This project was funded by the UK Medical Research Council (MRC) as part of the MRC-NIHR methodology research programme (reference G0901486) and will be published in full in Health Technology Assessment; Vol. 18, No. 9. See the NIHR Journals Library website for further project information.

Background: There has been a rapid growth in recent years of available technologies for

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ObjectiveThis review examines generic preference-based measures and their ability to reflect health-related quality of life in patients with visual disorders.MethodsA systematic search was undertaken to identify clinical studies of patients with visual disorders where health state utility values were measured and reported. Data were extracted to assess the validity and responsiveness of the measures. A narrative synthesis of the data was undertaken due to the heterogeneity between different studies.ResultsThere was considerable heterogeneity in the 31 studies identified in terms of patient characteristics, visual disorders, and outcomes reported. Vision loss was associated with a reduction in scores across the preference-based measure, but the evidence on validity and responsiveness was mixed. The EQ-5D health-related assessment instrument's performance differed according to condition, with poor performance in age-related macular degeneration (AMD) and diabetic retinopathy. The more limited evidence on the HUI-3 instrument found it performed best in differentiating between severity groups of patients with glaucoma, AMD, cataracts, and diabetic retinopathy. One study reported data on the SF-6D instrument and showed it was able to differentiate between patients with AMD.ConclusionsThe performance of the EQ-5D in visual disorders was mixed. The HUI-3 seemed to perform better in some conditions, but the evidence on this and SF-6D is limited. More head to head comparisons of these three measures are required. The new five-level version of EQ-5D may do better at the milder end of visual function.

This journal is a member of and subscribes to the principles of the Committee on Publication Ethics (COPE) (http://www.publicationethics.org/).Editorial contact: nihredit@southampton.ac.ukThe full HTA archive is freely available to view online at http://www.hta.ac.uk/project/htapubs.asp. Print copies can be purchased from the individual report pages. criteria for inclusion in the Health Technology Assessment journalReports are published in Health Technology Assessment (HTA) if (1) they have resulted from work for the HTA programme, and (2) they are of a sufficiently high scientific quality as assessed by the reviewers and editors.Reviews in Health Technology Assessment are termed 'systematic' when the account of the search appraisal and synthesis methods (to minimise biases and random errors) would, in theory, permit the replication of the review by others. Hta programmeThe HTA programme, part of the National Institute for Health Research (NIHR), was set up in 1993. It produces high-quality research information on the effectiveness, costs and broader impact of health technologies for those who use, manage and provide care in the NHS. 'Health technologies' are broadly defined as all interventions used to promote health, prevent and treat disease, and improve rehabilitation and long-term care.The research findings from the HTA programme directly influence decision-making bodies such as the National Institute for Health and Clinical Excellence (NICE) and the National Screening Committee (NSC). HTA programme findings also help to improve the quality of clinical practice in the NHS indirectly in that they form a key component of the 'National Knowledge Service'.For more information about the HTA programme please visit the website: http://www.hta.ac.uk/ this reportThe research reported in this issue of the journal was funded by the HTA programme as project number 09/22/21. The contractual start date was in July 2010. The draft report began editorial review in January 2012 and was accepted for publication in June 2012. The authors have been wholly responsible for all data collection, analysis and interpretation, and for writing up their work. The HTA editors and publisher have tried to ensure the accuracy of the authors' report and would like to thank the reviewers for their constructive comments on the draft document. However, they do not accept liability for damages or losses arising from material published in this report.This report presents independent research funded by the National Institute for Health Research (NIHR). The views and opinions expressed by authors in this publication are those of the authors and do not necessarily reflect those of the NHS, the NIHR, NETSCC, the HTA programme or the Department of Health.

Background/Aims: In the last 20 years the range of high-technology augmentative and alternative communication (AAC) aids has rapidly expanded. This review aimed to provide a ‘state of the art’ synthesis, to provide evidence-based information for researchers, potential users and service providers. Methods: Electronic databases were searched from 2000 to 2010, together with reference lists of included papers and review papers. The review considered work of any design which reported an intervention using high-tech AAC with people who have communication difficulties (excluding those with solely hearing or visual loss) published in peer-reviewed journals. Results: Sixty-five papers reporting interventions using high-tech AAC were identified. There was evidence that high-technology AAC may be beneficial across a range of diagnoses and ages. The evidence, however, is currently drawn from studies using designs considered to be at high risk of bias. Conclusion: The review suggests that the high level of individual variation in outcome requires a greater understanding of characteristics of clients who may or may not benefit from this technology. Also, the wide range of outcomes measured requires further work in the field to establish what a ‘good outcome’ from intervention may be.

