Health-related quality of life and symptom assessment in patients with myelodysplastic syndromes

Expert Review of Hematology

Volumn 2, Issue 1, 2009, Pages 69-80

  Caocci, Giovanni ^a   La Nasa, Giorgio ^b   Efficace, Fabio ^c  

^a Binaghi Hospital   (Italy)

^b UNIVERSITY OF CAGLIARI   (Italy)

^c GIMEMA Data Center   (Italy)

Author keywords

Myelodysplasia; Patient reported outcome; Quality of life; Systematic review

Indexed keywords

5 AZA 2' DEOXYCYTIDINE; ALPHA TOCOPHEROL; AZACITIDINE; CYCLOSPORIN A; GRANULOCYTE COLONY STIMULATING FACTOR; LENALIDOMIDE; NOVEL ERYTHROPOIESIS STIMULATING PROTEIN; RECOMBINANT ERYTHROPOIETIN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; RETINOIC ACID; THALIDOMIDE; THYMOCYTE ANTIBODY;

ANEMIA; ANXIETY; BLOOD TRANSFUSION; CLINICAL FEATURE; DEPRESSION; FATIGUE; HUMAN; INFECTION; METHODOLOGY; MYELODYSPLASTIC SYNDROME; OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUALITY OF LIFE; QUESTIONNAIRE; RATING SCALE; REVIEW; SCORING SYSTEM; SELF REPORT; THROMBOCYTE TRANSFUSION; TREATMENT DURATION; TREATMENT OUTCOME;

HUMANS; MYELODYSPLASTIC SYNDROMES; PATIENT-CENTERED CARE; QUALITY OF LIFE; TREATMENT OUTCOME;

EID: 77953425222     PISSN: 17474086     EISSN: None     Source Type: Journal    
DOI: 10.1586/17474086.2.1.69     Document Type: Review

References (75)

