Skip to main content

Advertisement

SpringerLink
Log in

Patient-Reported Outcomes in Myelodysplastic Syndromes and MDS/MPN Overlap Syndromes: Stepping Onto the Stage with Changing Times

  • Myelodysplastic Syndromes (M Savona, Section Editor)
  • Published:
Current Hematologic Malignancy Reports Aims and scope Submit manuscript

Abstract

Quality of life (QOL) and symptom burden are important measures captured by patient-reported outcomes (PROs). Myelodysplastic and myelodysplastic/myeloproliferative (MDS/MPN) neoplasm overlap syndromes are notable for significant morbidity and mortality, including a wide spectrum of physical and psychosocial effects. Thus, the development and application of PROs can provide meaningful information to facilitate communication and assist in follow up care. Disease-specific measures can more accurately reflect the full breadth of functional restrictions and symptoms. While traditional endpoints include remission, relapse, and survival rates, adoption of PROs in myeloid neoplasm clinical trials can facilitate drug approval. Integration of PROs in myeloid neoplasms is an important measure to capture QOL and symptoms, which can improve disease management.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Similar content being viewed by others

References

  1. Prince. (1984). Purple Rain Recorded by Prince and the Revolution. On Purple Rain vinyl. Minneapolis, Minnesota: Warner Bros.

  2. Health USDo, Human Services FDACfDE, 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for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance.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.

    Article  Google Scholar 

  3. Lee SJ, Williams LA. Patient-reported outcomes for acute graft-versus-host disease prevention and treatment trials. Biol Blood Marrow Transplant. 2010;16:295–300.

    Article  PubMed  Google Scholar 

  4. Dylan B. (1963). The Times They Are A-Changin. Recorded by B. Dylan. On The Times They Are A-Changin vinyl. New York, New York: Columbia.

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

    Article  PubMed  Google Scholar 

  6. Rothrock NE, Kaiser KA, Cella D. Developing a valid patient-reported outcome measure. Clin Pharmacol Ther. 2011;90:737–42.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  7. 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.

    Article  PubMed  PubMed Central  Google Scholar 

  8. Temel JS, Greer JA, Muzikansky A, et al. Early palliative care for patients with metastatic non-small-cell lung cancer. N Engl J Med. 2010;363:733–42.

    Article  CAS  PubMed  Google Scholar 

  9. 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. 2003;6:522–31.

    Article  PubMed  Google Scholar 

  10. Mesa RA, Niblack J, Wadleigh M, et al. The burden of fatigue and quality of life in myeloproliferative disorders (MPDs): an international internet-based survey of 1179 MPD patients. Cancer. 2007;109:68–76.

    Article  PubMed  Google Scholar 

  11. Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med. 2010;363:1117–27.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  12. Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366:799–807.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  13. Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med. 2012;366:787–98.

    Article  CAS  PubMed  Google Scholar 

  14. Velikova G, Booth L, Smith AB, et al. Measuring quality of life in routine oncology practice improves communication and patient well-being: a randomized controlled trial. J Clin Oncol. 2004;22:714–24.

    Article  PubMed  Google Scholar 

  15. Taylor DH Jr, Bull J, Zhong X, et al. The effect of palliative care on patient functioning. J Palliat Med. 2013;16:1227–31.

    Article  PubMed  PubMed Central  Google Scholar 

  16. Fayers P, Bottomley A, Group EQoL, Quality of Life U. Quality of life research within the EORTC-the EORTC QLQ-C30. European Organisation for Research and Treatment of Cancer. Eur J Cancer. 2002;38(Suppl 4):S125–33.

    Article  PubMed  Google Scholar 

  17. 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. 1993;85:365–76.

    Article  CAS  PubMed  Google Scholar 

  18. Webster K, Cella D, Yost K. The functional assessment of chronic illness therapy (FACIT) measurement system: properties, applications, and interpretation. Health Qual Life Outcomes. 2003;1:79.

    Article  PubMed  PubMed Central  Google Scholar 

  19. McQuellon RP, Russell GB, Cella DF, et al. Quality of life measurement in bone marrow transplantation: development of the functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) scale. Bone Marrow Transplant. 1997;19:357–68.

    Article  CAS  PubMed  Google Scholar 

  20. Lee SJ, Cook EF, Soiffer R, Antin JH. Development and validation of a scale to measure symptoms of chronic graft-versus-host disease. Biology of Blood and Marrow Transplantation. 2002;8:444–52.

    Article  PubMed  Google Scholar 

  21. Baker F, Zabora J, Jodrey D, et al. Quality of life and social support of patients being evaluated for bone marrow transplantation. J Clin Psychol Med Settings. 1995;2:357–72.

    Article  CAS  PubMed  Google Scholar 

  22. Cella D, Riley W, Stone A, et al. The patient-reported outcomes measurement information system (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005-2008. J Clin Epidemiol. 2010;63:1179–94.

    Article  PubMed  PubMed Central  Google Scholar 

  23. Dueck AC, Mendoza TR, Mitchell SA, et al. Validity and reliability of the US National Cancer Institute's patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE). JAMA oncology. 2015;1:1051–9.

    Article  PubMed  PubMed Central  Google Scholar 

  24. Ware JE, Kosinski M, Dewey JE, Gandek B. SF-36 health survey: manual and interpretation guide. Lincoln: Quality Metric Inc.; 2000.

    Google Scholar 

  25. Podsiadlo D, Richardson S. The timed "up & go": a test of basic functional mobility for frail elderly persons. J Am Geriatr Soc. 1991;39:142–8.

