ursodeoxycholic acid / Generic mfg. - 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

Long-term outcomes of a chemotherapy-free regimen of concomitant blinatumomab and inotuzumab ozogamicin in older patients with Philadelphia negative B-cell acute lymphoblastic leukemia (ASH 2025) - "Pts received the following treatment during cycle 1: dexamethasone 20 mgintravenously (IV) days 1-4, vincristine 1 mg IV day 4, fractionated InO 0.6 IV mg/m2 day 1 and 0.3 IVmg/m2 day 8 followed by blina IV days 15-28 (9 ug/day x 2 days followed by 28 ug/daily) followed by 2-week rest...Pts with CD20 positive ALL could receive rituximab 375 mg/m2 IV on days 2 and 9during cycles 1-4. Pts received 12 doses of intrathecal chemotherapy and ursodiol...There were no episodes of veno-occlusive disease.ConclusionA largely chemotherapy-free regimen of blina in combination with InO for older or unfit pts was toleratedand resulted in high rates of MRD-negative remission with 2-year survival rates of 50%. Most deaths areattributable to non-relapse mortality, suggesting that continued optimization of treatment regimens areneeded for older pts with B-cell ALL."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Disorders • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Leukopenia • Myocardial Infarction • Neutropenia • Respiratory Diseases • Thrombocytopenia • CD20 • CRLF2 • KMT2A

Consolidation with CAR T-cell therapy may abrogate the need for allogeneic stem cell transplant in patients with relapsed B-cell acute lymphoblastic leukemia after receiving dose-dense mini-hyper-CVD with inotuzumab ozogamicin and blinatumomab (ASH 2025) - "Pts received mini-hyper-CVD in combination with InO and blina in a dose-dense fashion: cycles 1, 3,5 consisted of mini-hyper-CVD (cyclophosphamide 150 mg/m2 D1-3, vincristine 2mg D4, dexamethasone20 mg D1-4, InO 0.6 mg/m2 D2 and 0.3 mg/m2 D8 [of C1], InO 0.3 mg/m2 D2 and 0.3 mg/m2 D8 [of C3],blina 9ug D4-5 and 28 ug D6-21 [of C1] and blina 28 ug D4-21 [of C3 and C5); cycles 2, 4, 6 consisted ofmini-MTX and ara-C (MTX 250 mg/m2 D1, cytarabine 500 mg/m2 q12 hours D2-3, InO 0.3 mg/m2 D2 and0.3 mg/m2 D8 [of C2 and C4], blina 28 mg D4-21), with Peg-G-CSF administered after each course. Ptsreceived 12 IT chemotherapy administrations and rituximab if CD20 positive. All pts received ursodiol...Proceeding with CAR T consolidation leads to impressive outcomes with a 2-year EFSand OS rates of close to 90%. Such strategy is being evaluated in the frontline setting."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Pneumonia • Respiratory Diseases • Septic Shock • Transplantation • ABL1 • CD20 • TP53

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

The addition of inotuzumab ozogamicin to frontline hyper-CVAD and sequential blinatumomab leads to durable survival outcomes in adults with newly diagnosed B-cell acute lymphoblastic leukemia: Four-year follow-up of a phase 2 study (ASH 2025) - "Pts received Hyper-CVAD alternating with high-dosemethotrexate (MTX) and cytarabine (Ara-C) for up to 4 cycles, followed by 4 cycles of blina...Ofatumumab 2000 mg or rituximab 375 mg/m2 wasadministered in pts with CD20>1%...Pts received 12 ITchemotherapies and ursodiol.ResultsAs of July 2025, 75 pts were treated (38 without InO [cohort 1] and 37 with InO [cohort 2])...With death as a competing risk, the 4-year cumulative incidenceof relapse (CIR) rate was 18% for cohort 1 and 8% for cohort 2 (p = 0.177) and 4-year cumulativeincidence of death without relapse rate was 2% and 0% (p = 0.083), respectively.MVA for OS and EFS demonstrates that the administration of InO was the only favorable predictive factorfor OS (no InO HR 12.5; p=0.02) and borderline for EFS (no InO HR 3.15; p=0.06).There were no episodes of veno-occlusive disease.ConclusionFor pts with ND Ph-negative B-ALL, the addition of InO to Hyper-CVAD plus blina leads to promisingsurvival outcomes with the..."