1. Nimer SD. Myelodysplastic syndromes: Blood 111, 4841-4851 (2008).
2. Bennett JM, Catovsky D, Daniel MT et al. Proposals for the classification of the myelodysplastic syndromes. Br. J. Haematol. 51, 189-199 (1982).
3. Bennett JM. World Health Organization classification of the acute leukemias and myelodysplastic syndrome. Int. J. Hematol. 72, 131-133 (2000).
4. Greenberg P, Cox C, LeBeau MM et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 89, 2079-2088 (1997).
5. Bernasconi C. Evidence-based approach to treatment of myelodysplastic syndromes. Haematologica 86, 897-899 (2001).
6. Nosslinger T, Reisner R, Koller E et al. Myelodysplastic syndromes, from French-American-British to World Health Organization: comparison of classifications on 431 unselected patients from a single institution. Blood 98, 2935-2941 (2001).
7. Cutler CS, Lee SJ, Greenberg P et al. A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood 104, 579-585 (2004).
8. Malcovati L, Germing U, Kuendgen A et al. Time-dependent prognostic scoring system for predicting survival and leukemic evolution in myelodysplastic syndromes. J. Clin. Oncol. 25, 3503-3510 (2007).
9. Alessandrino EP, Amadori S, Barosi G et al. Italian Society of Hematology. Evidence- and consensus-based practice guidelines for the therapy of primary myelodysplastic syndromes. A statement from the Italian Society of Hematology. Haematologica 87, 1286-1306 (2002).
10. Greenberg PL, Baer MR, Bennett JM et al. Myelodysplastic syndromes clinical practice guidelines in oncology. J. Natl Compr. Cancer Netw. 4, 58-77 (2006).
11. Valent P, Horny HP, Bennett JM et al. Definitions and standards in the diagnosis and treatment of the myelodysplastic syndromes: consensus statements and report from a working conference. Leuk. Res. 31, 727-736 (2007).
12. Cheson BD, Bennett JM, Kantarjian H et al. Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood 96, 3671-3674 (2000).
13. Cheson BD, Greenberg PL, Bennett JM et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood 108, 419-425 (2006).
14. Stewart BW, Kleihues P. World Health Organization. World Cancer Report. IARC Press, Lyon, France (2003).
15. Johnson JR, Temple R. Food and Drug Administration requirements for approval of new anticancer drugs. Cancer Treat. Rep. 69, 1155-1159 (1985).
16. ASCO. Outcomes of cancer treatment for technology assessment and cancer treatment guidelines. American Society of Clinical Oncology. J. Clin. Oncol. 14, 671-679 (1996).
17. Parmet S, Lynm C, Glass RM. JAMA patient page. Quality of life. JAMA 288, 3070 (2002).
18. Kong SX, Ghandi SK. Methodologic assessments of quality of life measures in clinical trials. Ann. Pharmacother. 31, 830-836 (1997).
19. Velikova G, Stark D, Selby P. Quality of life instruments in oncology. Eur. J. Cancer. 35, 1571-1580 (1999).
20. Schumacher M, Olschewski M, Schulgen G. Assessment of quality of life in clinical trials. Stat. Med. 10, 1915-1930 (1991).
21. Acquadro C, Berzon R, Dubois D et al. Incorporating the patient's perspective into drug development and communication: an ad hoc task force report of the Patient- Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration. February 16, 2001. Value Health 6, 522-531 (2003).
22. Dubois D, Dhawan R, Van de Velde H et al. Descriptive and prognostic value of patient-reported outcomes: the bortezomib experience in relapsed and refractory multiple myeloma. J. Clin. Oncol. 24(6), 976-982 (2006).
23. Fayers P, Machin D. Quality of Life. Assessment, Analysis and Interpretation. John Wiley & Sons, Chichester, UK (2000).
24. Lipscomb J, Gotay CC, Snyder CF. Patient-reported outcomes in cancer: a review of recent research and policy initiatives. CA Cancer J. Clin. 57(5), 278-300 (2007).
25. Thomas ML. Health-related quality of life for those with myelodysplastic syndrome: conceptualization, measurement and implications for research and practice. In: Myelodysplastic Syndromes. Clinical and Biological AdVances. Greenberg PL (Ed.). Cambridge University Press, Cambridge, UK 263-295 (2006).
26. Thomas ML. Quality of life and psychosocial adjustment in patients with myelodysplastic syndromes. Leuk. Res. 22(Suppl. 1), 41-47 (1998).
27. Hellström-Lindberg E, Gulbrandsen N, Lindberg G et al. A validate decision model for treating the anemia of myelodysplastic syndromes with erythropoietin + granulocyte colonystimulating factor: significant effects on quality of life. Br. J. Haematol. 29, 1217-1219 (2003).
28. Servaes P, Verhagen C, Bleijenberg G. Fatigue in cancer patients during and after treatment. Prevalence, correlates and interventions. Eur. J. Cancer 38, 27-43 (2002).
