Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation
Abstract Objectives Patients with refractory myasthenia gravis (MG) have few treatment options. Autologous hematopoietic stem cell transplantation (HSCT) has been used to treat immune diseases; however, its use in the treatment of MG is not broadly considered. Our objective is to report on the effic...
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Wiley
2025-01-01
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| Series: | Annals of Clinical and Translational Neurology |
| Online Access: | https://doi.org/10.1002/acn3.52246 |
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| author | Benjamin Beland Jan Storek Liam Quartermain Christopher Hahn C. Elizabeth Pringle Pierre R. Bourque Michael Kennah Natasha Kekre Christopher Bredeson David Allan Kareem Jamani Christopher White Harold Atkins |
| author_facet | Benjamin Beland Jan Storek Liam Quartermain Christopher Hahn C. Elizabeth Pringle Pierre R. Bourque Michael Kennah Natasha Kekre Christopher Bredeson David Allan Kareem Jamani Christopher White Harold Atkins |
| author_sort | Benjamin Beland |
| collection | DOAJ |
| description | Abstract Objectives Patients with refractory myasthenia gravis (MG) have few treatment options. Autologous hematopoietic stem cell transplantation (HSCT) has been used to treat immune diseases; however, its use in the treatment of MG is not broadly considered. Our objective is to report on the efficacy and safety of HSCT in refractory MG. Methods Twenty‐one patients who underwent HSCT for MG were retrospectively reviewed. All patients had severe MG refractory to multiple therapies. Stem cells were mobilized with cyclophosphamide and granulocyte colony‐stimulating factor. The grafts were depleted of immune cells by selecting CD34+ cells. HSCT conditioning consisted of high‐dose cytoreductive therapy and anti‐thymocyte globulin. The primary efficacy outcome was achieving clinically stable remission or minimal manifestations without treatment and remaining as such until most recent follow‐up. Results The median time from MG diagnosis to HSCT was 4.0 years. The primary outcome was reached in 16 of 18 evaluable patients (89%) at a median of 1.7 years and maintained with a median follow‐up of 6.7 years (range 1.0–21.9 years). Three patients were not evaluable for the primary outcome: one due to confounding illness and two died within 12 months of transplant. The transplant‐related mortality at 100 days was 9.5%. Two late deaths occurred, with uncertain relation to the HSCT. Interpretation After HSCT for refractory MG, most patients achieved sustained disease remission. However, HSCT‐related mortality in medically complex MG patients may be high. Prospective studies investigating the efficacy and safety of HSCT in the treatment of refractory MG are warranted. |
| format | Article |
| id | doaj-art-02ea485a4e674e32996f5b8e9fc23c9c |
| institution | Kabale University |
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| language | English |
| publishDate | 2025-01-01 |
| publisher | Wiley |
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| series | Annals of Clinical and Translational Neurology |
| spelling | doaj-art-02ea485a4e674e32996f5b8e9fc23c9c2025-01-21T05:41:42ZengWileyAnnals of Clinical and Translational Neurology2328-95032025-01-01121566810.1002/acn3.52246Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantationBenjamin Beland0Jan Storek1Liam Quartermain2Christopher Hahn3C. Elizabeth Pringle4Pierre R. Bourque5Michael Kennah6Natasha Kekre7Christopher Bredeson8David Allan9Kareem Jamani10Christopher White11Harold Atkins12Division of Neurology, Department of Clinical Neurosciences, Cumming School of Medicine University of Calgary Calgary Alberta CanadaDivision of Hematology, Department of Medicine, Cumming School of Medicine University of Calgary Calgary Alberta CanadaTransplant and Cell Therapy Program, Division of Hematology, Department of Medicine The Ottawa Hospital Ottawa Ontario CanadaDivision of Neurology, Department of Clinical Neurosciences, Cumming School of Medicine University of Calgary Calgary Alberta CanadaDivision of Neurology, Department of Medicine, Faculty of Medicine University of Ottawa Ottawa Ontario CanadaDivision of Neurology, Department of Medicine, Faculty of Medicine University of Ottawa Ottawa Ontario CanadaTransplant and Cell Therapy Program, Division of Hematology, Department of Medicine The Ottawa Hospital Ottawa Ontario CanadaTransplant and Cell Therapy Program, Division of Hematology, Department of Medicine The Ottawa Hospital Ottawa Ontario CanadaTransplant and Cell Therapy Program, Division of Hematology, Department of Medicine The Ottawa Hospital Ottawa Ontario CanadaTransplant and Cell Therapy Program, Division of Hematology, Department of Medicine The Ottawa Hospital Ottawa Ontario CanadaDivision of Hematology, Department of Medicine, Cumming School of Medicine University of Calgary Calgary Alberta CanadaDivision of Neurology, Department of Clinical Neurosciences, Cumming School of Medicine University of Calgary Calgary Alberta CanadaTransplant and Cell Therapy Program, Division of Hematology, Department of Medicine The Ottawa Hospital Ottawa Ontario CanadaAbstract Objectives Patients with refractory myasthenia gravis (MG) have few treatment options. Autologous hematopoietic stem cell transplantation (HSCT) has been used to treat immune diseases; however, its use in the treatment of MG is not broadly considered. Our objective is to report on the efficacy and safety of HSCT in refractory MG. Methods Twenty‐one patients who underwent HSCT for MG were retrospectively reviewed. All patients had severe MG refractory to multiple therapies. Stem cells were mobilized with cyclophosphamide and granulocyte colony‐stimulating factor. The grafts were depleted of immune cells by selecting CD34+ cells. HSCT conditioning consisted of high‐dose cytoreductive therapy and anti‐thymocyte globulin. The primary efficacy outcome was achieving clinically stable remission or minimal manifestations without treatment and remaining as such until most recent follow‐up. Results The median time from MG diagnosis to HSCT was 4.0 years. The primary outcome was reached in 16 of 18 evaluable patients (89%) at a median of 1.7 years and maintained with a median follow‐up of 6.7 years (range 1.0–21.9 years). Three patients were not evaluable for the primary outcome: one due to confounding illness and two died within 12 months of transplant. The transplant‐related mortality at 100 days was 9.5%. Two late deaths occurred, with uncertain relation to the HSCT. Interpretation After HSCT for refractory MG, most patients achieved sustained disease remission. However, HSCT‐related mortality in medically complex MG patients may be high. Prospective studies investigating the efficacy and safety of HSCT in the treatment of refractory MG are warranted.https://doi.org/10.1002/acn3.52246 |
| spellingShingle | Benjamin Beland Jan Storek Liam Quartermain Christopher Hahn C. Elizabeth Pringle Pierre R. Bourque Michael Kennah Natasha Kekre Christopher Bredeson David Allan Kareem Jamani Christopher White Harold Atkins Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation Annals of Clinical and Translational Neurology |
| title | Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation |
| title_full | Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation |
| title_fullStr | Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation |
| title_full_unstemmed | Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation |
| title_short | Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation |
| title_sort | refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation |
| url | https://doi.org/10.1002/acn3.52246 |
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