hydroxyurea / Generic mfg. - LARVOL DELTA

Fmp45 promotes Rad53 dephosphorylation via Ptc2 interaction to attenuate checkpoint signaling and maintain genome stability. (PubMed, J Genet Eng Biotechnol) - "These findings elucidate a critical checkpoint-phosphatase coordination mechanism ensuring timely DNA damage recovery and genomic stability, identifying Fmp45 as a key regulator balancing DNA repair fidelity with cell cycle resumption following DSBs."

Journal • Immunology • CHEK2 • RAD51

Comparing the diagnosis, disease, and treatment experience of essential thrombocythemia from clinicians' and patients' perspectives: Insights from a qualitative interview study in the United States (ASH 2025) - "At the time of the interviews, all patients had received hydroxyurea, with 7 currently receiving it, including 6 as first-line therapy. Two were receiving ruxolitinib, and 1 was receiving peginterferon alfa-2a... This study uncovered several gaps between specialists' and patients' perspectives across the ET disease journey. Patients described more complex and prolonged diagnostic experiences than specialists reported. While specialists generally perceived patients with ET having minimal symptoms, patients reported persistent symptoms that significantly impacted their HRQoL."

Clinical • Interview • Acute Myelogenous Leukemia • Chronic Eosinophilic Leukemia • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Musculoskeletal Pain • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytosis • Thrombosis

Patient experience sub-study of a randomised UK multicentre Phase 2 trial of ASTX727 versus hydroxycarbamide/best supportive care in myelodysplastic (MDS)/myeloproliferative (MPN) overlap syndromes (AMMO) (ASH 2025) - "Results highlight variation in support/information needs, with desire for more robust communication, especially around personal trial completion/next steps, a recurrent theme. Other areas identified as key to optimising patient experience included coping/support mechanisms and financial toxicities; reaffirming the importance of trial sponsors making provision for cost reimbursement."

Clinical • P2 data • Cardiovascular • Hematological Malignancies • Infectious Disease • Mood Disorders • Myelodysplastic Syndrome

Understanding the essential thrombocythemia diagnosis journey, treatment experience, disease burden, and unmet need: Insights from a qualitative interview study with patients in the United States (ASH 2025) - "At the time of the interview, all 10 patients had been exposed to hydroxyurea (HU) with 6 patients receiving it as their first-line treatment. The remaining 4 were on second-line treatment, including 1 who was on HU after receiving anagrelide and others who had transitioned to alternative cytoreductive agents, including 2 on ruxolitinib and 1 on peginterferon alfa-2a following prior use of HU... Some patients reported persistent symptoms, including fatigue, which are associated with impaired HRQoL in areas of physical functioning, cognitive abilities, the ability to work, daily activities, and emotional well-being. Some patients identified prolonged diagnostic journey requiring multiple referrals and healthcare visits as well as seeking additional opinions and specialized care. Patients identified unmet needs in current treatments, namely poor symptom control, and significant side effects."

Clinical • Interview • Acute Myelogenous Leukemia • Chronic Eosinophilic Leukemia • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Mood Disorders • Musculoskeletal Pain • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytosis

Prevalence, treatment patterns, and complications of sickle cell disease in insured US populations: A retrospective study (ASH 2025) - "Use of disease-modifying therapies was limited, with 21.6% and 24.4% of patients using hydroxyurea (HU), 0.5% and 0.8% using L-glutamine, and 12.2% and 15.4% receiving chronic blood transfusions during the baseline and follow-up periods, respectively. Uptake of newer SCD therapies was low, with <1% of patients using crizanlizumab or voxelotor during either period... Treatment of SCD remains dominated by symptom management rather than disease-modifying therapy, as reflected by high utilization of as-needed medications and low uptake of newer therapies. The barriers to accessing disease-modifying therapies and targeted treatments warrant further investigation. Optimization of SCD management is needed to help slow disease progression and reduce complications."

