Pαβlo: Paediatric αβ haploidentical transplantation

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.

Disease Stage: high risk haematological malignancy, or a non-malignant disease correctable by HSCT who do not have a fully matched sibling donor, who have an indication to undergo HSCT

Patient Age Range: >3 months to ≤18 years

Sample Size: 124

Trial Sponsors:

• National: ANZCHOG

Read more at the Clinical Trial Registry