Danazol therapy for aplastic anemia refractory to immunosuppressive therapy

Abstract

Although there are anecdotal reports of the efficacy of danazol in the treatment of aplastic anemia (AA), there has been no systematic study to clarify its efficacy and toxicity. Therefore, we assessed the efficacy of danazol for treatment of patients with AA refractory to immunosuppressive therapy (IST) and those who relapsed after IST, in a prospective clinical trial. Sixteen patients (12 males and four females; six severe cases and 10 moderate cases) were treated with 300 mg of danazol daily for 12 weeks. All patients completed the treatment period without occurrence of severe toxicity. Three female patients achieved partial remission, whereas only two of the 12 male patients did so. None of the responders had shown a response to previous IST or an increase in the percentage of paroxysmal nocturnal hemoglobinuria (PNH)-type cells which are known to be a marker for a good response to IST. These findings indicate that danazol is effective for a subset of AA patients, and particularly for female patients with AA refractory to IST. Am. J. Hematol. 83:387-389, 2008. V

Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study

“…These results compare favorably with the results reported for drug therapy for patients with refractory SAA, with responses rates in the range of 30-50% for most therapeutic options. 6,7,9,10 In the past, the use of haploidentical donors was limited by the incidence of severe GVHD and the need for profound ex vivo T-cell depletion, which impaired immune reconstitution and contributed to an increased risk of infections, relapse and non-relapse mortality. Post-transplant Cy is an effective strategy for GVHD prevention with reduced impairment in immune reconstitution that is observed with more profound T-cell depletion strategies previously used in haploidentical HSCT.…”

“…[1][2][3][4][5] Current therapeutic strategies for these patients include a higher risk hematopoietic stem cell transplantation (HSCT) from mismatched donors, a second course of immunosuppression, androgens, and more recently, the TPO receptor agonist eltrombopag recently approved in this setting. 3,[6][7][8][9][10] Higher risk HSCT for those who lack a histocompatible donor often include umbilical cord blood and haploidentical donor as a source of HSCs (HSCs). [11][12][13][14][15][16][17][18][19] The use of haploidentical donors has some advantages compared with matched unrelated donors (MUD) and cord blood units, as a donor is more widely and readily available usually within the family, abbreviating the time for stem cell collection and infusion into the recipient.…”

Successful obstetric and hematologic outcome of aplastic anemia in a pregnant Jehovah’s Witness

“…18 In a study by Chuhjo et al, danazol was also indicated to have a positive effect on erythroid cell line in aplastic anemia, acting by enhanced erythropoiesis rather than improvement in red cell survival. 19 In our patient, after gradual improvement in blood counts was noted, the tendency towards normal values continued and she was discharged. After 9 months both the patient and the baby are in good health.…”

Flow cytometric analysis of CD 55 and CD 59 deficient cells in patients with Aplastic Anemia and their clinicohematological profile

“…(15 In our study, only three patients were on androgen monotherapy of which only one patient was followed up for the full duration of 2 years and remained in partial remission. Two patients (66%) achieved partial remission after 12 weeks of monotherapy with danazol.…”