Quality of Life Research

, Volume 27, Issue 4, pp 979–985 | Cite as

Utility of a patient-reported outcome in measuring functional impairment during autologous stem cell transplant in patients with multiple myeloma

  • Nina Shah
  • Qiuling Shi
  • Sergio Giralt
  • Loretta Williams
  • Qaiser Bashir
  • Muzaffar Qazilbash
  • Richard E. Champlin
  • Charles S. Cleeland
  • Xin Shelley Wang



We aimed to determine the utility of a patient-reported outcome (PRO) as it relates to patient performed testing (PPT) for measuring functional status in multiple myeloma patients after autologous hematopoietic stem cell transplantation (auto-HCT).


Symptom interference on walking (a PRO) was measured by the MD Anderson Symptom Inventory (MDASI). PPT was assessed via 6-min walk test (6MWT). Mixed effects modeling was used to examine (1) the longitudinal relationship between the MDASI score and 6MWT distance and (2) the MDASI scores between patients who did or did not complete the 6WMT. Receiver operating characteristic (ROC) curve analysis was performed to quantify the construct validity of the PRO by differentiating performance status.


Seventy-nine patients were included. Mean 6MWT distance significantly correlated with MDASI-walking interference score (PRO) over the first month of auto-HCT (est = 6.09, p = 0.006). There was a significantly higher completion rate for MDASI versus 6MWT at each time point (p < 0.01). Patients who completed the 6MWT reported less interference on walking during the study period (est = 1.61, p < 0.0001). Finally, the PRO demonstrated significant construct validity for measuring functioning status with MDASI-walking against ECOG-PS as the anchor (AUC = 0.77, 95% CI 0.60–0.94, p = 0.003).


The PRO of MDASI-walking interference is a valid physical functioning measure, correlating with an objective functional measure (6MWT) in MM patients undergoing auto-HCT. As patients with poorer functional status during therapy are less likely to complete PPT, this PRO may offer a more practical quantitative measure of functioning in patients.


Patient-reported outcomes (PRO) Patient performed testing (PPT) MD Anderson Symptom Inventory (MDASI) Functional status Auto-HCT 



This work was supported in part by the National Cancer Institute of the National Institutes of Health through The University of Texas MD Anderson Cancer Center’s Cancer Center Support Grant, P30 CA016672 (PI: RA DePinho) and program project P01 CA124787 (PI: Charles S. Cleeland).

Compliance with ethical standards

Conflict of interest

The MD Anderson Symptom Inventory (MDASI) is copyrighted and licensed by The University of Texas MD Anderson Cancer Center and by Charles S. Cleeland. The authors declare no other conflicts of interest in this work.

Ethical Approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. This study was approved by the M.D. Anderson Institutional Review Board.

Informed consent

Informed consent was obtained from all individual participants included in the study.

Supplementary material

11136_2017_1759_MOESM1_ESM.pdf (16 kb)
Supplementary Figure 1. Example of MDASI daily symptom interference on module. Question 25 (red arrow) asked patients to rate interference with “walking” on a 0-10 scale (PDF 15 KB)
11136_2017_1759_MOESM2_ESM.tif (24.3 mb)
Supplementary Figure 2. Kaplan-Meier analysis of overall survival, comparing patients who were able to perform the 6MWT versus those who were not at 5 days after auto-HCT. There was no significant difference in median overall survival between these groups (TIF 24917 KB)


