Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis: a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Abstract

Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse. The procedure was generally well tolerated and only two instances of severe transplant-related toxicity were recorded. There was no treatment-related mortality, although one patient needed intensive care. aHSCT may be a therapeutic option for children with disease that does not respond to standard care.

Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019

“…polyarticular course or onset, inadequate response and/or intolerance to prednisone or diseasemodifying antirheumatic drugs) and other AD, including systemic sclerosis, systemic lupus erythematosus, vasculitis and polymyositis-dermatomyositis. Paediatric multiple sclerosis is a rare indication for autologous HSCT, but longterm responses have been reported [266]. Autologous and allogeneic HSCT have both been performed in severe autoimmune cytopenias, with similar outcomes [184].…”

Paediatric multiple sclerosis and antibody-associated demyelination: clinical, imaging, and biological considerations for diagnosis and care

“…Bone marrow transplant: Autologous hematopoietic stem cell transplantation (aHSCT) in adults with MS has shown dramatic reduction in relapse rate, although toxicity from the conditioning regimen has limited wide-spread adoption. The European Society for Bone and Marrow Transplant reviewed outcomes and safety of aHSCT in 21 pediatric MS patients from multiple centers 84 . Only one child required intensive care, infectious complications occurred in 4, and fever in a further 12 children.…”

Current international trends in the treatment of multiple sclerosis in children—Impact of the COVID-19 pandemic

“…Autologous haematopoietic stem cell transplantation has been used for MS since 1995 with almost complete suppression of the disease in adult RRMS, although very few studies have been randomized and controlled. ( Fassas et al, 1997 ) However, only limited paediatric data have been published to date( Burman et al, 2017 ), and a possible benefit has to be weighed against the risk of serious adverse events. ( Saccardi et al, 2006 ) In our survey, 36% of the respondents answered that stem cell transplantation is a treatment option for paediatric MS in their country.…”