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Current Hematologic Malignancy Reports

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11.02.2020 | Stem Cell Transplantation (R Maziarz, Section Editor)

Patient-Reported Outcomes in Myelodysplastic Syndromes: the Move from Life Span to Health Span

verfasst von: Shimoli V. Barot, Bhumika J. Patel, Aaron T. Gerds

Erschienen in: Current Hematologic Malignancy Reports | Ausgabe 2/2020

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Abstract

Purpose of Review

The lack of fully effective therapies to alter the natural course of myelodysplastic syndromes (MDS) leads to chronic morbidity, mortality and affects quality of life (QoL). Since existing therapies outside of hematopoietic cell transplantation (HCT) are not curative, there is a growing interest in incorporating patient-reported outcomes (PROs) as meaningful endpoints for these patients in research and clinical practice.

Recent Findings

Currently, there are limited numbers of studies reporting the impact of MDS therapeutics on PROs to guide clinical decision-making and increase patient satisfaction. However, clinical trials that have incorporated QoL outcomes have demonstrated positive results with the use of growth factors, hypomethylating agents, and lenalidomide.

Summary

Here we review and highlight the importance of harnessing the power of PROs as part of a comprehensive efficacy evaluation to ultimately deliver superior patient-centered care across the spectrum of MDS. As our understanding of MDS continues to increase, adapting the metrics of these outcome measurements will be equally important as the alteration of the natural history in developing new therapies.

Health USDo, Human Services FDACfDE, Research, Health USDo, Human Services FDACfBE, Research, et al. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79.CrossRef

Xiao C, Polomano R, Bruner DW. Comparison between patient-reported and clinician-observed symptoms in oncology. Cancer Nurs. 2013;36(6):E1–E16.PubMedCrossRef

Fromme EK, Eilers KM, Mori M, Hsieh YC, Beer TM. How accurate is clinician reporting of chemotherapy adverse effects? A comparison with patient-reported symptoms from the Quality-of-Life Questionnaire C30. J Clin Oncol. 2004;22(17):3485–90.PubMedCrossRef

Basch E, Jia X, Heller G, Barz A, Sit L, Fruscione M, et al. Adverse symptom event reporting by patients vs clinicians: relationships with clinical outcomes. J Natl Cancer Inst. 2009;101(23):1624–32.PubMedPubMedCentralCrossRef

Atkinson TM, Li Y, Coffey CW, Sit L, Shaw M, Lavene D, et al. Reliability of adverse symptom event reporting by clinicians. Qual Life Res. 2012;21(7):1159–64.PubMedCrossRef

Baratelli, C., Turco, C.G.C., Lacidogna, G. et al. The role of patient-reported outcomes in outpatients receiving active anti-cancer treatment: impact on patients’ quality of life. Support Care Cancer27, 4697–4704 (2019). https://doi.org/10.1007/s00520-019-04777-2.

Velikova G, Booth L, Smith AB, Brown PM, Lynch P, Brown JM, et al. Measuring quality of life in routine oncology practice improves communication and patient well-being: a randomized controlled trial. J Clin Oncol. 2004;22(4):714–24.PubMedCrossRef

Basch E, Deal AM, Kris MG, Scher HI, Hudis CA, Sabbatini P, et al. Symptom monitoring with patient-reported outcomes during routine Cancer treatment: a randomized controlled trial. J Clin Oncol. 2016;34(6):557–65.PubMedCrossRef

Kotronoulas G, Kearney N, Maguire R, Harrow A, Di Domenico D, Croy S, et al. What is the value of the routine use of patient-reported outcome measures toward improvement of patient outcomes, processes of care, and health service outcomes in cancer care? A systematic review of controlled trials. J Clin Oncol. 2014;32(14):1480–501.PubMedCrossRef

10.

Montazeri A. Quality of life data as prognostic indicators of survival in cancer patients: an overview of the literature from 1982 to 2008. Health Qual Life Outcomes. 2009;7:102.PubMedPubMedCentralCrossRef

11.

