leukemia • Oncology • Transplantation • CD22

Phase II Study of Inotuzumab Ozogamicin for the Treatment of Measurable Residual Disease-Positive B-Cell Acute Lymphoblastic Leukemia: 3-Year Update (ASH 2024) - "Furthermore, MRD negativity by NGS was defined as undetectable MRD with a sensitivity of ≥ 1x10-6 when applicable All patients received ursodiol for hepatic sinusoidal obstruction syndrome (SOS) prophylaxis...Seventeen patients (57%) had received prior blinatumomab...SOS was seen in three patients : one pt with Ph-negative ALL developed SOS 2 weeks following transplant (recovered after defibrotide therapy), and two pts both with Ph-positive ALL on concurrent ponatinib (of whom one died). Conclusion : INO resulted in high survival and MRD negativity rates in most patients with B-cell ALL and persistent MRD or MRD recurrence, with a favorable safety profile."

P2 data • Residual disease • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Hepatology • Leukemia • Oncology • ABL1

Evaluation of Circulating Endothelial Cells (CECs) As Marker of Endothelial Damage in Allo-Transplanted Patients at High Risk of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS): The Cecinvod Study (ASH 2023) - "Pts receiving TBI-based regimen were more likely to develop VOD compared to those receiving Treosulfan (10 to 14 g/m2) or Busulfan ev (9...After defibrotide treatment, the CEC levels increased in the first week, while they progressively decreased during the VOD treatment (T6 and T7, -50,7% and -71,5%, respectively)...We show that CECs can be considered reliable marker of endothelial damage in alloSCT pts, highlighting the impact of previous treatments, the conditioning regimen, and allo-SCT itself. Increased CEC level may be helpful to confirm VOD diagnosis, as well as their monitoring may be useful to evaluate the response to the treatment for VOD."

Clinical • Bone Marrow Transplantation • Hepatology • Otorhinolaryngology • Transplantation • ENG • MCAM • PTPRC

Systematic Literature Review of Incidence and Management of Non-HCT-Related Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) (ASH 2023) - "One study reported outcomes for 206 children who received supportive care for VOD/SOS during 6-thioguanine therapy for ALL; only three pts had acute hepatic failure and all pts recovered from VOD/SOS. Non-HCT VOD/SOS occurs in diverse disease areas, including hematologic and solid tumor cancers. A lack of consensus regarding VOD/SOS diagnosis in the non-HCT setting may lead to underdiagnosis; therefore, clinicians should be vigilant for VOD/SOS even in non-HCT pts. Though defibrotide is approved for post-HCT VOD/SOS, there is no approved therapy for non-HCT VOD/SOS; future trials should focus on diagnosis and treatment outside the HCT setting, which represents a significant unmet need."

Review • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hepatology • Infectious Disease • Liver Failure • Nephrology • Otorhinolaryngology • Solid Tumor • Wilms Tumor

Transplant Associated Thrombotic Microangiopathy: Acomprehensive Review and Local Experience (ASH 2023) - "eculizumab, a humanized antibody against complement protein, can be highly effective in patients with TA-TMA...Other available treatment options include rituximab and defibrotide. Other therapeutic agents are under clinical trials such as ravulizumab (C5 inhibitor), nomacopan (C5 and leukotriene B4 inhibitor) and narsoplimab (mannan-binding lectin-associated serine protease-2 [MASP-2] inhibitor)...Thus far, the choice is to individualize therapy according to comorbidities, severity of clinical presentation and availability of the treatment options. ConclusionTA-TMA remains a significant clinical challenge for transplant physicians, and more research is needed to improve our understanding of its pathogenesis, diagnosis, and management, particularly in guiding the choice of therapy."

Review • Anemia • Bone Marrow Transplantation • Cardiovascular • Graft versus Host Disease • Hematological Disorders • Hypertension • Immunology • Infectious Disease • Thrombocytopenia • Transplantation

A Phase II Study to Evaluate the Safety and Efficacy of Defibrotide in Children with High Risk Kawasaki Disease (IND 127812) (ASH 2023) - P2 | "Current treatment is directed at reducing inflammation with IVIG and aspirin. This preliminary data suggests defibrotide is safe in children with HR KD failing IVIG. Further patients will be required to determine the safety and preliminary efficacy of defibrotide in children with HR KD. Supported in part by a research grant from Jazz Pharmaceuticals."

