By Caroline Song
Medical Xpress, a web-based medical and health news service has recently published an article titled “Stem cell transplantation for severe sclerosis associated with long-term survival” on June 24, 2014. The article reviews a study published in the June 25, 2014 issue of the Journal of the American Medical Association, or JAMA. The study conducted by Jacob M. van Laar, M.D., Ph.D., of the University Medical Center Utrecht, Utrecht, the Netherlands and Dominique Farge, M.D., Ph.D., of the Assistance Publique – Hopitaux de Paris, Paris 7 Diderot University, France, looked at patients suffering from a severe form of sclerosis, an autoimmune connective tissue disorder. The study compared two forms of treatment for sclerosis. The first form of treatment uses hematopoietic stem cell transplantation, or HSCT. The second treatment consisted of an intravenous infusion of the chemotherapeutic drug cyclophosphamide. From March 2001 to October 2009, 158 patients were recruited from 10 different countries and followed up until October 2013. The 158 patients were randomly assigned treatment by HSCT or cyclophosphamide. At the end of the study period, 53 adverse events had occurred with 22 in the HSCT group and 31 in the cyclophosphamide group. It was found that patients treated with HSCT had higher mortality in the first year of treatment, but had a better long-term survival in comparison to those treated with cyclophosphamide. The authors also felt that HSCT had a higher effectiveness in comparison to cyclophosphamide. The effectiveness was measured by evaluating skin, motor ability, quality of life, and patient lung function. A second study performed by Dinesh Khanna, M.D., M.Sc. of the University of Michigan, Ann Arbor and published by JAMA, discusses how HSCT should be used in treatment. Due to HSCT’s ability to increase long-term survival at the cost of increased mortality in the first year of treatment, there needs to be a strong framework to help evaluate when HSCT may be appropriate for patients suffering from systemic sclerosis.