Idiopathic Myelofibrosis
Of Historical Interest
Androgens and erythropoiesis. Further evaluation of testosterone treatment of myelofibrosis.
Gardner FH, Nathan DG.
N Engl J Med. 1966;274:420-426.
Description: A paper that contains iron and chromium kinetic data that elucidates many mechanisms of anemia. PubMed citation number: 5904280
Agnogenic myeloid metaplasia: a clonal proliferation of hematopoietic stem cells with secondary myelofibrosis.
Jacobson RJ, Salo A, Fialkow PJ.
Blood. 1978;51:189-194.
Description: The paper that showed (by G-6-PD isoenzymes in women) that AMM is a clonal disorder of stem cells (and not stromal cells).
PubMed citation number: 620081
Prognosis
Prognostic factors in agnogenic myeloid metaplasia: a report on 195 cases with a new scoring system.
Dupriez B, Morel P, Demory JL, et al.
Blood. 1996;88:1013-1018.
Description: A retrospective analysis to determine adverse prognostic variables and a method of stratifying patients into risk groups (the Dupriez score).
PubMed citation number: 8704209
Myelofibrosis with myeloid metaplasia: diagnostic definition and prognostic classification for clinical studies and treatment guidelines.
Barosi, G.
J Clin Oncol. 1999;17:2954-2970.
PubMed citation number: 10561375
Treatment
Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients.
Tefferi A, Mesa RA, Nagorney DM, Schroeder G, Silverstein MN.
Blood. 2000;95:2226-2233.
PubMed citation number: 10733489
Allogeneic blood cell transplantation following reduced-intensity conditioning is effective therapy for older patients with myelofibrosis with myeloid metaplasia.
Devine SM, Hoffman R, Verma A, et al.
Blood. 2002;99:2255-2258.
Description: A series of 4 patients (median age, 56 years) who survived nonmyeloablative transplantation from HLA-identical siblings. All patients had improvement in splenomegaly and regression of bone marrow fibrosis. PubMed citation number: 11877308
Allogeneic hematopoietic stem cell transplantation for myelofibrosis.
Deeg HJ, Gooley TA, Flowers ME, et al.
Blood. 2003;102:3912-3918.
Description: Analysis of 56 patients who underwent allogeneic bone marrow transplantation (from related and unrelated donors). Three-year median survival was 58%, and outcome was improved with busulfan/cyclophosphamide as a conditioning regimen.
PubMed citation number: 12920019
A phase 2 trial of combination low dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia.
Mesa RA, Steensma DP, Pardanani A, et al.
Blood. 2003;101:2534-2541.
Description: A promising phase 2 study of 21 patients taking a well-tolerated dose of thalidomide (50 mg) and prednisone (0.5 mg/kg). There was a 62% overall response rate, and 40% of patients became transfusion independent.
PubMed citation number: 12517815
Molecular Mechanisms of Disease
Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis.
Levine RL, Wadleigh M, Cools J, et al.
Cancer Cell. 2005;7:387-397.
PubMed citation number: 15837627
Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders.
Baxter EJ, Scott LM, Campbell PJ, et al.
Lancet. 2005;365:1054-1061.
PubMed citation number: 15781101
A gain-of-function mutation of JAK2 in myeloproliferative disorders.
James C, Ugo V, Le Couedic JP, et al.
N Engl J Med. 2005;352:1779-1790.
PubMed citation number: 15858187
Description: Three (of 4) recent papers identifying an activating mutation (V671F) in the Janus-associated kinase 2 (JAK2) in subjects with myeloproliferative disorders; the fourth (James et al, Nature 2005) looked only at P vera samples. The methods were slightly different in each case; the mutation was detected in 34%-57% of patients with idiopathic myelofibrosis.
Review
Treatment approaches in myelofibrosis with myeloid metaplasia: the old and the new.
Tefferi, A.
Semin Hematol. 2003;40:18-21.
PubMed citation number: 12682877
Myelofibrosis with myeloid metaplasia.
Tefferi, A.
N Engl J Med. 2000;342:1255-1265.
PubMed citation number: 10781623