The best way to describe this treatment is a hematopoietic stem cell transplant (HSCT). Hematopoietic refers to the body’s ability to make the cells of your bloodstream: red blood cells that carry oxygen, platelets that help with clotting, and white blood cells that help fight infection.

In most cases, a patient has a problem that would benefit from replacing their blood system with someone else’s (the donor). Hematopoietic (blood-making) stem cells are given or transfused into a patient. Over time, these grow into a mature blood system.

In almost every circumstance, a conditioning or preparative regimen (i.e., chemotherapy) is provided prior to the administration of stem cells. This regimen usually involves giving intravenous chemotherapy, but also may require administering radiation. The donor undergoes an extensive screening process including testing that ensures their stem cells are as closely matched as possible to the patient receiving them. This matching process is important because we want the donor’s stem cells to grow into a blood system that lives harmoniously with the patient.

In rare instances, such as for treatment of certain cancers and tumors, the patient’s own stem cells can be given back to them after chemotherapy is delivered.

Our experienced transplant doctors and support staff will spend time explaining to you and your child all the aspects of the process. This includes the planned preparative regimen (chemotherapy), donor options and strategies to prevent the unwanted risks of transplant.

There are three types of HSCT: allogeneic, autologous and syngeneic.

• An allogeneic HSCT uses stem cells from a donor who is an acceptable human leukocyte antigen (HLA) match for your child. This can be from a related donor or an unrelated donor.

• An autologous HSCT uses your child’s own stem cells with the purpose of giving them back to him or her after receiving an intense preparative or conditioning regimen (chemotherapy).

• A syngeneic HSCT uses stem cells from your child’s identical twin.

Our attending doctors will discuss with you which type of HSCT is best for your or your child’s type of disease.

The first option for a donor is oftentimes in a patient’s own family. A sibling’s (brother’s or sister’s) bone marrow or blood is “harvested” for stem cells. In some cases, a patient’s parents are used as a donor for a transplant. Sometimes, unrelated adult donors are used as source of stem cells. The National Marrow Donor Program (NMDP) and other international registries store information about individuals who have been generous enough to donate their hematopoietic stem cells for use in a transplant. Lastly, cord blood units are also used as a source of stem cells.

A patient’s HLA status is typed when it is determined that he or she would benefit from a transplant. HLA stands for human leukocyte antigen. Antigens are special proteins found on the surface of white blood cells (also called leukocytes). You inherit your HLA typing from your parents. HLA typing is used to determine how well a donor matches a patient. Blood samples or cheek swab samples are used to determine HLA typing. The match status determines how a transplant doctor will strategically approach the treatment for the patient and their disease.

When patients receive a bone marrow transplant, stem cells are harvested from a donor’s hip bone. The donor receives anesthesia and, while asleep, a transplant doctor uses special tools to collect blood from the middle of the bone or marrow space.

Some patients receive stem cells from an individual’s peripheral blood. In that instance, a donor receives special intravenous catheters, and they are connected to a special machine that filters the blood and collects the stem cells.

Finally, some patients receive stem cells from a cord blood unit. Some mothers agree to donate their blood to a stem cell registry. After their baby is born, the blood from the umbilical cord, which is also a rich source of hematopoietic (blood-making) stem cells, is frozen and kept in a bank for later use.

In each of these cases, the donor or cord blood unit is HLA-typed to ensure that it is an appropriate source of stem cells to be used on a patient who is to receive it as part of their transplant.

The risks of transplant include:

Complications to Vital Organs

It is possible that treatment with a transplant can cause damage to multiple organs of the body. The most commonly affected organs are the heart, liver, lungs and kidneys. These organs will be monitored closely throughout transplant, and consulting doctors will assist the BMT team in the management of your care.

Infection

You are at a high risk for infections (bacterial, viral and fungal) related to your weakened immune system. These infections can come from out in the community or from within your own body. You will be on medications to prevent infections. You will require close monitoring and a quick response to infectious symptoms such as fever.

Our BMT team is working hard to introduce new therapies and medicines that can prevent and treat infection. Our center also has open clinical trials exploring the expansion and delivery of special immune cells called cytotoxic T lymphocytes (CTLs) taken from your donor that have been trained to prevent and treat certain viral infections. These CTLs can be administered in the weeks after you have received your donor’s stem cells. Your bone marrow transplant attending doctor may determine if you are eligible for these trials and therapies.

