Bone Marrow Transplant (BMT) also called as Stem Cell Transplant is a procedure to replace damaged or destroyed bone marrow of a patientwith healthy bone marrow stem cells of the donor. It is the soft, fatty tissue inside your bones which produces blood cells. Stem cells are immature cells in the bone marrow that give rise to all of your different blood cells. BMT procedure involves transplanting blood stem cells, which travel to the bone marrow where they produce new blood cells and promote growth of new marrow which in turn give a normal & healthy life to the patient.

Groundbreaking research continues to advance the science of transplant so more diseases can be treated with a bone marrow or cord blood transplant. See what diseases are treatable with a transplant.

Bone marrow transplants have been successfully used for the treatment and cure of a variety of Blood Cancers, Immune System Diseases, and Blood Diseases for many years now.

Learn more about how transplant may treat different diseases and when it may be an option for you on BMT Clinic.com. For some diseases, BMT is the only potential cure. There are over 70 diseases that can be treated by BMT. Some of them are listed here.

Indications of Allogeneic BMT

1. Nonmalignant diseases

• Bone Marrow Failure States, including:
  • Severe Aplastic Anemia
  • Fanconi’s Anemia
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Pure Red Cell Aplasia/Acquired And Inherited (DBA)
  • Amegakaryocytosis / Congenital Thrombocytopenia

• Immunodeficiency States
  • Severe Combined Immunodeficiency (SCID)
  • Wiskott-Aldrich syndrome (WAS)
  • Chediak-Higashi syndrome
  • Congenital Neutropenia

• Hemoglobinopathies
  • Beta Thalassemia Major
  • Sickle Cell Disease

• Metabolic Disorders
  • Hurler’s Syndrome (MPS-IH)
  • Adrenoleukodystrophy (ALD)
  • Metachromatic Leukodystrophy (MLD)
  • Gaucher’s disease
  • Nieman Picks Disease
  • Osteopetrosis

2. Malignant Diseases

• Acute Lymphoblastic Leukemia (ALL)
• Acute Myelogenous Leukemia (AML)
• Chronic Myelogenous Leukemia (CML)
• Juvenile Myelomonocytic Leukemia (JMML)
• Chronic Myelo- Monocytic Leukemia
• Chronic Lymphocytic Leukemia (CLL)
• Myelodysplastic Syndrome (MDS)
• Multiple Myeloma
• Hodgkin’s and Non Hodgkin’s Lymphoma
• Myeloproliferative disorders: Myelofibrosis

Autologous Transplant

Autologous BMT is done by using the patient’s own stem cells.

Indications of Autologous BMT

• Acute Promyelocytic Leukemia
• Multiple Myeloma
• Hodgkin’s & Non Hodgkin’s Lymphoma
• Germ Cell Tumor
• Ewing’s sarcoma
• Neuroblastoma

After the infusion of stem cells or bone marrow, it takes 4 to 6 weeks for the new bone marrow to grow and function as it should. During this time, you may run the greatest risk of bleeding, or of having infections and other complications. It occurs when the new bone marrow, called graft, does not recognize the person who receives it, called the host. The graft then sends out fighter white blood cells, called lymphocytes, to attack the host. It can be mild, moderate or severe and can involve the skin, the liver or the bowel. Rashes and diarrhea are symptoms. Sometimes tissue samples of symptomatic areas are taken to diagnose the disease. This complication is seen with allogenic transplants, in which the transplant comes from either a related or unrelated donor.

Signs to watch for Sign or symptoms of infection

1. A fever above 38. 5 C or 101 F.
2. Any breathing or respiratory problems, or a cough, runny nose, sore throat or cold.
3. Stomach complaints, such as diarrhea, nausea, vomiting or cramps.

Signs or symptoms of Graft-versus-host disease ( GVHD ) ( for patients with a donor transplant only)

1. A new rash on the body, palms of the hands, or soles of the feet.
2. Watery diarrhea.
3. Loss of appetite with weight loss.
4. Jaundice, a yellow colour in the whites of the eyes or in the skin.

Visitors

Visitors at home are allowed but they must be carefully screened. Family and friends should not visit if they are ill. Visiting children have greater chance carrying contagious diseases because they are more exposed to them in the school environment. Chickenpox, measles, mumps and cold sores are of particular concern. Please report any known exposure to these diseases to the BMT coordinator as soon as possible.

Skin Care

Make sure patient bathes or showers every day using non-irritating soap and shampoo. Always clean the tub with a cleanser first. If skin is dry apply Vaseline or paraffin oil to the skin. Protect patient’s skin from the sun, especially if patient had radiation or has GVHD.

Follow these guidelines.

1. Cover patient’s skin as much as possible with clothing.
2. Make sure patient wear a hat, especially until his or her hair has grown back.
3. Use lots of sunscreen on patient’s skin.

Mouth Care

Make sure attendant or patient care patient’s mouth at least 3 times a day, preferably after meals.

1. If platelets are lower than 100,000 use toothette. If your platelets are higher, use a soft toothbrush. Use any mild toothpaste with fluoride.
2. Make sure that the areas where the gums meet the upper and lower cheek are brushed thoroughly.
3. Report any white patches, called thrush, or mouth sores to patient’s physician.