Retrospective data • Asthma • Cardiovascular • Genetic Disorders • Hematological Disorders • Immunology • Infectious Disease • Renal Disease • Respiratory Diseases • Sickle Cell Disease

Impact of fatigue on health care resource utilization and costs in sickle cell disease in the United States (ASH 2025) - "Compared with those without fatigue, patients with fatigue were more likely to receive hydroxyurea treatment (31% vs 20%; P <.001) and transfusions (37% vs 19%; P <.001) and have higher baseline rates of comorbidities, most notably hypertension (33% vs 22%; P <.001) and chronic pulmonary disease (31% vs 21%; P <.001)... This study highlights the substantial and previously underrecognized economic burden of fatigue among patients with SCD. Presence of fatigue was associated with a significantly greater probability of annual HCRU and increased associated costs. These findings reinforce the need for effective and safe treatments for patients with SCD to address the unmet need of managing fatigue."

HEOR • Cardiovascular • Fatigue • Gene Therapies • Genetic Disorders • Hematological Disorders • Hypertension • Mood Disorders • Pulmonary Disease • Respiratory Diseases • Sickle Cell Disease

Delays in hydroxyurea initiation for patients with sickle cell disease in British Columbia (ASH 2025) - "Efforts should be made to improve access by addressing system-level barriers that lead to delays, thereby improving patient health outcomes and reducing healthcare costs. Addressing this disparity would advance equity, bridging the gap between patients with SCD and those with malignancies, and ensuring that access to an essential medication is determined by clinical need, not diagnosis or systemic bias."

Clinical • Genetic Disorders • Sickle Cell Disease

Impact of acute pain on health care resource utilization and costs in sickle cell disease in the United States (ASH 2025) - "Comparisons were adjusted for demographic characteristics (including age at index, sex, race, ethnicity, geographic region, and type of insurance coverage), baseline clinical characteristics, SCD-related comorbidities (including chronic pulmonary disease, asthma, and hypertension), and SCD treatments (including hydroxyurea, L-glutamine, voxelotor, crizanlizumab-tmca, and erythropoietin-stimulating agents) using multivariable regression models. Acute pain was associated with higher probability of 12-month HCRU and increased associated costs among patients with SCD, highlighting the considerable and previously underrecognized economic burden of acute pain in this population. These findings reinforce the necessity for new treatments to address the substantial unmet need in managing SCD-related acute pain."

HEOR • Asthma • Cardiovascular • Gene Therapies • Genetic Disorders • Hematological Disorders • Hypertension • Immunology • Pain • Pulmonary Disease • Respiratory Diseases • Sickle Cell Disease

Increased risk of vaso-occlusive crisis in patients with sickle cell disease treated with crizanlizumab: A real-world analysis using the trinetx database (ASH 2025) - "In this real-world matched cohort analysis, patients with HbSS sickle cell disease treated with crizanlizumab experienced consistently higher rates of VOCs compared to those receiving hydroxyurea. While crizanlizumab was designed to reduce VOC frequency, these findings raise important questions about its effectiveness in clinical practice. These results highlight the continued utility of hydroxyurea and underscore the need for further research to clarify the role of crizanlizumab and to identify which patients are most likely to benefit from its use."

Clinical • Real-world • Real-world evidence • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Efficacy of crizanlizumab in preventing vaso-oclusive crisis and hospitlalizations in sickle cell disease (ASH 2025) - "This study showed that crizanlizumab alone or with Hydroxyurea was associated with higher rates of VOCs and hospitalization than those not on Crizanlizumab, indicating inferior outcomes and supporting the need for further research into novel therapeutics for SCD."

Clinical • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Geographic and temporal trends in sickle cell disease hospitalizations in New York State (ASH 2025) - "Despite advances in treatment, including hydroxyurea and chronic transfusions, many individuals with SCD continue to experience frequent hospitalizations, prolonged lengths of stay, and increased mortality risk—trends often driven by racial, socioeconomic, and regional disparities... These findings underscore both geographic and temporal disparities in the severity and outcomes of SCD hospitalizations across New York State. The growing proportion of high-severity and high-mortality hospitalizations highlights the increasing clinical complexity and unmet care needs among individuals with SCD. Notably, major mortality cases rose from 3% in 2009 to 13% in 2022, while minor mortality classifications declined from 89% to 67%, and moderate mortality cases increased from 8% to 20% over the same period."

