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Should patients with refractory 
anemia with excess blasts or those 
with oligoblastic AML receive 
induction therapy prior to allogeneic 
transplantation? 
Nina Shah 
Assistant Professor 
Department of Stem Cell Transplantation 
M.D. Anderson Cancer Center
Allogeneic Transplant for MDS 
• The only curative option 
• Greater experience has made this treatment a 
reality for older individuals 
• Traditionally “less disease is better” 
• But does this hold true for MDS?
The pros of chemo before allo SCT 
• Historically the fewer blasts (AML) the better 
• Timing: coordinating the transplant can take 
1-2 months
The cons of chemo before transplant 
• Toxicity 
• Worsening cytopenias 
• Infections more delays 
• No clear prospective data to show benefit 
• And what about the hypomethylating agents?
Evidence 
• Nakai et al (Leukemia, 2005) performed a 
retrospective review of 283 pts who underwent 
matched related donor allo transplantfor MDS 
• Compared patients who did and did not receive 
induction chemotherapy before transplant (RAEB-t 
or secondary AML) 
• OS at 5 years was 57% for patients who 
underwent allo-SCT as a primary treatment and 
54% for those who underwent allo-SCT in 
remission after induction chemotherapy (P=0.81).
Evidence cont. 
• Scott et al (BBMT 2005) 
• Retrospective review of 125 pts who 
underwent related or MUD SCT for MDS or 
tAML 
• 33 pts received induction chemo before SCT; 
92 did not 
• No benefit in post-transplant outcomes for 
those pt who received pre-transplant 
induction chemotherapy
Hypomethylating agents 
• Less intense than induction chemotherapy 
• Higher CR rates with induction chemotherapy IC 
than with HMAs, although HMAs may be active in 
patients with complex karyotypes in whom IC 
almost invariably fails 
• But patients can still have cytopenias/toxicities 
• “All interventions aimed at reducing disease 
burden before allo-SCT expose patients to the risk 
of complications, which may prevent them from 
undergoing transplantation” 
Yakoub-Agha, BBMT 2014
MD Anderson Data 
• N= 256 pts with MDS undergoing allo SCT after 2001 
• 133 matched related, 123 MUD 
– 78 (30.5%): no pre-SCT therapy 
– 40 (15.6%) received chemotherapy 
– 122 (47.7%) received HMA 
– 16 (6.2%) received both (chemo+HMA) 
• Outcomes were similar in patients who were untreated 
and who received cytoreductive therapy before HSCT 
• Three-year event-free survival (EFS) was 44.2%, 30.6%, 
34.2%, and 32.8% respectively, (P = .50) 
• Monosomal karyotype was a particularly poor 
prognositc factor 
Oran, BBMT 2014
OS after allo SCT according to best 
response to HMA before SCT 
Oran, Clin Lymph Myel Leuk 2013
Conclusion 
• Though it is tempting to treat MDS and 
oligoblastic AML with conventional 
chemotherapy these diseases may have a 
different pace from classic AML 
• The most curative option is allogeneic SCT 
• There is no prospective evidence that 
conventional chemotherapy before allo SCT 
improves patient outcomes 
• Patients should proceed as quickly as possible to 
allo SCT

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oligoblastic AML

  • 1. Should patients with refractory anemia with excess blasts or those with oligoblastic AML receive induction therapy prior to allogeneic transplantation? Nina Shah Assistant Professor Department of Stem Cell Transplantation M.D. Anderson Cancer Center
  • 2. Allogeneic Transplant for MDS • The only curative option • Greater experience has made this treatment a reality for older individuals • Traditionally “less disease is better” • But does this hold true for MDS?
  • 3. The pros of chemo before allo SCT • Historically the fewer blasts (AML) the better • Timing: coordinating the transplant can take 1-2 months
  • 4. The cons of chemo before transplant • Toxicity • Worsening cytopenias • Infections more delays • No clear prospective data to show benefit • And what about the hypomethylating agents?
  • 5. Evidence • Nakai et al (Leukemia, 2005) performed a retrospective review of 283 pts who underwent matched related donor allo transplantfor MDS • Compared patients who did and did not receive induction chemotherapy before transplant (RAEB-t or secondary AML) • OS at 5 years was 57% for patients who underwent allo-SCT as a primary treatment and 54% for those who underwent allo-SCT in remission after induction chemotherapy (P=0.81).
  • 6. Evidence cont. • Scott et al (BBMT 2005) • Retrospective review of 125 pts who underwent related or MUD SCT for MDS or tAML • 33 pts received induction chemo before SCT; 92 did not • No benefit in post-transplant outcomes for those pt who received pre-transplant induction chemotherapy
  • 7. Hypomethylating agents • Less intense than induction chemotherapy • Higher CR rates with induction chemotherapy IC than with HMAs, although HMAs may be active in patients with complex karyotypes in whom IC almost invariably fails • But patients can still have cytopenias/toxicities • “All interventions aimed at reducing disease burden before allo-SCT expose patients to the risk of complications, which may prevent them from undergoing transplantation” Yakoub-Agha, BBMT 2014
  • 8. MD Anderson Data • N= 256 pts with MDS undergoing allo SCT after 2001 • 133 matched related, 123 MUD – 78 (30.5%): no pre-SCT therapy – 40 (15.6%) received chemotherapy – 122 (47.7%) received HMA – 16 (6.2%) received both (chemo+HMA) • Outcomes were similar in patients who were untreated and who received cytoreductive therapy before HSCT • Three-year event-free survival (EFS) was 44.2%, 30.6%, 34.2%, and 32.8% respectively, (P = .50) • Monosomal karyotype was a particularly poor prognositc factor Oran, BBMT 2014
  • 9. OS after allo SCT according to best response to HMA before SCT Oran, Clin Lymph Myel Leuk 2013
  • 10. Conclusion • Though it is tempting to treat MDS and oligoblastic AML with conventional chemotherapy these diseases may have a different pace from classic AML • The most curative option is allogeneic SCT • There is no prospective evidence that conventional chemotherapy before allo SCT improves patient outcomes • Patients should proceed as quickly as possible to allo SCT