Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients.
Hydroxyurea (HU) is frequently given as treatment for myelofibrosis (MF), but data on its efficacy and tolerability are scarce. The results of HU therapy were evaluated in 40 patients with hyperproliferative manifestations of primary (n = 32), post-polycythemia vera (n = 6), or post-essential thrombocythemia (n = 2) myelofibrosis. Median interval between diagnosis and HU start was 6.2 months (range 0-141.7). Reasons for treatment were constitutional symptoms (55%), symptomatic splenomegaly (45%), thrombocytosis (40%), leukocytosis (28%), pruritus (10%), and bone pain (8%). The starting dose was 500 mg/day, subsequently adjusted to the individual efficacy and tolerability. Response was bone pain 100%, constitutional symptoms 82%, pruritus 50%, splenomegaly 40%, and anemia 12.5%. According to the International Working Group for Myelofibrosis Research and Treatment criteria, clinical improvement was achieved in 16 patients (40%). Median duration of response was 13.2 months (range 3-126.2). Worsening of the anemia or appearance of pancytopenia were observed in 18 patients, requiring administration of erythropoietin-stimulating agents (n = 17) and/or danazol (n = 9). Oral or leg ulcers appeared in five patients and one had gastrointestinal symptoms. HU is an effective and generally well-tolerated therapy for the hyperproliferative manifestations of MF. The accentuation of the anemia often induced by HU is usually manageable with concomitant treatment.
Martínez-Trillos
Alejandra
A
Hematology Department, Hospital Clínic, IDIBAPS, University of Barcelona, Barcelona, Spain.
Gaya
Anna
A
Maffioli
Margherita
M
Arellano-Rodrigo
Eduardo
E
Calvo
Xavier
X
Díaz-Beyá
Marina
M
Cervantes
Francisco
F
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000150:complications / Q000188:drug therapy; D000986:Antisickling Agents / Q000009:adverse effects / Q000627:therapeutic use; D001842:Bone and Bones / Q000503:physiopathology; D005260:Female; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000627:therapeutic use*; D007871:Leg Ulcer / Q000139:chemically induced; D007964:Leukocytosis / Q000150:complications / Q000188:drug therapy; D008297:Male; D008875:Middle Aged; D019226:Oral Ulcer / Q000139:chemically induced; D010146:Pain / Q000150:complications / Q000188:drug therapy; D010198:Pancytopenia / Q000139:chemically induced; D055728:Primary Myelofibrosis / Q000150:complications / Q000188:drug therapy*; D011537:Pruritus / Q000150:complications / Q000188:drug therapy; D013163:Splenomegaly / Q000150:complications / Q000188:drug therapy; D013922:Thrombocytosis / Q000150:complications / Q000188:drug therapy; D016896:Treatment Outcome
D000986:Antisickling Agents; D006918:Hydroxyurea