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Propensity score matching analysis comparing the efficacy of Ruxolitinib to historical controls in second-line or beyond treatment for chronic GvHD after steroid failure

Bone Marrow Transplantation volume 58, pages 1024–1032 (2023)Cite this article

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Abstract

Established first-line therapy for chronic graft-versus-host disease (cGvHD) comprises corticosteroids with/without calcineurin inhibitors, but about half of cGvHD patients are refractory to corticosteroid therapy. The present study retrospectively analyzed treatment outcomes in 426 patients and undertook a propensity-score matching (PSM) analysis between ruxolitinib (RUX) treated group and a historical group of cGvHD patients treated with best available treatment (BAT). PSM process adjusted unbalanced risk factors between the 2 groups, including GvHD severity, HCT-CI score, and treatment line, extracting 88 patients (44 in BAT/RUX groups each) for final analysis. In PSM subgroup, RUX group showed 74.7% 12 months’ FFS rate vs 19.1% for BAT group (p < 0.001), whereas 12 months’ OS rates were 89.2% and 77.7%, respectively. Multivariate analysis for FFS confirmed RUX superiority over BAT together with HCT-CI score 0–2 vs ≥3. For OS, RUX was superior to BAT, while age ≥60 years and severe grade cGvHD adversely impacted OS. In PSM subgroup, at months 0, 3, and 6, 4.5%, 12.2% and 22.2% more patients in RUX group could discontinue prednisone compared to BAT group, respectively. In conclusion, the current study showed that for FFS, RUX was superior to BAT as second-line therapy or beyond in cGvHD patients after therapy failure.

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Fig. 1: Outcomes in the overall patient group.
Fig. 2: Outcomes in the propensity score matched groups.
Fig. 3: Comparison of daily dose corticosteroids between the 2 groups.
Fig. 4: Proportion of the patients on daily prednisone dose ≤0.1 mg/kg/day and those discontinued it at month 0, 3 and 6.

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Data availability

Data are available upon request from the authors at their discretion.

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Author notes

  1. These authors contributed equally: Igor Novitzky-Basso, Swe Mar Linn.

Authors and Affiliations

  1. Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

    Igor Novitzky-Basso, Swe Mar Linn, Arjun Law, Rajat Kumar, Jonas Mattsson & Dennis Dong Hwan Kim

  2. University of Toronto, Toronto, ON, Canada

    Igor Novitzky-Basso, Arjun Law, Rajat Kumar, Jonas Mattsson & Dennis Dong Hwan Kim

  3. Vancouver General Hospital, British Columbia Cancer Agency, Vancouver, BC, Canada

    Jennifer White & Samantha Culos

  4. University of British Columbia, Vancouver, BC, Canada

    Jennifer White

  5. Saskatchewan Cancer Agency, Regina, SK, Canada

    Mohamed Elemary

  6. University of Saskatchewan, Saskatoon, SK, Canada

    Mohamed Elemary

  7. London Health Science Centre, London, ON, Canada

    Anargyros Xenocostas, Uday Deotare & Kate Kelly

  8. University of Western Ontario, London, ON, Canada

    Anargyros Xenocostas & Uday Deotare

  9. St Vincent’s Hospital Sydney, Darlinghurst, NSW, Australia

    Nada Hamad & Sui Tan

  10. University of New South Wales, Sydney, NSW, Australia

    Nada Hamad

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Contributions

INB, SML, JW, ME, AX, UD, KK, NH, ST, SC, AL, RK, JM, DDHK contributed patient and treatment data for analysis. SML, DDHK collected data and undertook statistical analysis. INB, SML, DDHK reviewed the data and prepared the manuscript. INB, SML, JW, ME, AX, UD, KK, NH, ST, SC, AL, RK, JM, DDHK reviewed the analysis results and edited the manuscript.

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Correspondence to Dennis Dong Hwan Kim.

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Novitzky-Basso, I., Linn, S.M., White, J. et al. Propensity score matching analysis comparing the efficacy of Ruxolitinib to historical controls in second-line or beyond treatment for chronic GvHD after steroid failure. Bone Marrow Transplant 58, 1024–1032 (2023). https://doi.org/10.1038/s41409-023-02020-5

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