International working group for myelofibrosis research and treatment response assessment and long-term follow-up of 50 myelofibrosis patients treated with thalidomide-prednisone based regimens.
We have previously reported the benefits of combination therapy with thalidomide and prednisone for the improvement of cytopenias and splenomegaly for patients with myelofibrosis (MF); both primary and those arising from an antecedent polycythemia vera (PV) and essential thrombocythemia (ET). We report here the overall efficacy of three different thalidomide-prednisone based regimens (alone or in combination with either oral cyclophosphamide for three months or continuous weekly etanercept) when assessed by the IWG-MRT response criteria which were developed after completion of these studies. Additionally we report on the durability of response and long-term follow-up. We observed an overall response rate of 28% by IWG-MRT criteria (mostly clinical improvement - 22% anemia, 8% splenomegaly) with a median duration of response of 8.5 months. Toxicities primarily consisted of neuropathy (Grade 3 or higher neuropathy in 6%) and cytopenias (Grade 3 or higher in 20%). After a median follow-up of three years, fourteen patients (28%) had expired from MF at the time of this analysis with median survival of 36 months.
Division of Hematology/Oncology, Mayo Clinic Scottsdale, Arizona, USA.
Prakash
P
Thapaliya
Ayalew
A
Tefferi
Animesh
A
Pardanani
David P
DP
Steensma
John
J
Camoriano
Wenting
W
Wu
Susan
S
Geyer
Ruben A
RA
Mesa
D000368:Aged; D000369:Aged, 80 and over; D015331:Cohort Studies; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011241:Prednisone / Q000627:therapeutic use*; D055728:Primary Myelofibrosis / Q000188:drug therapy*; D012189:Retrospective Studies; D013792:Thalidomide / Q000627:therapeutic use*; D016896:Treatment Outcome
D013792:Thalidomide; D011241:Prednisone