29. Casadevall N, Durieux P, Dubois S et al. Health, economic, and quality of life effects of erythropoietin and granulocyte colony-stimulating factor for the treatment of myelodysplastic syndromes: a randomized, controlled trial. Blood 104, 321-327 (2004).
30. Jansen AJG, Essink-Bot ML, Beckers EAM, Hop WCJ, Schippeurus MR, Van Rhenen DJ. Quality of life measurements in patients with transfusion-dependent myelodysplastic syndromes. Br. J. Haematol. 121, 270-274 (2003).
31. Thomas ML, Zhang J, Greenberg PL. Quality of life in individuals with myelodysplastic syndromes (MDS): a descriptive study. Blood (Suppl. 1), 662a (1999).
32. Osoba D, Tannock IF, Ernst DS, Neville AJ. Health-related quality of life in men with metastatic prostate cancer treated with prednisone alone or mitoxantrone and prednisone. J. Clin. Oncol. 17, 1654-1663 (1999). (Pubitemid 29269233)
33. Moinpour CM. Measuring quality of life: an emerging science. Semin. Oncol. 21(Suppl. 10), 48-60 (1994).
34. Feld R. Endpoints in cancer clinical trials: is there a need for measuring quality of life? Support Care Cancer 3, 23-27 (1995).
35. Efficace F, Osoba D, Gotay C, Sprangers M, Coens C, Bottomley A. Has the quality of health-related quality of life reporting in cancer clinical trials improved over time? Towards bridging the gap with clinical decision making. Ann. Oncol. 18, 775-781 (2007).
36. Efficace F, Kemmler G, Vignetti M, Mandelli F, Molica S, Holzner B. Health-related quality of life assessment and reported outcomes in leukaemia randomised controlled trials - a systematic review to evaluate the added value in supporting clinical decision making. Eur. J. Cancer 44, 1497-1506 (2008).
37. Efficace F, Novik A, Vignetti M, Mandelli F, Cleeland CS. Health-related quality of life and symptom assessment in clinical research of patients with haematological malignancies: where are we now and where do we go from here? Haematologica 92, 1596-1598 (2007).
38. Stasi R, Abruzzese E, Lanzetta G, Terzoli E, Amadori S. Darbepoetin -for the treatment of anemic patients with low and intermediate-1-risk myelodysplastic syndromes. Ann. Oncol. 16, 1921-1927 (2005).
39. Coates A, Dillenbeck CF, McNeil DR et al. On the receiving end - II. Linear analogue self-assessment (LASA) in evaluation of aspects of the quality of life cancer patients receiving therapy. Eur. J. Cancer Clin. Oncol. 19, 1633-1637 (1983).
40. Giagounidis AAN, Haase S, Germing H et al. Treatment of myelodysplastic syndrome with isolated del(5q) including bands q31-q33 with a combination of all-trans-retinoic acid and tocopherol-α: a Phase II study. Ann. Hematol. 84, 389-394 (2005).
41. Aloe Spiriti MA, Latagliata R, Niscola P et al. Impact of a new dosing regimen of epoietin ? on quality of life and anemia in patients with low-risk myelodysplastic syndrome. Ann. Hematol. 84, 167-176 (2005).
42. Clavio M, Nobili F, Balleari E et al. Quality of life and brain function following high-dose recombinant human erythropoietin in low-risk myelodysplastic syndromes: a preliminary report. Eur. J. Haematol. 72, 113-120 (2004).
43. Moher D, Jones A, Lepage L. CONSORT Group. Use of the CONSORT statement and quality of reports of randomized trials: a comparative before-and-after evaluation. JAMA 285, 1992-1995 (2001).
44. Efficace F, Bottomley A, Osoba D et al. Beyond the development of health-related quality of life (HRQOL) measures. A checklist for evaluating HRQOL outcomes in cancer clinical trials-does HRQOL evaluation in prostate cancer research inform clinical decision-making? J. Clin. Oncol. 21, 3502-3511 (2003).
45. Kantarjian H, Issa JP, Rosenfeld CS et al. Decitabine improves patient outcomes in myelodysplastic syndromes. Results of a Phase III randomized study. Cancer 106, 1794 - 1803 (2006).
46. Balleari E, Rossi E, Clavio M et al. Erytropoietin plus granulocyte colonystimulating factor is better than erythropoietin alone to treat anemia in lowrisk myelodysplastic syndromes: result from a randomized single-centre study. Ann. Hematol. 85, 174-180 (2006).
47. Kornblith AB, Herndon JE, Silverman LR et al. Impact of azacitidine on the quality of life of patients with myelodysplastic syndrome treated in a randomized Phase III trial: a cancer and leukemia group B study. J. Clin. Oncol. 10, 2441-2452 (2002).
48. Veit CT, Ware JE. The structure of psychological distress and well-being in general population. J. Consult. Clin. Psychol. 51, 730-742 (1983).
49. Bottomley A, Therasse P. Quality of life in patients undergoing systemic therapy for adVanced breast cancer. Lancet Oncol. 3, 620-628 (2002). (Pubitemid 35174680)
50. Efficace F, Bottomley A. Health-related quality of life assessment methodology and reported outcomes in randomised controlled trials of primary brain cancer patients. Eur. J. Cancer 38, 1824-1831 (2002).
