HIQA to begin HTA on the repatriation of stem cell transplant services for children in Ireland

Date of publication:

The Health Information and Quality Authority (HIQA) has published the protocol for its next Health Technology Assessment (HTA) on the repatriation of paediatric haematopoietic stem cell transplant services to Ireland. The protocol describes the approach that HIQA's team will use to complete this piece of work.

The HTA will examine the choice of treatment location for stem cell transplants (allogeneic haematopoietic stem cell transplant (HSCT)) to treat children with certain inborn errors of metabolism, inborn errors of immunity and haemoglobinopathies.

Currently, patients with conditions other than bone marrow failure syndromes or haematological malignancies (for example, leukaemia) must go abroad to receive HSCT. This treatment has historically been provided in the UK and is funded through the HSE's Treatment Abroad Scheme. This HTA will examine whether or not transplants for children with certain inborn errors of metabolism, inborn errors of immunity and haemoglobinopathies should be carried out in Ireland.

Dr Conor Teljeur, HIQA's Chief Scientist, said: “HSCT treatment can be long, with patients and their families sometimes needing to stay abroad for three to six months. During that time, siblings or parents may be separated from each other. Also, the availability of treatment is dependent on the host country's capacity, which can impact on when families can access treatment.”

The HTA will analyse organisational, social and ethical issues, and the budget impact associated with providing HSCT for these conditions in Ireland rather than abroad. The results of the HTA will inform a decision by the HSE on whether to repatriate treatment and provide HSCT in Ireland for paediatric non-malignant HSCT indications.

You can find the protocol from the link at the top of the page.

The full Health Technology Assessment will be published in due course.


For further information please contact:

Marty Whelan, Head of Communications and Stakeholder Engagement 085 805 5202 / mwhelan@hiqa.ie

Notes to the editor:

  • The Health Information and Quality Authority (HIQA) has today published the following document: Repatriation of paediatric haematopoietic stem cell transplant services to Ireland: Protocol for a Health Technology Assessment. The document outlines the approach and methods that will be used to complete the assessment.
  • This HTA was requested by the National Paediatric Public Health Lead in the Health Service Executive. It is supported by specialist clinicians in Children’s Health Ireland.
  • Haematopoietic stem cell transplantation is not a suitable treatment for every patient with an inborn error of metabolism, inborn error of immunity or haemoglobinopathy. Its suitability depends on the form and severity of disease and the availability of a suitable donor.
  • A stem cell transplant (sometimes known as a bone marrow transplant), is a treatment whereby a person is given a transfusion of healthy blood stem cells from their own body or from a donor. These transplanted cells can grow into new healthy blood cells in the person’s bone marrow. This HTA examines allogeneic transplants, which involve receiving stem cells from a donor. Donors are often siblings or close family members, but can be strangers.