Approximately 150 children undergo bone marrow transplant in Australia and New Zealand every year. Some children have stem cells donated from a relative, and others get their stem cells from a volunteer donor who is a genetic match, or from donated umbilical cord blood. Some patients, however, do not have any of these donors available.
Parents share half of their genes with their children, and so are haploidentical (a half genetic match). Haploidentical donors have not been used routinely in the past because of the complication known as graft versus host disease (GVHD). GVHD is when the donor cells attack the patient’s body because they are not a complete genetic match. GVHD is caused by a specific type of blood cell, called a TCR α+β+ T cell. Transplant doctors now have a way of removing these T cells from the donor stem cells, to stop GVHD from occurring. This is called TCR α+β+ cell depletion. The purpose of this study is to take haploidentical donor stem cells, and perform TCR α+β+/CD19+ cell depletion before they are given to a patient. It is hoped that the donor stem cells will grow (engraft) in the patient, and that the chance of the patient getting GVHD will be low, thus improving treatment outcomes for these children.
The primary purpose of this trial is to evaluate the efficacy of using parent donors for bone marrow transplants in children, using a particular processing method of the donor blood cells (TCR a+ß+/CD19+ cell depletion) compared to those receiving a standard bone marrow transplant from another donor, We also want to compare cost to the health system of TCR a+ß+/CD19+ cell depletion with that of standard bone marrow transplant procedures,
Who is it for?
Patients may be eligible to enrol in this trial if they are aged 3 months to 18 years of age, have been diagnosed with haematological malignancies or non malignant disorders requiring a bone marrow transplant, and have an eligible parent donor.
Study details
Patients who have no fully matched brothers or sisters, and no well matched volunteer donors or umbilical cord blood units available will receive a TCR a+ß+/CD19+ cell depleted graft from a parent donor. Control patients will receive a standard bone marrow transplant from another donor (e.g., volunteer donor) as per institutional practice.
Patients will undergo clinical assessments at regular intervals for two years following the transplant to evaluate the efficacy of the treatment. Further information, such as quality of life and carer burden, will be collected for up to 5 years following the transplant.
It is hoped that the findings from this trial will provide information on whether parent donor cells undergoing specialised cell processing can be effectively used for bone marrow transplants in children with haematological malignancies or non malignant disorders.
