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Early cessation of a randomised study in acute graft versus host disease: upfront mesenchymal stromal cells with corticosteroids versus corticosteroids alone

Bone Marrow Transplantation volume 55, pages 2199–2201 (2020)Cite this article

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Graft versus host disease (GVHD) remains a critical cause of treatment failure after allogeneic haemopoietic progenitor cell transplantation (HPCT). Mesenchymal stromal cells (MSC) are of interest as therapy for GVHD due to putative immunomodulatory and immuno-privileged properties. An early phase II study investigating MSC for steroid-refractory aGVHD showed promising short-term responses and favourable survival for responders [1]. Our centre subsequently developed an MSC product and observed satisfactory feasibility and safety for steroid-refractory acute and chronic GVHD [2]. We hypothesised that MSC would be more effective if infused at diagnosis of aGVHD rather than for steroid-refractory disease. Hence we designed a prospective, randomised study in aGVHD to investigate the efficacy of MSC infusion in addition to standard corticosteroid therapy.

Patients with grades II–IV acute GVHD within 180 days of allogeneic HPCT with calcineurin inhibitor-based GVHD prophylaxis were eligible. Patients were excluded if they had already received >72 h of systemic corticosteroids at any dose for GVHD. Participants randomised to the control arm received 2 mg/kg IV methylprednisolone or 2.5 mg oral prednisolone daily for a minimum of 5 days. Participants randomised to the study (MSC) arm received the same corticosteroid therapy, in addition to 2 × 106 MSC/kg infused intravenously on day 1 and day 8. Randomisation was conducted by a central study co-ordinator using an electronic random sequence generator.

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Acknowledgements

The Medical Research Foundation of Royal Perth Hospital and National Collaborative Research Infrastructure Strategy (NCRIS) provided funding. The MSC were manufactured by CTTWA under license from Isopogen Pty Ltd.

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Authors and Affiliations

  1. Department of Haematology, Fiona Stanley Hospital, Perth, WA, Australia

    Duncan Purtill, Julian Cooney, Matthew Wright & Paul Cannell

  2. Department of Haematology, Royal Perth Hospital, Perth, WA, Australia

    Melita Cirillo & Richard Herrmann

  3. School of Pathology and Laboratory Medicine, Faculty of Health and Medical Science, University of Western Australia, Perth, WA, Australia

    Melita Cirillo

  4. Cell and Tissue Therapies WA, Royal Perth Hospital, Perth, WA, Australia

    Janice Fogarty, Dino Tan & Marian Sturm

  5. Regenerative Biology, Faculty of Health and Medical Science, University of Western Australia, Perth, WA, Australia

    Marian Sturm

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  1. Duncan Purtill
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  2. Melita Cirillo
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  8. Richard Herrmann
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Correspondence to Duncan Purtill.

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MS has a personal financial interest in and is a director of Isopogen Pty Ltd.

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Purtill, D., Cirillo, M., Fogarty, J. et al. Early cessation of a randomised study in acute graft versus host disease: upfront mesenchymal stromal cells with corticosteroids versus corticosteroids alone. Bone Marrow Transplant 55, 2199–2201 (2020). https://doi.org/10.1038/s41409-020-0955-9

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