An aggressive form of MOGAD treated with aHSCT: A case report
Elvira Sbragia1,2, Giacomo Boffa1, Riccardo Varaldo3
1Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Genoa, Italy.
Background:
Although myelin-oligodendrocyte-glycoprotein (MOG)-antibody-associated disease (MOGAD) has been considered a more favorable demyelinating central nervous system disorder, recent data evidence that some patients might experience severe relapses and high disability. Actual treatment-options are acquired mostly from anti-aquaporin-4-antibody-positive neuromyelitis optica spectrum disorder and rely on clinical experience. Therefore, treatment of aggressive forms of MOGAD can be challenging.
Objectives And Methods:
To describe a patient with an aggressive MOGAD treated with autologous hematopoietic stem cell transplantation (aHSCT).
Results:
A 56-year-old man was diagnosed with MOGAD in 2017 because of right optic-neuritis and anti-MOG-antibody positivity. In the following 2 years, he experienced two optic neuritis with good recovery after high-dose steroid. At the end of 2019, he presented sensory and motor impairment at lower limbs with evidence of several spinal, longitudinally extended, tumefactive inflammatory lesions. Despite sequential treatment with rituximab and tocilizumab alongside high-dose steroid, intravenous immunoglobulins and plasma-exchange, he experienced several clinical relapses and exhibited persistent magnetic resonance activity. He was finally addressed to intense immunosuppression with myeloablative conditioning regimen followed by autologous hematopoietic stem cell transplantation (aHSCT). After 2 years follow-up, he is free from disease-activity.
Conclusions:
In a patient affected by aggressive, treatment-refractory MOGAD, aHSCT resulted as safe and was able to suppress disease-activity for over 2 years.
Insights
Autologous hematopoietic stem cell transplantation (aHSCT) offers a safe and effective treatment for aggressive myelin-oligodendrocyte-glycoprotein antibody-associated disease (MOGAD), suppressing disease activity for over two years in a refractory case.
Area of Science:
- Neuroimmunology
- Demyelinating diseases of the central nervous system
- Autoimmune disorders
Background:
- Myelin-oligodendrocyte-glycoprotein antibody-associated disease (MOGAD) can present with severe relapses and disability, challenging treatment approaches.
- Current treatments for MOGAD are often adapted from neuromyelitis optica spectrum disorder (NMOSD) and rely on clinical experience.
- Aggressive forms of MOGAD necessitate the exploration of novel and effective therapeutic strategies.
Observation:
- A 56-year-old male diagnosed with MOGAD experienced recurrent optic neuritis followed by severe spinal cord inflammation with tumefactive lesions.
- The patient exhibited persistent disease activity and relapses despite multiple treatments including high-dose steroids, rituximab, tocilizumab, IVIg, and plasma exchange.
- Conventional therapies failed to control the aggressive, treatment-refractory MOGAD.
Findings:
- Autologous hematopoietic stem cell transplantation (aHSCT) was performed following a myeloablative conditioning regimen.
- The patient achieved sustained disease remission with no evidence of disease activity for over two years post-aHSCT.
- aHSCT demonstrated a favorable safety profile in this patient with aggressive MOGAD.
Implications:
- Autologous hematopoietic stem cell transplantation (aHSCT) represents a viable therapeutic option for severe, treatment-refractory MOGAD.
- This case highlights the potential of aHSCT to induce long-term remission in aggressive demyelinating disorders.
- Further research is warranted to establish the role of aHSCT in the management of MOGAD and other refractory autoimmune neurological conditions.
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