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Stem Cell (Bone Marrow) Transplantation

Overview of Stem Cells and Stem Cell Transplants
Open Enrollments
Research Studies
Transplant Funding
Related Articles
Patient Stories

Overview of Stem Cells and Stem Cell Transplants

Stem Cells. This site provides many links about stem cells, stem cell transplants, research, and the latest news. MedlinePlus.

Cellular Therapies in Systemic Sclerosis: Recent Progress. Currently, hematopoietic stem cell transplantation is the only cellular therapy that has demonstrated clinical effects on the immune system, neoangiogenesis, and fibrosis. PubMed, Curr Rheumatol Rep, 2016 Feb;18(2):12.

Advances in pathogenesis and treatment of systemic sclerosis. Immunosuppression is now established as a beneficial approach but balancing risk and benefit is vital, especially for powerful approaches such as autologous stem cell transplantation. PubMed, Clin Med, 2015 Dec;15 Suppl 6:s58-63. (Also see Immunosuppressants)

Autologous Hematopoietic Stem Cell Transplantation (HSCT) vs Intravenous Pulse Cyclophosphamide in Diffuse Cutaneous Systemic Sclerosis: A Randomized Clinical Trial. Among patients with early diffuse cutaneous systemic sclerosis, HSCT was associated with increased treatment-related mortality in the first year after treatment. However, HCST conferred a significant long-term event-free survival benefit. JAMA, 2014;311(24):2490-2498. (Also see Cyclophosphamide Research)

Is immunosuppressive therapy the anchor treatment to achieve remission in systemic sclerosis (SSc)? Patients with rapidly progressing diffuse cutaneous SSc should be evaluated for hematopoietic stem cell transplantation. PubMed, Rheumatology, 2013 Oct 31.

Stem cell transplantation in systemic sclerosis. Stem cell transplantation is an effective treatment option for patients with severe systemic sclerosis, but is associated with toxicity and treatment-related mortality. Patient selection and comprehensive cardiopulmonary screening are critical factors in determining outcome. PubMed, Curr Opin Rheumatol, 2013 Nov;25(6):719-25.

Is belief larger than fact: expectations, optimism and reality for translational stem cell research. A more honest acknowledgement of the expected therapeutic benefits of stem cell therapies and the timelines to achieving them are necessary. PubMed, BMC Med, 2012 Nov 6;10:133.

Open Enrollments

Allogeneic Hematopoietic Stem Cell Transplantation

Currently recruiting. Last updated 10/15/12.

Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic Sclerosis This study is designed to examine whether treating patients with high dose Cyclophosphamide and Fludarabine (drugs which reduce the function of your immune system) and CAMPATH-1H (a protein that kills the immune cells that are thought to be causing the disease), followed by return of blood stem cells that have been previously collected from patients brother or sister will stop or reverse the disease. Northwestern University, Chicago, Illinois. ClinicalTrials.gov.

ASSIST Trial: Now Recruiting

Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial (DISclRituxan). ASSIST I was the first randomized trial in patients with scleroderma to not just slow disease progression but rather actually reverse it. It is the first treatment to have ever demonstrated reversal of lung disease in scleroderma with improvement in FVC, TLC, HRCT, and QOL. This study will compare the ASSIST I conditioning regimen of cyclophosphamide and rATG to the same regimen (rATG, cyclophosphamide) with rituximab (termed rituximab sandwich regimen). Northwestern. Updated 04/01/12.

Early Diffuse Progressive Systemic Sclerosis

Transplanting Stem Cells Works for Scleroderma. Autologous blood stem cell transplants significantly improved survival in patients with high-risk scleroderma, despite significant treatment-related mortality, a randomized trial showed. MedPage Today, 06/07/12.

Stem Cell Transfusion is a form of bone marrow transplantation which uses a patient's own cells, thus avoiding rejection problems. This is now being used, under a National Institute of Health Program, for early diffuse progressive systemic sclerosis.

SCOT Clinical Trial: Enrollment Completed

Enrollment for the SCOT trial completed in 2011.