51. Efficace F, Bottomley A, Vanvoorden V, Blazeby JM. Methodological issues in assessing health-related quality of life of colorectal cancer patients in randomized controlled trials. Eur. J. Cancer 40, 187-197 (2004).
52. Bottomley A, Efficace F, Thomas R, Vanvoorden V, Ahmedzai S. Health-related quality of life in non small-cell lung cancer: methodologic issues in randomized controlled trials. J. Clin. Oncol. 21, 2982-2992 (2003).
53. Efficace F, Bottomley A, Van Andel G. Health-related quality of life in prostate carcinoma patients: a systematic review of randomized controlled trials. Cancer 97, 377-388 (2003). (Pubitemid 36133805)
54. Molica S. Quality of life in chronic lymphocytic leukemia: a neglected issue. Leuk. Lymphoma 46, 1709-1714 (2005).
55. Taphoorn MJ, Stupp R, Coens C et al. Health-related quality of life in patients with glioblastoma: a randomised controlled trial. Lancet Oncol. 6, 937-944 (2005).
56. Van Meerbeeck JP, Gaafar R, Manegold C et al. Randomized Phase III study of cisplatin with or without raltitrexed in patients with malignant pleural mesothelioma: an intergroup study of the European Organisation for Research and Treatment of Cancer Lung Cancer Group and the National Cancer Institute of Canada. J. Clin. Oncol. 23, 6881-6889 (2005).
57. Lee SJ, Joffe S, Haesook TK. Physicians' attitudes about quality-of-life issue in hematopoietic stem cell transplantation. Blood 194, 2194-12120 (2004).
58. King MT, Fayers PM. Making quality-oflife results more meaningful for clinicians. Lancet 371, 709-710 (2008).
59. Schünemann HJ, Akl EA, Guyatt GH. Interpreting the results of patient reported outcome measures in clinical trials: the clinician's perspective. Health Qual. Life Outcomes 4, 62 (2006).
60. Osoba D, Bezjak A, Brundage M; for the Quality of Life Committee of the NCIC CTG. Analysis and interpretation of health-related quality of life data from clinical trials: basic approach of the National Cancer Institute of Canada Clinical Trials Group. Eur. J. Cancer 41, 280-287 (2005).
61. Steensma DP, Heptinstall KV, Johnson VM 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. 32, 691-698 (2008).
62. Heptinstall K. Quality of life in myelodysplastic syndrome: a special report from the Myelodysplastic Syndromes Foundation, Inc. Oncology 22(Suppl. 2), 13-18 (2008).
63. Fayers PM, Hopwood P, Harvey A et al. Quality of life assessment in clinical trials - guidelines and a checklist for protocol writers: the U.K. Medical Research Council experience. MRC Cancer Trials Office. Eur. J. Cancer 33, 20-28 (1997).
64. Chassany O, Sagnier P, Marquis P et al. Patient-reported outcomes: the example of health related quality of life-a European guidance document for the improved integration of health related quality of life assessment in the drug regulatory process. Drug Inf. J. 36, 209-238 (2002).
65. Gotay CC, Korn EL, McCabe MS et al. Quality-of-life assessment in cancer treatment protocols: research issues in protocol development. J. Natl Cancer Inst. 84, 575-579 (1992).
66. Aaronson NK, Ahmedzai S, Bergman B et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J. Natl Cancer Inst. 85, 365-376 (1993).
67. Cella DF, Tulsky DS, Gray G et al. The Functional Assessment of Cancer Therapy scale: development and validation of the general measure. J. Clin. Oncol. 11, 570-579 (1993).
68. Yellen, SB, Cella D, Webster K, Blendowski C, Kaplan E. Measuring fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) measurement system. J. Pain Symptom Manage. 13, 63-74 (1997).
69. Smets EMA, Garssen B, Bonke B, de Haes JCJM. Multidimensional Fatigue Inventory (MFI): psychometric qualities of an instrument to assess fatigue. J. Psychosom. Res. 39, 315-325 (1995).
70. Mendoza TR, Wang XS, Cleeland CS et al. The rapid assessment of fatigue severity in cancer patients: use of the brief fatigue inventory. Cancer 85, 1186-1196 (1999).
71. Hann DM, Jacobsen PB, Azzarello LM et al. Measurement of fatigue in cancer patients: development and validation of the Fatigue Symptom Inventory. Qual. Life Res. 7, 301-310 (1998).
72. Weis J, Gao Y, Conroy T et al. Development of an EORTC module for the assessment of cancer related fatigue (EORTC FAR 15): Phase III results. Qual. Life Res. A-108 (2007) (Abstract 1624).
73. Cleeland CS, Mendoza TR, Wang XS et al. Assessing symptom distress in cancer patients: the M.D. Anderson Symptom Inventory: Cancer 89, 1634-1646 (2000).
74. Zigmond AS, Snaith RP. The Hospital Anxiety and Depression Scale: Acta Psychiatr. Scand. 67, 361-370 (1983).
75. Silverman LR, Demakos EP, Peterson BL 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. 20, 2429-2440 (2002).