Mucositis

Mucositis is a potential side effect of the preparative regimen (chemotherapy) where your mucous membranes have died off, causing inflammation, pain and sometimes bleeding. This can occur in the mouth, throat and gastrointestinal (GI) tract. This side effect typically resolves when your white blood cell count increases.

Our BMT team has been working hard to develop new approaches to transplant that minimize this problem. We work with specialized doctors who are part of the Pain and Palliative Service. They will help minimize your discomfort from this expected side effect of transplant.

Graft-Versus-Host Disease (GVHD)

GVHD is a risk with allogeneic (donor) HSCT. GVHD is caused when the immune cells (specific kind of white blood cells called T cells) from the donor (graft) recognize your body (host) as foreign and cause a reaction. GVHD causes damage to tissues or organs in your body and affects their ability to function normally. The effects to these organs can be anywhere from mild to life-threatening and may be fatal. GVHD can occur early or late in the transplant process. Medications are started during the preparative regimen (chemotherapy) to prevent this complication and continue post-transplant. These medications suppress your immune system and cause you to be at increased risk for infections.

GVHD symptoms can include skin rash or peeling, yellowing of the skin, poor liver function, poor appetite, nausea, vomiting, stomach / abdominal pain, diarrhea or blood in the stool. Your attending doctor will evaluate your symptoms and start testing or treatment, if needed.

Our BMT team is working hard to introduce new strategies, therapies and medicines that can prevent and treat GVHD. Some of these approaches are being actively investigated at our center. Your bone marrow transplant attending doctor may determine if you are eligible for these trials.

Thrombotic Microangiopathy (TMA)

A complication involving the vascular system as a result of chemotherapy, infection or GVHD. The blood vessels become inflamed and small blood clots can form. This causes kidney injury, protein leaking in urine and high blood pressure. The lungs, heart and gut may also be affected.

New approaches now exist to identify and treat TMA early before it worsens. Some approaches are being actively investigated at our center. Your bone marrow transplant attending doctor may determine if you are eligible for trials that explore how best to diagnose and treat TMA.

Veno-Occlusive Disease (VOD)

Veno-occlusive disease is a complication involving the liver as a result of high doses of chemotherapy or radiation as part of the preparative regimen (chemotherapy). The blood vessels in the liver become blocked and the liver can no longer process medications and waste products in the blood. This causes swelling and may lead to other organ failure.

Our center has become an active location for new and developing treatments for VOD. Your bone marrow transplant attending doctor will determine the best prevention and treatment strategies for VOD.

In general, it is a disease that would benefit from the administration of an intense preparative regimen (chemotherapy and/or radiation) and replacing your blood system with someone else’s (the donor’s). Here is a short list of some diseases that can be treated by performing an allogeneic (donor) transplant:

• High-risk malignancies: relapsed leukemias and lymphomas
• Immune deficiencies 
• Hemagophagocytic lymphohistiocytosis (HLH)
• Bone marrow failure syndromes like Fanconi anemia
• Hemoglobinopathies like sickle cell anemia and beta thalassemia
• Other rare metabolic disorders and enzyme deficiencies including Hurler’s syndrome, osteopetrosis and adrenoleukodystrophy

Certain solid tumors like neuroblastoma or brain cancers like medulloblastoma may require treatment by delivering an autologous (own stem cells) HSCT. This is when high-dose chemotherapy is given to a patient followed by the delivery of his or her own stem cells.

Generally, the BMT hospitalization includes the time needed to administer the preparative regimen (chemotherapy) and the time needed to recover from this treatment. It can be as short as six to eight weeks, and sometimes is longer.

The stem cells are given on “Day 0.” After this, there is a time period of approximately two weeks when you are particularly vulnerable to infection. During this time, you may also experience pain from mouth sores or mucositis. Medicines are needed to keep you as comfortable as possible. Fluids and nutrition are provided to keep you hydrated and stable. Other medicines are used to keep your body as stable as possible throughout the process.

After your stem cell infusion, approximately two to three weeks are also needed to allow your donor graft to begin producing blood cells adequate for symptoms to improve. Then, many of the medicines and nutritional support used to keep you stable need to be weaned and/or stopped.

Your doctor will work closely with the in-patient BMT team and your caregivers to decide when you are well enough to be safely discharged from the hospital after BMT. Education is a large part of the discharge process so you are comfortable after leaving the hospital.

Some families stay close by in places like the Ronald McDonald House or the Hope Lodge. Others find accommodations in local residence housing.

Cincinnati Children’s will work with you to provide the best possible options that suit you and your family. You can learn more through our Guest Services.