mepitiostane and Anemia--Refractory

We retrospectively evaluated the efficacy of androgen in the treatment of refractory anemia (RA) and compared patient characteristics and the probability of survival in androgen-responder and nonresponder groups. Forty patients with RA were treated in our hospital between 1975-1989, and 27 were treated with various derivatives of androgen. Eleven of the latter responded effectively to androgen therapy, representing an efficacy rate of 40.7%, higher than that of any other treatments thus far reported. The probability of 10-year survival estimated by the Kaplan-Meier method was 75.0% for the responder group and 41.3% for nonresponders, with a median follow-up of 1202 and 1272 days, respectively. In addition, the percent probability of transformation-free survival was higher among androgen-responders than among nonresponders, though the difference was not significant. Transformation from RA to RAEB or overt leukemia was seen in only one case among the former group, but in six among the latter. With respect to patient characteristics, only the percentage of marrow myeloblasts differed significantly between the groups.

Topics: Adolescent; Adult; Aged; Aged, 80 and over; Androstanols; Anemia, Refractory; Danazol; Drug Evaluation; Fluoxymesterone; Follow-Up Studies; Humans; Life Tables; Methenolone; Middle Aged; Retrospective Studies

Forty-eight patients with refractory anemia (RA) were retrospectively analyzed for their prognosis and subclassified into three groups: 12 patients with hematological improvement (A), 23 patients with no changes (B), and 13 patients with progression to RAEB or acute leukemia (C). For all patients, the median survival were 49.2 months, and the rate of leukemic transformation was 16%. The median survivals were 60.6, 32.1, and 17.9 months, respectively, for groups A, B and C. The factors indicating poor prognosis were low reticulocyte counts, low neutrophil alkaline phosphatase activity, low% red cell utilization, high M/E ratio, high blast percentage in the bone marrow and cytological abnormalities in the granulocyte and megakaryocyte series. By using multiple discriminant analysis, we obtained a formula for the prognostic estimation with a discrimination probability of 62.5%. This formula could predict either the patients with good (Y greater than 0.85) or poor (Y less than 0.59) prognosis, and might be useful to select the treatment for this intractable anemia at the time of diagnosis.

Topics: Adolescent; Adult; Aged; Aged, 80 and over; Androstanols; Anemia, Refractory; Danazol; Discriminant Analysis; Drug Therapy, Combination; Female; Humans; Male; Methenolone; Middle Aged; Oxymetholone; Prognosis; Retrospective Studies

In a cooperative trial, 99 patients with refractory anemias were treated according to a standard protocol consisting of successive administration of prednisolone (PSL), 1 alpha hydroxyvitamin D3 (D3) and androgens. The primary objective of this protocol was to ameliorate peripheral cytopenia and to assess the value of the 3 drugs, claimed to be of some value, in the management of patients with refractory anemias. In principle, patients were initially treated with oral PSL 40-60 mg/day. Patients not responding to PSL within 4 weeks had an 8-week trial of D3 starting with 1 microgram/day, increasing to 3 micrograms/day. Those not responding to either of the two were given AS. Although the number of evaluable courses is limited as yet for each drug, the preliminary results suggest 1) a slight to moderate increase in hemoglobin in 20-25% of patients on PSL, 2) a partial increase in granulocyte and/or platelets and a reduction of marrow blasts in 10-20% of patients during the administration of D3 and 3) the effects are poorer in cases of severe cytopenia. In addition, the improvement was mostly transient. The treatment was well tolerated in the majority of patients.

Topics: Administration, Oral; Androgens; Androstanols; Anemia, Refractory; Cholecalciferol; Drug Administration Schedule; Drug Therapy, Combination; Humans; Oxymetholone; Prednisolone