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Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
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The hematopoietic stem cells or HSCs are multipotent, meaning they can differentiate and give rise to all blood and immune cells. HSCs are maintained in the quiescent stage until an external stimulus initiates their differentiation. The multipotent HSCs exist as two heterogeneous populations, long-term repopulating cells (LTRC) and short-term repopulating cells (STRC). The two HSC populations have different surface markers or receptors and are classified based on quiescence and long-term...
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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Updated: Jun 17, 2025

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
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Hematopoietic stem cell transplantation for multiple sclerosis.

Giacomo Boffa1, Matilde Inglese2, Giovanni Luigi Mancardi1

  • 1Department of Neurology, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy.

Handbook of Clinical Neurology
|August 7, 2024
PubMed
Summary
This summary is machine-generated.

Hematopoietic stem cell transplantation (HSCT) offers a promising treatment for aggressive multiple sclerosis (MS) when other therapies fail. This approach involves intense immunosuppression and stem cell infusion, with over 1800 patients treated globally.

Keywords:
Disability improvementDisability progressionMRI activityMultiple sclerosisRelapse

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Area of Science:

  • Neurology
  • Immunology
  • Regenerative Medicine

Background:

  • Multiple sclerosis (MS) treatments have advanced, yet long-term remission, especially in aggressive cases, remains difficult.
  • Hematopoietic stem cell transplantation (HSCT) is emerging as a viable strategy for aggressive MS.
  • Over 1800 MS patients have undergone HSCT worldwide.

Purpose of the Study:

  • To provide an overview of HSCT for aggressive MS.
  • To highlight unique considerations for MS patients undergoing HSCT.
  • To discuss the efficacy, safety, and impact of HSCT on MS biomarkers.

Main Methods:

  • Review of HSCT procedures specific to MS patients.
  • Analysis of existing evidence on HSCT efficacy and safety in MS.
  • Presentation of preliminary data on HSCT's effects on CSF, MRI, and serum biomarkers.

Main Results:

  • HSCT is increasingly explored for aggressive MS, with significant patient numbers treated globally.
  • Specific considerations for MS patients include fertility preservation, therapy washout, and post-transplant monitoring.
  • Preliminary data suggest HSCT impacts CSF, MRI metrics, and biomarkers of neurodegeneration/demyelination.

Conclusions:

  • HSCT represents a significant therapeutic option for aggressive MS.
  • Careful patient selection based on international guidelines is crucial.
  • Further research into HSCT's long-term effects and biomarker changes is warranted.