Young Patients' Quality of Life After Allogeneic Bone Marrow Transplantation at the Hematology Department at Rigshospitalet Investigated With a Youth-specific Questionnaire - EORTC QLQ-AYA

Background Allogeneic hematopoietic cell transplantation (alloHCT), formerly called bone marrow transplantation, is a potentially curative treatment for patients with life-threatening hematological cancers and bone marrow diseases. The young patient is not entirely well defined, but often includes patients from 13 or 15 years to 29 or 39 years. Most of the young patients aged 18 to 29 who are transplanted for hematological diseases have leukemia and lymphoma and have undergone one to several intensive chemotherapy courses to get their hematological disease under control, which gives the best benefit from the transplant. A prerequisite for alloHCT is that a suitable tissue-type-compatible family or foreign matched donor can be found for the patient. The donor cells re-establish a normal bone marrow function after the transplant and are an important prerequisite for the patient to be cured, as they perform an immunological anti-cancer effect, the so-called graft versus leukemia effect.

Before the transplant, the patient and the next of kin meet for a pre-interview where they are informed about the transplant and early and late complications. AlloHCT is a prerequisite for cure, but also a risky treatment with a transplant-related mortality of 10-20% among patients.

The transplant itself starts with pretreatment consisting of chemotherapy and radiation therapy prior to the infusion of the donor stem cells. Both chemotherapy and radiotherapy are given in varying intensity depending on the patient protocol, which depends on the patients age, general health and the disease they are being transplanted for. After the transplant, the patient is on immunosuppressive (e.g. Ciclosporin) and supportive (e.g. antibiotics) treatment for at least half a year. Then an attempt is made to taper off the transplant medication, which is not always successful at first, which is why the immunosuppressive treatment often has to be continued.

There are many different complications and side effects to alloHCT. Partly toxic to chemo and radiotherapy, partly infections and not least so-called graft versus host disease (GVH), a relatively frequent condition (30-50%), where donor cells attack the patients healthy organs (e.g. skin, mucous membranes, liver, intestines and lungs) with the need for increased and prolonged immunosuppressive treatment, with morbidity and, in rare severe cases, mortality. Despite this, long-term survival is on average 60-70%. But survival comes with a price for many - with degrees of late sequelae and reduced quality of life.

During a tough and long-lasting cancer course, young patients find themselves in a particularly vulnerable situation and the young persons life is put on pause socially and educationally (Sodergren et al, Jrn AYA Onc 2018 and Qual Life Res, 2017). The brain is not fully developed, which makes it more exposed to toxic influences. Specifically for the young person who completes an alloHCT course with intensive chemo and radiation treatment, there are many side effects. If the young person also gets treatment-requiring GVH and has to have long-term heavy immunosuppressive treatment with e.g. steroid - in addition to discomfort from the GVH attack itself, there is fatigue, difficulty concentrating, negative changes in appearance (changed fat distribution, loss of muscles, discolouration of the skin, hair loss) and a long list of various late effects (diabetes, hypertension, hormonal disorders, vision problems, skin cancer, etc etc). Young people thus have several reasons to be unhappy and have a reduced quality of life in many aspects of youth life after alloHCT. At the same time, they live the first years after the transplant with the fear that their cancer will return.