28 years of high-dose therapy and SCT for neuroblastoma in Europe: lessons from more than 4000 procedures


Creative Commons License

Ladenstein R., Poetschger U., Hartman O., Pearson A. D. J., Klingebiel T., Castel V., ...Daha Fazla

BONE MARROW TRANSPLANTATION, cilt.41, 2008 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 41
  • Basım Tarihi: 2008
  • Doi Numarası: 10.1038/bmt.2008.69
  • Dergi Adı: BONE MARROW TRANSPLANTATION
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Anahtar Kelimeler: neuroblastoma, high-dose treatment, stem cells, EBMT, BONE-MARROW-TRANSPLANTATION, HIGH-RISK NEUROBLASTOMA, STEM-CELL TRANSPLANTATION, CHIMERIC ANTI-GD2-ANTIBODY CH14.18, COLONY-STIMULATING FACTOR, STAGE-IV NEUROBLASTOMA, TOTAL-BODY IRRADIATION, PERIPHERAL-BLOOD, I-131 METAIODOBENZYLGUANIDINE, METASTATIC NEUROBLASTOMA
  • Ankara Üniversitesi Adresli: Evet

Özet

Between 1978 and 2006, the European Group for Blood and Marrow Transplantation registered 4098 high-dose therapy (HDT) procedures followed by stem cell rescue (SCR) (3974 autologous/124 allogeneic) in patients with neuroblastoma. The 5-year rates for overall (OS) and event-free survival are 37 and 32%, respectively. The median age at diagnosis is 3.9 years (0.3-62 years) with 76 patients older than 18 years. Patients above 10 years carry a 2.5-fold higher risk. Younger patients cure significantly (< 0.001) better with OS rates of 40 and 30% for age groups 2-4 years and 4-10 years, respectively. Their risks are about twofold higher than that of patients below 2 years with OS rates of 60%. The better the quality of remission status before HDT/SCT the better are the observed OS rates: 43% in CR1 (1199 patients) and 42% for CR2 (140 patients), and 36% for those in very good partial or partial remission (1413 patients) and 21% for those with sensitive relapse (134 patients). Patients reported with stable disease in first remission still had an OS rate of 30%. Multivariate analysis shows significantly better OS in the age group of less than 2 years (< 0.0001), as well as a better quality of remission status before HDT/SCT (P < 0.0001), with the use of peripheral stem cells (P = 0.014), autologous SCT (P = 0.031) and busulphan/melphalan HDT (P < 0.001). Busulphan/melphalan HDT/SCT in first remission achieves an OS of 48%, while it is only 35% with other regimens (P < 0.001), including melphalan alone, other melphalan-containing regimens, a variety of other drugs given as a single HDT as well as the addition of TBI or sequential HDT/SCT procedures. Further progress in the field may only be expected from large-scale international randomized trials.