Clinical • Genetic Disorders • Hematological Disorders • Infectious Disease • Novel Coronavirus Disease • Sickle Cell Disease

Less is more: Improving outcomes in haemato-oncology with optimal antibiotic use for febrile neutropenia (ASH 2025) - "Management of FN: Rapid administration of piperacillin-tazobactam and amikacin (Piptaz/Ami) for fever (≥38°C) with neutropenia (≤0.5x10 9 /L or expected neutropenia) after taking BCs...Two patients had relapsed AML, 2 newly diagnosed AML (on Venetoclax/Azacytidine and one on palliative Hydroxycarbamide), and 1 post-haploidentical HSCT for lymphoma... Patient population: 49% had AML/MDS; 66% had active disease (presentation or relapse). Treatment modalities: intensive chemotherapy (37%) autologous (37%) or allogeneic HSCT (13%). Median age was 57 years (23-85)."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Cerebral Hemorrhage • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Myelodysplastic Syndrome • Neutropenia • Pneumonia • Renal Disease

Contextualising and tailoring standards-based audit (SBA) processes for sickle cell disease (SCD) care in Zambia: A pilot Study (ASH 2025) - "Morbidity and mortality remain high in SSA due to limited resources and lack of effective implementation of evidence-based clinical interventions: Newborn screening for early detection, hydroxyurea use, penicillin prophylaxis, malaria prophylaxis; and appropriate blood transfusion therapy, to name a few...Expected Results I expect to understand process of tailoring and contextualising an implementation strategy, SBA and to evaluate the degree of normalisation of this tailored SBA as part of the healthcare professional's practice and sustainability. Additionally, this will be developed into an SBA SCD toolkit that can be scaled up for SCD care in Zambia and extended to other Sub-Saharan African contexts to ultimately improve the quality of care for people living with SCD."

Clinical • Genetic Disorders • Hematological Disorders • Infectious Disease • Malaria • Sickle Cell Disease

Patient outcomes for sickle cell disease with support staff-driven interventions: A scoping review (ASH 2025) - "Three RCTs included a community health worker (CHW) as part of the intervention to increase hydroxyurea (HU) adherence through education and/or reminders, of which 2 showed no sustained improvement in HU adherence, while 1 showed increased HU prescription fills without corresponding laboratory improvements... There is limited evidence evaluating the impact of support staff on patient outcomes in SCD care. Among the few RCTs conducted, most did not demonstrate significant benefit to acute care utilization or HU adherence, with 1 exception for improved HU refill rates. This review highlights the need for future RCTs to examine outcomes related to care coordination, such as retention in care and completion of health maintenance."

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

Scoping review of current practices and recommendations on fertility preservation in sickle cell disease (ASH 2025) - "Introduction: Sickle cell disease and its treatments, mainly hydroxyurea and transformative therapies, are associated with male and female infertility... Articles were categorized into three major headings with occasional overlap noted, namely: referral, counseling, and access. Five articles discussed the importance of referrals, emphasizing that formal referrals or consultations should be made to a fertility service informing patients of their fertility preservation options prior to gene therapy or hematopoietic stem cell transplant. Pediatric and reproductive endocrinologists, gynecologists, and maternal fetal medicine experts were specially mentioned."

Review • Bone Marrow Transplantation • Gene Therapies • Genetic Disorders • Gynecology • Hematological Disorders • Infertility • Sexual Disorders • Sickle Cell Disease

Cervical cancer screening outcomes in adolescents and young adults with sickle cell disease age 21–29: A retrospective chart review (ASH 2025) - "Objective To evaluate cervical cancer screening outcomes among AYAs aged 21–29 with SCD and to assess associations with clinical factors including hydroxyurea use, HPV vaccination status, STI history, BMI, smoking status, and HPV test results... AYAs with SCD are at risk for inadequate cervical cancer screening and HPV vaccination. Targeted interventions—such as structured transition programs and integrated preventive care services—are critical to improving outcomes in this underserved and vulnerable group."

Retrospective data • Review • Bone Marrow Transplantation • Cervical Cancer • Genetic Disorders • Human Papillomavirus Infection • Infectious Disease • Oncology • Preventive care • Sickle Cell Disease • Solid Tumor

Improvement in sickle cell disease care metrics: Regular scheduled hematology provider visits and transcranial doppler completion rate – a QI initiative at Phoenix Children's hospital (ASH 2025) - "At baseline, the percentage of patients with scheduled appointments within 180 days was 73%. This improved to 80%three months post interventions. The baseline percentage of patients with TCD's completed within one year was 71%."