Clinical • P2 data • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Hepatology • Leukemia • CD20 • CD22 • CRLF2 • KMT2A

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

Efficient production of ursodeoxycholic acid via a deep eutectic solvent-enabled 7α- and 7β-hydroxysteroid dehydrogenase cascade system with enhanced solubility of 7-keto-lithocholic acid. (PubMed, Int J Biol Macromol) - "Additionally, high-level UDCA production was demonstrated, with a 92 % conversion rate and a final titer of 46 g/L from 50 g/L CDCA. These findings highlight the potential of DES to enhance enzymatic cascade bioprocesses involving insoluble substrates or intermediates, offering a robust and scalable platform for the efficient biosynthesis of UDCA."

Journal

Advances in Pharmacological Activities, Biosynthesis, and Structural Modification of Ursodeoxycholic Acid (UDCA): A Review. (PubMed, Drug Des Devel Ther) - "In this paper, the pharmacological activities, biosynthesis and structural modification of UDCA were systematically discussed. It is believed that with the deepening of research, more UDCA derivatives with better medicinal properties will become drugs and better serve human health."

Journal • Review • Hepatology

Plasmapheresis for Suspected Drug-Induced Liver Injury During Pregnancy: A Multidisciplinary Diagnostic and Therapeutic Challenge. (PubMed, J Clin Med) - "The drug was discontinued and symptomatic treatment with ursodeoxycholic acid and prednisone was implemented with marginal effect. Plasmapheresis treatment was safely and effectively used for the first time in the therapeutic process in a pregnant patient with DILI. Interdisciplinary cooperation of specialists with gastroenterology, nephrology, and obstetrics expertise is crucial to achieving a timely diagnosis and begin effective treatment for pregnant patients with acute liver injury."

Journal • Review • Cholestasis • Gynecology • Hematological Disorders • Hepatology • Hypertension • Liver Failure • Nephrology • Obstetrics

Randomized Controlled Trial Evidence on Peroxisome Proliferator-Activated Receptor (PPAR) Agonists in Primary Biliary Cholangitis: A Systematic Review and Meta-Analysis. (PubMed, Int J Hepatol) - "Ursodeoxycholic acid (UDCA) is the first-line treatment, while obeticholic acid (OCA) serves as a second-line option because of moderate UDCA nonresponsiveness and cirrhosis-related concerns. There was no significant difference between PPAR agonists and placebo for ALT level (SMD = -0.93, 95%CI = -1.94 to 0.08; p = 0.07, I2 = 95%), AST level (SMD = -0.01, 95%CI = -0.67 to 0.66; p = 0.99, I2 = 91%), and pruritus (RR = 0.77, 95%CI = 0.29 to 2.06; p = 0.60, I2 = 34%). Our study found a superior efficacy of PPAR agonists compared with placebo for change in ALP, GGT, TBil, and Tg levels, highlighting the potentially beneficial effect of PPAR agonists on liver health."

Journal • Retrospective data • Review • Dermatology • Fibrosis • Gastroenterology • Hepatology • Immunology • Liver Cirrhosis • Primary Biliary Cholangitis • Pruritus

Chemotherapy Free Regimen of Inotuzumab Ozogamicin and Blinatumomab in Frontline Therapy of Older Patients with Philadelphia Negative B-Cell Acute Lymphoblastic Leukemia (ASH 2024) - "Treatment consisted of Dexamethasone intravenous (IV) 20 mg (Day) D1-D4 and vincristine 1 mg IV on D4 with fractionated InO 0.6 mg/m2 on D1 and 0.3 mg/m2 on D8, in Cycle (C) 1...CNS prophylaxis with alternating IT methotrexate and cytarabine was administered for 12 doses...All pts received ursodeoxycholic acid prophylaxis...11 pts (79%) received rituximab...Conclusion : A chemotherapy minimized combination of InO and Blina leads to promising response and survival outcomes in older pts with newly diagnosed B-ALL and appears tolerable. Longer follow up to assess safety, continued efficacy and incidence of secondary myeloid neoplasms is needed in this population."

