hydroxyurea / Generic mfg. - LARVOL DELTA

Prospera (ABNL-MARRO 002): A randomized phase 2 study of pacritinib vs. hydroxyurea in patients with advanced proliferative chronic myelomonocytic leukemia (CMML) (ASH 2025) - P2 | "In the phase 3 DACOTA trial (Itzykson et al., JCO 2022),decitabine modestly delayed leukemic transformation compared to HU but did not improve event-free oroverall survival and was associated with increased early mortality. The primary endpoint is CBR at Week 24, defined by modified IWG MDS/MPNcriteria incorporating hematologic improvement, spleen volume reduction, and symptom response.Secondary endpoints include CBR at any time, duration of response, event-free survival, leukemia-freesurvival, and overall survival.Correlative studies will assess treatment-related changes in clonal dynamics, cytokine signaling, andhematopoietic composition using longitudinal peripheral blood and bone marrow sampling. Single-celltranscriptomic and immunophenotypic profiling will be employed to characterize pacritinib's impact onboth the malignant clone and the surrounding immune microenvironment."

Clinical • Metastases • P2 data • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytopenia • IRAK1

Unexpected disseminated Mycobacterium kansasii in post-ET myelofibrosis on hydroxyurea (ESCMID Global 2026) - No abstract available

Myelofibrosis

Downregulation of the host ribonucleotide reductase subunit M2 via the Wnt/β-catenin pathway is a novel mechanism for duck plague virus replication and a potential antiviral target. (PubMed, Poult Sci) - "Functional studies revealed that RRM2 acts as a host restriction factor against DPV, as its overexpression inhibited viral genome replication and propagation, whereas its knockdown or inhibition with hydroxyurea (HU) promoted viral replication. In conclusion, our study unveils a novel strategy whereby DPV enhances its replication by hijacking the Wnt/β-catenin pathway to degrade the host metabolic enzyme RRM2. These findings not only deepen our understanding of DPV pathogenesis but also identify the Wnt/β-catenin-RRM2 axis as a promising target for developing novel antiviral interventions against duck plague."

Journal • Infectious Disease • Targeted Protein Degradation • RRM2

IFN-I-Mediated Transcriptional Reprogramming Drives Myeloid-Skewed Hematopoiesis in Sickle Cell Anemia. (PubMed, Am J Hematol) - "Importantly, hydroxyurea treatment attenuates IFN-I signaling in neutrophils, consistent with its therapeutic role in reducing excessive inflammation and granulopoiesis. Collectively, these findings uncover IFN-I-driven remodeling of hematopoiesis as a fundamental mechanism of leukocytosis and chronic inflammation in SCA, and establish a tractable therapeutic axis to mitigate innate immunity activation in this disease."

Journal • Anemia • Genetic Disorders • Hematological Disorders • Inflammation • Sickle Cell Disease • CSF3R

Monotherapy update from Phase 1 portion in Phase1/2 trial of the menin-MLL inhibitor enzomenib (DSP-5336) in patients with relapsed or refractory acute leukemia (ASH 2025) - P1/2 | "N=108 (93.1%) had AML and med prior regimen was 2 (1-9); 36 pts (31.0 %) had priorallogeneic stem cell transplant, 86 pts (74.1%) prior venetoclax...DSwas manageable with brief treatment interruption, corticosteroids and hydroxyurea as needed, with nodeaths, study discontinuations, or dose reductions due to DS... ENZO has been well tolerated with no DLTs in 116 pts. ENZO has low lipophilicity and highclearance, leading to a short half-life and has demonstrated a wide therapeutic window. This may allowdosing to be tailored to the specific biology of different AML subtypes."

Clinical • Monotherapy • P1/2 data • Central Nervous System Leukemia • Hematological Malignancies • Leukemia • HOXA9 • KMT2A • MEIS1 • NPM1 • NUP98 • TP53

Risk of Cutaneous Squamous Cell Carcinoma in Patients with Myelofibrosis Receiving Janus Kinase Inhibitors (AAD 2026) - "Across all patients, cSCC occurrence was significantly associated with skin cancer history (p = 0.001), but not with hydroxyurea exposure (p = 0.845), immunosuppression history (p = 0.454), or autoimmune disease (p = 1.000). Preliminary findings suggest higher cSCC occurrence among patients with myelofibrosis treated with JAKi compared to those who were not. This highlights the need for heightened dermatologic surveillance for JAKi-treated patients and may inform risk-benefit discussions when selecting systemic therapies."

