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26. Idiopathic Myelofibrosis

Gardner, F. H. and D. G. Nathan

(1966). Androgens and erythropoiesis. Further evaluation of testosterone treatment of myelofibrosis.
N Engl J Med 274(8): 420-6.
Description: A paper that contains iron and chromium kinetic data that elucidates many mechanisms of anemia.
PubMed citation number: 5904280

Jacobson, R. J., Salo, A, and Fialkow, P. J.

(1978). Agnogenic myeloid metaplasia: a clonal proliferation of hematopoietic stem cells with secondary myelofibrosis.
Blood 51(2): 189-94.
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

Dupriez, B., Morel, P., Demory, J. L., Lai, J. L., Simon, M., Plantier, I., and Bauters, F.

(1996). Prognostic factors in agnogenic myeloid metaplasia: a report on 195 cases with a new scoring system.
Blood 88(3): 1013-8.
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

Barosi, G.

(1999). Myelofibrosis with myeloid metaplasia: diagnostic definition and prognostic classification for clinical studies and treatment guidelines.
J Clin Oncol 17(9): 2954-70.
PubMed citation number: 10561375

Tefferi, A., Mesa, R. A., Nagorney, D. M., Schroeder, G. and Silverstein, M. N.

(2000). Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients.
Blood 95(7): 2226-33.
PubMed citation number: 10733489

Devine, S. M., Hoffman, R., Verma, A., Shah, R., Bradlow, B. A., Stock, W., Maynard, V., Jessop, E., Peace, D., Huml, M., Thomason, D., Chen, Y. H. and van Besien, K.

(2002). Allogeneic blood cell transplantation following reduced-intensity conditioning is effective therapy for older patients with myelofibrosis with myeloid metaplasia.
Blood 99(6): 2255-8.
Description: A series of 4 patients (median age 56) who survived nonmyeloablative transplant from HLA identical siblings. Interestingly they show that all patients had improvement in splenomegaly and regression of marrow fibrosis.
PubMed citation number: 11877308

Vannucchi, A. M., Bianchi, L., Cellai, C., Paoletti, F., Rana, R. A., Lorenzini, R., Migliaccio, G. and Migliaccio, A. R.

(2002). Development of myelofibrosis in mice genetically impaired for GATA-1 expression (GATA-1(low) mice).
Blood 100(4): 1123-32.
Description: A mouse model for myelofibrosis?
PubMed citation number: 12149188

Deeg, H. J., Gooley, T. A., Flowers, M. E., Sale, G. E., Slattery, J. T., Anasetti, C., Chauncey, T. R., Doney, K., Georges, G. E., Kiem, H. P., Martin, P. J., Petersdorf, E. W., Radich, J., Sanders, J. E., Sandmaier, B. M., Warren, E. H., Witherspoon, R. P., Storb, R. and Appelbaum, F. R.

(2003). Allogeneic hematopoietic stem cell transplantation for myelofibrosis.
Blood 102(12): 3912-8.
Description: Analysis of 56 patients who underwent allogeneic bone marrow transplant (from related and unrelated donors). 3 year median survival was 58%, outcome was improved with busulfan/cyclophosphamide as a conditioning regimen.
PubMed citation number: 12920019

Mesa, R. A., Steensma, D. P., Pardanani, A., Li, C. Y., Elliott, M., Kaufmann, S. H., Wiseman, G., Gray, L. A., Schroeder, G., Reeder, T., Zeldis, J. B., and Tefferi, A.

(2003). A phase 2 trial of combination low dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia.
Blood 101(7): 2534-41.
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

Reviews

Tefferi, A.

(2003). Treatment approaches in myelofibrosis with myeloid metaplasia: the old and thenew.
Semin Hematol 40(1 Suppl 1): 18-21.
PubMed citation number: 12682877

Tefferi, A.

(2000). Myelofibrosis with myeloid metaplasia.
N Engl J Med 342(17): 1255-65.
PubMed citation number: 10781623

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