Clinical • IO biomarker • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Cardiovascular • CNS Disorders • Hematological Disorders • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Movement Disorders • Myocardial Infarction • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • CD20 • CRLF2 • KMT2A • TP53

Phase 2 Trial of Mini-Hyper-CVD Plus Inotuzumab Ozogamicin, with or without Blinatumomab, in Older Patients with Newly Diagnosed Philadelphia Chromosome-Negative B-Cell Acute Lymphoblastic Leukemia (ASH 2023) - "Patients received mini-Hyper-CVD (mHCVD, cyclophosphamide 150mg/m2 q12h D1-3, dexamethasone 20mg daily D1-4, 11-14, and vincristine 2mg D1, 8 alternating with methotrexate (MTX) 250mg/m2 D1 & cytarabine 500mg/m2 q12h D2, 3) for up to 8 cycles. Eight doses of intrathecal (IT) MTX /cytarabine were given for CNS prophylaxis; patients with CNS disease had IT hydrocortisone, MTX, and cytarabine twice weekly till CNS clearance, then weekly x4. Eight doses of rituximab 375mg/m2 were given on D1, 8 of C1-4 if CD20 was ≥20% by flow cytometry...Ursodeoxycholic acid was given to all patients...Maintenance was initially with vincristine 2mg D1, prednisolone 50mg daily D1-5, 6-mercaptopurine 50mg BID & MTX 10mg/m2 weekly (POMP) for three years...Conclusion Older patients with ND Ph-negative ALL treated with mHCVD plus InO, with or without blina had excellent response rates and deep remissions. However, further adjustment of the regimen is needed, especially in patients ≥70..."

Clinical • P2 data • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • B Acute Lymphoblastic Leukemia • CNS Disorders • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Septic Shock • CD20 • TP53

Updated Results of the Combination of Mini-Hyper-CVD with Inotuzumab Ozogamicin and Blinatumomab in Patients with Relapsed/Refractory B-Cell ALL (ASH 2024) - "All pts received mini-HCVD (cyclophosphamide and dexamethasone at 50% dose reduction, no anthracycline, methotrexate at 75% dose reduction, cytarabine at 0.5 g/m2 x4 doses) and INO...CD20+ pts received rituximab on D2 and D8 of the first 4 cycles. All pts received Ursodiol for the prevention of hepatic sinusoidal obstruction syndrome (SOS)...Introducing Blina and fractionating INO seem to improve the safety and efficacy of this combination. Using a D-D approach resulted in high rates of deep and early MRD responses and promising survival outcomes, which may be better than the sequential use of these agents."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Hepatology • Leukemia • Oncology • CD20 • CRLF2 • TP53

Low Intensity Mini-Hypercvd (mHCVD), Inotuzumab Ozogamicin (Ino) with/without Blinatumomab (Blina) in Older Patients with Newly Diagnosed Philadelphia Negative B-Cell Acute Lymphoblastic Leukemia (B-ALL): 10 Years Update (ASH 2024) - "The chemotherapy backbone was mHCVD (mHCVD : cyclophosphamide 150mg/m2 q12h [day] D1-3, dexamethasone 20mg/day D1-4, 11-14, and vincristine 2 mg D1, 8; alternating with methotrexate (MTX) 250mg/m2 D1 & cytarabine 500mg/m2 q12h D2, 3) for up to 8 cycles (C). 12 doses of intrathecal IT MTX /cytarabine were given for CNS prophylaxis; pts with CNS disease had IT hydrocortisone, MTX, and cytarabine 2/week till CNS clearance, then weekly x 4. 8 doses of rituximab 375mg/m2 were given on D1, 8 of C1-4 if CD20 was ≥20% by flow cytometry (FCM)...Ursodeoxycholic acid was given to all pts...Maintenance was initially with vincristine 2mg D1, prednisolone 50mg daily D1-5, 6-mercaptopurine 50mg BID & MTX 10mg/m2 weekly (POMP) for 3 years...Conclusion : mHCVD-InO ± Blina is an efficacious and tolerable regimen with promising PFS and OS benefit even on long-term F/U. Further reduction of chemo doses and assessment of mutations that increase risk of myeloid neoplasms,..."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • CNS Disorders • Epilepsy • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology • CD20 • CRLF2 • KMT2A • TP53