Cord blood transplantation (CBT) is curative for many primary immunodeficiencies (PIDs) but is associated with risks of viral infection and graft-versus-host disease (GvHD). Serotherapy reduces GvHD but potentially increases the risk of viral infection by delaying immune reconstitution. Because many PID patients have pre-existing viral infections, the optimal dose of serotherapy is unclear. We performed a retrospective analysis in 34 consecutive PID patients undergoing CBT and compared immune reconstitution, viral infection, GvHD, mortality, and long-term immune function between high-dose (n = 11) and low-dose (n = 9) serotherapy. Serotherapy dose had no effect on neutrophil engraftment. Median CD3(+) engraftment occurred at 92.5 and 97 days for high- and low-dose serotherapy, respectively. The low-dose serotherapy group had higher CD3(+), CD4(+), and early thymic emigrant counts at 4 months compared with the high-dose group. GvHD severity and number of viral infections did not differ between serotherapy doses. Survival from the transplantation process was 90.9% for high-dose and 100% for low-dose groups. In conclusion, low-dose serotherapy enhanced T cell reconstitution and thymopoiesis during the first year after CBT with no increase in GvHD.

The paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of trabectedin for the treatment of relapsed platinumsensitive ovarian cancer, based upon a review of the manufacturer's submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal process. The submission addressed only part of the decision problem and did not provide evidence to compare trabectedin (Yondelis  , PharmaMar) and pegylated liposomal doxorubicin hydrochloride (PLDH) (Caelyx  , Schering-Plough) with key comparators. The submission's 70Trabectedin for the treatment of relapsed ovarian cancer direct comparison evidence came from one reasonable-quality randomised controlled trial (RCT) of trabectedin and PLDH versus PLDH alone (ET743-OVA-301). The results of the RCT were subdivided into the entire platinum-sensitive population (> 6-month relapse after initial platinum-based chemotherapy) and partially platinum-sensitive (≥ 6-to 12-month relapse) and fully platinum-sensitive (> 12-month relapse) populations. The outcomes included were overall survival, progression-free survival measured by three types of assessor, response rates, adverse effects of treatment, health-related quality of life and cost per quality-adjusted-life-year (QALY) gained. A mixed treatment comparison (MTC) meta-analysis comparing trabectedin and PLDH with single-agent PLDH within the entire platinum-sensitive population, with paclitaxel or with topotecan also formed part of the submission. The RCT data showed that trabectedin plus PLDH compared with PLDH monotherapy had a significant effect on overall survival only within the partially platinum-sensitive subgroup. PFS results reported by the independent radiologists showed significant effects in favour of the trabectedin and PLDH arm for the entire and partially platinum-sensitive populations only. Rates of grade 3 and 4 adverse events were mostly higher in the trabectedin and PLDH arm than in the PLDH alone arm. There were several issues regarding the undertaking of the MTC, and thus the data were not considered robust. Furthermore, the ERG did not believe the MTC to be necessary to answer the decision problem. The manufacturer submitted a de novo cost-effectiveness model. The main analysis compared trabectedin in combination with PLDH versus paclitaxel, topotecan and PLDH (each as monotherapy) in the entire platinum-sensitive population, using results estimated from the MTC. Additional analyses were presented comparing trabectedin in combination with PLDH versus PLDH monotherapy using direct evidence from the OVA-301 trial for the fully, partially and entire platinum-sensitive populations. The cost per QALY gained for trabectedin in combination with PLDH versus PLDH monotherapy was estimated to be £70,076 in the main analysis. In the additional analyses, the cost per QALY gained for trabectedin in combination with PLDH versus PLDH monotherapy was £94,832, £43,996 and £31,092 for the ent...