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Who Needs A Stem Cell Transplant?

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A bone marrow transplant also called stem cell transplant is a procedure that infuses healthy blood-forming stem cells in another person's body to replace damaged or diseased bone marrow.

Stem cell transplants are reserved for patients with life-threatening diseases whose blood, bone marrow, or immune system is compromised.

Deciding whether you should agree to get a stem cell transplant can be overwhelming. Here are a few things to consider to help you make that decision.

From a disease standpoint

Standard of care. What it means is that the role of a transplant is reasonably well defined and results compare favourably (or are superior) to those of non-transplant treatment approaches. It may be offered in clinical scenarios such as when cancer that is lurking in a dormant form repopulates. It might still be sensitive to a different chemotherapy cocktail, but the response achieved is unlikely to be durable. Another scenario is if you have a disease that is categorized as high risk (for relapse) based on an analysis of genetic changes. It is generally not recommended in scenarios such as early stages of disease when the additional risk of a transplant is not justified, or in advanced forms of a disease in which the chance of success is so small that it does not justify the risks.

When things go wrong: Diseases

Severe Aplastic anemia

Hemoglobinopathies

Severe Combined Immune Deficiency Syndrome

Inborn metabolic disorders

When things go very wrong: cancer

Acute Myelogenous Leukemia (AML)

Acute Lymphocytic Leukemia (ALL)

Chronic Myelogenous Leukemia (CML)

Non-Hodgkin’s Lymphoma (NHL)

Hodgkin’s disease (HD)

Burkitt Lymphoma

Myelodysplastic Syndrome (MDS)

Multiple Myeloma (MM)

Germ cell tumors

Neuroblastoma

Ewing’s Sarcoma

From a patient standpoint

The average age of patients undergoing transplants has risen over the past few decades (old-old) made possible largely by the development of low-intensity conditioning regimens tolerable by older patients. A formal assessment of whether you will be able to survive the storm (transplant!) is made by considering organ function (based on whether your kidneys, lungs, heart, and liver working fine) the presence of comorbid conditions, and active infections.

Neither

Let’s face it. Your life is not going to be the same anymore. So, do not underestimate the potential impact of your decision. A decision that cannot be guided by experience. And one that can have either wonderful (achieving long-term remission and cure) or appalling consequences (result in death). There can only be evidence presented to you that this transplant might work. It is not proof that it will. In reality, the numbers derived from decision-making tools that we use to guide us, are only as good as the methods we use to arrive at them. Take for instance the methods used to assess the depth of response to treatment. We’ve transitioned from complete morphological remission (a test that detects 1 cancer cell out of 100 normal cells) to measurable residual disease (a test that detects 1 cancer cell out of 10,000 normal cells). Several other variables add to the unpredictability of the situation such as studies with contradictory outcomes, different treatment protocols adopted by different centres, experience, access to drugs, and socioeconomic factors.

Take the time to reflect on your life. Ask yourself the pertinent questions. Are you a seventy-year-old who has lived a content life? Are you a twenty-year-old who has only just begun? How much are you willing to sacrifice to crush this disease?

Accept the fact that there will be good days and not-so-good days. Understand that it’s a marathon, not a sprint. Once you take a leap of faith, stop second-guessing and brace yourself for the challenge.

Standard of care. What it means is that the role of a transplant is reasonably well defined and results compare favourably (or are superior) to those of non-transplant treatment approaches. It may be offered in clinical scenarios such as when cancer that is lurking in a dormant form repopulates. It might still be sensitive to a different chemotherapy cocktail, but the response achieved is unlikely to be durable. Another scenario is if you have a disease that is categorized as high risk (for relapse) based on an analysis of genetic changes. It is generally not recommended in scenarios such as early stages of disease when the additional risk of a transplant is not justified, or in advanced forms of a disease in which the chance of success is so small that it does not justify the risks.

Severe Aplastic anemia

Hemoglobinopathies

Severe Combined Immune Deficiency Syndrome

Inborn metabolic disorders

When things go very wrong: cancer

Acute Myelogenous Leukemia (AML)

Acute Lymphocytic Leukemia (ALL)

Chronic Myelogenous Leukemia (CML)

Non-Hodgkin’s Lymphoma (NHL)

Hodgkin’s disease (HD)

Burkitt Lymphoma

Myelodysplastic Syndrome (MDS)

Multiple Myeloma (MM)

Germ cell tumors

Neuroblastoma

Ewing’s Sarcoma

The average age of patients undergoing transplants has risen over the past few decades (old-old) made possible largely by the development of low-intensity conditioning regimens tolerable by older patients. A formal assessment of whether you will be able to survive the storm (transplant!) is made by considering organ function (based on whether your kidneys, lungs, heart, and liver working fine) the presence of comorbid conditions, and active infections.

Let’s face it. Your life is not going to be the same anymore. So, do not underestimate the potential impact of your decision. A decision that cannot be guided by experience. And one that can have either wonderful (achieving long-term remission and cure) or appalling consequences (result in death). There can only be evidence presented to you that this transplant might work. It is not proof that it will. In reality, the numbers derived from decision-making tools that we use to guide us, are only as good as the methods we use to arrive at them. Take for instance the methods used to assess the depth of response to treatment. We’ve transitioned from complete morphological remission (a test that detects 1 cancer cell out of 100 normal cells) to measurable residual disease (a test that detects 1 cancer cell out of 10,000 normal cells). Several other variables add to the unpredictability of the situation such as studies with contradictory outcomes, different treatment protocols adopted by different centres, experience, access to drugs, and socioeconomic factors.

Take the time to reflect on your life. Ask yourself the pertinent questions. Are you a seventy-year-old who has lived a content life? Are you a twenty-year-old who has only just begun? How much are you willing to sacrifice to crush this disease?

Accept the fact that there will be good days and not-so-good days. Understand that it’s a marathon, not a sprint. Once you take a leap of faith, stop second-guessing and brace yourself for the challenge.

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