Page 35 - 2020_11-Haematologica-web
P. 35

Novel dynamic outcome indicators and clinical endpoints in MDS
model for treating the anaemia of myelodys- plastic syndromes with erythropoietin + granulocyte colony-stimulating factor: sig- nificant effects on quality of life. Br J Haematol. 2003;120(6):1037-1046.
15. List A, Dewald G, Bennett J, et al. Lenalidomide in the myelodysplastic syn- drome with chromosome 5q deletion. N Engl J Med. 2006;355(14):1456-1465.
16. Fenaux P, Giagounidis A, Selleslag D, et al. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-depen- dent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. Blood. 2011;118(14):3765-3776.
17. Della Porta MG, Malcovati L. Clinical rele- vance of extra-hematologic comorbidity in the management of patients with myelodys- plastic syndrome. Haematologica. 2009;94(5):602-606.
18. Rochau U, Stojkov I, Conrads-Frank A, et al. Development of a core outcome set for myelodysplastic syndromes - a Delphi study from the EUMDS Registry Group. Br J Haematol. 2020 May 14. [Epub ahead of print]
19. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring sys- tem for myelodysplastic syndromes. Blood. 2012;120(12):2454-2465.
20. Itzykson R, Crouch S, Travaglino E, et al. Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syn- dromes. Blood Adv. 2018;2(16):2079-2089.
21. de Swart L, Crouch S, Hoeks M, et al. Impact of red blood cell transfusion dose density on progression-free survival in lower-risk myelodysplastic syndromes patients. Haematologica. 2020;105(3):632- 639.
22. Stauder R, Lambert J, Desruol-Allardin S, et al. Patient-reported outcome measures in studies of myelodysplastic syndromes and acute myeloid leukemia: literature review and landscape analysis. Eur J Haematol. 2020;104(5):476-487.
23. Stauder R, Yu G, Koinig KA, et al. Health- related quality of life in lower-risk MDS patients compared with age- and sex- matched reference populations: a European LeukemiaNet study. Leukemia. 2018;32(6): 1380-1392.
24. Brooks R. EuroQol: the current state of play. Health Policy. 1996;37(1):53-72.
25. Greiner W, Weijnen T, Nieuwenhuizen M, et al. A single European currency for EQ-5D
health states. Results from a six-country
study. Eur J Health Econ. 2003;4(3):222-231. 26. Langelaan M, de Boer MR, van Nispen RM, Wouters B, Moll AC, van Rens GH. Impact of visual impairment on quality of life: a comparison with quality of life in the gener- al population and with other chronic condi- tions. Ophthalmic Epidemiol. 2007;14(3):
119-126.
27.Abel GA, Efficace F, Buckstein RJ, et al.
Prospective international validation of the Quality of Life in Myelodysplasia Scale (QUALMS). Haematologica. 2016;101(6):781-788.
28. Nilsson-Ehle H, Birgegard G, Samuelsson J, et al. Quality of life, physical function and MRI T2* in elderly low-risk MDS patients treated to a haemoglobin level of >/=120 g/L with darbepoetin alfa +/- filgrastim or ery- throcyte transfusions. Eur J Haematol. 2011; 87(3):244-252.
29. Platzbecker U, Symeonidis A, Oliva EN, et al. A phase 3 randomized placebo-controlled trial of darbepoetin alfa in patients with ane- mia and lower-risk myelodysplastic syn- dromes. Leukemia. 2017;31(9):1944-1950.
30. Jadersten M, Malcovati L, Dybedal I, et al. Erythropoietin and granulocyte-colony stimulating factor treatment associated with improved survival in myelodysplastic syn- drome. J Clin Oncol. 2008;26(21):3607-3613.
31. Park S, Grabar S, Kelaidi C, et al. Predictive factors of response and survival in myelodysplastic syndrome treated with ery- thropoietin and G-CSF: the GFM experi- ence. Blood. 2008;111(2):574-582.
32. Garelius HK, Johnston WT, Smith AG, et al. Erythropoiesis-stimulating agents signifi- cantly delay the onset of a regular transfu- sion need in nontransfused patients with lower-risk myelodysplastic syndrome. J Intern Med. 2017;281(3):284-299.
33. Shenoy N, Vallumsetla N, Rachmilewitz E, Verma A, Ginzburg Y. Impact of iron over- load and potential benefit from iron chela- tion in low-risk myelodysplastic syndrome. Blood. 2014;124(6):873-881.
34.Ganz T. Hepcidin and iron regulation, 10 years later. Blood. 2011;117(17):4425-4433.
35. de Swart L, Reiniers C, Bagguley T, et al. Labile plasma iron levels predict survival in patients with lower-risk myelodysplastic syndromes. Haematologica. 2018;103(1):69- 79.
36. Porter JB, de Witte T, Cappellini MD, Gattermann N. New insights into transfu-
sion-related iron toxicity: implications for the oncologist. Crit Rev Oncol Hematol. 2016;99:261-271.
37.Neukirchen J, Fox F, Kundgen A, et al. Improved survival in MDS patients receiv- ing iron chelation therapy - a matched pair analysis of 188 patients from the Dusseldorf MDS registry. Leuk Res. 2012;36(8):1067- 1070.
38. Rose C, Brechignac S, Vassilief D, et al. Does iron chelation therapy improve survival in regularly transfused lower risk MDS patients? A multicenter study by the GFM (Groupe Francophone des Myelodysplasies). Leuk Res. 2010;34(7):864-870.
39. Gattermann N, Finelli C, Della Porta M, et al. Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes. Haematologica. 2012;97(9):1364-1371.
40. Angelucci E, Santini V, Di Tucci AA, et al. Deferasirox for transfusion-dependent patients with myelodysplastic syndromes: safety, efficacy, and beyond (GIMEMA MDS0306 Trial). Eur J Haematol. 2014;92 (6):527-536.
41. Breccia M, Voso MT, Aloe Spiriti MA, et al. An increase in hemoglobin, platelets and white blood cells levels by iron chelation as single treatment in multitransfused patients with myelodysplastic syndromes: clinical evidences and possible biological mecha- nisms. Ann Hematol. 2015;94(5):771-777.
42. Angelucci E, Li J, Greenberg P, et al. Iron chelation in transfusion-dependent patients with low- to intermediate-1-risk myelodys- plastic syndromes: a randomized trial. Ann Intern Med. 2020;172(8):513-522.
43. Schafer AI, Cheron RG, Dluhy R, et al. Clinical consequences of acquired transfu- sional iron overload in adults. N Eng J Med. 1981;304(6):319-324.
44. de Swart L, Hendriks JC, van der Vorm LN, et al. Second international round robin for the quantification of serum non-transferrin- bound iron and labile plasma iron in patients with iron-overload disorders. Haematologica. 2016;101(1):38-45.
45. Malcovati L, Karimi M, Papaemmanuil E, et al. SF3B1 mutation identifies a distinct sub- set of myelodysplastic syndrome with ring sideroblasts. Blood. 2015;126(2):233-241.
46. Fenaux P, Kiladjian JJ, Platzbecker U. Luspatercept for the treatment of anemia in myelodysplastic syndromes and primary myelofibrosis. Blood. 2019;133(8):790-794.
haematologica | 2020; 105(11)
2523


































































































   33   34   35   36   37