THEUDCA: UDCA to Prevent Post-TIPS Hepatic Encephalopathy (clinicaltrials.gov) - P=N/A | N=270 | Not yet recruiting | Sponsor: West China Hospital

New trial • CNS Disorders • Hepatic Encephalopathy

Clinical characteristics of newly diagnosed patients with primary biliary cholangitis (PBC) indicate the need for better awareness on timely diagnosis and adequate UDCA therapy. (PubMed, Z Gastroenterol) - "Guidelines on primary biliary cholangitis (PBC) recommend therapy with 13-15 mg/kg ursodeoxycholic acid (UDCA) and assessment of treatment response after 12 months...UDCA dosage is suboptimal in many cases. Time point of diagnosis and UDCA dosage should be improved."

Journal • Cardiovascular • Diabetes • Fibrosis • Hepatology • Hypertension • Immunology • Metabolic Disorders • Primary Biliary Cholangitis • Pulmonary Arterial Hypertension

Effectiveness and tolerability of bezafibrate in primary biliary cholangitis - a nationwide real-world study. (PubMed, Am J Gastroenterol) - "In this real-world nationwide study, the one-year fibrate discontinuation rate was substantial. However off-label use of bezafibrate was associated with reductions in the biochemical markers of cholestasis in PBC, which were associated with clinical outcome in this setting of second line therapy."

Journal • Real-world evidence • Cholestasis • Hepatology • Immunology • Liver Failure • Primary Biliary Cholangitis • Transplantation

Antiviral and virucidal activities against SARS-CoV-2 and antibacterial properties of bile acids and their salts with naturally occurring organic cations of l-carnitine, creatinine, and choline. (PubMed, RSC Adv) - "Four bile acids, lithocholic acid (LCA, 1), deoxycholic acid (DCA, 5), ursodeoxycholic acid (UDCA, 9), and chenodeoxycholic acid (CDCA, 13), were used to form salts with l-carnitine [X], creatinine [Y], and choline [Z], which are naturally occurring compounds...Cholinium salts of these bile acids exhibited enhanced antibacterial activity; for example, 41.4-41.5% antibacterial improvement was observed for the [DCA][Z] (8) salt when compared with its corresponding bile acid DCA (5). This work provides evidence that certain salts of bile acids have improved antibacterial activity, but they do not enhance antiviral properties."

Journal • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases

Gut microbiota-derived metabolites in immunomodulation and gastrointestinal cancer immunotherapy. (PubMed, Front Immunol) - "Bile acids display dual roles, with ursodeoxycholic acid and tauroursodeoxycholic acid counteracting the tumor-promoting effects of deoxycholic acid and lithocholic acid. These platforms enable quantitative assessment of exposure-response thresholds, dissection of context-dependent effects, and in vitro pre-evaluation of the feasibility and safety of metabolite-based immunologic adjuvants combined with PD-1/PD-L1 blockade. Collectively, microbiota-derived metabolites represent promising targets for precision diagnosis and treatment in GI cancer immunotherapy."

Journal • Review • Gastrointestinal Cancer • Immunology • Oncology • Solid Tumor • CD8

Pembrolizumab-Induced Secondary Cholangitis: A Case Report. (PubMed, Cureus) - "The patient responded well to the combination of ursodeoxycholic acid (UDCA), corticosteroid therapy and mycophenolate mofetil (MMF), showing gradual improvement in liver enzyme levels and significant improvement in abdominal pain. Immune-related cholangitis such as this is crucial to identify as distinct from infective cholangitis, as treatment is primarily immunosuppression. It adds to the growing body of evidence of immune-related cholangitis as a specific adverse reaction, encouraging and enabling more research into this to guide future clinical decision-making and the formation of treatment guidance and diagnostic criteria."

