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DRUG CLASS:

Ribonucleotide reductase inhibitor

12d
Clinical and hematological profile of patients with philadelphia-negative myeloproliferative neoplasms: First report from the Ecuadorian registry. (PubMed, Hematol Transfus Cell Ther)
These results reveal a relatively homogeneous epidemiological profile across the Latin American region and underscore the need for more multicenter studies to better characterize pH- MPNs in Ecuador and the region, to optimize diagnostic and treatment strategies.
Journal
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JAK2 (Janus kinase 2)
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hydroxyurea
15d
HU-F-AIM: Evaluation of HU-resistance in Adult Patients With Polycythemia Vera Who Meet PV-AIM Predictors (clinicaltrials.gov)
P4, N=76, Terminated, Novartis Pharmaceuticals | Trial completion date: Jan 2027 --> Feb 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Jan 2027 --> Feb 2026; Sponsor Decision
Trial completion date • Trial termination • Trial primary completion date
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hydroxyurea
22d
Photoactivatable Nanozyme-Integrated Microneedles Orchestrate a Self-Amplifying Immunogenic Cell Death-STING Signaling Circuit for Tumor Immune Rewiring. (PubMed, ACS Nano)
Hollow mesoporous Prussian blue (HMPB) nanozymes coloaded with hydroxyurea (HU) and thapsigargin (TG) are embedded within a poly(γ-glutamic acid) microneedle matrix, allowing localized and minimally invasive intratumoral delivery. This trimodal self-amplifying immune cascade enhances tumor immunogenicity, remodels the tumor immune microenvironment, and elicits potent local and systemic T cell-mediated antitumor responses. Collectively, this work establishes a nanozyme-microneedle-integrated strategy for the precise orchestration of innate and adaptive antitumor immunity.
Journal
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STING (stimulator of interferon response cGAMP interactor 1) • CGAS (Cyclic GMP-AMP Synthase) • STIM1 (Stromal Interaction Molecule 1)
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hydroxyurea
28d
Chronic myeloid leukaemia in a child with mucopolysaccharidosis type VI: diagnostic and management challenges. (PubMed, BMJ Case Rep)
The patient was stabilised with hydroxyurea and subsequently commenced on imatinib, achieving haematological remission, while supportive measures addressed the multisystem complications of MPS VI. This case highlights the need for vigilance in recognising malignancy in children with rare genetic disorders, the limitations of confirmatory testing in low-resource settings and the importance of multidisciplinary, individualised care when treatments for one condition may complicate the other.
Journal
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ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
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imatinib • hydroxyurea
1m
Testing the Response to the Anti-cancer Drug, Triapine, in Uterine Cancers Using Markers From the Tissue at the Time of Hysterectomy (clinicaltrials.gov)
P1, N=12, Active, not recruiting, National Cancer Institute (NCI) | Suspended --> Active, not recruiting
Enrollment closed
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Triapine (3-AP)
1m
ProTIP: Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease (clinicaltrials.gov)
P4, N=50, Recruiting, Emory University | Not yet recruiting --> Recruiting | Initiation date: Dec 2025 --> Apr 2026
Enrollment open • Trial initiation date
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hydroxyurea
1m
Encapsulation of hydroxyurea in exosomes derived from adipose mesenchymal stem cells enhances anticancer efficacy in breast cancer. (PubMed, Transl Oncol)
These results suggest that exosomes improve drug uptake and bypass drug resistance mechanisms, enabling dose reduction and minimizing side effects. This study introduces HU-Exo as a targeted and multifaceted drug delivery system capable of inhibiting breast tumor growth and metastasis, paving the way for the development of novel and combination therapies in the future.
Journal
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MMP2 (Matrix metallopeptidase 2) • MMP9 (Matrix metallopeptidase 9)
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hydroxyurea
1m
Concomitant plasma cell myeloma and chronic myelomonocytic leukemia in elderly: diagnostic complexity, therapeutic challenges - case report and literature review. (PubMed, Ann Med Surg (Lond))
She underwent leukapheresis, hydroxyurea for cytoreduction, and bortezomib-dexamethasone for myeloma. In elderly patients with cytopenias and monocytosis, thorough diagnostic workup is crucial. This case emphasizes the need for personalized therapy in complex hematologic overlap syndromes.
Journal
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NRAS (Neuroblastoma RAS viral oncogene homolog) • TET2 (Tet Methylcytosine Dioxygenase 2) • SDC1 (Syndecan 1)
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NRAS mutation • TET2 mutation
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bortezomib • dexamethasone • hydroxyurea
1m
DNA polymerase kappa stabilized by Ptbp2 interacts with MRE11 and promotes genomic instability in leukemia. (PubMed, Cell Death Discov)
Knocking out Ptbp2 in CML cell lines and patient samples decreased Polk levels; when treated with hydroxyurea, these samples exhibited increased DNA damage, evidenced by long comet tails and elevated γH2AX foci, a DNA damage marker; however, re-expressing Polk in Ptbp2-KO cells restored the phenotype...Cells with high levels of Ptbp2 and Polk showed increased sister chromatid exchanges and BrdU incorporation in ex vivo tests, while multinucleated cells with multipolar spindles appeared in in vivo tests. Our results confirm the key role of the Ptbp2-Polk-MRE11 axis in promoting genomic instability and supporting the survival of cells with higher malignancy.
Journal
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MRE11A (MRE11 homolog, double strand break repair nuclease) • POLK (DNA Polymerase Kappa)
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hydroxyurea
2ms
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.
PK/PD data • Journal
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ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
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imatinib • hydroxyurea
2ms
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
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JAK2 (Janus kinase 2)
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Jakafi (ruxolitinib) • hydroxyurea
2ms
Enrollment closed • Enrollment change • First-in-human
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erlotinib • Lytgobi (futibatinib)