Thread by @TransfusionWM

Prof Bowen delivering a talk for Prof Platzbecker on novel approaches to cytopenias in MDS #BSH2019

Around 30% of patients will respond to Eprex and license for MDS came last year
medicines.org.uk/emc/product/11…
Darbepoietin didn't meet its primary endpoint and so not licensed but real world experience is that both are in use for anaemia in MDS
#BSH2019

Median duration of response is 17 months. Sideroblastic patients respond less well. If EPO raised and transfusion dependent, only 10% will respond. All others should have a therapeutic trial. Even those with RAEB-1 may continue to respond for a good time #BSH2019

Luspatercept is a GDF11 inhibitor which removes suppression of erythropoiesis without having an effect on bone metabolism #BSH2019

Luspatercept reduces red cell and ESA requirements. SF3B1 mutated patients and those with sideroblastic patients respond better #BSH2019

Phase 3 trial MEDALIST of luspatercept every 21d vs placebo in transfusion dep pts. Del 5q and >5% blasts excluded. 226 patients randomised #BSH2019

Primary endpoint of transfusion independence met in 37.9% patients vs 13.2% in the placebo arm, p<0.0001. Improvement continues even beyond 8 weeks. Results presented at ASH and full paper awaited #BSH2019
ascopost.com/News/59528

Other novel treatments in development for anaemia in MDS include imetelstat - telomerase inhibitor. Response rates approx 35% response, independent of EPO baseline
#BSH2019

Spliceosome inhibitors: mutations such as SF3B1 are common in MDS and subtypes. H3B-8800 appears effective but carries potentially for retinal and liver toxicity #BSH2019

Eltrombopag increases platelet count in low risk MDS
thelancet.com/journals/lanha…
Romiplostim trial did not show increased survival but did demonstrate fewer bleeding events
Neither licensed in MDS currently
#BSH2019