Myelodysplastic Syndrome (MDS)

Myelodysplastic syndrome (MDS) is a heterogenous blood disease affecting WBCs, RBCs, and/or platelets. Although it is considered as a not-curable blood cancer, many patients can be treated with newly developed targeted therapy.

[stanleykim]

The new 5th edition of WHO classification was published recently.
It changed the name of MDS to Myelodysplastic neoplasm.
The dysplasia should be more than 10% for Dx.
Also there are some changes in classification:
For example, no clear distinction between single and multiple lineage dysplasia. Hypoplastic MDS is now a distinct type of MDS.

[stanleykim]

COMMANDS 3 trial where Luspatercept was compared with ESA (eg, Epogen, Procrit, Retacrit) for low or intermediate risk MDS showed luspatercept was better than ESA in efficacy (transfusion independency rate). However, most patients (>70%) were MDS-ring siderobladts, adverse effects were more prevalent in the luspatercept group, the mortality rates were not different between luspatercept and ESA groups.

[stanleykim]

In August 2023,
The FDA has approved luspatercept-aamt (Reblozyl) for the treatment of anemia without prior erythropoiesis-stimulating agent (ESA) use in adult patients with very low– to intermediate-risk myelodysplastic syndrome (MDS) who may require regular red blood cell (RBC) transfusions.1

The regulatory decision is supported by interim findings from the phase 3 COMMANDS trial (please read my comment below) in which luspatercept had superior efficacy of concurrent RBC transfusion independence (RBC-TI) and hemoglobin (Hb) increase vs epoetin alfa, irrespective of ring sideroblast status.

[zakiuddin]

Thanks! very good explanation of the different types of MDS 👍

[stanleykim]

Imetelstat:
New drug for EPO refractory MDS was approved by FDA.

[zachmd]

Thank you prof. i learnt much from this video and i appreciate it. from Cambodia.

[bambangsetiono]

Thank you very much for your kingly good heart, GBU dokter it is very helpful for my priest friend in indonesia

[stanleykim]

Error correction:
At 4:00 myeloblast should be myelocyte.

[amyshin]

안녕하세요. 캐나다에서 살고 있습니다. 오빠가 골수형성이상 증후군 진단을 받아서 제가 골수이식을 해줄 수 있는지 검사를 해보려고 합니다. 한국에 갈 상황이 아닌데 밴쿠버 병원 의료 기술을 믿을 수 있을지 걱정이 됩니다 의사분들께 감사하지만.. 어려운 수술이될지 궁금해서 알아보고 있는데요.. 조언 좀 들을 수 있을지요??

[stanleykim]

Error:
At 4:00, Maturation arrest at myelocyte level not myeloblast.

[shukriramlee]

Did we almost find the cure of this disease?