Clinical • P2 data • Bone Marrow Transplantation • Cardiovascular • Heart Failure • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Oncology • Pulmonary Disease • Respiratory Diseases • Vasculitis

Incidence and Risk Factors of Veno-Occlusive Disease Are Different in Younger Versus Older Adults Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (ASH 2023) - "However, the interaction of historic risk factors in the current era, particularly with the increasing use of calicheamicin-based therapies and post-transplant cyclophosphamide as GVHD prophylaxis, remains unclear...VOD cases were defined using the classic VOD EBMT criteria or if patients received defibrotide treatment for VOD based on clinical judgment of the treating physician...Forty-nine (3%) patients received inotuzumab ozogamicin (InO) containing regimens prior to allo-SCT, and 30 (2%) were exposed to gemtuzumab ozogamicin (GO)... In summary, our data highlight that young adults (age 18 – 25 years) represent a distinct adult population with increased rates of VOD compared to their older counterparts, which are exacerbated by disease and treatment related factors (DRI and number lines of therapy). In contrast, patients > 25 years of age had low rates of VOD even in the presence of historical predictors, with only hepatic risk factors identified as increasing..."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • Transplantation

Sinusoidal Obstruction Syndrome (SOS) Biomarkers-Derived Predictive Model to Individualize SOS Preemption in Patients Undergoing Allogeneic Stem Cell Transplant (ASH 2023) - "The SOS biomarker-based model offers a new method to guide risk-adapted prophylactic or preemptive therapy for SOS."

Biomarker • Clinical • Predictive model • Hepatology • Transplantation

Successful Treatment Outcomes of Hematopoietic Stem Cell Transplantation with Reduced-Toxicity Conditioning Regimen Incorporating Treosulfan in Pediatric Patients with XIAP Deficiency (ASH 2023) - "Results Since January 2016, 4 patients with XIAP deficiency had undergone HSCT using RTC regimen consisted of fludarabine (150–180 mg/m2), treosulfan (42 g/m2), rabbit ATG (5–7.5 mg/kg), and thiotepa (10 mg/kg)...One patient who received HFD HSCT developed acute GI GVHD grade 3 and VOD within 1 month from HSCT, which was recovered after treatment with systemic steroid and defibrotide...Conclusions HSCT with treosulfan based RTC regimen is promising treatment option for pediatric patients with XIAP deficiency, with successful engraftment and low treatment-related toxicity. However, further studies including a larger multicenter trial and measures to refine the regimen are mandate to verify efficacy and safety of this regimen."

Clinical • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Gastrointestinal Disorder • Graft versus Host Disease • Hemophagocytic lymphohistiocytosis • Immunology • Inflammatory Bowel Disease • Pediatrics • Rare Diseases • Transplantation • XIAP

Terlipressin for Sinusoidal Obstruction Syndrome: Expanding Indications Beyond Cirrhotic Hepatorenal Syndrome (KIDNEY WEEK 2025) - "Case Description A 45-year-old woman with B-cell acute lymphoblastic leukemia underwent matched related donor allogeneic HCT following inotuzumab ozogamicin exposure, placing her at high risk for SOS. She received reduced-intensity conditioning and post-transplant cyclophosphamide...Prior to dialysis initiation, she received terlipressin (0.85 mg IV q6h) and continuous bumetanide infusion...Despite standard therapy with defibrotide and diuretics, she progressed to severe fluid overload and impending dialysis...As dialysis in HCT carries >80% mortality, early reversal is critical. This is the first reported case of successful terlipressin use in SOS-AKI."