    Article  CAS  PubMed  Google Scholar 

  26. Holsinger T, Deveau J, Boustani M, Williams JW Jr. Does this patient have dementia? JAMA. 2007;297:2391–404.

    Article  PubMed  Google Scholar 

  27. Lawton MP, Brody EM. Assessment of older people: self-maintaining and instrumental activities of daily living. Gerontologist. 1969;9:179–86.

    Article  CAS  PubMed  Google Scholar 

  28. Rumpf HJ, Meyer C, Hapke U, John U. Screening for mental health: validity of the MHI-5 using DSM-IV Axis I psychiatric disorders as gold standard. Psychiatry Res. 2001;105:243–53.

    Article  CAS  PubMed  Google Scholar 

  29. Sherbourne CD, Stewart AL. The MOS social support survey. Soc Sci Med. 1991;32:705–14.

    Article  CAS  PubMed  Google Scholar 

  30. Fillenbaum GG, Smyer MA. The development, validity, and reliability of the OARS multidimensional functional assessment questionnaire. J Gerontol. 1981;36:428–34.

    Article  CAS  PubMed  Google Scholar 

  31. Ballen KK, Shrestha S, Sobocinski KA, et al. Outcome of transplantation for myelofibrosis. Biol Blood Marrow Transplant. 2010;16:358–67.

    Article  PubMed  Google Scholar 

  32. Efficace F, Gaidano G, Breccia M, et al. Prevalence, severity and correlates of fatigue in newly diagnosed patients with myelodysplastic syndromes. Br J Haematol. 2015;168:361–70.

    Article  PubMed  Google Scholar 

  33. 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. 2004;104:321–7.

    Article  CAS  PubMed  Google Scholar 

  34. Efficace F, Santini V, La Nasa G, et al. Health-related quality of life in transfusion-dependent patients with myelodysplastic syndromes: a prospective study to assess the impact of iron chelation therapy. BMJ Support Palliat Care. 2016;6:80–8.

    Article  PubMed  Google Scholar 

  35. Lubbert M, Suciu S, Baila L, et al. Low-dose decitabine versus best supportive care in elderly patients with intermediate- or high-risk myelodysplastic syndrome (MDS) ineligible for intensive chemotherapy: final results of the randomized phase III study of the European Organisation for Research and Treatment of Cancer leukemia group and the German MDS study group. J Clin Oncol. 2011;29:1987–96.

    Article  PubMed  Google Scholar 

  36. Deschler B, Ihorst G, Platzbecker U, et al. Parameters detected by geriatric and quality of life assessment in 195 older patients with myelodysplastic syndromes and acute myeloid leukemia are highly predictive for outcome. Haematologica. 2013;98:208–16.

    Article  PubMed  PubMed Central  Google Scholar 

  37. Abel GA, Efficace F, Buckstein RJ, et al. Prospective international validation of the Quality of Life in Myelodysplasia Scale (QUALMS). Haematologica. 2016;101:781–8.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  38. Abel GA, Klaassen R, Lee SJ, et al. Patient-reported outcomes for the myelodysplastic syndromes: a new MDS-specific measure of quality of life. Blood. 2014;123:451–2.

    Article  CAS  PubMed  Google Scholar 

  39. Garcia-Manero G. (2017). 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. In. Clinical Trials.gov; 2016. https://www.clinicaltrials.gov/ct2/show/NCT02378701

  40. Buckstein R, Wells RA, Zhu N, et al. Patient-related factors independently impact overall survival in patients with myelodysplastic syndromes: an MDS-CAN prospective study. Br J Haematol. 2016;174:88–101.

    Article  PubMed  Google Scholar 

  41. Scherber R, Dueck AC, Johansson P, et al. The myeloproliferative neoplasm symptom assessment form (MPN-SAF): international prospective validation and reliability trial in 402 patients. Blood. 2011;118:401–8.

    Article  CAS  PubMed  Google Scholar 

  42. Bevans M, El-Jawahri A, Tierney DK, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the patient-centered outcomes working group report. Biol Blood Marrow Transplant. 2017;23:538–51.

    Article  PubMed  Google Scholar 

  43. Savona MR, Malcovati L, Komrokji R, et al. An international consortium proposal of uniform response criteria for myelodysplastic/myeloproliferative neoplasms (MDS/MPN) in adults. Blood. 2015;125:1857–65.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  44. Murphy K. (Producer), Reiner, R. (Director). (1984). This is Spinal Tap Motion Picture. United States: Embasy Pictures.

Download references

Author information

Authors and Affiliations

  1. Leukemia and Myeloid Disorders Program, Department of Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, 9500 Euclid Ave, CA-60, Cleveland, OH, 44120, USA

    Sagar S. Patel & Aaron T. Gerds

Authors
  1. Sagar S. Patel
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Aaron T. Gerds
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Aaron T. Gerds.

Ethics declarations

Conflict of Interest

Sagar S. Patel declares no potential conflicts of interest. Aaron T. Gerds reports personal fees from Incyte, CTI Biopharma, Astra-Zenica, and Roche outside the submitted work.

Human and Animal Rights and Informed Consent

This article contains no studies with human or animal subjects performed by any of the authors.

Additional information

This article is part of the Topical Collection on Myelodysplastic Syndromes

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Patel, S.S., Gerds, A.T. Patient-Reported Outcomes in Myelodysplastic Syndromes and MDS/MPN Overlap Syndromes: Stepping Onto the Stage with Changing Times. Curr Hematol Malig Rep 12, 455–460 (2017). https://doi.org/10.1007/s11899-017-0406-x

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s11899-017-0406-x

Keywords

Search

Navigation