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Bone Marrow Transplant ; 51(9): 1204-10, 2016 Sep.
Article in English | MEDLINE | ID: mdl-27159174

ABSTRACT

The optimal autologous stem cell rescue (HDC-SCR) regimen for children with high-risk neuroblastoma (HR-NBL) is not defined. Carboplatin/etoposide/melphalan (CEM) is the current US standard; however, European data suggest busulfan/melphalan (Bu/Mel) may have less toxicity. Published data regarding toxicities associated with CEM and Bu/Mel are limited. We conducted a single-institution retrospective cohort study of children with HR-NBL who received CEM or Bu/Mel preparative regimens. Toxicity data were analyzed using χ(2) or Fisher's exact, Wilcoxon two-sample or log-rank tests. Sinusoidal obstruction syndrome (SOS) was observed in 7/44 CEM (15.9%) and 5/21 (24%) Bu/Mel patients (P=0.50). Median time to SOS was longer following Bu/Mel than CEM (20 versus 9 days, P=0.02). Pulmonary hypertension (PHTN) was observed in ~20% of children after Bu/Mel and none after CEM (P=0.01). CEM patients had more nephrotoxicity (P=0.001), packed red blood cell (P=0.02) and platelet transfusions (P=0.008), and days on maximum pain support (P=0.0007). Time to engraftment, length of stay, documented infection rates and HDC-SCR-related mortality were similar. Nephrotoxicity and resource utilization associated with cytopenias and mucositis were greater after CEM. Pulmonary toxicities were more severe after Bu/Mel, and increased vigilance for PHTN may be warranted, particularly in children with hypoxemia out of proportion to respiratory distress.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols/toxicity , Myeloablative Agonists/toxicity , Neuroblastoma/complications , Neuroblastoma/drug therapy , Transplantation Conditioning/adverse effects , Adolescent , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Busulfan/administration & dosage , Carboplatin/administration & dosage , Child , Child, Preschool , Cohort Studies , Etoposide/administration & dosage , Female , Hepatic Veno-Occlusive Disease/chemically induced , Humans , Hypertension, Pulmonary/chemically induced , Infant , Kidney Diseases/chemically induced , Male , Melphalan/administration & dosage , Mucositis/chemically induced , Myeloablative Agonists/administration & dosage , Neuroblastoma/mortality , Neuroblastoma/therapy , Pancytopenia/chemically induced , Retrospective Studies , Stem Cell Transplantation/adverse effects , Stem Cell Transplantation/methods , Stem Cell Transplantation/mortality , Transplantation Conditioning/methods , Transplantation Conditioning/mortality , Young Adult
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