Assessment of Real-World Patient-Reported Outcomes in Patients Initiating Biologic Agents for the Treatment of Autoimmune Diseases: An Observational Study in Four Patient-Powered Research Networks

Abstract

Background

The most reliable and meaningful approach for inclusion of patient-reported outcomes (PROs) in the evaluation of real-world clinical effectiveness of biologics in the treatment of autoimmune diseases is u ncertain. This study aimed to assess and compare the proportions of patients who had abnormalities in PROs measuring important general health domains at the initiation of treatment with biologics, as well as the effects of baseline abnormalities on subsequent improvement.

Methods

PROs were collected for patient participants with inflammatory arthritis, inflammatory bowel disease, and vasculitis using Patient-Reported Outcomes Measurement Information System instruments. Scores were reported as T-scores normalized to the general population in the United States. Baseline PROs scores were collected near the time of biologic initiation, and follow-up scores were collected 3 to 8 months later. In addition to summary statistics, the proportion of patients with PROs abnormalities (scores ≥5 units worse than the population norm) was determined. Baseline and follow-up scores were compared, and an improvement of ≥5 units was considered significant.

Results

There was wide variation across autoimmune diseases in baseline PROs scores for all domains. For example, the proportion of participants with abnormal baseline pain interference scores ranged from 52% to 93%. When restricted to participants with baseline PROs abnormalities, the proportion of participants experiencing an improvement of ≥5 units was substantially higher.

Conclusion

As expected, many patients experienced improvement in PROs following initiation of treatment with biologics for autoimmune diseases. Nevertheless, a substantial proportion of participants did not exhibit abnormalities in all PROs domains at baseline, and these participants appear less likely to experience improvement. For PROs to be reliably and meaningfully included in the evaluation of real-world medication effectiveness, more knowledge and careful consideration are needed to select the most appropriate patient populations and subgroups for inclusion and evaluation in studies measuring change in PROs.

Keywords:

• patient-reported outcome measures
• patient outcome assessment
• biological therapy
• autoimmune diseases

Acknowledgments

This study was supported by a PCORI Demonstration Grant – PPRND-1507-32163. The ArthritisPower PPRN registry infrastructure is supported in part by NIAMS P30AR072583. The authors thank all of the patient participants in IBD-Partners, ArthritisPower, Vasculitis PPRN, and PARTNERS/CARRA. The authors thank the CARRA Registry site principal investigators, sub-investigators and research coordinators: N. Abel, K. Abulaban, A. Adams, M. Adams, R. Agbayani, J. Aiello, S. Akoghlanian, C. Alejandro, E. Allenspach, R. Alperin, M. Alpizar, G. Amarilyo, W. Ambler, E. Anderson, S. Ardoin, S. Armendariz, E. Baker, I. Balboni, S. Balevic, L. Ballenger, S. Ballinger, N. Balmuri, F. Barbar-Smiley, L. Barillas-Arias, M. Basiaga, K. Baszis, M. Becker, H. Bell-Brunson, E. Beltz, H. Benham, S. Benseler, W. Bernal, T. Beukelman, T. Bigley, B. Binstadt, C. Black, M. Blakley, J. Bohnsack, J. Boland, A. Boneparth, S. Bowman, C. Bracaglia, E. Brooks, M. Brothers, A. Brown, H. Brunner, M. Buckley, M. Buckley, H. 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Franco, M. Freeman, K. Fritz, S. Froese, R. Fuhlbrigge, J. Fuller, N. George, K. Gerhold, D. Gerstbacher, M. Gilbert, M. Gillispie-Taylor, E. Giverc, C. Godiwala, I. Goh, H. Goheer, D. Goldsmith, E. Gotschlich, A. Gotte, B. Gottlieb, C. Gracia, T. Graham, S. Grevich, T. Griffin, J. Griswold, A. Grom, M. Guevara, P. Guittar, M. Guzman, M. Hager, T. Hahn, O. Halyabar, E. Hammelev, M. Hance, A. Hanson, L. Harel, S. Haro, J. Harris, O. Harry, E. Hartigan, J. Hausmann, A. Hay, K. Hayward, J. Heiart, K. Hekl, L. Henderson, M. Henrickson, A. Hersh, K. Hickey, P. Hill, S. Hillyer, L. Hiraki, M. Hiskey, P. Hobday, C. Hoffart, M. Holland, M. Hollander, S. Hong, M. Horwitz, J. Hsu, A. Huber, J. Huggins, J. Hui-Yuen, C. Hung, J. Huntington, A. Huttenlocher, M. Ibarra, L. Imundo, C. Inman, A. Insalaco, A. Jackson, S. Jackson, K. James, G. Janow, J. Jaquith, S. Jared, N. Johnson, J. Jones, J. Jones, J. Jones, K. Jones, S. Jones, S. Joshi, L. Jung, C. Justice, A. Justiniano, N. Karan, K. Kaufman, A. 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Rosolowski, H. Rothermel, D. Rothman, E. Roth-Wojcicki, K. Rouster – Stevens, T. Rubinstein, N. Ruth, N. Saad, S. Sabbagh, E. Sacco, R. Sadun, C. Sandborg, A. Sanni, L. Santiago, A. Sarkissian, S. Savani, L. Scalzi, L. Schanberg, S. Scharnhorst, K. Schikler, A. Schlefman, H. Schmeling, K. Schmidt, E. Schmitt, R. Schneider, K. Schollaert-Fitch, G. Schulert, T. Seay, C. Seper, J. Shalen, R. Sheets, A. Shelly, S. Shenoi, K. Shergill, J. Shirley, M. Shishov, C. Shivers, E. Silverman, N. Singer, V. Sivaraman, J. Sletten, A. Smith, C. Smith, J. Smith, J. Smith, E. Smitherman, J. Soep, M. Son, S. Spence, L. Spiegel, J. Spitznagle, R. Sran, H. Srinivasalu, H. Stapp, K. Steigerwald, Y. Sterba Rakovchik, S. Stern, A. Stevens, B. Stevens, R. Stevenson, K. Stewart, C. Stingl, J. Stokes, M. Stoll, E. Stringer, S. Sule, J. Sumner, R. Sundel, M. Sutter, R. Syed, G. Syverson, A. Szymanski, S. Taber, R. Tal, A. Tambralli, A. Taneja, T. Tanner, S. Tapani, G. Tarshish, S. Tarvin, L. Tate, A. Taxter, J. 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Disclosure