  1. 1.
    Deshpande, P. R., Rajan, S., Sudeepthi, B. L., & Abdul Nazir, C. P. (2011). Patient-reported outcomes: A new era in clinical research. Perspectives in Clinical Research, 2(4), 137–144.CrossRefPubMedPubMedCentralGoogle Scholar
  2. 2.
    Kotronoulas, G., Kearney, N., Maguire, R., Harrow, A., Di Domenico, D., Croy, S., & MacGillivray, S. (2014). 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. Journal of Clinical Oncology, 32(14), 1480–1501.CrossRefPubMedGoogle Scholar
  3. 3.
    Barney, B. J., Wang, X. S., Lu, C., Liao, Z., Johnson, V. E., Cleeland, C. S., & Mendoza, T. R. (2013). Prognostic value of patient-reported symptom interference in patients with late-stage lung cancer. Quality of Life Research, 22(8), 2143–2150.CrossRefPubMedGoogle Scholar
  4. 4.
    Penson, R. T., Huang, H. Q., Wenzel, L. B., Monk, B. J., Stockman, S., Long, H. J., 3rd, Ramondetta, L. M., Landrum, L. M., Oaknin, A., Reid, T. J., Leitao, M. M., Method, M., Michael, H., & Tewari, K. S. (2015). Bevacizumab for advanced cervical cancer: patient-reported outcomes of a randomised, phase 3 trial (NRG Oncology-Gynecologic Oncology Group protocol 240). The Lancet Oncology, 16(3), 301–311.CrossRefPubMedPubMedCentralGoogle Scholar
  5. 5.
    Mesa, R. A., Gotlib, J., Gupta, V., Catalano, J. V., Deininger, M. W., Shields, A. L., Miller, C. B., Silver, R. T., Talpaz, M., Winton, E. F., Harvey, J. H., Hare, T., Erickson-Viitanen, S., Sun, W., Sandor, V., Levy, R. S., Kantarjian, H. M., & Verstovsek, S. (2013). Effect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trial. Journal of Clinical Oncology, 31(10), 1285–1292.CrossRefPubMedPubMedCentralGoogle Scholar
  6. 6.
    Tefferi, A., Hudgens, S., Mesa, R., Gale, R. P., Verstovsek, S., Passamonti, F., Cervantes, F., Rivera, C., Tencer, T., & Khan, Z. M. (2014). Use of the Functional Assessment of Cancer Therapy—Anemia in persons with myeloproliferative neoplasm-associated myelofibrosis and anemia. Clinical Therapeutics, 36(4), 560–566.CrossRefPubMedGoogle Scholar
  7. 7.
    Wang, X. S., Shi, Q., Shah, N. D., Heijnen, C. J., Cohen, E. N., Reuben, J. M., Orlowski, R. Z., Qazilbash, M. H., Johnson, V. E., Williams, L. A., Mendoza, T. R., & Cleeland, C. S. (2014). Inflammatory markers and development of symptom burden in patients with multiple myeloma during autologous stem cell transplantation. Clinical Cancer Research, 20(5), 1366–1374.CrossRefPubMedPubMedCentralGoogle Scholar
  8. 8.
    Lee, S. J. (2004). Patient-reported outcomes in multiple myeloma. Journal of the National Comprehensive Cancer Network, 2(4), 379–383.CrossRefPubMedGoogle Scholar
  9. 9.
    King, T. A., King, M. T., & White, K. J. (2017). Patient reported outcomes in optimizing myeloma patients’ health-related quality of life. Seminars in Oncology Nursing, 33(3), 299–315.CrossRefPubMedGoogle Scholar
  10. 10.
    Wang, X. S., Shi, Q., Williams, L. A., Shah, N. D., Mendoza, T. R., Cohen, E. N., Reuben, J. M., Cleeland, C. S., & Orlowski, R. Z. (2015). Longitudinal analysis of patient-reported symptoms post-autologous stem cell transplant and their relationship to inflammation in patients with multiple myeloma. Leukemia & Lymphoma, 56(5), 1335–1341.CrossRefGoogle Scholar
  11. 11.
    Richardson, P. G., Barlogie, B., Berenson, J., Singhal, S., Jagannath, S., Irwin, D., Rajkumar, S. V., Srkalovic, G., Alsina, M., Alexanian, R., Siegel, D., Orlowski, R. Z., Kuter, D., Limentani, S. A., Lee, S., Hideshima, T., Esseltine, D. L., Kauffman, M., Adams, J., Schenkein, D. P., & Anderson, K. C. (2003). A phase 2 study of bortezomib in relapsed, refractory myeloma. New England Journal of Medicine, 348(26), 2609–2617.CrossRefPubMedGoogle Scholar
