Abstract
Objectives Autoimmunity affects the C/PNS in a range of heterogenous diseases. Whilst most patients respond to standard treatments, responses may be inadequate leading to significant and potentially permanent disability. Autologous haematopoietic stem cell transplantation (AHSCT) has been reported as a means of intensive immunomodulation. Here, we evaluate the safety and efficacy of AHSCT in patients with non-MS neuroimmunological disease.
Methods A Phase II clinical trial of AHSCT using a cyclophosphamide + ATG chemotherapeutic regimen for patients with treatment-refractory neuroimmunological disease commenced at St Vincent’s Hospital in December 2010 (ACTRN12613000339752. Eligibility criteria for each disease are available via ANZCTR.
Results 12 patients underwent AHSCT between May 2011 and September 2021 for the following; Bechet’s disease (n=1), CIDP (n=3), SLE/CNS vasculitis (n=4), opsoclonus myoclonus ataxia (n=1), stiff-person syndrome (n=2), neurosarcoidosis (n=1). Median follow-up is 39 months (19–67 months). Treatment related mortality was 0%. Adverse effects due to AHSCT were consistent with expected toxicities. A median of 7 (range = 5–11) prior disease-modifying therapies were trialled prior to AHSCT. Ten patients remained off immunotherapy following AHSCT. Quality of life metrics improved for the majority of patients transiently post-AHSCT but only remained below baseline in 5 of 12 patients at last follow up.
Conclusions Clinical evidence to support the use of AHSCT in treatment-refractory neuroimmunological disease is scant. This study represents a summary of the experience of the largest Australian autoimmune disease transplant unit, shedding light on which conditions and patient phenotype may be more likely to benefit from AHSCT.
