Jakafi (ruxolitinib) / Novartis, Incyte - LARVOL DELTA

The MAGIC of VEXAS : A case of overlap between Bechet's disease and VEXAS syndrome. (EULAR 2025) - "He responded well to prednisone and methotrexate (MTX)...MTX was discontinued, colchicine and azathioprine were initiated achieving remission. In 2019, he was diagnosed with pulmonary embolism, treated with high-dose corticosteroids and cyclophosphamide, followed by a maintenance therapy with infliximab and azathioprine, achieving remission...Treatment included corticosteroids and Azacitidine reducing transfusion dependency...Tocilizumab was attempted but discontinued due to an anaphylaxis-like reaction. Sarilumab was initiated after ruxolitinib was denied by health care provider...Ongoing management focuses on systemic disease control and addressing hematologic complications. Learning points for clinical practice: Key lesson from this case was the decision to further explore VEXAS in an already diagnosed Bechet’s patient, as well as treatment consequences of this diagnosis."

Clinical • Anemia • Cardiovascular • Dermatitis • Dermatology • Hematological Malignancies • Hypertension • Immunology • Infectious Disease • Musculoskeletal Diseases • Myelodysplastic Syndrome • Ocular Inflammation • Oncology • Ophthalmology • Pulmonary Embolism • Rare Diseases • Respiratory Diseases • Rheumatology • Uveitis • Vasculitis

Ruxolitinib Is an Effective Therapy for Ciltacabtagene Autoleucel-Associated Parkinsonism in Multiple Myeloma. (PubMed, J Hematol) - "However, in cilta-cel-associated parkinsonism, dopamine transporter imaging is normal, rendering traditional agents such as carbidopa/levodopa ineffective. As CAR T-cell therapy for multiple myeloma is expanding and moving to earlier lines, the need to optimize therapy for parkinsonism, a potentially life-threatening complication, becomes more urgent. This report presents the first documented cases of two patients with immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome and cilta-cel-associated parkinsonism, effectively treated with ruxolitinib."

Journal • CNS Disorders • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Immunology • Movement Disorders • Multiple Myeloma • Oncology • Parkinson's Disease • Rare Diseases

Frontline to Future: Considering the Role of Ruxolitinib Therapy in the Expanding cGvHD Landscape : Episode 2: A Review of Approved Therapies for SR cGvHD (Targeted Oncology) - "A panelist discusses how four FDA-approved agents treat steroid-refractory cGVHD, including ibrutinib (65% response rate), belumosidil (74% response rate), ruxolitinib (50% vs 25% in phase 3 trial), and exotilimab (74% response rate with durable responses lasting 17.2 months median failure-free survival)."

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JAK inhibitor withdrawal causes a transient pro-inflammatory cascade: A potential mechanism for major adverse cardiac events. (PubMed, PLoS One) - "Type I JAKinibs paradoxically accumulate functionally defective pJAK. Upon withdrawal, the primed pJAKs are de-repressed and initiate a pSTAT signaling cascade, resulting in high interferon and urokinase. Type II JAKinibs do not cause pJAK accumulation, pSTAT cascade, and subsequent pro-inflammatory transcripts. The resultant cytokines and proteins produced from this cascade might explain adverse cardiac outcomes. Thus, JAKinib withdrawal is a possible mechanism contributing to the major adverse cardiac events described with JAKinib therapy."

Adverse events • Journal • Immunology • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology • IFNG

Ruxolitinib targets STAT1-STAT3 cooperatively in large granular lymphocytic leukemia. (ICML 2025) - No abstract available

Hematological Malignancies • Leukemia • Lymphoma • Oncology • STAT1 • STAT3

GOLIDOCITINIB MONOTHERAPY IN THE TREATMENT OF REFRACTORY/RELAPSED INDOLENT T/NK-CELL LYMPHOMA: PRELIMINARY RESULTS FROM T-LGLL COHORT (ICML 2025) - "Immunosuppressive therapy remains the cornerstone of first-line treatment, encompassing corticosteroids, methotrexate, cyclosporine A (CsA), and cyclophosphamide...All six patients were relapsed or refractory to standard immunosuppressive therapy, among whom two patients experienced treatment failure from previous three lines of multi-agents therapy, including linperlisib (PI3Kδ inhibitor) and ruxolitinib (JAK1/2 inhibitor)...Preliminary results of golidocitinib monotherapy demonstrated encouraging anti-tumor activity and favorable safety profile in R/R T-LGLL. Further data with additional patients and follow-up are required."

