hydroxyurea / Generic mfg. - LARVOL DELTA

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

PROSPERA: Pacritinib vs. Hydroxyurea in Advanced Proliferative Chronic Myelomonocytic Leukemia (clinicaltrials.gov) - P2 | N=66 | Recruiting | Sponsor: Theradex | Not yet recruiting ➔ Recruiting

Enrollment open • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

[177Lu]Lu-DOTATATE for Recurrent Meningioma (LUMEN-1, EORTC-2334-BTG): Study Protocol for a Randomized Phase II Trial. (PubMed, J Nucl Med) - P2 | "In total, 136 patients will be randomized in a 2:1 ratio to [177Lu]Lu-DOTATATE (≤4 doses of 7.4 GBq given every 4 wk) or local standard of care (hydroxyurea, bevacizumab, sunitinib, octreotide, everolimus, or observation). The trial protocol includes a comprehensive exploratory translational research program with dosimetry and imaging-based and tissue-based investigations. LUMEN-1 was activated in March 2025 and will enroll patients in 35 sites in 10 countries across Europe, with primary endpoint collection planned after 2 y and study completion after 5 y. To our knowledge, EORTC-2334-BTG (LUMEN-1, NCT06326190) is the first prospective randomized trial investigating the efficacy of [177Lu]Lu-DOTATATE in patients with recurrent meningioma."

Journal • P2 data • Brain Cancer • Meningioma • Oncology • Solid Tumor • SSTR • SSTR2

Olverembatinib (HQP1351) Demonstrates Efficacy Vs. Best Available Therapy (BAT) in Patients (Pts) with Tyrosine Kinase Inhibitor (TKI)-Resistant Chronic Myeloid Leukemia Chronic-Phase (CML-CP) in a Registrational Randomized Phase 2 Study (ASH 2023) - P2 | "Introduction This was a multicenter, randomized, registrational phase 2 study to assess the efficacy and safety of olverembatinib compared with BAT in pts with CML-CP who were resistant and/or intolerant to 3 TKIs (imatinib [I], dasatinib [D], nilotinib [N]) in China...Pts were randomized 2:1 to investigational olverembatinib (40 mg QOD) or the BAT arm, which could be one of the following per investigator choice: TKIs (I, D, or N), interferon (IFN), hydroxyurea (HU), and homoharringtonine (HHT)...Olverembatinib was observed to be better tolerated and more effective than BAT in treating these pts. Internal study (CT.gov) numbers: HQP1351CC203 (NCT04126681)."

Clinical • P2 data • Anemia • Cardiovascular • Chronic Myeloid Leukemia • CNS Disorders • Congestive Heart Failure • Coronary Artery Disease • Dyslipidemia • Heart Failure • Hematological Disorders • Hematological Malignancies • Hypertriglyceridemia • Leukemia • Leukopenia • Myocardial Infarction • Neutropenia • Oncology • Thrombocytopenia • ABL1

Hyperleukocytosis in a Patient With Chronic Myeloid Leukemia and HIV: A Case Report. (PubMed, Cureus) - "Potential leukostasis was managed using leukapheresis, hydroxyurea, and continued antiretroviral therapy before initiating dasatinib (a tyrosine kinase inhibitor), given its therapeutic effect on CML and potential properties to target HIV infection. Multidisciplinary coordination allowed for successful management, balancing leukemia and HIV treatment needs. This case emphasizes the complexity of CML-HIV co-treatment and the importance of tailored therapy, highlighting the need for further research on dual therapy impacts to improve outcomes and quality of life in co-infected patients."

Journal • Chronic Myeloid Leukemia • Hematological Malignancies • Human Immunodeficiency Virus • Infectious Disease • Leukemia • Oncology

Macrocytosis as an Early Pharmacodynamic Marker of Imatinib Efficacy in Chronic Myeloid Leukemia. (PubMed, J Clin Med) - "Patients with conditions that could confound MCV (hydroxyurea exposure, megaloblastic anemia, hypothyroidism, chronic liver disease, alcoholism) were excluded. MCV elevation during imatinib therapy is associated with deeper molecular response and reduced need for treatment modification. MCV dynamics may serve as an inexpensive pharmacodynamic marker to support risk assessment and guide monitoring in chronic-phase CML."

Journal • PK/PD data • Chronic Myeloid Leukemia • Endocrine Disorders • Hematological Disorders • Hematological Malignancies • Hepatology • Leukemia • Oncology • ABL1 • BCR

Post-Transplant Cyclophosphamide with Extended Abatacept for GVHD Prophylaxis Following Haploidentical Transplantation for Non-Malignant Disorders (TCT-ASTCT-CIBMTR 2026) - "Conditioning included alemtuzumab (total 33 mg if 10 kg) days -22 to -19, fludarabine (total 150 mg/m2) days -8 to -4, thiotepa 8 mg/kg day -4, and melphalan (140 mg/m2) day -3. Sickle cell disease (SCD) patients also received hydroxyurea (30 mg/kg/d) from days -60 to -21...One EBV-PTLD case required rituximab and early abatacept cessation (day +118)...3. Describe expected immune reconstitution."

