Jakafi (ruxolitinib) / Novartis, Incyte - LARVOL DELTA

Use of Jak-inhibitors in a tertiary level pediatric pulmonary department (ERS 2026) - "Patients, treated with baricitinib (n=5), ruxolitinib (n=4), or tofacitinib (n=1), were monitored at least every 3 months for clinical response (oxygen requirement, disease stabilization/progression) and safety. JAKi may offer a broader therapeutic spectrum, enabling disease control and steroid spearing in otherwise refractory diseases that currently necessitate complex combination immunosuppressive therapies. We speculate that the benefit may be even better if JAKi will be introduced earlier-on."

Clinical • Graft versus Host Disease • Immunology • Inflammation • Interstitial Lung Disease • Pediatrics • Pulmonary Disease • Respiratory Diseases • Scleroderma • Systemic Sclerosis

The efficacy and safety of ruxolitinib plus corticosteroids versus corticosteroids for patients with severe checkpoint inhibitor pneumonitis: a Multicenter, Open-Label, Randomised Controlled Phase 2 Trial (ERS 2026) - P=N/A | "Background:Severe checkpoint inhibitor-related pneumonitis(CIP)is life-threatening.This study aims to evaluate the efficacy and safety of ruxolitinib plus corticosteroids in patients with severe CIP.In this investigator-initiated multicenter, randomized phase 2 trial, patients with G 3-4 CIP were randomized assigned (1:1) to receive glucocorticoids (C group) or add-on with ruxolitinib (R group).The primary endpoint was the proportion of patients achieving improvement to G1 CIP at week 8 with a prednisone dosage of ≤10mg/d.A total of 60 patients were enrolled from Apr 2023 to Nov 2025, with 20 patients (20/30, 66.7%) in R group and 11 patients (11/30, 36.7%) in C group reaching primary endpoint (p = 0.0379).In 2nd and 4th weeks, the proportion of patients improving to mild CIP (G0-2) in R group increased significantly(Fig 1).Repeated measures analysis demonstrated a significant overall treatment effect, with ruxolitinib being associated with significantly higher..."

Checkpoint inhibition • Clinical • P2 data • Diabetes • Infectious Disease • Inflammation • Musculoskeletal Diseases • Oncology • Pneumonia

Decitabine With Ruxolitinib, Fedratinib or Pacritinib for the Treatment of Accelerated/Blast Phase Myeloproliferative Neoplasms (clinicaltrials.gov) - P2 | N=25 | Recruiting | Sponsor: University of Washington | Trial completion date: Nov 2026 ➔ Nov 2027 | Trial primary completion date: Nov 2026 ➔ Nov 2027

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • Transplantation

Firstline Treatment with Ruxolitinib Versus Best Available Therapy in Patients with Polycythemia Vera: Pre-Specified Interim Analysis of the Randomized Phase 2b Ruxobeat Clinical Trial of the German Study Group for Myeloproliferative Neoplasms (GSG-MPN) (ASH 2023) - P2 | "Cytoreductive treatment with hydroxyurea (HU) or ropeginterferon-alpha is approved in EU for the treatment of high-risk patients (pts) with PV. In this interim analysis, first-line treatment with ruxolitinib for 6 months in high-risk pts with PV led to clinically meaningful improvements in overall response, hemoglobin and hematocrit, phlebotomy rates, splenomegaly, and patient-reported pruritus and fatigue severity, while BAT only improved platelet counts, WBC, hematocrit, and phlebotomy rates, without having an impact on symptoms."

Clinical • P2b data • Dermatology • Fatigue • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Pruritus

Histological Resolution of GVHD-Associated Immune Complex Glomerulonephritis After JAK1/2 Inhibition with Ruxolitinib Following Allogeneic HSCT (TTS 2026) - "This case provides, to our knowledge, the first histologically-confirmed documentation of immune complex clearance on serial biopsy following JAK1/2 inhibition for GVHD-associated MPGN post-HSCT. Ruxolitinib may suppress the cytokine-driven immune complex deposition underlying this entity and warrants further prospective investigation as a targeted therapy in this setting."

