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  1. Article ; Online: Risk models in myelofibrosis-the past, present, and future.

    Tefferi, Ayalew / Vannucchi, Alessandro M

    American journal of hematology

    2024  Volume 99, Issue 4, Page(s) 519–522

    Abstract: Risk models in myelofibrosis (MYSEC-PM) or primary myelofibrosis (IPSS, DIPSS, DIPSS+, MIPSS70, MIPSS70+, MIPSSv2, GIPSS). ...

    Abstract Risk models in myelofibrosis (MYSEC-PM) or primary myelofibrosis (IPSS, DIPSS, DIPSS+, MIPSS70, MIPSS70+, MIPSSv2, GIPSS).
    MeSH term(s) Humans ; Primary Myelofibrosis ; Prognosis
    Language English
    Publishing date 2024-02-23
    Publishing country United States
    Document type Editorial
    ZDB-ID 196767-8
    ISSN 1096-8652 ; 0361-8609
    ISSN (online) 1096-8652
    ISSN 0361-8609
    DOI 10.1002/ajh.27270
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 1251: Show issues Location:
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  2. Article ; Online: JAK2 inhibitor treatment of anemia in myelofibrosis.

    Tefferi, Ayalew / Vannucchi, Alessandro M

    American journal of hematology

    2023  Volume 98, Issue 7, Page(s) 995–997

    MeSH term(s) Humans ; Primary Myelofibrosis ; Anemia ; Protein Kinase Inhibitors/pharmacology ; Janus Kinase 2
    Chemical Substances Protein Kinase Inhibitors ; Janus Kinase 2 (EC 2.7.10.2) ; JAK2 protein, human (EC 2.7.10.2)
    Language English
    Publishing date 2023-04-21
    Publishing country United States
    Document type Editorial
    ZDB-ID 196767-8
    ISSN 1096-8652 ; 0361-8609
    ISSN (online) 1096-8652
    ISSN 0361-8609
    DOI 10.1002/ajh.26934
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: CALR mutations possess unique prognostic relevance in myelofibrosis-before and after transplant.

    Tefferi, Ayalew / Vannucchi, Alessandro M

    Bone marrow transplantation

    2023  Volume 59, Issue 1, Page(s) 1–3

    MeSH term(s) Humans ; Primary Myelofibrosis/genetics ; Primary Myelofibrosis/therapy ; Prognosis ; Mutation ; Phenotype ; Transplants ; Calreticulin/genetics ; Janus Kinase 2/genetics ; Myeloproliferative Disorders
    Chemical Substances Calreticulin ; Janus Kinase 2 (EC 2.7.10.2)
    Language English
    Publishing date 2023-10-11
    Publishing country England
    Document type Editorial ; Comment
    ZDB-ID 632854-4
    ISSN 1476-5365 ; 0268-3369 ; 0951-3078
    ISSN (online) 1476-5365
    ISSN 0268-3369 ; 0951-3078
    DOI 10.1038/s41409-023-02112-2
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 2168: Show issues Location:
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  4. Article ; Online: Myeloid sarcoma: more and less than a distinct entity.

    Loscocco, Giuseppe G / Vannucchi, Alessandro M

    Annals of hematology

    2023  Volume 102, Issue 8, Page(s) 1973–1984

    Abstract: Myeloid sarcoma (MS) is a distinct entity among myeloid neoplasms defined as a tumour mass of myeloid blasts occurring at an anatomical site other than the bone marrow, in most cases concomitant with acute myeloid leukaemia (AML), rarely without bone ... ...

    Abstract Myeloid sarcoma (MS) is a distinct entity among myeloid neoplasms defined as a tumour mass of myeloid blasts occurring at an anatomical site other than the bone marrow, in most cases concomitant with acute myeloid leukaemia (AML), rarely without bone marrow involvement. MS may also represent the blast phase of chronic myeloproliferative neoplasms (MPN) and myelodysplastic syndromes (MDS). However, the clinical and molecular heterogeneity of AML, as highlighted by the 2022 World Health Organization (WHO) and International Consensus (ICC) classifications, indirectly define MS more as a set of heterogeneous and proteiform diseases, rather than a homogeneous single entity. Diagnosis is challenging and relies mainly on histopathology, immunohistochemistry, and imaging. Molecular and cytogenetic analysis of MS tissue, particularly in isolated cases, should be performed to refine the diagnosis, and thus assign prognosis guiding treatment decisions. If feasible, systemic therapies used in AML remission induction should be employed, even in isolated MS. Role and type of consolidation therapy are not univocally acknowledged, and systemic therapies, radiotherapy, or allogeneic hematopoietic stem cell transplantation (allo-HSCT) should be considered. In the present review, we discuss recent information on MS, focusing on diagnosis, molecular findings, and treatments also considering targetable mutations by recently approved AML drugs.
    MeSH term(s) Humans ; Sarcoma, Myeloid/diagnosis ; Sarcoma, Myeloid/genetics ; Sarcoma, Myeloid/therapy ; Leukemia, Myeloid, Acute/genetics ; Myelodysplastic Syndromes/genetics ; Myeloproliferative Disorders/diagnosis ; Myeloproliferative Disorders/genetics ; Myeloproliferative Disorders/therapy ; Hematopoietic Stem Cell Transplantation
    Language English
    Publishing date 2023-06-07
    Publishing country Germany
    Document type Journal Article ; Review
    ZDB-ID 1064950-5
    ISSN 1432-0584 ; 0939-5555 ; 0945-8077
    ISSN (online) 1432-0584
    ISSN 0939-5555 ; 0945-8077
    DOI 10.1007/s00277-023-05288-1
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  5. Article ; Online: Age-stratified analysis reveals arterial thrombosis as a predictor for gender-related second cancers in myeloproliferative neoplasms: a case-control study.

