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CANCER:

Myeloproliferative Neoplasm

10h
Ropeginterferon Alfa-2b for the Treatment of Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes and Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
P2, N=35, Not yet recruiting, Jonsson Comprehensive Cancer Center | Trial completion date: Mar 2032 --> Sep 2032 | Initiation date: Mar 2026 --> Sep 2026 | Trial primary completion date: Mar 2031 --> Sep 2031
Trial completion date • Trial initiation date • Trial primary completion date
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Besremi (ropeginterferon alfa-2b-njft)
18h
Reparixin in Patients With Myelofibrosis Myeloproliferative Neoplasms Research Consortium (MPN-RC 120) (clinicaltrials.gov)
P2, N=10, Recruiting, Icahn School of Medicine at Mount Sinai | N=26 --> 10 | Trial primary completion date: Dec 2026 --> Dec 2027
Enrollment change • Trial primary completion date
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reparixin (DF 1681Y)
1d
MDM2 inhibitors in myeloid cancers: from basic biology to clinical use in myeloproliferative neoplasms. (PubMed, Leukemia)
Emerging MDM2 degraders and next-generation agents aim to overcome feedback limitations and improve therapeutic index. This review integrates mechanistic foundations, clinical development, resistance biology, and future directions, highlighting how decades of basic science have reshaped p53 reactivation into a precision therapeutic paradigm in myeloid disease.
Review • Journal • IO biomarker
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TP53 (Tumor protein P53) • BCL2 (B-cell CLL/lymphoma 2) • MDM2 (E3 ubiquitin protein ligase)
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TP53 mutation • TP53 wild-type
1d
Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms (clinicaltrials.gov)
P2, N=6, Active, not recruiting, H. Lee Moffitt Cancer Center and Research Institute | Recruiting --> Active, not recruiting | N=70 --> 6
Enrollment closed • Enrollment change
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SF3B1 (Splicing Factor 3b Subunit 1) • SRSF2 (Serine and arginine rich splicing factor 2) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
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SF3B1 mutation • SRSF2 mutation
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Reblozyl (luspatercept-aamt)
2d
Study of Oral Administration of LP-118 in Patients With Relapsed or Refractory CLL, SLL, MDS, MDS/MPN, AML, CMML-2, MPN-BP, ALL, MF, NHL, RT, MM or T-PLL. (clinicaltrials.gov)
P1, N=100, Recruiting, Newave Pharmaceutical Inc | Trial completion date: Oct 2025 --> Dec 2027 | Trial primary completion date: Oct 2025 --> Dec 2026
Trial completion date • Trial primary completion date
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BCL2 (B-cell CLL/lymphoma 2)
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LP-118
3d
Enrollment closed • Enrollment change
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TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ASXL1 (ASXL Transcriptional Regulator 1)
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TP53 mutation • FLT3-ITD mutation • ASXL1 mutation • CBL mutation
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azacitidine • Vanflyta (quizartinib)
3d
New P1 trial
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BCL2 (B-cell CLL/lymphoma 2) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
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cyclophosphamide • fludarabine IV • thiotepa • busulfan
3d
Association Between JAK2 V617F Somatic Mutation and Thoracic Aortic Aneurysms. (PubMed, Genes (Basel))
Compelling emerging evidence supports an association between the JAK2 V617F somatic mutation and the formation of thoracic aortic aneurysms, with VAF acting as a valuable biomarker for aneurysm risk. However, no studies have evaluated whether increasing VAF influences aneurysm growth rate, highlighting the need for future clinical research.
Review • Journal
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JAK2 (Janus kinase 2) • TGFBR2 (Transforming Growth Factor Beta Receptor 2) • COL3A1 (Collagen Type III Alpha 1 Chain)
3d
Endothelial Nitric Oxide Synthase-Dependent Mechanism of Hydroxyurea-Induced S-Phase Arrest in Erythroid Cells. (PubMed, Antioxidants (Basel))
NOS2 and NOS3 act as complementary mediators of proliferation and apoptosis, with NOS3 playing a distinct role in HU-induced proliferation arrest in erythroid cells. These findings highlight the therapeutic potential of NOS targeting to enhance the efficacy of HU and overcome resistance in hematologic malignancies.
Journal
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AKT1 (V-akt murine thymoma viral oncogene homolog 1) • NOS2 (Nitric Oxide Synthase 2) • NOS3 (Nitric oxide synthase 3)
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hydroxyurea
4d
Real-world treatment patterns and outcomes in accelerated and blast-phase myeloproliferative neoplasms: Insights from a large multi-centre cohort analysis in the United Kingdom. (PubMed, Br J Haematol)
Ruxolitinib-based regimens, particularly combined with azacitidine, showed acceptable activity in AP (median OS 27.2 months). IC carried high rates of febrile neutropenia and sepsis; venetoclax was associated with prolonged cytopenias. This study confirms the poor prognosis of MPN-AP/BP, the absence of a unified UK consensus approach and the need for improved therapies and prospective studies to determine optimal treatment approaches for this challenging cohort.
Journal • HEOR • Real-world evidence
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TP53 (Tumor protein P53)
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TP53 mutation
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Venclexta (venetoclax) • azacitidine • Jakafi (ruxolitinib)
4d
SOHO State of the Art Updates and Next Questions: Is Combination Therapy Here for Myelofibrosis? (PubMed, Clin Lymphoma Myeloma Leuk)
Emerging combination strategies and their clinical development will be reviewed here, including investigations that pair JAKi therapy with BCL-2 family inhibitors, BET inhibitors, restored p53 cell death signals, telomerase inhibitors, PIM1 kinase inhibitors, and mutant CALR targeted therapies. While several combination clinical trials suggest improved spleen and symptom responses and the possibility of disease modification, toxicity profiles and optimal sequencing remain areas of active investigation.
Review • Journal • IO biomarker
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JAK2 (Janus kinase 2) • PIM1 (Pim-1 Proto-Oncogene) • CALR (Calreticulin)
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CALR mutation
6d
Dresden MDS Registry With an Accompanying Biomaterial Collection (clinicaltrials.gov)
P=N/A, N=500, Not yet recruiting, Technische Universität Dresden
New trial