Page 37 - 2020_07-Haematologica-web

Personalized management of MDS
multicenter study of the European Society of Blood and Marrow Transplantation. Haematologica. 2015;100(3):400-408.
161. Deeg HJ, Scott BL, Fang M, et al. Five-group cytogenetic risk classification, monosomal karyotype, and outcome after hematopoiet- ic cell transplantation for MDS or acute leukemia evolving from MDS. Blood. 2012;120(7):1398-1408.
162. Kroger N, Iacobelli S, Franke GN, et al. Dose-reduced versus standard conditioning followed by allogeneic stem-cell transplan- tation for patients with myelodysplastic syndrome: a prospective randomized phase III study of the EBMT (RICMAC Trial). J Clin Oncol. 2017;35(19):2157-2164.
163.CutlerCS,LeeSJ,GreenbergP,etal.Adeci- sion analysis of allogeneic bone marrow transplantation for the myelodysplastic syn- dromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood. 2004;104(2):579-585.
164. Della Porta MG, Alessandrino EP, Bacigalupo A, et al. Predictive factors for the outcome of allogeneic transplantation in patients with MDS stratified according to the revised IPSS-R. Blood. 2014;123(15): 2333-2342.
165. Alessandrino EP, Della Porta MG, Bacigalupo A, et al. WHO classification and WPSS predict posttransplantation outcome in patients with myelodysplastic syndrome: a study from the Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Blood. 2008;112(3):895-902.
166. Della Porta MG, Galli A, Bacigalupo A, et al. Clinical effects of driver somatic mutations on the outcomes of patients with myelodys- plastic syndromes treated with allogeneic hematopoietic stem-cell transplantation. J Clin Oncol. 2016;34(30):3627-3637.
167.Bejar R, Stevenson KE, Caughey B, et al. Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplanta- tion. J Clin Oncol. 2014;32(25):2691-2698.
168.Yoshizato T, Nannya Y, Atsuta Y, et al. Genetic abnormalities in myelodysplasia and secondary acute myeloid leukemia: impact on outcome of stem cell transplanta- tion. Blood. 2017;129(17):2347-2358.
169.Lindsley RC, Saber W, Mar BG, et al. Prognostic mutations in myelodysplastic syndrome after stem-cell transplantation. N Engl J Med. 2017;376(6):536-547.
170.Yahng SA, Kim M, Kim TM, et al. Better transplant outcome with pre-transplant mar- row response after hypomethylating treat- ment in higher-risk MDS with excess blasts. Oncotarget. 2017;8(7):12342-12354.
171. Damaj G, Mohty M, Robin M, et al. Upfront allogeneic stem cell transplantation after reduced-intensity/nonmyeloablative condi- tioning for patients with myelodysplastic syndrome: a study by the Societe Francaise de Greffe de Moelle et de Therapie Cellulaire. Biol Blood Marrow Transplant. 2014;20(9):1349-1355.
172.Oran B, Kongtim P, Popat U, et al. Cytogenetics, donor type, and use of hypomethylating agents in myelodysplastic syndrome with allogeneic stem cell trans- plantation. Biol Blood Marrow Transplant. 2014;20(10):1618-1625.
173. Schroeder T, Wegener N, Lauseker M, et al. Comparison between upfront transplanta-
tion and different pretransplant cytoreduc- tive treatment approaches in patients with high-risk myelodysplastic syndrome and secondary acute myelogenous leukemia. Biol Blood Marrow Transplant. 2019;25(8):1550-1559.
174.Damaj G, Duhamel A, Robin M, et al. Impact of azacitidine before allogeneic stem-cell transplantation for myelodysplas- tic syndromes: a study by the Société Française de Greffe de Moelle et de Thérapie-Cellulaire and the Groupe- Francophone des Myélodysplasies. J Clin Oncol. 2012;30(36):4533-4540.
175.Beelen DW, Trenschel R, Stelljes M, et al. Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid leukaemia or myelodysplastic syndrome (MC- FludT.14/L): a randomised, non-inferiority, phase 3 trial. Lancet Haematol. 2020;7(1):e28-e39.
open-label phase I dose-finding study of APR-246 in hematological malignancies. Blood Cancer J. 2016;6(7):e447.
186. Sallman DA, DeZern AE, Garcia-Manero G, et al. Phase 2 results of APR-246 and azaciti- dine (AZA) in patients with TP53 mutant myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia (AML). Blood. 2019;134(Suppl_1):676.
187. Carvajal LA, Ben-Neriah D, Senecal A, et al. Dual inhibition of Mdmx and Mdm2 using an alpha-helical P53 stapled peptide (ALRN- 6924) as a novel therapeutic strategy in acute myeloid leukemia. Blood. 2017;130 (Suppl_1):795.
188. Rudolph KL, Chang S, Lee H-W, et al. Longevity, stress response, and cancer in aging telomerase-deficient mice. Cell. 1999;96(5):701-712.
