Effect of neo-adjuvant chemotherapy on long-term health state and behaviour in the PNET3 RCT of treatment for primitive neuroectodermal tumour (PNET)

Kennedy, Colin and Bull, Kim (2004) Effect of neo-adjuvant chemotherapy on long-term health state and behaviour in the PNET3 RCT of treatment for primitive neuroectodermal tumour (PNET). Neuro-Oncology, 6, 460-460.

Abstract

The addition of chemotherapy to craniospinal irradiation (CSI) for treatment of PNET is currently considered ‘the standard of care’ to improve both rate and quality of survival (QoS), but its effect on QoS has not been systematically assessed. We assessed QoS in UK children previously enrolled in the SIOP PNET3 RCT of CSI (35 Gy plus 20 Gy boost to posterior fossa) vs. CSI plus neo-adjuvant chemotherapy (Vincristine, Carboplatin, Etoposide, and Cyclophosphamide) using a cross-sectional multi-informant questionnaire design. Total scores on the two core measures, the Health Utilities Index mark 3 (HUI3) and the Strengths and Difficulties Questionnaire (SDQ), were compared between the two treatment arms using the Mann-Whitney U test. Information was obtained from patients, parents, and health professionals on 103/169 (61%) of survivors at a mean (SD) of 15.4 (4.0) years of age and 7.0 (2.25) years from diagnosis. Median {interquartile range} HUI scores and mean (SD) SDQ total difficulties scores were: HUI3 SDQ Respondent Parent Self* Parent Self** n 44 vs. 55 38 vs. 44 33 vs. 43 27 vs. 35 CSI 0.71 {0.52} 0.92 {0.37} 11.4 (5.21) 10.6 (4.59) CSI + Chemo 0.72 {0.48} 0.65 {0.45} 13.7 (5.90) 11.4 (5.90) *: aged over 11 years; **: aged 11–17 years Self-complete HUI3 scores were lower (Z = -2.263, p = 0.024, two-tailed) and parent-complete SDQ scores tended to be higher (Z = -1.559, p = 0.119, two-tailed) in the ‘CSI plus chemotherapy’ group. Parent-/child-complete SDQ scores were abnormally high in 14.0/2.9% and 33.3/7.4% of children in the ‘CSI’ and the ‘CSI plus chemotherapy’ arms, respectively. Patients in the two treatment arms will also be compared with respect to subscale scores on these measures, responses to quality of life questionnaires, and information from health professionals. These findings suggest that differences between self- and proxy-assessment are important and that the addition of chemotherapy to ‘standard dose’ CSI may adversely affect health status and behaviour. The effect on these measures of reducing the dose of CSI and simultaneously adding chemotherapy requires further investigation.

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Contributors

Author: Kim Bull

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