What is involved in a bone marrow (hematopoietic stem cell) transplant for a patient with a hematological disorder, such as leukemia, lymphoma, or multiple myeloma, or an autoimmune disease?

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What's Involved in a Bone Marrow Transplant

Bone marrow transplantation (hematopoietic stem cell transplantation) involves the infusion of hematopoietic stem cells into a patient after receiving high-dose chemotherapy (usually marrow-ablative) to re-establish normal blood cell production and immune function. 1

Core Components of the Transplant Process

Pre-Transplant Phase: Conditioning Regimen

  • Most patients receive chemotherapy, serotherapy, and/or radiation therapy before the actual stem cell infusion to prepare the body for transplantation 1
  • In allogeneic transplants, the conditioning regimen eradicates malignant cells in the bone marrow (if using myeloablative regimen) and induces immunosuppression to allow donor cell engraftment 1
  • In autologous transplants, high-dose myeloablative chemotherapy treats the underlying malignancy, followed by rescue infusion of the patient's own previously harvested cells to restore blood cell production 1

Stem Cell Source Selection

The hematopoietic stem cells can be obtained from three sources 1:

  • Peripheral blood stem cells (most common): Harvested after mobilization with growth factors (G-CSF or GM-CSF), avoiding general anesthesia and providing faster engraftment 1
  • Bone marrow: Collected via aspiration under general anesthesia, preferred for certain conditions like severe aplastic anemia due to lower graft-versus-host disease (GVHD) risk 1
  • Umbilical cord blood: Used primarily for pediatric allogeneic transplants, allows greater histoincompatibility tolerance 1

Transplant Type Classification

Allogeneic Transplantation (donor cells from another person):

  • Requires HLA-matched donor, ideally an HLA-identical twin or matched sibling for best outcomes 1
  • Alternative donors include matched unrelated donors (found through registries like the National Marrow Donor Program) or mismatched family members 1
  • Most effective for severe aplastic anemia and offers the only curative therapy for chronic myelogenous leukemia 1
  • Most commonly used for acute myeloid leukemia, acute lymphocytic leukemia, and myelodysplastic syndromes 1

Autologous Transplantation (patient's own cells):

  • Preferred when patients require high-dose chemotherapy but have healthy, undiseased bone marrow 1
  • Most frequently used for multiple myeloma, non-Hodgkin's lymphoma, and Hodgkin's disease 1
  • Does not carry risk of chronic GVHD 1

Post-Transplant Management and Complications

Immunosuppression for Allogeneic Recipients:

  • Cyclosporine or tacrolimus (FK506) is administered in combination with other immunosuppressive agents (methotrexate or corticosteroids) to prevent GVHD 1
  • T-lymphocyte depletion techniques reduce GVHD but increase risks of graft rejection, CMV infection, invasive fungal infection, and EBV-associated lymphoproliferative disease 1

Key Risk Factors:

  • Recipients of allogeneic grafts from non-HLA-matched siblings face substantially greater risk for GVHD, suboptimal graft function, and delayed immune recovery 1
  • Allogeneic peripheral blood transplants carry increased GVHD risk compared to bone marrow grafts 1

Critical Pitfalls to Avoid

  • Never use stem cell-toxic drugs (nitrosoureas, alkylating agents, especially melphalan) in potential transplant candidates before stem cell collection, as they damage the stem cell reserve 2
  • Collect sufficient stem cells before starting treatment to allow for multiple transplants if needed, as long-term therapy may limit future collection 2
  • For multiple sclerosis patients, autologous transplantation is not suitable for patients over 55 years old, with disease duration exceeding 20 years, or without focal inflammation 2

Outcomes and Success Factors

Transplant outcomes vary based on 1:

  • Type and stage of disease being treated
  • Overall health and age of the patient
  • Degree of HLA-mismatch between donor and recipient (for allogeneic transplants)
  • Source of hematopoietic cells used

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Guideline

Hematopoietic Stem Cell Transplantation for Various Diseases

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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