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Successful lymphoablative autologous haematopoietic stem cell transplantation in three cases of severe autoimmune disease despite reduced dose cyclophosphamide conditioning—do we need 200 mg/kg cyclophosphamide?

Bone Marrow Transplantation volume 57, pages 1207–1209 (2022)Cite this article

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To the Editor:

Since the late 1990s, autologous haematopoietic stem cell transplantation (AHSCT) has become an important part of the treatment landscape for select severe autoimmune diseases. In the case of systemic sclerosis (SSc), efficacy has been established through three randomised studies and is considered by some as a treatment standard in early severe disease [1,2,3,4]. The most common lymphoablative AHSCT regimen in SSc and some other autoimmune diseases utilises a cyclophosphamide (CYC) conditioning backbone at a dose of 200 mg/kg along with anti-thymocyte globulin (ATG). Despite the widespread adoption of this protocol (including another upcoming randomised trial in SSc) [5], the optimal dose of CYC conditioning in AHSCT for SSc or SLE has never been formally established; furthermore, dose-dependent, life-threatening toxicity from high-dose CYC remains a significant issue [6]. Accordingly, development of safer AHSCT protocols for autoimmune disease is a key area of unmet need. Herein, we report the successful outcomes of three patients undergoing AHSCT for autoimmune disease who for clinical reasons received lower than the planned 200 mg/kg doses of CYC conditioning. All patients were enrolled in an ongoing prospective ethics-approved trial (HREC 10/206) of AHSCT in severe autoimmune diseases at our centre.

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Fig. 1: Clinical parameters for the three patients before and after reduced dose cyclophosphamide autologous transplantation.

Data availability

All data reported in this study are included in this paper.

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Authors and Affiliations

  1. Rheumatology Department, St Vincent’s Hospital, Sydney, NSW, Australia

    Ross Penglase

  2. St Vincent’s Centre for Applied Medical Research, Sydney, NSW, Australia

    Ross Penglase, David Ma & John Moore

  3. University of New South Wales, Medical School, Sydney, NSW, Australia

    Ross Penglase, David Ma & John Moore

  4. Haematology Department, St Vincent’s Hospital, Sydney, NSW, Australia

    Helen Englert, David Ma, Sam Milliken & John Moore

  5. Canberra Rheumatology, Canberra, ACT, Australia

    Kathleen Tymms

  6. Rheumatology Department, Canberra Hospital, Canberra, ACT, Australia

    Kathleen Tymms

  7. Immunology Department, St Vincent’s Hospital, Sydney, NSW, Australia

    Andrew Carr

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Contributions

RP gathered the data and wrote the paper with input from HE, KT, AC, DM, SM and JM. JM conceived the idea for this paper.

Corresponding author

Correspondence to Ross Penglase.

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The authors declare no competing interests.

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Penglase, R., Englert, H., Tymms, K. et al. Successful lymphoablative autologous haematopoietic stem cell transplantation in three cases of severe autoimmune disease despite reduced dose cyclophosphamide conditioning—do we need 200 mg/kg cyclophosphamide?. Bone Marrow Transplant 57, 1207–1209 (2022). https://doi.org/10.1038/s41409-022-01696-5

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