Clinical • Genetic Disorders • Immunology • Myelofibrosis • Non-melanoma Skin Cancer • Oncology • Skin Cancer • Squamous Cell Carcinoma • Squamous Cell Skin Cancer

Left Ventricular Dysfunction in Patients with Sickle Cell Disease: Evaluating Genotypic Differences and Effects of Hydroxyurea in Youths (PAS 2026) - No abstract available

Clinical • Cardiovascular • Genetic Disorders • Heart Failure • Hematological Disorders • Sickle Cell Disease

Hydroxyurea Prescription Non-Dispensing Among Children with Sickle Cell Anemia (PAS 2026) - No abstract available

Clinical • Anemia • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Genomics in Child Health and the APS/SPR Mary Ellen Avery Neonatal Research Award (PAS 2026) - "It has also led to therapy outside of gene replacement, such as the CFTR modulars for cystic fibrosis (Trikafta), hydroxyurea for sickle cell disease or hereditary amyloidosis (tafamidis). Discuss the controversies around genome sequencing for universal newborn screening. Understand genetic mechanisms for increased susceptibility to Herpes Simplex infections."

Clinical • Amyloidosis • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Hematological Disorders • Herpes Simplex • Immunology • Infectious Disease • Respiratory Diseases • Sickle Cell Disease

Overcoming cytidine deaminase (CDA) mediated resistance via EO-4426 dual DNA-replication targeting: Implications for CDA-high solid tumors and mesenchymal GBM (AACR 2026) - "Unlike gemcitabine and hydroxyurea, EO-4426 resists cytidine deaminase (CDA)-mediated inactivation. New data are being developed to support further investigation of EO-4426 in cytidine metabolism-driven tumor contexts and biomarker guided clinical development strategies for CDA-high solid tumors and mesenchymal/APOBEC3G-enriched GBM. Comprehensive in vitro mechanistic data, biomarker correlations, and combination treatment findings will be presented at the Meeting."

Glioblastoma • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Ovarian Cancer • Solid Tumor • Triple Negative Breast Cancer • APOBEC3G • CDA

Integrated FHIT and IDH2 biomarkers predict synthetic lethal response to DCPS inhibition in acute myeloid leukemia (AACR 2026) - "In both in vivo and ex vivo systems, low FHIT expression emerged as a strong predictive biomarker of therapeutic response to DCPSi, while hydroxyurea pretreatment was identified as a negative confounding factor...Together, these findings establish DCPS as a safe and efficacious therapeutic target in AML. We propose that FHIT expression, frequently reduced in cancer, provides a biomarker-driven strategy for patient stratification, and that IDH2 mutation status further refines prediction of response to DCPS-targeted therapy."

Biomarker • Synthetic lethality • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FHIT • IDH2

A RHNO1-ATR/Chk1 positive feedback loop sustains the cellular DNA replication stress response (AACR 2026) - "RHNO1 knockdown sensitized cells to hydroxyurea (HU), increasing cell death and micronuclei formation...In summary, we demonstrated the RHNO1 promote critical OVC phenotypes and uncovered a novel positive feedback loop between RHNO1 and ATR/Chk1 signaling. This work identifies RHNO1 as a key component in maintaining the DRS response, highlighting its potential as a therapeutic target for cancers reliant on ATR/Chk1 signaling."

Oncology • Ovarian Cancer • Solid Tumor • CHEK1 • ERCC4

Nationwide epidemiologic and economic burden analysis of neurologic disorders associated with cancer treatment (AACR 2026) - "Higher mean incremental daily dose was significantly associated with increased CIND hazard, with oral agents showing HRs per 1-SD from 1.09-2.23 (bexarotene, capecitabine, hydroxyurea, mercaptopurine, tretinoin; all p≤0.046) and IV methotrexate showing HR 1.34 (95% CI 1.00-1.79; p=0.048). In this nationwide cohort, CIND is common, often delayed, and shaped by route of administration, indication, dose intensity, and patient comorbidity. CIND care concentrates substantial utilization and financial burden in chemotherapy exposed populations, underscoring the need for dose optimized regimens, proactive surveillance, and better supportive care strategies."

