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.
BackgroundClarithromycin 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 presentationHere 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.ConclusionAdditional investigations should be considered to elucidate the therapeutic effect of roxithromycin in myeloma.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.