Beta-Thalassemia • Bone Marrow Transplantation • Glomerulonephritis • Graft versus Host Disease • Immunology • Lupus Nephritis • Nephrology • JAK1

IMproveMF update: Phase 1/1B trial of imetelstat (IME)+ruxolitinib (RUX) in patients (pts) with intermediate (INT)-1, INT-2, or high-risk (HR) myelofibrosis (MF). (ASCO 2025) - P1, P2 | "In part 1 of IMproveMF, no DLTs were observed and the RP2D dose of 9.4 mg/kg IME was determined. AEs were consistent with those observed in other IME clinical trials, and preliminary efficacy was positive, demonstrating the potential of IME+RUX in this pt population with high unmet needs. Part 2 of this trial is ongoing across the US at 6 sites."

Clinical • P1 data • Anemia • Fatigue • Hematological Disorders • Infectious Disease • Leukopenia • Myelofibrosis • Neutropenia • Pain • Pneumonia • Respiratory Diseases

Low rates of chronic graft-versus-host disease with ruxolitinib maintenance following allogeneic HCT. (PubMed, Blood) - P2 | "GVHD prophylaxis consisted of tacrolimus and methotrexate. Prolonged administration of ruxolitinib following HCT is associated with low rates of clinically significant cGVHD. The incorporation of JAK inhibition into GVHD prevention approaches warrants further investigation."

Journal • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Neutropenia • Thrombocytopenia • Transplantation • JAK1

Bromodomain and Extra-Terminal Inhibitor INCB057643 (LIMBER-103) in Patients with Relapsed or Refractory Myelofibrosis and Other Advanced Myeloid Neoplasms: A Phase 1 Study (ASH 2023) - P1 | "INCB057643 monotherapy (4 and 8 mg qd) and combined (4 and 6 mg qd) with ruxolitinib was generally well tolerated, whereas the 12 mg qd monotherapy dose caused 2 DLTs. There were no treatment-related fatal events. Improvements in spleen size and symptom burden were observed in patients receiving ≥8 mg in the monotherapy group and 4 mg in the combination therapy group."

Clinical • Metastases • P1 data • Anemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Pneumonia • Respiratory Diseases • Thrombocytopenia • MYC

SURVIVAL IMPACT AND KINETICS OF HEMOGLOBIN IMPROVEMENT WITH MOMELOTINIB IN PATIENTS WITH MYELOFIBROSIS AND MODERATE TO SEVERE ANEMIA: POST HOC ANALYSES OF SIMPLIFY-1 AND MOMENTUM (EHA 2025) - "SIMPLIFY-1 and MOMENTUM were randomized, double-blind, phase 3 trials of momelotinib (n=215) vs ruxolitinib (n=217) in JAK inhibitor-naive patients and momelotinib (n=130) vs danazol (n=65) in JAK inhibitor-experienced patients, respectively. Anemia severity at BL dictates the probability and kinetics of achieving Hb >10 g/dL with momelotinib. Patients with BL moderate anemia were numerically more likely to achieve this threshold and faster than those with BL severe anemia, underscoring the benefits of earlier anemia intervention with momelotinib to maximize clinical outcomes. Regardless of anemia severity, patients who achieved Hb >10 g/dL by week 24 with momelotinib had numerically longer OS than those who did not, validating achievement of Hb levels above this threshold as a positive prognostic factor."

Clinical • Retrospective data • Anemia • Hematological Disorders • Myelofibrosis • ACVR1 • JAK1 • JAK2

Etanercept in Steroid-Refractory Acute Graft Versus Host Disease: Clinical Response, Toxicity, and Long-Term Outcomes. (PubMed, Blood Cell Ther) - "Infections were the major cause of mortality, and early tapering of systemic steroids may be the key to improving outcomes. In the current ruxolitinib era, etanercept may hold a place in patients where ruxolitinib has failed or is not feasible."