    Ghirardi, Arianna / Carobbio, Alessandra / Guglielmelli, Paola / Rambaldi, Alessandro / De Stefano, Valerio / Vannucchi, Alessandro M / Tefferi, Ayalew / Barbui, Tiziano

    Blood cancer journal

    2024  Volume 14, Issue 1, Page(s) 68

    Language English
    Publishing date 2024-04-22
    Publishing country United States
    Document type Letter
    ZDB-ID 2600560-8
    ISSN 2044-5385 ; 2044-5385
    ISSN (online) 2044-5385
    ISSN 2044-5385
    DOI 10.1038/s41408-024-01052-4
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  6. Article ; Online: Pegasus causes inherited thrombocytopenia.

    Vannucchi, Alessandro M

    Blood

    2019  Volume 134, Issue 23, Page(s) 2000–2002

    MeSH term(s) Germ-Line Mutation ; Humans ; Leukopenia ; Thrombocytopenia ; Transcription Factors
    Chemical Substances Transcription Factors
    Language English
    Publishing date 2019-12-02
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2019002181
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  7. Article ; Online: Role of JAK inhibitors in myeloproliferative neoplasms: current point of view and perspectives.

    Loscocco, Giuseppe G / Vannucchi, Alessandro M

    International journal of hematology

    2022  Volume 115, Issue 5, Page(s) 626–644

    Abstract: Classic Philadelphia-negative myeloproliferative neoplasms (MPN) include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), classified as primary (PMF), or secondary to PV or ET. All MPN, regardless of the underlying driver ... ...

    Abstract Classic Philadelphia-negative myeloproliferative neoplasms (MPN) include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), classified as primary (PMF), or secondary to PV or ET. All MPN, regardless of the underlying driver mutation in JAK2/CALR/MPL, are invariably associated with dysregulation of JAK/STAT pathway. The discovery of JAK2V617F point mutation prompted the development of small molecules inhibitors of JAK tyrosine kinases (JAK inhibitors-JAKi). To date, among JAKi, ruxolitinib (RUX) and fedratinib (FEDR) are approved for intermediate and high-risk MF, and RUX is also an option for high-risk PV patients inadequately controlled by or intolerant to hydroxyurea. While not yet registered, pacritinib (PAC) and momelotinib (MMB), proved to be effective particularly in thrombocytopenic and anemic MF patients, respectively. In most cases, JAKi are effective in reducing splenomegaly and alleviating disease-related symptoms. However, almost 50% lose response by three years and dose-dependent toxicities may lead to suboptimal dosing or treatment discontinuation. To date, although not being disease-modifying agents, JAKi represent the therapeutic backbone particularly in MF patient. To optimize therapeutic strategies, many trials with drug combinations of JAKi with novel molecules are ongoing. This review critically discusses the role of JAKi in the modern management of patients with MPN.
    MeSH term(s) Humans ; Janus Kinase 2/genetics ; Janus Kinase Inhibitors/therapeutic use ; Janus Kinases/genetics ; Janus Kinases/metabolism ; Janus Kinases/therapeutic use ; Mutation ; Myeloproliferative Disorders/genetics ; Polycythemia Vera/drug therapy ; Primary Myelofibrosis/drug therapy ; STAT Transcription Factors/genetics ; STAT Transcription Factors/metabolism ; STAT Transcription Factors/therapeutic use ; Signal Transduction ; Thrombocythemia, Essential/drug therapy
    Chemical Substances Janus Kinase Inhibitors ; STAT Transcription Factors ; Janus Kinase 2 (EC 2.7.10.2) ; Janus Kinases (EC 2.7.10.2)
    Language English
    Publishing date 2022-03-29
    Publishing country Japan
    Document type Journal Article ; Review
    ZDB-ID 1076875-0
    ISSN 1865-3774 ; 0917-1258 ; 0925-5710
    ISSN (online) 1865-3774
    ISSN 0917-1258 ; 0925-5710
    DOI 10.1007/s12185-022-03335-7
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 269: Show issues Location:
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  8. Article ; Online: Are the available data sufficient to suggest cytoreductive agents for patients with CHIP and stroke?

