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Stem Cell Transplantation with High-Dose Chemotherapy

High-dose chemotherapy and stem cell transplantation are important parts of treatment plans for eligible, recently diagnosed myeloma patients. One of the following types of transplants may be used.

Autologous Stem Cell Transplantation

The patient’s own stem cells are collected for this type of stem cell transplant. If needed, a doctor may use plerixafor (Mozobil®)—which is FDA approved for use in combination with a granulocyte colony-stimulating factor (G-CSF)—to help mobilize stem cells and move them into the peripheral blood for collection. “Mobilizing” stem cells means stimulating them to move from the marrow into the bloodstream, so that enough cells can be collected for an autologous transplant. The patient is then treated with high doses of chemotherapy, often using melphalan. The cells are then returned to the patient via an infusion into the bloodstream (like a blood transfusion) to rescue the bone marrow from the effects of the high doses of chemotherapy. This procedure is associated with good response rates and remains the standard of care, after completion of primary therapy, for eligible patients. However, it is not appropriate for all patients, and it is not a cure for myeloma. Patients should discuss the benefits and risks of any procedure with their doctors. Response to the transplant is measured by the standard tests used to monitor myeloma treatment, such as blood and urine protein levels, marrow tests and imaging studies. Myeloma patients who have had an autologous transplant may require maintenance therapy (see below).

Tandem Autologous Stem Cell Transplantation. This term refers to a planned second course of high-dose chemotherapy and stem cell transplant within 6 months of the first course. According to recent studies, this should only be considered as a treatment option in patients who fail to achieve a good response with the first transplant, or in select patients with high-risk cytogenetic features, such as del(17p).

Allogeneic Stem Cell Transplantation and Reduced-Intensity Allogeneic Stem Cell Transplantation

Allogeneic and reduced-intensity allogeneic transplantation are other types of stem cell transplants used to treat certain blood cancers. The main difference between autologous and allogeneic transplant is that in allogeneic transplant, the cells are obtained from a donor whose cells are compatible with those of the patient, usually a brother or sister. Side effects and mortality are more likely to occur with an allogeneic transplant than with an autologous transplant. Therefore, allogeneic transplantation has a limited role in myeloma treatment and it should only be done in the context of a clinical trial.

Maintenance Therapy

The use of continued therapy to maintain a response obtained with induction therapy or stem cell transplantation is showing a benefit in overall survival rate. Lenalidomide (Revlimid®) is the preferred agent for post-transplant maintenance, based on the results of several clinical trials. Lenalidomide is FDA approved for patients with myeloma as maintenance therapy following an autologous stem cell transplant. It does not produce the neurotoxicity of other immunomodulatory drugs. However, there appears to be an increased risk for the development of a secondary cancer, especially after transplantation or after therapy with a regimen that contains melphalan. More information is needed about the effects of maintenance therapy on overall survival, as well as second cancer risk. Maintenance therapy with bortezomib (Velcade®) or ixazomib (Ninlaro®) is recommended for patients with certain cytogenetic abnormalities.


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