Monotherapy • Cutaneous T-cell Lymphoma • Hematological Malignancies • Indolent Lymphoma • Leukemia • Lymphoma • Natural Killer/T-cell Lymphoma • Oncology • T-Cell Large Granular Lymphocyte Leukemia • PIK3CD

Clinical Characterization of Patients with Myositis Associated with Checkpoint Inhibitors (ICI-Myositis) (EULAR 2025) - "Additional therapies included ruxolitinib (9 patients), mycophenolate (6 patients), intravenous immunoglobulins (15 patients), plasmapheresis (2 patients), intravenous abatacept (8 patients), and intravenous tocilizumab (1 patient). ICI-induced myopathies often manifest early after initiating ICI therapy, particularly in severe cases, and are associated with a high mortality rate (31% in our series). Mortality due to ICI-myositis is concentrated in the initial weeks following symptom onset, with advanced age and ventilatory insufficiency caused by diaphragmatic dysfunction identified as key prognostic factors. The use of rapidly acting immunosuppressive agents, such as corticosteroids or ruxolitinib, along with optimal monitoring of respiratory and cardiac function and timely supportive measures, appears to be the most effective treatment strategy for these patients."

Checkpoint inhibition • Clinical • Gastrointestinal Disorder • Heart Failure • Immunology • Musculoskeletal Diseases • Musculoskeletal Pain • Myasthenia Gravis • Myositis • Oncology • Pain • Respiratory Diseases • Ventricular Tachycardia

IL-1 inhibition in patients with VEXAS syndrome – a retrospective international multicenter study (EULAR 2025) - "Demographic and clinical data, including genetic data, medication use and dose (prednisone, immunosuppressive, biologics and targeted synthetic medications), response to treatment and adverse events were collected anonymously from medical files. Eventhough VEXAS was first described more than 4 years ago, the best treatment approach remains unknown. Drug survival of canakinumab was higher than that of anakinra – with survival in patients receiving the 300 mg/month dose of more than 3 years on average, while in the 150 mg/month dose and anakinra group – survival was ~6 months. This is probably due to a lower adverse event rate and a higher rate of response in the canakinumab group."

Retrospective data • Anemia • Dermatology • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Myelodysplastic Syndrome • Neutropenia • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • CRP

Comparing JAK Inhibitors and Standard Therapies in Refractory Myopathies: Impact on Cardiovascular, Malignancy, and Disease Activity Outcomes (EULAR 2025) - "The cohorts were defined by treatment exposure: patients receiving JAK inhibitors (tofacitinib, ruxolitinib, abrocitinib, upadacitinib, filgotinib, or baricitinib) were compared to those receiving standard therapies (methotrexate, azathioprine, IVIG, rituximab, or mycophenolate mofetil). JAK inhibitors demonstrated significant benefits in reducing MACE, stroke, CAD, heart failure, thrombosis, vascular disease, malignancy, and CK-based disease activity in patients with DM, ASS, and IMNM compared to standard therapies. However, these advantages, the higher all-cause mortality observed in the JAK inhibitor cohort underscores the importance of careful risk stratification and close monitoring. These findings highlight the therapeutic potential of JAK inhibitors in refractory myopathies, warranting further investigation in randomized controlled trials to validate these results and refine treatment strategies for inflammatory myopathies."

Atherosclerosis • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Dermatomyositis • Diabetes • Heart Failure • Metabolic Disorders • Myositis • Oncology • Rare Diseases • Solid Tumor • Thrombosis

Unmasking a Genetic Predisposition: A Case of Refractory Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome in a Patient with Neurodevelopmental Delay (EULAR 2025) - "Stabilized on 30 mg prednisone, she was transferred to our facility...Pulse dose steroids were started, followed by anakinra, which proved to be ineffective and led to worsening transaminitis...Tocilizumab at 8 mg/kg/day for three consecutive days led to some improvement but caused profound neutropenia...This highlights the growing role of genetic testing in identifying rare contributors to complex autoinflammatory syndromes. Ruxolitinib is a therapeutic option in severe or refractory HLH."