Journal • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Infectious Disease • Transplantation

Ruxolitinib Versus Best Available Therapy in Patients with Essential Thrombocythemia: Pre-Specified Interim Analysis of the Randomized Phase 2b Ruxobeat Clinical Trial of the German Study Group for Myeloproliferative Neoplasms (GSG-MPN) (ASH 2022) - P2 | "Cytoreductive treatment with hydroxyurea (HU) or anagrelide is approved for the treatment of patients (pts) with ET. In this pre-specified interim analysis, treatment with ruxolitinib was not superior over BAT to induce complete response (using strict criteria for symptoms) in ET pts that were either untreated or not intolerant/resistant to prior therapy. However, RUX was more effective in reducing ET-associated spleen size and symptoms, such as headache and concentration problems. The RuxoBEAT trial is ongoing."

Clinical • P2b data • Cardiovascular • Dermatology • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Pain • Polycythemia Vera • Pruritus • Thrombocytosis

Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI (clinicaltrials.gov) - P1/2 | N=40 | Not yet recruiting | Sponsor: Peking University People's Hospital

New P1/2 trial • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation

PELABRESIB IN COMBINATION WITH RUXOLITINIB FOR JANUS KINASE INHIBITOR-NAIVE PATIENTS WITH MYELOFIBROSIS: 72-WEEK FOLLOW-UP WITH LONG-TERM EFFICACY OUTCOMES OF THE PHASE III MANIFEST-2 STUDY (EHA 2025) - P3 | "PELA+RUX showed sustained improvements over 72 weeks in splenic response, symptoms, SVR35/TSS50 dual response, BMF, and anemia vs PBO+RUX. The safety profile for Grade ≥3 TEAEs was similar and consistent across treatment arms, with the imbalance in leukemic transformation cases decreasing over time. Survival outcomes show a trend in favor of the PELA+RUX arm."

Clinical • Combination therapy • P3 data • Anemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelofibrosis • Oncology • Thrombocytopenia

TRANSFORM-1 Phase 3 study: Efficacy and safety of navitoclax plus ruxolitinib in patients with untreated myelofibrosis. (PubMed, Blood) - P3 | "NAV+RUX showed higher hematologic toxicity versus PBO+RUX (Grade 3/4 thrombocytopenia: 54.0% vs 19.2%; Grade 3/4 neutropenia: 40.3% vs 8.8%); diarrhea (any grade: 41.9% vs 16.8%) was also more common. Cytopenias were generally manageable and reversible with dose adjustments."

Journal • P3 data • Hematological Disorders • Myelofibrosis • Neutropenia • Oncology • Thrombocytopenia

Beyond the Bone Marrow: A Case Report of Extramedullary Hematopoiesis in Advanced Primary Myelofibrosis. (PubMed, Cureus) - "The patient received hydroxyurea, ruxolitinib, and palliative hepatosplenic radiotherapy, achieving transient clinical and biochemical improvement. However, he subsequently developed radiation-induced bone marrow aplasia and died from progressive disease. This case underscores the aggressive clinical course of advanced PMF when continuous disease-modifying therapy is interrupted and highlights the importance of timely access to targeted treatment and a multidisciplinary approach for managing extensive EMH and its life-threatening complications."

