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Transplant Timing and Diseases Treated

Several important factors affecting patient survival outcomes of a hematopoietic cell transplant include:

  • The timing of the transplant. Typically, transplants performed earlier in the disease process have been shown to have better outcomes.
  • The disease and disease stage.

Additional factors affecting transplant outcomes include the patient's age and co-morbidities, urgency of the need for a transplant and availability of a well-matched donor or cord blood unit.

Transplant timing

Transplant outcomes are affected by performing an unrelated donor or cord blood transplant at an appropriate time during the disease (usually remission) and according to the patient’s health.

The patient's treatment plan may indicate the need for an immediate transplant. Or, another treatment may be performed initially, with a plan to move to transplant quickly, if the need arises.

The Recommended Timing for Transplant Consultation (marrow.org) guidelines were developed and published jointly by the National Marrow Donor Program (NMDP) and American Society of Blood and Marrow Transplantation (ASBMT). These guidelines are based on current clinical practice and the medical literature, as well as comprehensive evidence-based reviews [1].

Hematopoietic cell transplantation is a potential life-saving treatment option for some patients. However, one of the critical factors in improved outcomes is appropriate timing of the transplant. These guidelines:
  • Indicate prognostic factors for patients at risk of disease progression using standard therapy
  • Indicate which patients should be evaluated for transplantation
  • Provide a basis for initial discussions when developing a treatment plan that may include transplantation
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Diseases treatable by a hematopoietic cell transplant

Due to long-term experience, ongoing studies and advances in transplantation:
  • Patient eligibility for transplant continues to expand.
  • The list of indications for which transplant may be a standard treatment option continues to increase.

Below is a list of diseases for which autologous or allogeneic hematopoietic cell transplantation may be a treatment. The list includes diagnoses for which transplantation is a standard treatment as well as diagnoses for which transplantation is a newer option. When information about a disease is available on marrow.org, it is linked to Table 1: Disease Specific Information and Outcomes.

Leukemias and lymphomas, including:

Multiple myeloma and other plasma cell disorders

Severe aplastic anemia and other marrow failure states, including:
SCID and other inherited immune system disorders, including:
Hemoglobinopathies, including:
  • Beta thalassemia major
  • Sickle cell disease

Hurler's syndrome and other inherited metabolic disorders, including:
Myelodysplastic and myeloproliferative disorders (MDS), including:
  • Refractory anemia (all types)
  • Chronic myelomonocytic leukemia
  • Agnogenic myeloid metaplasia (myelofibrosis)

Familial erythrophagocytic lymphohistiocytosis and other histiocytic disorders

Other malignancies

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Table 1. Disease Specific Information and Outcomes

Disease

Information on marrow.org

Acute myelogenous leukemia (AML), adult and pediatric

Patient Information (marrow.org) Physician Information (marrow.org)
Acute lymphoblastic leukemia (ALL), adult and pediatric Patient Information (marrow.org) Physician Information (marrow.org)
Chronic myelogenous leukemia (CML), adult Patient Information (marrow.org) Physician Information (marrow.org)
Hodgkin's disease Physician Information (marrow.org)
Non-Hodgkin's lymphoma (NHL), adult Patient Information (marrow.org) Physician Information (marrow.org)
Multiple Myelomas Physician Information (marrow.org)

Severe aplastic anemia, adult and pediatric

Fanconi anemia, pediatric

Patient Information (marrow.org) Physician Information (marrow.org)
Severe combined immunodeficiency (SCID) Patient Information (marrow.org) Physician Information (marrow.org)
Wiskott-Aldrich syndrome (WAS) Patient Information (marrow.org) Physician Information (marrow.org)
Hurler’s Syndrome Patient Information (marrow.org) Physician Information (marrow.org)
Cerebral X-linked adrenoleukodystrophy (ALD) Patient Information (marrow.org) Physician Information (marrow.org)
Globoid-cell leukodystrophy (GLD) or Krabbe's disease Patient Information (marrow.org) Physician Information
Metachromatic leukodystrophy (MLD)  Patient Information (marrow.org) Physician Information (marrow.org)
Myelodysplastic syndromes (MDS) Patient Information (marrow.org)
    Physician Information (marrow.org)

    References

    1. Evidence-based Reviews, American Society for Blood and Marrow Transplantation. 2006. Published in Biology of Blood and Marrow Transplantation and available online at: http://www.asbmt.org/policystat/policy.html


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    Pre-transplant Factors Affecting Outcomes

    Transplant Terms
    Hematopoietic cell transplant (HCT) is also referred to as:
    • HSCT - hematopoietic stem cell transplant
    • BMT - blood or marrow transplant
    A transplant may also be described by cell source:
    • Bone marrow transplant
    • Peripheral blood stem cell (PBSC) transplant
    • Cord blood transplant
    Hematopoietic cells are also referred to as:
    • Blood-forming cells
    • Blood-progenitor cells
    • Hematopoietic progenitor cells

    Medical Professional




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