Hero 2010.
Methods | No information on method of randomisation provided. |
Participants | 295 children with high‐risk neuroblastoma (defined as stage 4 or MYCN amplified). No information was provided on age, sex, stage of disease, primary disease or recurrence, histology, presence of bone metastases, immunocytologic analysis of bone marrow at diagnosis, MYCN amplification, serum LDH level, treatment details, response before randomisation. |
Interventions | Myeloablative therapy (n=143) versus conventional therapy, i.e. 4 cycles of oral cyclophosphamide (n=119). For 33 patients no information was provided. |
Outcomes | Relapses occurred statistically significantly (P < 0.001) later in the transplant group (74 out of 143 (52%) patients; median 21 months (range 8 to 85 months)) than in the control group (83 out of 119 (70%) patients; median 16 months (range 7 to 60 months, median 16 months)). Patients died up to nine years after diagnosis: in the transplant group at a median of 30 months; in the control group at a median of 20 months (P < 0.001). Early complications (the abstract did not report if these were significant statistical differences): (a) pneumonia n = 10 in the transplant group versus n = 1 in the control group, (b) other severe infectious complications n = 8 in the transplant group, (c) veno‐occlusive disease n = 8 in the transplant group, (d) renal failure n = 3 in the transplant group, (e) treatment‐related death n = 5 in the transplant group versus n = 0 in the control group. Secondary malignant disease: 1 in each treatment group (both leukaemia cases; no further information provided). Major late effects in 109 patients surviving 5 years or longer, i.e. n = 68 in the transplant group versus n = 41 in the control group (it was unclear if this were all patients who survived 5 years or longer): (a) hearing loss 72% versus 51% (P = 0.04), (b) tubular damage 18% versus 12% (NS), (c) hypothyroidism 19% versus 2% (P = 0.02), (d) focal nodular hyperplasia of the liver 10% versus 0 % (P = 0.04), (e) impaired growth 10% versus 0% (P = 0.04). In conclusion: myeloablative therapy proved effective with respect to long‐term outcome, but is complicated by acute and long‐term toxicity. |
Notes | This study has not been published in full text (April 2015), but was presented at the ANR conference 2010. It provides additional follow‐up of the Berthold 2005 study. Length of follow‐up was up to 12 years (median observation time 8.4 years). |
LDH: lactate dehydrogenase; NS: not significant