High-dose chemotherapy (HDC) followed by hematopoietic stem-cell (HSC) transplant (HSCT) or stem-cell support (SCS) (i.e., blood or marrow) transplant is an effective treatment modality for many patients with certain malignancies and non-malignancies. The rationale of this treatment approach is to provide a very dose-intensive treatment in order to eradicate malignant cells followed by rescue with peripheral blood, umbilical cord blood, or bone marrow stem-cells.
This policy was based on MedLine literature search through September 2008. In 1990, Lenarsky and Parkman reported the initial attempts to treat patients with AIDS due to the lack of effective concomitant anti-viral therapy. They concluded that HSCT would continue to be in the forefront of human bone marrow transplantation. (1) Fasth reported the outcome of HSCT was strongly dependent on the patient’s age, clinical status at transplantation and the type of immunodeficiency. This review article generally described primary immunodeficiencies, not HIV or AIDS specifically. (2)
Allogeneic, autologous, tandem or triple stem-cell transplant and donor leukocyte infusion (DLI) for acquired immunodeficiency syndrome (AIDS) is a secondary immunodeficiency syndrome resulting from a human immunodeficiency virus (HIV) infection AIDS or HIV infection is considered experimental, investigational and unproven due to lack of adequate evidence of safety and effectiveness documented in published, peer-reviewed medical literature.
A search of peer reviewed literature through October 2012 was conducted. Few human clinical trials have been published. Studies with animal models of autoimmune diseases have provided the stimulus for further research in treating a variety of immune diseases, using autologous stem-cell support (AuSCS). (3)
Donor leukocyte infusion (DLI) to treat any stage of AIDS and HIV is considered experimental, investigational, and unproven due to lack of adequate evidence of safety and effectiveness documented in published, peer-reviewed medical literature. (4)
As with DLI, HPC Boost has a positive response rate for relapse following AlloSCS. (5) The boost of stem-cells, a second dose, may be helpful to reduce the graft failure process, avoiding the risk of serious bleeding and/or infection. However, the data is insufficient for the use of HPC Boost following AlloSCS for treatment of non-hematological malignancies to lessen post-transplant graft failures. (5, 6, 7, 8)
Short Tandem Repeat (STR) Markers
Following SCS therapy, it is important to determine whether the new blood forming system is of the donor or the recipient, based upon the proportion of donor and recipient cells. The characteristics of the engraftment are analyzed, which is called chimerism analysis. Using STR marker assay to characterize the hematological course and to evaluate the usefulness of the blood forming system (particularly for hematological malignancies, myelodysplastic/myeloproliferative processes, or certain genetic or metabolic disorders) has been tested initially after the SCS, when the patient is declared as disease-free, and at the point of the confirmed stable engraftment of only the donor pattern of the blood forming system. (9, 10) Without further randomized trials using STR markers prior to or post SCS therapy for treatment of AIDS and HIV, the data is insufficient to determine the outcome/effect of stem-cell engraftment. (9, 10, 11, 12, 13, 14)
A search in November 2012 found 24 studies. Of those only 11 studies are actively recruiting patients for the treatment research of bone marrow and stem-cell transplantation for AIDS or HIV infections, when the patient has another unrelated disease condition, such as cancer or liver disease. There were no active studies identified for the sole treatment of AIDS or HIV by HSCT.
To date, there are no new clinical trial publications or any additional information that would change the coverage position of this medical policy. Thus the use of SCS, as a single treatment or infusion, tandem or triple stem-cell transplant and DLI in AIDS or HIV remains experimental, investigational and unproven.
Based on a search of scientific literature in the MedLine database through March 2013, HPC boost to reduce the graft failure process and STR markers to monitor engraftment chimerism for the treatment of AIDS and HIV are considered experimental, investigational, and unproven due to the lack of adequate evidence of safety and effectiveness documented in published, peer-reviewed medical literature.
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