Autologous Stem-Cell Transplantation for Severe Scleroderma
New England Journal of Medicine,Volume 378, Issue 11, Page 1066-1067, March 2018.
Nicoletta Del Papa, Francesca Pignataro, Eleonora Zaccara, Wanda Maglione, Antonina Minniti
Purpose of review To provide an overview of recently published work on autologous hematopoietic stem-cell transplantation (HSCT) in patients with systemic sclerosis (SSc). Recent findings Superiority of HSCT vs. intravenous cyclophosphamide pulses was demonstrated in the randomized controlled American Scleroderma: Cyclophosphamide or Transplantation (SCOT) Trial (n = 75), supporting the results from earlier studies. In the SCOT Trial, total body irradiation was used instead of the nonmyeloablative regimens used in other trials, and considered well tolerated during a follow-up time of 4.5 years. Three small uncontr...
Purpose of review To summarize the current clinical experience in the use of autologous hematopoietic stem cell transplantation (HSCT) in autoimmune disease and to explore the concept of durable remission induction and tissue de-remodeling via restoration of normal niche function and “immune reset." Recent findings Controlled clinical trials in systemic sclerosis, multiple sclerosis, and Crohn's disease as well as extensive uncontrolled trial and registry data have established the unique role of HSCT in selected cases. Although HSCT for multiple sclerosis and systemic sclerosis has recently entered several ...
Conditions: Systemic Sclerosis; Diffuse Sclerosis Systemic; Interstitial Lung Disease; Pulmonary Hypertension Interventions: Drug: Cyclophosphamide; Drug: Mesna; Drug: Rituximab; Drug: Alemtuzumab; Drug: Thiotepa; Drug: GM-CSF Sponsor: Paul Szabolcs Not yet recruiting
Introduction: Graft-versus-host disease (cGVHD) is a major complication of allogeneic stem cell transplantation which develops due to the action of immunocompetent donor lymphocytes against immunosupressed recipient host cells. Onset of the disease after 100 days of transplantation is termed as chronic GVHD. Lichenoid and sclerodermoid changes are the two main cutaneous presentations of chronic GVHD. Sclerodermoid GVHD was first described by Master et al. in 1975 and is considered a severe form of the disease eventually making the patient disabled.
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Conclusions: For scleroderma patients, fewer adipose-derived stem cells, because of a history of corticosteroid therapy and a local inflammatory microenvironment, are more important factors, whereas blood supply showed no significant change. Therefore, cell-assisted lipotransfer not only improves the survival rate of transplanted fat but also improves skin texture in bleomycin-induced skin fibrosis nude mice.
PMID: 29654391 [PubMed - as supplied by publisher]
British Journal of Dermatology,Volume 178, Issue 3, Page e239-e239, March 2018.
Systemic sclerosis: Autologous HSCT is efficacious, but can we make it safer?, Published online: 08 March 2018; doi:10.1038/nrrheum.2018.34Autologous haematopoietic stem cell transplantation (HSCT) has proved efficacious in treating patients with systemic sclerosis, but different regimens have different associated toxicities and different effects on lung function. Through comparison of different clinical trials, we can learn how to improve the safety of HSCT.