Clinical • Neurodevelopmental • Acute Kidney Injury • Anemia • Autism Spectrum Disorder • Cardiovascular • CNS Disorders • Developmental Disorders • Fragile X Syndrome • Genetic Disorders • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Neutropenia • Oncology • Pneumonia • Psychiatry • Rare Diseases • Renal Disease • Respiratory Diseases • Thrombocytopenia • Vasculitis

Primary HLH is a significant Cause of Haemophagocytic Lymphohistiocytosis in Young Adults: Findings from a Single-Centre Cohort Study (EULAR 2025) - "Despite treatment with corticosteroids, anakinra, IVIG and etoposide, four cases were refractory to these first-line therapies. Three patients subsequently received a JAK inhibitor (1 patient baricitinib, two patients ruxolitinib) with both patients receiving Ruxolitinib noted to have improvement in fevers and cytopenias. One patient continued ruxolitinib in combination with ciclosporin until undergoing allogeneic BMT 15 months after diagnosis...The patient with CNS-HLH, was deemed unfit for BMT, they were additionally managed with ruxolitinib, high-dose anakinra and intrathecal methotrexate but developed new inflammatory brain lesions and irreversible brain damage after their anakinra dose was weaned and were palliated. The patient with XIAP deficiency, who presented with EBV-related HLH, responded well to therapy with corticosteroids and rituximab, with no relapses... pHLH accounted for 3.6% of adult HLH cases, with higher prevalence (12.8%) in younger adults. This may..."

Clinical • Bone Marrow Transplantation • CNS Disorders • CNS Lymphoma • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Hepatology • Immunology • Infectious Disease • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Rare Diseases • Vascular Neurology • IL2RA • PRF1 • RAB27A • TNFRSF9 • XIAP

Jakafi: "For patients with anemia at myelofibrosis diagnosis who received ESAs or danazol in combination with ruxolitinib, spleen and symptom response rates were similar to those reported in the total JUMP study population"; Myelofibrosis (Incyte) - EHA 2025

P3 data • Hematological Malignancies • Myelofibrosis • Oncology

A phase 2 study of itacitinib alone or in combination with low-dose ruxolitinib in patients with myelofibrosis. (PubMed, Leuk Res) - "Overall, 8 of 23 patients enrolled achieved SVR at week 24; larger average changes in SVR at week 12 were observed for itacitinib monotherapy vs. the combination. No unexpected safety signals were observed."

Journal • P2 data • Fatigue • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Thrombocytopenia • JAK1

Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial. (ASCO 2025) - P3 | " An economic evaluation based on transfusion-related data in pts treated with PAC or BAT (including ruxolitinib [RUX] and erythroid support [ES]) from the PERSIST-2 trial (NCT02055781). The reduction in transfusion rates associated with PAC treatment relative to BAT is projected to result in decreased transfusion-related medical cost and time burden for pts with MF and anemia."

Clinical • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Myelofibrosis • ACVR1 • IRAK1 • JAK1

Combined MEK, STAT3 and PD-1 Inhibition in Metastatic Pancreatic Ductal Adenocarcinoma (clinicaltrials.gov) - P1 | N=28 | Completed | Sponsor: Peter Hosein, MD | Active, not recruiting ➔ Completed | Trial completion date: Sep 2027 ➔ May 2025

Trial completion • Trial completion date • Oncology • Pancreatic Adenocarcinoma • Pancreatic Cancer • Pancreatic Ductal Adenocarcinoma • BRCA1 • BRCA2 • MSI • TMB

Similar Splenic, Symptom Responses Are Reported With Ruxolitinib Combos in Myelofibrosis With Anemia (Cancer Network) - P3b | N=2,233 | JUMP (NCT01493414) | Sponsor: Novartis Pharmaceuticals | "Spleen and symptom response rates remained similar for patients with myelofibrosis and anemia who were given erythropoiesis-stimulating agents (ESAs) or danazol, both in combination with ruxolitinib, compared with the overall patient population in the phase 3b JUMP trial (NCT01493414), according to results from a post hoc analysis of the study that were presented at the European Hematology Association Congress 2025. For patients with a hemoglobin (Hb) of less than 12.0 g/dL (n = 101), the spleen length response rate at 12 weeks was 41.6% and 36.6% at 24 weeks. For those with an Hb of less than 10 g/dL (n = 52), the spleen length response rate at 12 weeks was 42.3% and 34.6% at 12 weeks. For reference, the spleen length response in the JUMP trial at 12 weeks was 39.1% and 31.5% at 24 weeks."