SCOT Clinical Trial, which stands for "Scleroderma Cyclophosphamide Or Transplantation", will compare two potential therapies: autologous stem cell transplantation versus high-dose monthly cyclophosphamide. Sclerodermatrial.org. (Also see Scleroderma Clinical Trials: Current and Cyclophos-phamide)

Research Studies

Mesenchymal stem cell therapy for refractory scleroderma: a report of 2 cases. Stem cell therapy is not ready for use on every patient with scleroderma but there are reasons to believe that it is possibly effective where there is indeed no hope to offer. PMC, Stem Cell Investig, 09/19/2016.

Does ex vivo CD34+ positive selection influence outcome after autologous hematopoietic stem cell transplantation (AHSCT) in systemic sclerosis patients? We demonstrate that CD34+ does not add benefit to the outcome of SSc patient treated with AHSCT and these findings should be further confirmed by prospective randomized trials. PubMed, Bone Marrow Transplant, 12/07/2015.

Human Stem Cell Transplantation (HSCT) Revealed As Promising Treatment For Systemic Sclerosis. The HSCT method appears to be a promising treatment of systemic sclerosis disease and the results of this study demonstrate substantial improvement over the course of 12 months treatment. Scleroderma News, 08/20/2015.

Transplant Funding

National Foundation for Transplants (NFT). NFT has raised from $5,000 to $600,000 in individual transplant campaigns from Maine to Guam.

The impact of dialysis on the survival of patients with immunoglobulin light chain (AL) amyloidosis undergoing autologous stem cell transplantation (ASCT). The study found significant differences in the OS depending on when the acute renal failure occurred and patients who required dialysis within 30 days of ASCT had the highest rate of treatment–related mortality. PubMed, Nephrol Dial Transplant, 2016 Aug;31(8):1284-9.

T cell repertoire (TCR) following autologous stem cell transplantation for multiple sclerosis. TCR characterization during immunomodulatory treatment is both feasible and informative, and may enable monitoring of pathogenic or protective T cell clones following HSCT and cellular therapies. PubMed, J Clin Invest, 2014 Feb 17. (Also see Multiple Sclerosis)

Use of Mesenchymal Stem Cells (MSC) in Chronic Inflammatory Fistulizing and Fibrotic Diseases: a Comprehensive Review. The current data indicate that MSC represent a promising alternative strategy and encouraging results in the treatment of Crohn's disease, Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis. PubMed, Clin Rev Allergy Immunol, 2013 Jan 8. (Also see Lupus Treatments)

Patient Stories

Sadly, Doris Razo passed away in December 2012.

Chasing the Cure. In this book, follow Doris' courageous journey in dealing with an autoimmune disease and sifting through the maze of doctors and treatments, including a stem cell transplant. Doris Razo.

Carmen Rijos

Carmen Rijos: Systemic Scleroderma Carmen is in need of a stem cell transplant for scleroderma. This is a request for donations for the Carmen Rijos Private Foundation…

Craig R.

Craig R: CREST Scleroderma I had my first scleroderma symptoms at age twenty-two, which included severe Raynaud's with some ulceration and tissue loss…

Elva

Elva: Mother of Diffuse Scleroderma Patient My son is unable to put on his own socks and shoes. He is very tired, and has tremendous pain…

Jennifer Weldon

Jennifer Weldon: Systemic Scleroderma/Stem Cell Transplant Patient My hope is that others who suffer with this disease find the courage to have the stem cell/bone marrow transplant. If the doctors can learn more about fighting and curing scleroderma, then my decision to have the transplant and go on living is worth the fight… ISN.

Krissy

Krissy: Systemic Scleroderma/Stem Cell Transplant Candidate Our home is like a hospital. We have oxygen tanks for when I need extra help breathing, and a lovely kangaroo pump which is how I get my continuous feeding in me…

Toni: Spouse of Stem Cell Transplant Survivor

Toni: Spouse of Stem Cell Transplant Survivor Jeff is now 17 months past his stem cell transplant and to look at him you'd never know he was sick…

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