Post-transplantation • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Sickle Cell Disease • Transplantation • CD4 • IL2RA • IL7R

Outcomes of Treosulfan, Thiotepa and Fludarabine Conditioning in Matched Sibling Donor Vs. Haploidentical Related Donor Hematopoietic Stem Cell Transplant for Children and Young Adults with Sickle Cell Disease (TCT-ASTCT-CIBMTR 2026) - "Rabbit ATG (thymoglobulin) 2.5 mg/kg on day-2 and day-1 was given to those undergoing MSD and on day -6 and day-5 to those undergoing Haploidentical HSCT...Graft versus host disease (GVHD) prophylaxis was cyclosporine and Methotrexate for MSD HSCT and PTCy 50 mg/kg on day+3 and 4 along with MMF & tacrolimus for Haploidentical Donor HSCT. All children undergoing haploidentical HSCT with anti-Human leukocyte antigen (HLA) antibodies >3000 MFI were given pre transplant immune suppression (PTIS) for 6 weeks (Cyclophosphamide 1000 mg/m2 on day 1 and day22, Rituximab 100 mg/m2 on day8,15,29,36 and MMF and hydroxyurea)...Conclusion– Outcomes of TTF conditioning is superior in children and young adults undergoing MSD HSCT vs haploidentical HSCT with PTCy for sickle cell disease. Treosulfan, Thiotepa and fludarbine (TTF) based conditioning is effective in sickle cell disease patients udergoing HSCT Outcomes are superior for patients undergoing matched sibling donor HSCT..."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Disorders • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Sickle Cell Disease • Transplantation

Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure (clinicaltrials.gov) - P1/2 | N=8 | Recruiting | Sponsor: Paul Szabolcs | Trial completion date: Dec 2026 ➔ Dec 2028 | Trial primary completion date: Dec 2025 ➔ Dec 2027

Trial completion date • Trial primary completion date • Aplastic Anemia • Bone Marrow Transplantation • Chronic Obstructive Pulmonary Disease • Hematological Disorders • Idiopathic Pulmonary Fibrosis • Immunology • Neutropenia • Pulmonary Disease • Respiratory Diseases • Transplantation

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

Myelofibrosis

PRO-RIC: Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC (clinicaltrials.gov) - P=N/A | N=50 | Recruiting | Sponsor: Paul Szabolcs | Trial completion date: Jun 2026 ➔ Jun 2028 | Trial primary completion date: Dec 2025 ➔ Dec 2027

Trial completion date • Trial primary completion date • Anemia • Aplastic Anemia • Immunology • Metabolic Disorders • Pediatrics • Primary Immunodeficiency • Transplantation

Case series: a rare dominant form of β-thalassemia successfully treated by luspatercept. (PubMed, Ann Hematol) - "Our findings support the use of Luspatercept and Hydroxyurea as therapeutic options. Long-term monitoring, including surveillance for complications related to iron overload, is essential for optimal clinical management."

Journal • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • HBB

ASTX727 delivers superior response rates and associated survival benefit versus hydroxycarbamide/best supportive care in CMML and other MDS/MPN overlap syndromes: First results from the Phase 2 UK multicenter randomized ammo trial (ASH 2025) - "Rapid recruitment(6mo early) and high retention showcases feasibility/appetite for RCTs in this challenging group.Extending access beyond CMML AMMO also showcases HMA efficacy across related biological entities.The 53% ORR is in keeping with that expected for HMA monotherapy and almost double that for HC/BSC.Toxicity was higher, but acceptable and in line with prior ASTX727 trials. Crucially this did not negate itsefficacy advantage, translating into significantly longer PFS, TFS and OS: the first randomized survivalbenefit demonstrated in MDS/MPN for ~30y and supporting ASTX727 as a new SoC for advancedMDS/MPN."

Clinical • P2 data • Chronic Myelomonocytic Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Respiratory Diseases • Thrombocytopenia

Phase IIa study on the safety and efficacy of Flonoltinib Maleate Tablets in the treatment of patients with polycythemia vera (ChiCTR) - P2 | N=60 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences (IHCAMS);West China Hospital of Sichuan University;Shengjin

New P2 trial • Polycythemia Vera

Impairment of ribosomes and DNA biosynthesis confers resistance to Inhibition of sphingolipid biosynthesis. (PubMed, Mol Genet Genomics) - "To conditionally suppress sphingolipid biosynthesis, we employed a tetracycline-repressible promoter to control LCB1 expression...Furthermore, sublethal concentrations of cycloheximide (a translation inhibitor), diazaborine (a ribosome maturation inhibitor), hydroxyurea (a DNA biosynthesis inhibitor), and zeocin (a DNA double-strand break inducer) alleviated growth defects caused by LCB1 repression...Additionally, these treatments suppressed the reduction in Lcb1 and Aur1 protein expression levels. These findings reveal a previously unappreciated link between ribosome function, DNA biosynthesis, and sphingolipid metabolism and provide insight into how cells adapt to metabolic stress."