Clinical • HEOR • Oncology

Molecular characterization of inhibition of the DNA repair protein RAD51 by the small molecule JKYN-1 (AACR 2026) - "JKYN-1, the structure of which is a modification of IBR120, is 5-fold stronger as a single-agent inhibitor of proliferation...In hydroxyurea-treated human breast carcinoma MCF-7 cells, B02, an established RAD51 inhibitor (Mol Cancer Ther 20:1257-1269, 2021...Derivatives of JKYN-1 are currently being studied for improvements to pharmacokinetic properties. Funded by Sarissa, Inc., Breast Cancer Canada, and London Health Sciences Foundation."

Breast Cancer • Oncology • Osteosarcoma • Sarcoma • Solid Tumor • RAD51

CDK2 controls RIPK2 ubiquitination and stability in prostate cancer cells (AACR 2026) - "This study aims to address this knowledge gap by investigating the regulation of RIPK2 ubiquitination and degradation in PC cells, with a particular focus on CDK2, which we identified as a key mediator of RIPK2 regulation of the c-Myc oncoprotein. PC cells—22Rv1 (androgen receptor-positive) and PC3 (androgen receptor-negative)—were treated with two clinically evaluated, CDK2-selective inhibitors, INX-315 and PF-07104091...RIPK2 stability was assessed using cycloheximide (CHX) chase assays, and proteasomal degradation was evaluated with MG132 treatment...Cell cycle synchronization was achieved by serum starvation or treatment with Ro-3306, hydroxyurea, thymidine, or monastrol... Kinase-active CDK2 inhibits the K48-linked ubiquitination of RIPK2 and protects it from proteasomal degradation. This protection is potentially mediated by regulating the association between RIPK2 and its deubiquitinase YOD1 in PC cells. The findings support further investigation into the..."

Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor • AR • CDK2 • MYC • RIPK2

DNA fiber-based replication phenotypes distinguish WEE1-inhibitor response in high-grade endometrial cancer patient-derived organoids (PDOs) treated with azenosertib (AACR 2026) - "DNA-fiber assays measured: (i) fork speed (CldU+IdU as ongoing replication forks), (ii) replication fork stability under RS caused by hydroxyurea (HU), using the IdU:CldU ratio after WEE1i+HU, and (iii) single-stranded (ss)DNA gap formation via the S1 nuclease assay (WEE1i±S1). Four PDOs were profiled: DF4850 and DF4161 (WEE1i-sensitive; IC50s1500nM). In high-grade EC PDOs, azenosertib uniformly slows replication forks, but fork stability under HU and S1-detectable gap formation distinguish WEE1i-sensitive (DF4850, DF4161) from insensitive (DF042, DF4968) models. These mechanism-anchored DNA-fiber readouts merit prospective evaluation as predictive and pharmacodynamic biomarkers for WEE1 inhibitors in high-grade EC. Additional biomarker analyses, including immunohistochemical analyses of RS proteins, are ongoing and will be presented."

Clinical • Carcinosarcoma • Endometrial Cancer • Oncology • Sarcoma • Solid Tumor • TP53

Management of Right Heart Failure due to Acute Chest Syndrome Using VA ECMO: A Case Report (SCCM 2026) - "A growing body of evidence suggests VA ECMO may provide lifesaving support for patients with acute right heart failure secondary to ACS.Description: A 29-year-old female with a history of previously well controlled Hemoglobin S-zero thalassemia on hydroxyurea who presented with complaint of acute chest and back pain...While there is limited data on the use of VA ECMO in this population, the case demonstrates that it can be lifesaving. While VA ECMO with SCD presents unique challenges, it should be considered when a RV failure deteriorates despite aggressive supportive measures and exchange transfusion."

Case report • Clinical • Back Pain • Cardiovascular • Congestive Heart Failure • Genetic Disorders • Heart Failure • Hematological Disorders • Musculoskeletal Pain • Respiratory Diseases • Sickle Cell Disease • Thrombosis

Thrombotic Complications in Sickle Cell Anemia (THSNA 2026) - "Management strategies, such as antiplatelet therapy and anticoagulation, may help reduce the risk of thrombosis, and treatment regimens such as hydroxyurea, blood transfusions, and crizanlizumab show promising results. We have reviewed the complex mechanisms that promote thrombotic complications in SCA, discussing their pathophysiology, complications, and advances in targeted therapies to reduce them and improve patient outcomes. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Anemia • Cardiovascular • Cerebral Hemorrhage • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders • Inflammation • Nephrology • Renal Disease • Sickle Cell Disease • Venous Thromboembolism • HBB

ACUTE STEMI FROM CORONARY EMBOLISM IN A YOUNG MALE WITH POLYCYTHEMIA VERA (ACC 2026) - "He was started on aspirin, ticagrelor, statin, and ezetimibe; eptifibatide was stopped due to bleeding...Due to recurrent arterial events, apixaban was started, aspirin stopped, ticagrelor continued. Heart failure therapy and hydroxyurea were continued... PV can cause embolic STEMI without coronary disease. Early recognition and tailored therapy led to stable recovery."