    Barbui, Tiziano / Carobbio, Alessandra / Vannucchi, Alessandro M / De Stefano, Valerio

    Blood advances

    2023  Volume 7, Issue 24, Page(s) 7551–7553

    MeSH term(s) Humans ; Cytoreduction Surgical Procedures ; Antineoplastic Agents ; Combined Modality Therapy ; Stroke/drug therapy
    Chemical Substances Antineoplastic Agents
    Language English
    Publishing date 2023-10-22
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2915908-8
    ISSN 2473-9537 ; 2473-9529
    ISSN (online) 2473-9537
    ISSN 2473-9529
    DOI 10.1182/bloodadvances.2023012007
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    Zs.A 7153: Show issues Location:
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  9. Article ; Online: One thousand patients with essential thrombocythemia: the Florence-CRIMM experience.

    Loscocco, Giuseppe G / Gesullo, Francesca / Capecchi, Giulio / Atanasio, Alessandro / Maccari, Chiara / Mannelli, Francesco / Vannucchi, Alessandro M / Guglielmelli, Paola

    Blood cancer journal

    2024  Volume 14, Issue 1, Page(s) 10

    Abstract: We describe 1000 patients with essential thrombocythemia seen at the Center Research and Innovation of Myeloproliferative Neoplasms (CRIMM), Florence, Italy, between 1980 and 2023: median age 59 years (18-95), females 65%, JAK2/CALR/MPL-mutated 66%/19%/4% ...

    Abstract We describe 1000 patients with essential thrombocythemia seen at the Center Research and Innovation of Myeloproliferative Neoplasms (CRIMM), Florence, Italy, between 1980 and 2023: median age 59 years (18-95), females 65%, JAK2/CALR/MPL-mutated 66%/19%/4%, triple-negative (TN) 11%. Extreme thrombocytosis (ExT, platelets ≥1000 × 10
    MeSH term(s) Humans ; Male ; Female ; Middle Aged ; Thrombocythemia, Essential/complications ; Leukocytosis/complications ; Myeloproliferative Disorders/complications ; Thrombocytosis/complications ; Thrombosis/etiology ; Thrombosis/genetics ; Mutation ; Janus Kinase 2/genetics ; Calreticulin/genetics
    Chemical Substances Janus Kinase 2 (EC 2.7.10.2) ; Calreticulin
    Language English
    Publishing date 2024-01-18
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2600560-8
    ISSN 2044-5385 ; 2044-5385
    ISSN (online) 2044-5385
    ISSN 2044-5385
    DOI 10.1038/s41408-023-00968-7
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: A safety evaluation of ruxolitinib for the treatment of polycythemia vera.

    Boldrini, Valentina / Vannucchi, Alessandro M / Guglielmelli, Paola

    Expert opinion on drug safety

    2023  Volume 23, Issue 1, Page(s) 1–7

    Abstract: Introduction: Polycythemia Vera (PV) is a chronic myeloproliferative neoplasm hallmarked by deregulated proliferation of hematopoietic stem cells leading to prevalent expansion of red cell mass, increased rate of vascular events, splenomegaly, disease- ... ...

    Abstract Introduction: Polycythemia Vera (PV) is a chronic myeloproliferative neoplasm hallmarked by deregulated proliferation of hematopoietic stem cells leading to prevalent expansion of red cell mass, increased rate of vascular events, splenomegaly, disease-associated symptoms, and risk of evolution to secondary myelofibrosis and blast phase. PV is pathogenetically associated with autonomously persistent activation of JAK2, which causes overproduction of blood cells and an inflammatory condition responsible for the clinical manifestations of the disease. Extensively supported by preclinical studies, targeting JAK2-dependent signaling represents a rational therapeutic approach to PV, finally leading to the approval of ruxolitinib, a JAK1/2 inhibitor.
    Areas covered (literature research): We analyzed reports of phase 2 and phase 3 trials with ruxolitinib in PV and relevant literature dealing with efficacy and safety aspects, including most recent real-world reports.
    Expert opinion: Ruxolitinib is the only JAK2 inhibitor approved for the treatment of PV with well-known efficacy for splenomegaly, symptoms, and potentially reduction of vascular events. The treatment regimen is notably manageable and safe, with the most prevalent side effects primarily encompassing myelosuppression, hyperlipidemia, non-melanoma skin cancer and infections, mainly reactivation of Herpes Zoster. These effects necessitate ongoing surveillance and proactive preventive measures.
    MeSH term(s) Humans ; Polycythemia Vera/drug therapy ; Polycythemia Vera/complications ; Splenomegaly/etiology ; Nitriles ; Pyrimidines/adverse effects ; Janus Kinase Inhibitors ; Pyrazoles
    Chemical Substances ruxolitinib (82S8X8XX8H) ; Nitriles ; Pyrimidines ; Janus Kinase Inhibitors ; Pyrazoles
    Language English
    Publishing date 2023-12-29
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 2088728-0
    ISSN 1744-764X ; 1474-0338
    ISSN (online) 1744-764X
    ISSN 1474-0338
    DOI 10.1080/14740338.2023.2299391
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