Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant

Aouad, Patrick; Li, Jamma; Arthur, Chris; Burt, Richard K.; Fernando, Suran L.; Parratt, John

doi:10.1016/j.jocn.2015.02.007

Cited by 25 publications

(14 citation statements)

References 11 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Other data come from two case reports and a Chinese study in 21 patients with so-called opticospinal MS [71][72][73]. A recent case report showed a sustained clinical, radiological and immunopathological NMO remission with rituximab treatment prior to aHSCT [74]. Recent data from Northwestern University support favourable clinical outcomes of aHSCT with the Cy-ATG regimen combined with rituximab, with clearance of anti-AQP-4Ab [75].…”

Section: Neuromyelitis Optica (Nmo)mentioning

confidence: 99%

Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE)

Sharrack

Saccardi

Alexander

et al. 2019

Bone Marrow Transplant

179

276

View full text Add to dashboard Cite

These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development. The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies. aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified. Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.

show abstract

Section: Neuromyelitis Optica (Nmo)mentioning

confidence: 99%

Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE)

Sharrack

Saccardi

Alexander

et al. 2019

Bone Marrow Transplant

179

276

View full text Add to dashboard Cite

show abstract

“…AHSCBMT has been studied in NMO also albeit in smaller numbers. AHSCBMT in refractory NMO/NMOSD was associated with clinical and radiological remission, improved disability and resolution of AQP-4 antibodies which were still undetectable 12 months later ( Aouad et al., 2015 , Peng et al., 2010 ). The European Group for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) reported after a median follow-up of 47 months with baseline EDSS=6.5, three of 16 cases were progression and treatment free, while in the remaining 13 patients further treatments were administered for disability progression or relapse after AHSCBMT.…”

mentioning

confidence: 89%

Review of approved NMO therapies based on mechanism of action, efficacy and long-term effects

Brod

2020

Multiple Sclerosis and Related Disorders

View full text Add to dashboard Cite

Highlights Neuromyelitis optica (NMO - including NMO spectrum disorders [NMOSD]) is a devastating disease. Up until recently, there was no proven agent to treat to prevent relapses. We now have three agents indicated for the treatment of NMO. We might suggest the following sequence – 1st line using eculizumab for rapid efficacy and stabilization without effect on the acquired immune system followed by satrilizumab (long term immunomodulation). Reserve inebilizumab (immunosuppressant) for breakthrough disease and salvage the severe with AHSCBMT. In NMO, control the complement, transition to modulation, and reserve suppression – and salvage the severe with AHSCBMT.

show abstract

“…Recently, Aouad and colleagues reported on a 47-year-old female patient that underwent autologous HSCT with a disease duration of 11 years [ 71 ]. Rituximab was administered as part of the conditioning regimen (cyclophosphamide + Anti-thymocyte Globulin, ATG).…”

Section: Autologous Hsct In Nmosdmentioning

confidence: 99%

Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica-Spectrum Disorders (NMO-SD): State-of-the-Art and Future Perspectives

Ceglie

Papetti

Valeriani

et al. 2020

IJMS

View full text Add to dashboard Cite

Neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorders (NMOSD) are a group of autoimmune inflammatory disorders of the central nervous system (CNS). Understanding of the molecular basis of these diseases in the last decades has led to an important improvement in the treatment of this disease, in particular, to the use of immunotherapeutic approaches, such as monoclonal antibodies and Hematopoietic Stem Cell Transplantation (HSCT). The aim of this review is to summarize the pathogenesis, biological basis and new treatment options of these disorders, with a particular focus on HSCT applications. Different HSCT strategies are being explored in NMOSD, both autologous and allogeneic HSCT, with the new emergence of therapeutic effects such as an induction of tolerance to auto-antigens and graft versus autoimmunity effects that can be exploited to hopefully treat a disease that still has prognosis.

show abstract

Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant

Cited by 25 publications

References 11 publications

Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE)

Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE)

Review of approved NMO therapies based on mechanism of action, efficacy and long-term effects

Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica-Spectrum Disorders (NMO-SD): State-of-the-Art and Future Perspectives

Contact Info

Product

Resources

About