  12. 12.
    Cleeland, C. S., Mendoza, T. R., Wang, X. S., Chou, C., Harle, M. T., Morrissey, M., & Engstrom, M. C. (2000). Assessing symptom distress in cancer patients: the M.D. Anderson Symptom Inventory. Cancer, 89(7), 1634–1646.CrossRefPubMedGoogle Scholar
  13. 13.
    Shi, Q., Wang, X. S., Vaporciyan, A. A., Rice, D. C., Popat, K. U., & Cleeland, C. S. (2016). Patient-reported symptom interference as a measure of postsurgery functional recovery in lung cancer. Journal of Pain and Symptom Management, 52, 822–831CrossRefPubMedPubMedCentralGoogle Scholar
  14. 14.
    Jones, D., Zhao, F., Fisch, M. J., Wagner, L. I., Patrick-Miller, L. J., Cleeland, C. S., & Mendoza, T. R. (2014). The validity and utility of the MD Anderson Symptom Inventory in patients with prostate cancer: evidence from the Symptom Outcomes and Practice Patterns (SOAPP) data from the Eastern Cooperative Oncology Group. Clinical Genitourinary Cancer, 12(1), 41–49.CrossRefPubMedGoogle Scholar
  15. 15.
    Jones, D., Vichaya, E. G., Wang, X. S., Williams, L. A., Shah, N. D., Thomas, S. K., Johnson, V. E., Champlin, R. E., Cleeland, C. S., & Mendoza, T. R. (2013). Validation of the M. D. Anderson Symptom Inventory multiple myeloma module. Journal of Hematology & Oncology, 6, 13.CrossRefGoogle Scholar
  16. 16.
    Overcash, J. (2015). Assessing the functional status of older cancer patients in an ambulatory care visit. Healthcare, 3(3), 846–859.CrossRefPubMedPubMedCentralGoogle Scholar
  17. 17.
    Miyamoto, S., Nagaya, N., Satoh, T., Kyotani, S., Sakamaki, F., Fujita, M., Nakanishi, N., & Miyatake, K. (2000). Clinical correlates and prognostic significance of six-minute walk test in patients with primary pulmonary hypertension. Comparison with cardiopulmonary exercise testing. American Journal of Respiratory and Critical Care Medicine, 161(2 Pt 1), 487–492.CrossRefPubMedGoogle Scholar
  18. 18.
    Ko, V., Naylor, J. M., Harris, I. A., Crosbie, J., & Yeo, A. E. (2013). The six-minute walk test is an excellent predictor of functional ambulation after total knee arthroplasty. BMC Musculoskeletal Disorders, 14, 145.CrossRefPubMedPubMedCentralGoogle Scholar
  19. 19.
    Jones, L. W., Devlin, S. M., Maloy, M. A., Wood, W. A., Tuohy, S., Espiritu, N., Aquino, J., Kendig, T., Michalski, M. G., Gyurkocza, B., Schaffer, W. L., Ali, B., Giralt, S., & Jakubowski, A. A. (2015). Prognostic importance of pretransplant functional capacity after allogeneic hematopoietic cell transplantation. The Oncologist, 20(11), 1290–1297.CrossRefPubMedPubMedCentralGoogle Scholar
  20. 20.
    Shah, N., Shi, Q., Williams, L. A., Mendoza, T. R., Wang, X. S., Reuben, J. M., Dougherty, P. M., Bashir, Q., Qazilbash, M. H., Champlin, R. E., Cleeland, C. S., & Giralt, S. A. (2016). Higher stem cell dose infusion after intensive chemotherapy does not improve symptom burden in older patients with multiple myeloma and amyloidosis. Biology of Blood and Marrow Transplantation, 22(2), 226–231.CrossRefPubMedGoogle Scholar
  21. 21.
    Castel, L. D., Abernethy, A. P., Li, Y., Depuy, V., Saville, B. R., & Hartmann, K. E. (2007). Hazards for pain severity and pain interference with daily living, with exploration of brief pain inventory cutpoints, among women with metastatic breast cancer. Journal of Pain and Symptom Management, 34(4), 380–392.CrossRefPubMedGoogle Scholar
