Document Detail

One thousand patients with primary myelofibrosis: the mayo clinic experience.
MedLine Citation:
PMID:  22212965     Owner:  NLM     Status:  MEDLINE    
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.
Ayalew Tefferi; Terra L Lasho; Thitina Jimma; Christy M Finke; Naseema Gangat; Rakhee Vaidya; Kebede H Begna; Aref Al-Kali; Rhett P Ketterling; Curtis A Hanson; Animesh Pardanani
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Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Mayo Clinic proceedings     Volume:  87     ISSN:  1942-5546     ISO Abbreviation:  Mayo Clin. Proc.     Publication Date:  2012 Jan 
Date Detail:
Created Date:  2012-01-03     Completed Date:  2012-02-28     Revised Date:  2013-12-13    
Medline Journal Info:
Nlm Unique ID:  0405543     Medline TA:  Mayo Clin Proc     Country:  England    
Other Details:
Languages:  eng     Pagination:  25-33     Citation Subset:  AIM; IM    
Copyright Information:
Copyright © 2012 Mayo Foundation for Medical Education and Research. Published by Elsevier Inc. All rights reserved.
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MeSH Terms
Age Factors
Cell Transformation, Neoplastic*
Disease Progression
Leukemia / etiology*
Middle Aged
Primary Myelofibrosis* / blood,  genetics,  mortality,  pathology
Retrospective Studies
Risk Factors
Survival Analysis
Time Factors

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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