Recombinant human erythropoietin for the treatment of anaemia in patients with chronic idiopathic myelofibrosis.
BACKGROUND
Patients with chronic idiopathic myelofibrosis (CIMF) usually present with anaemia. Treatment is often palliative and the majority of patients receive regular red blood cell (RBC) transfusions. Although recombinant human erythropoietin (rhu-EPO) has been proved effective for the treatment of anaemia in several chronic diseases, including haematological malignancies, its role in the treatment of the anaemia in CIMF is not well established. We report the beneficial effect of rhu-EPO administration in 20 patients with CIMF and discuss the parameters predicting favourable response.


METHODS
Twenty patients with CIMF (9 women and 11 men) regularly treated with supportive RBC transfusions were included in the study. The median age was 70 years (range 45-81 years). Rhu-EPO, 10,000 U, was given subcutaneously 3 times a week. The median duration of therapy was 83 months, ranging from 13 to 87 months.


RESULTS
Treatment was considered effective if haemoglobin levels increased over 2 g/dl within 12 weeks after enrolment or the RBC transfusion requirements were reduced by 50% within the same interval. Twelve patients (60%) responded to therapy. Responders were mainly female, had smaller spleen size (p = 0.024), low RBC transfusion requirements (< or = 1-2 units per month), and significantly lower endogenous serum erythropoietin (EPO) and beta2-microglobulin (beta2-M) levels when compared with non-responders (p < 0.0001 and 0.00001, respectively). Treatment was well tolerated and none of the patients was withdrawn from the treatment protocol because of side effects.


CONCLUSIONS
Rhu-EPO administration is an effective, safe and well-tolerated treatment for patients with CIMF and anaemia leading to a significant reduction in RBC transfusion requirements. Factors predicting favourable response are low endogenous EPO and beta2-M serum levels and slight to moderate splenomegaly.
Tsiara
S N
SN
Department of Haematology, School of Medicine, Ioannina, Greece. stsiara@cc.uoi.gr
Chaidos
A
A
Bourantas
L K
LK
Kapsali
H D
HD
Bourantas
K L
KL
D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000188:drug therapy* / Q000209:etiology*; D002908:Chronic Disease; D017707:Erythrocyte Transfusion; D004921:Erythropoietin / Q000008:administration & dosage* / Q000097:blood; D005260:Female; D006454:Hemoglobins; D006801:Humans; D008297:Male; D008875:Middle Aged; D055728:Primary Myelofibrosis / Q000150:complications*; D011994:Recombinant Proteins; D013154:Spleen / Q000473:pathology; D016896:Treatment Outcome; D001613:beta 2-Microglobulin / Q000097:blood
D006454:Hemoglobins; D011994:Recombinant Proteins; D001613:beta 2-Microglobulin; D004921:Erythropoietin