Stem cell potential for children with severe autoimmune diseases: review


Haematopoietic stem cell transplantation (HSCT) has a role in children and young people with severe autoimmune and inflammatory diseases who have exhausted other treatment options, according to an analysis of European data. 

The data from 326 patients under the age of 18 suggests it is possible to achieve successful control of severe haematological autoimmunity by both autologous and allogeneic stem cell transplants, albeit with a significant risk of toxicity and complications.

While the evidence is limited due to the rarity of the treatment in this patient group, early disease recognition, careful patient selection and a multidisciplinary approach as well as ongoing attempts at disease control, should improve the efficacy and safety of this procedure, the review in the British Journal of Haematology concludes.

The analysis looked at children and young people with severe autoimmune and inflammatory diseases who had been registered in the European Society for Blood and Marrow Transplantation between 1996 and 2021. 176 procedures were performed with autologous graft and 167 with donor stem cells.

In the dataset there were 146 children who had an autologous or allogeneic transplant for a rheumatic disease, most commonly systemic or polyarticular juvenile idiopathic arthritis (86), followed by systemic lupus erythematosus (22) and systemic sclerosis (16).

While the data showed some cases of complete remission – including in 55% of the 65 patients who had an autologous transplant for systemic or polyarticular juvenile idiopathic arthritis – there remain significant risks of treatment related morbidity and mortality, the reviewers said.

These risks mean the option of an HSCT is limited to those who are refractory for all other therapies.

“These are rare treatments and patients have to be managed in a very individualised way. Many patients will have been resistant to multiple therapies so will have been in a challenging situation where there is no effective alternative,” Professor John Snowden, Consultant Haematologist and Director of Blood and Marrow Transplantation at Sheffield Teaching Hospitals NHS Foundation Trust, who is also Secretary to the EBMT, and co-author of the review, told the limbic.

“In a proportion of patients, the long-term outcomes have been good, even curative in extremely challenging patients, but there is always a difficult balance of risks of transplant-related complications and toxicities.

“Patients should always be reviewed by a multidisciplinary team and discussed with paediatric centres with experience in haematopoietic stem cell transplantation in autoimmune diseases, such as Great Ormond Street and Newcastle in the UK”, he added.

Looking forward, the authors noted that prospective studies are needed to define the safety and efficacy of stem cell transplantation in this group of patients.

And while developments in transplant technique are likely to improve outcomes further, there are also likely to be significant, concurrent advances in ‘standard of care’ treatments, and possibly the option to combine them, they added.

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