P3 data • Myelofibrosis

VAX4FRAIL: National Project on Vaccines, COVID-19 and Frail Patients (clinicaltrials.gov) - P=N/A | N=747 | Completed | Sponsor: Azienda USL Reggio Emilia - IRCCS | Recruiting ➔ Completed | N=1300 ➔ 747

Enrollment change • Trial completion • CNS Disorders • Hematological Malignancies • Infectious Disease • Novel Coronavirus Disease • Oncology • Rheumatology • Solid Tumor • Squamous Cell Skin Cancer

Treatment patterns and blood count control in 10,112 patients with polycythemia vera. (PubMed, Expert Rev Hematol) - "Of a total 10,112 patients, most received phlebotomy (68.1%) or hydroxyurea (HU; 28.2%) as initial treatment, with median follow-up of 32.1 (IQR, 13.5-58.5) months and 31.5 (IQR, 16.8-54.9) months, respectively. Additionally, 54.2% of patients who switched to ruxolitinib achieved white blood cell counts < 11 × 109/L, and 57.5% achieved platelet counts ≤ 400 × 109/L after 1 year of ruxolitinib treatment. This real-world evidence highlights the importance of considering alternative therapies for patients whose initial treatment regimen does not provide adequate clinical benefit."

Journal • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

Ruxolitinib for Treatment of Graft Versus Host Disease after Liver Transplantation-Case Report and Review of Literature. (PubMed, J Clin Exp Hepatol) - "Image 1."

Journal • Graft versus Host Disease • Hepatology • Immunology • Transplantation

From Stem Cells to Synergy: A High-Throughput Platform for Inborn Errors of Immunity – STAT1-GoF as Proof-of-Concept" (EAACI 2025) - "Synergy assays with ruxolitinib demonstrated combination indices <0.7, indicating strong synergistic effects...Beyond STAT1-GoF, this modular system—leveraging CRISPR-edited reporters and high-throughput screening—can be tailored to study diverse IEI-causing mutations. By bridging traditional herbal medicine with precision pharmacology, our work provides a scalable strategy to accelerate personalized treatments for patients with IEI, highlighting its broad applicability across the spectrum of these disorders."

Late-breaking abstract • Immunology • STAT1

Ruxolitinib for refractory lung granulomatosis in two pediatric patients. (PubMed, Rheumatology (Oxford)) - No abstract available

Journal • Pediatrics • Rare Diseases • Vasculitis

New topical molecular targeted therapies for atopic dermatitis in children: A systematic review and meta-analysis. (PubMed, Pediatr Allergy Immunol) - "Nine studies (reported in eight articles) involving 2182 patients were included, with 1469 children treated with Janus kinase inhibitors (ruxolitinib and delgocitinib) and phosphodiesterase-4 inhibitors (crisaborole, lotamilast, and difamilast)...New topical targeted therapies administered over 4 weeks are effective and safe for children with atopic dermatitis aged ≤18 years. Further studies are needed to establish their long-term safety and efficacy."

Clinical • Journal • Retrospective data • Review • Atopic Dermatitis • Dermatitis • Dermatology • Immunology

Ruxolitinib add-on to corticosteroids for patients with severe checkpoint inhibitor-related pneumonitis:an interim analysis (ERS 2025) - No abstract available

Checkpoint inhibition • Clinical • Inflammation • Oncology • Pneumonia

A Phase I/II Trial of Ruxolitinib with Chemotherapy for Patients with Relapsed and/or Refractory Philadelphia-like Acute Lymphoblastic Leukemia. (PubMed, Clin Lymphoma Myeloma Leuk) - P2 | "Continued efforts should focus on identifying optimal treatment strategies for this high-risk group of patients."

Journal • P1/2 data • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • CRLF2 • EPOR • HMBOX1 • JAK2 • NUP214

Ropeginterferon Alfa 2b Plus Ruxolitinib for Myelofibrosis (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: The University of Hong Kong | Not yet recruiting ➔ Recruiting

Enrollment open • Monotherapy • Myelofibrosis