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CANCER:

Myeloproliferative Neoplasm

1d
Geriatric Optimization Plan to Improve Survival in Older Adult Allogeneic Hematopoietic Cell Transplant Candidates, OTIS Study (clinicaltrials.gov)
P=N/A, N=30, Completed, Ohio State University Comprehensive Cancer Center | Active, not recruiting --> Completed
Trial completion
1d
Response to therapy and prognosis according to molecular characterization in CALR-mutated essential thrombocythemia. (PubMed, Hemasphere)
Furthermore, the HMR profile was also associated with a higher risk of progression to acute leukemia, while it did not influence the probability of CHR or progression to MF. In conclusion, CALR VAF and HMR profile appear to be more important than CALR mutation type regarding treatment response and major clinical outcomes in ET.
Journal • CALR
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CALR (Calreticulin)
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CALR mutation
1d
Unraveling the role of TP53 mutations in myeloproliferative neoplasms: Molecular mechanisms of leukemic transformation. (PubMed, Hemasphere)
Additionally, we explore the mechanisms underlying TP53 mutations in the leukemic transformation of MPNs, including clonal evolution to multihit status and the role of inflammation and therapy. Finally, recent findings on the clinical impact of multihit TP53 mutations and potential strategies for targeting the p53 pathway in MPNs and sAML are presented.
Review • Journal
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TP53 (Tumor protein P53)
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TP53 mutation
1d
Role of Chitinase 3-like-1 in Myelofibrosis via Fibroblast-Produced Extracellular Matrix Enhancement. (PubMed, J Cell Physiol)
Romiplostim-induced MF in a mouse model demonstrated extensive bone marrow (BM) CHI3L1 mRNA expression, which was reversed by clodronate treatment. A culture assay revealed that high CHI3L1 concentrations promoted extracellular matrix production by fibroblast cell lines, and that a CHI3L1-neutralizing antibody abrogated this effect. These results indicate the importance of CHI3L1 in the association between fibrocytes and fibroblasts in MF and could be a focus for future treatment.
Journal
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JAK2 (Janus kinase 2) • CD14 (CD14 Molecule) • CHI3L1 (Chitinase 3-like 1)
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clodronate disodium • Nplate (romiplostim)
3d
Polycythemia in males living with HIV post dolutegravir and long term antiretroviral therapy. (PubMed, Int J STD AIDS)
Haematological adverse effects of ART are well recognized, most commonly anemia, particularly with zidovudine-containing regimens. Although the patients were clinically stable, persistent polycythaemia may increase long term thrombotic risk and therefore may warrant periodic complete blood count monitoring. ConclusionThis case series highlights the need for awareness of this uncommon but potentially significant haematological observation in patients receiving dolutegravir-based ART and underscores the importance of systematic evaluation to exclude alternative aetiologies.
Journal
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CD4 (CD4 Molecule)
5d
Mutational architecture defines disease behaviour in an Indian cohort of Myeloproliferative Neoplasms: Foundations for a Genomics-first classification. (PubMed, Leuk Res)
Survival differences were independent of WHO diagnostic labels, with molecular architecture alone consistently predicting outcomes across all clusters In this Indian MPN cohort, molecular architecture, not WHO label, dictated prognosis. The reproducible impact of high-risk co-mutations demonstrates both the feasibility and need for a genomics-first, tiered classification to guide risk stratification and therapy.
Journal • Tumor mutational burden
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TMB (Tumor Mutational Burden) • ABL1 (ABL proto-oncogene 1) • JAK2 (Janus kinase 2) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2)
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TMB-H
5d
Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis (clinicaltrials.gov)
P1, N=6, Not yet recruiting, Novartis Pharmaceuticals | Trial primary completion date: Nov 2027 --> Nov 2026
Trial primary completion date
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Jakafi (ruxolitinib) • pelabresib (DAK539)
6d
Successful Haplo-Hematopoietic Stem Cell Transplantation for Juvenile Myelomonocytic Leukemia in a Child With Underlying Thrombocytopenia-Absent Radius Syndrome: A Unique Case. (PubMed, Cancer Rep (Hoboken))
This case represents, to our knowledge, one of the very few-if not the first-reported instances of successful HSCT for JMML in a patient with TAR syndrome. It underscores the importance of vigilant surveillance in TAR patients for potential malignant transformation and demonstrates the curative potential of HSCT in rare congenital-hematologic overlap syndromes.
Journal
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NF1 (Neurofibromin 1) • RBM8A (RNA Binding Motif Protein 8A)
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RAS mutation
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cyclophosphamide • melphalan • Grafapex (treosulfan)
6d
Neutrophil-to-lymphocyte ratio half reduction as a surrogate of molecular response in polycythemia vera treated with ropeginterferon alfa-2b. (PubMed, Ther Adv Hematol)
NLR half reduction significantly predicted hematologic response (week 24 OR 6.42, p = 0.001) and molecular response consistently across all time points (week 24 OR 27.94, p < 0.001). NLR half reduction is a simple, cost-effective biomarker that may reflect molecular response and treatment efficacy in PV.
Journal
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JAK2 (Janus kinase 2)
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Besremi (ropeginterferon alfa-2b-njft)
7d
Ruxolitinib reverses systemic vasculitis driven by JAK2 V617F-mutated essential thrombocythemia: a case report. (PubMed, Front Immunol)
Treatment with hydroxycarbamide and ruxolitinib resulted in decreased platelet counts and improved vasculitis, with no subsequent recurrence of cardiovascular events. This rare case shows that ruxolitinib can be effective in treating vasculitis complications in patients with JAK2 mutation-positive ET.
Journal
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JAK2 (Janus kinase 2)
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Jakafi (ruxolitinib) • hydroxyurea
7d
Clinical, Genetic, and Pathologic Variability in Myelodysplastic Syndromes and Precursor Conditions Across Race, Ethnicity, and Sex. (PubMed, Am J Hematol)
These findings highlight the need for enhanced understanding of MDS pathogenesis across patient groups and refined prognostic tools to improve personalized management of MDS spectrum conditions. Trial Registration: ClinicalTrials.gov identifier: NCT02775383.
Journal
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TP53 (Tumor protein P53) • DNMT3A (DNA methyltransferase 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2)
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TP53 mutation