Stem Cell Transplantation

Greco R, Bondanza A, Oliveira MC, Badoglio M, Burman J, Piehl F, Hagglund H, Krasulova E, Simões BP, Carlson K, Pohlreich D, Labopin M, Saccardi R, Comi G, Mancardi GL, Bacigalupo A, Ciceri F, Farge D.Autologous hematopoietic stem cell transplantation in neuromyelitis optica: A registry study of the EBMT Autoimmune Diseases Working Party. Mult Scler. 2014  pii: 1352458514541978. [Epub ahead of print]

BACKGROUND:Neuromyelitis optica (NMO) is an inflammatory autoimmune disorder of the central nervous system, hallmarked by pathogenic anti-aquaporin 4 antibodies. NMO prognosis is worse compared with multiple sclerosis.
OBJECTIVE:The European Group for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) conducted a retrospective survey to analyze disease outcome following autologous stem cell transplantation (ASCT).
METHODS:This retrospective multicenter study assessed the efficacy and safety of ASCT in 16 patients with refractory NMO reported to the EBMT registry between 2001 and 2011.
RESULTS:Fifteen patients were successfully mobilized with cyclophosphamide (Cy) and G-CSF, one with G-CSF alone. All patients received an unmanipulated autologous peripheral blood stem cell graft, after conditioning with BEAM plus anti-thymocyte globulin (ATG, n = 9 patients), thiotepa-Cy (n = 3) or Cy (200 mg/kg) plus ATG (n = 4). After a median follow-up of 47 months, three of 16 cases were progression and treatment free, while in the remaining 13 patients further treatments were administered for disability progression or relapse after ASCT. Altogether, relapse-free survival at three and five years was 31% and 10%, respectively, while progression-free survival remained 48% at three and five years.
CONCLUSIONS:In these NMO patients, highly resistant to conventional treatment, ASCT allows for temporary control of the disease, despite a tendency to progress or relapse in the long term.

In the past, a bone marrow harvest (a surgical procedure) was the only way to collect stem cells for a transplant. Today it is now possible to collect  stem cells from the peripheral blood (from the bloodstream).

Stem cell mobilization is a process whereby stem cells are stimulated out of the bone marrow space (e.g., the hip bones and the chest bone) into the bloodstream, so they are available for collection for future reinfusion. The cells are then preserved, frozen and stored until the time of transplant.

Some preferred characteristics of a mobilization regimen include: Mobilizing enough stem cells for transplant. This allows for recovery of red blood cells, white blood cells, and platelets when the stem cells are re-infused into your body. In this study they used cyclosposphamide an anti-cancer drug and granulocyte-Macrophage colony stimulating factor a cytokine that promotes cell growth and mobilises stem cells.

Then before the stem cell replacement the people had there immune system removed using different ways such as BEAM a mixture of chemotherapy agents that kills cells (carmustine BICNU/etposide/cytarabine (Ara C/Melphalan) and anti-thymocyte globulin which are actibodies aimed at killing thymocytes,-immature T cells or cyclophosphamide which kills dividing cells

In this study in NMO the long term effects were studied in sixteen people and only three remained progression and relapse free. One suspects that the high failure rate (>80%) may relate to likihood that these people were not treated early. However, it shows that this very radical treatment, may not always be the solution and many of the subjects need retreatment. 

This treatment as profG will testify also comes with significant side effect risks. 

Studies in MS are ongoing

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