Medications

Patient will still need medications at home. Nurse and the pharmacist will explain the medication to patient or attendants. Before patient leave the hospital, please be sure that patient or attendants understand what all the medications are for and how to give them. Some of these medications can be expensive.

Common side effects of transplant

1. Mucositis: Mucositis is the painful inflammation and ulceration of the mucous membranes lining of gastrointestinal tract, which usually occurs as an adverse effect of conditioning regimen of transplant wither because of high dose chemotherapy or total body irradiation.

Mucositis is often divided into two main types, including:

• Oral Mucositis, which occurs inside the mouth and can cause painful mouth ulcers
• Gastrointestinal Mucositis, which occurs inside the digestive system and often causes diarrhea

2. Infections: Patient who undergo transplant, they are at risk of severe infection both from inside the body or from outside. During the phase of low counts they are at risk of severe bacterial infection and fungal infection. Once patient accept the graft (engraftment), they are at risk of severe viral infections, fungal infection and rare bacterial infection. This increase susceptibility to infection is due to either immunosuppressive therapy or treatment of graft versus host disease.

The immune reconstitution after transplant occurs usually after 12-24 months in Allogenic stem cell transplant and minimum of 6 months after Autologous stem cell transplant.

3. Bleeding and transfusion: Patient undergoing high dose chemotherapy and transplant will develop severe cytopenia before recovery of graft function (engraftment). During this period they will require regular packed red blood cells and platelet transfusion to prevent complication related to low hemoglobin and platelets. All blood product transfusion after transplant will be irradiated blood products.

4. Graft Versus Host Disease: For doing a successful Transplantation, doctors need the Bone Marrow or Stem Cell enriched blood of a donor, which can be transfused into the patient to replace his diseased Bone Marrow. This marrow or stem cell enriched blood is called the ‘GRAFT’.

Graft Versus Host Disease with an Allogenic transplantation (a transplant from a donor) there is a possibility that the donors cells (the graft) will react against the recipient’s tissue (the host), even when the donor is one’s own brother or sister. This is called Graft Versus Host Disease (usually referred to as GVHD). This is one of the serious complication can occur post Allogenic transplant. The degree of severity increases proportionately to degree of mismatch between donors and recipients at HLA or in setting of unrelated donor transplant or Haplo identical transplant. All Allogenic transplant patient usually gets immunosuppressive treatment to prevent GVHD, thus usually HVHD is mild. Some times GVHD it can become very severe and even life threatening. It can cause diarrhea, skin rashes and acute or chronic liver damage. The disease can occur up to one year after the transplant. If its occur before 100 days usually called as acute graft versus host disease and if present after 100 days its known as chronic graft versus host disease.

If graft versus host disease occurs, it does not mean that the transplant has failed. The GVHD is often beneficial in patients with high risk hematopoietic malignancies because it may kill residual cancer cells (graft versus leukemia/tumor effect – GVL/GVT effect)

Graft Versus Host Disease (Gvhd): When cells of the immune system from the donor’s Graft attacks the body of the patient, recognizing it as foreign. This only happens with BMT and not with solid organ transplantation.

GVHD is of Two Types

• Acute: This is a sudden onset inflammation of skin, gut and liver, resulting in skin rash, diarrhoea and jaundice respectively.
• Chronic: This is more like a slowly developing thickening and tightening of skin, dryness of eyes and mouth and joint pains etc.

5. Veno- Occlusive disease: Hepatic Veno-Occlusive Disease (VOD), also known as sinusoidal obstruction syndrome (SOS) is one of the most serious and common complications after myeloablative hematopoietic stem cell transplantation (HSCT). Clinical diagnosis of hepatic VOD is based on the clinical triad of (1) painful hepatomegaly, (2) hyper bilirubinemia and (3) unexplained fluid retention. While milder cases usually resolve spontaneously, severe VOD is associated with a grim prognosis.

6. Graft failure: Rarely in Allogenic transplant, there are chances that recipient failed to accept the graft. This could be primary engraftment failure or secondary. Primary engraftment failure is a condition where patient failed to accept the graft and usual outcome is either recovery of self-blood cells or fatality. Secondary engraftment failure means patient accepted the donor graft but recipient reject later.

When the donor blood stem cells do not seed the patient’s bone marrow, either due to

• Inadequate amount of donor stem cells.
• Rejection or destruction by the residual cells in the patient.

​What is a GRAFT composed of?

The graft has various cells with a definite role to play and consists of:-

• CD 34 Cells (Stem Cells) which are required for replenishing the patient’s marrow with healthy stem cells.
• T Cells which are further sub classified into TCRαβ and TCRγΔ. T cells are double edged weapons, they protect us from viruses and react against tumour cells. When they are transfused into host (patient) they may react violently, if they do not recognise the host’s tissues/organs and this may lead to Graft Versus Host Disease (GVHD). In the same way residual T cells in the patient can reject the donor GRAFT and cause GRAFT FAILURE.
• B Cells protect us from various infections and assist the T Cell in accelerating the process of Graft Versus Host Disease (GVHD).
• NK Cells are the natural killer cells, They are very unique, because they not only kill the cancer cells, they also protect us from infections. They do not contribute to GVHD.

Other side effects which patient may encounter are Thrombotic micro angiopathy and hemolytic anemia, persistent low platelet, hemorrhagic cystitis & interstitial pneumonia.