Basch E, Deal AM, Dueck AC, Scher HI, Kris MG, Hudis C, et al. Overall survival results of a trial assessing patient-reported outcomes for symptom monitoring during routine cancer treatment. JAMA. 2017;318(2):197–8.PubMedPubMedCentralCrossRef

12.

Basch E, Geoghegan C, Coons SJ, Gnanasakthy A, Slagle AF, Papadopoulos EJ, et al. Patient-reported outcomes in cancer drug development and US regulatory review: perspectives from industry, the Food and Drug Administration, and the patient. JAMA Oncol. 2015;1(3):375–9.PubMedCrossRef

13.

Mesa RA, Gotlib J, Gupta V, Catalano JV, Deininger MW, Shields AL, et al. Effect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trial. J Clin Oncol. 2013;31(10):1285–92.PubMedPubMedCentralCrossRef

14.

Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366(9):799–807.PubMedPubMedCentralCrossRef

15.

Rock EP, Kennedy DL, Furness MH, Pierce WF, Pazdur R, Burke LB. Patient-reported outcomes supporting anticancer product approvals. J Clin Oncol. 2007;25(32):5094–9.PubMedCrossRef

16.

Calvert M, Blazeby J, Altman DG, Revicki DA, Moher D, Brundage MD, et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA. 2013;309(8):814–22.PubMedCrossRef

17.

Steensma DP, Bennett JM. The myelodysplastic syndromes: diagnosis and treatment. Mayo Clin Proc. 2006;81(1):104–30.PubMedCrossRef

18.

Hofmann WK, Koeffler HP. Myelodysplastic syndrome. Annu Rev Med. 2005;56:1–16.PubMedCrossRef

19.

Sekeres MA. Epidemiology, natural history, and practice patterns of patients with myelodysplastic syndromes in 2010. J Natl Compr Cancer Netw. 2011;9(1):57–63.CrossRef

20.

Aul C, Gattermann N, Schneider W. Age-related incidence and other epidemiological aspects of myelodysplastic syndromes. Br J Haematol. 1992;82(2):358–67.PubMedCrossRef

21.

Ma X, Does M, Raza A, Mayne ST. Myelodysplastic syndromes: incidence and survival in the United States. Cancer. 2007;109(8):1536–42.PubMedCrossRef

22.

Foran JM, Shammo JM. Clinical presentation, diagnosis, and prognosis of myelodysplastic syndromes. Am J Med. 2012;125(7 Suppl):S6–13.PubMedCrossRef

23.

Steensma DP, Heptinstall KV, Johnson VM, Novotny PJ, Sloan JA, Camoriano JK, et al. Common troublesome symptoms and their impact on quality of life in patients with myelodysplastic syndromes (MDS): results of a large internet-based survey. Leuk Res. 2008;32(5):691–8.PubMedCrossRef

24.

Nguyen PL. The myelodysplastic syndromes. Hematol Oncol Clin North Am. 2009;23(4):675–91.PubMedCrossRef

25.

Cleeland CS, Williams LA. Symptom burden in hematologic malignancies. Blood. 2014;123(24):3686–7.PubMedCrossRef

26.

Thomas ML. Quality of life and psychosocial adjustment in patients with myelodysplastic syndromes. Leuk Res. 1998;22(Suppl 1):S41–7.PubMedCrossRef

27.

Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89(6):2079–88.PubMedCrossRef

28.

Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Sole F, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120(12):2454–65.PubMedPubMedCentralCrossRef

29.

Sekeres MA, Gerds AT. Established and novel agents for myelodysplastic syndromes. Hematology Am Soc Hematol Educ Program. 2014;2014(1):82–9.PubMedCrossRef

30.

Martino R, Valcarcel D, Brunet S, Sureda A, Sierra J. Comparable non-relapse mortality and survival after HLA-identical sibling blood stem cell transplantation with reduced or conventional-intensity preparative regimens for high-risk myelodysplasia or acute myeloid leukemia in first remission. Bone Marrow Transplant. 2008;41(1):33–8.PubMedCrossRef

31.