Journal • Metabolic Disorders • RPL23

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

Hemoglobin as a Molecular Glue: Toward Potent Inhibition of HbS Polymerization in Sickle Cell Disease. (PubMed, Adv Healthc Mater) - "Current therapies, such as hydroxyurea and voxelotor, provide only partial symptomatic relief, underscoring the urgent need for transformative strategies. These molecular glues, generated through gene editing or synthetic biology, offer a cell-intrinsic, high-concentration mechanism to counteract HbS polymerization, potentially overcoming the limitations of current therapies. We examine the key challenges in translating this paradigm, including precise structural characterization of polymerization intermediates, efficient intracellular delivery to erythrocytes, temporal regulation under hypoxic conditions, and the mitigation of immunogenicity."

Journal • Review • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Targeted Protein Degradation

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

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

CHARACTERISTICS AND CLINICAL OUTCOMES IN PATIENTS (PTS) WITH POLYCYTHEMIA VERA (PV) RECEIVING RUXOLITINIB (RUX) AFTER HYDROXYUREA (HU): A LONGITUDINAL ANALYSIS FROM REVEAL (EHA 2023) - P=N/A | "This longitudinal analysis of REVEAL demonstrated that HU-RUX had more severe disease burden vs HU. In HU- RUX, RUX tx resulted in significant improvements in PV-related symptoms, spleen size reduction, and sustainedblood parameter improvements. This correlated with a reduction of PHL within 6 mo; most pts became PHL-free at 12 mo."

Clinical • Clinical data • Polycythemia Vera

TREATMENT COMPARISON OF HYDROXYUREA VS RUXOLITINIB IN ESSENTIAL THROMBOCYTHEMIA (ET): A MATCHED COHORT ANALYSIS (EHA 2023) - P=N/A, P2 | "Pts who switched from hydroxyurea to RUX experienced controlled WBC and PLT counts compared with pts who stayed on hydroxyurea. Pts who switched from hydroxyurea to RUX also showed a reduction in splenomegaly over time. These results demonstrate that switching to RUX after being refractory or intolerant to hydroxyurea may improve clinical outcomes of pts with ET."

Essential Thrombocythemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Thrombocytosis

Budd-Chiari Syndrome Secondary to Essential Thrombocythaemia Complicated by Acquired Von Willebrand Disease and Mimicking Hepatic Malignancy: A Case Report. (PubMed, Cureus) - "The patient was managed with cytoreductive therapy; initially, hydroxycarbamide and anagrelide, later transitioned to ruxolitinib, alongside lifelong anticoagulation. At six-month follow-up, he demonstrated improved LFTs, stable blood counts, and no recurrent thrombotic events, with ongoing surveillance for variceal bleeding. This case underscores the importance of considering BCS in patients with ET and hepatic abnormalities, screening for AvWS to balance thrombotic/bleeding risks, and utilizing a multidisciplinary team (MDT) approach for optimal management."

Journal • Dermatology • Essential Thrombocythemia • Fatigue • Hematological Disorders • Hemophilia • Hepatocellular Cancer • Myeloproliferative Neoplasm • Oncology • Pruritus • Pulmonary Disease • Thrombocytosis • JAK2

PHARMACOKINETICS/PHARMACODYNAMICS, SAFETY AND EFFICACY OF CRIZANLIZUMAB IN PATIENTS WITH SICKLE CELL DISEASE AGED 12 TO <18 YEARS: 2-YEAR DATA FROM THE PHASE 2 SOLACE-KIDS STUDY (EHA 2023) - P2 | "Patients received crizanlizumab on Day 1, Day 15, then every 4 weeks (up to 2 years), with or without hydroxyurea (HU)/Hydroxycarbamide (HC). In this 2-year analysis, crizanlizumab 5 mg/kg with or without concomitant HU/HC has shown a reduction in VOCs resulting in decreased healthcare visits per year, consistent with the established profile of crizanlizumab in adults. Crizanlizumab was safe and well tolerated with no new/unexpected safety concerns. These results confirm 5 mg/kg as an adequate dose in pediatrics with SCD aged 12 to <18 years."

Clinical • P2 data • PK/PD data • Back Pain • CNS Disorders • Genetic Disorders • Hematological Disorders • Infectious Disease • Musculoskeletal Pain • Pain • Pediatrics • Sickle Cell Disease

Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group. (PubMed, Ann Hematol) - P2 | "Ruxolitinib (RUX) is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. One hundred nine adverse events (AEs) occurred in 24/28 patients (all grade 1 to 3), and no pt permanently discontinued treatment because of AEs. Thus, treatment with ruxolitinib in untreated PV pts is feasible, well-tolerated, and efficient regarding the above-mentioned endpoints."

Journal • Cardiovascular • Dermatology • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Pruritus

Ruxolitinib Versus Best Available Therapy for Polycythemia Vera Intolerant or Resistant to Hydroxycarbamide in a Randomized Trial. (PubMed, J Clin Oncol) - "The MAJIC-PV study demonstrates ruxolitinib treatment benefits HC-INT/RES PV patients with superior CR, and EFS as well as molecular response; importantly also demonstrating for the first time, to our knowledge, that molecular response is linked to EFS, PFS, and OS."

Journal • Cardiovascular • Hematological Disorders • Polycythemia Vera • Thrombosis • ASXL1