This work is licensed under a Creative Commons Attribution 4.0 International License.
Hematopoietic Stem Cell Transplant as Symptomatic Treatment for Myasthenia Gravis: A Literature Review
Corresponding Author(s) : Jessica Amadea Sutrisno
Nusantara Science and Technology Proceedings,
Multi-Conference Proceeding Series F
Abstract
Myasthenia gravis (MG) is an autoimmune neurological disorder that impairs communication at the neuromuscular junction. Over the past seven decades, its global incidence has steadily increased. Approximately 10–20% of MG patients do not respond adequately to or cannot tolerate conventional treatments. Hematopoietic stem cells (HSCs), which are multipotent cells capable of producing all blood cell types and restoring the hematopoietic system, are being explored as a therapeutic option. Hematopoietic stem cell transplantation (HSCT) is under investigation as a potential treatment for severe and refractory MG, with ongoing research aimed at enhancing its safety, and efficacy, and identifying optimal candidates for the therapy. This paper aimed to evaluate the efficacy of hematopoietic stem cell transplantation (HSCT) as a therapeutic option for managing symptomatic myasthenia gravis (MG), especially in cases unresponsive to conventional therapies. A systematic search identified five studies, comprising two cohort studies and three case reports. Most patients in these studies demonstrated promising improvement following HSCT treatment, as indicated by a reduction in MGFA scores and improved clinical manifestations during follow-up assessments. This treatment approach has demonstrated significant potential. However, the current studies are susceptive to bias, and more preclinical research along with high-quality clinical trials, large-scale are required to determine if this emerging treatment can meet the anticipated outcomes.
Keywords
Download Citation
Endnote/Zotero/Mendeley (RIS)BibTeX
- Almaeen, A., Wani, F. A., & Thirunavukkarasu, A. (2021). Knowledge and attitudes towards stem cells and the significance of their medical application among healthcare sciences students of Jouf University. PeerJ., 9, e10661. doi:10.7717/peerj.10661
- Banerjee, A., Bhattacharya, A., & Ghatak, N. (2021). Stem cells-basics and prospectives. International Journal of Engineering Applied Sciences and Technology, 6(5). doi:10.33564/ijeast.2021.v06i05.027
- Bryant, A., Atkins, H., Pringle, C. E., Allan, D., Anstee, G., Bence-Bruckler, I. et al. (2016). Myasthenia gravis treated with autologous hematopoietic stem cell transplantation. JAMA Neurology, 73(6), 652. doi:10.1001/jamaneurol.2016.0113
- Bubuioc, A. M., Kudebayeva, A., Turuspekova, S., Lisnic, V., & Leone, M. A. (2021). The epidemiology of myasthenia gravis. Journal of Medicine and Life, 14(1), 7–16. doi: 10.25122/jml-2020-0145.
- Demirci, S., Leonard, A., & Tisdale, J. F. (2020). Hematopoietic stem cells from pluripotent stem cells: Clinical potential, challenges, and future perspectives. Stem Cells Translational Medicine, 9(12), 1549–57. doi:10.1002/sctm.20-0247
- Dresser, L., Wlodarski, R., Rezania, K., & Soliven, B. (2021). Myasthenia gravis: Epidemiology, pathophysiology and clinical manifestations. Journal of Clinical Medicine, 10(11), 2235.
- Håkansson, I., Sandstedt, A., Lundin, F., Askmark, H., Pirskanen, R., Carlson, K., et al. (2017). Successful autologous haematopoietic stem cell transplantation for refractory myasthenia gravis – a case report. Neuromuscul Disord, 27(1), 90-3. doi: 10.1016/j.nmd.2016.09.020
- Lehnerer, S., Jacobi, J., Schilling, R., Grittner, U., Marbin, D., Gerischer, L. et al. (2021). Burden of disease in myasthenia gravis: Taking the patient’s perspective. Journal of Neurology, 269(6), 3050–63. doi:10.1007/s00415-021-10891-1
- Mai, V. V., Le, H. S., Nguyen, T. M., Le, T. T., Nguyen, H. Q., Hoang, T. T., et al. (2023). Autologous hematopoietic stem cell transplantation to treat myasthenia gravis: Experience of a developing country. Archives of the Balkan Medical Union, 58(2), 150–7. doi:10.31688/abmu.2023.58.2.08
- Mariottini, A., Bulgarini, G., Cornacchini, S., Damato, V., Saccardi, R., & Massacesi, L. (2023). Hematopoietic stem cell transplantation for the treatment of autoimmune neurological diseases: An update. Bioengineering, 10(2), 176. doi:10.3390/bioengineering10020176
- Melo, C. L., Peña, A. M., Salazar, L. A., Jiménez, S. I., Gómez, E. D., Chalela, C. M. et al. (2019). Autologous hematopoietic stem cell transplantation in a patient with refractory seropositive myasthenia gravis: A case report. Neuromuscul Disord., 29(2), 142-45. doi: 10.1016/j.nmd.2018.11.008.
