When Should Anaemia Patients Consider a Bone Marrow Transplant?

Anaemia continues to be a significant public health issue in India, particularly affecting women, children, and pregnant mothers. Characterized by a deficiency of red blood cells or hemoglobin, anaemia leads to symptoms like fatigue, weakness, and in severe cases, life-threatening complications. The condition is often due to iron deficiency, but it can also result from genetic disorders, chronic diseases, or bone marrow dysfunction.

On National Anaemia Day, it’s essential to raise awareness about the condition, its impact, and advanced treatment options such as bone marrow transplant (BMT) for severe cases. Firstpost consulted with Dr. Rahul Bhargava, Principal Director and Chief BMT at Fortis Memorial Research Institute, Gurugram, to shed light on when a bone marrow transplant becomes necessary and the associated risks.

Understanding Anaemia and Bone Marrow Transplants

Anaemia occurs when the body does not produce enough healthy red blood cells or when red blood cells malfunction. These cells are vital as they transport oxygen to tissues, and their deficiency leads to various symptoms such as fatigue, dizziness, shortness of breath, and heart complications. Mild to moderate anaemia can often be managed through dietary changes, iron supplements, or medications. However, severe forms of anaemia may require more advanced interventions, including a bone marrow transplant.

Bone marrow is the spongy tissue inside bones responsible for producing red blood cells, white blood cells, and platelets. A bone marrow transplant involves replacing diseased or damaged bone marrow with healthy stem cells from a compatible donor. The procedure aims to restore healthy blood cell production, improving the patient’s overall health and quality of life.

When is a Bone Marrow Transplant Necessary?

According to Dr. Bhargava, BMT is typically considered in severe, life-threatening anaemia cases where the bone marrow fails to function adequately despite conventional treatments. The conditions warranting BMT include:

1. Aplastic Anaemia

Aplastic anaemia is a rare but serious condition where the bone marrow fails to produce enough blood cells. Symptoms include extreme fatigue, frequent infections, and uncontrolled bleeding. Traditional treatments include immunosuppressive therapy and blood transfusions, but if these fail, BMT becomes a viable option.

In aplastic anaemia, the patient’s bone marrow is damaged by autoimmune attacks or environmental factors like chemical exposure, radiation, or certain medications. BMT offers a potential cure by replacing the dysfunctional marrow with healthy stem cells from a donor, allowing normal blood cell production to resume.

2. Sickle Cell Anaemia

Sickle cell anaemia is a genetic disorder characterized by the production of misshapen, rigid red blood cells. These cells can obstruct blood flow, leading to severe pain, organ damage, and increased risk of stroke. While medications, pain management, and blood transfusions can alleviate symptoms, BMT remains the only known cure.

BMT is often recommended for children and young adults with severe complications. The procedure not only addresses the blood cell defects but can significantly improve the quality of life, reducing hospital visits and medical complications. Early transplantation, especially before irreversible organ damage occurs, yields better outcomes.

3. Thalassemia Major

Thalassemia major is another genetic condition where the body produces defective hemoglobin, leading to severe anaemia. Patients require regular blood transfusions to manage their condition, but over time, this can result in iron overload, damaging vital organs such as the heart and liver.

For thalassemia major, BMT is considered the only curative option, preferably performed at a young age. The transplant replaces the defective bone marrow with healthy stem cells, enabling the production of normal hemoglobin and reducing the need for ongoing transfusions.

4. Myelodysplastic Syndromes (MDS)

MDS encompasses a group of disorders where the bone marrow produces abnormal blood cells, which may progress to leukaemia. Symptoms include anemia, easy bruising, frequent infections, and fatigue. For high-risk MDS patients who do not respond to other treatments, BMT can prevent disease progression and offer a chance for remission.

Dr. Bhargava emphasizes that early diagnosis and timely consideration of BMT can be crucial for MDS patients, preventing the progression to more severe conditions like acute myeloid leukaemia (AML).

Risks and Considerations of Bone Marrow Transplant

Bone marrow transplantation is a complex and high-risk procedure, requiring careful consideration. The decision to proceed depends on factors such as the severity of the anaemia, overall health, age, and donor compatibility.

Procedure Overview

BMT involves eradicating the diseased bone marrow through high-dose chemotherapy or radiation, followed by the transplantation of healthy stem cells from a compatible donor. Donors can be related (usually siblings) or unrelated but matched via national and international donor registries.

The transplanted stem cells migrate to the bone marrow and begin producing healthy blood cells over several weeks. Recovery time varies, but patients typically spend several weeks in the hospital for close monitoring.

Potential Risks

While BMT can be life-saving, it carries significant risks:

1. Infections: The procedure suppresses the immune system, making patients highly susceptible to bacterial, viral, and fungal infections. Rigorous infection control measures are essential during and after the transplant.

2. Graft-versus-Host Disease (GVHD): GVHD occurs when the donor’s immune cells attack the patient’s tissues. Acute GVHD affects the skin, liver, and gut, while chronic GVHD can lead to long-term complications. Treatment includes immunosuppressive drugs, but GVHD remains a significant concern.

3. Organ Damage: Pre-transplant chemotherapy and radiation can affect organs like the heart, lungs, and liver, leading to complications during recovery.

4. Bleeding and Anemia: Platelet production may be impaired, increasing the risk of bleeding. Blood transfusions are often required to manage this issue.

5. Relapse of Disease: In conditions like MDS, there’s a risk that the disease may return, even after a successful transplant.

6. Infertility: The aggressive treatments can affect fertility, which is an essential consideration for younger patients.

7. Mortality Risk: Despite advancements in BMT procedures, there is still a mortality risk associated with the transplant, especially for older patients or those with advanced disease stages.

Evaluating the Decision

Dr. Bhargava advises that the decision to undergo BMT should be made collaboratively between the patient, haematologist, and transplant specialist. The assessment considers the patient’s overall health, disease severity, donor availability, and willingness to endure the procedure’s risks and recovery period.

In cases where conventional therapies fail to improve anaemia, or where the condition severely compromises quality of life, BMT offers a chance for remission or cure. For patients with aplastic anaemia, sickle cell anaemia, thalassemia major, or high-risk MDS, BMT is often the best course of action.

Advancements in Bone Marrow Transplantation

Advances in transplant techniques and supportive care have improved outcomes significantly. Innovations include:

• Reduced-intensity conditioning (RIC): This approach uses lower doses of chemotherapy or radiation, reducing organ damage and infection risks.
• Peripheral blood stem cell transplants (PBSCT): Stem cells are collected from peripheral blood rather than bone marrow, reducing complications.
• Improved donor registries: Enhanced international registries increase donor matching accuracy, expanding access to suitable donors.
• Targeted therapies: New drugs minimize immune responses and manage GVHD effectively.

Anaemia remains a critical health concern in India, with severe forms demanding advanced treatments like bone marrow transplants. While BMT offers hope for a cure in conditions like aplastic anaemia, sickle cell anaemia, thalassemia major, and MDS, the decision requires thorough consultation with healthcare professionals. Awareness about BMT and its benefits can lead to informed decisions, offering patients a chance for a healthier future.