A global online study of haematopoietic stem cell transplantation in multiple sclerosis and other neurodegenerative disorders

• Published in: Chronic diseases and translational medicine Vol. 9; no. 1; pp. 39 - 43
• Main Authors: Panegyres, Peter K., Russell, Jodi, Chen, Huei‐Yang, Panegyres, Mariella, Cui, Yi
• Format: Journal Article
• Language: English
• Published: United States John Wiley & Sons, Inc 01.03.2023; John Wiley and Sons Inc; Wiley
• Subjects: Costs; Disease; Family income; Medical research; Multiple sclerosis; neurodegenerative disorders; Original; Patient satisfaction; Quality of life; Social networks; Stem cell transplantation; stem cells; Canada; Australia; United States > US; Western Europe; neurodegenerative disorders; stem cells; multiple sclerosis
• ISSN: 2589-0514; 2095-882X; 2589-0514
• DOI: 10.1002/cdt3.44

Background The objective of this study was to understand the uptake of hemopoietic stem cell transplantation (HSCT) in neuroimmunological disorders like multiple sclerosis (MS). Method An independent University affiliated research organization conducted a global online survey of people having had HSCT, examining demographics, treatment protocol, and effectiveness. Results Of 271 participants, useful data were available in 223; women aged 35–54 accounted for 73.5%. Most had a household income greater than US$50,000, and the majority of participants were from Australia and the United States. Nearly 94.6% of people suffer from MS. Most had their treatment in Russia (38.7%) and 78.1% had nonmyeloablative transplants. Nearly half of the participants spent between US$50,000 to US$74,999. There were 54.5% of neurologists who did not support their patients having HSCT. Around 85.5% of participants believed HSCT helped them manage their disease from weeks to years after transplantation, and treatment was recommended by 9.5% of participants. The average reduction in Expanded Disability Status Score after transplantation was 1.2 (95% CI: 0.97–1.41; N = 197; p < 0.01; t: 10.7, df: 196). Conclusion Participants were supportive of HSCT despite the costs and would recommend it to others. The data suggest some benefit in minimizing disability in MS and provides justification for large randomized controlled trials. Expanded Disability Status Score (EDSS) difference among 197 patients after stem cell transplantation. Highlights Patients support hematopoietic stem cell transplantation (HSCT) in multiple sclerosis. Patients will pay and travel long distances for HSCT. Patients will recommend HSCT to others. Most patients report HSCT helps their disease. This study provides evidence that patients believe in HSCT, stimulating the need for further investigation in randomized controlled trials on a global scale.