Journal • Gastrointestinal Disorder • Hepatology • Immunology • Liver Failure • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Pain • Solid Tumor

Tumor necrosis factor alpha-induced protein 3: Biomarker discovery and therapeutic advancement in primary biliary cholangitis. (PubMed, World J Hepatol) - "Tumor necrosis factor alpha-induced protein 3 (TNFAIP3) was examined in this study as a novel biomarker to predict the efficiency of ursodeoxycholic acid (UDCA) and thereby improved primary biliary cholangitis (PBC) treatment...In conclusion, TNFAIP3 and fatigue have significant impact on UDCA in PBC. These findings provide a new view on PBC pathophysiology and suggest that TNFAIP3 may be a suitable biomarker or therapeutic target for the disease."

Biomarker • Journal • Fatigue • Hepatology • Immunology • Oncology • Primary Biliary Cholangitis • TNFAIP3

Triple autoimmune overlap syndrome of the liver: a rare case of concomitant PBC, PSC, and AIH. (PubMed, Future Sci OA) - "A 51-year-old woman initially diagnosed with AIH-PBC overlap presented persistent hepatocellular cytolysis despite corticosteroids and ursodeoxycholic acid...Due to azathioprine intolerance and hepatotoxicity, therapy was switched to mycophenolate mofetil with UDCA and low-dose corticosteroids, resulting in biochemical stabilization...It underscores the importance of integrating serology, histology, and advanced imaging for accurate diagnosis and individualized therapy. In the absence of standardized guidelines, management relies on tailored immunosuppression and supportive care to prevent progression toward liver failure."

Journal • Autoimmune Hepatitis • Fibrosis • Hepatology • Immunology • Inflammation • Liver Failure • Primary Biliary Cholangitis

Metabolomic changes associated with treatment response of neoadjuvant chemotherapy with TEC regimen in HER2-negative breast cancer. (PubMed, Front Pharmacol) - "This study aimed to characterize time-dependent metabolic alterations and identify metabolites associated with treatment response in HER2-negative breast cancer patients undergoing neoadjuvant chemotherapy (NAC) with the TEC regimen (docetaxel, epirubicin, and cyclophosphamide)...Following six cycles, elevated epinephrine levels were positively associated with therapeutic efficacy, while increased cysteine levels were linked to unfavorable outcomes...Combined analysis of ursodeoxycholic acid and cysteine improved the predictive performance for treatment response. These findings reveal dynamic metabolic reprogramming during NAC and suggest that ursodeoxycholic acid and cysteine may serve as potential predictive biomarkers of therapeutic efficacy in HER2-negative breast cancer patients treated with the TEC regimen."

Journal • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • HER-2

ELSPIRE: A Study of Elafibranor in Adults With Primary Biliary Cholangitis and Inadequate Response or Intolerance to Ursodeoxycholic Acid. (clinicaltrials.gov) - P3 | N=69 | Active, not recruiting | Sponsor: Ipsen | Trial completion date: Oct 2026 ➔ Jun 2026 | Trial primary completion date: Oct 2026 ➔ Jun 2026

Trial completion date • Trial primary completion date • Hepatology • Immunology • Primary Biliary Cholangitis

A rare cause of neonatal cholestasis: congenital portosystemic shunt. (PubMed, Proc (Bayl Univ Med Cent)) - "Bilirubin and liver function enzymes gradually decreased after initiation of ursodeoxycholic acid therapy. In newborns presenting with hypoglycemia, cholestasis, and thrombocytopenia, initial investigations are usually performed for metabolic diseases, congenital infections (toxoplasmosis, cytomegalovirus, rubella, herpes simplex virus), and genetic causes. As these tests may take time to return, early radiological imaging should be pursued to evaluate for anatomical abnormalities."

Journal • Cholestasis • Cytomegalovirus Infection • Hematological Disorders • Hepatology • Herpes Simplex • Hypoglycemia • Infectious Disease • Metabolic Disorders • Rubella • Thrombocytopenia