Marten J. Poley - Erasmus Universiteit Rotterdam

Summary 149mortality. Finally, it is intended to consider not only the costs and effectsoccurring to the patients themselves but also those to the parents, whose positionmust not be ignored in cost-effectiveness studies of neonatal surgery. Caring fora child with a major congenital anomaly may have a negative influence on theparents' HRQoL. Also, caregiving may take the parents significant amounts oftime, and may be associated with high out-of-pocket expenses and costsassociated with production losses. Quantitative data to support these perceptionsare however largely lacking in neonatal surgery. Besides its aim to presentevidence on the cost-effectiveness of neonatal surgery, this thesis can serve asan introduction into cost-effectiveness analyses to pediatric surgeons. It will givethem the opportunity to become acquainted with the principles of costeffectivenessanalysis.Chapter 2 presents a full economic evaluation of neonatal surgery andsubsequent treatment for congenital anorectal malformations (ARM). Using thetechnique of cost-utility analysis, the costs and effects of treatment of patientswith ARM are compared to 'no treatment'. Total costs of treatment are calculatedat € 31,593, mainly consisting of direct and indirect medical costs. ARM patientsafter surgical correction suffer considerable stool difficulties and their medicalconsumption is relatively high. The EQ-5D questionnaire, however, shows that theHRQoL of ARM patients is only slightly lower than that of the general population(0.88 v 0.93). Treatment results in a gain of 12.7 quality-adjusted life years(QALYs). Cost-effectiveness (cost per QALY of € 2,482) is good.Chapter 3 contains a cost-utility analysis of treatment for congenitaldiaphragmatic hernia (CDH), closely resembling the methods used in Chapter 2.Total costs—for the most part initial hospitalization costs and indirect medicalcosts—average € 42,658. Productivity losses in both the patients and theircaregivers appear to be minor. Former CDH patients suffer from respiratorydifficulties and stomach aches, even at adult age. According to the EQ-5D,however, their HRQoL does not differ from the general population, suggestingthat these symptoms barely affect overall HRQoL. Treatment yields a gain of 17.5QALYs. Costs per QALY amount to € 2,434, which indicates good costeffectiveness.Chapter 4 covers an in-depth analysis of the short-term and long-term HRQoL ofsurvivors of ARM or CDH. The patients or their parents are administered symptomchecklists and generic HRQoL instruments to find out how the patients are doingcompared to the general population. Many patients appear to retain substantialresidual symptomatology into adulthood. In the youngest ARM patients (aged 1–4years), generic HRQoL is severely affected, but the older ARM patients showbetter HRQoL. In the CDH patients, the influence of symptoms on HRQoL seemsless profound. The instruments used reveal little difference between adultstreated for ARM or CDH and the general population. These results show that forthese two neonatal surgical procedures, improved survival does not come at the