HAPLO+4Kids
Research type
Research Study
Full title
Memory T cells to improve immunity after TCRαβ/CD19 depleted haploidentical donor stem cell transplantation for inborn errors of immunity
IRAS ID
1007527
Contact name
Sophie Hambleton
Contact email
Sponsor organisation
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Research summary
We plan to study the benefit of a cellular therapy called CD45RO+ memory T cell addback after stem cell transplantation from a mismatched donor in children with inborn errors of immunity (IEI).
IEI are associated with reduced quality of life and risk of death in early childhood in severe cases. Stem cell transplantation is an established curative therapy for affected patients, but about 25-60% of those eligible lack a suitable tissue-type-matched donor. An alternative is to use a mismatched family or unrelated donor, such as a parent, but there is a catch. Stem cell harvests include not only the stem cells that will go on to repopulate the patient's bone marrow, but also a mixture of mature immune cells that are armed and potentially dangerous. These cells include a group of white blood cells (good T cells) which are very useful for fighting infections. However, after a transplant, bad T cells from the donor can attack normal cells in the patients and cause a condition called "graft-versus-host disease" (GvHD). T cells are very sensitive to tissue type differences, so GvHD is a big risk in mismatched transplants. For this reason, it is standard practice to remove most T cells from the graft, but it takes a long time for the immune system to recover after this type of transplant. This leads to a high risk of serious infections and even death during the transplant period, until the immune system recovers.
The new cellular therapy we plan to study is a way of giving back the good T cells (memory T cells, CD45RO+) from a portion of the donor graft as an "addback" (or boost) after T cell-depleted mismatched transplant.REC name
London - Hampstead Research Ethics Committee
REC reference
23/LO/0514
Date of REC Opinion
16 Oct 2023
REC opinion
Further Information Favourable Opinion