Practice question

How do bone marrow transplants work?

Children undergoing bone marrow transplants need support and follow up

Bone marrow transplantation and recovery from it are complicated, and children who have it need support and follow up

Bone marrow transplantation (BMT), or haematopoietic stem cell transplantation, has been used for many years to cure various immunological and haematological disorders, and more recently solid tumour cancers ( Simpson and Dazzi 2019 ).

The bone marrow is where blood cells are made. In babies, many bones produce bone marrow but as children grow, bone marrow development occurs predominantly in the pelvic bones, arms, spine, sternum and ribs (

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Bone marrow transplantation and recovery from it are complicated, and children who have it need support and follow up

Picture: iStock

Bone marrow transplantation (BMT), or haematopoietic stem cell transplantation, has been used for many years to cure various immunological and haematological disorders, and more recently solid tumour cancers (Simpson and Dazzi 2019).

The bone marrow is where blood cells are made. In babies, many bones produce bone marrow but as children grow, bone marrow development occurs predominantly in the pelvic bones, arms, spine, sternum and ribs (Chamley et al 2005).

Stem cells are made in the bone marrow and will eventually become red blood cells, white blood cells and platelets as they come out of the bone marrow and move into the thymus (Children’s Cancer and Leukaemia Group 2019). The thymus, situated just below the breastbone, ‘decides’ what the stem cells will become.

Children given an autologous transplant can recover more quickly from chemotherapy

A child with a condition affecting the bone marrow will be unable to produce healthy red cells, white cells and platelets, and the child’s symptoms – pale, lethargic, clotting problems and a compromised immune system – will reflect this.

It is possible for a patient to receive a transplant using their own stem cells, a process called an autologous transplant. This is usually performed in children who receive high-dose chemotherapy as part of cancer treatment.

In an autologous transplant, the child receives medication to push their own stem cells into their peripheral blood before cancer treatment begins. This happens in a process called apheresis, where stem cells are collected but other blood cells are returned. The stem cells are given back once the child has received treatment.

An autologous stem cell transplant allows the patient’s bone marrow to recover more quickly from chemotherapy.

For an allogeneic transplant the patient needs to be matched with a donor

An allogeneic transplant is one in which the patient receives stem cells from a donor, and is used for children who require their own diseased or compromised bone marrow to be replaced.

To receive stem cells, the patient will need to be a ‘match’ with the donor. Full siblings are always tested first, as there is a one in four chance they will be a complete match.

Patients who lack suitably matched donors among their relatives require other stem cell sources such as matched unrelated donors, umbilical cord blood or from relatives who are haploidentical or half-matched. The less ‘matched’ the donor, the more complications can be expected and the higher the risk of bone marrow rejection.

First, the patient’s existing marrow must be eliminated so the donor marrow can grow in its place. A combination of intense chemotherapy drugs is used to destroy the bone marrow.

Stem cells or bone marrow are administered in the same way as a blood transfusion

Until the donor marrow is established and healthy, children will require transfusions of red blood cells and platelets when symptoms indicate these are low. However, white blood cells are not routinely transfused post-transplant so patients will be placed in strict isolation to protect them from infections.

BMT procedures take place on the ward. The stem cells or bone marrow are administered in the same way as a blood transfusion, infused via a central line.

Patients will then remain in isolation, with a regimen of supportive treatments, until they show signs of the new marrow ‘engrafting’. Children will have regular blood monitoring to assess this and can often become unwell owing to bone marrow suppression.

Recovery from the process is complicated

Infection is a major complication and can be one of the highest causes of death for such patients (Styczyński et al 2020). Because of the risk of infection, a number of prophylactic medications are prescribed and antibiotic treatment given if the child is symptomatic (Rasheed et al 2019).

Graft-versus-host disease (GvHD) occurs in allogeneic BMT where the donor marrow starts to attack host cells (Moules et al 2008). The skin, liver and gut are the organs most commonly affected, causing rashes, diarrhoea and liver dysfunction (Vogelsang et al 2003).

Given the complexity of BMT, it is not unusual for children to experience psychological effects such as depression and even post-traumatic stress disorder (Breitwieser and Vaughn 2014, Widows et al 2000). Recovery from the process is complicated, so children will often attend healthcare services for ongoing care, monitoring and follow up.


This article has been subject to external open peer review and checked for plagiarism using automated software

References

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