Journal • Acute Myelogenous Leukemia • Aplastic Anemia • Cardiovascular • Cholestasis • Chronic Eosinophilic Leukemia • Fibrosis • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Hepatology • Hypertension • Immunology • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Portal Hypertension

Updated Analysis of Overall Survival With Imetelstat in Relapsed or Refractory Myelofibrosis From IMbark Versus Real-World Data and Assessment of Real-World Treatment Patterns: Updated Post Hoc Analysis With Extended Follow-Up (SOHO 2026) - P2 | "RW treatment patterns, baseline/disease characteristics, and mOS of patients diagnosed before/after 2016 (cut point selected to match diagnosis period of IMbark population) and 2019 (fedratinib US approval) were compared. A more favorable OS benefit with imetelstat versus BAT in RW patients with R/R myelofibrosis and poor prognosis was observed. The significant improvement in OS in RW patients over the last decade was independent of transplant status, potentially due to shorter time from diagnosis to ruxolitinib start and earlier switch to next-line JAKi or clinical trial. Evolving treatment patterns are key considerations for interpreting future clinical trial outcomes."

Clinical • HEOR • Real-world • Real-world evidence • Retrospective data • Myelofibrosis • Oncology

Alphabeta T and B-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (TBdepl-haploHSCT) in Children with Myelodysplastic Syndromes (ASH 2021) - P1/2 | "Rituximab (200 mg/sqm) was administered on day -1 to prevent post-transplantation EBV-induced lymphoproliferative disorders (PTLD)...One patient developed moderate chronic GvHD [cumulative incidence 5.2% (95% CI 0-14.8)], which completely resolved with low-dose steroids and ruxolitinib... These data indicate that TBdepl-haploHSCT is a safe and effective transplant option also in children with MDS. Indeed, the low risk of both non-relapse mortality and acute/chronic GvHD makes this approach particularly attractive in the pediatric setting. Moreover, this haplo strategy compares favorably with T-cell replete approaches [Suo et al., 2020]."

Clinical • Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Juvenile Myelomonocytic Leukemia • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Pediatrics • Pulmonary Disease • Renal Cell Carcinoma • Respiratory Diseases • Transplantation

ELRITERCEPT (KER-050) DEMONSTRATED POTENTIAL TO TREAT MYELOFIBROSIS AND MITIGATE RUXOLITINIB-ASSOCIATED CYTOPENIAS IN THE PHASE 2 RESTORE TRIAL (EHA 2024) - P2 | "These data from RESTORE suggest that elritercept was generally well tolerated and has potential to treatseveral aspects of MF. Improvements in Hgb and transfusion burden with maintenance of plateletsdemonstrate potential to address ineffective hematopoiesis and treat cytopenias associated with MF andruxolitinib. Data also support potential for elritercept to reduce spleen size and improve symptoms."

P2 data • Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myelofibrosis • Oncology • Thrombocytopenia • ACVR2A • TGFB1

Safety and Efficacy of Fedratinib in Patients with Primary (P), Post-Polycythemia Vera (Post-PV), and Post-Essential Thrombocythemia (Post-ET) Myelofibrosis (MF) Previously Treated with Ruxolitinib: Primary Analysis of the FREEDOM Trial (ASH 2022) - P2, P3b | "Clinically relevant and durable spleen and symptom responses were observed in FREEDOM study participants, with a majority of responders showing durable SVR at data cutoff. This supports the use of fedratinib in pts with MF previously treated with RUX. One limitation of the study was small pt sample size."

Clinical • Anemia • CNS Disorders • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Myelofibrosis • Myeloproliferative Neoplasm • Novel Coronavirus Disease • Oncology • Polycythemia Vera • Thrombocytopenia • Thrombocytosis

PROGRESSIVE DYSPNEA WITH CHRONIC GRAFT-VS-HOST DISEASE LEADS TO A RUXOLITINIB-ASSOCIATED PULMONARY ALVEOLAR PROTEINOSIS DIAGNOSIS: A CASE REPORT (CHEST 2026) - No abstract available

Case report • Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Pulmonary Disease • Respiratory Diseases

Transform-1: A Randomized, Double-Blind, Placebo-Controlled, Multicenter, International Phase 3 Study of Navitoclax in Combination with Ruxolitinib Versus Ruxolitinib Plus Placebo in Patients with Untreated Myelofibrosis (ASH 2023) - P2, P3 | "This first randomized trial in JAKi-naïve MF with NAV + RUX combination led to an SVR35W24 rate that was twice as high as PBO + RUX (P<0.0001). The responses were durable; AEs of thrombocytopenia and anemia were common but manageable with dose modification without any clinically significant sequalae. Additional evaluation is ongoing."

