All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional.

  TRANSLATE

The gvhd Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the gvhd Hub cannot guarantee the accuracy of translated content. The gvhd and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.

The GvHD Hub is an independent medical education platform, sponsored by Medac and supported through grants from Sanofi and Therakos. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.

Now you can support HCPs in making informed decisions for their patients

Your contribution helps us continuously deliver expertly curated content to HCPs worldwide. You will also have the opportunity to make a content suggestion for consideration and receive updates on the impact contributions are making to our content.

Find out more

GvHD prophylaxis with tocilizumab in combination with CSA and MMF in cord blood transplantation: A phase II trial

By Sheetal Bhurke

Share:

Jul 8, 2025

Learning objective: After reading this article, learners will be able to cite a new clinical development in graft-versus-host disease.


 

Double-unit cord blood transplantation (dCBT) is the SoC for patients with high-risk hematologic malignancies and has shown improved PFS rates. However, dCBT has also been associated with higher rates of aGvHDTocilizumab (Toci), an interleukin-6 receptor blocker, has demonstrated efficacy in reducing aGvHD in early clinical studies.

Politikos et al. published results of a phase II trial assessing the efficacy and safety of tocilizumab in combination with CSA and MMF in the prevention of aGvHD in patients with acute leukemia post-dCBT in Blood Advances.1

The Toci cohort (N = 45) included patients diagnosed with AML (n = 21), ALL (n = 12), MPAL (n = 3), MDS (n = 4), CML (n = 2), or NHL (n = 3) who were undergoing dCBT. The no Toci cohort comprised of historic controls who received dCBT (N = 39). The primary endpoint was the incidence of Grade 2–4 aGvHD at Day 100.

 

Key learnings2

The cumulative incidence of Grade 2–4 aGvHD (71% vs 82%; p = 0.11) and Grade 3–4 aGvHD (11% vs 23%; p = 0.13) were comparable between the Toci vs no Toci cohorts, respectively.

The Toci cohort showed a reduced cumulative incidence of pre-engraftment syndrome (38% vs 72%; p < 0.001) and Grade 1–4 lower GI aGvHD (16% vs 33%; p = 0.056) than the no Toci cohort.

While the Toci cohort showed a trend towards faster platelet recovery (HR, 1.55; 95% CI: 0.98–2.46; p = 0.06) compared with the no Toci cohort, it was associated with delayed neutrophil engraftment (HR, 0.6; 95% CI: 0.38–0.95; p = 0.028) and no survival benefit.

The incidence of GvHD was not considerably reduced by the Toci-based regimen. Further investigation is needed to identify alternative approaches to aGvHD prophylaxis post-dCBT.

Abbreviations: aGvHD, acute graft-versus-host disease; ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; CI, confidence interval; CML, chronic myeloid leukemia; CSA, cyclosporin A; dCBT, double-unit cord blood transplantation; GI, gastrointestinal; GvHD, graft-versus-host disease; HR, hazard ratio; MDS, myelodysplastic syndrome; MMF, mycophenolate mofetil; MPAL, mixed-phenotype acute leukemia; NHL, non-Hodgkin lymphoma; OS, overall survival; PFS, progression-free survival; SoC, standard of care; Toci, tocilizumab; TRM, treatment-related mortality. 

References

Please indicate your level of agreement with the following statements:

The content was clear and easy to understand

The content addressed the learning objectives

The content was relevant to my practice

I will change my clinical practice as a result of this content