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Turkish Journal of Haematology :... Feb 2020
Topics: Aged, 80 and over; Antineoplastic Agents; Humans; Hydroxyurea; Male; Primary Myelofibrosis; Tooth Discoloration
PubMed: 31711282
DOI: 10.4274/tjh.galenos.2019.2019.0275 -
Clinical Lymphoma, Myeloma & Leukemia Sep 2020
Topics: Hematopoietic Stem Cell Transplantation; Humans; Primary Myelofibrosis; Survival Analysis; Transplantation, Homologous
PubMed: 32862877
DOI: 10.1016/S2152-2650(20)30468-7 -
American Journal of Hematology Oct 2016
Topics: Bone Marrow; Female; Fibrosis; Humans; Male; Primary Myelofibrosis; Prognosis
PubMed: 27342701
DOI: 10.1002/ajh.24458 -
Giornale Di Gerontologia Oct 1968
Topics: Adolescent; Adult; Age Factors; Aged; Child; Child, Preschool; Diagnosis, Differential; Hepatomegaly; Humans; Italy; Middle Aged; Primary Myelofibrosis; Splenomegaly
PubMed: 5734768
DOI: No ID Found -
Mayo Clinic Proceedings Jan 2012To share our decades of experience with primary myelofibrosis and underscore the importance of outcomes research studies in designing clinical trials and interpreting...
OBJECTIVE
To share our decades of experience with primary myelofibrosis and underscore the importance of outcomes research studies in designing clinical trials and interpreting their results.
PATIENTS AND METHODS
One thousand consecutive patients with primary myelofibrosis seen at Mayo Clinic between November 4, 1977, and September 1, 2011, were considered. The International Prognostic Scoring System (IPSS), dynamic IPSS (DIPSS), and DIPSS-plus were applied for risk stratification. Separate analyses were included for patients seen at time of referral (N=1000), at initial diagnosis (N=340), and within or after 1 year of diagnosis (N=660).
RESULTS
To date, 592 deaths and 68 leukemic transformations have been documented. Parameters at initial diagnosis vs time of referral included median age (66 vs 65 years), male sex (61% vs 62%), red cell transfusion need (24% vs 38%), hemoglobin level less than 10 g/dL (38% vs 54%), platelet count less than 100 × 10(9)/L (18% vs 26%), leukocyte count more than 25 × 10(9)/L (13% vs 16%), marked splenomegaly (21% vs 31%), constitutional symptoms (29% vs 34%), and abnormal karyotype (31% vs 41%). Mutational frequencies were 61% for JAK2V617F, 8% for MPLW515, and 4% for IDH1/2. DIPSS-plus risk distributions at time of referral were 10% low, 15% intermediate-1, 37% intermediate-2, and 37% high. The corresponding median survivals were 17.5, 7.8, 3.6, and 1.8 years vs 20.0, 14.3, 5.3, and 1.7 years for patients younger than 60 years of age. Compared with both DIPSS and IPSS, DIPSS-plus showed better discrimination among risk groups. Five-year leukemic transformation rates were 6% and 21% in low- and high-risk patients, respectively.
CONCLUSION
The current document should serve as a valuable resource for patients and physicians and provides context for the design and interpretation of clinical trials.
Topics: Age Factors; Aged; Cell Transformation, Neoplastic; Disease Progression; Female; Humans; Leukemia; Male; Middle Aged; Minnesota; Primary Myelofibrosis; Prognosis; Retrospective Studies; Risk Factors; Survival Analysis; Time Factors
PubMed: 22212965
DOI: 10.1016/j.mayocp.2011.11.001 -
Medicine Jul 2017This case report describes the progression of primary myelofibrosis (PMF) to polycythemia vera (PV), and discuss its potential mechanisms.
RATIONALE
This case report describes the progression of primary myelofibrosis (PMF) to polycythemia vera (PV), and discuss its potential mechanisms.
PATIENT CONCERNS
The patient was admitted because of abdominal discomfort and enlarged spleen for 19 months.
DIAGNOSIS
A case of PMF progressed to PV was retrospectively analyzed. There were 19 months between the diagnosis of PMF and PV. The JAK2 V617F mutation was positive before and after the diagnosis of PV; however, new chromosomal abnormalities were detected during the progression.
INTERVENTIONS
For treatment of PMF, the danazol, calcitriol, and thalidomide were given. Then, the use of thalidomide and calcitriol was stopped, and hydroxyurea was started. For treatment of PV, interferon treatment was given, whereas hydroxyurea was continued.
