Tandem high-dose chemotherapy and autologous stem cell transplantation in patients with high-risk neuroblastoma: Results of SMC NB-2004 study

• Published in: Bone marrow transplantation (Basingstoke) Vol. 48; no. 1; pp. 68 - 73
• Main Authors: SUNG, K. W, SON, M. H, KIM, J.-S, KIM, D. W, LEE, S. H, YOO, K. H, KOO, H. H, KIM, J. Y, CHO, E. J, LEE, S. K, CHOI, Y. S, LIM, D. H
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01.01.2013
• Subjects: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Antineoplastic Agents - administration & dosage; Antineoplastic Agents - adverse effects; Antineoplastic Agents - therapeutic use; Antineoplastic Combined Chemotherapy Protocols - administration & dosage; Antineoplastic Combined Chemotherapy Protocols - adverse effects; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Biological and medical sciences; Bone marrow; Bone marrow, stem cells transplantation. Graft versus host reaction; Bone Neoplasms - diagnosis; Bone Neoplasms - pathology; Bone Neoplasms - secondary; Bone Neoplasms - therapy; Cancer; Care and treatment; Chemotherapy; Child; Child, Preschool; Combined Modality Therapy - adverse effects; Development and progression; DNA Copy Number Variations; Feasibility Studies; Health aspects; Hematopoietic stem cells; Humans; Induction Chemotherapy - adverse effects; Infant; Interleukin-2 - administration & dosage; Interleukin-2 - adverse effects; Interleukin-2 - therapeutic use; Isotretinoin - administration & dosage; Isotretinoin - adverse effects; Isotretinoin - therapeutic use; Medical sciences; N-Myc Proto-Oncogene Protein; Neoplasm Proteins - genetics; Neoplasm Proteins - metabolism; Neoplasm Staging; Neuroblastoma; Neuroblastoma - diagnosis; Neuroblastoma - pathology; Neuroblastoma - secondary; Neuroblastoma - therapy; Neurology; Nuclear Proteins - genetics; Nuclear Proteins - metabolism; Oncogene Proteins - genetics; Oncogene Proteins - metabolism; Prognosis; Prospective Studies; Radiotherapy - adverse effects; Radiotherapy - methods; Stem cell transplantation; Stem Cell Transplantation - adverse effects; Survival Analysis; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation; Transplantation, Autologous; Tumors of the nervous system. Phacomatoses; Whole-Body Irradiation - adverse effects; South Korea; Human; High risk; Nervous system diseases; Hematology; Stem cell; Hematopoietic cell; auto-SCT; Malignant tumor; Neuroblastoma; Chemotherapy; Treatment; Autologous hematopoietic stem cell transplantation; Autonomic neuropathy; High dose; Cancer; high-dose chemotherapy
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/bmt.2012.86

From January 2004 to December 2008, 50 consecutive patients with high-risk neuroblastoma were assigned to receive tandem HDCT (high-dose chemotherapy)/auto-SCT after nine cycles of induction chemotherapy. CEC (carboplatin + etoposide + cyclophosphamide) regimen and TM (thiotepa + melphalan)-TBI regimen (or TM regimen for stage 3 patients) were the first and second HDCT regimens. Local radiotherapy, differentiation therapy with 13-cis-retinoid acid and immunotherapy with interleukin-2 were given after tandem HDCT/auto-SCT. Of the 50 patients, 49 underwent a first HDCT/auto-SCT and 47 underwent a second HDCT/auto-SCT. The tumor relapsed or progressed in 14 patients, secondary malignancy developed in one patient and one patient died from chronic lung disease. Therefore, 34 patients remained event free with a median follow-up of 54.5 months (range, 14-94 months) from diagnosis. The probabilities of 5-year OS and EFS for all 50 patients were 77.0% (95% confidence interval (CI), 63.7-90.3) and 71.4% (95% CI, 58.7-84.1), respectively. However, all patients who remained event free for >3 years after tandem HDCT/auto-SCT experienced late adverse effects. Chemotherapeutic dose-escalation strategy using tandem HDCT/auto-SCT was very encouraging for survival. However, further studies incorporating newer treatment modalities are needed to reduce late adverse effects without jeopardizing the survival rate.