For some myelofibrosis (MF) patients whose disease symptoms and quality of life deteriorate, even with the current range of drug therapies and treatments available, a stem cell or bone marrow transplant (BMT) may be considered.
Watch the video where Chris shares his experience of a bone marrow transplant, also known as stem cell transplant (SCT).
BMT for MF involves using blood stem cells from a family member, unrelated donor or umbilical cord blood unit. An autologous BMT, where the patient’s own cells are used, is not an option. Every patient’s circumstances will be unique to them and the haematologist and transplant team will need to consider numerous factors including:
The International Prognostic Scoring System (IPSS) for MF
This is a risk stratification system which uses five variables of anaemia, age, leukocytosis, (high white blood cell count), peripheral blasts (immature cells) and constitutional symptoms such as weight loss, drenching sweats and fever. Consideration of these variables provide clinicians with four levels of risk factor from low risk, (no risk factors), through to high risk, (three of more risk factors). Using these variables plus additional factors such as low platelets, chromosomal changes or abnormalities and being dependent on blood transfusions, a patient’s risk and prognosis can then be categorised at any stage in the disease. In general, clinicians do not routinely transplant patients who have an IPSS of low or intermediate risk 1 of the disease and await a ‘‘trigger’’ to move forward to transplant. However it is not wise to leave a transplant until MF is high risk or progressing to leukaemia as the success of transplant is then lessened.
The general health of the patient is also important in assessing the possibilities of transplant. By the nature of the median age of onset of MF, many patients referred for a transplant are of a more advanced age. Many transplant centres will consider patients aged up to 70––75 years of age. However, with advancing years, other medical problems may make transplant more complicated or risky and therefore will be taken into account for assessment of suitability for transplant.
Stem cell transplant: simplified facts
Haematopoietic stem cells (HSC) – immature bone marrow cells which mature into red or white cells or platelets. In a stem cell transplant these cells are ‘‘harvested’’ from a donor and transplanted to the recipient.
Donors may be found from family members, but siblings only offer a one in four chance of being a ‘‘tissue-type’’ match. Many BMTs involve unrelated donors from large international Stem Cell Transplant registries. Sometimes it can be difficult to find a suitable donor and this may mean that a transplant is not possible.
Conditioning prior to the transplant refers to the preparation of the patient’s bone marrow to receive the donor cells. Usually 7–10 days of high dose chemotherapy +/- radiotherapy which helps to destroy diseased cells in the patient’s system but also weakens or dampens the immune system.
Transplant day: Day zero – This is not surgery but where the patient receives healthy donated stem cells via a central infusion line via blood bags similar to ones used for blood transfusions. The transfused cells will find their way to the patient’s bone marrow where they will eventually start to divide and make healthy new blood cells. This is known as engraftment and typically will take two to three weeks for the neutrophil levels to rise over 0.5. Engraftment of red cells and platelets take longer.
Graft-Versus-Host Disease (GVHD) – A common complication of BMT. There are two types, acute and chronic ranging from mild to severe. GVHD occurs because of the differences between the patient and donor cells so that the new immune system from the donor may see the recipient’s cells as different and attack them. GVHD often affects the skin, gut and liver. Depending on the type of GVHD, the treatment options and risk of having GVHD will be different.
Other considerations – A BMT is a serious undertaking and a wide range of complications can occur. Infectious complications occur frequently as well as common side effects like ‘‘mucositis’’, a painful mouth, making eating temporarily difficult.
Post transplant management and outcomes
Every patient’s outcomes and recovery times will vary enormously but regular monitoring will be part of the post transplant regime. In the early stages, hospital visits will be more often and it can be common to be readmitted to hospital for a period of time depending on the individual patient needs. It is possible to have a short recovery but for some patients recovery can last for many years. Don’t forget, MPN Voice offers patients the opportunity for buddy support in all aspects of living with MPNs so if you are considering a transplant or have undergone one, and would like support, please do contact us at firstname.lastname@example.org.
You can also visit the real stories to read about the experiences of people who have undergone a bone marrow transplant.