OUTCOMES
After 30 months of the progression (at the recent follow-up), this patient had no obvious symptoms or thrombosis.
LESSONS
PMF rarely progresses to PV, however, the progression will significantly improve the quality of life and prognosis.
Topics: Disease Progression; Female; Humans; Middle Aged; Polycythemia Vera; Primary Myelofibrosis
PubMed: 28700486
DOI: 10.1097/MD.0000000000007464 -
European Journal of Haematology May 2009The clinical phenotype of myelofibrosis (MF) is recognized either de novo (primary) or in the setting of polycythemia vera (post-PV) or essential thrombocythemia... (Review)
Review
The clinical phenotype of myelofibrosis (MF) is recognized either de novo (primary) or in the setting of polycythemia vera (post-PV) or essential thrombocythemia (post-ET). Approximately one-third of patients with primary MF (PMF) present with cytogenetic abnormalities; the most frequent are del(20q), del(13q), trisomy 8 and 9, and abnormalities of chromosome 1 including duplication 1q. Other less frequent lesions include -7/del(7q), del(5q), del(12p), +21 and der(6)t(1;6)(q21;p21.3). In general, cytogenetic abnormalities are qualitatively similar among PMF, post-ET MF and post-PV MF although their individual frequencies may differ. Based on prognostic effect, cytogenetic findings in MF are classified as either 'favorable' or 'unfavorable'. The former include normal karyotype or isolated del(20q) or del(13q) and the latter all other abnormalities. Unfavorable cytogenetic profile in both PMF and post-PV/ET MF confers an independent adverse effect on survival; it is also associated with higher JAK2V617F mutational frequency. In addition to their prognostic value, cytogenetic studies in MF ensure diagnostic exclusion of other myeloid neoplasms that are sometimes associated with bone marrow fibrosis (e.g. BCR-ABL1-positive or PDGFRB-rearranged) and also assist in specific treatment selection (e.g. lenalidomide therapy is active in MF associated with del(5q).
Topics: Chromosome Aberrations; Cytogenetic Analysis; Diagnosis, Differential; Humans; Primary Myelofibrosis; Prognosis; Sequence Deletion
PubMed: 19141119
DOI: 10.1111/j.1600-0609.2009.01224.x -
Hematological Oncology Jun 2017Myelofibrosis (MF) is the most severe among the classical Philadelphia-negative myeloproliferative neoplasms that also include essential thrombocytemia and polycythemia... (Review)
Review
Myelofibrosis (MF) is the most severe among the classical Philadelphia-negative myeloproliferative neoplasms that also include essential thrombocytemia and polycythemia vera. Myelofibrosis is characterized by numerous genetic lesions, often variously associated with each other, and by an aggressive clinical phenotype leading to severely reduced survival. Also, the inflammatory microenvironment plays a key role in disease initiation and progression. Because of the complexity of its pathogenesis and the variability of clinical features, MF is a disease that requires a personalized approach and remains orphan of curative treatments besides allogeneic transplantation. JAK2 inhibitors have marked a remarkable progress, because they alleviate systemic symptoms and reduce splenomegaly but have a limited effect on survival, on mutation load, and on marrow fibrosis. Here, we review the main contributing factors to MF pathogenesis and prognosis, focusing on how these factors relate to therapeutic choices. We discuss results from ongoing studies of JAK2 inhibitors and report on new therapeutic strategies that proved effective in early preclinical and clinical trials, including combination treatments, antifibrotic agents, and telomerase inhibitors.
Topics: Drug Therapy, Combination; Humans; Janus Kinase 2; Primary Myelofibrosis; Prognosis; Telomerase
PubMed: 27510853
DOI: 10.1002/hon.2324 -
Leukemia & Lymphoma Oct 2017
Topics: Aged; Biomarkers, Tumor; Female; Follow-Up Studies; HSP27 Heat-Shock Proteins; Heat-Shock Proteins; Humans; Male; Middle Aged; Molecular Chaperones; Primary Myelofibrosis; Prognosis; Survival Rate
PubMed: 28278711
DOI: 10.1080/10428194.2017.1296146 -
Gastroenterology Nov 2019
Topics: Humans; Hypertension, Portal; Janus Kinase Inhibitors; Male; Middle Aged; Nitriles; Portal Pressure; Primary Myelofibrosis; Pyrazoles; Pyrimidines; Treatment Outcome
PubMed: 28478151
DOI: 10.1053/j.gastro.2016.08.059