Clinical • Combination therapy • P3 data • Anemia • B Cell Lymphoma • Fatigue • Fibrosis • Hematological Disorders • Immunology • Leukemia • Lymphoma • Myelofibrosis • Myeloproliferative Neoplasm • Neutropenia • Oncology • Thrombocytopenia • Thrombocytosis • BCL2 • BCL2L1 • BCL2L2

ACQUIRED PULMONARY ALVEOLAR PROTEINOSIS DUE TO RUXOLITINIB IN A PATIENT WITH GRAFT VS HOST DISEASE TREATED BY STAGED LOBAR LAVAGE (CHEST 2026) - No abstract available

Clinical • Graft versus Host Disease • Immunology • Respiratory Diseases

PDGFR-Targeted Ruxolitinib Liposomes Modulate Fibroblast-Associated Colorectal Cancer-Stroma Interactions, Reducing Invasion and Enhancing Chemosensitivity. (PubMed, ACS Omega) - "Taken together, these findings suggest that PDGFR-targeted liposomal delivery of Rux may affect fibroblast-associated tumor-stroma interactions, which could contribute to reduced colorectal cancer cell invasion and enhanced sensitivity to chemotherapy. Further studies are needed to clarify the underlying mechanisms and determine whether these effects are mediated through alterations in activated fibroblast functions."

Journal • Colorectal Cancer • Myelofibrosis • Oncology • Solid Tumor

Efficacy and Safety of Add-on Parsaclisib to Ruxolitinib Therapy in Myelofibrosis Patients With Suboptimal Response to Ruxolitinib: Final Results From a Phase 2 Study (ASH 2022) - P2 | "Final results from the phase 2 study demonstrate improvement in symptoms and SV with add-on parsaclisib in patients with MF having a suboptimal response to ruxolitinib. All daily dosing regimens were more efficacious than daily/weekly dosing. Combination therapy was associated with limited grade 3/4 AEs and TEAE-related discontinuations."

Clinical • P2 data • Endocrine Disorders • Fatigue • Gastroenterology • Gastrointestinal Disorder • Herpes Simplex • Immunology • Infectious Disease • Myelofibrosis • Myeloproliferative Neoplasm • Pneumonia • Polycythemia Vera • Pulmonary Disease • Respiratory Diseases • Thrombocytopenia • Thrombocytosis • PIK3CD

Janus Kinase Inhibitor Use After Liver Transplantation Is Associated With Sustained Malignancy and Thromboembolic Risk: A Propensity Score-Matched Real-World Cohort Study (ACG 2026) - "JAKi exposure included tofacitinib, ruxolitinib, upadacitinib, or baricitinib recorded on or after documentation of LT status. Among 74,576 adults with LT, 238 received JAKi and 74,338 did not. After PSM, 232 patients were retained in each cohort with balanced demographic and clinical characteristics. JAKi use was associated with higher malignancy risk at 1 year (23.3% vs 12.1%; HR 2.33, 95% CI 1.48â3.69; p< 0.001), which persisted at 5 years (26.7% vs 17.2%; HR 2.15, 95% CI 1.44â3.22; p< 0.001)."

Clinical • Real-world • Real-world evidence • Cardiovascular • Chronic Kidney Disease • CNS Disorders • Diabetes • Dyslipidemia • Genetic Disorders • Graft versus Host Disease • Hypertension • Immunology • Infectious Disease • Metabolic Disorders • Myeloproliferative Neoplasm • Myocardial Infarction • Nephrology • Obesity • Oncology • Transplant Rejection • Transplantation • Venous Thromboembolism