637. Myelodysplastic Syndromes—Clinical Studies: Higher Risk MDS Clinical Studies

Sunday, December 4, 2016: 9:30 AM-11:00 AM
Mrinal Patnaik, MBBS, MD, Mayo Clinic and Rami S. Komrokji, MD, H. Lee Moffitt Cancer Center & Research Institute

Eytan M. Stein, Amir T. Fathi, Courtney D. DiNardo, Daniel A Pollyea, et al. 

Discussion/Conclusion: Daily Tx with oral enasidenib monotherapy was well tolerated and induced responses in more than one-half of these MDS pts with mIDH2, 50% of whom had higher-risk disease, and two-thirds of whom had failed prior HMA Tx. Notably, one-half of evaluable MDS pts who had failed prior HMA Tx had a response, including a CR, with enasidenib monotherapy. Only 2 pts experienced disease progression during Tx. Mutational testing is rapidly becoming essential to diagnosis and prognostication in MDS, and assessment of IDH2 mutations can identify MDS pts who may benefit from targeted Tx with enasidenib.


Guillermo Garcia-Manero, Naval G. Daver, Guillermo Montalban-Bravo, Elias J. Jabbour, et al. 

CONCLUSION: Preliminary results indicate that PD-1 blockade with Nivo in combination with AZA in untreated higher-risk MDS pts is associated with a tolerable safety profile and clinical activity. Single-agent Ipi is capable of inducing responses in previously treated MDS pts. Single-agent Nivo did not show clinical activity. Further follow-up is needed to update efficacy and safety data.


Guillermo Garcia-Manero, MD1, Martin S. Tallman, MD2*, Giovanni Martinelli, MD3, Vincent Ribrag, et al.

Conclusion: PD-1 blockade with pembrolizumab was associated with a manageable safety profile and potential clinical activity in patients with MDS after failure of first-line treatment with a hypomethylating agent. Future studies in combination with azacitidine are planned.


Guillermo Montalban-Bravo,  Prithviraj Bose, Yesid Alvarado, Naval Daver, , et al. 

CONCLUSION: Guadecitabine is a well-tolerated and active new HMA for patients with higher-risk MDS and CMML even in the presence of adverse biological features such as high frequency of complex karyotype, therapy related disease and TP53 mutations. Further follow up is required to assess survival benefit in terms of OS and leukemia-free survival.


Marie Sebert, Cecile Bally, Pierre Peterlin,Odile Beyne-Rauzy, et al.

Conclusion: Response rates with GDAC, in this population of higher risk MDS, CMML or low blast count AML with failure to AZA (and often with IWG 2006 progression) were modest. Tolerance was similar to that of conventional HMA treatment.


Brian Ball, Rami S. Komrokji, Lionel Ades, Mikkael A. Sekeres, et al.

Conclusions. This large cohort of patients treated with IC after HMA failure confirms the relatively dismal outcome of this patient group. No induction strategy is superior for outcomes of interest, nor more toxic. IC-post-HMA is a valid treatment option, with response rates and transplant rates that exceed what is observed with other treatment modalities. The benefit of IC-post-HMA may be maximized by patient selection: non-adverse cytogenetics, no prior exposure to IC, and availability of an allogeneic donor seem to be the key factors.


Poster: Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low-Intermediate Risk Myelodysplastic Syndromes (MDS)

Long-Term Results from Phase 2 PACE‐MDS Study

Uwe Platzbecker, Ulrich Germing, Katharina Götze, Philipp Kiewe, et al.


  • Lower-risk MDS patients treated with luspatercept demonstrated robust and sustained increases in hemoglobin and decreases in transfusion burden (per IWG HI-E) and a high rate of RBC transfusion independence 
  • In patients with EPO levels < 200, response rates were similar in both RS+ and RS- patients 
  • In patients with EPO levels ≥200 to ≤500, response rates were higher in RS+ compared to RS- patients 
  • These data support the initiation of Phase 3 studies of luspatercept in lower-risk MDS