An End To Autoimmune Disease Progression

Hematopoietic Stem Cell Transplantation

Definition:  Hematopoietic stem cell transplantation (HSCT) involves the intravenous infusion of autologous or allogenic stem cells collected from bone marrow, peripheral blood, or umbilical cord blood to re-establish hematopoietic function in patients with damaged or defective bone marrow or immune systems. HSCT, as used here, is a general term covering transplantation of progenitor/stem cells from any source (eg, bone marrow, peripheral blood, cord blood), in conjunction with high-dose chemotherapy to reset the immune system.

Autologous transplantation:  Generally, candidates for autologous transplantation have no demonstrable malignancy in the blood or bone marrow. Whereas treatment-related morbidity and mortality rates are lowest with autografts. This finding relates to the absence of a graft versus host effect.

Peripheral blood (PBPC):  Hematopoietic stem cells circulate in blood and can be identified and quantified using flow cytometry (cells express the CD34 antigen). Administration of recombinant hematopoietic growth factors (ie, granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor) to patients or donors releases marrow stem cells into the peripheral blood, which can be collected from the veins of the patient or donor.

Apheresis instruments, similar to those used for collecting platelet concentrates from volunteer donors for transplantation, are used in an ambulatory setting. Using this technique, during a 3- to 4-hour period, approximately 1 log more hematopoietic progenitors can be collected than in a bone marrow harvest.

Furthermore, the need for general anesthesia and a sterile operating room is eliminated. This process, known as mobilization of stem cells, may be enhanced in patients destined for autografts in whom exposure to cytotoxic drugs (eg, cyclophosphamide) plus hematopoietic growth factor therapy causes the release of 10 times as many (or more) primitive hematopoietic progenitors into the circulation than the recombinant cytokines alone.

The use of peripheral blood rather than bone marrow as a source of hematopoietic stem cells results in the collection of more CD34+ progenitor cells and faster marrow recovery (ie, 2 wk vs 4 wk for neutrophil, platelet, and RBC counts). In the autologous setting, most transplantation groups use PBPCs in the majority of settings.In the allogenic setting, the situation is considerably more complex.

The optimal product has yet to be determined with respect to the use of bone marrow versus PBPCs. As previously mentioned, PBPCs engraft more rapidly than bone marrow, but the increased T-cell load may result in higher rates of Graft Versus Host Disease (GVHD), particularly chronic GVHD.

The following Autoimmune Disorders are also treated with HSCT:

Aplastic anemia

Autoimmune Related Retinopathy and Optic Neuropathy (ARRONS

Bullous Skin Disorder

Chronic Inflammatory Demylinating Polyneuropathy (CIDP)

Crohn's Disease

Devic's Disease

Idiopathic Inflammatory Myopathy Diseases

Leukocyte Adhesion Deficiency Type 1

Morphea

Multiple Sclerosis

Myasthenia Gravis

Primary Biliary Cirrhosis

Pulmonary Fibrosis

Rheumatoid Arthritis

Sarcoidosis

Severe combined immunodeficiency disorders

Scleroderma

Stiff Persons Disease

Systemic Lupus Erthematosus

Systemic Necrotizing Vascultits

Type 1 Diabetes