189. di Fagagna FdA, Reaper PM, Clay-Farrace L, et al. A DNA damage checkpoint response in telomere-initiated senescence. Nature. 2003;426(6963):194-198.
190. Tefferi A, Lasho TL, Begna KH, et al. A pilot study of the telomerase inhibitor imetelstat for myelofibrosis. N Engl J Med. 2015;373(10):908-919.
191. Platzbecker U, Steensma DP, Van Eygen K, et al. Imerge: a study to evaluate imetelstat (GRN163L) in transfusion-dependent sub- jects with IPSS low or intermediate-1 risk myelodysplastic syndromes (MDS) that is relapsed/refractory to erythropoiesis-stimu- lating agent (ESA) treatment. Blood. 2019;134(Suppl_1):4248.
192. Bataller A, Montalban-Bravo G, Soltysiak KA, et al. The role of TGFβ in hematopoiesis and myeloid disorders. Leukemia. 2019;33(5):1076-1089.
193.Zhou L, Nguyen AN, Sohal D, et al. Inhibition of the TGF-β receptor I kinase promotes hematopoiesis in MDS. Blood. 2008;112(8):3434-3443.
194. Suragani RN, Cadena SM, Cawley SM, et al. Transforming growth factor-β superfamily ligand trap ACE-536 corrects anemia by pro- moting late-stage erythropoiesis. Nat Med. 2014;20(4):408-414.
195.Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382(2):140-151.
196.Kurtz SE, Eide CA, Kaempf A, et al. Molecularly targeted drug combinations demonstrate selective effectiveness for myeloid-and lymphoid-derived hematologic malignancies. Proc Natl Acad Sci U S A. 2017;114(36):E7554-E7563.
176.
177.
Casper J, Knauf W, Kiefer T, et al. Treosulfan and fludarabine: a new toxicity-reduced conditioning regimen for allogeneic hematopoietic stem cell transplantation. Blood. 2004;103(2):725-731.
Ruutu T, Volin L, Beelen DW, et al. Reduced- toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplas- tic syndromes: final results of an internation- al prospective phase II trial. Haematologica. 2011;96(9):1344-1350.
178.Schroeder T, Rachlis E, Bug G, et al. Treatment of acute myeloid leukemia or myelodysplastic syndrome relapse after allo- geneic stem cell transplantation with azaciti- dine and donor lymphocyte infusions--a ret- rospective multicenter analysis from the German Cooperative Transplant Study Group. Biol Blood Marrow Transplant. 2015;21(4):653-660.
179.DiNardo CD, Stein EM, de Botton S, et al. Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. N Engl J Med. 2018;378(25):2386-2398.
180.Stein EM, Fathi AT, DiNardo CD, et al. Enasidenib in patients with mutant IDH2 myelodysplastic syndromes: a phase 1 sub- group analysis of the multicentre, AG221-C- 001 trial. Lancet Haematol. 2020;7(4):e309- e319.
181. Vousden KH, Lu X. Live or let die: the cell's response to p53. Nat Rev Cancer. 2002;2(8):594-604.
182. Wei AH, Garcia JS, Borate U, et al. A phase 1b study evaluating the safety and efficacy of venetoclax in combination with azaciti- dine in treatment-naïve patients with high- er-risk myelodysplastic syndrome. Blood. 2019;134(Suppl_1):568.
183. Zeidan AM, Pollyea DA, Garcia JS, et al. A phase 1b study evaluating the safety and efficacy of venetoclax as monotherapy or in combination with azacitidine for the treat- ment of relapsed/refractory myelodysplastic syndrome. Bood. 2019;134(Suppl_1):565.
184. Kulasekararaj AG, Smith AE, Mian SA, et al. TP 53 mutations in myelodysplastic syn- drome are strongly correlated with aberra- tions of chromosome 5, and correlate with adverse prognosis. Br J Haemat. 2013;160(5):660-672.
185. Deneberg S, Cherif H, Lazarevic V, et al. An
197.
Swords RT, Azzam D, Al-Ali H, et al. Ex- vivo sensitivity profiling to guide clinical decision making in acute myeloid leukemia: a pilot study. Leuk Res. 2018;64:34-41.
198.Spinner MA, Aleshin A, Santaguida MA, et al. A feasibility study of biologically focused therapy for myelodysplastic syndrome patients refractory to hypomethylating agents. Blood (2019):134: (Suppl 1):4239.
199. Drusbosky LM, Cogle CR. Computational modeling and treatment identification in the myelodysplastic syndromes. Curr Hematol Malig Rep. 2017;12(5):478-483.
200.Drusbosky LM, Singh NK, Hawkins KE, et al. A genomics-informed computational biology platform prospectively predicts treatment responses in AML and MDS patients. Blood Adv. 2019;3(12):1837-1847.
haematologica | 2020; 105(7)
1779