title: Efficacy and tolerability of danazol as a treatment for the anaemia of myelofibrosis with myeloid metaplasia: long-term results in 30 patients.
abstract: Androgens are considered the treatment of choice for the anaemia of myelofibrosis with myeloid metaplasia (MMM). Good results have been reported in a few patients treated with danazol, a synthetic attenuated androgen. The long-term efficacy and tolerability of danazol as treatment for the anaemia of MMM was evaluated in 30 patients, who received 600 mg/d, with progressive tapering to the minimum effective dose in the responders after 6 months. Complete response (CR) was defined as transfusion cessation with normal Hb and partial response (PR) as an Hb increase >/=1.5 g/dl with transfusion-independent Hb values >10 g/dl maintained for at least 8 weeks. Median follow-up was 20.5 months (range: 3.5-58 months). Response was achieved in 11 patients (37%), including eight CRs and three PRs. Median time to response was 5 months (range: 1-9 months). Four patients stopped responding at 6-24 months, two responders discontinued treatment because of toxicity, and five maintained response at 3.5-42 months. Pretreatment variables associated with response were lack of transfusion requirement (P= 0.001) and higher Hb at treatment start (P= 0.02). Toxicity was usually moderate, leading to treatment withdrawal in only two cases. Danazol is effective and well tolerated in a substantial proportion of MMM patients with anaemia.
authors.affiliation: Haematology Department, Hospital Clínic, IDIBAPS, University of Barcelona, Spain. fcervan@clinic.ub.es
authors.forename: Francisco
authors.initials: F
authors.lastname: Cervantes
authors.forename: Alberto
authors.initials: A
authors.lastname: Alvarez-Larrán
authors.forename: Abel
authors.initials: A
authors.lastname: Domingo
authors.forename: Eduardo
authors.initials: E
authors.lastname: Arellano-Rodrigo
authors.forename: Emili
authors.initials: E
authors.lastname: Montserrat
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000188:drug therapy* / Q000209:etiology; D003613:Danazol / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D055728:Primary Myelofibrosis / Q000150:complications*; D011379:Prognosis; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D003613:Danazol