Danazol for the treatment of thrombocytopenia in patients with myelodysplastic syndrome

Abstract

Thrombocytopenia is a poor prognostic indicator in the myelodysplastic syndromes (MDS). Treatment options for patients with symptomatic thrombocytopenia are limited. Danazol, an attenuated androgen, may have some efficacy in increasing the platelet count of patients with MDS. We retrospectively reviewed 33 patients with primary MDS who were treated with danazol for 6 or more weeks. After 6 weeks on danazol, the mean platelet count increased from 42 × 10 9 /L to 60 × 10 9 /L (P < 0.015), and 25 out of 33 patients (76%) had an increase in their platelet counts. Following 12 weeks of treatment, the mean platelet count increased to 67 × 10 9 /L (P < 0.005), and 21 out of 29 patients (72%) had an increase in their platelet counts. Seven out of nine patients no longer required platelet transfusions because bleeding stopped after 6 weeks on danazol. Mean duration of response was 10 months (range 2-68 months). Responses were seen in all FrenchAmerican-British (FAB) subtypes and in all International Prognostic Scoring System (IPSS) scores. Therapy was well tolerated. Danazol may be effective in MDS patients who are thrombocytopenic. Am.

Cyclosporine Combined with Levamisole for Lower-Risk Myelodysplastic Syndromes

“…A previous study has described a better response to ATG in patients with trisomy 8 and found that trisomy 8 cells within BM were preferentially suppressed by cytotoxic T lymphocytes [36]. The unexpectedly encouraging outcome highlighted the complementary and synergistic properties between CsA and LMS, also at least partly due to the addition of danazol, which demonstrated the efficacy in MDS in the earlier literature [37,38], although this was controversial [39]. …”

Therapeutic Modalities for Patients with Lower-Risk Myelodysplastic Syndromes: Current Options and Future Directions

“…MDS can be associated with immune thrombocytopenic purpura (ITP) and other autoimmune disorders; these patients may respond to the combination of corticosteroids and intravenous immunoglobulin. Danazol has also been studied in MDS-associated thrombocytopenia but has not been evaluated in a large prospective trial [20]. It may be beneficial in patients who have immunemediated cell destruction.…”

Is there still a place for “old therapies” in the management of immune thrombocytopenia?

“…Danazol is also effective to treat thrombocytopenia during myelodysplastic syndromes (MDS) [51][52][53], which represent the major differential diagnosis of ITP, notably in the elderly. Thus, when the diagnosis is not clearly established between ITP and MDS despite bone marrow explorations, danazol could be an interesting therapy.…”