Cardiovascular • Chronic Eosinophilic Leukemia • Chronic Kidney Disease • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hypertension • Myocardial Infarction • Polycythemia Vera • Thrombosis • JAK2

PROXIMAL LEFT ANTERIOR DESCENDING ARTERY THROMBUS WITH JANUS KINASE 2 POSITIVE ESSENTIAL THROMBOCYTOSIS AS LONE KNOWN RISK FACTOR (ACC 2026) - "This NSTEMI was most consistent with coronary thromboembolism from JAK2-positive ET. The patient became chest-pain free after aspiration thrombectomy of the mid LAD and 48 hours of intravenous heparin. She was discharged on DAPT with hematology follow-up to consider cytoreduction with hydroxyurea and to evaluate for acquired VWF deficiency."

Clinical • Atherosclerosis • Diabetes • Dyslipidemia • Eosinophilia • Essential Thrombocythemia • Gastroenterology • Hematological Disorders • Hypertension • Metabolic Disorders • Myocardial Infarction • Polycythemia Vera • Pulmonary Disease • Thrombocytosis • Thrombosis • JAK2

ACUTE CORONARY SYNDROME IN A YOUNG ADULT WITHOUT TRADITIONAL RISK FACTORS: A CASE OF JAK2-POSITIVE ESSENTIAL THROMBOCYTHEMIA (ACC 2026) - "ET was confirmed by bone marrow biopsy, and hydroxyurea was initiated alongside standard ACS therapy... ACS may be the presenting event of ET in young adults without traditional risk factors. Prompt recognition and hematology assessment can guide cytoreductive therapy and prevent recurrent thrombosis."

Clinical • Acute Coronary Syndrome • Atherosclerosis • Cardiovascular • Essential Thrombocythemia • Hematological Disorders • Myeloproliferative Neoplasm • Thrombocytosis • Thrombosis • JAK2

Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial. (ASCO 2025) - P3 | " SURPASS ET is an open-label, multicenter, Phase III trial comparing ropeg with anagrelide over 12 months in patients with ET who were hydroxyurea-resistant or-intolerant. Ropeg showed superior efficacy and safety compared to anagrelide as second-line therapy for ET. It represents a potential new therapeutic option for ET. Patient demographics and baseline characteristics.*n (%) presented for categorical variables."

P3 data • P3 data: top line • Essential Thrombocythemia • Hematological Disorders • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytosis • ABL1

IS THIS DÉJÀ VU? INTRA-PROCEDURAL THROMBUS FORMATION COMPLICATING PCI (ACC 2026) - "Case: We report a 46-year-old male with ET with a platelet count of 556,000µL on hydroxyurea and warfarin who presented with STEMI of the right coronary artery. This case underscores the critical importance of recognizing ET as a driver of catastrophic thrombotic complications during coronary interventions, highlighting the role of aggressive anticoagulation and real-time ACT monitoring in this high-risk population."

Acute Coronary Syndrome • Essential Thrombocythemia • Hematological Disorders • Myeloproliferative Neoplasm • Myocardial Infarction • Thrombocytosis • Thrombosis

A mixed-methods evaluation to optimize the existing hospital based sickle cell disease care model from a tribal area of Gujarat, India. (PubMed, J Public Health Res) - "Hydroxyurea was prescribed to 56% of eligible patients but clinical outcomes remained poor...Using an implementation science framework, this study optimized an SCD care model tailored to a marginalized tribal population. By strengthening outreach, care protocols, counseling, and digital tracking, the model addresses persistent delivery gaps, illustrating the value of evidence-informed, context-specific intervention refinement in resource-limited settings."

Journal • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Reproductive Health and Pregnancy Outcomes Among Women With Sickle Cell Disease in Tribal Communities of India: A Community-Based Retrospective Comparative Study. (PubMed, Pediatr Blood Cancer) - "Women with SCD in tribal settings face considerable reproductive and maternal health risks. Integrating reproductive health and antenatal services into SCD care is essential to reduce maternal and neonatal morbidity in high-burden, resource-limited regions."

Journal • Retrospective data • Genetic Disorders • Hematological Disorders • Pain • Sickle Cell Disease