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A Pilot Study of the Telomerase Inhibitor Imetelstat for Myelofibrosis
Ist Teil von
The New England journal of medicine, 2015-09, Vol.373 (10), p.908-919
Ort / Verlag
United States: Massachusetts Medical Society
Erscheinungsjahr
2015
Quelle
MEDLINE
Beschreibungen/Notizen
Imetelstat, a telomerase inhibitor, induced complete or partial responses in 21% of patients with refractory myelofibrosis. In some patients, reversal of marrow fibrosis was documented and the burden of mutant clones decreased. Myelosuppression was the key toxic effect.
Allogeneic stem-cell transplantation is currently the only method of treatment for patients with myeloproliferative neoplasm–associated myelofibrosis that has been shown to induce long-term disease-free remission.
1
Unfortunately, allogeneic stem-cell transplantation is associated with a relatively high rate of treatment-related death and complications, including chronic graft-versus-host disease. Furthermore, many older patients are not eligible for this intervention. Other treatment strategies, including the use of Janus kinase (JAK) inhibitors, are palliative and lack selective anticlonal activity.
2
Ruxolitinib and other JAK inhibitors can alleviate constitutional symptoms and reduce spleen size, but they often cannot induce complete or partial remissions, reversal of bone marrow fibrosis, . . .