Acute Kidney Injury • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Hepatology • Hypotension • Leukemia • Liver Failure • Nephrology • Portal Hypertension • Renal Disease

Patient Characteristics and Outcomes of Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease in Allogeneic Stem Cell Transplant Patients - a Multicenter Canadian Study (ASH 2024) - "Twelve patients received inotuzumab and five received gemtuzumab...Intravenous (i.v.) busulfan (Bu) was administered to 330 patients; median dose 860 mg...Treatment with ursodeoxycholic acid was used in 93% of cases. Graft versus host prophylaxis included i.v. methotrexate [65%], calcineurin inhibitor [99%] and anti-thymocyte globulin [85%]...Of the nine patients treated with defibrotide graded as moderate [n=2], severe [n=4], and very severe [n=3], seven died with 3 deaths before day 100...Hospital and ICU stays were longer in VOD patients who were more likely to die within the first 100 days. VOD may be under-diagnosed and this underscores the need for ongoing education and resources to allow for early intervention."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myelofibrosis • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases • Transplantation

Incidence, Clinical Risk Factors, and Biomarkers of SOS/VOD Following Allogeneic HSCT in Adults: A Real-Life Study By the Spanish Group of Hematopoietic Stem Cell Transplantation and Cell Therapy (GETH-TC) (ASH 2024) - "Defibrotide was used in 45 pts (55.6%)...Univariate analysis identified significant clinical risk factors for SOS/VOD diagnosis, including advanced disease (≥2nd CR or relapse) (p=.026), non-HLA-matched donor (p=.016), ≥2nd HSCT (p 1.5 mg/dL/>26 μmol/L) (p=.034), prior use of gemtuzumab ozogamicin or inotuzumab ozogamicin (p=0.037), elevated levels of tacrolimus/sirolimus (>10 ng/mL) (p=.003) or INR (>1.5) (<.0001) before SOS/VOD diagnosis, baseline (d0) EASIX ≥2 (p= .032) and EASIX ≥ 6 on d+7 (p <.001) and on d+14 (p <.001)...EASIX score at d0, d+7 and d+14, was validated as predictor of SOS/VOD onset. These findings warrant confirmation in a prospective study."

Biomarker • Clinical • Bone Marrow Transplantation • Hepatology • Transplantation

Sinusoidal Obstruction Syndrome in Children with CD22+ B-Cell Precursors Acute Lymphoblastic Leukemia (BCP-ALL) Treated with Inotuzumab Ozogamicin in Trial ITCC-059: Risk Factors and Outcomes (ASH 2024) - P2 | "In phase IA, phase II and stratum III, patients received single agent InO; while in phase IB InO was combined with vincristine and dexamethasone...Overall, 14 (13%) patients developed SOS, 2 during treatment, 10 after HSCT, and 2 after subsequent chemotherapy including high-dose methotrexate and cyclophosphamide...All SOS cases received treatment with defibrotide, except one patient in which SOS resolved with supportive management...The time between last InO and HSCT seems a significant risk factor for developing SOS, as in previous studies in adults treated with gemtuzumab ozogamicin. Taking into account also the longer median half-life of InO in pediatrics (T1/2 423 h,17.6 days; Trial NCT02981628), a minimum interval of 35 days after last InO dose and HSCT could be considered based on these data."

Clinical • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Hepatology • Leukemia • Oncology • Pediatrics • Septic Shock • CD22

Resolution of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) with Defibrotide Following HCT in Adult and Pediatric Patients: Pooled Analysis of Defifrance and EBMT PASS Registries (ASH 2023) - "Day 100 survival was higher in patients with VOD/SOS resolution versus without, regardless of severity, highlighting the importance of VOD/SOS resolution. The safety profile of defibrotide in the real-world setting was consistent with reports from previous studies, supporting the utility of defibrotide treatment per the prescribing information."

Retrospective data • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Otorhinolaryngology • Pediatrics • Pulmonary Disease • Respiratory Diseases

A Phase II Study to Evaluate the Safety and Efficacy of Defibrotide and Changes in Plasma Biomarkers in Sickle Cell Disease-Related Acute Chest Syndrome (IND 127812) (ASH 2023) - P2 | "Our data suggests that defibrotide is safe and well tolerated in SCD patients with ACS. sICAM-1, Ang2, IL-6, and PLA2G2A may serve as important biomarkers in SCD associated ACS. A randomized phase II/III multi-center clinical trial will need to be conducted to further investigate efficacy of defibrotide in patients with SCD-associated ACS."

Biomarker • Clinical • P2 data • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Pediatrics • Sickle Cell Disease • Transplantation • ICAM1 • IL6 • PLA2G2A