Dr Timothy Beukelman reports grants from Patient-Centered Outcomes Research Institute, during the conduct of the study; personal fees from UCB, outside the submitted work; Dr Millie D Long reports personal fees from AbbVie, personal fees from Janssen, grants, personal fees from Takeda, grants, personal fees from Pfizer, personal fees from Target Pharmasolutions, personal fees from Prometheus, personal fees from Genentech, personal fees from Roche, grants, personal fees from Lilly, during the conduct of the study; personal fees from Takeda, personal fees from Janssen, personal fees from Pfizer, personal fees from AbbVie, outside the submitted work; Dr Michael D Kappelman reports other from other, during the conduct of the study; Dr Peter A Merkel reports grants, personal fees from AbbVie, grants, personal fees from AstraZeneca, grants, personal fees from Boehringer-Ingelheim, grants, personal fees from Bristol-Myers Squibb, grants, personal fees from ChemoCentryx, grants, personal fees from GlaxoSmithKline, grants, personal fees from InflaRx, grants, personal fees from Neutrolis, grants, personal fees from Takeda, personal fees from CSL Behring, personal fees from Dynacure, personal fees from Immagene, personal fees from Jubilant, personal fees from Magenta, personal fees from MiroBio, personal fees from Mitsubishi, personal fees from NSPharma, personal fees from Otsuka, personal fees from Q32, personal fees from Regeneron, personal fees from Sparrow, grants from Eicos, grants from Electra, grants from Genentech/Roche, grants from Sanofi, personal fees, Equity options from Kyverna, Royalties from UpToDate, outside the submitted work; Dr William Benjamin Nowell reports grants from PCORI, during the conduct of the study; grants from Amgen, grants from Eli Lilly, grants from Pfizer, grants from UCB, grants from Janssen, grants from AbbVie, grants from BMS, outside the submitted work; Dr Sarah Ringold reports grants from PCORI, during the conduct of the study; Employment starting 8/9/2021 from Janssen, Salary support through 7/2021 from CARRA, Royalties from UpToDate, outside the submitted work; Dr Jeffrey R Curtis reports grants from PCORI, during the conduct of the study. The authors report no other conflicts of interest in this work.