- Schlatter, M. I., Yandamuri, S. S., O’Connor, K. C., Nowak, R. J., Pham, M. C., Obaid, A. H., et al. (2023). Remission of severe myasthenia gravis after autologous stem cell transplantation. Annals of Clinical and Translational Neurology., 10(11), 2105–13. doi:10.1002/acn3.51898
- Zakrzewski, W., Dobrzyski, M., Szymonowicz, M., & Rybak, Z. (2019). Stem cells: Past, present, and future - stem cell research & therapy [Internet]. Stem Cell Res Ther., 10, 68. https://doi.org/10.1186/s13287-019-1165-5
References
Almaeen, A., Wani, F. A., & Thirunavukkarasu, A. (2021). Knowledge and attitudes towards stem cells and the significance of their medical application among healthcare sciences students of Jouf University. PeerJ., 9, e10661. doi:10.7717/peerj.10661
Banerjee, A., Bhattacharya, A., & Ghatak, N. (2021). Stem cells-basics and prospectives. International Journal of Engineering Applied Sciences and Technology, 6(5). doi:10.33564/ijeast.2021.v06i05.027
Bryant, A., Atkins, H., Pringle, C. E., Allan, D., Anstee, G., Bence-Bruckler, I. et al. (2016). Myasthenia gravis treated with autologous hematopoietic stem cell transplantation. JAMA Neurology, 73(6), 652. doi:10.1001/jamaneurol.2016.0113
Bubuioc, A. M., Kudebayeva, A., Turuspekova, S., Lisnic, V., & Leone, M. A. (2021). The epidemiology of myasthenia gravis. Journal of Medicine and Life, 14(1), 7–16. doi: 10.25122/jml-2020-0145.
Demirci, S., Leonard, A., & Tisdale, J. F. (2020). Hematopoietic stem cells from pluripotent stem cells: Clinical potential, challenges, and future perspectives. Stem Cells Translational Medicine, 9(12), 1549–57. doi:10.1002/sctm.20-0247
Dresser, L., Wlodarski, R., Rezania, K., & Soliven, B. (2021). Myasthenia gravis: Epidemiology, pathophysiology and clinical manifestations. Journal of Clinical Medicine, 10(11), 2235.
Håkansson, I., Sandstedt, A., Lundin, F., Askmark, H., Pirskanen, R., Carlson, K., et al. (2017). Successful autologous haematopoietic stem cell transplantation for refractory myasthenia gravis – a case report. Neuromuscul Disord, 27(1), 90-3. doi: 10.1016/j.nmd.2016.09.020
Lehnerer, S., Jacobi, J., Schilling, R., Grittner, U., Marbin, D., Gerischer, L. et al. (2021). Burden of disease in myasthenia gravis: Taking the patient’s perspective. Journal of Neurology, 269(6), 3050–63. doi:10.1007/s00415-021-10891-1
Mai, V. V., Le, H. S., Nguyen, T. M., Le, T. T., Nguyen, H. Q., Hoang, T. T., et al. (2023). Autologous hematopoietic stem cell transplantation to treat myasthenia gravis: Experience of a developing country. Archives of the Balkan Medical Union, 58(2), 150–7. doi:10.31688/abmu.2023.58.2.08
Mariottini, A., Bulgarini, G., Cornacchini, S., Damato, V., Saccardi, R., & Massacesi, L. (2023). Hematopoietic stem cell transplantation for the treatment of autoimmune neurological diseases: An update. Bioengineering, 10(2), 176. doi:10.3390/bioengineering10020176
Melo, C. L., Peña, A. M., Salazar, L. A., Jiménez, S. I., Gómez, E. D., Chalela, C. M. et al. (2019). Autologous hematopoietic stem cell transplantation in a patient with refractory seropositive myasthenia gravis: A case report. Neuromuscul Disord., 29(2), 142-45. doi: 10.1016/j.nmd.2018.11.008.
Schlatter, M. I., Yandamuri, S. S., O’Connor, K. C., Nowak, R. J., Pham, M. C., Obaid, A. H., et al. (2023). Remission of severe myasthenia gravis after autologous stem cell transplantation. Annals of Clinical and Translational Neurology., 10(11), 2105–13. doi:10.1002/acn3.51898
Zakrzewski, W., Dobrzyski, M., Szymonowicz, M., & Rybak, Z. (2019). Stem cells: Past, present, and future - stem cell research & therapy [Internet]. Stem Cell Res Ther., 10, 68. https://doi.org/10.1186/s13287-019-1165-5