Low risk MDS is one of the Common Referral in Hematology Opd -

Low risk MDS is one of the Common Referral in Hematology Opd -

Here are some clinical pearls which help Physicians

If any doubts feel free to share ur questions …

Treatment of low Risk MDS

Goal Of Treatment : make them free of transfusion

If del 5q

Relatively good Prognosis

Alan list : Lenalidomide ( Game changer )

67 percentage benefit from Lenalidomide in del 5q

33 percentage don’t respond with Lenalidomide ( if 5q del is present along with Tp53 or complex cytogenetics )

Note : If u have refractory anemia , macrocytosis , elderly women

Must do karyotyping / Fish for del 5q

Also look for del 17p ( if negative check NGS for Tp53 aberration)

Lenalidomide works in 67 percentage of MDS with 5q deletion and if Tp53 is present Lenalidomide doesn’t work

So , MDS with 5q if Lenalidomide is not working check for Tp53

Usual time to have response is 2months

Does Lenalidomide work even in absence of 5q

Yes… 27 percentage of patients respond with Lenalidomide

Valeria santini : Phase 3 paper showed that

Len works even in absence of 5q deletion in low risk MDS


If 5q deletion is absent .

Check for EPO levels

If EPO below 250 ( atleast less than 500 )

ESA… Erythropoietin stimulating agents works

Darbopoieitin and Erythropoietin works

Predictors of response for ESA

a) EPO less than 250

b) less blood transfusion dependence ( less than 2 bags / month)

c) Absence of 5q

d) No complex mutation

e) Absence of ringed sideroblasts

f) Absence of SF3B1

Darbopoieitin ( Cresp )

Response occurs with in two months

Seen in 20 to 40 percentage

Dose : 500 ug s/c 2 to 3 weeks

Those with in adequate response can add GCSF ( additional response of 10 to 20 pts ) .

Should be started early .

3 Combination therapy

Lenalidomide plus ESA

Works in 20 percentage of people

4 … Immunosuppresants

Young age


Short duration of transfusion dependence

Hypoplastic MDS

PNH clone if present ( small)

Trisomy 8


Absence of RARS

Absence of SF3B1

5 … SF3B1 mutation


Favourable prognosis

Seen in MDS - RS

Rx … Luspatercept …


Is a TGF B ligand trapping molecule

Targets later stages of erythropoeisis

Dose is 1 to 1.75 mg/kg