Chang C, Storer BE, Scott BL, Bryant EM, Shulman HM, Flowers ME, et al. Hematopoietic cell transplantation in patients with myelodysplastic syndrome or acute myeloid leukemia arising from myelodysplastic syndrome: similar outcomes in patients with de novo disease and disease following prior therapy or antecedent hematologic disorders. Blood. 2007;110(4):1379–87.PubMedPubMedCentralCrossRef

32.

Laport GG, Sandmaier BM, Storer BE, Scott BL, Stuart MJ, Lange T, et al. Reduced-intensity conditioning followed by allogeneic hematopoietic cell transplantation for adult patients with myelodysplastic syndrome and myeloproliferative disorders. Biol Blood Marrow Transplant. 2008;14(2):246–55.PubMedPubMedCentralCrossRef

33.

Deeg HJ. Transplant strategies for patients with myelodysplastic syndromes. Curr Opin Hematol. 2006;13(2):61–6.PubMedCrossRef

34.

Deeg HJ. Optimization of transplant regimens for patients with myelodysplastic syndrome (MDS). Hematology Am Soc Hematol Educ Program. 2005:167–73.

35.

El-Jawahri A, Kim HT, Steensma DP, Cronin AM, Stone RM, Watts CD, et al. Does quality of life impact the decision to pursue stem cell transplantation for elderly patients with advanced MDS? Bone Marrow Transplant. 2016;51(8):1121–6.PubMedCrossRef

36.

Sekeres MA, Steensma DP. Rethinking clinical trial endpoints in myelodysplastic syndromes. Leukemia. 2019;33(3):570–5.PubMedCrossRef

37.

Cannella L, Caocci G, Jacobs M, Vignetti M, Mandelli F, Efficace F. Health-related quality of life and symptom assessment in randomized controlled trials of patients with leukemia and myelodysplastic syndromes: what have we learned? Crit Rev Oncol Hematol. 2015;96(3):542–54.PubMedCrossRef

38.

Abel GA, Efficace F, Buckstein RJ, Tinsley S, Jurcic JG, Martins Y, et al. Prospective international validation of the Quality of Life in Myelodysplasia Scale (QUALMS). Haematologica. 2016;101(6):781–8.PubMedPubMedCentralCrossRef

39.

A basket study of novel therapy for untreated MDS/MPN and relapsed/refractory overlap syndromes (ABNL-MARRO). https://ClinicalTrials.gov/show/NCT04061421.

40.

Interventional validation of an MDS-specific measure of quality of life: assessing the responsiveness of the Quality of Life in Myelodysplasia Scale (QUALMS-1) to different hypomethylating agent regimens for low and intermediate risk disease. https://ClinicalTrials.gov/show/NCT02378701.

41.

Phase II Decitabine (DAC) versus azacitidine (AZA) in myelodysplastic syndrome (MDS). https://ClinicalTrials.gov/show/NCT02269280.

42.

Cottone F, Efficace F, Sommer K, Caocci G, Fazi P, Vignetti M, et al. Validation of the EORTC QLQ-C30 Summary Score in patients with hematological malignancies. 2019;37(15_suppl):e18551-e.

43.

Cella D. The Functional Assessment of Cancer Therapy-Anemia (FACT-An) Scale: a new tool for the assessment of outcomes in cancer anemia and fatigue. Semin Hematol. 1997;34(3 Suppl 2):13–9.PubMed

44.

Platzbecker U, Hofbauer LC, Ehninger G, Holig K. The clinical, quality of life, and economic consequences of chronic anemia and transfusion support in patients with myelodysplastic syndromes. Leuk Res. 2012;36(5):525–36.PubMedCrossRef

45.

Gale RP, Barosi G, Barbui T, Cervantes F, Dohner K, Dupriez B, et al. What are RBC-transfusion-dependence and -independence? Leuk Res. 2011;35(1):8–11.PubMedCrossRef

46.

Malcovati L, Germing U, Kuendgen A, Della Porta MG, Pascutto C, Invernizzi R, et al. Time-dependent prognostic scoring system for predicting survival and leukemic evolution in myelodysplastic syndromes. J Clin Oncol. 2007;25(23):3503–10.PubMedCrossRef

47.

