Bone marrow is the site of production of new blood cells from a specialized group of cells called the "stem cells". Stem cells have the capacity to divide throughout life and replenish all types of blood cells. In certain diseases, bone marrow fails to produce blood cells (e.g. – aplastic anemia) or produces abnormal hemoglobin (e.g. – sickle cell anemia, thalassemia). In blood cancers, bone marrow is replaced with cancer cells and sometimes these children do not respond to chemotherapy which is the first line treatment. These patients need a new healthy bone marrow to fight these fatal conditions. The process by which a healthy marrow from a donor is infused into a patient is called "Bone marrow transplant (BMT)". Bone marrow transplant is a lifesaving treatment for many children suffering from blood related diseases, cancers, immunodeficiencies and metabolic diseases.
The group of diseases in which children are prone to recurrent infections due to a defect in the immune system are called PRIMARY IMMUNE DEFICIENCY DISEASES. “1 in 2000 children may have an immune deficiency. The city of Bangalore may alone have around 5000 children with immune deficiency, but many remain undiagnosed due to lack of awareness of these disease. Children with serious immune deficiencies often die if bone marrow transplant is not offered on time. Only a handful of centres in India have the expertise to treat immune deficiency diseases. The Pediatric Immunology department at Aster CMI is one of its kinds that has the wherewithal to diagnose and treat the most complex diseases of children. The Pediatric Bone Marrow Transplant (BMT) unit at Aster CMI is led by a team of highly qualified specialists and guided by international expertise of Dr. Fulvio Porta to handle all kinds of complicated cases. The most advanced technology and equipment, upto-date infrastructure; leading experts in the field and above all, the will to cure have all come together at Aster CMI’s Bone marrow transplant center that is offering cure to young souls with immune deficiencies.
When vital organs of an individual start failing to function, transplantation of an organ from a healthy donor can be life-saving. You must have heard of liver transplant, kidney transplant and so on. Very analogous to these, bone marrow transplant (BMT) is lifesaving in many individuals with serious diseases.
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What Are The Conditions That Warrant Bone Marrow Transplant?
The broad group of conditions where Pediatric Bone Marrow Transplant (BMT) is useful are as follow -
- If the bone marrow starts failing to produce blood elements – Aplastic anemia. These individuals have low hemoglobin, white cell counts, and platelet counts and require monthly blood transfusions. Providing new bone marrow from a healthy donor can cure these patients.
- If the hemoglobin is abnormal – Thalassemia, sickle cell anemia, and other hemoglobinopathies. These children require periodic blood transfusions and Pediatric Bone Marrow Transplant (BMT) can be curative for these diseases.
- Cancers–Blood cancers (acute leukemia), solid tumors (neuroblastoma), and many other cancers can be treated with Pediatric Bone Marrow Transplant (BMT).
- Primary Immune Deficiency diseases – Some children are born with a problem in their immune system and fall ill repeatedly. They may be hospitalized for infections on several occasions. Repeated ear discharge, pneumonia, or diarrhea are often noted. Such children require immunological testing. In many of these immune deficiencies, Pediatric Bone Marrow Transplant (BMT) is curative. Severe Combined Immune Deficiency is often present in young children who fail to gain normal weight and fall ill repeatedly. This condition is universally fatal without a Pediatric Bone Marrow Transplant (BMT).
- Metabolic diseases – Some children are born with defects in the enzymes required for a normal metabolism - Inherited disorders of metabolism, congenital storage diseases.
When Should One Seek An Opinion From A Pediatric Bone Marrow Transplant (BMT) Expert?
- If your child is diagnosed with any of the above-mentioned conditions.
- If your physician/pediatrician has advised a bone marrow transplant as an option.
- Children who are diagnosed with cancers and do not respond to chemotherapy.
- Children who remain unwell and have recurrent infections. Immunological testing and a visit to an Immunologist is warranted. The immunologist would guide you, in case a transplant is needed in your situation.
What Are The Steps Involved In Pediatric Bone Marrow Transplant (BMT)?
Pediatric Bone Marrow Transplant (BMT) is a complex procedure requiring hospitalization for a month and sometimes longer.
Following are the steps involved:
- The patient is evaluated for the need for a transplant. In some conditions, a bone marrow transplant is an emergency (for example – severe combined immune deficiency), while in others, it is an elective procedure wherein patients are stabilized before undergoing a transplant.
- Donor selection – Akin to blood transfusion, where one needs to transfuse blood with the right blood group; a match between the donor and recipient is of paramount importance in Pediatric Bone Marrow Transplant (BMT). HLA matching is carried out using blood samples or buccal swabs. Healthy siblings who are fully matched on HLA typing offer the best outcome in most of the transplants. Haploidentical transplants (with mother/father being the donor) and matched unrelated donor (MUD) transplants are the other options.
- Conditioning – Here, the patient is admitted and medications are administered to prepare him/her for the transplant.
- Bone marrow transplant – Bone marrow-derived from the donor is administered as an intravenous infusion to the patient.
- Post-transplant recovery – The Patient is given medications and is observed for recovery. This period involves periodic blood tests to ensure recovery is smooth.
