Autologous Transplant in Neuroblastoma

Commonly referred to as autologous transplant, the use of the patient's own stem cells to support recovery from high-dose chemotherapy is more properly referred to as high-dose chemotherapy with stem cell rescue (HDC/SCR). The HDC regimen used to prepare the patient is usually myeloablative, meaning that no bone marrow recovery is possible without SCR. There are also submyeloablative HDC regimens, in which the SCR is used to speed recovery, decrease toxicity, and decrease treatment interval without being absolutely required for engraftment (Kletzel et al. 2002; Kreissman et al. 1997).

Compared with autologous marrow, PBSC provides faster hematopoietic recovery from HDC resulting in lower infection risk, shorter duration of mucositis and hospital stay, and lower transfusion requirement, especially of platelets. The use of PBSC, along with other advances in prophylaxis and supportive care, has decreased the treatment-related mortality (TRM) rate in autologous transplant to <5% in many studies. As a result, the use of autolo-gous marrow to support HDC/SCR has very limited indication.

There are several well-established indications for autologous HDC/SCR. In recurrent Hodgkin's disease, 70 % relapse again after successfully achieving a second complete remission (CR). Using HDC/SCR in second CR increases EFS to approximately 40-60% (Lazarus et al. 2001). Another established indication for HDC/SCR is high-risk NB. A number of single-

arm or retrospective studies suggested that autolo-gous bone marrow transplant might improve EFS (Matthay et al. 1998). A large EBMT retrospective analysis of 1070 HDC/SCR for NB noted overall 49% survival at 2 years. Some relapses were as late as 7 years from transplant, although most events occurred within the first 18 months. Forty-eight of the 1070 procedures were performed after relapse, with no survivors among those undergoing a second HDC/SCR procedure (Philip et al. 1997). In Table 11.6.2, EFS rates at or around 3 years from several major studies are summarized.

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