Pegasys for Myelofibrosis – pro

Interferon is effective in myeloproliferative disorders, which include myelofibrosis. Pegylated interferon is more convenient that interferon because it can be given weekly. However, in the one study in which marrow histology was examined, progression of myelofibrosis was not inhibited by pegylated interferon. Many patients with myelofibrosis are JAK2 positive and pegylated interferon has been shown to reduce Jak2 expression. However, the extent of evidence and number of publications is much smaller for myelofibrosis than for P. Vera and Essential Thrombocytosis. A recent (Nordic) guideline says: “JAK2-mutation is found in about half those patients with PMF. The findings of a steady decrease in the level of the JAK2-mutation in a large number of PV-patients during treatment with pegylated interferon α-2a supports the contention that IFN-treatment may influence the disease at the molecular level, at least in half of the patients (JAK2-positive). A prospective, randomised study of pegylated interferon versus HU in patients with PMF is warranted to clarify this issue”.

The National Cancer Institute (NCI) guidelineslist as treatment recommendations:
“Treatment options:
1. Clinical trials involving JAK2 inhibitors.(COMFORT-I trial [NCT00952289])
2. Interferon-alpha.[20,21]
3. Splenectomy.[18,30]
4. Splenic radiation therapy.[4]
5. Hydroxyurea.[3,4]
6. Allogeneic peripheral stem cell or bone marrow transplantation.[26,27,31,32]
7. Thalidomide.[15,22,23,25]
8. Lenalidomide.[17,33]”
accessed 10/14/11)
The use of pegylated interferon alpha 2a is supported by the peer reviewed literature. In a study of pegylated interferon 2a to treat 18 patients with myelofibrosis, 16 patients responded to achieve either ‘complete remission’ (six patients) or ‘major responses’ (ten patients).

Ianotto JC, Kiladjian JJ, Demory JL, Roy L, Boyer F, Rey J, Dupriez B, Berthou C, Abgrall JF. PEG-IFNalpha-
2a therapy in patients with myelofibrosis: a study of the French Groupe d’Etudes des
Myelofibroses (GEM) and France Intergroupe des syndromes Myéloprolifératifs (FIM). Br J
Haematol. 2009 Jul;146(2):223-5).

Verstovsek S, Lawhorn K, Giles F, et al. PEG-intron for myeloproliferative diseases: an update on ongoing phase II study. Blood. 2004;104:11. Abstract 1517.

Nordic MPD Study Group,,

Hans Carl Hasselbalch, Myelofibrosis with myeloid metaplasia: The advanced phase of an untreated disseminated hematological cancer:
Time to change our therapeutic attitude with early upfront treatment? Leukemia Research Volume 33, Issue 1 , Pages 11-18, January 2009

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