Danazol: An Effective and Underutilised Treatment Option in Diamond-Blackfan Anaemia.

Diamond-Blackfan anaemia (DBA) is a rare congenital red cell aplasia that presents in infancy. The exact molecular mechanism of ineffective erythropoiesis and red cell aplasia remains unclear, rendering targeted therapy elusive. The mainstay treatment of DBA is with regular blood transfusion and long-term corticosteroids, both of which have long-term side effects. We report a case of DBA successfully treated with danazol, a synthetic androgen, and suggest that danazol be considered as a viable option in patients who become refractory to steroids and are considered high risk or unfit for allogeneic stem cell transplantation.

Roxithromycin monotherapy inducing a partial response in a patient with myeloma: a case report.

BACKGROUND: Clarithromycin is an efficacious treatment for myeloma in combination with other anti-myeloma therapy but not as monotherapy. To date, all studies have focused on a clarithromycin-specific effect rather than a class effect (macrolide) and there is no information on the activity of roxithromycin in myeloma. CASE PRESENTATION: Here we report an untreated 86-year-old New Zealand European white man with IgA myeloma whose paraprotein decreased by 57%, consistent with a partial response, after a course of roxithromycin for pneumonia. His paraprotein reduced from 46 to 20 g/L while his hemoglobin improved from 97 to 123 g/L after 1 month. CONCLUSION: Additional investigations should be considered to elucidate the therapeutic effect of roxithromycin in myeloma.