Clinical • Cardiovascular • Genetic Disorders • Hematological Disorders • Renal Disease • Retinal Disorders • Sickle Cell Disease

Inhibitors of RAD51 as potential novel therapies in Acute Myeloid Leukemia (ASH 2025) - "Furthermore, these compounds often exhibit strong synergy and at least additivity in combination with both on-label and off-label use of FDA-approved compounds, as well as investigational molecularly-targeted agents, in relevant cell lines; these include FLT3 inhibitors quizartinib, gilteritinib and tuspetinib ((in MV-4-11 and HL60 cell lines) and BCR/ABL inhibitors (imatinib and regorafenib) in the K562 cell line (AACR 2025)...We have confirmed the IBRs inhibit multiple RAD51 functions including hydroxyurea-induced RAD51 focus formation, HRR by DR-GFP assay, and RS protection by RPA focus formation and a DNA fibre assay...JKYN-1 is a partially optimized (more soluble and more potent) version of IBR120 but is not resistant to degradation by liver microsomes or sufficiently capable of entry into target cells...Treatment dose and combinations will be pre-screened using a highly predictive cell line as a surrogate of human cancer stem cells for subsequently AML patient..."

Acute Myelogenous Leukemia • Colorectal Cancer • Hematological Malignancies • Leukemia • Solid Tumor • BRCA1 • BRCA2 • FLT3 • HRD • RAD51

An evolving landscape – how changing conditioning regimes can improve myelofibrosis transplant outcomes. (ASH 2025) - "Of the 16 cases, seven received Fludarabine/Busulphan/Antithymocyte globulin (Flu/Bu/ATG) conditioning (Fludarabine 30mg/m 2 day -9 to day -5, Busulphan 3.2mg/kg on day -4 and day -3 and rabbit ATG 2.5mg/kg on day -2 and day -1), seven underwent Fludarabine/Cyclophosphamide (Flu/Cy) conditioning (Fludarabine 25mg/m 2 day -6 to day -2 and Cyclophosphamide 1g/m 2 on day -3 and day -2), and two received Fludarabine/Melphalan (Flu/Mel) (Fludarabine 25mg/m 2 day -6 to day -2 and Melphalan 100mg/m 2 on day -2)...Therapies included Ruxolitnib, Momelotinib, splenic irradiation, Hydroxycarbamide, Danazol, Interferon, Cyclophosphamide, Anagrelide, Thalidomide, Panobinostat and Navitoclax...Although limited by a small sample size, these findings indicate that the Flu/Bu/ATG conditioning regimen may be associated with improved outcomes, thereby supporting its adoption as the preferred approach for patients undergoing allogeneic stem cell transplantation for myelofibrosis as per BSH..."

Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Myelofibrosis • Pneumonia • Respiratory Diseases • Transplantation • ASXL1 • CALR • JAK2 • SF3B1 • U2AF1

Prescribing patterns in MDS/MPN syndromes (ASH 2025) - "Additionally, the prescribing patterns of Hydroxyurea, Hypomethylating Agents (HMA)- Azacitidine/Decitabine and JAK inhibitors Ruxolitinib/Cedazuridine/Pacritinib/Momelotinib/Fedratinib were analyzed in these patients. In this real-world analysis, our data demonstrate that over 90% of patients diagnosed with MDS/MPN overlap syndromes do not receive any treatment in routine clinical practice. The most prescribed medication in this cohort of patients with MDS/MPN was hydroxyurea, followed by hypomethylating medications (decitabine and azacitidine). Being untreated was associated with a significant decrement in survival, with untreated patients having up to twice the risk of death than those treated."