Jansen AJ, Essink-Bot ML, Beckers EA, Hop WC, Schipperus MR, Van Rhenen DJ. Quality of life measurement in patients with transfusion-dependent myelodysplastic syndromes. Br J Haematol. 2003;121(2):270–4.PubMedCrossRef

48.

Lucioni C, Finelli C, Mazzi S, Oliva EN. Costs and quality of life in patients with myelodysplastic syndromes. Am J Blood Res. 2013;3(3):246–59.PubMedPubMedCentral

49.

Park S, Greenberg P, Yucel A, Farmer C, O'Neill F, De Oliveira BC, et al. Clinical effectiveness and safety of erythropoietin-stimulating agents for the treatment of low- and intermediate-1-risk myelodysplastic syndrome: a systematic literature review. Br J Haematol. 2019;184(2):134–60.PubMedCrossRef

50.

Kantarjian H, Giles F, List A, Lyons R, Sekeres MA, Pierce S, et al. The incidence and impact of thrombocytopenia in myelodysplastic syndromes. Cancer. 2007;109(9):1705–14.PubMedCrossRef

51.

Prica A, Sholzberg M, Buckstein R. Safety and efficacy of thrombopoietin-receptor agonists in myelodysplastic syndromes: a systematic review and meta-analysis of randomized controlled trials. Br J Haematol. 2014;167(5):626–38.PubMedCrossRef

52.

Oliva EN, Alati C, Santini V, Poloni A, Molteni A, Niscola P, et al. Eltrombopag versus placebo for low-risk myelodysplastic syndromes with thrombocytopenia (EQoL-MDS): phase 1 results of a single-blind, randomised, controlled, phase 2 superiority trial. Lancet Haematol. 2017;4(3):e127–e36.PubMedCrossRef

53.

Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, et al. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002;20(10):2429–40.PubMedCrossRef

54.

Kantarjian H, Issa JP, Rosenfeld CS, Bennett JM, Albitar M, DiPersio J, et al. Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer. 2006;106(8):1794–803.PubMedCrossRef

55.

Fenaux P, Giagounidis A, Selleslag D, Beyne-Rauzy O, Mufti G, Mittelman M, et al. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with low-/intermediate-1-risk myelodysplastic syndromes with del5q. Blood. 2011;118(14):3765–76.PubMedCrossRef

56.

Santini V, Almeida A, Giagounidis A, Gropper S, Jonasova A, Vey N, et al. Randomized phase III study of lenalidomide versus placebo in RBC transfusion-dependent patients with lower-risk non-del(5q) myelodysplastic syndromes and ineligible for or refractory to erythropoiesis-stimulating agents. J Clin Oncol. 2016;34(25):2988–96.PubMedCrossRef

57.

Santini V, Almeida A, Giagounidis A, Platzbecker U, Buckstein R, Beach CL, et al. The effect of lenalidomide on health-related quality of life in patients with lower-risk non-del(5q) myelodysplastic syndromes: results from the MDS-005 study. Clin Lymphoma Myeloma Leuk. 2018;18(2):136–44 e7.PubMedCrossRef

Titel: Patient-Reported Outcomes in Myelodysplastic Syndromes: the Move from Life Span to Health Span
verfasst von: Shimoli V. Barot
Bhumika J. Patel
Aaron T. Gerds
Publikationsdatum: 11.02.2020
Verlag: Springer US
Erschienen in: Current Hematologic Malignancy Reports / Ausgabe 2/2020
Print ISSN: 1558-8211
Elektronische ISSN: 1558-822X
DOI: https://doi.org/10.1007/s11899-020-00562-9

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23.04.2024 | Medikamente in Schwangerschaft und Stillzeit | Nachrichten

Live-Webinar: Aktuelle Leitlinien bei Herz-Kreislauf-Erkrankungen

Springer Medizin

Patient-Reported Outcomes in Myelodysplastic Syndromes: the Move from Life Span to Health Span

Abstract

Purpose of Review

Recent Findings

Summary

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Live-Webinar: Aktuelle Leitlinien bei Herz-Kreislauf-Erkrankungen

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Abstract

Purpose of Review

Recent Findings

Summary

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