  22. 22.
    Oken, M. M., Creech, R. H., Tormey, D. C., Horton, J., Davis, T. E., McFadden, E. T., & Carbone, P. P. (1982). Toxicity and response criteria of the Eastern Cooperative Oncology Group. American Journal of Clinical Oncology, 5(6), 649–655.CrossRefPubMedGoogle Scholar
  23. 23.
    Terwee, C. B., Bot, S. D., de Boer, M. R., van der Windt, D. A., Knol, D. L., Dekker, J., Bouter, L. M., & de Vet, H. C. (2007). Quality criteria were proposed for measurement properties of health status questionnaires. Journal of Clinical Epidemiology, 60(1), 34–42.CrossRefPubMedGoogle Scholar
  24. 24.
    Shi, Q., Mendoza, T. R., Wang, X. S., & Cleeland, C. S. (2016). Using a symptom-specific instrument to measure patient-reported daily functioning in patients with cancer. European Journal of Cancer, 67, 83–90.CrossRefPubMedGoogle Scholar
  25. 25.
    Wood, W. A., Le-Rademacher, J., Syrjala, K. L., Jim, H., Jacobsen, P. B., Knight, J. M., Abidi, M. H., Wingard, J. R., Majhail, N. S., Geller, N. L., Rizzo, J. D., Fei, M., Wu, J., Horowitz, M. M., & Lee, S. J. (2016). Patient-reported physical functioning predicts the success of hematopoietic cell transplantation (BMT CTN 0902). Cancer, 122(1), 91–98.CrossRefPubMedGoogle Scholar
  26. 26.
    Sorror, M. L., Maris, M. B., Storb, R., Baron, F., Sandmaier, B. M., Maloney, D. G., & Storer, B. (2005). Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood, 106(8), 2912–2919.CrossRefPubMedPubMedCentralGoogle Scholar
  27. 27.
    Jones, L. W., Hornsby, W. E., Goetzinger, A., Forbes, L. M., Sherrard, E. L., Quist, M., Lane, A. T., West, M., Eves, N. D., Gradison, M., Coan, A., Herndon, J. E., & Abernethy, A. P. (2012). Prognostic significance of functional capacity and exercise behavior in patients with metastatic non-small cell lung cancer. Lung Cancer, 76(2), 248–252.CrossRefPubMedGoogle Scholar
  28. 28.
    Baumann, F. T., Kraut, L., Schule, K., Bloch, W., & Fauser, A. A. (2010). A controlled randomized study examining the effects of exercise therapy on patients undergoing haematopoietic stem cell transplantation. Bone Marrow Transplantation, 45(2), 355–362.CrossRefPubMedGoogle Scholar
  29. 29.
    Dimeo, F., Fetscher, S., Lange, W., Mertelsmann, R., & Keul, J. (1997). Effects of aerobic exercise on the physical performance and incidence of treatment-related complications after high-dose chemotherapy. Blood, 90(9), 3390–3394.PubMedGoogle Scholar
  30. 30.
    Izano, M., Satariano, W. A., Hiatt, R. A., & Braithwaite, D. (2013). The impact of functional limitations on long-term outcomes among African-American and white women with breast cancer: a cohort study. British Medical Journal Open, 3(10), e003232.Google Scholar

Copyright information

© Springer International Publishing AG, part of Springer Nature 2017

Authors and Affiliations

  • Nina Shah
    • 1
  • Qiuling Shi
    • 2
  • Sergio Giralt
    • 3
  • Loretta Williams
    • 2
  • Qaiser Bashir
    • 4
  • Muzaffar Qazilbash
    • 4
  • Richard E. Champlin
    • 4
  • Charles S. Cleeland
    • 2
  • Xin Shelley Wang
    • 2
  1. 1.Department of MedicineUniversity of California San FranciscoSan FranciscoUSA
  2. 2.Department of Symptom ResearchThe University of Texas MD Anderson Cancer CenterHoustonUSA
  3. 3.Department of MedicineMemorial Sloan-Kettering Cancer CenterNew YorkUSA
  4. 4.Department of Stem Cell TransplantationThe University of Texas MD Anderson Cancer CenterHoustonUSA

Personalised recommendations