Atypical Chronic Myeloid Leukemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm

Myelodysplastic/myeloproliferative overlap neoplasms: Clinicopathological features, treatment strategies, and outcomes — a UK multicentre cohort Study (ASH 2025) - "29% had an antecedent diagnosis of MPN (n=18) or MDS (n=7), and 11 treated with either hydroxyurea (HU; n=9), anagrelide (ANA; n=3), hypomethylating agent (HMA; n=1) or underwent allogeneic transplantation (allo-HCT; n=1)...Treatment was initiated in 57%; 29 (33%) received HU, 7 (8%) ANA, 13 (15%) HMA, 6 (7%) ruxolitinib, and 5 underwent allo-HCT... MDS/MPN neoplasms present therapeutic challenges due to heterogenic phenotypes, lack of clear survival benefit with current non-transplant therapies, poor OS post allo-HCT and a paucity of new agents. Genomic characterisation aids prognostic estimation across all subtypes. Moreover, we confirm the prognostic relevance of 'genomically stratifying' the MDS/MPN-NOS group as per the seminal work of Paloma et al, aiding therapeutic decisions."

Clinical • Cardiovascular • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Thrombocytosis • Thrombosis • ASXL1 • JAK2 • RUNX1 • SETBP1 • SF3B1 • SRSF2 • STAG2 • TET2 • TP53

Risk stratification, prognostic factors, outcomes and quality of life assessment in patients with primary polycythemia (ASH 2025) - "Therapeutic modalities used at our center included phlebotomy in 91.5%, aspirin in 80.5%, cytoreduction with hydroxyurea in 25.4% and systemic anticoagulation in 11% of patients. Our study provides a valuable insight into the prognostic factors and disease process of PV and JAK2 unmutated erythrocytosis in the Indian population. It also gives a glimpse of the QoL in this population, which has till date never been explored before. With such significant divergence from global standards, it is integral to explore these in much greater detail to understand its intricacies in our large and diverse subpopulation."

Biomarker • Clinical • HEOR • Cardiovascular • Chronic Eosinophilic Leukemia • Diabetes • Dyslipidemia • Fibrosis • Hematological Disorders • Hypertension • Metabolic Disorders • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombosis • JAK2

Clinical features and outcomes of myeloproliferative neoplasm patients with MPL mutations and concurrent JAK2/calr mutations (ASH 2025) - "ET treatments included hydroxyurea and off-label Ropeginterferon alfa-2b. Among those with MF, 7 patients received ruxolitinib, 2 required anemia-related therapies, including darbepoetin alfa and Luspatercept...However, further work is required to investigate this hypothesis. Limited follow-up and the predominance of White populations in our cohort underscore the need for studies involving larger and more diverse populations with extended longitudinal data to better understand the prognostic significance and therapeutic implications of the co-mutations."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytosis • ASXL1 • CALR • JAK2 • NOTCH1 • SF3B1 • SH2B3 • SRSF2 • TET2

Long term outcomes of ET and PV patients after clinical trial treatment with interferon alfa (ASH 2025) - "Hydroxyurea (HU) is the most frequently utilized first-line cytoreductive agent, however pegylated interferon alpha-2a (PEG) and the more recently approved, ropeginterferon alfa-2b (ropeg-IFN), have demonstrated a potential to induce molecular responses and improve event free survival (EFS) (Gisslinger Leukemia 2023). This analysis also demonstrated high rates of PEG-related toxicities, although definitive causality was often difficult to determine. Additional clinical and molecular data will be available at the time of the meeting."

Clinical • Bone Marrow Transplantation • Chronic Eosinophilic Leukemia • CNS Disorders • Depression • Endocrine Disorders • Essential Thrombocythemia • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Liver Failure • Mood Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Septic Shock • Thrombocytosis • Thrombosis • CALR • JAK2

Interferon therapy achieves potent reduction in JAK2 allele burden in patients with myeloproliferative neoplasms: A real-world analysis (ASH 2025) - " This single center, retrospective study evaluated consecutive pts treated with pegylated interferon alfa-2a (peg-IFN) or ropeginterferon alfa-2b (ropeg), regardless of line of therapy...Prior therapy included hydroxyurea, anagrelide, and ruxolitinib in 36 (43.9%), 2 (2.4%), and 8 (9.8%) pts, respectively; 44 had not received prior cytoreductive therapy... This real-world analysis demonstrates the feasibility and efficacy of IFN therapy in clinical practice. Most pts achieved sustained CHR with low treatment discontinuation rate; thrombotic and disease progression events were rare. Importantly, we demonstrate the substantial impact of IFN on reducing JAK2 allele burden."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytosis • Thrombosis • JAK2 • NPM1