MDS

Question 1

epidemiology

Answer 1

median onset 7th decade,

60% men

Question 2

cytogenetics standard (testable)

Answer 2

5q- only 25% progress to leukemia (low frequency, better survival);

other cytogenetics probably not testable

Question 3

5q- syndrome -6

Answer 3

refractory anemia,

mild leukop,

atypical megakaryocytes,

normal to elevated plts,

transfusion dependence,

extended survival

Question 4

clinical sx

Answer 4

fever, pallor, infx, bruising, fatigue, dyspnea on exertion, bleeding

Question 5

lab sx. -5

Answer 5

macrocytic anemia,

neutrop,

thrombocytop,

nucleated RBC,

peripheral blasts

Question 6

classification systems

Answer 6

WHO replaced FAB

Question 7

international prognostic scoring system for MDS variables -3

Answer 7

cytogentics, %BM blasts, Hgb, Plt, ANC

ranges from 0.8 yrs to 8.8yrs OS v.high risk to v.low,

time to 25% AML conversion increases with score from 0.73yrs to 10.8yr for low and NOT reached for v. low

Question 8

factors to consider for treatment

Answer 8

IPSS score, 
WHO/FAB classification
age
PS
HLA-DR status
cytogenetic status
comorbidity

Question 9

goal of hypomethylating agents

Answer 9

delay transformation to AML, NOT improve OS

Question 10

tx options for MDS (5pts)

Answer 10

1. erythropoietic agents (epo, darbe, g-csf, gm-csf)
2. supportive measures (transfusion, chelation tx)
3. hypomethylating agents (azacitidine, decitabine)
4. immunomodulation drugs (lenalid FDA approved for 5q-)
5. SCT / induction chemo

Question 11

management of anemia - transfusions (5pts)

Answer 11

most pt’s require,

goal hgb >10,

iron chelation (defuroxime) needed if >20-30 units,

infx complications,

dec OS if transfusion dependent

Question 12

iron chelation therapy indx -6 and Duration -1

Answer 12

1. INDICATIONS:
   serum ferritin reaches 1000 mg/L,

transfusion 2x/mo for at least 1 yr (ie. transfusion dependent),

low risk MDS (as will live longer),

need to preserve organ function,

alloSCT candidates (will need more transfusions),

life expectancy greater than 1yr

—con’t as long as needed, agent based on availability, reimbursement

Question 13

ECOG phase III EPO trial

Answer 13

epo 150units/m2/d -> if NO response EPO + gcsf 1mg/kg/d ->if NO response EPO 300units/m2/d + gcsf

Question 14

azacitidine vs supportive care (BSC) and vs induction chemo pre HCT and vs HCT

Answer 14

75mg/m2 SC or IV D1-7 q28d

#BSC: CR 7% vs 0, 
OS 24.5 vs 16mo (p=sig)

Question 15

decitibine vs BSC

Answer 15

ROLE: if can’t tolerate more intense chemo= older pts, more comorbidities

#CR 9% vs 0, 
median duration response 9.5 vs 7.6mo, 
OS (NS), 
AML at 1 yr 22 vs 33% (sig), 
PFS 6.6 vs 3mo (sig)

Question 16

lenalidomide (in 5q- pts ONLY) - phase II trial

Answer 16

better median Hgb, CCyR, PCyR

Question 17

alloSCT

Answer 17

high risk up front, better long term OS