High dose immunosuppressive therapy and stem cell transplantation in autoimmune and inflammatory diseases

2002 ◽  
Vol 2 (4) ◽  
pp. 399-414 ◽  
Author(s):  
Sarah J. Bingham ◽  
John Snowden ◽  
Gareth Morgan ◽  
Paul Emery
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Inflammatory Diseases ◽  
High Dose ◽  
Autoimmune And Inflammatory Diseases

Survival, and time to an advanced disease state or progression, of untreated patients with moderately severe multiple sclerosis in a multicenter observational database: relevance for design of a clinical trial for high dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation

2006 ◽  
Vol 12 (2) ◽  
pp. 174-179 ◽  
Author(s):  
M Daumer ◽  
L M Griffith ◽  
W Meister ◽  
R A Nash ◽  
J S Wolinsky
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
High Dose ◽  
Hematopoietic Stem ◽  
Edss Score

Despite prolonged survival, patients with multiple sclerosis (MS) experience considerable morbidity, which adversely impacts quality of life. To assess the risk-benefit of a clinical trial of high dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation for MS, we sought to determine the natural history of the disease in a comparison group of untreated patients. We identified 285 individuals with 2132 combined observation years (median: 5.6 years; 5th to 95th percentile: 1-21 years), with Expanded Disability Status Scale (EDSS) scores of 3.0-5.5 at baseline observation. Disease-related mortality was zero at five years, 5.4% at 10 years, and 22% at 15 years (40 patients contributing to the data point; 95% confidence interval: 4-32%). Risk for progression to advanced disability, defined as an EDSS score of 8, was very low for the subgroup with a baseline EDSS score of 3-3.5; however, for those with a baseline EDSS score of 4-5.5, 3% had advanced disability after two years, 5% after three years, 6% after four years, 12% after five years, and 40% after 10 years. The estimated probability of disease progression, defined as an increase in EDSS score by ≥ 1.0 sustained for at least 180 days, was 5% after one year, 14% after two years, 22% after three years, 38% after five years, 57% after 10 years, and-80% after 20 years of observation. The relevance of these features to the design of the clinical trial is discussed.

scholarly journals High-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation for severe multiple sclerosis

Blood ◽  
2003 ◽  
Vol 102 (7) ◽  
pp. 2364-2372 ◽  
Author(s):  
Richard A. Nash ◽  
James D. Bowen ◽  
Peter A. McSweeney ◽  
Steven Z. Pavletic ◽  
Kenneth R. Maravilla ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
High Risk Group ◽  
Oligoclonal Bands ◽  
High Dose ◽  
Barr Virus ◽  
Engraftment Syndrome

Abstract There were 26 patients enrolled in a pilot study of high-dose immunosuppressive therapy (HDIT) for severe multiple sclerosis (MS). Median baseline expanded disability status scale (EDSS) was 7.0 (range, 5.0-8.0). HDIT consisted of total body irradiation, cyclophosphamide, and antithymocyte globulin (ATG) and was followed by transplantation of autologous, granulocyte colony-stimulating factor (G-CSF)-mobilized CD34-selected stem cells. Regimen-related toxicities were mild. Because of bladder dysfunction, there were 8 infectious events of the lower urinary tract. One patient died from Epstein-Barr virus (EBV)-related posttransplantation lymphoproliferative disorder (PTLD) associated with a change from horse-derived to rabbit-derived ATG in the HDIT regimen. An engraftment syndrome characterized by noninfectious fever with or without rash developed in 13 of the first 18 patients and was associated in some cases with transient worsening of neurologic symptoms. There were 2 significant adverse neurologic events that occurred, including a flare of MS during mobilization and an episode of irreversible neurologic deterioration after HDIT associated with fever. With a median follow-up of 24 (range, 3-36) months, the Kaplan-Meier estimate of progression (≥ 1.0 point EDSS) at 3 years was 27%. Of 12 patients who had oligoclonal bands in the cerebrospinal fluid at baseline, 9 had persistence after HDIT. After HDIT, 4 patients developed new enhancing lesions on magnetic resonance imaging of the brain. The estimate of survival at 3 years was 91%. Important clinical issues in the use of HDIT and stem cell transplantation for MS were identified; however, modifications of the initial approaches appear to reduce treatment risks. This was a heterogeneous high-risk group, and a phase 3 study is planned to fully assess efficacy. (Blood. 2003;102:2364-2372)

High-dose chemotherapy and hematopoietic stem cell transplantation for breast cancer: Past or future?

2001 ◽  
Vol 28 (4) ◽  
pp. 377-388 ◽  
Author(s):  
Roy D. Baynes ◽  
Roger D. Dansey ◽  
Jared L. Klein ◽  
Caroline Hamm ◽  
Mark Campbell ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
High Dose Chemotherapy ◽  
High Dose ◽  
Hematopoietic Stem
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